10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

_________________________________

FORM 10-Q

_________________________________

☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended June 30, 2023

or

☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from ___________ to ___________

Commission File Number: 001-36257

 TRAVERE THERAPEUTICS, INC.

(Exact name of registrant as specified in its charter)

Delaware	27-4842691
(State or other jurisdiction of incorporation or organization)	(I.R.S. Employer Identification No.)

3611 Valley Centre Drive, Suite 300

San Diego, CA 92130

(Address of Principal Executive Offices)

(888) 969-7879

(Registrant's Telephone number including area code)

N/A

Former name, former address and former fiscal year, if changed since last report

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.0001 per share	TVTX	The Nasdaq Global Market

Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.   Yes ☑ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).  Yes ☑ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and "emerging growth company" in Rule 12b-2 of the Exchange Act.

Large accelerated filer	☑	Accelerated filer	☐
Non-accelerated filer	☐	Smaller reporting company	☐
		Emerging growth company	☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

 Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act).    Yes  ☐ No ☑

 The number of shares of outstanding common stock, par value $0.0001 per share, of the Registrant as of August 1, 2023 was 75,010,865.

TRAVERE THERAPEUTICS, INC.

Form 10-Q

For the Fiscal Quarter Ended June 30, 2023

TABLE OF CONTENTS

		Page No.
PART I – FINANCIAL INFORMATION
Item 1.	Financial Statements
	Consolidated Balance Sheets as of June 30, 2023 (Unaudited) and December 31, 2022	4
	Unaudited Consolidated Statements of Operations and Comprehensive Loss for the three and six months ended June 30, 2023 and 2022	5
	Unaudited Consolidated Statements of Stockholders' Equity for the three and six months ended June 30, 2023 and 2022	6
	Unaudited Consolidated Statements of Cash Flows for the six months ended June 30, 2023 and 2022	8
	Notes to Unaudited Consolidated Financial Statements	9
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	25
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	36
Item 4.	Controls and Procedures	36
PART II – OTHER INFORMATION
Item 1.	Legal Proceedings	37
Item 1A.	Risk Factors	37
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	64
Item 3.	Defaults Upon Senior Securities	64
Item 4.	Mine Safety Disclosures	64
Item 5.	Other Information	64
Item 6.	Exhibits	65

1

FORWARD-LOOKING STATEMENTS 

This report contains forward-looking statements regarding our business, financial condition, results of operations and prospects. Words such as “expects,” “anticipates,” “intends,” “plans,” “believes,” “seeks,” “estimates” and similar expressions or variations of such words are intended to identify forward-looking statements, but are not deemed to represent an all-inclusive means of identifying forward-looking statements as denoted in this report. Additionally, statements concerning future matters are forward-looking statements.

Although forward-looking statements in this report reflect the good faith judgment of our management, such statements can only be based on facts and factors currently known by us. Consequently, forward-looking statements are inherently subject to risks and uncertainties and actual results and outcomes may differ materially from the results and outcomes discussed in or anticipated by the forward-looking statements. Factors that could cause or contribute to such differences in results and outcomes include, without limitation, those specifically addressed under the headings “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2022, and in this Quarterly Report on Form 10-Q. You are urged not to place undue reliance on these forward-looking statements, which speak only as of the date of this report. 

In addition, statements that "we believe" and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this Quarterly Report on Form 10-Q, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned to not unduly rely upon these statements.

We file reports with the Securities and Exchange Commission ("SEC"). The SEC maintains a website (www.sec.gov) that contains reports, proxy and information statements, and other information regarding issuers that file electronically with the SEC, including us.

We undertake no obligation to revise or update any forward-looking statements in order to reflect any event or circumstance that may arise after the date of this report, except as required by law. Readers are urged to carefully review and consider the various disclosures made throughout the entirety of this quarterly report, which are designed to advise interested parties of the risks and factors that may affect our business, financial condition, results of operations and prospects.

Risk Factor Summary

Below is a summary of material factors that make an investment in our common stock speculative or risky. This summary does not address all of the risks that we face. Additional discussion of the risks summarized in this risk factor summary, and other risks that we face, can be found under the heading “Risk Factors” in Item 1A of Part II of this Quarterly Report on Form 10-Q and should be carefully considered, together with other information in this Quarterly Report on Form 10-Q and our other filings with the SEC before making investment decisions regarding our common stock.

•Our future prospects are highly dependent upon our ability to successfully develop and execute commercialization strategies for our products, including FILSPARI (sparsentan) to reduce proteinuria in adults with primary Immunoglobulin A nephropathy (IgAN), and to attain market acceptance among physicians, patients and healthcare payers.

•In order to operate our business and increase adoption and sales of our products, we need to continue to develop our commercial organization, including maintaining and growing a highly experienced and skilled workforce with qualified sales representatives.

•Our clinical trials are expensive and time-consuming and may fail to demonstrate the safety and efficacy of our product candidates. Success in preclinical testing and early clinical trials does not ensure that later clinical trials will be successful.

•Communications and/or feedback from regulatory authorities related to our clinical trials does not guarantee any particular outcome from or timeline for regulatory review, and expedited regulatory review pathways may not actually lead to faster development or approval.

•Interim, topline and preliminary data from our clinical trials that we announce or publish may change materially as more patient data become available and audit and verification procedures are completed.

•We face substantial generic and other competition, and our operating results will suffer if we fail to compete effectively.

•Healthcare reform initiatives, unfavorable pricing regulations, and changes in reimbursement practices of third-party payers or patients' access to insurance coverage could affect the pricing of and demand for our products.

•We are dependent on third parties to manufacture and distribute our products.

•The market opportunities for our products and product candidates may be smaller than we believe they are.

•Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval or commercialization.

•We do not currently have patent protection for certain of our commercial products. If we are unable to obtain and maintain intellectual property relating to our technology and products, their value may be adversely affected.

•We expect to rely on orphan drug status to develop and commercialize certain of our product candidates, but our orphan drug designations may not confer marketing exclusivity or other expected commercial benefits.

•We will likely experience fluctuations in operating results and could incur substantial losses, and the market price for shares of our common stock may be volatile.

2

•Negative publicity regarding any of our products could impair our ability to market any such product and may require us to spend time and money to address these issues.

•We may need substantial funding and may be unable to raise capital when needed. Our indebtedness could adversely affect our financial condition.

•We might not successfully complete the sale of our bile acid product portfolio for the treatment of rare liver diseases when expected, or at all.

•We may be unable to successfully integrate new products or businesses we may acquire.

•We may become involved in litigation matters, which could result in substantial costs, divert management's attention and otherwise have a material adverse effect on our business, operating results or financial condition.

•We are subject to significant ongoing regulatory obligations and oversight, which may result in significant additional expense and may limit our commercial success.

3

PART I - FINANCIAL INFORMATION

Item 1. Financial Statements

TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED BALANCE SHEETS

(in thousands, except par value and share amounts)

	June 30, 2023		December 31, 2022
Assets	(unaudited)
Current assets:
Cash and cash equivalents	$	70,874	$	61,688
Marketable debt securities, at fair value	420,463		388,557
Accounts receivable, net	20,397		16,646
Inventory, net	18,765		6,922
Prepaid expenses and other current assets	11,556		12,624
Total current assets	542,055		486,437
Property and equipment, net	8,570		9,049
Operating lease right of use assets	19,559		21,000
Intangible assets, net	154,456		145,038
Other assets	11,789		11,061
Total assets	$	736,429	$	672,585
Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable	$	19,915	$	17,290
Accrued expenses	88,749		95,742
Deferred revenue, current portion	10,244		11,976
Business combination-related contingent consideration, current portion	6,900		7,000
Operating lease liabilities, current portion	4,663		4,433
Other current liabilities	5,240		5,722
Total current liabilities	135,711		142,163
Convertible debt	376,403		375,545
Deferred revenue, less current portion	6,788		10,931
Business combination-related contingent consideration, less current portion	67,200		64,200
Operating lease liabilities, less current portion	25,106		27,510
Other non-current liabilities	8,736		9,385
Total liabilities	619,944		629,734
Commitments and Contingencies (See Note 13)
Stockholders' Equity:
Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of June 30, 2023 and December 31, 2022	—		—
Common stock $0.0001 par value; 200,000,000 shares authorized; 74,971,807, and 64,290,570 issued and outstanding as of June 30, 2023 and December 31, 2022, respectively	7		6
Additional paid-in capital	1,306,517		1,059,975
Accumulated deficit	(1,186,184)		(1,014,223)
Accumulated other comprehensive loss	(3,855)		(2,907)
Total stockholders' equity	116,485		42,851
Total liabilities and stockholders' equity	$	736,429	$	672,585

The accompanying notes are an integral part of these consolidated financial statements.

4

TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(in thousands, except share and per share amounts)

(unaudited)

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Net product sales	$	57,012	$	50,950	$	107,295	$	97,393
License and collaboration revenue	2,685		3,217		9,395		5,261
Total revenue	59,697		54,167		116,690		102,654
Operating expenses:
Cost of goods sold	1,990		2,051		7,115		4,189
Research and development	69,411		59,681		129,324		116,292
Selling, general and administrative	74,037		52,979		146,282		99,767
Change in fair value of contingent consideration	1,840		4,907		8,596		13,987
Total operating expenses	147,278		119,618		291,317		234,235
Operating loss	(87,581)		(65,451)		(174,627)		(131,581)
Other income (expenses), net:
Interest income	5,128		782		8,774		1,060
Interest expense	(2,911)		(2,972)		(5,851)		(5,487)
Other (expense) income, net	(201)		662		(114)		688
Loss on extinguishment of debt	—		—		—		(7,578)
Total other income (expense), net	2,016		(1,528)		2,809		(11,317)
Loss before income tax provision	(85,565)		(66,979)		(171,818)		(142,898)
Income tax provision	(65)		(53)		(143)		(105)
Net loss	$	(85,630)	$	(67,032)	$	(171,961)	$	(143,003)
Basic and diluted net loss per common share	$	(1.13)	$	(1.05)	$	(2.38)	$	(2.26)
Basic and diluted weighted average common shares outstanding	76,001,801		63,638,385		72,109,573		63,387,009
Comprehensive loss:
Net loss	$	(85,630)	$	(67,032)	$	(171,961)	$	(143,003)
Foreign currency translation (loss) gain	(170)		1,416		(736)		1,487
Unrealized loss on marketable debt securities	(1,509)		(803)		(212)		(2,007)
Comprehensive loss	$	(87,309)	$	(66,419)	$	(172,909)	$	(143,523)

The accompanying notes are an integral part of these consolidated financial statements.

5

TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY

(unaudited, in thousands, except share amounts)

	Three Months Ended June 30, 2023											Three Months Ended June 30, 2022
	Common Stock			Additional Paid in Capital		Accumulated Other Comprehensive Loss		Accumulated Deficit		Total Stockholders' Equity		Common Stock			Additional Paid in Capital		Accumulated Other Comprehensive Loss		Accumulated Deficit		Total Stockholders' Equity
	Shares	Amount										Shares	Amount
Balance - March 31	74,586,806	$	7	$	1,291,863	$	(2,176)	$	(1,100,554)	$	189,140	63,510,277	$	6	$	1,021,542	$	(1,695)	$	(811,712)	$	208,141
Share based compensation	—	—		11,172		—		—		11,172		—	—		12,352		—		—		12,352
Issuance of common stock under the equity incentive plan and proceeds from exercise	228,461	—		793		—		—		793		250,598	—		824		—		—		824
Employee stock purchase program purchase and expense	156,540	—		2,689		—		—		2,689		77,175	—		1,815		—		—		1,815
Foreign currency translation adjustments	—	—		—		(170)		—		(170)		—	—		—		1,416		—		1,416
Unrealized loss on marketable debt securities	—	—		—		(1,509)		—		(1,509)		—	—		—		(803)		—		(803)
Net loss	—	—		—		—		(85,630)		(85,630)		—	—		—		—		(67,032)		(67,032)
Balance - June 30	74,971,807	$	7	$	1,306,517	$	(3,855)	$	(1,186,184)	$	116,485	63,838,050	$	6	$	1,036,533	$	(1,082)	$	(878,744)	$	156,713

The accompanying notes are an integral part of these consolidated financial statements.

6

TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (continued)

(unaudited, in thousands, except share amounts)

	Six Months Ended June 30, 2023											Six Months Ended June 30, 2022
	Common Stock			Additional Paid in Capital		Accumulated Other Comprehensive Loss		Accumulated Deficit		Total Stockholders' Equity		Common Stock			Additional Paid in Capital		Accumulated Other Comprehensive Loss		Accumulated Deficit		Total Stockholders' Equity
	Shares	Amount										Shares	Amount
Balance - December 31	64,290,570	$	6	$	1,059,975	$	(2,907)	$	(1,014,223)	$	42,851	62,491,498	$	6	$	1,068,634	$	(562)	$	(765,966)	$	302,112
Cumulative-effect adjustment from adoption of ASU 2020-06	—	—				—		—		—		—	—		(74,945)		—		30,225		(44,720)
Share based compensation	—	—		24,497		—		—		24,497		—	—		20,287		—		—		20,287
Issuance of common stock under the equity incentive plan and proceeds from exercise	820,947	—		3,089		—		—		3,089		567,777	—		947		—		—		947
Employee stock purchase program purchase and expense	156,540	—		3,128		—		—		3,128		77,175	—		2,065		—		—		2,065
Equity offering, net of issuance costs of $12.6 million	9,703,750	1		191,198		—		—		191,199		—	—		—		—		—		—
Issuance of pre-funded common stock warrants, net of issuance costs of $1.6 million	—	—		24,630		—		—		24,630		—	—		—		—		—		—
Issuance of common stock under At-The-Market offering, net of issuance costs of $0.6 million	—	—		—		—		—		—		701,600	—		19,545		—		—		19,545
Foreign currency translation adjustments	—	—		—		(736)		—		(736)		—	—		—		1,487		—		1,487
Unrealized loss on marketable debt securities	—	—		—		(212)		—		(212)		—	—		—		(2,007)		—		(2,007)
Net loss	—	—		—		—		(171,961)		(171,961)		—	—		—		—		(143,003)		(143,003)
Balance - June 30	74,971,807	$	7	$	1,306,517	$	(3,855)	$	(1,186,184)	$	116,485	63,838,050	$	6	$	1,036,533	$	(1,082)	$	(878,744)	$	156,713

The accompanying notes are an integral part of these consolidated financial statements.

7

TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED STATEMENTS OF CASH FLOWS

(unaudited, in thousands)

	For the Six Months Ended June 30,
	2023		2022
Cash Flows From Operating Activities:
Net loss	$	(171,961)	$	(143,003)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	23,449		15,200
Share based compensation	25,368		20,823
Change in estimated fair value of contingent consideration	8,596		13,987
Payments from change in fair value of contingent consideration	(4,890)		(4,247)
Amortization of (discounts) premiums on investments	(3,329)		895
Loss on extinguishment of debt	—		7,578
Other	1,367		3,980
Changes in operating assets and liabilities:
Accounts receivable	(3,735)		(777)
Inventory	(13,022)		(2,060)
Prepaid expenses and other current and non-current assets	933		(2,336)
Change in lease assets and liabilities, net	(492)		(492)
Accounts payable	2,512		(2,917)
Accrued expenses	(7,904)		9,070
Deferred revenue, current and non-current	(6,823)		(5,700)
Other current and non-current liabilities	(483)		(2,127)
Net cash used in operating activities	(150,414)		(92,126)
Cash Flows From Investing Activities:
Proceeds from the sale/maturity of marketable debt securities	196,871		217,325
Purchase of marketable debt securities	(225,660)		(206,529)
Purchase of intangible assets	(31,170)		(16,579)
Other	(633)		(148)
Net cash used in by investing activities	(60,592)		(5,931)
Cash Flows From Financing Activities:
Payment of guaranteed minimum royalty	(1,050)		(1,050)
Payment of business combination-related contingent consideration	(863)		(1,271)
Proceeds from issuances of 2029 convertible senior notes	—		316,250
Payment of debt issuance costs	—		(9,882)
Repurchase of 2025 convertible senior notes including premium	—		(211,324)
Proceeds from the issuance of common stock, net of issuance costs	191,198		—
Proceeds from the issuance of pre-funded warrants, net of issuance costs	24,630		—
Proceeds from exercise of stock options	3,089		947
Proceeds from issuances under the employee stock purchase plan	2,258		1,529
Proceeds from the issuance of common stock in At-the-Market equity offering, net of issuance costs	—		19,545
Net cash provided by financing activities	219,262		114,744
Effect of exchange rate changes on cash	930		(2,681)
Net increase in cash and cash equivalents	9,186		14,006
Cash and cash equivalents, beginning of year	61,688		165,753
Cash and cash equivalents, end of period	$	70,874	$	179,759

The accompanying notes are an integral part of these consolidated financial statements.

8

TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIES

NOTES TO THE UNAUDITED CONSOLIDATED FINANCIAL STATEMENTS

NOTE 1.  DESCRIPTION OF BUSINESS

Organization and Description of Business

Travere Therapeutics, Inc. (“we”, “our”, “us”, “Travere” and the “Company”) refers to Travere Therapeutics, Inc., a Delaware corporation, as well as its subsidiaries. Travere is a fully integrated biopharmaceutical company headquartered in San Diego, California focused on identifying, developing and delivering life-changing therapies to people living with rare kidney, liver, and metabolic diseases. The Company regularly evaluates and, where appropriate, acts on opportunities to expand its product pipeline through licenses and acquisitions of products in areas that will serve patients with serious unmet medical need and that the Company believes offer attractive growth characteristics.

FILSPARI™ (sparsentan)

On February 17, 2023, the U.S. Food and Drug Administration (the "FDA") granted accelerated approval of FILSPARI™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally at UPCR ≥1.5 gram/gram. FILSPARI, a once-daily, oral medication is designed to selectively target two critical pathways (endothelin 1 and angiotensin-II) in the disease progression of IgAN.

Clinical-Stage Programs:

The continued approval of FILSPARI for IgAN may be contingent upon confirmation of a clinical benefit in the Company's ongoing Phase 3 clinical trial of sparsentan for the treatment of IgAN (the "PROTECT Study"), which is designed to demonstrate whether FILSPARI slows kidney function decline. Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in the fourth quarter of 2023 and are intended to support traditional approval of FILSPARI.

Sparsentan remains a novel investigational product candidate which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis (FSGS) in the U.S. and Europe. The double-blind portion of the Phase 3 study of sparsentan for FSGS has recently concluded and, following release of the top-line data from the study which showed that the study did not meet its primary endpoint, the Company is conducting further analyses of the data and is preparing to engage with regulators to explore a potential path forward toward a potential regulatory submission in FSGS.

Pegtibatinase (TVT-058) is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. Pegtibatinase is currently being evaluated in the Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. In May 2023, the Company announced positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study. The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited.

Chenodal (chenodeoxycholic acid or CDCA) is a naturally occurring bile acid that is approved for the treatment of people with radiolucent stones in the gallbladder. In September 2022, the Company was granted Fast Track Designation by the FDA for the investigation of Chenodal for cerebrotendinous xanthomatosis (CTX). In January 2020, the Company randomized the first patients in its Phase 3 RESTORE Study to evaluate the effects of Chenodal in adult and pediatric patients with CTX, and the study enrollment remains open. The pivotal study is intended to support an NDA submission for marketing authorization of Chenodal for CTX in the United States. In July 2023, the Company entered into an Asset Purchase Agreement to sell substantially all of the Company’s assets that are primarily related to the Company’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam. See Note 18.

Preclinical Programs:

The Company is a participant in a Cooperative Research and Development Agreement ("CRADA"), which forms a multi-stakeholder approach to pool resources with leading experts, and incorporate the patient perspective early in the therapeutic identification and development process. The Company is partnering with the National Institutes of Health’s National Center for Advancing Translational Sciences ("NCATS") and a leading patient advocacy organization, Alagille Syndrome Alliance, aimed at the identification of potential small molecule therapeutics for Alagille syndrome ("ALGS"). There are no treatment options currently approved for ALGS.

The Company is party to a collaboration agreement with PharmaKrysto Limited and their early-stage cystinuria discovery program, whereby the Company is responsible for funding all research and development expenses for the pre-clinical activities associated with the cystinuria program.

Other Commercial Products:

•Thiola® and Thiola EC® (tiopronin tablets) are approved in the United States for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria.

•Cholbam® (cholic acid capsules) is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders.

•Chenodal (chenodiol tablets) is approved in the United States for the treatment of patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age.

9

NOTE 2.  BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES

The accompanying unaudited consolidated financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 23, 2023. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial information, the instructions for Form 10-Q and the rules and regulations of the SEC. Accordingly, since they are interim statements, the accompanying consolidated financial statements do not include all of the information and notes required by GAAP for annual financial statements, but reflect all adjustments consisting of normal, recurring adjustments, that are necessary for a fair statement of the financial position, results of operations and cash flows for the interim periods presented. Interim results are not necessarily indicative of the results that may be expected for any future periods. The December 31, 2022 balance sheet information was derived from the audited financial statements as of that date. Certain reclassifications have been made to the prior period consolidated financial statements to conform to the current period presentation.

A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows:

Principles of Consolidation

The unaudited consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation. See Note 6 for further discussion of variable interest entities (“VIE”) that the Company consolidates.

Revenue Recognition

The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Accounting Standards Codification ("ASC") 606, Revenue from Contracts with Customers ("ASC 606"), the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only recognizes revenue from contracts when it is probable that the entity will collect substantially all the consideration it is entitled to in exchange for the goods or services it transfers to the customer. See Note 3 and Note 4 for further discussion.

Payments received under collaboration and licensing agreements may include non-refundable fees at the inception of the arrangements, milestone payments for specific achievements and royalties on the sale of products. At the inception of arrangements that include milestone payments, the Company uses judgement to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, and is recorded in license and collaboration revenue.

The Company utilizes significant judgement to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgement in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin.

If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly.

The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary.

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Cost of goods sold

Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping and handling costs, and provisions for excess and obsolete inventory. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partner, at cost plus a margin.

The following table summarizes cost of goods sold for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Cost of goods sold - product sales	$	1,983	$	2,051	$	4,071	$	4,189
Cost of goods sold - license and collaboration	7		—		3,044		—
Total cost of goods sold	$	1,990	$	2,051	$	7,115	$	4,189

Capitalization of Inventory Costs

Prior to the regulatory approval of the Company's drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. The Company capitalizes inventory costs associated with its products after regulatory approval, when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Until the date at which regulatory approval has been received, costs related to the production of inventory are recorded as research and development expenses as incurred. Any eventual sale of previously expensed ("zero-cost") inventories may impact future margins, for any periods in which those inventories are sold.

Sales of FILSPARI for the three and six months ended June 30, 2023 primarily consisted of zero-cost inventories, which favorably impacted gross margin for related sales. Prior to the February 2023 FDA approval of FILSPARI (sparsentan), the Company recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredients to support the commercial launch of FILSPARI. Had these costs been included, total cost of goods sold would have increased by less than $0.1 million and by approximately $0.1 million for the three and six months ended June 30, 2023, respectively. The Company expects to continue to benefit from the sale of previously expensed inventories through at least 2024.

Research and Development Expenses

Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, develop drug materials and delivery devices, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.

Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered.

Clinical Trial Expenses

The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process.

Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain.

The Company currently has one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter.

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Intangible Assets with Cost Accumulation Model

In 2014, the Company entered into a license agreement with Mission Pharmacal in which the Company obtained the exclusive right to license the trademark of Thiola. The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the asset under the cost accumulation model ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining useful life.

In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows, resulting in incremental expense of $3.7 million for the three and six months ended June 30, 2023, recorded in selling, general, and administrative. The change in estimated useful life has been accounted for as a change in accounting estimate and the remaining carrying amounts of the Thiola Intangible will be amortized prospectively over the new useful life.

Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at June 30, 2023 as such royalties are not yet probable and estimable.

Variable Interest Entity

The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgements regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made.

Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.

Recently Adopted Accounting Pronouncements

In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU will require entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The ASU is effective for fiscal years beginning after December 15, 2021, including interim periods within those fiscal years. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 10, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million.

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption.

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NOTE 3. REVENUE RECOGNITION

Product Sales, Net

Product sales consist of FILSPARI, bile acid products (Chenodal and Cholbam) and tiopronin products (Thiola and Thiola EC). The Company sells its products to specialty pharmacies and through direct-to-patient distributors worldwide, with the United States and Canada representing approximately 98% and 1% of net product sales, respectively, and rest of world representing less than 1% of net product sales, based on the product shipment destination.

The Company sells FILSPARI to three direct-to-patient specialty pharmacies. The Company sells its other products to patients and pharmacies, with distribution facilitated through a single direct-to-patient distributor. Revenues from product sales are recognized in satisfaction of a single performance obligation when the customer obtains control of the Company’s product. For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's other products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its other products, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient. Contracts do not contain significant financing components based on the typical period of time between performance of services and collection of consideration.

Deductions from Revenue

Revenues from product sales are recorded at the net sales price, which includes provisions resulting from discounts, rebates and co-pay assistance that are offered to customers, payers and other indirect customers relating to the Company’s sales of its products. These provisions are based on the estimates of the amounts earned or to be claimed on the related sales. These amounts are treated as variable consideration, estimated and recognized as a reduction of the transaction price at the time of the sale, using the most likely amount method, and are classified as a reduction of accounts receivable (if the amount is payable to a customer) or as a current liability (if the amount is payable to a party other than a customer). The Company includes these estimated amounts in the transaction price to the extent it is probable that a significant reversal of cumulative revenue recognized for such transactions will not occur. Where appropriate, these reserves take into consideration the Company’s historical experience, current contractual and statutory requirements and specific known market events and trends. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contract. If actual results in the future vary from the Company’s provisions, the Company will adjust the estimate, which would affect net product revenue and earnings in the period such variances become known. For the six months ended June 30, 2023 and 2022, adjustments to net product revenue related to performance obligations satisfied in previous periods, were immaterial.

Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Commercial Rebates: The Company calculates the rebates it incurs according to any contracts with certain commercial payers and deducts these amounts from its gross product sales at the time the revenues are recognized. Allowances for commercial rebates are established based on actual payer information, which is reasonably estimated at the time of delivery for applicable products. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale.

Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. Historically, returns have been immaterial.

Co-pay Assistance: The Company offers a co-pay assistance program, which is intended to provide financial assistance to qualified commercially insured patients with prescription drug co-payments required by payers. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue.

The following table summarizes net product sales for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Bile acid products	$	27,501	$	25,534	$	53,606	$	50,609
Tiopronin products	26,050		25,416		47,224		46,784
FILSPARI	3,461		—		6,465		—
Total net product sales	$	57,012	$	50,950	$	107,295	$	97,393

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NOTE 4. COLLABORATION AND LICENSE AGREEMENTS

On September 15, 2021, the Company entered into a license and collaboration agreement (“License Agreement”) with Vifor (International) Ltd. (“CSL Vifor”), pursuant to which the Company granted an exclusive license to CSL Vifor for the commercialization of sparsentan in Europe, Australia and New Zealand ("Licensed Territories"). CSL Vifor also has first right of negotiation to expand the licensed territories into Canada, China, Brazil and/ or Mexico. Under the terms of the License Agreement, the Company received an upfront payment of $55.0 million and will be eligible for up to $135.0 million in aggregate regulatory and market access related milestone payments and up to $655.0 million in aggregate sales-based milestone payments for a total potential value of up to $845.0 million. The Company is also entitled to receive tiered double-digit royalties of up to 40 percent of annual net sales of sparsentan in the Licensed Territories.

Under the License Agreement, CSL Vifor will be responsible for all commercialization activities in the Licensed Territories. The Company remains responsible for the worldwide clinical development of sparsentan through regulatory approval as defined and will retain all rights to sparsentan in the United States and rest of world outside of the Licensed Territories. Development costs for any post regulatory approval development activities, subject to approval by both parties, will be borne by the Company and CSL Vifor as defined, respectively. The License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the licensed territories. Each party has the right to terminate the License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the License Agreement by the other party is extended due to a force majeure event that continues for six months or more.

The Company assessed the License Agreement and determined that it meets both criteria to be considered a collaborative agreement within the Scope of ASC 808, Collaborative Arrangements of active participation by both parties and exposures to significant risks and rewards dependent on the commercial success of the activities. Both parties participate on joint steering and other committees overseeing the collaboration activities. Also, both parties are exposed to significant risks and rewards based on the economic outcomes of regulatory approvals and commercialization of sparsentan.

The Company determined the transaction price under the License Agreement totaled $55.0 million, consisting of the fixed non-refundable upfront payment. The variable regulatory and access related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the transaction price and will be recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated have been satisfied.

The Company concluded that CSL Vifor represented a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct from the promise to provide clinical development services resulting in two performance obligations. As a result, the Company allocated $12.0 million of the transaction price, based on the performance obligations' relative standalone selling prices, to the license, which was recognized in full in 2021. The remaining $43.0 million of the transaction price was allocated to the clinical development activities and recorded as deferred revenue, which will be recognized over the development period based upon the ratio of costs incurred to date to the total estimated costs.

For the three months ended June 30, 2023, the Company recognized $2.7 million in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the six months ended June 30, 2023, the Company recognized $9.4 million in license and collaboration revenue, which consisted of $3.3 million from the sale of active pharmaceutical ingredients to CSL Vifor at cost plus a margin, and $6.1 million for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the three and six months ended June 30, 2022, the Company recognized $3.2 million and $5.3 million, respectively, in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs.

Deferred revenue related to the clinical development activities as of June 30, 2023 was $17.0 million. Of this amount, $10.2 million was classified as current as of June 30, 2023, based upon amounts expected to be realized within the next year.

NOTE 5. MARKETABLE DEBT SECURITIES

The Company's marketable debt securities as of June 30, 2023 and December 31, 2022 were composed of available-for-sale commercial paper and corporate and government debt securities. The primary objective of the Company’s investment portfolio is to preserve capital and liquidity while enhancing overall returns. The Company’s investment policy limits interest-bearing security investments to certain types of instruments issued by institutions with primarily investment grade credit ratings and places restrictions on maturities and concentration by asset class and issuer.

Marketable debt securities consisted of the following (in thousands):

	June 30, 2023		December 31, 2022
Marketable debt securities:
Commercial paper	$	97,201	$	123,647
Corporate debt securities	235,631		224,055
Securities of government sponsored entities	87,631		40,855
Total available-for-sale marketable debt securities	$	420,463	$	388,557

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The following is a summary of short-term marketable debt securities classified as available-for-sale as of June 30, 2023 (in thousands):

	Remaining Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	97,362	$	—	$	(161)	$	97,201
Corporate debt securities	Less than 1	97,682		8		(847)		96,843
Securities of government-sponsored entities	Less than 1	37,294		—		(229)		37,065
Total maturity less than 1 year		232,338		8		(1,237)		231,109
Corporate debt securities	1 to 2	140,014		33		(1,259)		138,788
Securities of government-sponsored entities	1 to 2	51,277		—		(711)		50,566
Total maturity 1 to 2 years		191,291		33		(1,970)		189,354
Total available-for-sale marketable debt securities		$	423,629	$	41	$	(3,207)	$	420,463

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 (in thousands):

	Remaining Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	124,301	$	2	$	(656)	$	123,647
Corporate debt securities	Less than 1	155,841		—		(1,355)		154,486
Securities of government-sponsored entities	Less than 1	7,473		—		(80)		7,393
Total maturity less than 1 year		287,615		2		(2,091)		285,526
Corporate debt securities	1 to 2	70,195		33		(659)		69,569
Securities of government-sponsored entities	1 to 2	33,702		6		(246)		33,462
Total maturity 1 to 2 years		103,897		39		(905)		103,031
Total available-for-sale securities		$	391,512	$	41	$	(2,996)	$	388,557

During the six months ended June 30, 2023, investment activity for the Company included $196.9 million in maturities and $225.7 million in purchases, all relating to debt-based marketable securities. During the six months ended June 30, 2022, investment activity for the Company included $217.3 million in maturities and $206.5 million in purchases, all relating to debt-based marketable securities. As of June 30, 2023 and December 31, 2022, the accrued interest receivable related to the Company's marketable debt securities was $2.5 million and $1.9 million, respectively, and was recorded in prepaid expenses and other current assets on the Consolidated Balance Sheets.

The Company reviews the available-for-sale marketable debt securities for declines in fair value below the cost basis each quarter. For any security whose fair value is below its amortized cost basis, the Company first evaluates whether it intends to sell the impaired security, or will otherwise be more likely than not required to sell the security before recovery. If either are true, the amortized cost basis of the security is written down to its fair value at the reporting date. If neither circumstance holds true, the Company assesses whether any portion of the unrealized loss is a result of a credit loss. Any amount deemed to be attributable to credit loss is recognized in the income statement, with the amount of the loss limited to the difference between fair value and amortized cost and recorded as an allowance for credit losses. The portion of the unrealized loss related to factors other than credit losses is recognized in other comprehensive income (loss).

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of June 30, 2023 (in thousands):

	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	97,201	$	161	$	—	$	—	$	97,201	$	161
Corporate debt securities	168,000		1,680		43,621		426		211,621		2,106
Securities of government-sponsored entities	86,632		936		999		4		87,631		940
Total	$	351,833	$	2,777	$	44,620	$	430	$	396,453	$	3,207

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The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 (in thousands):

	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	117,853	$	656	$	—	$	—	$	117,853	$	656
Corporate debt securities	99,066		1,041		107,964		973		207,030		2,014
Securities of government-sponsored entities	31,402		263		4,456		63		35,858		326
Total	$	248,321	$	1,960	$	112,420	$	1,036	$	360,741	$	2,996

As of June 30, 2023 and December 31, 2022, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $399.7 million and $363.7 million, respectively.

As of June 30, 2023 and December 31, 2022, the Company does not intend to sell these investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis. The increase in unrealized losses for the six months ended June 30, 2023 was primarily due to fluctuations in short-term interest rates. The Company does not believe the unrealized losses incurred during the period are due to credit-related factors. The credit ratings of the securities held remain of the highest quality. Moreover, the Company continues to receive payments of interest and principal as they become due, and our expectation is that those payments will continue to be received timely. Factors unknown to us at this time may cause actual results to differ and require adjustments to the Company’s estimates and assumptions in the future.

NOTE 6. VARIABLE INTEREST ENTITIES

On March 8, 2022, the Company entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company related to PharmaKrysto's early-stage cystinuria discovery program, and concurrently therewith entered into a Stock Purchase Agreement with PharmaKrysto (together, the "Agreements"). Pursuant to the terms of the Agreements, the Company paid PharmaKrysto's shareholders $0.6 million in cash to purchase 5% of the outstanding common shares of PharmaKrysto and $0.4 million to PharmaKrysto as a one-time signing fee. Under the Collaboration Agreement, the Company will fund all research and development expenses for the pre-clinical activities associated with the cystinuria program, which are estimated to be approximately $5.0 million. The Agreements require the Company to purchase an additional 5% of the outstanding common shares for $1.0 million upon the occurrence of a specified pre-clinical milestone, and grant an option to the Company to purchase all of the remaining outstanding shares of PharmaKrysto for $5.0 million upon the occurrence of a subsequent pre-clinical milestone prior to expiration of the option on March 8, 2025. If the Company elects to exercise the option, it would be required to perform commercially reasonable clinical diligence obligations. In addition, it would be required to make cash milestone payments totaling up to an aggregate $16.0 million upon the achievement of certain development and regulatory milestones, plus tiered royalty payments of less than 4% on future net sales of a product, if approved. The Company has the right to terminate the Agreements and return the shares for a nominal price at any time upon 60 days’ notice, subject to survival of contingent obligations, if any.

The Company determined that PharmaKrysto is a VIE because it lacks the resources to conduct the cystinuria clinical program and the limitation on the residual returns through the Company's option to purchase the remaining outstanding shares. The Company further concluded that it is the primary beneficiary of the VIE due to the Company's ultimate control over the research and development program, and its obligation, subject to continuation of the collaboration, to fund 100% of research and development costs of the program pursuant to the terms of the Collaboration Agreement.

The upfront payments were expensed to research and development and other income (expense), net upon initial consolidation. The consolidated assets and liabilities as of June 30, 2023 and December 31, 2022 were immaterial. The results of operations were not significant for the three and six months ended June 30, 2023 and 2022. The Company is not required to provide additional funding other than the contractually required amounts disclosed above. The creditors and beneficial holders of PharmaKrysto have no recourse to the general credit or assets of the Company.

NOTE 7.  LEASES

As of June 30, 2023, the Company had two operating leases, including one operating lease with Kilroy Realty, L.P. (the "Landlord") for office space located in San Diego, California, which was entered into in April 2019 and subsequently amended in May 2020. Coinciding with the Company's ability to direct the use of the office space, which occurred in phases over 2020, and utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established ROU assets totaling $34.6 million and lease liabilities totaling $34.5 million. The total ROU asset and lease liability at measurement were each offset by lease incentives associated with tenant improvement allowances totaling $7.9 million.

The initial term of the office lease ends in August 2028, and the Landlord has granted the Company an option to extend the term of the lease by a period of 5 years. At lease inception, it was not reasonably certain that the Company will extend the term of the lease and therefore the renewal period has been excluded from the aforementioned ROU asset and lease liability measurements. The measurement of the lease term occurs from the February 2021 occupancy date of the office space delivered in September 2020.

The Company has one operating lease with Esprit Investments Limited for office space located in Dublin, Ireland, which was entered into in October 2022. The initial term of the office lease ends in September 2027. The lease provides the option to extend the term of the lease by a period of 5 years, although at lease inception, it was not reasonably certain that the Company would elect this option and therefore the renewal period was excluded from the initial lease

16

measurement. The aggregate base rent due over the initial term of the lease is approximately $0.5 million. Utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established an ROU asset and corresponding lease liability of $0.4 million.

Following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of June 30, 2023 (in thousands):

	June 30, 2023
2023 (remaining six months)	$	3,174
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	35,082
Present value discount	(5,313)
Total lease liability	29,769
Unamortized lease incentives	(5,083)
Cash payments in excess of straight-line lease expense	(5,127)
Total ROU asset	$	19,559

The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:

	June 30, 2023		December 31, 2022
Weighted-average remaining lease term in years	5.2		5.7
Weighted-average discount rate	6.48	%	6.48	%

For the three and six months ended June 30, 2023, the Company recorded $1.2 million and $2.5 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances. For the three and six months ended June 30, 2022, the Company recorded $1.3 million and $2.5 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances.

NOTE 8.  FAIR VALUE MEASUREMENTS

The Company utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy are described below:

Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and

Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.

The valuation techniques used to measure the fair value of the Company’s debt securities and all other financial instruments, all of which have counter-parties with high credit ratings, were valued based on quoted market prices or model driven valuations using significant inputs derived from or corroborated by observable market data. Based on the fair value hierarchy, the Company classified marketable debt securities within Level 2.

Financial instruments with carrying values approximating fair value include cash and cash equivalents, accounts receivable, and accounts payable, due to their short-term nature. As of June 30, 2023, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $65.4 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $284.9 million. As of December 31, 2022, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $62.9 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $283.0 million. The fair values were estimated utilizing market quotations and are considered Level 2.

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The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of June 30, 2023 (in thousands):

	As of June 30, 2023
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	$	70,874	$	70,874	$	—	$	—
Marketable debt securities, available-for-sale	420,463		—		420,463		—
Total	$	491,337	$	70,874	$	420,463	$	—
Liabilities:
Business combination-related contingent consideration	$	74,100	$	—	$	—	$	74,100
Total	$	74,100	$	—	$	—	$	74,100

The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2022 (in thousands):

	As of December 31, 2022
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	$	61,688	$	61,688	$	—	$	—
Marketable debt securities, available-for-sale	388,557		—		388,557		—
Total	$	450,245	$	61,688	$	388,557	$	—
Liabilities:
Business combination-related contingent consideration	$	71,200	$	—	$	—	$	71,200
Total	$	71,200	$	—	$	—	$	71,200

The Company acquired two businesses, related to the Cholbam and Chenodal products, whose purchase price included potential future payments that are contingent on the achievement of certain milestones and percentages of future net sales derived from the products acquired. The Company recorded contingent consideration liabilities at their fair value on the acquisition date and revalues them at the end of each reporting period. In estimating the fair value of the Company’s contingent consideration, the Company uses a Monte Carlo Simulation. The determination of the contingent consideration liabilities requires significant judgements including the appropriateness of the valuation model and reasonableness of estimates and assumptions included in the forecasts of future net sales and the discount rates applied to such forecasts. Changes in these estimates and assumptions could have a significant impact on the fair value of the contingent consideration liabilities.

Discount rates used to determine the fair value at June 30, 2023 and December 31, 2022 are as follows:

	Revenue Discount		Payment Discount
	Cholbam	Chenodal
June 30, 2023	7.50%	7.50%	6.90%
December 31, 2022	7.75%	8.00%	8.10%

Based on the fair value hierarchy, the Company classified the fair value measurement of contingent consideration within Level 3 because valuation inputs are based on projected revenues discounted to a present value.

The following table sets forth a summary of changes in the estimated fair value of the Company's Level 3 business combination-related contingent consideration for the six months ended June 30, 2023 and 2022 (in thousands):

	Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3)
	2023		2022
Balance at January 1	$	71,200	$	67,100
Changes in the fair value of contingent consideration	8,596		13,987
Contractual payments disbursed	(2,756)		(2,685)
Contractual payments included in accrued liabilities at June 30	(2,940)		(2,702)
Balance at June 30	$	74,100	$	75,700

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NOTE 9. INTANGIBLE ASSETS

Ligand License Agreement

In 2012, the Company entered into an agreement with Ligand Pharmaceuticals, Inc. ("Ligand") for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). As consideration for the license, the Company is required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million. In March 2023, the Company capitalized a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) that was triggered upon the accelerated approval of FILSPARI in February 2023. Pursuant to the Ligand License Agreement, the Company is obligated to pay to Ligand (and BMS) an escalating royalty between 15% and 17% of net sales of sparsentan, with payments due quarterly. The Company began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the three and six months ended June 30, 2023, the Company capitalized $0.5 million and $1.0 million, respectively, to intangible assets for royalties owed on net sales of FILSPARI. The cost of the $23.0 million milestone payment and royalty payments are being amortized to selling, general and administration on a straight-line basis through April 30, 2033.

The following table sets forth amortizable intangible assets as of June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Finite-lived intangible assets	$	334,685	$	302,935
Less: accumulated amortization	(181,165)		(158,833)
Net carrying value	$	153,520	$	144,102

As of June 30, 2023 and December 31, 2022, the Company had goodwill of $0.9 million.

The following table summarizes amortization expense for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Research and development	$	2,420	$	1,625	$	4,814	$	1,911
Selling, general and administrative	10,925		5,946		17,518		12,216
Total amortization expense	$	13,345	$	7,571	$	22,332	$	14,127

NOTE 10.  CONVERTIBLE NOTES PAYABLE

The composition of the Company’s convertible senior notes are as follows (in thousands):

	June 30, 2023		December 31, 2022
2.25% convertible senior notes due 2029	$	316,250	$	316,250
2.50% convertible senior notes due 2025	68,904		68,904
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029	(8,050)		(8,750)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025	(701)		(859)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$	376,403	$	375,545

Convertible Senior Notes Due 2029

On March 11, 2022, the Company completed a registered underwritten public offering of $316.3 million aggregate principal amount of 2.25% Convertible Senior Notes due 2029 (“2029 Notes”), which includes $41.3 million aggregate principal amount of 2029 Notes sold pursuant to the full exercise of the underwriters’ option to purchase additional 2029 Notes. The Company issued the 2029 Notes under an indenture, dated as of September 10, 2018, as supplemented by the second supplemental indenture, dated as of March 11, 2022 (collectively, the “2029 Indenture”). The 2029 Notes will mature on March 1, 2029, unless earlier repurchased, redeemed, or converted. The 2029 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.25%, payable semi-annually in arrears on March 1 and September 1 of each year, beginning on September 1, 2022.

The Company received net proceeds from the issuance of the 2029 Notes of $306.4 million, after deducting commissions and offering expenses of $9.9 million. At June 30, 2023, accrued interest on the 2029 Notes of $2.4 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2029 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2029 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.

Holders may convert their 2029 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on June 30, 2022 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days

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immediately after any 10 consecutive trading day period (such 10 consecutive trading day period, the “measurement period”) if the trading price per $1,000 principal amount of 2029 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions of the Company’s common stock; (4) if the Company calls the 2029 Notes for redemption; and (5) at any time from, and including, December 1, 2028 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate. The initial conversion rate for the 2029 Notes is 31.3740 shares of the Company’s common stock per $1,000 principal amount of 2029 Notes, which represents an initial conversion price of approximately $31.87 per share. If a “make-whole fundamental change” (as defined in the 2029 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.

The 2029 Notes will be redeemable, in whole or in part at the Company’s option at any time, and from time to time, on or after March 2, 2026 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2029 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on (1) each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice; and (2) the trading day immediately before the date the Company sends such notice. However, the Company may not redeem less than all of the outstanding 2029 Notes unless at least $100.0 million aggregate principal amount of 2029 Notes are outstanding and not called for redemption as of the time the Company sends the related redemption notice. In addition, calling any 2029 Note for redemption will constitute a make-whole fundamental change with respect to that 2029 Note, in which case the conversion rate applicable to the conversion of that 2029 Note will be increased in certain circumstances if it is converted after it is called for redemption. If a fundamental change (as defined in the 2029 Indenture) occurs, then, except as described in the 2029 Indentures, holders may require the Company to repurchase their 2029 Notes at a cash repurchase price equal to the principal amount of the 2029 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2029 Notes will be paid pursuant to the terms of the 2029 Indenture. In the event that all of the 2029 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2029 Notes for redemption will constitute a “make-whole fundamental change."

The Company incurred approximately $9.9 million of debt issuance costs relating to the issuance of the 2029 Notes, which were recorded as a reduction to the 2029 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2029 Notes using the effective interest method. We determined the expected life of the debt is equal to the seven-year term of the 2029 Notes. The effective interest rate on the 2029 Notes is 2.74%.

Convertible Senior Notes Due 2025

On September 10, 2018, the Company completed a registered underwritten public offering of $276.0 million aggregate principal amount of 2.50% Convertible Senior Notes due 2025 ("2025 Notes"), and entered into a base indenture and supplemental indenture agreement (collectively, the "2025 Indenture") with respect to the 2025 Notes. The 2025 Notes will mature on September 15, 2025, unless earlier repurchased, redeemed, or converted. The 2025 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.50%, payable semi-annually in arrears on March 15 and September 15 of each year, beginning on March 15, 2019.

The net proceeds from the issuance of the 2025 Notes were approximately $267.2 million, after deducting commissions and the offering expenses of $8.8 million payable by the Company. At June 30, 2023, accrued interest of $0.5 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2025 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2025 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.

Holders may convert their 2025 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on December 31, 2018 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days immediately after any 10 consecutive trading day period (“measurement period”) if the trading price per $1,000 principal amount of 2025 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions on the Company’s common stock; (4) if the Company calls the 2025 Notes for redemption; and (5) at any time from, and including, May 15, 2025 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate.

The initial conversion rate for the 2025 Notes is 25.7739 shares of the Company’s common stock per $1,000 principal amount of 2025 Notes, which represents an initial conversion price of approximately $38.80 per share. If a “make-whole fundamental change” (as defined in the 2025 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.

The 2025 Notes will be redeemable, in whole or in part, at the Company’s option at any time, and from time to time, on or after September 15, 2022 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2025 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date, but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on each of at least 20 trading days during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice. If a fundamental change (as

20

defined in the 2025 Indenture) occurs, then, subject to certain exceptions, holders may require the Company to repurchase their 2025 Notes at a cash repurchase price equal to the principal amount of the 2025 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2025 Notes will be paid pursuant to the terms of the 2025 Indenture. In the event that all of the 2025 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2025 Notes for redemption will constitute a “make-whole fundamental change."

The Company incurred approximately $8.8 million of debt issuance costs relating to the issuance of the 2025 Notes, which were recorded as a reduction to the 2025 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2025 Notes using the effective interest method. The Company determined the expected life of the debt is equal to the seven-year term of the 2025 Notes. The effective interest rate on the 2025 Notes is 2.98%.

On March 11, 2022, the Company completed its repurchase of $207.1 million aggregate principal amount of 2025 Notes for cash, including accrued and unpaid interest, for a total of $213.8 million. This transaction involved a contemporaneous exchange of cash between the Company and holders of the 2025 Notes participating in the issuance of the 2029 Notes. Accordingly, we evaluated the transaction for modification or extinguishment accounting in accordance with ASC 470-50, Debt – Modifications and Extinguishments on a creditor-by creditor basis depending on whether the exchange was determined to have substantially different terms. The repurchase of the 2025 Notes and issuance of the 2029 Notes were deemed to have substantially different terms based on the present value of the cash flows or significant difference between the value of the conversion option immediately prior to and after the exchange. Therefore, the repurchase of the 2025 Notes was accounted for as a debt extinguishment. The Company recorded a $7.6 million loss on extinguishment of debt on its Consolidated Statements of Operations for the six months ended June 30, 2022, which includes the write-off of related deferred financing costs of $3.4 million. After giving effect to the repurchase, the total remaining principal amount outstanding under the 2025 Notes as of June 30, 2023 was $68.9 million.

The 2025 and 2029 Notes are accounted for in accordance with ASC 470-20, Debt with conversion and Other Options (“ASC 470-20”) and ASC 815-40, Contracts in Entity’s Own Equity (“ASC 815-40”). Under ASC 815-40, to qualify for equity classification (or nonbifurcation, if embedded) the instrument (or embedded feature) must be both (1) indexed to the issuer’s stock and (2) meet the requirements of equity classification guidance. Based upon the Company’s analysis, it was determined that the 2025 Notes and the 2029 Notes do not contain embedded features requiring recognition as derivatives and bifurcation, and therefore are measured at amortized cost and recorded as liabilities on the Consolidated Balance Sheets.

The 2025 and 2029 Notes do not contain any financial or operating covenants or any restrictions on the payment of dividends, the issuance of other indebtedness or the issuance or repurchase of securities by the Company. There were no events of default for the 2025 Notes or 2029 Notes at June 30, 2023.

The 2025 and 2029 Notes are classified on the Company's Consolidated Balance Sheets at June 30, 2023 as long-tern convertible debt.

The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Contractual interest expense	$	2,210	$	2,210	$	4,419	$	4,053
Amortization of debt issuance costs	429		429		858		766
Total interest expense for the 2025 and 2029 Notes	$	2,639	$	2,639	$	5,277	$	4,819

Total interest expense recognized for the three and six months ended June 30, 2023 was $2.9 million and $5.9 million, respectively. Total interest expense recognized for the three and six months ended June 30, 2022 was $3.0 million and $5.5 million, respectively.

NOTE 11. ACCRUED EXPENSES

Accrued expenses at June 30, 2023 and December 31, 2022 consisted of the following (in thousands):

	June 30, 2023		December 31, 2022
Research and development	$	28,254	$	26,070
Compensation related costs	21,489		35,267
Sales discounts, rebates, and allowances	14,376		13,486
Selling, general and administrative	11,252		8,791
Accrued royalties	8,467		7,755
Miscellaneous accrued expenses	4,911		4,373
Total accrued expenses	$	88,749	$	95,742

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NOTE 12.  NET LOSS PER COMMON SHARE

Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period.

As discussed in Note 17, as part of its February 2023 underwritten public offering, the Company issued and sold pre-funded warrants to purchase 1.25 million shares of its common stock at a price to the public of $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. Since the $0.0001 price per share represents little consideration and is non-substantive in relation to the $20.9999 price per pre-funded warrant and the $21.00 price per share of the common stock offered to the public, and as the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them, the shares underlying the warrants are therefore included in the calculation of basic net loss per common share.

The Company’s potentially dilutive shares, which include outstanding stock options, restricted stock units, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents and are not included in the calculation of diluted net loss per share because their effect is anti-dilutive.

Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):

	Three Months Ended June 30,
	2023					2022
	Shares	Net Loss		Loss per common share		Shares	Net Loss		Loss per common share
Basic and diluted loss per share	76,001,801	$	(85,630)	$	(1.13)	63,638,385	$	(67,032)	$	(1.05)
	Six Months Ended June 30,
	2023					2022
	Shares	Net Loss		Loss per common share		Shares	Net Loss		Loss per common share
Basic and diluted loss per share	72,109,573	$	(171,961)	$	(2.38)	63,387,009	$	(143,003)	$	(2.26)

The following common stock equivalents have been excluded because they were anti-dilutive:

	Three Months Ended June 30,		Six Months Ended June 30,
	2023	2022	2023	2022
Convertible debt	11,697,952	11,697,952	11,697,952	10,026,309
Options	10,719,869	10,313,152	10,633,802	10,090,259
Restricted stock	3,626,814	2,160,842	3,453,767	2,031,149
Total anti-dilutive shares	26,044,635	24,171,946	25,785,521	22,147,717

NOTE 13.  COMMITMENTS AND CONTINGENCIES

Commitments

Certain of the Company's contractual arrangements with contract manufacturing organizations ("CMOs") require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's financial statements. As of June 30, 2023, we have commitments to purchase $22.1 million in active pharmaceutical ingredients, to be delivered in 2023, which is planned to support commercial sales of FILSPARI.

Contingencies

In October 2021, our Kolbam distributor in France notified us that the French authorities were seeking reimbursement for a portion of Kolbam sales in France during the periods from 2015-2020. During this period, the Company had aggregate revenues from sales of Kolbam in France of approximately $8.0 million. At this time, the Company is not able to estimate the potential liability that may be incurred, if any.

Legal Proceedings

From time to time in the normal course of business, the Company is subject to various legal matters such as threatened or pending claims or litigation. Although the results of claims and litigation cannot be predicted with certainty, the Company does not believe it is a party to any claim or litigation the outcome of which, if determined adversely to it, would individually or in the aggregate be reasonably expected to have a material adverse effect on its results of operations or financial condition.

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NOTE 14.  SHARE-BASED COMPENSATION

Stock Options

The following table summarizes stock option activity during the six months ended June 30, 2023:

	Shares Underlying Options	Weighted Average Exercise Price		Weighted Average Remaining Contractual Life (years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658
Granted	1,157,750	21.69		—	—
Exercised	(211,663)	14.59		—	1,341
Forfeited/canceled	(292,208)	25.72		—	—
Outstanding at June 30, 2023	10,586,301	$	21.60	5.82	$	3,677
Vested and expected to vest at June 30, 2023	10,586,301	$	21.60	5.82	$	3,677

At June 30, 2023, unamortized stock compensation for stock options was $35.0 million, with a weighted-average recognition period of 2.6 years.

At June 30, 2023, outstanding options to purchase 7.5 million shares of common stock were exercisable with a weighted-average exercise price per share of $20.71.

In connection with the retirement of the Company's former Chief Financial Officer, the Board of Directors approved a modification to extend the deadline to exercise each stock option held to the earlier of three months following the last vesting date or the original expiration date of the option, and to continue vesting on the original schedule of any underlying unvested stock options and restricted stock units. The modification resulted in incremental compensation cost of $2.6 million for the three and six months ended June 30, 2023.

Restricted Stock Units

Service Based Restricted Stock Units

The following table summarizes the Company’s service based restricted stock unit activity during the six months ended June 30, 2023:

	Number of Restricted Stock Units	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	2,343,709	$	24.65
Granted	1,773,692	21.97
Vested	(609,284)	23.33
Forfeited/canceled	(117,837)	23.83
Unvested at June 30, 2023	3,390,280	$	23.52

At June 30, 2023, unamortized stock compensation for service based restricted stock units was $67.8 million, with a weighted-average recognition period of 3.0 years.

Performance Based Restricted Stock Units

The following table summarizes the Company’s performance based restricted stock unit activity during the six months ended June 30, 2023:

	Number of Restricted Stock Units	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	157,048	$	25.24
Granted	66,250	22.40
Vested	—	—
Forfeited/canceled	(22,840)	24.86
Unvested at June 30, 2023	200,458	$	24.34

At June 30, 2023, unamortized stock compensation for performance based restricted stock units was $2.0 million, with a weighted-average recognition period of 0.9 years.

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Share-Based Compensation

The following table sets forth total share-based compensation for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Research and development	$	4,615	$	3,684	$	9,096	$	6,852
Selling, general and administrative	6,988		8,953		16,271		13,971
Total share-based compensation	$	11,603	$	12,637	$	25,367	$	20,823

NOTE 15. INVENTORY

Inventory consisted of the following at June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Raw materials	$	14,652	$	3,627
Finished goods	4,113		3,295
Total inventory	$	18,765	$	6,922

NOTE 16. ACCOUNTS RECEIVABLE

Accounts receivable, net of reserves for prompt pay discounts and expected credit losses, was $20.4 million and $16.6 million at June 30, 2023 and December 31, 2022, respectively. The total reserves for both periods were immaterial.

The Company's evaluation and accounting for credit losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses.

NOTE 17. EQUITY OFFERINGS

Underwritten Public Offering of Common Stock

In February 2023, the Company sold an aggregate of approximately 9.7 million shares of its common stock and pre-funded warrants to purchase 1.25 million shares of its common stock in an underwritten public offering, at a price to the public of $21.00 per share of common stock and $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately, subject to certain beneficial ownership limitations which can be modified by the respective holders with at least 61 days' notice, and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. The net proceeds to the Company from the offering, after deducting the underwriting discounts and offering expenses, were approximately $215.8 million.

The pre-funded warrants were classified as a component of permanent stockholders' equity within additional paid-in capital and were recorded at the issuance date using a relative fair value allocation method. The pre-funded warrants are equity classified because they (i) are freestanding financial instruments that are legally detachable and separately exercisable from the equity instruments, (ii) are immediately exercisable, (iii) do not embody an obligation for the Company to repurchase its shares, (iv) permit the holders to receive a fixed number of shares of common stock upon exercise, (v) are indexed to the Company's common stock and (vi) meet the equity classification criteria. In addition, such pre-funded warrants do not provide any guarantee of value or return. The Company valued the pre-funded warrants at issuance, concluding that their sale price approximated their fair value, and allocated the aggregate net proceeds from the sale proportionately to the common stock and prefunded warrants, including approximately $24.6 million allocated to the pre-funded warrants and recorded as a component of additional paid-in capital.

At-the-Market Equity Offering

In February 2020, the Company entered into an Open Market Sale Agreement ("ATM Agreement") with Jefferies LLC, as agent (“Jefferies”), pursuant to which the Company may offer and sell, from time to time through Jefferies, shares of its common stock having an aggregate offering price of up to $100.0 million. Of the $100.0 million originally authorized for sale under the ATM Agreement, approximately $28.6 million were sold under the Company’s prior registration statement on Form S-3 (Registration No. 333-227182). An additional $51.9 million were sold under the Company's effective registration statement on Form S-3 (Registration Statement No. 333-259311), which included $20.1 million in the year ended December 31, 2022. The Company did not sell any shares under the ATM Agreement during the six months ended June 30, 2023. As of June 30, 2023, an aggregate of $19.5 million remained eligible for sale under the ATM Agreement.

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NOTE 18. SUBSEQUENT EVENTS

On July 16, 2023, the Company executed an Asset Purchase Agreement (the “Purchase Agreement”) with Mirum Pharmaceuticals, Inc., a Delaware corporation (“Mirum”). Subject to the terms and conditions of the Purchase Agreement, at the closing (the “Closing”) of the transactions contemplated by the Purchase Agreement (the “Asset Purchase”), Mirum will purchase from the Company substantially all of the assets that are primarily related to the Company’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Products”) for an aggregate purchase price, subject to certain adjustments pursuant to the terms of the Purchase Agreement, of up to $445.0 million in cash, with $210.0 million due at the Closing and up to $235.0 million after the Closing, upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products (the “Milestone Events”).

Mirum has agreed to use certain specified resources and efforts during stipulated time-periods to obtain regulatory approval and to cause the Milestone Events to be achieved. The Company and Mirum have also entered into a transition services agreement pursuant to which the Company has agreed to perform certain services for a period of time following the Closing, with respect to Mirum’s use and operation of the assets purchased in the Asset Purchase.

The Company and Mirum are permitted under certain circumstances to terminate the Agreement, including in the event that the Closing has not occurred by October 16, 2023, which period will be automatically extended to April 16, 2024 if any condition remaining to be satisfied relates to regulatory approval.

Item 2.  Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited consolidated financial statements and related notes included in this Quarterly Report on Form 10-Q and the audited financial statements and notes thereto as of and for the year ended December 31, 2022 and the related Management’s Discussion and Analysis of Financial Condition and Results of Operations, both of which are contained in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission (SEC) on February 23, 2023. Past operating results are not necessarily indicative of results that may occur in future periods. In addition, see the discussion under the heading “Forward-Looking Statements” immediately preceding the consolidated financial statements included under Part I of this Quarterly Report on Form 10-Q.

Overview

We are a biopharmaceutical company headquartered in San Diego, California, focused on identifying, developing and delivering life-changing therapies to people living with rare kidney, liver, and metabolic diseases. Our approach centers on advancing our innovative pipeline with multiple late-stage clinical programs targeting rare diseases with significant unmet medical needs. Upon approval of any of our late-stage programs, we intend to leverage the skills of our talented commercial organization which has successfully identified, supported and treated patients prescribed our approved products over the last nine years.

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Our Pipeline and Approved Products

We have a diversified pipeline designed to address areas of high unmet need in rare kidney, liver, and metabolic diseases. We invest revenues from our commercial portfolio into our pipeline with the goal of delivering new treatments for diseases with limited or no approved therapies.

The following table summarizes the status of our clinical programs, preclinical programs and approved products, each of which is described in further detail below.

	CTX: cerebrotendinous xanthomatosis
1	On February 17, 2023, the FDA granted accelerated approval of FILSPARI™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 gram/gram.
2	On May 1, 2023, we announced topline primary efficacy results from the pivotal Phase 3 DUPLEX Study of sparsentan, as described below.
3	CDCA is not indicated for CTX, but has received a medical necessity determination in the United States by the FDA for CTX. We are conducting a Phase 3 clinical trial to examine the safety and efficacy of CDCA (Chenodal®) for the treatment of CTX.
4	In May 2023, the Company announced positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study of pegtibatinase in HCU.

FILSPARI™ (sparsentan)

On February 17, 2023, the U.S. Food and Drug Administration (the "FDA") granted accelerated approval of FILSPARI™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally at UPCR ≥1.5 gram/gram. FILSPARI became commercially available in the U.S. beginning the week of February 27, 2023, and we are providing a comprehensive patient support program throughout the patient’s treatment journey.

This indication is granted under accelerated approval based on reduction in proteinuria. It has not been established whether FILSPARI slows kidney function decline in patients with IgAN. The continued approval of FILSPARI may be contingent upon confirmation of a clinical benefit in the ongoing Phase 3 PROTECT Study, which is designed to demonstrate whether FILSPARI slows kidney function decline. Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in late third quarter or early fourth quarter of 2023 and are intended to support traditional approval of FILSPARI.

FILSPARI, a once-daily, oral medication is designed to selectively target two critical pathways in the disease progression of IgAN (endothelin 1 and angiotensin-II) and is the first and only non-immunosuppressive therapy approved for the treatment of this condition.

FILSPARI (sparsentan) is a dual endothelin angiotensin receptor antagonist (DEARA). Pre-clinical data have shown that blockade of both endothelin type A and angiotensin II type 1 pathways in forms of rare chronic kidney disease, reduces proteinuria, protects podocytes and prevents glomerulosclerosis and mesangial cell proliferation. Sparsentan has been granted Orphan Drug Designation for the treatment of IgAN in the U.S. and Europe and FILSPARI has been granted seven years of Orphan Drug Exclusivity for proteinuria in adults with primary IgAN at risk of rapid disease progression.

IgAN is characterized by hematuria, proteinuria, and variable rates of progressive renal failure. With an estimated prevalence of up to 150,000 people in the United States and greater numbers in Europe and Asia, IgAN is the most common primary glomerular disease. Most patients are diagnosed between the

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ages of 16 and 35, with up to 40% progressing to end stage kidney disease within 15 years. FILSPARI is the first and only non-immunosuppressive therapy approved for this condition. We expect approximately 30,000 to 50,000 patients in the United States to be addressable under FILSPARI's accelerated approval indication statement.

The approval of FILSPARI, granted under the FDA’s accelerated approval pathway, is based on clinically meaningful and statistically significant improvements in proteinuria compared to an active comparator in the pivotal and ongoing Phase 3 PROTECT Study, the largest head-to-head interventional study to date in IgAN. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400mg of sparsentan, compared to 300mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite available ACE or ARB therapy.

The PROTECT Study protocol provided for an unblinded analysis of at least 280 patients to be performed after 36 weeks of treatment to evaluate the primary efficacy endpoint - the change in proteinuria (UPCR) at week 36 from baseline. Secondary efficacy endpoints include the rate of change in eGFR following the initiation of randomized treatment over 58-week and 110-week periods, as well as the rate of change in eGFR over 52-week and 104-week periods following the first six weeks of randomized treatment in approximately 380 patients. In August 2021, we announced positive topline interim results from the ongoing Phase 3 PROTECT Study. The PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. After 36 weeks of treatment, patients receiving FILSPARI achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to a mean reduction in proteinuria from baseline of 15.1 percent for irbesartan-treated patients (p<0.0001). We believe that preliminary eGFR data available at the time of the interim analysis are indicative of a potential clinically meaningful treatment effect after two years of treatment. Results from the interim assessment in the PROTECT Study showed that FILSPARI was well tolerated with a clearly defined safety profile that has been consistent across all clinical trials conducted to date. In PROTECT, the most common adverse reactions (≥ 5%) are peripheral edema, hypotension (including orthostatic hypotension), dizziness, hyperkalemia, and anemia. Because of the risks of liver injury and birth defects, FILSPARI is available only through a REMS approved by the FDA.

Per request from the FDA, the efficacy data contained in the FDA-approved label is a post-hoc sensitivity analysis that evaluates the first 281 randomized patients, a subset of the full trial population. The mean reduction in proteinuria from baseline in the post-hoc sensitivity analysis is 45% for FILSPARI versus 15% for the active control, irbesartan. Both the pre-specified and post-hoc sensitivity analyses have demonstrated that FILSPARI achieves a rapid and sustained reduction in proteinuria, with statistically significant and clinically meaningful improvement compared to the active comparator irbesartan. The PROTECT Study is fully enrolled and is scheduled to continue as planned on a blinded basis to assess the treatment effect on eGFR slope over 110 weeks in the confirmatory endpoint analysis.

Beginning in 2023, we plan to expand data generation through a sub study in the open-label extension of the ongoing PROTECT Study, as well as an open-label clinical study to investigate the safety and efficacy of sparsentan in combination with sodium glucose cotransporter-2 inhibitors (SGLT2i) for the treatment of IgAN.

In August 2022, we and Vifor (International) Ltd. ("CSL Vifor"), with whom we entered into a license and collaboration agreement ("License Agreement") in September 2021, announced that the European Medicines Agency (the "EMA") had accepted for review the conditional marketing authorization application of sparsentan for the treatment of IgAN in Europe. We anticipate a review opinion by the Committee for Medicinal Products for Human Use (the "CHMP") around the end of 2023.

Clinical-Stage Programs:

Sparsentan for the treatment of FSGS

Sparsentan has been granted Orphan Drug Designation for the treatment of FSGS in the U.S. and Europe.

FSGS is a leading cause of end-stage kidney disease (ESKD) and nephrotic syndrome. There are currently no FDA-approved pharmacologic treatments for FSGS and there remains a high unmet need for patients living with FSGS as off-label treatments such as ACE/ARBs, steroids, and immunosuppressant agents are effective in only a subset of patients and use of some of these off-label treatments may be further inhibited by their safety profiles. Every year approximately 5,400 patients are diagnosed with FSGS and we estimate that there are more than 40,000 FSGS patients in the United States and a similar number in Europe with approximately half of them being candidates for sparsentan.

In 2016, we generated positive data from our Phase 2 DUET study in FSGS. In 2018, we announced the initiation of the Phase 3 clinical trial designed to serve as the basis for an NDA and MAA filing for sparsentan for the treatment of FSGS (the "DUPLEX Study"). The DUPLEX Study is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of sparsentan in 371 patients. The DUPLEX Study protocol provided for an unblinded analysis of at least 190 patients to be performed after 36 weeks of treatment to evaluate the interim efficacy endpoint - the proportion of patients achieving a FSGS partial remission of proteinuria endpoint (FPRE), which is defined as urine protein-to-creatinine ratio (UPCR) ≤1.5 g/g and a >40% reduction in UPCR from baseline, at week 36. In February 2021, we announced that the ongoing Phase 3 DUPLEX Study achieved its pre-specified interim FSGS partial remission of proteinuria endpoint following the 36-week interim period. After 36 weeks of treatment, 42.0 percent of patients receiving sparsentan achieved FPRE, compared to 26.0 percent of irbesartan-treated patients (p=0.0094). Following engagement with the FDA on the interim proteinuria analysis and a subsequent eGFR datacut, we elected to forego the previously planned submission for accelerated approval and pursue a potential traditional approval upon completion of the DUPLEX Study.

In May 2023, we announced topline primary efficacy results from the pivotal Phase 3 DUPLEX Study of sparsentan in FSGS. The confirmatory primary endpoint of the DUPLEX Study to support traditional regulatory approval is the rate of change in eGFR over 108 weeks of treatment. At the end of the 108-week double-blind period, sparsentan was observed to have a 0.3 mL/min/1.73m2 per year (95% CI: -1.74, 2.41) favorable difference on eGFR total slope and a 0.9 mL/min/1.73m2 per year (95% CI: -1.27, 3.04) favorable difference on eGFR chronic slope compared to the active control irbesartan, which was not statistically significant. After 108 weeks of treatment, sparsentan achieved a mean reduction in proteinuria from baseline of 50%, compared to 32% for irbesartan. Although the DUPLEX Study did not achieve its two-year primary endpoint with statistical significance over the active control irbesartan, we are encouraged by topline results for the secondary endpoints on proteinuria and topline exploratory endpoints, including renal outcomes, which trended favorably for sparsentan. In addition, a preliminary review of the safety results through 108 weeks of treatment indicate sparsentan has been generally well-tolerated and the overall safety profile in the study to date has been generally consistent between treatment groups.

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We are continuing to analyze the data to further evaluate the potential for sparsentan as a treatment for FSGS, and are planning to engage with regulators to explore a potential path forward for a supplemental New Drug Application (sNDA) in the U.S. Together with CSL Vifor, we also plan to engage with the EMA to determine the potential for a subsequent variation to the Conditional Marketing Authorization (CMA) of sparsentan for the treatment of FSGS, subject to a review decision on the pending application for CMA of sparsentan in IgA nephropathy. Given the high unmet need of FSGS patients, with no medicines currently approved for the condition, and the challenges associated with studying FSGS due to its heterogeneity and other attributes, we intend to pursue discussions with these regulators based on the totality of the data from the study and our clinical experience with sparsentan to date. While there is some regulatory precedent to evaluate drug candidates for potential approval despite the primary endpoint of a pivotal trial not being achieved, we are unable to predict if the regulatory agencies will be amenable to a submission based on the totality of data after not reaching statistical significance on the pre-specified primary endpoint.

If sparsentan receives marketing authorization in any of the licensed territories, CSL Vifor will be responsible for all commercialization activities in such licensed territories. We remain responsible for the clinical development of sparsentan and will retain all rights to sparsentan in the United States and rest of world outside of the licensed territories, provided that CSL Vifor has a right of negotiation to expand the licensed territories into Canada, China, Brazil and/or Mexico.

Pegtibatinase (TVT-058)

Pegtibatinase (TVT-058) is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Classical HCU is a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can lead to vision, skeletal, circulatory and central nervous system complications. It is estimated that there are at least 3,500 people living with HCU in the United States with similar numbers in Europe. Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. Pegtibatinase is currently being evaluated in the Phase 1/2 COMPOSE Study, a double blind, randomized, placebo-controlled dose escalation study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU.

In December 2021, we announced positive topline results from the Phase 1/2 COMPOSE Study. Pegtibatinase demonstrated dose-dependent reductions in total homocysteine (tHcy) during the 12 weeks of treatment, and in the highest dose cohort to date evaluating 1.5mg/kg of pegtibatinase twice weekly (BIW), treatment with pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy) through 12 weeks of treatment, including a 55.1% mean relative reduction in tHcy from baseline as well as maintenance of tHcy below a clinically meaningful threshold of 100 μmol. Additionally, in a dose-dependent manner in the study to date, methionine levels were substantially reduced and cystathionine levels were substantially elevated following treatment with pegtibatinase, suggesting that pegtibatinase acts in a manner similar to the native CBS enzyme. To date in the study, pegtibatinase has been generally well-tolerated, with no discontinuations due to treatment-related adverse events.

Following positive results from the first five cohorts of the ongoing Phase 1/2 COMPOSE Study, we evaluated pegtibatinase in the sixth cohort of the COMPOSE Study to further inform our pivotal development program to ultimately support potential approval of pegtibatinase for the treatment of HCU. The additional cohort was initiated to inform and refine formulation work for future development and commercial purposes and to further evaluate the dose response curve for pegtibatinase. In May 2023, we announced positive topline results from the sixth cohort of the Phase 1/2 COMPOSE Study. In this cohort, five patients were randomized in a blinded fashion to receive 2.5 mg/kg of lyophilized pegtibatinase or placebo twice weekly (BIW), with four patients assigned to the treatment group. In this highest dose cohort to date, treatment with pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy), with a 67.1% mean relative reduction in tHcy from baseline, as well as maintenance of mean tHcy below the clinically meaningful threshold of 100 μmol, over weeks 6 to 12. To date in the study, pegtibatinase has been generally well-tolerated, with no discontinuations due to treatment-related adverse events.

We are preparing for the potential initiation of a pivotal Phase 3 clinical trial of pegtibatinase in patients with HCU by the end of 2023, subject to communications and feedback from the FDA and associated program assessments.

We acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited.

Chenodal

Chenodal (chenodeoxycholic acid or CDCA) is a naturally occurring bile acid that is approved for the treatment of people with radiolucent stones in the gallbladder. While indicated for radiolucent stones in the gallbladder, Chenodal has been recognized as the standard of care for cerebrotendinous xanthomatosis (CTX) for more than three decades, although it is not currently labeled for this indication. CTX is a rare, progressive and underdiagnosed bile acid synthesis disorder affecting many parts of the body. In September 2022, we were granted Fast Track designation by the FDA for the investigation of Chenodal for CTX. In January 2020, we randomized the first patients in our Phase 3 RESTORE Study to evaluate the effects of Chenodal in adult and pediatric patients with CTX, and the study enrollment remains open. The pivotal study is intended to support an NDA submission for marketing authorization of Chenodal for CTX in the United States. During 2023, we expect to complete the ongoing Phase 3 RESTORE Study in CTX. Pending supportive data, we anticipate being in position to subsequently submit an NDA for a CTX indication.

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Preclinical Programs:

We are a participant in a Cooperative Research and Development Agreement (CRADA), which forms a multi-stakeholder approach to pool resources with leading experts, and incorporate the patient perspective early in the therapeutic identification and development process. We are partnering with the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) and a leading patient advocacy organization, Alagille Syndrome Alliance, aimed at the identification of potential small molecule therapeutics for Alagille syndrome (ALGS). There are no treatment options currently approved for ALGS.

We are party to a collaboration agreement with PharmaKrysto Limited and their early-stage cystinuria discovery program, whereby we are responsible for funding all research and development expenses for the pre-clinical activities associated with the cystinuria program.

Other Commercial Products:

Thiola and Thiola EC (tiopronin)

Thiola and Thiola EC are approved by the FDA for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. Due to the larger stone size, cystine stones may be more difficult to pass, often requiring surgical procedures to remove. More than 80 percent of people with cystinuria develop their first stone by the age of 20. More than 25 percent will develop cystine stones by the age of 10. Recurring stone formation can cause loss of kidney function in addition to substantial pain and loss of productivity associated with renal colic and stone passage. While a portion of people living with the disease are able to manage symptoms through diet and fluid intake, the prevalence of cystinuria in the US is estimated to be 10,000 to 12,000, indicating that there may be as many as 4,000 to 5,000 affected individuals with cystinuria in the US that would be candidates for Thiola or Thiola EC.

In June 2019 we announced that the FDA approved 100 mg and 300 mg tablets of Thiola EC, an enteric-coated formulation of Thiola, to be used for the treatment of cystinuria. Thiola EC offers the potential for administration with or without food, and the ability to reduce the number of tablets necessary to manage cystinuria. Thiola EC became available to patients in July 2019.

In May 2021, a generic option for the 100 mg version of the original formulation of Thiola (tiopronin tablets) became available and in June 2022, a second option for the 100 mg version of the original formulation of Thiola (tiopronin tablets) was approved. These generic versions of the original formulation of Thiola have impacted sales, and these or additional generic versions of either formulation could have a material adverse impact on sales. In February 2023, a generic version of Thiola EC (100 mg and 300 mg) was approved by the FDA.

Cholbam (cholic acid)

The FDA approved Cholbam (cholic acid capsules) in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of Cholbam has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy.

Chenodal (chenodiol)

Chenodal is a synthetic oral form of chenodeoxycholic acid ("CDCA"), a naturally occurring primary bile acid synthesized from cholesterol in the liver. The FDA approved Chenodal for the treatment of people with radiolucent stones in the gallbladder. In 2010, Chenodal was granted orphan drug designation for the treatment of cerebrotendinous xanthomatosis ("CTX"), a rare autosomal recessive lipid storage disease. We acquired Chenodal in March 2014.

While Chenodal is not labeled for CTX, it received a medical necessity determination in the US by the FDA and has been used as the standard of care for more than three decades. We are working to obtain FDA approval of Chenodal for the treatment of CTX and initiated a Phase 3 clinical trial for this indication in January 2020. The prevalence of CTX is estimated in the literature to be as high as 1 in 70,000 in the overall population. Pathogenesis of CTX involves deficiency of the enzyme 27-hydroxylase (encoded by the gene CYP27A1), a rate-limiting enzyme in the synthesis of primary bile acids, including CDCA, from cholesterol. The disruption of primary bile acid synthesis in CTX leads to toxic accumulation of cholesterol and cholestanol in most tissues. Patients may present with intractable diarrhea, premature cataracts, tendon xanthomas, atherosclerosis, and cardiovascular disease in childhood and adolescence. Neurological manifestations of the disease, including dementia and cognitive and cerebellar deficiencies, emerge during late adolescence and adulthood. The types, combinations and severity of symptoms can be different from person to person, and making diagnosis challenging and often delayed. Oral administration of CDCA has been shown to normalize primary bile acid synthesis in patients with CTX.

Sale of Assets Related to Cholbam (cholic acid) and Chenodal (chenodiol)

In July 2023, we executed an Asset Purchase Agreement (the “Purchase Agreement”) with Mirum Pharmaceuticals, Inc., a Delaware corporation (“Mirum”). Subject to the terms and conditions of the Purchase Agreement, at the closing (the “Closing”) of the transactions contemplated by the Purchase Agreement (the “Asset Purchase”), Mirum will purchase from us substantially all of the assets that are primarily related to our business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Products”) for an aggregate purchase price, subject to certain adjustments pursuant to the terms of the Purchase Agreement, of up to $445.0 million in cash, with $210.0 million due at the Closing and up to $235.0 million after the Closing, upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products (the “Milestone Events”).

We and Mirum are permitted under certain circumstances to terminate the Agreement, including in the event that the Closing has not occurred by October 16, 2023, which period will be automatically extended to April 16, 2024 if any condition remaining to be satisfied relates to regulatory approval.

We are in the process of evaluating the accounting impact, including any potential gain from the sale of assets, on the consolidated financial statements. We expect the Closing to occur in the third quarter of 2023.

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Results of Operations

Results of operations for the three and six months ended June 30, 2023 compared to the three and six months ended June 30, 2022

Revenue

The following table provides information regarding net product sales (in thousands):

	Three Months Ended June 30,						Six Months Ended June 30,
	2023		2022		Change		2023		2022		Change
Bile acid products	$	27,501	$	25,534	$	1,967	$	53,606	$	50,609	$	2,997
Tiopronin products	26,050		25,416		634		47,224		46,784		440
FILSPARI	3,461		—		3,461		6,465		—		6,465
Total net product revenues	57,012		50,950		6,062		107,295		97,393		9,902
License and collaboration revenue	2,685		3,217		(532)		9,395		5,261		4,134
Total revenue	$	59,697	$	54,167	$	5,530	$	116,690	$	102,654	$	14,036

Net product sales

The increase in total net product revenues for the three and six months ended June 30, 2023 compared to the three and six months ended June 30, 2022 was primarily due the launch of FILSPARI in February 2023 and continued organic growth of our legacy products.

License and collaboration revenue

The increase in license and collaboration revenue for the six months ended June 30, 2023 compared to the six months ended June 30, 2022 was primarily due to a $3.3 million sale of active pharmaceutical ingredients to CSL Vifor in March 2023, at cost plus a margin, in preparation for a potential European approval and subsequent commercial launch.

Operating Expenses

The following table provides information regarding operating expenses (in thousands):

	Three Months Ended June 30,						Six Months Ended June 30,
	2023		2022		Change		2023		2022		Change
Cost of goods sold - product sales	$	1,983	$	2,051	$	(68)	$	4,071	$	4,189	$	(118)
Cost of goods sold - license and collaboration	7		—		7		3,044		—		3,044
Total cost of goods sold	1,990		2,051		(61)		7,115		4,189		2,926
Research and development	69,411		59,681		9,730		129,324		116,292		13,032
Selling, general and administrative	74,037		52,979		21,058		146,282		99,767		46,515
Change in fair value of contingent consideration	1,840		4,907		(3,067)		8,596		13,987		(5,391)
	$	147,278	$	119,618	$	27,660	$	291,317	$	234,235	$	57,082

Cost of goods sold

Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping and handling costs, and provisions for excess and obsolete inventory.

For the three and six months ended June 30, 2023 compared to the three and six months ended June 30, 2022, our cost of goods sold - product sales decreased by $0.1 million and $0.1 million, respectively, due to higher inventory reserves recognized in the comparative period, offset by increased sales and higher FDA fees recognized in 2023.

For the six months ended June 30, 2023 as compared to the six months ended June 30, 2022, our cost of goods sold - license and collaboration increased by $3.0 million due primarily to the sale of active pharmaceutical ingredients to CSL Vifor.

Sales of FILSPARI primarily consisted of zero-cost inventories, which favorably impacted gross margin for related sales. Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), we recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredients to support the commercial launch of FILSPARI. Had these costs been included, total cost of goods sold would have increased by less than $0.1 million and by approximately $0.1 million for the three and six months ended June 30, 2023, respectively. We expect to continue to benefit from the sale of previously expensed inventories through at least 2024.

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Research and development expenses

Research and development costs include expenses related to sparsentan, pegtibatinase (TVT-058) and our other pipeline programs. We expense all research and development costs as they are incurred. Our research and development costs are comprised of salaries and bonuses, benefits, non-cash share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, and develop drug materials and delivery methods, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. We charge direct internal and external program costs to the respective development programs. We also incur indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.

We currently have one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support studies. As such, clinical trial expenses will vary depending on the all the factors set forth above and may fluctuate significantly from quarter to quarter.

We routinely engage vendors and service providers for scientific research, clinical trial, regulatory compliance, manufacturing and other consulting services. We also make grants to research and non-profit organizations to conduct research which may lead to new intellectual properties that we may subsequently license under separately negotiated license agreements. Such grants may be funded in lump sums or installments.

The following table provides information regarding research and development expenses (in thousands):

	Three Months Ended June 30,						Six Months Ended June 30,
	2023		2022		Change		2023		2022		Change
External service provider costs:
Sparsentan	$	22,036	$	25,354	$	(3,318)	$	43,186	$	44,726	$	(1,540)
Pegtibatinase	18,260		9,197		9,063		28,671		18,275		10,396
General and other product candidates	7,511		5,909		1,602		12,800		14,845		(2,045)
Total external service provider costs	47,807		40,460		7,347		84,657		77,846		6,811
Internal personnel costs	21,604		19,221		2,383		44,667		38,446		6,221
Total research and development	$	69,411	$	59,681	$	9,730	$	129,324	$	116,292	$	13,032

For the three and six months ended June 30, 2023 as compared to the three and six months ended June 30, 2022, our research and development expenses increased by $9.7 million and $13.0 million, respectively. Internal personnel costs to support all programs increased by $2.4 million and $6.2 million, respectively, reflecting increased headcount along with rising labor costs driven in part by inflation. External service provider costs increased by $7.3 million and $6.8 million, respectively, which was largely driven by increases in investment as we prepare for the potential Phase 3 development program for pegtibatinase, slightly offset by a decrease in costs associated with the development of sparsentan as its Phase 3 programs advance towards completion.

Selling, general and administrative expenses

Selling, general and administrative expenses consist of salaries and bonuses, benefits, non-cash share-based compensation, legal and other professional fees, rent, depreciation and amortization, travel, insurance, business development, sales and marketing programs, and other operating expenses.

For the three and six months ended June 30, 2023 as compared to the three and six months ended June 30, 2022, our selling, general and administrative expenses increased by $21.1 million and $46.5 million, respectively, due to increased headcount as a result of operational growth, including rising labor costs driven in part by inflation, as well as commercial preparations and activity related to the U.S. launch of FILSPARI, such as the full staffing of the dedicated sales force. Increases include combined employee compensation and stock compensation costs of $3.8 million and $16.6 million, respectively, increases in commercial support expenses of $9.1 million and $13.8 million, respectively, and increases in various professional services expenses of $2.1 million and $6.0 million, respectively. A change in the estimated useful life of the Thiola intangible asset and the accelerated amortization recognized in connection with the change contributed to a $3.7 million increase in expense for the three and six months ended June 30, 2023 as compared to the three and six months ended June 30, 2022. The three and six months ended June 30, 2023 also includes amortization of intangible assets derived from the milestone payment made in connection with the February 2023 accelerated approval of FILSPARI and royalties owed on net sales of FILSPARI.

Change in the valuation of contingent consideration

For the three and six months ended June 30, 2023 as compared to the three and six months ended June 30, 2022, the change in fair value of contingent consideration is due to the passage of time, updated revenue projections and changes in market driven discount rates, including fluctuations in treasury rates and credit spreads.

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Other Income/Expenses

The following table provides information regarding other income (expenses), net (in thousands):

	Three Months Ended June 30,						Six Months Ended June 30,
	2023		2022		Change		2023		2022		Change
Interest income	$	5,128	$	782	$	4,346	$	8,774	$	1,060	$	7,714
Interest expense	(2,911)		(2,972)		61		(5,851)		(5,487)		(364)
Other (expense) income, net	(201)		662		(863)		(114)		688		(802)
Loss on extinguishment of debt	—		—		—		—		(7,578)		7,578
	$	2,016	$	(1,528)	$	3,544	$	2,809	$	(11,317)	$	14,126

The $3.5 million change in our total other income (expense) for the three months ended June 30, 2023 as compared to the three months ended June 30, 2022, is primarily attributable to a $4.3 million increase in interest income in 2023, which was due to increases in short-term interest rates on our interest-bearing security investments. The $14.1 million change in our total other income (expense) for the six months ended June 30, 2023 as compared to the six months ended June 30, 2022, is primarily attributable to a $7.7 million increase in interest income in 2023, which was due to increases in short-term interest rates on our interest-bearing security investments, and the $7.6 million loss on extinguishment of debt that was recognized in 2022 in connection with the partial repurchase of the Convertible Senior Notes due 2025.

Income Tax Benefit (Provision)

At June 30, 2023, we had $11.5 million of unrecognized tax benefits. We did not recognize any interest or penalties related to unrecognized tax benefits during the three and six months ended June 30, 2023.

Liquidity and Capital Resources

We have financed our operations through a combination of borrowings, sales of our equity securities, and revenues generated from our commercialized products, including FILSPARI, Chenodal, Cholbam, Thiola and Thiola EC, along with proceeds from license and collaboration agreements. We have experienced significant growth in recent years in the number of our employees and the scope of our operations. We have expanded our sales and marketing, compliance and legal functions in addition to expansion of all functions to support a commercial organization, including by adding additional members to our sales force in connection with the recent commercial launch of FILSPARI in the United States for IgAN. We anticipate that our expenses will continue to increase as we expand our sales and marketing infrastructure to commercialize FILSPARI and our other current approved products and any other new products for which we may obtain regulatory approval, advance the research and development of sparsentan for the treatment of IgAN and FSGS, along with the research and development for additional product candidates including pegtibatinase (TVT-058), and expand operational, financial, and management information systems and personnel, including personnel to support product development efforts and our obligations as a public company.

We believe that our available cash and short-term investments as of the date of this filing, together with anticipated cash generated from operations, will be sufficient to fund our anticipated level of operations beyond the next 12 months. We expect that our operating results will vary from quarter-to-quarter and year-to-year depending upon various factors including revenues, selling, general and administrative expenses, and research and development expenses, particularly with respect to our clinical and preclinical development activities. Our ability to fund our operations in subsequent years will depend upon certain factors which are beyond our control and may require us to obtain additional debt or equity capital or refinance all or a portion of our debt, including the 2025 Notes and 2029 Notes, on or before maturity. Though we generate revenues from product sales arrangements, we may incur significant operating losses over the next several years. Our ability to achieve profitable operations in the future will depend in large part upon completing development of products in our pipeline, obtaining regulatory approvals for these products and bringing these products to market, along with potential in-licensing of additional products approved by the FDA and selling and manufacturing these products.

We had the following balances at June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Cash and Cash Equivalents	$	70,874	$	61,688
Marketable debt securities	420,463		388,557
Accumulated Deficit	(1,186,184)		(1,014,223)
Stockholders' Equity	116,485		42,851
Net Working Capital*	$	406,344	$	344,274
Net Working Capital Ratio**	3.99		3.42
* Current assets less current liabilities. **Current assets divided by current liabilities.

As of June 30, 2023, we had cash and cash equivalents of $70.9 million and available-for-sale marketable debt securities of $420.5 million. Substantial sources of funds since the start of 2023, as summarized further below, include net proceeds of $215.8 million from an underwritten public offering of our common stock and pre-funded warrants to purchase our common stock.

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Over the next 12 months, our expected financial obligations include, but are not limited to, funding our operations, operating lease payments, interest payments on our outstanding debt, royalties on sales of our existing commercialized products, research and development expenses pertaining to clinical and preclinical development activities across our pipeline, expenses associated with the launch of FILSPARI, including purchase commitments for manufactured product. Sources of cash over this period include net revenues from sales of our products, the sale or maturity of investments in our portfolio of marketable debt securities, and certain earned and potential milestone payments associated with sparsentan in connection with our license and collaboration arrangement with CSL Vifor. Subject to the Closing of the Asset Purchase pursuant to the Asset Purchase Agreement entered into with Mirum in July 2023, we will receive $210.0 million due at the Closing, with the Closing expected to occur in the third quarter of 2023.

Beyond the next 12 months and over the foreseeable future, our known commitments and potential financial obligations will likely include ongoing operations funding, operating lease payments, interest payments on our outstanding debt, royalties on sales of our existing commercialized products, research and development expenses pertaining to clinical and preclinical development activities across our pipeline, milestone and royalty payments associated with FILSPARI, pegtibatinase (TVT-058), and other developmental programs based upon the achievement of certain agreement-specific criteria, along with sales-based royalties and the repayment of principal on the outstanding 2025 Notes and 2029 Notes upon their respective maturities. Potential sources of cash over this time horizon may include net revenues from sales of our existing products and, if commercialized, our pipeline products, licensing revenue, the sale or maturity of marketable debt securities in our investment portfolio, the refinancing of all or a portion of our debt, including the 2025 Notes and 2029 Notes, on or before maturity, or the issuance of additional debt or equity. In addition, depending on prevailing market conditions, our liquidity requirements, contractual restrictions, and other factors, we may also from time to time seek to retire or purchase our outstanding debt, including the 2025 Notes or 2029 Notes, through cash purchases and/or exchanges for equity securities, in open market purchases, privately negotiated transactions or otherwise. In light of global and macroeconomic conditions, including rising interest rates, liquidity concerns at, and failures of, banks and other financial institutions, and volatility in the capital markets, we may not be able to successfully conduct financing or refinancing activity on favorable terms or at all.

Collaboration and License Proceeds

License and Collaboration Agreement with CSL Vifor

On September 15, 2021, we entered into a License Agreement with CSL Vifor, pursuant to which we granted an exclusive license to CSL Vifor for the commercialization of sparsentan in the Licensed Territories. Under the terms of the License Agreement, we received an upfront payment of $55.0 million in September 2021 and will be eligible for up to $135.0 million in aggregate regulatory and market access related milestone payments and up to $655.0 million in aggregate sales-based milestone payments for a total potential value of up to $845.0 million. We are also entitled to receive tiered double-digit royalties of up to 40 percent of annual net sales of sparsentan in the Licensed Territories.

See Note 4 to our unaudited Consolidated Financial Statements for further discussion.

Equity Offerings

2023 Underwritten Public Offering of Common Stock

In February 2023, we sold an aggregate of approximately 9.7 million shares of our common stock and pre-funded warrants to purchase 1.25 million shares of our common stock in an underwritten public offering, at a price of $21.00 per share of common stock and $20.9999 per prefunded warrant. The pre-funded warrants are exercisable immediately, subject to certain beneficial ownership limitations which can be modified by the respective holders with at least 61 days' notice, and are exercisable for one share of our common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. The net proceeds to us from the offering, after deducting the underwriting discounts and offering expenses, were approximately $215.8 million.

At-the-Market Equity Offering

In February 2020, we entered into an Open Market Sale Agreement ("ATM Agreement") with Jefferies LLC, as agent (“Jefferies”), pursuant to which we may offer and sell, from time to time through Jefferies, shares of our common stock having an aggregate offering price of up to $100.0 million. Of the $100.0 million originally authorized for sale under the ATM Agreement, approximately $28.6 million were sold under our prior registration statement on Form S-3 (Registration No. 333-227182). An additional $51.9 million were sold under our effective registration statement on Form S-3 (Registration Statement No. 333-259311), which included $20.1 million in the year ended December 31, 2022. We did not sell any shares under the ATM Agreement during the six months ended June 30, 2023. As of June 30, 2023, an aggregate of $19.5 million remained eligible for sale under the ATM Agreement.

Operating Leases

Future Minimum Rental Commitments

As of June 30, 2023, we have future minimum rental commitments totaling $35.1 million arising from our operating leases. These commitments represent the aggregate base rent through August 2028.

See Note 7 to our unaudited Consolidated Financial Statements for further discussion.

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Purchase Commitments

Manufactured Product

Certain of the Company's contractual arrangements with contract manufacturing organizations ("CMOs") require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's financial statements. As of June 30, 2023, we have commitments to purchase $22.1 million in active pharmaceutical ingredients to be delivered in 2023.

Royalties and Contingent Cash Payments

Ligand License Agreement

In 2012, we entered into an agreement with Ligand Pharmaceuticals, Inc. ("Ligand") for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). As consideration for the license, we are required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million. Through June 30, 2023, we have paid $41.4 million for contractual milestone payments under the Ligand License Agreement, which includes a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) in March 2023 that was triggered upon the accelerated approval of FILSPARI in February 2023. Upon commercialization of sparsentan or any products containing related compounds, we will be obligated to pay to Ligand an escalating annual royalty between 15% and 17% of net sales of all such products, with payments due quarterly. We began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the three and six months ended June 30, 2023, we capitalized $0.5 million and $1.0 million, respectively, to intangible assets for royalties owed on net sales of FILSPARI.

The license agreement will continue until neither party has any further payment obligations under the agreement and is expected to continue for up to 20 years from the effective date. Ligand may terminate the license agreement due to (i) our insolvency, (ii) our material uncured breach of the agreement, (iii) our failure to use commercially reasonable efforts to develop and commercialize sparsentan as described above or (iv) certain other conditions. We may terminate the license agreement due to a material uncured breach of the agreement by Ligand.

See Note 9 to our unaudited Consolidated Financial Statements for further discussion.

Thiola License Agreement

In 2014, we entered into a license agreement with Mission Pharmacal ("Mission"), pursuant to which we obtained an exclusive, royalty-bearing license to market, sell and commercialize Thiola (tiopronin) in the United States and Canada, and a non-exclusive license to use know-how relating to Thiola to the extent necessary to market Thiola. Under the terms of the license agreement, as subsequently amended, which runs through May 2029, we are obligated to pay to Mission guaranteed minimum royalties equaling the greater of $2.1 million or 20% of our Thiola net sales generated outside of the United States during each calendar year.

Acquisition of Orphan Technologies Limited

In November 2020, we completed the acquisition of Orphan Technologies Limited (“Orphan”), including Orphan’s rare metabolic disorder drug pegtibatinase (TVT-058). We acquired Orphan by purchasing all of the outstanding shares. Under the Stock Purchase Agreement ("the Agreement"), we agreed to make contingent cash payments up to an aggregate of $427.0 million based on the achievement of certain development, regulatory and commercialization events as set forth in the Agreement, as well as additional tiered mid-single digit royalty payments based upon future net sales of any pegtibatinase products in the US and Europe, subject to certain reductions as set forth in the Agreement, and a contingent payment in the event a pediatric rare disease voucher for any pegtibatinase product is granted.

Stock Purchase and Collaboration Agreement with PharmaKrysto

On March 8, 2022, we entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company related to PharmaKrysto's early-stage cystinuria discovery program, and concurrently therewith entered into a Stock Purchase Agreement with PharmaKrysto (together, the "Agreements"). Pursuant to the terms of the Agreements, we acquired 5% of the outstanding common shares of PharmaKrysto and are required to purchase an additional 5% of the outstanding common shares for $1.0 million upon the occurrence of a specified pre-clinical milestone. The Agreements also require us to fund all research and development expenses for the pre-clinical activities associated with the cystinuria program, which are estimated to be approximately $5.0 million. In addition, the Agreements grant us an option to purchase the remaining outstanding shares of PharmaKrysto for $5.0 million upon the occurrence of a subsequent pre-clinical milestone prior to expiration of the option on March 8, 2025. If we elect to exercise the option, we would be required to perform commercially reasonable clinical diligence obligations. In addition, we would be required to make cash milestone payments totaling up to an aggregate $16.0 million upon the achievement of certain development and regulatory milestones, plus tiered royalty payments of less than 4% on future net sales of a product, if approved. We have the right to terminate the Agreements and return the shares for a nominal price at any time upon 60 days’ notice, subject to survival of contingent obligations, if any.

Borrowings

Convertible Senior Notes Due 2029

On March 11, 2022, we completed a registered underwritten public offering of $316.3 million aggregate principal amount of 2.25% Convertible Senior Notes due 2029 (“2029 Notes”). We issued the 2029 Notes under an indenture, dated as of September 10, 2018, as supplemented by the second supplemental indenture, dated as of March 11, 2022 (collectively, the “2029 Indenture”). The 2029 Notes will mature on March 1, 2029, unless earlier repurchased, redeemed, or converted. The 2029 Notes are senior unsecured obligations of ours and bear interest at an annual rate of 2.25%, payable semi-annually in arrears on March 1 and September 1 of each year, beginning on September 1, 2022. The 2029 Notes do not contain any financial or operating covenants or any restrictions on the payment of dividends, the issuance of other indebtedness or the issuance or repurchase of securities by us.

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Convertible Senior Notes Due 2025

On September 10, 2018, we completed a registered underwritten public offering of $276.0 million aggregate principal amount of 2.50% Convertible Senior Notes due 2025 ("2025 Notes"), and entered into a base indenture and supplemental indenture agreement (collectively, the "2025 Indenture") with respect to the 2025 Notes. The 2025 Notes will mature on September 15, 2025, unless earlier repurchased, redeemed, or converted. The 2025 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.50%, payable semi-annually in arrears on March 15 and September 15 of each year, beginning on March 15, 2019. On March 11, 2022, coinciding with the issuance of the 2029 Notes, we completed our repurchase of $207.1 million of aggregate principal amount of 2025 Notes for cash. After giving effect to the repurchase, the total remaining principal amount outstanding under the 2025 Notes as of June 30, 2023 was $68.9 million. The 2025 Notes do not contain any financial or operating covenants or any restrictions on the payment of dividends, the issuance of other indebtedness or the issuance or repurchase of securities by us.

Funding Requirements

We believe that our available cash and short-term investments as of the date of this filing will be sufficient to fund our anticipated level of operations beyond the next 12 months. We expect to use cash flows from operations and outside financings to meet our current and future financial obligations, including funding our operations, debt service and capital expenditures. Our ability to make these payments depends on our future performance, which will be affected by financial, business, economic, regulatory and other factors, many of which we cannot control. Factors that may affect financing requirements include, but are not limited to:

•the timing, progress, cost and results of our clinical trials, preclinical studies and other discovery and research and development activities;

•the timing and outcome of, and costs involved in, seeking and obtaining marketing approvals for our products, and in maintaining quality systems standards for our products;

•the timing of, and costs involved in, commercial activities, including product marketing, sales and distribution;

•our ability to successfully commercialize FILSPARI for IgAN, to obtain full regulatory approval for, and successfully commercialize, FILSPARI for the treatment of IgAN, and to obtain regulatory approval for, and successfully commercialize, sparsentan for FSGS and our other or future product candidates;

•increases or decreases in revenue from our marketed products, including decreases in revenue resulting from generic entrants or health epidemics or pandemics;

•debt service obligations on the 2025 Notes and 2029 Notes;

•the number and development requirements of other product candidates that we pursue;

•our ability to manufacture sufficient quantities of our products to meet expected demand;

•the costs of preparing, filing, prosecuting, maintaining and enforcing any patent claims and other intellectual property rights, litigation costs and the results of litigation;

•our ability to enter into collaboration, licensing or distribution arrangements and the terms and timing of these arrangements;

•the potential need to expand our business, resulting in additional payroll and other overhead expenses;

•the potential in-licensing of other products or technologies;

•the emergence of competing technologies or other adverse market or technological developments; and

•the impacts of inflation and resulting cost increases.

Future capital requirements will also depend on the extent to which we acquire or invest in additional complementary businesses, products and technologies.

Cash Flows

Cash Flows from Operating Activities

Cash used in operating activities for the six months ended June 30, 2023 was $150.4 million compared to cash used of $92.1 million for the six months ended June 30, 2022. The increase in cash used was primarily attributable to increased research and development and sales, general and administrative expenses.

Cash Flows from Investing Activities

Cash used in investing activities for the six months ended June 30, 2023 was $60.6 million compared to cash used of $5.9 million for the six months ended June 30, 2022. The change was due to the increase in net purchases of marketable debt securities along with a $23.0 million milestone payment to Ligand (and BMS) in March 2023 that was triggered upon the accelerated approval of FILSPARI in February 2023.

Cash Flows from Financing Activities

Cash provided by financing activities for the six months ended June 30, 2023 was $219.3 million compared to cash provided of $114.7 million for the six months ended June 30, 2022. The increase in cash provided was due to the March 2023 issuance of common stock and pre-funded warrants through an underwritten public offering that provided $215.8 million, offset by net proceeds of $95.4 million from the March 2022 issuance of the 2029 Notes and repurchase of the 2025 Notes.

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Other Matters

Adoption of New Accounting Standards

See Note 2 to our unaudited Consolidated Financial Statements in this report for a discussion of adoption of new accounting standards.

Recently Issued Accounting Pronouncements

See Note 2 to our unaudited Consolidated Financial Statements in this report for a discussion of recently issued accounting pronouncements.

Critical Accounting Estimates

Our consolidated financial statements are prepared in accordance with accounting principles generally accepted in the United States of America. The preparation of our consolidated financial statements and related disclosures requires us to make estimates and assumptions that affect the reported amount of assets, liabilities, revenue, costs and expenses, and related disclosures. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions and conditions. See our Annual Report on Form 10-K for the fiscal year ended December 31, 2022 for information about critical accounting estimates as well as a description of our other significant accounting policies.

Item 3.  Quantitative and Qualitative Disclosures about Market Risk

Our primary exposure to market risk is related to changes in interest rates. As of June 30, 2023, we had cash equivalents and marketable debt securities of approximately $491.3 million, consisting of money market funds, U.S. government agency debt, corporate debt and commercial paper. This exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates, particularly because our investments are in short-term debt securities. Our marketable debt securities are subject to interest rate risk and will fall in value if market interest rates increase. Due to the short-term duration of our investment portfolio and the low risk profile of our investments, a change in interest rates of 100 basis points would have approximately a $2.9 million impact on our investments.

The marketable debt securities held in our investment portfolio may subject us to credit risk, though our investment policy limits interest-bearing security investments to certain types of instruments issued by institutions with primarily investment grade credit ratings and places restrictions on maturities and concentration by asset class and issuer. Given these policy restrictions and our emphasis on preserving capital and liquidity while enhancing overall returns, we have not experienced material credit-related losses with our securities holdings.

We are also exposed to market risk related to changes in foreign currency exchange rates. From time to time, we enter into contracts with vendors that are located outside of the United States, which contracts are denominated in foreign currencies. We are subject to fluctuations in foreign currency rated in connection with these agreements. We do not currently hedge our foreign currency exchange rate risk.

Inflation generally affects us by increasing our salaries and fees paid to third-party contract service providers. Recent inflationary pressures have primarily impacted our operations through increased labor costs. While we continue to monitor the effects of macroeconomic factors, inflationary pressures have not affected our current outlook or business objectives.

Item 4.  Controls and Procedures

Evaluation of Disclosure Controls and Procedures

We maintain disclosure controls and procedures that are designed to ensure that information required to be disclosed in our reports required by the Exchange Act of 1934, as amended, is recorded, processed, summarized and reported within the timelines specified in the SEC’s rules and forms, and that such information is accumulated and communicated to our management, including our Chief Executive Officer and Chief Financial Officer, as appropriate, to allow timely decisions regarding required disclosure. In designing and evaluating the disclosure controls and procedures, management recognized that any controls and procedures, no matter how well designed and operated, can only provide reasonable assurance of achieving the desired control objectives, and in reaching a reasonable level of assurance, management necessarily was required to apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures.

As required by SEC Rule 13a-15(b), we carried out an evaluation, under the supervision and with the participation of our management, including our Chief Executive Officer and Chief Financial Officer, of the effectiveness of the design and operation of our disclosure controls and procedures as of the end of the quarter covered by this report. Based on the foregoing, our Chief Executive Officer and Chief Financial Officer concluded that our disclosure controls and procedures were not effective at the reasonable assurance level due to the material weakness in internal control over financial reporting described below.

Changes in Internal Control over Financial Reporting

An evaluation was also performed under the supervision and with the participation of our management, including our Chief Executive Officer and Chief Financial Officer, of any change to our internal control over financial reporting that occurred during the quarter covered by this report and that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.

As disclosed in Part II, Item 9A of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, our Chief Executive Officer and Chief Financial Officer concluded that our internal control over financial reporting was not effective as of December 31, 2022 due to the following material weakness: We did not design effective controls and procedures to evaluate the accounting for a certain pre-launch inventory contract affecting the timing of

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recognition of research and development expense. Such material weakness did not result in a restatement of previously issued annual consolidated financial statements or interim consolidated financial statements. The material weakness has not been fully remediated as of the date of this report.

Our evaluation did not identify any changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Securities Exchange Act of 1934) that occurred during the quarter ended June 30, 2023, that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting. Our remediation plan is underway to address the material weakness mentioned above. The material weakness will not be considered remediated until the applicable controls operate for a sufficient period of time and management has concluded, through testing, that these controls are operating effectively.

PART II - OTHER INFORMATION 

Item 1.  Legal Proceedings

The information required by this Item is incorporated herein by reference to the Notes to the Unaudited Consolidated Financial Statements--Note 13 Commitments and Contingencies: Legal Proceedings in Part I, Item 1, of this Quarterly Report on Form 10-Q.

Item 1A. Risk Factors

Our business, as well as an investment in our common stock, is highly speculative in nature and involves a high degree of risk. Our securities should be purchased only by persons who can afford to lose their entire investment. Carefully consider the risks and uncertainties described below together with all of the other information included herein, including the financial statements and related notes, before deciding to invest in our common stock. If any of the following risks actually occur, they could adversely affect our business, prospects, financial condition and results of operations. In such event(s), the market price of our common stock could decline and result in a loss of part or all of your investment. Accordingly, prospective investors should carefully consider, along with other matters referred to herein, the following risk factors in evaluating our business before purchasing any shares of our common stock. We have marked with an asterisk (*) those risk factors that were not included as separate risk factors in, or reflect changes to the similarly titled risk factors included in, Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2022 as filed with the Securities and Exchange Commission (SEC) on February 23, 2023.

Risks Related to the Commercialization of Our Products

Our future prospects are highly dependent upon our ability to successfully develop and execute commercialization strategies for our products, including FILSPARI, and to attain market acceptance among physicians, patients and healthcare payers.*

Our ability to generate significant product revenues and to achieve commercial success in the near-term will depend almost entirely on our ability to successfully commercialize our products in the United States, including FILSPARI (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, which was approved by the FDA in February 2023 under the FDA’s accelerated approval regulations.

As a newly-approved product for a rare disease that had no previously-approved non-immunosuppressive treatment, the successful launch and commercialization of FILSPARI is subject to many risks. There are numerous examples of unsuccessful product launches and failures to meet high expectations of market potential, including by pharmaceutical companies with more experience and resources than we have. While we have established our commercial team and U.S. sales force, we will need to continue to train and further develop the team in order to successfully coordinate the ongoing launch and commercialization of FILSPARI in the United States. There are many factors that could cause the launch and commercialization of FILSPARI to be unsuccessful, including a number of factors that are outside our control. Because no non-immunosuppressive product has previously been approved by the FDA for the treatment of IgAN, it is difficult to estimate FILSPARI’s market potential or the time it will take to increase patient and physician awareness of FILSPARI and change current treatment paradigms. The commercial success of FILSPARI depends on the extent to which patients and physicians accept and adopt FILSPARI for IgAN patients. For example, if the addressable patient population suffering from primary IgAN is smaller than we estimate, if it proves difficult to educate physicians as to the availability and potential benefits of FILSPARI, or if physicians are unwilling to prescribe or patients are unwilling to take FILSPARI, the commercial potential of FILSPARI will be limited. We also do not know how physicians, patients and payers will respond to the pricing of FILSPARI. Physicians may not prescribe FILSPARI and patients may be unwilling to use FILSPARI if coverage is not provided or reimbursement is inadequate to cover a significant portion of the cost. Thus, significant uncertainty remains regarding the commercial potential of FILSPARI. If the launch or commercialization of FILSPARI is unsuccessful or perceived as disappointing, the price of our common stock could decline significantly and long-term success of the product and our company could be harmed.

In order to operate our business and increase adoption and sales of our products, we need to continue to develop our commercial organization, including maintaining and growing a highly experienced and skilled workforce with qualified sales representatives.*

In order to successfully commercialize our products in the United States, we have built a specialized sales force. In order to successfully commercialize any approved products, we must continue to build our sales, marketing, distribution, managerial and other non-technical capabilities. Factors that may hinder our ability to successfully market and commercially distribute our products include:

•inability of sales personnel to obtain access to or educate adequate numbers of physicians on the benefits and safety of prescribing our products;

•inability to recruit, retain and effectively manage adequate numbers of effective sales personnel;

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•lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies that have more extensive product lines; and

•unforeseen delays, costs and expenses associated with maintaining our sales organization.

If we are unable to maintain an effective sales force for our products, including the recently expanded sales force for FILSPARI or any other potential future approved products, we may not be able to generate sufficient product revenue in the United States. In addition, until the commencement of our commercial launch in February 2023, no one in our sales force had promoted FILSPARI or any other medicine for the treatment of IgAN patients. We are required to expend significant time and resources to train our sales force to be credible and persuasive in convincing physicians to prescribe and pharmacists to dispense our products. In addition, we must continually train our sales force to ensure that a consistent and appropriate message about our products is being delivered to our potential customers. We currently have limited resources compared to some of our competitors, and the continued development of our own commercial organization to market our products and any additional products we may develop or acquire will be expensive and time-consuming. We also cannot be certain that we will be able to continue to successfully develop this capability.

Similarly, if CSL Vifor does not effectively engage or maintain its sales force for sparsentan if approved in the Licensed Territories, our ability to recognize milestone payments and royalties from the Licensed Territories will be adversely affected.

We will need to continue to expend significant time and resources to train our sales forces to be credible and persuasive in discussing our products with the specialists treating the patients indicated under the product’s label. In addition, if we are unable to effectively train our sales force and equip them with effective marketing materials our ability to successfully commercialize our products could be diminished, which would have a material adverse effect on our business, results of operations and financial condition.

We are dependent on CSL Vifor for the successful commercialization of sparsentan in certain key territories outside of the United States, if approved, and CSL Vifor's commercialization efforts may fail to meet our expectations. We may not be able to establish additional collaborations or other arrangements for sparsentan in other territories, which may adversely impact our ability to generate product revenue in additional jurisdictions.

Pursuant to the terms of the License Agreement, we granted an exclusive license to CSL Vifor for the commercialization of sparsentan in the Licensed Territories, which consist of Europe, Australia and New Zealand. Consequently, the commercial success of sparsentan in the Licensed Territories will depend in significant part on the efforts of CSL Vifor, over which we will have limited control. In August 2022, Vifor Pharma Group was acquired by CSL Limited, parent company to CSL Behring and is now operating under the brand CSL Vifor. We do not currently know what effect, if any, this acquisition will ultimately have on our relationship with CSL Vifor. While our agreement with CSL Vifor remains in place following the acquisition, there is no guarantee that our collaboration with CSL Vifor will not be affected, adversely or otherwise, by the change in ownership. Moreover, in connection with the acquisition of CSL Vifor and related restructuring, substantially less resources could be devoted to the commercialization of sparsentan in the Licensed Territories, or such efforts could be discontinued entirely. If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sell sparsentan in territories outside of the United States, if approved, our ability to generate product revenue outside of the United States and the Licensed Territories may be limited.

The commercial success of our products depends on them being considered to be effective drugs with advantages over other therapies.

The commercial success of our products FILSPARI, Thiola, Chenodal and Cholbam, and, if approved, sparsentan for the treatment of FSGS, depends on them being considered to be effective drugs with advantages over other therapies. A number of factors, as discussed in greater detail below, may adversely impact the degree of acceptance of these products, including their efficacy, safety, price and benefits over competing therapies, as well as the coverage and reimbursement policies of third-party payers, such as government and private insurance plans.

If unexpected adverse events are reported in connection with the use of any of these products, physician and patient acceptance of the product could deteriorate and the commercial success of such product could be adversely affected. We are required to report to the FDA events associated with our products relating to death or injury. Adverse events could result in additional regulatory controls, such as a requirement for costly post-approval clinical studies or revisions to our approved labeling which could limit the indications or patient population for a product or could even lead to the withdrawal of a product from the market.

We face substantial generic and other competition, and our operating results will suffer if we fail to compete effectively.*

Under the Hatch-Waxman Amendments of the Federal Food, Drug, and Cosmetic Act, a pharmaceutical manufacturer may file an ANDA seeking approval of a generic copy of an approved innovator product or an NDA under Section 505(b)(2) that relies on the FDA’s prior findings of safety and effectiveness in approving the innovator product. A Section 505(b)(2) NDA may be for a new or improved version of the original innovator product. Certain of our products, including Thiola, Chenodal and Cholbam, are subject to immediate competition from compounded and generic entrants, as the ANDA and NDA for these drug products have no remaining or current patent or non-patent exclusivity. In April 2021, a generic option for the 100 mg version of the original formulation of Thiola (tiopronin tablets) was approved by the FDA and an additional generic option of the original formulation of Thiola (tiopronin tablets) was approved in June 2022. Additional generic versions of Thiola may be approved in the future. During the year ended December 31, 2022, we experienced a decrease in total net product revenues compared to the year ended December 31, 2021, which was due in part to competition from generic tiopronin tablets (100 mg version of the original formulation). Our future net product revenues from Thiola may be materially impacted by competition from existing or additional generic versions of Thiola. In February 2023, a generic version of Thiola EC (100 mg and 300 mg) was approved by the FDA. Our future net product revenues from Thiola EC may also be materially impacted by competition from existing or additional generic versions of Thiola or Thiola EC.

In addition, there have been a number of recent regulatory and legislative initiatives designed to encourage generic competition for pharmaceutical products, including expedited review procedures for generic manufacturers and incentives designed to spur generic competition of branded drugs. In particular, the FDA and the U.S. Federal Trade Commission (“FTC”) have been focused on brand companies’ denial of drug supply to potential generic competitors for

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testing. In December 2019, the CREATES Act was enacted, which provides a legislatively defined private right of action under which generic companies can bring suit against companies who refuse access to product for the bioequivalence testing needed to support approval of a generic product.

We have completed our response to a civil investigative demand from the FTC related to the marketing, sale, distribution and pricing of our products, including Thiola. While the investigation remains open, at this time the FTC has not indicated that it has additional questions for us, and has not initiated any claim or proceeding against us relating to these matters.

We cannot currently predict the specific outcome or impact on our business of such regulatory and legislative initiatives, litigation or investigation. However, it is our policy, which is in compliance with the CREATES Act, to evaluate requests for samples of our branded products, and to provide samples in response to bona fide requests from qualified third parties, including generic manufacturers, subject to specified conditions. We have provided and are in the process of providing samples to certain generic manufacturers.

If additional generic versions of Thiola, any generic versions of Thiola EC, any generic versions of FILSPARI following the expiration of patent or regulatory exclusivity for the product, or generic versions of Cholbam or Chenodal or any of our other current or future products are approved, sales of that product likely would be negatively impacted, which could have a material adverse impact on our sales and profitability. In addition, the defense of litigation and response to investigation requests could result in substantial costs, reputational impact, and the diversion of management attention and resources.

The Drug Price Competition and Patent Term Restoration Act (commonly referred to as the "Hatch-Waxman Act") requires an ANDA applicant seeking FDA approval of its proposed generic product prior to the expiration of an Orange Book-listed patent (as defined below) to certify that the applicant believes that the patent is invalid, unenforceable and/or will not be infringed by the manufacture, use or sale of the drug for which the application has been submitted (a paragraph IV certification) and notify the NDA and patent holder of such certification (a paragraph IV notice). Upon receipt of a paragraph IV notice, the Hatch-Waxman Act allows the patent holder, with proper basis, to bring an action for patent infringement against the ANDA filer, asking that the proposed generic product not be approved until after the patent expires. For ANDAs that are filed (“received”) after the listing of the patent in the Orange Book, if the patent holder commences a lawsuit within 45 days from receipt of the paragraph IV notice, the Hatch-Waxman Act provides a 30-month stay during which time the FDA cannot finally approve the generic’s application. If the litigation is resolved in favor of the ANDA applicant during the 30-month stay period, the stay is lifted and the FDA may finally approve the ANDA if it is otherwise ready for approval. For ANDAs that are filed (“received”) before the listing of the patent in the Orange Book, the 30-month stay provision of the Hatch-Waxman Act does not apply. It also may be possible, depending on the approved label, for an ANDA applicant to elect to submit a section viii statement certifying that its proposed ANDA label does not contain (or carves out) any language regarding the patented method-of-use rather than certify to a listed method-of-use patent.

In October 2022, our licensor, Mission Pharmacal Company, was granted a patent covering the treatment of cystinuria by administering Thiola EC with food (US Patent No. 11,458,104, “the ‘104 patent”) and has listed this patent in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (the "Orange Book"). Following Mission’s listing of the '104 patent in the Orange Book, and as of December 31, 2022, Mission has received three paragraph IV notice letters from three generic manufacturers notifying Mission that each has filed an ANDA seeking approval of a proposed generic version of Thiola EC (tiopronin) 100 mg and 300 mg oral tablets before expiration of the '104 patent and asserting that the '104 patent is not infringed and/or is invalid, with each such ANDA having been filed prior to the granting and listing of the ‘104 patent. The ANDA filed by Par Pharmaceutical Inc. (which had previously indicated to Mission that it may challenge the ‘104 patent through the Patent Trial and Appeal Board procedures at the United States Patent and Trademark Office), for a generic version of Thiola EC (100 mg and 300 mg) was approved by the FDA on February 24, 2023. Under our agreement with Mission, we have the right to enforce the '104 patent. We are evaluating these paragraph IV notices, and will evaluate any other paragraph IV notices received, on a case by case basis in order to determine whether to initiate patent litigation against any such generic manufacturer. There is no guarantee that the ‘104 patent will withstand any challenge at the Patent and Trademark Office or in litigation, if initiated. Patent litigation is expensive and time consuming, requires significant resources, may absorb significant time of our management and has an unpredictable outcome. If we determine not to pursue patent litigation or the patent is not upheld in litigation or administrative review or if a generic competitor is found not to infringe this patent, the resulting generic competition will likely negatively affect our business, financial condition and results of operations.

Healthcare reform initiatives, unfavorable pricing regulations, and changes in reimbursement practices of third-party payers or patients' access to insurance coverage could affect the pricing of and demand for our products.*

The business and financial condition of healthcare-related businesses will continue to be affected by efforts of governments and third-party payers to contain or reduce the cost of healthcare through various means. In the United States and some foreign jurisdictions, there have been a number of legislative and regulatory changes and proposed changes regarding the healthcare system that could prevent or delay marketing approval for our current product candidates or any future product candidate that we develop, restrict or regulate post-approval activities and affect our ability to profitably sell sparsentan, pegtibatinase (TVT-058), or any other product candidate for which we obtain marketing approval.

Our products are sold to patients whose healthcare costs are met by third-party payers, such as government programs, private insurance plans and managed-care programs. These third-party payers are increasingly attempting to contain healthcare costs by limiting both coverage and the level of reimbursement for medical products and services. Levels of reimbursement, if any, may be decreased in the future, and future healthcare reform legislation, regulations or changes to reimbursement policies of third-party payers may otherwise adversely affect the demand for and price levels of our products, which could have a material adverse effect on our sales and profitability.

Economic, social, and congressional pressure may result in individuals and government entities increasingly seeking to achieve cost savings through mechanisms that limit coverage or payment for our products. For example, state Medicaid programs are increasingly requesting manufacturers to pay supplemental rebates and requiring prior authorization for use of drugs. Managed care organizations continue to seek price discounts and, in some cases, to impose restrictions on the coverage of particular drugs. Government efforts to reduce Medicaid expenses may lead to increased use of managed care organizations by Medicaid programs. This may result in managed care organizations influencing prescription decisions for a larger segment of the population and a corresponding constraint on prices and reimbursement for our products.

In addition, patients’ access to employer sponsored insurance coverage may be negatively impacted by economic factors that result in increased rates of unemployment. To the extent patients taking our approved therapies become unemployed and experience a reduction to, or increased costs associated with,

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their insurance coverage, demand for our products could decline, which could have a material adverse effect on our sales and profitability, either as a result of decreased sales of our products and/or increased provision by us of free product to uninsured or commercially insured patients. The extent and duration of this potential impact on our business is currently unknown.

We are dependent on third parties to manufacture and distribute our products.

We have no manufacturing capabilities and rely on third-party manufacturers who are sole source suppliers for manufacturing of FILSPARI, Thiola, Chenodal and Cholbam. The facilities used by our third-party manufacturers must be approved by the FDA. Our dependence on third parties for the manufacture of our products may harm our profit margin on the sale of products and our ability to deliver products on a timely and competitive basis. Because we are ultimately responsible for ensuring that our API and finished products are manufactured in accordance with cGMP regulations and similar regulatory requirements outside the United States, it is critical that we maintain effective management practices and oversight with respect to our third-party manufacturers, including routine auditing. If our third-party manufacturers are unable to manufacture to specifications or in compliance with applicable regulatory requirements, our ability to commercialize our products will be adversely impacted and could affect our ability to gain market acceptance for our products and negatively impact our revenues.

We currently have no in-house distribution channels for FILSPARI, Thiola, Chenodal or Cholbam and we are dependent on third-party distributors to distribute such products. The outsourcing of our distribution function is complex, and we may experience difficulties that could reduce, delay or stop shipments of such products. If we encounter such distribution problems, and we are unable to quickly enter into a similar agreement with another distributor on substantially similar terms, distribution of FILSPARI, Thiola, Chenodal and/or Cholbam could become disrupted, resulting in lost revenues, provider dissatisfaction, and/or patient dissatisfaction.

Governments outside the United States tend to impose strict price controls and reimbursement approval policies, which may adversely affect our prospects for generating revenue.

In some countries, particularly EU countries and EFTA member states, the pricing of prescription pharmaceuticals is subject to governmental control. In these countries, pricing negotiations with governmental authorities can take considerable time (6 to 12 months or longer) after the receipt of marketing approval for a product. To obtain reimbursement or pricing approval in some countries, we may be required to conduct a clinical trial that compares the cost effectiveness of our product candidate to other available therapies. In addition, certain governmental authorities may conduct reviews of reimbursement previously provided and assert for various reasons that amounts need to be repaid. For example, in October 2021 our distributor/exploitant in France for our previously marketed product Kolbam informed us that they had received a notice that the price previously paid for Kolbam during its period on the market in France had been recalculated by the agency responsible for pharmaceutical pricing in France, with such notice asserting amounts owed for repayment. While we cannot currently estimate the likelihood that any of such asserted amount will ultimately need to be repaid following the currently ongoing review process and any applicable appeal procedures, we may ultimately determine the need to repay all or a portion of the amounts being asserted. From 2015 through 2020, the period during which we had sales of Kolbam in France, our aggregate revenues from sales of Kolbam in France attributable to all purchasers/payers were approximately $8 million. If reimbursement of our products is unavailable or limited in scope or amount, or if pricing is set at unsatisfactory levels or subject to re-assessment and recoupment procedures, our prospects for generating revenue outside of the United States, if any, could be adversely affected and our business may suffer.

We may not be able to rely on orphan drug exclusivity for our products.*

Regulatory authorities in some jurisdictions, including the United States and Europe, may designate drugs for relatively small patient populations as orphan drugs, providing eligibility for orphan drug exclusivity upon regulatory approval if certain jurisdictional-specific conditions are met. For example, FILSPARI has been granted orphan drug designation for the treatment of IgAN and has been awarded seven years of orphan drug exclusivity to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urinary protein-to-creatinine ration ("UPCR") ≥1.5 gram/gram, and Cholbam was granted orphan drug designation in the United States and upon FDA approval of the marketing application in March 2015 was awarded seven years of orphan drug exclusivity, which expired in March 2022. Generally, if a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, that product is entitled to a period of marketing exclusivity, which precludes the applicable regulatory authority from approving another marketing application for the same drug for the same indication for that time period. The applicable period is seven years in the United States and ten years in Europe or, in the case of orphan drugs for which a pediatric investigation plan has been completed, 12 years. Even though we have been awarded orphan drug designation in the United States and Europe for sparsentan for the treatment of IgAN and FSGS and for pegtibatinase for the treatment of HCU, we may not be able to maintain it in Europe and the orphan drug designation may not result in orphan drug exclusivity in the United States for FSGS or Europe if approved. For example, if a competitive product that contains the same active moiety and treats the same disease as our product is shown to be clinically superior to our product, any orphan drug exclusivity we have obtained will not block the approval of such competitive product and we may effectively lose orphan drug exclusivity. Similarly, if a competitive product that contains the same active moiety and treats the same disease as our product candidate is approved for orphan drug exclusivity before our product candidate, we may not be able to obtain approval for our product candidate until the expiration of the competitive product’s orphan drug exclusivity unless our product candidate is shown to be clinically superior to the competitive product.

Risks Related to the Development of our Product Candidates

Our clinical trials are expensive and time-consuming and may fail to demonstrate the safety and efficacy of our product candidates.*

Before obtaining regulatory approval for the sale of any of our current or future product candidates, we must subject these product candidates to extensive nonclinical and clinical testing to demonstrate their safety and efficacy for humans. Clinical trials are expensive, time-consuming and may take years to complete.

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We may experience numerous unforeseen events during, or as a result of, preclinical or nonclinical testing and the clinical trial process that could delay or prevent our ability to obtain, or impact our willingness to pursue, regulatory approval or commercialize our product candidates, including:

•our preclinical or nonclinical tests or clinical trials may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional nonclinical testing or clinical trials or we may abandon projects that we expect to be clinically promising in light of cost or strategic considerations;

•regulators may require us to conduct studies of the long-term effects associated with the use of our product candidates;

•regulators or institutional review boards may not authorize us to commence a clinical trial or conduct a clinical trial at a prospective trial site;

•the FDA or any non-United States regulatory authority may impose conditions on us regarding the scope or design of our clinical trials or may require us to resubmit our clinical trial protocols to institutional review boards for re-inspection due to changes in the regulatory environment;

•the number of patients required for our clinical trials may be larger than we anticipate or participants may drop out of our clinical trials at a higher rate than we anticipate;

•our third-party contractors or clinical investigators may fail to comply with regulatory requirements or fail to meet their contractual obligations to us in a timely manner;

•we might have to suspend or terminate one or more of our clinical trials if we, regulators or institutional review boards determine that the participants are being exposed to unacceptable health risks;

•regulators or institutional review boards may require that we hold, suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements;

•the cost of our clinical trials or the anticipated commercialization costs may be greater than we anticipate;

•the supply or quality of our product candidates or other materials necessary to conduct our clinical trials may be insufficient or inadequate, or more expensive than we originally anticipated, or we may not be able to reach agreements on acceptable terms with prospective suppliers or clinical research organizations; and

•the effects of our product candidates may not be the desired effects or may include undesirable side effects or the product candidates may have other unexpected characteristics.

We will only obtain regulatory approval to commercialize a product candidate if we can demonstrate to the satisfaction of the FDA, and in the case of foreign commercialization, to the applicable foreign regulatory authorities, in well-designed and conducted clinical trials, that our product candidates are safe and effective and otherwise meet the appropriate standards required for approval for a particular indication.

Conducting clinical trials effectively in pursuit of regulatory approval requires significant resources, and the costs of conducting clinical trials varies depending on a number of factors, including the dosage of the study drug, trial size and duration. These costs may prove greater than we originally anticipated, which may result in us choosing to abandon or forgo clinical trials that we deem clinically promising as we actively strategize over time with respect to the allocation of our resources.

Our product development costs will also increase if we experience delays in testing or approvals. We do not know whether any nonclinical tests or clinical trials will be initiated as planned, will need to be restructured or will be completed on schedule, if at all. Significant nonclinical or clinical trial delays also could shorten the patent protection period during which we may have the exclusive right to commercialize our product candidates. In addition, such delays could allow our competitors to bring products to market before we do and impair our ability to commercialize our products or product candidates.

If we are required to conduct additional clinical trials or other testing of our product candidates beyond those that we currently contemplate, if we are unable to successfully complete our clinical trials or other testing, if the results of these trials or tests are not positive or are only modestly positive or if there are safety concerns, we may:

•be delayed in obtaining, or may not be able to obtain, marketing approval for one or more of our product candidates;

•obtain approval for indications that are not as broad as intended or entirely different than those indications for which we sought approval; and

•have the product removed from the market after obtaining marketing approval.

For example, in our pivotal Phase 3 DUPLEX Study of sparsentan in FSGS, although we achieved the pre-specified interim FSGS partial remission of proteinuria endpoint after 36 weeks of treatment, the study did not achieve the primary efficacy eGFR slope endpoint over 108 weeks of treatment. While we intend to engage with the FDA to explore a potential path forward for a supplemental New Drug Application ("sNDA") in the U.S. and work with our collaborator CSL Vifor to engage with the European Medicines Agency ("EMA") to also explore a potential regulatory path forward in FSGS in Europe based on the DUPLEX data, there is no guarantee that we will be able to establish a pathway to a potential submission of sparsentan for FSGS based on the results from the DUPLEX Study, that the FDA and/or EMA will support an application for sparsentan in FSGS, or that sparsentan will be approved for FSGS. Moreover, while there is some regulatory precedent to evaluate drug candidates for potential approval despite the primary endpoint of a pivotal trial not being achieved, we are unable to predict if the regulatory agencies will be amenable to a submission based on the totality of data after not reaching statistical significance on the pre-specified primary endpoint.

In August 2021, we announced that our ongoing pivotal Phase 3 PROTECT Study of sparsentan in IgAN achieved its pre-specified primary efficacy endpoint after 36 weeks of treatment. Pursuant to the PROTECT Study protocol, patients are to continue in a blinded manner to assess the treatment effect on eGFR slope over two years in the confirmatory endpoint analyses of the study. Given that interim results from the study have been publicly announced, it is possible that we may see a higher than anticipated attrition rate in the study. To the extent that an insufficient number of patients choose to remain in the study for the full two years, it could jeopardize our ability to complete the study and submit for traditional regulatory approval for sparsentan in IgAN.

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We may not be able to initiate or continue clinical trials in the rare diseases on which we are focused if we are unable to locate a sufficient number of eligible patients willing and able to participate in the clinical trials required by the FDA or foreign regulatory agencies. In addition, as other companies and researchers may be concurrently developing therapies for the same or similar indications that we are focused on, we could face competition for a limited number of patients, investigators and clinical trial sites willing to participate in clinical trials. Our inability to enroll and maintain a sufficient number of patients for any of our current or future clinical trials would result in significant delays or may require us to abandon one or more clinical trials altogether.

In January 2020, we randomized the first patients in a Phase 3 clinical trial to evaluate the effects of Chenodal in adult and pediatric patients with CTX. The pivotal study, known as the RESTORE study, is intended to support an NDA submission for marketing authorization of Chenodal for CTX in the United States. While Chenodal has been used as the standard of care for CTX for over three decades, it is not labeled for CTX and as such we cannot market this drug candidate for the treatment of CTX unless and until it receives FDA approval for this indication. If we experience delays in obtaining approval or if we fail to obtain approval of Chenodal for the treatment of CTX, our business, financial condition and results of operations could be adversely affected.

Success in nonclinical testing and early clinical trials does not ensure that later clinical trials will be successful.*

Success in nonclinical testing and early clinical trials does not ensure that later clinical trials will be successful. For example, although we observed favorable responses with the physician-initiated treatment of fosmetpantotenate in PKAN patients outside the United States, the Phase 3 FORT Study evaluating the safety and efficacy of fosmetpantotenate compared to placebo in patients with PKAN did not meet its primary endpoint, did not demonstrate a difference between treatment groups, and did not meet its secondary endpoint. Similarly, while we saw trends in favor of sparsentan in the two year confirmatory endpoint analysis in the DUPLEX Study in FSGS, the positive eGFR results from the open-label portion of the DUET study of sparsentan in FSGS were not replicated in the Phase 3 clinical trial with statistical significance. Similarly, the positive nonclinical data we have seen from pegtibatinase (TVT-058) being tested in a mouse model of homocystinuria and the positive topline results we reported in December 2021 and May 2023 from the ongoing Phase 1/2 clinical trial of pegtibatinase (TVT-058) may not be replicated in future studies. We cannot assure that any current or future clinical trials of sparsentan or pegtibatinase (TVT-058) will ultimately be successful.

Before obtaining regulatory approval to conduct clinical trials of our product candidates, we must conduct extensive nonclinical tests to demonstrate the safety of our product candidates in animals. Nonclinical testing is expensive, difficult to design and implement, and can take many years to complete. In addition, during the clinical development process, additional nonclinical toxicology studies are routinely conducted concurrently with the clinical development of a product candidate. If any of our product candidates show unexpected findings in concurrent toxicology studies, we could experience potentially significant delays in, or be required to abandon, development of that product candidate. A failure of one or more of our nonclinical studies can occur at any stage of testing.

Communications and/or feedback from regulatory authorities related to our current or planned future clinical trials does not guarantee any particular outcome from or timeline for regulatory review, and expedited regulatory review pathways may not actually lead to faster development or approval.*

Communications and/or feedback from regulatory authorities, including the FDA or EMA, related to our current or future clinical trials does not guarantee any particular outcome from or timeline for regulatory review for such clinical trials, and expedited regulatory review pathways may not actually lead to faster development or approval.

In 2018 we initiated the Phase 3 DUPLEX Study and the Phase 3 PROTECT Study. We initiated the DUPLEX Study and the PROTECT Study under the Subpart H pathway for potential accelerated approval in the United States, and potential conditional marketing authorization in Europe, in both jurisdictions based on change in proteinuria. Recognition of change in proteinuria as a surrogate endpoint in kidney disease is a relatively new regulatory development, and, as the field continues to evolve, new learnings may impact regulatory viewpoints.

In February 2023, the FDA granted accelerated approval to FILSPARI (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a UPCR ≥1.5 gram/gram. As a post marketing requirement, we must complete the PROTECT Study and fulfill other post-marketing requirements. The EMA has accepted for review the conditional marketing authorization application of sparsentan for the treatment of IgAN in Europe, and a review decision is expected in the second half of 2023.

In May 2023, we announced that the DUPLEX Study did not achieve its two-year primary endpoint with statistical significance over the active control irbesartan. Although we are encouraged by the topline results for the secondary endpoints on proteinuria and topline exploratory endpoints, including renal outcomes, which trended favorably for sparsentan, and we are continuing to analyze the data to further evaluate the potential for sparsentan as a treatment for FSGS and plan to meet with the regulators to explore a potential path to a submission for sparsentan in FSGS, there is no guarantee that we or our collaborator CSL Vifor will be able to establish a pathway to a potential submission of sparsentan for FSGS based on the results from the DUPLEX Study, that the FDA and/or EMA will support an application for sparsentan in FSGS, or that sparsentan will be approved for FSGS. While there is some regulatory precedent to evaluate drug candidates for potential approval despite the primary endpoint of a pivotal trial not being achieved, we are unable to predict if the regulatory agencies will be amenable to a submission based on the totality of data after not reaching statistical significance on the pre-specified primary endpoint.

We expect that the EMA’s determination as to whether the sufficiency of the data from the PROTECT Study supports a conditional marketing authorization in Europe will be made during the application review process based on the totality of the data, including eGFR data available for review from the relevant studies. There can be no assurance that the EMA will deem our achievement of any interim endpoint or measurement in the PROTECT Study to be sufficient to grant conditional marketing authorization for sparsentan for the treatment of IgAN, or that our timelines will not be delayed notwithstanding the availability of an expedited regulatory review pathway.

Although the EMA has accepted our conditional marketing authorization application for review, there can be no assurance that the study will proceed as planned and there can be no guarantee that the EMA will grant conditional marketing authorization in the EU for sparsentan for IgAN. Furthermore, even though sparsentan was granted accelerated approval for IgAN, there can be no assurance that the data from the ongoing PROTECT Study will support traditional approval of sparsentan as a treatment for IgAN.

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Although the FDA has granted Fast Track and Breakthrough Therapy designations to pegtibatinase (TVT-058) for the treatment of HCU, there is no guarantee that we will be able to reach agreement with the FDA on the final study design for a proposed Phase 3 trial of pegtibatinase (TVT-058), that we will ultimately proceed with the proposed Phase 3 trial, or that pegtibatinase (TVT-058) will be approved for HCU in the future, on an expedited timeline or at all. We intend to use a surrogate endpoint, change in total homocysteine (tHcy) level, as a biomarker to demonstrate efficacy in the proposed Phase 3 pivotal trial and to support a future marketing application for TVT-058 for the treatment of HCU. While we have commenced discussions with the FDA regarding the use of this biomarker to support a future approval under the traditional or accelerated approval pathway, we will need to have further interactions with the FDA as part of the routine regulatory advancement of the program and will need to confirm with the FDA the use of total homocysteine as the pivotal endpoint for the study, align with the FDA on the details of the study, as well as on other elements of the program such as matters related to chemistry, manufacturing and controls. Prior to initiating the proposed Phase 3 trial, we will need to evaluate the clinical/regulatory pathway and the drug supply and product profile against the backdrop of the commercial landscape and opportunity to confirm strategic alignment within the program. Due to the inherent complexities of drug development, there is no guarantee that these factors will align in support of the proposed Phase 3 program.

Similarly, while we were granted Fast Track designation by the FDA for the investigation of Chenodal for CTX in September 2022, the Phase 3 RESTORE study may not ultimately support an NDA submission and the Fast Track designation may not ultimately lead to FDA approval of Chenodal for CTX on an expedited timeline or at all.

Obtaining access to an expedited program (such as Fast Track and Breakthrough Therapy designations) may not in fact lead to faster development timelines or achieve faster review or approval than conventional FDA procedures. We may experience delays in approval timelines attributable to, among other things, acquiring sufficient supply of our product to conduct clinical trials, identifying and resolving issues relating to chemistry, manufacturing and controls, or conducting additional nonclinical or clinical studies. In addition, the FDA may withdraw access to an expedited program if it believes the access or designation is no longer supported by the data from our program.

Interim, topline and preliminary data from our clinical trials that we announce or publish may change materially as more patient data become available and audit and verification procedures are complete.

From time to time, we may publicly disclose preliminary or topline or interim data from our clinical studies, which is based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a more comprehensive review of the data related to the particular study. We also make assumptions, estimations, calculations and conclusions as part of our analyses of data, and we may not have received or had the opportunity to fully and carefully evaluate all data. As a result, the topline results that we report may differ from future results of the same studies, or different conclusions or considerations may qualify such results, once additional data have been received and fully evaluated. Topline data also remain subject to audit and verification procedures that may result in the final data being materially different from the preliminary data we previously published. As a result, topline data should be viewed with caution. From time to time, we may also disclose interim data from our clinical trials. Interim data from clinical trials that we may complete are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment and dosing continues and more patient data become available. Adverse differences between preliminary or interim data and final or confirmatory data could significantly harm our business prospects.

Further, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the value of the particular program, the approvability or commercialization of the particular product candidate or product and our company in general. In addition, the information we choose to publicly disclose regarding a particular study or clinical trial is based on what is typically extensive information, and you or others may not agree with what we determine is the material or otherwise appropriate information to include in our disclosure, and any information we determine not to disclose may ultimately be deemed significant with respect to future decisions, conclusions, views, activities or otherwise regarding a particular drug, drug candidate or our business. If the topline data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, our product candidates may be harmed, which could harm our business, operating results, prospects or financial condition.

We and/or a collaborative partner are or will be subject to ongoing regulatory obligations and continued regulatory review for our approved products and any product candidates that receive regulatory approval.

The FDA’s accelerated approval of FILSPARI is limited to adults with primary IgAN who are at risk of rapid disease progression, generally a UPCR ≥1.5 gram/gram, and is subject to our completion of the PROTECT Study. Any future regulatory approvals that sparsentan or any of our other product candidates receives may be subject to significant limitations on the approved indicated uses for which the product may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance to monitor the safety and efficacy of the product candidate.

In addition, our products, including FILSPARI, and any of our product candidates that are approved by the FDA or a comparable foreign regulatory authority, are or will be subject to extensive and ongoing regulatory requirements, including for the manufacturing, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion, import, export, recordkeeping, conduct of potential post-marketing studies and post-market submission requirements. These requirements include submissions of safety and other post-marketing information and reports, registration, as well as continued compliance with current good manufacturing practices and good clinical practices, for any clinical trials that we conduct post-approval. Later discovery of previously unknown problems with a product, including adverse events of unanticipated severity or frequency, undesirable side effects caused by the product, problems encountered by our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, either before or after product approval, may result in, among other things:

•restrictions on the marketing, manufacturing, or distribution of the product;

•requirements to include additional warnings on the label;

•requirements to create or enhance a medication guide outlining the risks to patients;

•withdrawal of the product from the market;

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•voluntary or mandatory product recalls;

•requirements to change the way the product is administered or for us to conduct additional clinical trials;

•fines, warning or untitled letters or holds on clinical trials;

•refusal by the FDA to approve pending applications or supplements to approved applications filed by us or our strategic partners, or suspension or revocation of product license approvals;

•product seizure or detention, or refusal to permit the import or export of products;

•injunctions or the imposition of civil or criminal penalties; and

•harm to our reputation.

For example, we have certain post-marketing requirements and commitments associated with FILSPARI and Cholbam. Further, we face risks relating to those post-marketing obligations, as well as the commercial acceptance of FILSPARI and Cholbam. If the regulatory approval for FILSPARI, Thiola, Chenodal and/or Cholbam are withdrawn for any reason, it would have a material adverse impact on our sales and profitability.

The third-party clinical investigators and contract research organizations that we rely upon to conduct our clinical trials may not be diligent, careful or timely, and may make mistakes, in the conduct of our trials.

We depend on third-party clinical investigators and contract research organizations (“CROs”) to conduct our clinical trials under agreements with us. The CROs play a significant role in the conduct of our clinical trials. Failure of the CROs to meet their obligations could adversely affect clinical development of our product candidates. The third-party clinical investigators are not our employees and we cannot control the timing or amount of resources they devote to our studies. If their performance is substandard, it could delay or prevent approval of our FDA applications. Moreover, these third-party investigators and CROs may also have relationships with other commercial entities, some of which may compete with us. If third-party investigators and CROs allocate their resources to assist our competitors at our expense, it could harm our competitive position. In response to COVID-19, we have engaged providers of home health and remote monitoring services to assist with the ongoing conduct of our clinical trials in an effort to mitigate disruption caused by COVID-19 related issues. The introduction of new third parties into our ongoing clinical trials increases the risks associated with our dependence on third parties, including the risk that substandard performance by, or competing interests of, such third parties could have a negative impact on our clinical trials.

Risks Related to our Products and Product Candidates

Our products may not achieve or maintain expected levels of market acceptance or commercial success.

The success of our products is dependent upon achieving and maintaining market acceptance. Commercializing products is time consuming, expensive and unpredictable. There can be no assurance that we will be able to, either by ourselves or in collaboration with our partners or through our licensees, successfully commercialize new products or current products or gain market acceptance for such products. New product candidates that appear promising in development may fail to reach the market or may have only limited or no commercial success.

Further, the discovery of significant problems with a product similar to one of our products that implicate (or are perceived to implicate) an entire class of products could have an adverse effect on sales of the affected products. Accordingly, new data about our products, or products similar to our products, could negatively impact demand for our products due to real or perceived side effects or uncertainty regarding efficacy and, in some cases, could result in product withdrawal.

Our current products, including FILSPARI, and any product candidates that receive marketing approval, that we or a collaboration partner bring to the market may not gain market acceptance by physicians, patients, third-party payers, and others in the medical community. If these products do not achieve an adequate level of acceptance, we may not generate significant product revenue and we may not become profitable. The degree of market acceptance of our current products and product candidates, if approved for commercial sale, will depend on a number of factors, including:

•the prevalence and severity of any side effects, including any limitations or warnings contained in a product’s approved labeling;

•the efficacy and potential advantages over alternative treatments;

•the pricing of our product candidates;

•relative convenience and ease of administration;

•the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;

•the strength of marketing and distribution support and timing of market introduction of competitive products;

•publicity concerning our products or competing products and treatments; and

•sufficient third-party insurance coverage and reimbursement.

As part of the NDA review process for sparsentan for IgAN, the FDA required us to include a REMS and a boxed warning on the label regarding mandatory birth control for patients of child-bearing potential regarding risk of embryo-fetal toxicity, as has been required for other approved endothelin antagonists, and a REMS and boxed warning on the label for liver monitoring regarding potential risk of hepatotoxicity, as has been required for certain other approved endothelin antagonists. As part of the liver monitoring REMS, monthly monitoring of each patient is required for the first year the patient is on treatment, and quarterly thereafter. While we have taken efforts to streamline the REMS with the cadence of typical patient monitoring and have implemented convenience-focused features within the REMS program, the existence of monthly liver monitoring has the potential to be viewed as an impediment to prescribing FILSPARI. Also, while we intend to utilize our continued clinical trial experience with FILSPARI and post-marketing data gathering commitment to potentially

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support lifting of the liver monitoring REMS in the future following sufficient experience with FILSPARI and if supported by the data, there is no guarantee that the data will support this endeavor, or even if we believe it does, that the FDA will agree with it.

Even if a potential or current product displays a favorable efficacy and safety profile in nonclinical and clinical trials, market acceptance of the product will not be known until after it is launched. The efforts by us or any applicable collaboration partner to educate patients, the medical community, and third-party payers on the benefits of our products may require significant resources and may never be successful. Such efforts to educate the marketplace may require more resources than are required by the conventional marketing technologies employed by our competitors.

The market opportunities for our products and product candidates may be smaller than we believe they are.

Certain of the diseases that our current and future product candidates are being developed to address, such as IgAN, FSGS and HCU, are relatively rare. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our product candidates, may not be accurate.

Currently, most reported estimates of the prevalence of IgAN, FSGS and HCU are based on studies of small subsets of the population of specific geographic areas, which are then extrapolated to estimate the prevalence of the diseases in the broader world population. As new studies are performed the estimated prevalence of these diseases may change. There can be no assurance that the prevalence of IgAN, FSGS or HCU in the study populations accurately reflect the prevalence of these diseases in the broader world population.

The FDA-approved label of FILSPARI is currently limited to adult patients with IgAN at risk of rapid disease progression, generally a UPCR ≥1.5 gram/gram. Based on our interactions with the FDA, we believe that the FDA has imposed the rapid disease progression limitation on the FILSPARI label because of the accelerated approval pathway under which the product has been approved, and that there should be an opportunity to further expand the label to cover a broader population of IgAN patients following the conclusion of the confirmatory portion of the PROTECT Study, pending supportive data. However, there can be no guarantee that this will be the case.

If our estimates of the prevalence of IgAN, FSGS or HCU or of the number of patients who may benefit from treatment with sparsentan or pegtibatinase prove to be incorrect or if regulatory approval is conditioned on label restrictions that limit the approved patient population, the market opportunities for our product candidates may be smaller than we believe they are, our prospects for generating revenue may be adversely affected and our business may suffer.

Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval or commercialization.

Undesirable side effects caused by our product candidates could interrupt, delay or halt clinical trials and could result in the denial of regulatory approval by the FDA or other regulatory authorities for any or all targeted indications, and in turn prevent us from commercializing our product candidates and generating revenues from their sale.

In addition, if any of our product candidates receive marketing approval and we or others later identify undesirable side effects caused by the product:

•regulatory authorities may require the addition of restrictive labeling statements;

•regulatory authorities may withdraw their approval of the product; and

•we may be required to change the way the product is administered or conduct additional clinical trials.

Any of these events could prevent us from achieving or maintaining market acceptance of the affected product or could substantially increase the costs and expenses of commercializing the product candidate, which in turn could delay or prevent us from generating significant revenues from its sale or adversely affect our reputation.

We do not currently have patent protection for certain of our commercial products. If we are unable to obtain and maintain protection for the intellectual property relating to our technology and products, their value will be adversely affected.

Our success will depend in large part on our ability to obtain and maintain protection in the United States and other countries for the intellectual property covering, or incorporated into, our technology and products. The patent situation in the field of biotechnology and pharmaceuticals generally is highly uncertain and involves complex legal, technical, scientific and factual questions. We do not have, and do not expect to obtain, patent protection for Thiola, Chenodal or Cholbam. Additionally, although we have a license to a granted U.S. patent covering the treatment of cystinuria by administering Thiola EC with food (U.S. Patent No. 11,458,104, "the '104 patent”), as well as a pending U.S. patent application directed to Thiola EC, we do not know whether the pending U.S. patent application or any future patent application will result in a granted patent covering Thiola EC. More generally, we may not be able to obtain additional issued patents relating to our technology or products. Even if issued, patents issued to us or our licensors, including for example the 104 patent, may be challenged, narrowed, invalidated, held to be unenforceable or circumvented, which could limit our ability to stop competitors from marketing similar products or reduce the term of patent protection we may have for our products. Changes in either patent laws or in interpretations of patent laws in the United States and other countries may diminish the value of our intellectual property or narrow the scope of our patent protection.

Patent laws vary by country. Some countries have compulsory licensing laws under which a patent owner may be required to grant licenses to third parties. Some countries do not grant or enforce patents related to medical treatments, or limit enforceability in the case of a public emergency. In addition, many countries limit the enforceability of patents against government agencies or government contractors. If we are unable to obtain or enforce patents related to medical treatments in certain countries, or we or any of our licensors is forced to grant a license to third parties with respect to any patents relevant to our business, our business may be adversely affected.

The intellectual property systems in other countries can be destabilized as a result of political events, during which the ability to obtain, maintain and enforce intellectual property protection in the affected country may be uncertain and evolving. For example, as a result of the ongoing war between Ukraine and Russia, Russian officials have suggested that they may treat patents or patent applications owned by parties from certain countries, including the United States, as unenforceable and/or provide for zero compensation compulsory licenses to such patents or patent applications. Recent court decisions in Russia

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have raised questions about the strength of trademark protections in Russia. The U.S. government’s response to political events may also negatively affect our ability to obtain, maintain and enforce intellectual property protection in the affected country. For example, the U.S. government has issued sanctions against Russia related to the ongoing war in Ukraine, and as a result of these sanctions, it may not be possible to pay fees necessary for prosecution and maintenance of Russian patent applications and patents in the absence of licenses or exclusions set forth by the U.S. government authorizing transactions in connection with intellectual property. Payments for trademark protection may be similarly impacted. The U.S. Department of the Treasury has issued General License No. 31, authorizing such transactions to allow filing, prosecution and maintenance of Russian patents and trademarks. Uncertainties regarding political events, including the ongoing war between Ukraine and Russia, as well as any resulting losses of intellectual property protection, could harm our business.

Our product FILSPARI is covered by U.S. Patent No. 6,638,937, which expired in 2019 and to which we have an exclusive license. In addition, U.S. Patent No. 9,662,312, to which we also have an exclusive license and which was granted on May 30, 2017 and expires in 2030, covers the use of sparsentan for treating glomerulosclerosis, including FSGS. U.S. Patent No. 9,993,461, to which we also have an exclusive license and which was granted on June 12, 2018 and expires in 2030, covers the use of sparsentan for treating IgAN as well as glomerulosclerosis, including FSGS.

For products we develop based on a new chemical entity not previously approved by the FDA, we expect that in addition to the protection afforded by our patent filings that we will be able to obtain five years regulatory exclusivity via the provisions of the Food, Drug, and Cosmetic Act ("FDC Act") and possibly seven years regulatory exclusivity via the orphan drug provisions of the FDC Act. In the case of sparsentan, the periods of regulatory exclusivity may, if certain conditions are satisfied, be extended by six months on the basis of pediatric exclusivity, thereby resulting in exclusivity periods of 5.5 years and 7.5 years, respectively. In addition, we may be able to obtain up to five years patent term extension (to compensate for regulatory approval delay) for one patent covering such a product for its FDA-approved use. Such a patent, like the periods of regulatory exclusivity, also may be extended by a further six months on the basis of pediatric exclusivity if certain conditions are satisfied.

The degree of future protection for our proprietary rights is uncertain, and we cannot ensure that:

•we or our licensors were the first to make the inventions covered by each of our pending patent applications;

•we or our licensors were the first to file patent applications for these inventions;

•others will not independently develop similar or alternative technologies or duplicate any of our technologies;

•any patents issued to us or our licensors that provide a basis for commercially viable products will provide us with any competitive advantages or will not be challenged by third parties;

•we will develop additional proprietary technologies that are patentable;

•we will file patent applications for new proprietary technologies promptly or at all;

•the claims we make in our patents will be upheld by patent offices in the United States and elsewhere;

•our patents will not expire prior to or shortly after commencing commercialization of a product; and

•the patents of others will not have a negative effect on our ability to do business.

We have negotiated a license agreement with Ligand Pharmaceuticals for the rights to sparsentan which we are initially developing for the treatment of IgAN and FSGS. This license subjects us to various commercialization, reporting and other obligations. If we were to default on our obligations, we and CSL Vifor could lose our rights to sparsentan. We have obtained a U.S. patent and European patent each covering the use of sparsentan for treating glomerulosclerosis, including FSGS, as well as a second U.S. patent and a second European patent each covering both the use of sparsentan for treating IgAN and the use of sparsentan for treating glomerulosclerosis, including FSGS. In addition, in 2020 we obtained a U.S. patent covering the use of sparsentan for the treatment of Alport syndrome. However, we cannot be certain that we will be able to obtain patent protection for various other potential indications for sparsentan, or whether, if granted, we would be able to enforce such patents. Additionally, in November 2020, a third party filed an opposition to our second European patent (European Patent No. EP3222277, “the ‘277 EP Patent”), in the European Patent Office ("EPO"). While we are vigorously defending the ‘277 EP Patent against the opposition, there is no guarantee that we will be successful in doing so.

Our patents also may not afford us protection against competitors with similar technology. Because patent applications in the United States and many other jurisdictions are typically not published until 18 months after filing, or in some cases not at all, and because publications of discoveries in the scientific literature often lag behind the actual discoveries, neither we nor our licensors can be certain that we or they were the first to make the inventions claimed in our or their issued patents or pending patent applications, or that we or they were the first to file for protection of the inventions set forth in these patent applications. If a third party has also filed a United States patent application prior to the effective date of the relevant provisions of the America Invents Act (i.e. before March 16, 2013) covering our product candidates or a similar invention, we may have to participate in an adversarial proceeding, known as an interference, declared by the USPTO to determine priority of invention in the United States. The costs of these proceedings could be substantial, and it is possible that our efforts could be unsuccessful, resulting in a loss of our United States patent position.

We cannot assure you that third parties will not assert patent or other intellectual property infringement claims against us with respect to technologies used in our products. If patent infringement suits were brought against us, we may be unable to commercialize some of our products which could severely harm our business. Litigation proceedings, even if not successful, could result in substantial costs and harm our business.

We expect to rely on orphan drug status to develop and commercialize certain of our products and product candidates, but our orphan drug designations may not confer marketing exclusivity or other expected commercial benefits.

We expect to rely on orphan drug exclusivity for sparsentan and potential future product candidates that we may develop. Orphan drug status currently confers seven years of marketing exclusivity in the United States under the FDC Act, and up to ten years of marketing exclusivity in Europe for a particular product in a specified indication or, in the case of orphan drugs for which a pediatric investigation plan has been completed, 12 years. The FDA and EMA

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have granted orphan designation for Chenodal, sparsentan, and pegtibatinase (TVT-058) for the treatment of CTX, IgAN and FSGS and homocystinuria, respectively. While we have been granted these orphan designations, we will not be able to rely on these designations to exclude other companies from manufacturing or selling these molecules for the same indication beyond these time frames. Furthermore, in Europe, orphan drug status is re-evaluated in connection with the marketing authorization review process and a product candidate must re-qualify as of such time in order to maintain orphan drug status. In addition, any marketing exclusivity in Europe can be reduced from ten years to six years if the initial designation criteria have significantly changed since the market authorization of the orphan product.

For any product candidate for which we have been granted orphan drug designation in a particular indication, it is possible that another company also holding orphan drug designation for the same product candidate will receive marketing approval for the same indication before we do. If that were to happen, our applications for that indication may not be approved until the competing company's period of exclusivity expires. Even if we are the first to obtain marketing authorization for an orphan drug indication in the United States, there are circumstances under which a competing product may be approved for the same indication during the seven-year period of marketing exclusivity, such as if the later product is shown to be clinically superior to our orphan product, or if the later product is deemed a different product than ours. Further, the seven-year marketing exclusivity would not prevent competitors from obtaining approval of the same product candidate as ours for indications other than those in which we have been granted orphan drug designation, or for the use of other types of products in the same indications as our orphan product.

Any drugs we develop may become subject to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, thereby harming our business.*

In March 2010, President Obama signed the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the "PPACA"), a sweeping law intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add new transparency requirements for healthcare and health insurance industries, impose new taxes and fees on the health industry and impose additional health policy reforms. The PPACA revised the definition of “average manufacturer price” for reporting purposes, which could increase the amount of Medicaid drug rebates to states. The PPACA also increased the mandated Medicaid rebate from 15.1% to 23.1% of the average manufacturer price, expanded the rebate to Medicaid managed care utilization and increased the types of entities eligible for the federal 340B drug discount program. Further, the law imposed a significant annual fee on companies that manufacture or import certain branded prescription drug products. There have been executive, judicial, Congressional, and political challenges to certain aspects of the PPACA. For example, on June 17, 2021 the U.S. Supreme Court dismissed a challenge on procedural grounds that argued the PPACA is unconstitutional in its entirety because the “individual mandate” was repealed by Congress. Further, on August 16, 2022, President Biden signed the Inflation Reduction Act of 2022 ("IRA") into law, which among other things, extends enhanced subsidies for individuals purchasing health insurance coverage in PPACA marketplaces through plan year 2025. The IRA also eliminates the "donut hole" under the Medicare Part D program beginning in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost and through a newly established manufacturer discount program. It is possible that the PPACA will be subject to judicial or Congressional challenges in the future. It is unclear how any additional healthcare reform measures of the Biden administration will impact the PPACA and our business.

In addition, other legislative changes have been proposed and adopted since the PPACA was enacted. For example, in August 2011, President Obama signed into law the Budget Control Act of 2011, which, among other things, includes aggregate reductions to Medicare payments to providers of up to 2% per fiscal year, which went into effect beginning on April 1, 2013 and, due to subsequent legislative amendments, will stay in effect until 2032 unless additional Congressional action is taken. Additionally, in January 2013, the American Taxpayer Relief Act of 2012 was signed into law, which, among other things, reduced Medicare payments to several providers, including hospitals and imaging centers.

If we are unable to obtain and maintain coverage and adequate reimbursement from governments or third-party payers for any products that we may develop or if we are unable to obtain acceptable prices for those products, our prospects for generating revenue and achieving profitability will suffer.*

Our prospects for generating revenue and achieving profitability will depend heavily upon the availability of coverage and adequate reimbursement for the use of our approved product candidates from governmental and other third-party payers, both in the United States and in other markets. Reimbursement by a third-party payer may depend upon a number of factors, including the third-party payer’s determination that use of a product is:

•a covered benefit under its health plan;

•safe, effective and medically necessary;

•appropriate for the specific patient;

•cost-effective; and

•neither experimental nor investigational.

Obtaining reimbursement approval for a product from each government or other third-party payer is a time consuming and costly process that could require us to provide supporting scientific, clinical and cost effectiveness data for the use of our products to each payer. We may not be able to provide data sufficient to gain acceptance with respect to reimbursement. Additionally, we might need to conduct post-marketing studies in order to demonstrate the cost-effectiveness of any future products to such payers’ satisfaction. Such studies might require us to commit a significant amount of management time and financial and other resources. Even when a payer determines that a product is eligible for reimbursement, the payer may impose coverage limitations that preclude payment for some uses that are approved by the FDA or non-United States regulatory authorities. Also, prior authorization for a product may be required. In addition, there is a risk that full reimbursement may not be available for high-priced products. Moreover, eligibility for coverage does not imply that any product will be reimbursed in all cases or at a rate that allows us to make a profit or even cover our costs. Interim payments for new products, if applicable, may also not be sufficient to cover our costs and may not be made permanent. Further, coverage policies and third-party payer reimbursement rates may change at any time. Even if favorable coverage and reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the future.

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A primary trend in the United States healthcare industry and elsewhere is toward cost containment. We expect the changes made by PPACA, other legislation impacting the Medicare program and the 340B program, and the increasing emphasis on managed care to continue to put pressure on pharmaceutical product pricing. As these concerns continue to grow over the need for tighter oversight, there remains the possibility that the Heath Resources and Services Administration or another agency under the U.S. Department of Health and Human Services ("HHS") will propose regulations or that Congress will explore changes to the 340B program through legislation. There have also been a number of initiatives pending at the state and federal level that could negatively impact the reimbursement for products approved under the accelerated approval pathway in the United States by restricting patient access or establishing differential payment models. Certain states are also in the process of establishing Patient Drug Affordability Boards with the authority in some cases to set upper payment limits.

Further, there has been increasing legislative and enforcement interest in the United States with respect to drug pricing practices, including several recent U.S. congressional inquiries and federal and state legislation designed to, among other things, increase drug pricing transparency, expedite generic competition, review relationships between pricing and manufacturer patient assistance programs, and reform government program drug reimbursement methodologies. At the federal level, in July 2021, the Biden administration released an executive order that included multiple provisions aimed at prescription drugs. In response to Biden’s executive order, on September 9, 2021, HHS released a Comprehensive Plan for Addressing High Drug Prices that outlines principles for drug pricing reform. The plan sets out a variety of potential legislative policies that Congress could pursue as well as potential administrative actions HHS can take to advance these principles. In addition, the IRA, among other things (i) directs HHS to negotiate the price of certain high-expenditure, single-source drugs and biologics covered under Medicare and (ii) imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation. These provisions will take effect progressively starting in fiscal year 2023, although the Medicare drug price negotiation program is currently subject to legal challenges. HHS has and will continue to issue and update guidance as these programs are implemented. It is currently unclear how the IRA will be implemented but it is likely to have a significant impact on the pharmaceutical industry. In addition, in response to the Biden administration’s October 2022 executive order, on February 14, 2023, HHS released a report outlining three new models for testing by the Center for Medicare and Medicaid Innovation which will be evaluated on their ability to lower the cost of drugs, promote accessibility, and improve quality of care. It is unclear whether the models will be utilized in any health reform measures in the future. At the state level, legislatures are increasingly passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.

Any reduction in reimbursement from Medicare, Medicaid or other government-funded programs may result in a similar reduction in payments from private payers. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our drugs. Additionally, we are currently unable to predict what additional legislation or regulation, if any, relating to the healthcare industry may be enacted in the future or what effect recently enacted federal legislation or any such additional legislation or regulation would have on our business.

We face potential product liability exposure far in excess of our limited insurance coverage.*

The use of any of our potential products in clinical trials, and the sale of any approved products, may expose us to liability claims. These claims might be made directly by consumers, health care providers, pharmaceutical companies or others selling our products. We have obtained limited product liability insurance coverage for our clinical trials in the amount of $10 million per occurrence and $30 million in the aggregate. However, our insurance may not reimburse us or may not be sufficient to reimburse us for any expenses or losses we may suffer. Moreover, insurance coverage is becoming increasingly expensive, and we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability. We intend to expand our insurance coverage as we obtain marketing approval for additional product candidates in development, but we may be unable to obtain commercially reasonable product liability insurance. On occasion, juries have awarded large judgments in class action lawsuits based on drugs that had unanticipated side effects. A successful product liability claim or series of claims brought against us would decrease our cash reserves and could cause our stock price to fall.

We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do. Our operating results will suffer if we fail to compete effectively.*

Several of our competitors have substantially greater financial, research and development, distribution, manufacturing and marketing experience and resources than we do and represent substantial long-term competition for us. Other companies may succeed in developing and marketing products that are more effective and/or less costly than any products that may be developed and marketed by us, or that are commercially accepted before any of our products. Factors affecting competition in the pharmaceutical and drug industries vary, depending on the extent to which a competitor is able to achieve a competitive advantage based on its proprietary technology and ability to market and sell drugs. The industry in which we compete is characterized by extensive research and development efforts and rapid technological progress. Although we believe that our orphan drug status and proprietary position with respect to sparsentan may give us a competitive advantage, new developments are expected to continue and there can be no assurance that discoveries by others will not render such potential products noncompetitive. Furthermore, competitors could enter the market with generic versions of our products. For example, a generic option for the 100 mg version of the original formulation of Thiola (tiopronin tablets) was approved by the FDA in May 2021 and a second 100 mg version of the original formulation of Thiola (tiopronin tablets) was approved by the FDA in June 2022. Also, a generic option for the 100 mg and 300 mg versions of Thiola EC was approved by the FDA in late February 2023.

Our competitive position also depends on our ability to enter into strategic alliances with one or more large pharmaceutical and contract manufacturing companies, attract and retain qualified personnel, develop effective proprietary products, implement development and marketing plans, obtain patent protection, secure adequate capital resources and successfully sell and market our approved products. There can be no assurance that we will be able to successfully achieve all of the foregoing objectives.

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Use of third parties to manufacture our products and product candidates may increase the risk that we will not have sufficient quantities of our product and product candidates or such quantities at an acceptable cost, and clinical development and commercialization of our product and product candidates could be delayed, prevented or impaired.*

We do not own or operate manufacturing facilities for clinical or commercial production of our products or product candidates. We have limited personnel with experience in drug manufacturing and we lack the resources and the capabilities to manufacture any of our product candidates on a clinical or commercial scale. We outsource all manufacturing and packaging of our nonclinical, clinical, and commercial products to third parties. The manufacture of pharmaceutical products requires significant expertise and capital investment, including the development of advanced manufacturing techniques and process controls. Manufacturers of pharmaceutical products often encounter difficulties in production, particularly in scaling up initial production and in maintaining required quality control. These problems include difficulties with production costs and yields and quality control, including stability of the product candidate.

We intend to rely on third-party manufacturers for the long-term commercial supply of FILSPARI and for our development stage product candidates. We expect the manufacturers of each product or product candidate to, at least initially and potentially for a significant period of time, be single source suppliers to us. Reliance on third-party manufacturers entails risks to which we may not be subject if we manufactured our product candidates or products ourselves, including:

•reliance on the third party for regulatory compliance and quality assurance;

•limitations on supply availability resulting from capacity and scheduling constraints of the third parties;

•less control over cost increases resulting from inflationary pressures affecting raw materials and other supply chain components;

•impact on our reputation in the marketplace if manufacturers of our products fail to meet the demands of our customers;

•the possible breach of the manufacturing agreement by the third party because of factors beyond our control; and

•the possible termination or nonrenewal of the agreement by the third party, based on its own business priorities, at a time that is costly or inconvenient for us.

The failure of any of our contract manufacturers to maintain high manufacturing standards could result in injury or death of clinical trial participants or patients using our products. Such failure could also result in product liability claims, product recalls, product seizures or withdrawals, delays or failures in testing or delivery, cost overruns or other problems that could seriously harm our business or profitability.

Our contract manufacturers are required to adhere to FDA regulations setting forth cGMP. These regulations cover all aspects of the manufacturing, testing, quality control and recordkeeping relating to our product candidates and any products that we may commercialize. Our manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements outside the United States. Our manufacturers are subject to unannounced inspections by the FDA, state regulators and similar regulators outside the United States. We are ultimately responsible for ensuring that our API and finished products are manufactured in accordance with cGMP regulations and similar regulatory requirements outside the United States, and it is therefore critical that we maintain effective management practices and oversight with respect to our third-party manufacturers, including routine auditing. Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including fines, injunctions, civil penalties, failure of regulatory authorities to grant marketing approval of our product candidates, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or products, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect regulatory approval and supplies of our product candidates.

Our product and any products that we may develop may compete with other product candidates and products for access to manufacturing facilities. There are a limited number of manufacturers that operate under cGMP regulations and that are both capable of manufacturing for us and willing to do so. A health epidemic or pandemic and associated vaccine or treatment development and manufacturing efforts may increase demand for the services supplied by many third-party manufacturers, including some of those that we utilize for our products and product candidates, which may result in decreased availability of manufacturing slots at many such facilities. If the third parties that we engage to manufacture products for our developmental or commercial products should halt or cease to continue to do so for any reason, we likely would experience interruptions in cash flows and/or delays in advancing our clinical trials while we identify and qualify replacement suppliers, and we may be unable to obtain replacement supplies on terms that are favorable to us. Later relocation to another manufacturer will also require notification, review and other regulatory approvals from the FDA and other regulators and will subject our production to further cost and instability in the availability of our product candidates. In addition, if we are not able to obtain adequate supplies of our products and product candidates, or the drug substances used to manufacture them, it will be more difficult for us to sell our products and to develop our product candidates. This could greatly reduce our competitiveness and negatively affect our results of operations.

Our current and anticipated future dependence upon others for the manufacture of our products and product candidates may adversely affect our future profit margins and our ability to develop product candidates and commercialize our marketed products and any other products that may obtain regulatory approval on a timely and competitive basis.

Materials necessary to manufacture our products and product candidates may not be available on commercially reasonable terms, or at all, which may delay the development and commercialization of our products and product candidates.

We rely on the manufacturers of our products and product candidates to purchase from third-party suppliers the materials necessary to produce the compounds for our nonclinical and clinical studies and rely on these other manufacturers for commercial distribution if we obtain marketing approval for any of our product candidates. Suppliers may not sell these materials to our manufacturers at the time we need them or on commercially reasonable terms and all such prices are susceptible to fluctuations in price and availability due to transportation costs, government regulations, price controls, and changes in economic climate or other foreseen circumstances. We do not have any control over the process or timing of the acquisition of these materials by our manufacturers. In addition, significant increases in inflation and global supply chain disruptions, as well as past and potential future disruptions related to COVID-19 and potential future disruptions related to Russia's invasion of Ukraine and global geopolitical tension, including between the U.S. and China, have had and may continue to have a negative impact on our manufacturers’ ability to acquire the materials necessary for our business. Moreover, we currently do

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not have any agreements for the commercial production of these materials. If our manufacturers are unable to obtain these materials for our nonclinical and clinical studies, product testing and potential regulatory approval of our product candidates would be delayed, significantly impacting our ability to develop our product candidates. If our manufacturers or we are unable to purchase these materials after regulatory approval has been obtained for our product candidates, the commercial launch of our product candidates would be delayed or there would be a shortage in supply, which would materially affect our ability to generate revenues from the sale of our product candidates. For example, in 2021 a membrane used in pegtibatinase (TVT-058) drug substance manufacturing became more difficult to acquire due to the same or similar membranes being used in certain of the COVID-19 vaccine manufacturing and we continue to see challenges with securing materials used in the pegtibatinase manufacturing process that are in short supply as a result of COVID-19. While we believe our contingency plans will enable us to continue the ongoing clinical study of pegtibatinase (TVT-058) with the currently available clinical supplies, there is no guarantee that we will not face additional shortages of this membrane, or other materials necessary to manufacture pegtibatinase (TVT-058) or our other products and product candidates. If our risk mitigation plans are not successful in overcoming these challenges, our pegtibatinase program or other products and product candidates, could be delayed.

Risks Related to Our Business

Our limited operating history makes it difficult to evaluate our future prospects, and our profitability in the future is uncertain.*

We face the problems, expenses, difficulties, complications and delays, many of which are beyond our control, associated with any business in its early stages and have a limited operating history on which an evaluation of our prospects can be made. Such prospects should be considered in light of the risks, expenses and difficulties frequently encountered in the establishment of a business in a new industry, characterized by a number of market entrants and intense competition, and in the shift from development to commercialization of new products based on innovative technologies.

We have experienced significant growth over the past five years in the number of our employees and the scope of our operations. We have expanded our sales and marketing, compliance and legal functions in addition to expansion of all functions to support a commercial organization. We have also expanded our operations in connection with the commercial launch of FILSPARI in the United States, including by adding additional members to our sales force, and expect to continue to hire additional staff in the coming months. To manage our anticipated future growth, we must continue to implement and improve our managerial, operational and financial systems, expand our facilities, continue to recruit and train additional qualified personnel, and successfully integrate such expanded operations into our existing business. To succeed, we must recruit, retain, manage and motivate qualified clinical, scientific, technical, commercial and management personnel, and we face significant competition for experienced personnel.

Due to our limited resources, we may not be able to effectively manage the expansion of our operations or recruit and train additional qualified personnel, including in connection with the ongoing commercial launch of FILSPARI in the United States. The physical expansion of our operations may lead to significant costs and may divert our management and business development resources. Any inability on the part of our management to manage growth could delay the execution of our business plans or disrupt our operations.

Factors that may inhibit our efforts to commercialize our products without strategic partners or licensees include:

•our inability to recruit and retain adequate numbers of effective sales and marketing personnel;

•the inability of sales personnel to obtain access to or educate adequate numbers of physicians to prescribe our products;

•the lack of complementary products to be offered by our sales personnel, which may put us at a competitive disadvantage against companies with broader product lines;

•unforeseen costs associated with expanding our own sales and marketing team for new products or with entering into a partnering agreement with an independent sales and marketing organization; and

•efforts by our competitors to commercialize competitive products.

Moreover, though we generate revenues from product sales arrangements, we may incur significant operating losses over the next several years. Our ability to achieve profitable operations in the future will depend in large part upon successful in-licensing of products approved by the FDA, selling and manufacturing these products, completing development of our products, obtaining regulatory approvals for these products, and bringing these products to market. The likelihood of the long-term success of our company must be considered in light of the expenses, difficulties and delays frequently encountered in the development and commercialization of new drug products, competitive factors in the marketplace, as well as the regulatory environment in which we operate.

In addition, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors.

We depend on a highly experienced and skilled workforce to grow and operate our business. If we are unable to attract, retain and engage our employees, we may not be able to grow effectively.*

The execution of our strategic objectives and future success will depend upon our continued ability to identify, hire, develop, motivate and retain a highly qualified workforce. We depend on contributions from our employees, and, in particular, our senior management team, to execute efficiently and effectively. Our success further depends on our ability to attract, retain and motivate highly skilled mid-level and senior managers as well as team members at various levels in the scientific, development, medical and commercial areas of the business, particularly as we hire additional personnel in connection with ongoing commercial launch of FILSPARI in the United States.

Our headquarters are based in San Diego, California. This region is home to many other biopharmaceutical companies and many academic and research institutions. Competition for qualified key talent in our market is intense and may limit our ability to hire and retain employees on acceptable terms, or at all. As a result, we may not be able to retain our existing employees or hire new employees quickly enough to meet our needs.

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To induce valuable employees to remain at our company, in addition to salary, cash incentives and other employee benefits, we have provided stock options and restricted stock unit ("RSU") awards that vest over time. The value to employees of stock options and RSU awards that vest over time may be significantly affected by movements in our stock price that are beyond our control and may at any time be insufficient to counteract more lucrative offers from other companies. Current market conditions and the potential for extreme stock price volatility exacerbates this risk. Despite our efforts to retain valuable employees, members of our management, scientific, development and commercial teams may terminate their employment with us on short notice. All of our employees have at-will employment, which means that they could leave our employment at any time, with or without notice. We do not maintain “key person” insurance policies on the lives of any of our employees.

If we fail to effectively manage our hiring and retention needs, our ability to meet our strategic objectives and our business and operating results may be adversely impacted.

Health epidemics or pandemics could materially adversely affect our business, results of operations and financial condition.*

A health epidemic or pandemic poses the risk that we or our clinical trial subjects, employees, contractors, collaborators, suppliers and vendors may be prevented from conducting certain clinical trials or other business activities for an indefinite period of time, including due to travel restrictions, quarantines, “stay-at-home” and "shelter-in-place" orders or shutdowns that have been or may be requested or mandated by governmental authorities, or that our or their ability to conduct operations will be negatively impacted by staffing shortages while employees quarantine as a result of exposure to or transmission of the virus. In addition, a health epidemic or pandemic could impact personnel at third-party manufacturing facilities in the United States and other countries, including China, or the availability or cost of materials, which could potentially disrupt the supply chain for our commercial products, our product candidates or the comparator products in our ongoing clinical trials.

The timelines and conduct of our ongoing clinical trials previously have been affected by COVID-19 and we may experience similar delays or interruptions due to COVID-19 or other health epidemics or pandemics in the future. For example, in 2020 we experienced a reduction in the rates of patient enrollment in our ongoing clinical trials as a result of the COVID-19 pandemic. New health epidemics or pandemics may emerge that result in similar or more severe disruptions to our business, which could adversely impact our business and operating results.

We will likely experience fluctuations in operating results and could incur substantial losses.*

We expect that our operating results will vary significantly from quarter-to-quarter and year-to-year as a result of investments in research and development, specifically our clinical and nonclinical development activities. We anticipate that our expenses will continue to increase as we:

•continue the ongoing portion of the Phase 3 trial of FILSPARI for the treatment of IgAN to the confirmatory endpoint and through the open-label extension period;

•continue the open label portions of DUET and DUPLEX;

•continue the research and development of additional product candidates, including pegtibatinase (TVT-058);

•expand our sales and marketing infrastructure to commercialize our current approved products, and any other products for which we may obtain regulatory approval; and

•expand operational, financial, and management information systems and personnel, including personnel to support product development efforts and our obligations as a public company.

To attain and sustain profitability, we must succeed in developing and commercializing drugs with significant market potential. This will require us to be successful in a range of challenging activities, including the discovery of product candidates, successful completion of nonclinical testing and clinical trials of our product candidates, obtaining regulatory approval for these product candidates and manufacturing, marketing and selling those products for which we may obtain regulatory approval. We may not be successful enough in these activities to generate revenues that are substantial enough to recoup the expenses we have expended in conducting these activities to achieve profitability. Pursuant to the Ligand License Agreement, we are obligated to pay to Ligand an escalating annual royalty between 15% and 17% of net sales of FILSPARI and any other products containing sparsentan or related compounds, which will impact our potential future profit from the commercialization of FILSPARI in the United States and sparsentan for the treatment of IgAN in Europe, if approved, as well as sparsentan for the treatment of FSGS, if approved. Even if we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis. Our failure to become or remain profitable could depress the market price of our common stock and could impair our ability to raise capital, expand our business, diversify our product offerings or continue our operations. A decline in the market price of our common stock may also cause a loss of a part or all of your investment.

Negative publicity regarding any of our products could impair our ability to market any such product and may require us to spend time and money to address these issues.

If any of our products or any similar products distributed by other companies prove to be, or are asserted to be, harmful to consumers and/or subject to FDA enforcement action, our ability to successfully market and sell our products could be impaired. Because of our dependence on patient and physician perceptions, any adverse publicity associated with illness or other adverse effects resulting from the use or misuse of our products or any similar products distributed by other companies could limit the commercial potential of our products and expose us to potential liabilities.

We may not have sufficient insurance to cover our liability in any current or future litigation claims either due to coverage limits or as a result of insurance carriers seeking to deny coverage of such claims.

We face a variety of litigation-related liability risks. Our certificate of incorporation, bylaws, other applicable agreements, and/or Delaware law require us to indemnify (and advance expenses to) our current and past directors and officers and employees from reasonable expenses related to the defense of any action arising from their service to us, including circumstances under which indemnification is otherwise discretionary. While our directors and officers are included in a director and officer liability insurance policy, which covers all our directors and officers in some circumstances, our insurance coverage does not cover all of our indemnification obligations and may not be adequate to cover any indemnification or other claims against us. In addition, the underwriters of

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our present coverage may seek to avoid coverage in certain circumstances based upon the terms of the respective policies. If we incur liabilities that exceed our coverage under our directors and officers insurance policy or incur liabilities not covered by our insurance, we would have to self-fund any indemnification amounts owed to our directors and officers and employees in which case our results of operations and financial condition could be materially adversely affected. Further, if D&O insurance becomes prohibitively expensive to maintain in the future, we may be unable to renew such insurance on economic terms or unable to renew such insurance at all. The lack of D&O insurance may make it difficult for us to retain and attract talented and skilled directors and officers to serve our company, which could adversely affect our business.

We may need substantial funding and may be unable to raise capital when needed.*

We expect our general and research and development expenses to increase in connection with our ongoing and planned activities, particularly as we conduct later-stage clinical trials of our product candidates. In addition, in connection with the commercial launch of FILSPARI in the United States, we have begun to incur significant commercialization expenses and expect to continue to incur significant commercialization expenses for FILSPARI and any other future approved products, including for product sales and marketing, securing commercial quantities of product from our manufacturers, and product distribution. Our expenses have and may continue to increase as a result of increasing inflation in the United States and abroad. We currently have no additional commitments or arrangements for any additional financing to fund the research and development and commercial launch of our product candidates. General market conditions, including increases in interest rates and stock price volatility, actual or anticipated bank failures, and ongoing issues arising from COVID-19, Russia's invasion of Ukraine and global geopolitical tensions, as well as market conditions affecting companies in the life sciences industry in general, may make it difficult for us to seek financing from the capital markets on attractive terms, or at all.

Management believes our ability to continue our operations depends on our ability to sustain and grow revenue, results of operations and our ability to access capital markets when necessary to accomplish our strategic objectives. Management believes that we may incur losses in the immediate future. We expect that our operating results will vary significantly from quarter-to-quarter and year-to-year as a result of investments in research and development, specifically our clinical and nonclinical development activities. We expect to finance our cash needs from cash on hand and results of operations, and depending on results of operations we may either need additional equity or debt financing, or need to enter into strategic alliances on products in development to continue our operations until we can achieve sustained profitability and positive cash flows from operating activities. Additional funds may not be available to us when we need them on terms that are acceptable to us, or at all. If adequate funds are not available to us on a timely basis, we may be required to reduce or eliminate research development programs or commercial efforts.

Our future capital requirements will depend on many factors, including:

•the timing, progress, cost and results of our clinical trials, preclinical studies and other discovery and research and development activities;

•the timing of, and costs involved in, seeking and obtaining marketing approvals for our products, and in maintaining quality systems standards for our products;

•the timing of, and costs involved in, commercial activities, including product marketing, sales and distribution;

•our ability to successfully commercialize FILSPARI in adult patients with IgAN, and to obtain regulatory approval for and successfully commercialize our other or future product candidates;

•increases or decreases in revenue from our marketed products, including decreases resulting from generic entrants or health epidemics or pandemics such as COVID-19;

•debt service obligations on the 2025 Notes and 2029 Notes;

•the number and development requirements of other product candidates that we pursue;

•our ability to manufacture sufficient quantities of our products to meet expected demand;

•the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing and defending intellectual property related claims;

•our ability to enter into collaboration, licensing or distribution arrangements and the terms and timing of these arrangements;

•the potential need to expand our business, resulting in additional payroll and other overhead expenses;

•the potential in-licensing of other products or technologies;

•the emergence of competing products and technologies and other adverse market developments;

•the extent to which we acquire or invest in businesses, products and technologies; and

•the potential impacts of inflation and resulting cost increases.

The market price for shares of our common stock may be volatile and purchasers of our common stock could incur substantial losses.*

The price of our stock is likely to be volatile. The stock market in general, and the market for biotechnology companies in particular, have experienced extreme volatility that has often been unrelated to the operating performance of particular companies. The market price for our common stock may be influenced by many factors, including:

•results of clinical trials of our product candidates or those of our competitors;

•our entry into or the loss of a significant collaboration;

•regulatory or legal developments in the United States and other countries, including changes in the health care payment systems;

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•our ability to obtain and maintain marketing approvals from the FDA or similar regulatory authorities outside the United States;

•variations in our financial results or those of companies that are perceived to be similar to us;

•changes in the structure of healthcare payment systems;

•market conditions in the pharmaceutical and biotechnology sectors and issuance of new or changed securities analysts’ reports or recommendations;

•general economic, industry and market conditions, including the impacts thereon of inflation and rising interest rates, actual or anticipated bank failures, COVID-19, Russia's invasion of Ukraine and global geopolitical tensions;

•results of clinical trials conducted by others on drugs that would compete with our product candidates;

•developments or disputes concerning patents or other proprietary rights;

•public concern over our product candidates or any products approved in the future;

•litigation;

•communications from government officials regarding health care costs or pharmaceutical pricing;

•future sales or anticipated sales of our common stock by us or our stockholders; and

•the other factors described in this “Risk Factors” section.

In addition, the stock markets, and in particular, the Nasdaq Stock Market, have experienced extreme price and volume fluctuations that have affected and continue to affect the market prices of equity securities of many pharmaceutical companies. The realization of any of the above risks or any of a broad range of other risks, including those described in these “Risk Factors” could have a dramatic and material adverse impact on the market price of our common stock.

We might not successfully complete the sale of our bile acid product portfolio for the treatment of rare liver diseases when expected, or at all.*

On July 16, 2023, we entered into a definitive asset purchase agreement (the “Purchase Agreement”) with Mirum Pharmaceuticals, Inc. (“Mirum”), pursuant to which we have agreed to sell to Mirum, subject to the terms of the Purchase Agreement and subject to the closing of the transaction, substantially all of our assets that are primarily related to our business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam) (the “Products”). A portion of the consideration for the sale is in the form of milestone payments that will only be payable upon the achievement of certain milestones based on specified amounts of annual net sales of the Products (the “Milestone Events”). The closing of the transaction is subject to customary closing conditions, including but not limited to regulatory approval. Although we anticipate the transaction closing in the third quarter of 2023, there can be no assurance that the transaction will be consummated when expected, or at all. Failure to complete the transaction could negatively impact us in various ways, including but not limited to a negative impact on our financial condition and anticipated cash runway, and it could negatively impact our efforts regarding our other products and pipeline as part of our overall business strategy. In connection with the proposed transaction, we also face risks related to the effect of the announcement or pendency of the sale on our relationships with customers, suppliers, distributors and other business partners; risks relating to potential diversion of management attention away from our ongoing business operations; and the possibility that any or all of the Milestone Events might not be achieved and that any or all of the consideration tied to the achievement of the Milestone Events might not be received. The realization of any of the above risks could have an adverse impact on our business and operating results.

We may be unable to successfully integrate new products or businesses we may acquire.

We intend to expand our product pipeline by pursuing acquisition of pharmaceutical products. If an acquisition is consummated, the integration of the acquired business, product or other assets into our company may also be complex and time- consuming and, if such businesses, products and assets are not successfully integrated, we may not achieve the anticipated benefits, cost-savings or growth opportunities. Potential difficulties that may be encountered in the integration process include the following:

•integrating personnel, operations and systems, while maintaining focus on producing and delivering consistent, high quality products;

•coordinating geographically dispersed organizations;

•distracting employees from operations;

•retaining existing customers and attracting new customers; and

•managing inefficiencies associated with integrating the operations of the acquired company or product into our own operations.

Furthermore, these acquisitions and other arrangements, even if successfully integrated, may fail to further our business strategy as anticipated, expose us to increased competition or challenges with respect to our products or geographic markets, and expose us to additional liabilities associated with an acquired business, product, technology or other asset or arrangement. Any one of these challenges or risks could impair our ability to realize any benefit from our acquisitions or arrangements after we have expended resources on them.

Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we may develop.

Our business exposes us to potential liability risks inherent in the research, development, manufacturing and marketing of pharmaceutical products. If any of our product candidates in clinical trials or commercialized products harm people we may be subject to costly and damaging product liability claims. We have clinical trial insurance and commercial product liability coverage. However, this insurance may not be adequate to cover all claims. We may be exposed to

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product liability claims and product recalls, including those which may arise from misuse or malfunction of, or design flaws in, such products, whether or not such problems directly relate to the products and services we have provided. If we cannot successfully defend ourselves against claims that our product candidates or products caused injuries, we will incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:

•decreased demand for any product candidates or products that we may develop;

•damage to our reputation;

•regulatory investigations that could require costly recalls or product modifications;

•withdrawal of clinical trial participants;

•costs to defend the related litigation;

•substantial monetary awards to trial participants or patients, including awards that substantially exceed our product liability insurance, which we would then be required to pay from other sources, if available, and would damage our ability to obtain liability insurance at reasonable costs, or at all, in the future;

•loss of revenue;

•the diversion of management’s attention from managing our business; and

•the inability to commercialize any products that we may develop.

A successful product liability claim or a series of claims brought against us could cause our stock price to fall and, if judgments exceed our insurance coverage, could decrease our available cash and adversely affect our business.

We may become involved in litigation matters, which could result in substantial costs, divert management's attention and otherwise have a material adverse effect on our business, operating results or financial condition.*

From time to time we may become involved in certain litigation matters, including those described in Note 13 of the Consolidated Financial Statements included in this report. Although we intend to vigorously defend our interests in each matter, there is no guarantee that we will be successful and we may have to pay damages awards or otherwise may enter into settlement arrangements in connection with such matters. Any such payments or settlement arrangements could have material adverse effects on our business, operating results or financial condition. Even if we are successful in defending our interests in each matter, litigation with respect to such matters could result in substantial costs and significant adverse impact on our reputation and divert management's attention and resources, which could have a material adverse effect on our business, operating results or financial condition.

We are subject to significant ongoing regulatory obligations and oversight, which may result in significant additional expense and may limit our commercial success.*

We are subject to significant ongoing regulatory obligations, such as safety reporting requirements and additional post-marketing obligations, including regulatory oversight of the promotion and marketing of our products. In addition, the manufacture, quality control, labeling, packaging, safety surveillance, adverse event reporting, storage and recordkeeping for our products are subject to extensive and ongoing regulatory requirements. If we become aware of previously unknown problems with any of our products, a regulatory agency may impose restrictions on our products, our contract manufacturers or us. If we, our products and product candidates, or the manufacturing facilities for our products and product candidates fail to comply with applicable regulatory requirements, a regulatory agency, including the FDA, may send enforcement letters, mandate labeling changes, suspend or withdraw regulatory approval, suspend any ongoing clinical trials, refuse to approve pending applications or supplements filed by us, suspend or impose restrictions on manufacturing operations, request a recall of, seize or detain a product, seek criminal prosecution or an injunction, or impose civil or criminal penalties or monetary fines. In such instances, we could experience a significant drop in the sales of the affected products, our product revenues and reputation in the marketplace may suffer, and we could become the target of lawsuits.

We are also subject to regulation by national, regional, state and local agencies, including but not limited to the FDA, the Centers for Medicare & Medicaid Services ("CMS"), Department of Justice, the Federal Trade Commission, the HHS Office of Inspector General and other regulatory bodies. The FDC Act, Social Security Act, Public Health Service Act and other federal and state statutes and regulations govern to varying degrees the research, development, manufacturing and commercial activities relating to prescription pharmaceutical products, including nonclinical testing, clinical research, approval, production, labeling, sale, distribution, post-market surveillance, advertising, dissemination of information, promotion, marketing, and pricing to government purchasers and government health care programs. Our manufacturing partners are subject to many of the same requirements.

Companies may not promote drugs for “off-label” uses-that is, uses that are not described in the product’s labeling and that differ from those approved by the FDA or other applicable regulatory agencies. However, a company may share truthful and not misleading information that is otherwise consistent with the product’s labeling. A company that is found to have improperly promoted off-label uses may be subject to significant liability, including civil and administrative remedies as well as criminal sanctions. In addition, management’s attention could be diverted from our business operations and our reputation could be damaged.

The federal health care program Anti-Kickback Statute prohibits, among other things, knowingly and willfully offering, paying, soliciting, or receiving remuneration to induce or in return for purchasing, leasing, ordering or arranging for the purchase, lease or order of any health care item or service reimbursable under Medicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted broadly to apply to arrangements that pharmaceutical companies have with prescribers, purchasers and formulary managers, among others. Further, the PPACA, among other things, amends the intent requirement of the federal Anti-Kickback Statute so that a person or entity no longer needs to have actual knowledge of this statute or specific intent to violate it. In addition, the PPACA provides that the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the civil False Claims Act. Although there are a number of statutory exceptions and regulatory safe harbors under the federal Anti-Kickback Statute protecting certain common manufacturer business arrangements and

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activities from prosecution, the exceptions and safe harbors are drawn narrowly and an arrangement must meet all of the conditions specified in order to be fully protected from scrutiny under the federal Anti-Kickback Statute. We seek to comply with the exceptions and safe harbors whenever possible, but our practices, such as our patient assistance programs and prompt pay discounts with certain customers, may not in all cases meet all of the criteria for protection from Anti-Kickback liability and may be subject to scrutiny.

The federal false claims laws, including the federal False Claims Act, prohibit any person or entity from knowingly presenting, or causing to be presented, a false claim for payment to the federal government, or knowingly making, or causing to be made, a false statement to get a false claim paid. Additionally, the civil monetary penalties statute imposes penalties against any person or entity that, among other things, is determined to have presented or caused to be presented a claim to a federal health program that the person knows or should know is for an item or service that was not provided as claimed or is false or fraudulent. Many pharmaceutical and other health care companies have been investigated and have reached substantial financial settlements with the federal government under the federal False Claims Act for a variety of alleged marketing activities, including providing free product to customers with the expectation that the customers would bill federal programs for the product; providing consulting fees, grants, free travel, and other benefits to physicians to induce them to prescribe the company’s products; and inflating prices reported to private price publication services, which may be used by states to set drug payment rates under government health care programs. Companies have been prosecuted for causing false claims to be submitted because of the marketing of their products for unapproved uses. Pharmaceutical and other health care companies have also been prosecuted on other legal theories of Medicare and Medicaid fraud.

Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and promotional activities for pharmaceutical products. It is not clear whether additional legislative changes will be enacted, or whether the FDA regulations, guidance or interpretations will be changed, or what the impact of such changes on the marketing approvals of any Travere products, if any, may be. In addition, increased scrutiny by the U.S. Congress of the FDA’s approval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing and other requirements.

We also could become subject to government investigations and related subpoenas. Such subpoenas are often associated with previously filed qui tam actions, or lawsuits filed under seal under the federal False Claims Act. Qui tam actions are brought by private plaintiffs suing on behalf of the federal government for alleged violations of the federal False Claims Act. The time and expense associated with responding to such subpoenas, and any related qui tam or other actions, may be extensive, and we cannot predict the results of our review of the responsive documents and underlying facts or the results of such actions. Responding to government investigations, defending any claims raised, and any resulting fines, restitution, damages and penalties, settlement payments or administrative actions, as well as any related actions brought by stockholders or other third parties, could have a material impact on our reputation, business and financial condition and divert the attention of our management from operating our business.

The number and complexity of both federal and state laws continues to increase, and additional governmental resources are being added to enforce these laws and to prosecute companies and individuals who are believed to be violating them. In particular, the PPACA includes a number of provisions aimed at strengthening the government’s ability to pursue Anti-Kickback and False Claims Act cases against pharmaceutical manufacturers and other healthcare entities, including substantially increased funding for healthcare fraud enforcement activities, enhanced investigative powers, amendments to the federal False Claims Act that make it easier for the government and whistleblowers to pursue cases for alleged kickback and false claim violations and public reporting of certain payments and transfers of value by certain pharmaceutical manufacturers to physicians and teaching hospitals nationwide. We anticipate that government scrutiny of pharmaceutical sales and marketing practices will continue for the foreseeable future and subject us to the risk of further government investigations and enforcement actions. Responding to a government investigation or enforcement action would be expensive and time-consuming, and could have a material adverse effect on our business, financial condition, results of operations and growth prospects.

The U.S. Foreign Corrupt Practices Act, and similar worldwide anti-bribery laws generally prohibit companies and their intermediaries from making improper payments to government officials for the purpose of obtaining or retaining business. Our policies mandate compliance with these anti-bribery laws. We operate in parts of the world that have experienced governmental corruption to some degree and in certain circumstances, strict compliance with anti-bribery laws may conflict with local customs and practices or may require us to interact with doctors and hospitals, some of which may be state controlled, in a manner that is different than in the United States. We cannot assure that our internal control policies and procedures will protect us from reckless or criminal acts committed by our employees or agents. Violations of these laws, or allegations of such violations, could disrupt our business and result in criminal or civil penalties or remedial measures, any of which could have a material adverse effect on our business, financial condition and results of operations and could cause the market value of our common stock to decline.

The federal Health Insurance Portability and Accountability Act of 1996 ("HIPAA"), created new federal criminal statutes that prohibit, among other actions, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program, including private third-party payers, and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services. Like the federal Anti-Kickback Statute, the PPACA amended the intent standard for certain healthcare fraud provisions under HIPAA such that a person or entity no longer needs to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation.

Additionally, the federal Physician Payments Sunshine Act within the PPACA, and its implementing regulations, require that certain manufacturers of drugs, devices, biologicals and medical supplies to report annually information related to certain payments or other transfers of value made or distributed to physicians (defined to include doctors, dentists, optometrists, podiatrists, and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, or to entities or individuals at the request of, or designated on behalf of, physicians and teaching hospitals and to report annually certain ownership and investment interests held by physicians and their immediate family members.

Moreover, the Drug Supply Chain Security Act imposes obligations on manufacturers of pharmaceutical products related to product tracking and tracing. We are not sure whether additional legislative changes will be enacted, or whether the current regulations, guidance or interpretations will be changed, or what the impact of such changes on our business, if any, may be.

Also, many states have similar fraud and abuse statutes or regulations, including state anti-kickback and false claims laws, that apply to items and services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payer. Further, certain states require implementation of

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commercial compliance programs and marketing codes, compliance with the pharmaceutical industry’s voluntary compliance guidelines, and compliance with the applicable compliance guidance promulgated by the federal government. Other various state level requirements include restricting payments or the provision of other items of value that may be made to healthcare providers and other potential referral sources; restricting various marketing practices; requiring prescription drug companies to report expenses relating to the marketing and promotion of drug products; requiring the posting of information relating to clinical studies and their outcomes; requiring the registration of sales representatives; requiring the reporting of certain information related to drug pricing; and requiring drug manufacturers to track and report information related to payments, gifts, compensation, and other items of value to physicians and other healthcare providers.

In February 2021, we entered into a limited co-promotion arrangement with Albireo Pharma, Inc. ("Albireo"), providing for our Cholbam dedicated sales representatives to dedicate a portion of their efforts to promoting Albireo's product, Bylvay (odevixibat), in the United States following approval. In July 2021, Albireo announced that the U.S. Food & Drug Administration ("FDA") has approved Bylvay (odevixibat) for the treatment of pruritus in patients with Progressive Familial Intrahepatic Cholestasis ("PFIC"). The limited co-promotion agreement terminated in July 2022, in accordance with our mutual agreement with Albireo to terminate the agreement upon the one-year anniversary of the July 2021 launch. Nonetheless, if our activities in connection with promoting these products violated or were perceived to have violated any applicable regulatory requirements, we could become subject to investigations, litigation, and/or penalties as described above, and reputational harm, any of which could have a material adverse effect on our business.

If we are not able to obtain and maintain required regulatory approvals, we will not be able to commercialize our products, and our ability to generate revenue will be materially impaired.

Our product candidates, once approved, and the activities associated with their manufacture, marketing, distribution, and sales are subject to extensive regulation by the FDA and other regulatory agencies in the United States and by comparable authorities in other countries. Failure to adhere to regulations set out by these bodies for one or more of our commercial products could prevent us from commercializing the product candidate in the jurisdiction of the regulatory authority. We have only limited experience in meeting the regulatory requirements incumbent on the sale of drugs in the United States and elsewhere, and expect to rely on third-parties to assist us in these processes. If these third parties fail to adequately adhere to the regulations governing drug distribution and promotion we may be unable to sell our products, which could have a material effect on our ability to generate revenue.

Our product candidates and the activities associated with their development and commercialization, including testing, manufacture, safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale and distribution, are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and by comparable authorities in other countries. Failure to obtain regulatory approval for a product candidate will prevent us from commercializing the product candidate in the jurisdiction of the regulatory authority. We have only limited experience in filing and prosecuting the applications necessary to obtain regulatory approvals and expect to rely on third-party contract research organizations to assist us in this process.

Securing FDA approval requires the submission of extensive nonclinical and clinical data and supporting information to the FDA for each therapeutic indication to establish the product candidate’s safety and efficacy. Securing FDA approval also requires the submission of information about the product manufacturing process to, and successful inspection of manufacturing facilities by, the FDA. Our future products may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude our obtaining regulatory approval or prevent or limit commercial use.

Our product candidates may fail to obtain regulatory approval for many reasons, including:

•our failure to demonstrate to the satisfaction of the FDA or comparable regulatory authorities that a product candidate is safe and effective for a particular indication;

•the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable regulatory authorities for approval;

•our inability to demonstrate that a product candidate’s benefits outweigh its risks;

•our inability to demonstrate that the product candidate presents an advantage over existing therapies;

•the FDA’s or comparable regulatory authorities’ disagreement with the manner in which we interpret the data from nonclinical studies or clinical trials;

•failure of the third-party manufacturers with which we contract for clinical or commercial supplies to satisfactorily complete an FDA pre-approval inspection of the facility or facilities at which the product is manufactured to assess compliance with the FDA’s cGMP regulations to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity; and

•a change in the approval policies or regulations of the FDA or comparable regulatory authorities or a change in the laws governing the approval process.

The process of obtaining regulatory approvals is expensive, often takes many years, if approval is obtained at all, and can vary substantially based upon a variety of factors, including the type, complexity and novelty of the product candidates involved. Changes in regulatory approval policies during the development period, changes in or the enactment of additional statutes or regulations, or changes in regulatory review for each submitted product application may cause delays in the approval or rejection of an application. The FDA and non-United States regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data is insufficient for approval and require additional nonclinical, clinical or other studies. In addition, varying interpretations of the data obtained from nonclinical and clinical testing could delay, limit or prevent regulatory approval of a product candidate. Any regulatory approval we ultimately obtain may be limited or subject to restrictions or post approval commitments that render the approved product not commercially viable. Any FDA or other regulatory approval of our product candidates, once obtained, may be withdrawn, including for failure to comply with regulatory requirements or if clinical or manufacturing problems follow initial marketing.

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We are subject to stringent and changing U.S. and foreign laws, regulations, rules, contractual obligations, policies and other obligations related to data privacy and security. Our actual or perceived failure to comply with such obligations could lead to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse business consequences.*

In the ordinary course of business, we collect, receive, store, process, generate, use, transfer, disclose, make accessible, protect, secure, dispose of, transmit, and share (collectively, process) personal data and other sensitive information, including proprietary and confidential business data, trade secrets, intellectual property, data we collect about trial participants in connection with clinical trials, and sensitive third-party data. Our data processing activities may subject us to numerous data privacy and security obligations, such as various laws, regulations, guidance, industry standards, external and internal privacy and security policies, contracts, and other obligations that govern the processing of personal data by us and on our behalf.

In the United States, federal, state, and local governments have enacted numerous data privacy and security laws, including data breach notification laws, personal data privacy laws, consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act) and other similar laws (e.g., wiretapping laws). For example, HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act ("HITECH"), and their respective implementing regulations, imposes specific requirements relating to the privacy, security and transmission of individually identifiable health information. Among other things, HITECH, through its implementing regulations, makes certain of HIPAA’s privacy and security standards directly applicable to business associates, defined as a person or organization, other than a member of a covered entity’s workforce, that creates, receives, maintains or transmits protected health information for or on behalf of a covered entity for a function or activity regulated by HIPAA as well as their covered subcontractors. In addition, the California Consumer Privacy Act of 2018 (“CCPA”) applies to personal information of consumers, business representatives, and employees who are California residents, and requires businesses to provide specific disclosures in privacy notices, and afford California residents certain privacy rights related to their personal data. The CCPA allows for administrative fines for noncompliance (up to $7,500 per violation) and allows private litigants affected by certain data breaches to recover significant statutory damages. Although the CCPA exempts some data processed in the context of clinical trials, the CCPA increases compliance costs and potential liability with respect to other personal data we maintain about California residents. In addition, the California Privacy Rights Act of 2020 (“CPRA”), operative on January 1, 2023, expanded the CCPA's requirements, including by adding a new right for individuals to correct their personal information and establishing a new California Privacy Protection Agency to implement and enforce the CCPA (as amended) . Other states have enacted comprehensive data privacy laws, including Virginia and Colorado, both of which differ from the CPRA and became effective in 2023. Additional data privacy and security legislation has been proposed at the federal, state, and local levels in recent years. While these states, like the CCPA, also exempt some data processed in the context of clinical trials, such laws could increase our potential liability, increase compliance costs, or adversely affect our business.

Outside the United States, an increasing number of laws, regulations, and industry standards apply to data privacy and security. For example, the European Union’s General Data Protection Regulation (“EU GDPR”), the United Kingdom’s GDPR (“UK GDPR”), Brazil’s General Data Protection Law (Lei Geral de Proteção de Dados Pessoais, or “LGPD”) (Law No. 13,709/2018), and China’s Personal Information Protection Law (“PIPL”) impose strict requirements for processing personal data. For example, the EU GDPR imposes significant and complex burdens on processing personal data, particularly for processing “special category personal data” (such as personal data related to health and genetic information), which could be relevant to our operations in the context of our conduct of clinical trials and is of interest to relevant regulators. Under the EU GDPR, government regulators may impose temporary or definitive bans on data processing, as well as fines of up to 20 million euros under the EU GDPR, 17.5 million pounds sterling under the UK GDPR or, in each case, or 4% of annual global revenue, whichever is greater. Further, under the EU GDPR, individuals may initiate litigation related to processing of their personal data, as well as consumer protection organizations authorized at law to represent data subjects' interests.

In addition, privacy advocates and industry groups around the world have proposed, and may propose, standards with which we are legally or contractually bound to comply, or may become subject to in the future. We are also bound by contractual obligations related to data privacy and security, and our efforts to comply with such obligations may not be successful. Additionally, we publish privacy policies, marketing materials and other statements, such as compliance with certain certifications, regarding data privacy and security. If these policies, materials or statements are found to be deficient, lacking in transparency, deceptive, unfair, or misrepresentative of our practices, we may be subject to investigation, enforcement actions by regulators or other adverse consequences.

In the ordinary course of business, we may transfer personal data from Europe and other jurisdictions to the United States or other countries. Europe and other jurisdictions have enacted laws requiring data to be localized or limiting the transfer of personal data to other countries. In particular, the European Economic Area (EEA) and the United Kingdom (UK) have significantly restricted the transfer of personal data to the United States and other countries whose privacy laws it generally believes are inadequate. Other jurisdictions may adopt similarly stringent interpretations of their data localization and cross-border transfer laws, which could make it more difficult to transfer information across jurisdictions or prevent us from conducting business in certain countries. Although there are currently various mechanisms that may be used to transfer personal data from the EEA and UK to the United States in compliance with law, such as the EEA and UK’s standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S. Data Privacy Framework (which allows for transfers for relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges, and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal data to the United States.

If we are unable to implement a valid compliance mechanism for cross-border personal data transfers, or if the requirements for a legally-compliant transfer are too onerous, we may face significant adverse consequences, including increased exposure to regulatory actions, substantial fines and injunctions against processing or transferring personal data from Europe. Inability to import personal data from Europe to the United States may significantly and negatively impact our business operations, including by limiting our ability to conduct clinical trial activities in Europe and elsewhere; limiting our ability to collaborate with CROs, service providers, contractors and other companies that are subject to such cross-border data transfer or localization laws; the need to relocate part of or all of our business or data processing activities to other jurisdictions (such as Europe) at significant expense; or requiring us to increase our personal data processing capabilities and infrastructure in foreign jurisdictions at significant expense. Additionally, companies that transfer personal data out of the EEA and UK to other jurisdictions, particularly to the United States, are subject to increased scrutiny from regulators, individual litigants, and activities groups. Some European regulators have ordered certain companies to suspend or permanently cease certain transfers out of Europe for allegedly violating the GDPR’s cross-border data transfer limitations. For example, in May 2023, the Irish Data Protection Commission determined that a major social media

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company’s use of the standard contractual clauses to transfer personal data from Europe to the United States was insufficient and levied a 1.2 billion Euro fine against the company and prohibited the company from transferring personal data to the United States.

Our obligations related to data privacy and security are quickly changing in an increasingly stringent fashion, creating regulatory uncertainty. Additionally, these obligations may be subject to differing applications and interpretations, which may be inconsistent or conflict among jurisdictions. Preparing for and complying with these obligations requires significant resources and may necessitate changes to our information technologies, systems, and practices and to those of any third parties that process personal data on our behalf. Although we endeavor to comply with all applicable data privacy and security obligations, we may at times fail (or be perceived to have failed) to do so. Moreover, despite our efforts, our personnel or third parties upon whom we rely may fail to comply with such obligations, which could negatively impact our business operations and compliance posture. For example, any failure by a third-party service provider to comply with applicable law, regulations, or contractual obligations could result in adverse effects, including proceedings against us by governmental entities or others. If we or any of our partners fail to comply or are perceived to have failed to comply with applicable obligations, we or they could be subject to a range of regulatory actions, litigation (including class actions), or mass arbitration demands that could affect our or our partners' ability to commercialize our products and conduct necessary research and development, and could harm or prevent sales of the affected products, or could substantially increase the costs and expenses of commercializing and marketing our products. In particular, plaintiffs have become increasingly more active in bringing privacy-related claims against companies, including class claims and mass arbitration demands. Some of these claims allow for the recovery of statutory damages on a per violation basis, and, if viable, carry the potential for monumental statutory damages, depending on the volume of data and the number of violations. Any threatened or actual government enforcement action or litigation could also generate adverse publicity and require that we devote substantial resources that could otherwise be used in other aspects of our business. Compliance with applicable federal, state, and foreign laws is difficult and time consuming, and companies that violate them may face substantial penalties. The potential sanctions include significant criminal fines, civil monetary penalties, administrative penalties, disgorgement, exclusion from participation in federal health care programs, individual imprisonment, injunctions, recall or seizure of products, total or partial suspension of production, reputational harm, administrative burdens, interruption or cessation of clinical trials, additional oversight and reporting obligations if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of non-compliance with these laws, diminished profits and future earnings, and the curtailment or restructuring of our operations, and other sanctions. Because of the breadth of these laws, it is possible that some of our business activities could be subject to challenge under one or more of these laws. Such a challenge, irrespective of the underlying merits of the challenge or the ultimate outcome of the matter, could have a material adverse effect on our business, financial condition, results of operations and growth prospects.

If our information technology systems or data, or those of third parties upon which we rely, are or were compromised, we could experience adverse impacts resulting from such compromise, including, but not limited to, regulatory investigations or actions; litigation; fines and penalties; interruptions to our operations such as clinical trials; harm to our reputation; loss of revenue or profits; loss of sales and other adverse consequences.*

In the ordinary course of our business, we and our third-party service providers may process proprietary, confidential, and sensitive data, including personal data (such as health-related data and data related to our clinical trials), intellectual property, and trade secrets (collectively, sensitive information).

Cyberattacks, malicious internet-based activity, and online and offline fraud are prevalent and continue to increase. These threats are becoming increasingly difficult to detect. These threats come from a variety of sources, including traditional computer “hackers,” threat actors, personnel (such as through theft or misuse), "hacktivists", organized criminal threat actors, sophisticated nation-states, and nation-state-supported actors. Some actors now engage and are expected to continue to engage in cyberattacks, including without limitation nation-state actors for geopolitical reasons and in conjunction with military conflicts and defense activities. During times of war and other major conflicts, including the war in Ukraine, we and the third parties upon which we rely may be vulnerable to a heightened risk of these attacks, including retaliatory cyberattacks that could materially disrupt our systems and operations, supply chain, and ability to produce, sell and distribute our products and services. We and the third parties upon which we rely may be subject to a variety of other evolving threats, including, but not limited to, social-engineering attacks (including through deep fakes, which may be increasingly more difficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks (such as credential stuffing), credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or hardware failures, loss of data or other information technology assets, adware, telecommunications failures, and other similar threats. In particular, ransomware attacks, including those from organized criminal threat actors, nation-states and nation-state supported actors, are becoming increasingly prevalent and severe and can lead to significant interruptions, delays, or outages in our operations, disruption of clinical trials, loss of data (including data related to clinical trials), loss of income, significant extra expenses to restore data or systems, reputational loss and the diversion of funds. To alleviate the financial, operational and reputational impact of a ransomware attack, it may be preferable to make extortion payments, but we may be unwilling or unable to do so (including, for example, if applicable laws prohibit such payments). Additionally, remote work has become more common and has increased risks to our information technology systems and data, as more of our employees utilize network connections, computers, and devices outside our premises or network, including working at home, while in transit, and in public locations. Future or past business transactions (such as acquisitions or integrations) could also expose us to additional cybersecurity risks and vulnerabilities, as our systems could be negatively affected by vulnerabilities present in acquired or integrated entities’ systems and technologies. Furthermore, we may discover security issues that were not found during due diligence of such acquired or integrated entities, and it may be difficult to integrate companies into our information technology environment and security program.

We rely upon third-party service providers and technologies to operate critical business systems to process sensitive information in a variety of contexts, including, without limitation, third-party providers of cloud-based infrastructure, encryption and authentication technology, employee email, and other functions. We also rely on third-party service providers to provide certain products, including active pharmaceutical ingredients, to operate our business, including in China. Our ability to monitor these third parties’ information security practices is limited, and these third parties may not have adequate information security measures in place. While we may be entitled to damages if our third-party service providers fail to satisfy their privacy or security-related obligations to us, any award may be insufficient to cover our damages, or we may be unable to recover such award. In addition, supply-chain attacks have increased in frequency and severity, and we cannot guarantee that third parties’ infrastructure in our supply chain or our third-party partners’ supply chains have not been compromised. We may share or receive sensitive information with or from third parties.

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Any of the previously identified or similar threats could cause a security incident or other interruption that could result in unauthorized, unlawful, or accidental acquisition, modification, destruction, loss, alteration, encryption, disclosure of, or access to our sensitive information or our information technology systems, or those of the third parties upon whom we rely. A security incident or other interruption could disrupt our ability (and that of third parties upon whom we rely) to provide our products and services. We may expend significant resources or modify our business activities (including our clinical trial activities) to try to protect against security incidents. Certain data privacy and security obligations require us to implement and maintain specific security measures, industry-standard or reasonable security measures to protect our information technology systems and sensitive information.

While we have implemented security measures designed to protect against security incidents, there can be no assurance that these measures will be effective. We take steps to detect and remediate vulnerabilities, but we may not be able to detect and remediate all vulnerabilities because the threats and techniques used to exploit the vulnerability change frequently and are often sophisticated in nature. Therefore, such vulnerabilities could be exploited but may not be detected until after a security incident has occurred. These vulnerabilities pose material risks to our business. Despite our efforts to identify and remediate vulnerabilities, if any, in our information technology systems, our efforts may not be successful and could result in a material disruption of our programs and operations. For example, the loss of clinical trial data from completed or ongoing clinical trials for any of our product candidates could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. Further, we may experience delays in developing and deploying remedial measures designed to address any such identified vulnerabilities.

Applicable data security obligations may require us to notify relevant stakeholders of any security incidents, including affected individuals, customers, and regulators. Such disclosures are costly, and the disclosures or the failure to comply with such requirements, could lead to adverse consequences. If we (or a third party upon whom we rely) experience a security incident or are perceived to have experienced a security incident, we may experience adverse consequences. These consequences may include: government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); additional reporting requirements and/or oversight; restrictions on processing sensitive information (including personal data); litigation (including class claims); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; interruptions in our operations (including availability of data); financial loss; and other similar harms. Security incidents and attendant consequences may cause customers to stop using our products or services, deter new customers from using our products or services, and negatively impact our ability to grow and operate our business.

Our contracts may not contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in our contracts are sufficient to protect us from liabilities, damages, or claims related to our data privacy and security obligations. In addition, our insurance coverage may not be adequate or sufficient to protect us from or to mitigate liabilities arising out of our privacy and security practices or that such coverage will continue to be available on commercially reasonable terms or at all, or that such coverage will pay future claims.

In addition to experiencing a security incident, third parties may gather, collect, or infer sensitive information about us from public sources, data brokers, or other means that reveals competitively sensitive details about our organization and could be used to undermine our competitive advantage or market position.

Uncertainties in the interpretation and application of existing, new and proposed tax laws and regulations could materially affect our tax obligations and effective tax rate.

The tax regimes to which we are subject or under which we operate are unsettled and may be subject to significant change. The issuance of additional guidance related to existing or future tax laws, or changes to tax laws or regulations proposed or implemented by the current or a future U.S. presidential administration, Congress, or taxing authorities in other jurisdictions, including jurisdictions outside of the United States, could materially affect our tax obligations and effective tax rate. To the extent that such changes have a negative impact on us, including as a result of related uncertainty, these changes may adversely impact our business, financial condition, results of operations, and cash flows.

The amount of taxes we pay in different jurisdictions depends on the application of the tax laws of various jurisdictions, including the United States, to our international business activities, tax rates, new or revised tax laws, or interpretations of tax laws and policies, and our ability to operate our business in a manner consistent with our corporate structure and intercompany arrangements. The taxing authorities of the jurisdictions in which we operate may challenge our methodologies for pricing intercompany transactions pursuant to our intercompany arrangements or disagree with our determinations as to the income and expenses attributable to specific jurisdictions. If such a challenge or disagreement were to occur, and our position was not sustained, we could be required to pay additional taxes, interest, and penalties, which could result in one-time tax charges, higher effective tax rates, reduced cash flows, and lower overall profitability of our operations. Our financial statements could fail to reflect adequate reserves to cover such a contingency. Similarly, a taxing authority could assert that we are subject to tax in a jurisdiction where we believe we have not established a taxable connection, often referred to as a “permanent establishment” under international tax treaties, and such an assertion, if successful, could increase our expected tax liability in one or more jurisdictions.

Effective January 1, 2022, legislation enacted in 2017, informally titled the Tax Cuts and Jobs Act eliminated the option to deduct research and development expenses for tax purposes in the year incurred and requires taxpayers to capitalize and subsequently amortize such expenses over five years for research activities conducted in the United States and over 15 years of research activities conducted outside the United States. Unless the United States Department of the Treasury issues regulations that narrow the application of this provision to a smaller subset of our research and development expenses or the provision is deferred, modified, or repealed by Congress, in future years we may experience a material decrease in our cash flows from operations and an offsetting similarly sized increase in our net deferred tax assets over these amortization periods. The actual impact of this provision will depend on multiple factors, including the amount of research and development expenses we will incur and whether we conduct our research and development activities inside or outside the United States and our overall net operating loss position.

Our ability to use net operating loss carryforwards and certain other tax attributes to offset future taxable income and taxes may be subject to limitations.

Under current law, our federal net operating losses ("NOLs") generated in tax years beginning after December 31, 2017, may be carried forward indefinitely, but the deductibility of such federal NOLs is limited to 80% of taxable income. As of December 31, 2022, we had federal NOLs of $90.2 million. It is uncertain if and to what extent various states will conform to federal tax laws. In addition, under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50%

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change, by value, in its equity ownership over a three-year period, the corporation’s ability to use its pre-change NOL carryforwards and other pre-change U.S. tax attributes (such as research tax credits) to offset its post-change income or taxes may be limited. It is possible that we have experienced an ownership change in the past. In addition, we may experience ownership changes in the future as a result of subsequent shifts in our stock ownership, some of which may be outside of our control.

As a result, our federal NOL carryforwards may be subject to a percentage limitation if used to offset income in tax years following an ownership change. In addition, it is possible that we have in the past undergone, and in the future may undergo, additional ownership changes that could limit our ability to use all of our pre-change NOL carryforwards and other pre-change tax attributes (such as research tax credits) to offset our post-change income or taxes. Similar provisions of state tax law may also apply to limit our use of accumulated state tax attributes. In addition, at the state level, there may be periods during which the use of NOL carryforwards is suspended or otherwise limited, which could accelerate or permanently increase state taxes owed. As a result, we may be unable to use all or a material portion of our NOL carryforwards and other tax attributes, which would harm our future operating results by effectively increasing our future tax obligations.

Changes in funding for the FDA, the SEC and other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal functions on which the operation of our business may rely, which could negatively impact our business.

The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept payment of user fees, and statutory, regulatory, and policy changes. Average review times at the agency have fluctuated in recent years as a result. In addition, government funding of the SEC and other government agencies on which our operations may rely, including those that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable.

Disruptions at the FDA and other agencies may also slow the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business. For example, over the last several years, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities. If a prolonged government shutdown occurs, or if the FDA or EDA experience resource constraints, it could significantly impact the ability of the applicable regulatory agency to timely review and process our regulatory submissions, which could have a material adverse effect on our business. Further, future government shutdowns could impact our ability to access the public markets and obtain necessary capital in order to properly capitalize and continue our operations.

Our business could be negatively impacted by environmental, social and corporate governance (ESG) matters or our reporting of such matters.

There is an increasing focus from certain investors, employees, partners, and other stakeholders concerning ESG matters. While we have internal efforts directed at ESG matters and preparations for increased future disclosures, we may be perceived by certain stakeholders as not acting responsibly in connection with these matters, which could negatively impact us. Moreover, the SEC has recently proposed, and may continue to propose, certain mandated ESG reporting requirements, such as the SEC's proposed rules designed to enhance and standardize climate-related disclosures, which, if finally approved, would significantly increase our compliance and reporting costs and may also result in disclosures that certain investors or other stakeholders deem to negatively impact our reputation and/or that harm our stock price. In addition, given our business model, we currently do not report our environmental emissions and absent a legal requirement to do so we currently do not plan to report our environmental emissions, and lack of reporting could result in certain investors declining to invest in our common stock.

The withdrawal of the United Kingdom from the European Union, commonly referred to as “Brexit,” may adversely impact our ability to obtain regulatory approvals of our product candidates in the United Kingdom, result in restrictions or imposition of taxes and duties for importing our product candidates into the United Kingdom, and may require us to incur additional expenses in order to develop, manufacture and commercialize our product candidates in the United Kingdom.*

Following the result of a referendum in 2016, the United Kingdom left the European Union on January 31, 2020, commonly referred to as “Brexit.” A trade and cooperation agreement, or the Trade and Cooperation Agreement, that outlines the future trading relationship between the United Kingdom and the European Union was agreed in December 2020 and has been in effect since January 1, 2022.

Since a significant proportion of the regulatory framework in the United Kingdom applicable to our business and our product candidates is derived from EU directives and regulations, Brexit has had, and may continue to have, a material impact on the regulatory regime with respect to the development, manufacture, importation, approval and commercialization of our product candidates in the United Kingdom or, to the extent any development of our product candidates takes place in the United Kingdom, the European Union. For example, Great Britain is no longer covered by the centralized procedures for obtaining EU-wide marketing authorization from the EMA, and a separate marketing authorization will be required to market our product candidates in Great Britain. Centralized marketing authorizations continue to allow marketing in Northern Ireland.

While the Trade and Cooperation Agreement provides for the tariff-free trade of medicinal products between the United Kingdom and the European Union there are additional non-tariff costs which did not exist prior to the end of the Transition Period. Further, should the United Kingdom further diverge from the European Union from a regulatory perspective in relation to medicinal products, tariffs could be put into place in the future. We could therefore, both now and in the future, face significant additional expenses (when compared to the position prior to the end of the Transition Period) to operate our business, which could significantly and materially harm or delay our ability to generate revenues or achieve profitability of our business. Any further changes in international trade, tariff and import/export regulations as a result of Brexit or otherwise may impose unexpected duty costs or other non-tariff barriers on us. These developments, or the perception that any of them could occur, may significantly reduce global trade and, in particular, trade between the European Union and the United Kingdom.

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Business disruptions could seriously harm our future revenue and financial condition and increase our costs and expenses.

Our operations, and those of our third-party manufacturers, CROs and other contractors and consultants, could be subject to disruptions resulting from earthquakes, power shortages, telecommunications failures, water shortages, floods, hurricanes, typhoons, fires, extreme weather conditions, health epidemics or pandemics, wars and other geopolitical conflicts (including related to Russia's invasion of Ukraine), and other natural or man-made disasters or business interruptions, for which we are predominantly self-insured. The occurrence of any of these business disruptions could seriously harm our operations and financial condition and increase our costs and expenses.

Our corporate headquarters are located in San Diego, California, an area prone to wildfires and earthquakes. These and other natural disasters could severely disrupt our operations, and have a material adverse effect on our business, results of operations, financial condition and prospects. If a natural disaster, power outage or other event occurred that prevented us from using all or a significant portion of our headquarters, that damaged critical infrastructure, such as the manufacturing facilities of our third-party contract manufacturers, or that otherwise disrupted operations, it may be difficult or, in certain cases, impossible for us to continue our business for a substantial period of time. Any disaster recovery and business continuity plans we have in place may prove inadequate in the event of a serious disaster or similar event. We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which, could have a material adverse effect on our business.

In addition, we rely on third-party manufacturers, some of whom are located in China, to manufacture API for FILSPARI and certain of our product candidates. Any disruption in production or inability of our manufacturers in China to produce or ship adequate quantities to meet our needs, whether as a result of a natural disaster or other causes (such as staffing shortages, COVID-19 or other health epidemic or pandemic), could impair our ability to meet commercial demand for FILSPARI, to operate our business on a day-to-day basis and to continue our research and development of our product candidates. In addition, we are exposed to the possibility of product supply disruption and increased costs in the event of changes in the policies of the United States or Chinese governments (such as tariffs on chemical intermediates we use that are manufactured in China), political unrest or unstable economic conditions in China. Any recall of the manufacturing lots or similar action regarding our API used in clinical trials could delay the trials or detract from the integrity of the trial data and its potential use in future regulatory filings. In addition, manufacturing interruptions or failure to comply with regulatory requirements by any of these manufacturers could significantly delay clinical development of potential products and reduce third-party or clinical researcher interest and support of proposed trials. These interruptions or failures could also impede commercialization of our product candidates and impair our competitive position.

We have identified a material weakness in our internal control over financial reporting, and our financial controls and procedures may not in the future be sufficient to ensure timely and reliable reporting of financial information, which could, if not remediated, result in a material misstatement in our financial statements and could adversely affect our future results of operations and our stock price.*

A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial reporting such that there is a reasonable possibility that a material misstatement of our annual or interim consolidated financial statements will not be prevented or detected on a timely basis.

As disclosed under Item 9A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2022, there was a material weakness in our internal control over financial reporting as of December 31, 2022 because we did not design effective controls and procedures to evaluate the accounting for a certain pre-launch inventory contract affecting the timing of recognition of research and development expense.

As a result of the material weakness, we added controls for the timely accounting evaluation of research and development contracts that are intended to ensure appropriate expense recognition of certain pre-launch inventory. We do not believe that any of our remedial controls have been fully implemented or operated for a sufficient period of time or number of occurrences to allow for sufficient testing to determine the controls’ operating effectiveness. If we are unable to remediate this material weakness, or are otherwise unable to maintain effective internal control over financial reporting or disclosure controls and procedures, our ability to record, process and report financial information accurately, and to prepare financial statements within required time periods, could be adversely affected, which could subject us to litigation or investigations requiring management resources and payment of legal and other expenses and negatively impact the price of our common stock. In addition, we could be subject to sanctions or investigations by the SEC or other regulatory authorities, which would require additional financial and management resources.

Furthermore, investor perceptions of our company may suffer as a result of the current material weakness or any future material weakness in our internal controls, and this could cause a decline in the market price of our stock. Any failure of our internal control over financial reporting could have a material adverse effect on our stated operating results, result in an adverse opinion on our internal control over financial reporting from our independent registered public accounting firm, and harm our reputation.

Adverse developments affecting the financial services industry could adversely affect our current and projected business operations and our financial condition and results of operations.*

Adverse developments that affect financial institutions, such as events involving liquidity that are rumored or actual, have in the past and may in the future lead to bank failures and market-wide liquidity problems. For example, on March 10, 2023, Silicon Valley Bank (SVB) was closed by the California Department of Financial Protection and Innovation, which appointed the Federal Deposit Insurance Corporation (FDIC) as receiver. Similarly, on March 12, 2023, Signature Bank and Silvergate Capital Corp. were each swept into receivership. In addition, on May 1, 2023, the FDIC seized First Republic Bank and sold its assets to JPMorgan Chase & Co. While the U.S. Department of Treasury, FDIC and Federal Reserve Board have implemented a program to provide up to $25 billion of loans to financial institutions secured by certain of such government securities held by financial institutions to mitigate the risk of potential losses on the sale of such instruments, widespread demands for customer withdrawals or other needs of financial institutions for immediate liquidity may exceed the capacity of such program, there is no guarantee that such programs will be sufficient. Additionally, it is uncertain whether the U.S. Department of Treasury, FDIC and Federal Reserve Board will provide access to uninsured funds in the future in the event of the closure of other banks or financial institutions, or that they would do so in a timely fashion.

While we have not experienced any adverse impact to our liquidity or to our current and projected business operations, financial condition or results of operations as a result of the matters relating to SVB, Signature Bank, Silvergate Capital Corp and First Republic Bank, uncertainty remains over liquidity

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concerns in the broader financial services industry, and our business, our business partners or industry as a whole may be adversely impacted in ways that we cannot predict at this time.

Although we assess our banking relationships as we believe necessary or appropriate, our access to cash in amounts adequate to finance or capitalize our current and projected future business operations could be significantly impaired by factors that affect the financial institutions with which we have banking relationships. These factors could include, among others, events such as liquidity constraints or failures, the ability to perform obligations under various types of financial, credit or liquidity agreements or arrangements, disruptions or instability in the financial services industry or financial markets, or concerns or negative expectations about the prospects for companies in the financial services industry. These factors could also include factors involving financial markets or the financial services industry generally. The results of events or concerns that involve one or more of these factors could include a variety of material and adverse impacts on our current and projected business operations and our financial condition and results of operations. These could include, but may not be limited to, delayed access to deposits or other financial assets or the uninsured loss of deposits or other financial assets; or termination of cash management arrangements and/or delays in accessing or actual loss of funds subject to cash management arrangements.

In addition, widespread investor concerns regarding the U.S. or international financial systems could result in less favorable commercial financing terms, including higher interest rates or costs and tighter financial and operating covenants, or systemic limitations on access to credit and liquidity sources, thereby making it more difficult for us to acquire financing on acceptable terms or at all. Any decline in available funding or access to our cash and liquidity resources could, among other risks, adversely impact our ability to meet our operating expenses, financial obligations or fulfill our other obligations, result in breaches of our financial and/or contractual obligations or result in violations of federal or state wage and hour laws. Any of these impacts, or any other impacts resulting from the factors described above or other related or similar factors not described above, could have material adverse impacts on our liquidity and our current and/or projected business operations and financial condition and results of operations.

We maintain our cash at financial institutions, often in balances that exceed federally insured limits.*

We maintain the majority of our cash and cash equivalents in accounts at banking institutions in the United States that we believe are of high quality. Cash held in these accounts often exceed the FDIC insurance limits. If such banking institutions were to fail, we could lose all or a portion of amounts held in excess of such insurance limitations. As noted above, the FDIC recently took control of SVB, Signature Bank, Silvergate Capital Corp and First Republic Bank. In the event of failure of any of the financial institutions where we maintain our cash and cash equivalents, there can be no assurance that we would be able to access uninsured funds in a timely manner or at all. Any inability to access or delay in accessing these funds could adversely affect our business and financial position.

Risks Related to our Indebtedness and Investments

Our indebtedness could adversely affect our financial condition.*

As of June 30, 2023, we had approximately $385 million of total debt outstanding, classified as long term. As a result of our indebtedness, a portion of our cash flow will be required to pay interest and principal on the 2025 Notes and 2029 Notes if the notes are not converted to shares of common stock prior to maturity. We may not generate sufficient cash flow from operations or have future borrowings available to enable us to repay our indebtedness or to fund other liquidity needs.

Our indebtedness pursuant to the 2025 Notes and 2029 Notes could have important consequences. For example, it could:

•make it more difficult for us to satisfy our obligations with respect to any other debt we may incur in the future;

•increase our vulnerability to general adverse economic and industry conditions;

•require us to dedicate a substantial portion of our cash flow from operations to payments on our indebtedness and related interest, thereby reducing the availability of our cash flow to fund working capital, capital expenditures and other general corporate purposes;

•limit our flexibility in planning for, or reacting to, changes in our business and the industry in which we operate;

•increase our cost of borrowing;

•place us at a competitive disadvantage compared to our competitors that may have less debt; and

•limit our ability to obtain additional financing for working capital, capital expenditures, acquisitions, debt service requirements or general corporate purposes.

We expect to use cash flow from operations and outside financings to meet our current and future financial obligations, including funding our operations, debt service and capital expenditures. Our ability to make these payments depends on our future performance, which will be affected by financial, business, economic and other factors, many of which we cannot control. Our business may not generate sufficient cash flow from operations in the future, which could result in our being unable to repay indebtedness, or to fund other liquidity needs. If we do not generate sufficient cash from operations, we may be forced to reduce or delay our business activities and capital expenditures, sell assets, obtain additional debt or equity capital or restructure or refinance all or a portion of our debt, including the 2025 Notes and 2029 Notes, on or before maturity. We cannot make any assurances that we will be able to accomplish any of these alternatives on terms acceptable to us, or at all. In addition, the terms of existing or future indebtedness may limit our ability to pursue any of these alternatives. In addition, we may from time to time seek to retire or purchase our outstanding debt, including the 2025 Notes or 2029 Notes, through cash purchases and/or exchanges for equity securities, in open market purchases, privately negotiated transactions or otherwise. Such repurchases or exchanges, if any, will depend on prevailing market conditions, our liquidity requirements, contractual restrictions, and other factors. The amounts involved in any such transactions, individually or in the aggregate, may be material. Further, any such purchases or exchanges may result in us acquiring and retiring a substantial amount of such indebtedness, which could impact the trading liquidity of such indebtedness.

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We may be unable to raise the funds necessary to repurchase the 2025 Notes and 2029 Notes for cash following a fundamental change, or to pay any cash amounts due upon conversion, and our future indebtedness may limit our ability to repurchase the 2025 Notes and 2029 Notes or pay cash upon their conversion.

Noteholders may require us to repurchase their 2025 Notes and 2029 Notes following a fundamental change at a cash repurchase price generally equal to the principal amount of the 2025 Notes and 2029 Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date. In addition, upon conversion, we would satisfy part or all of our conversion obligation in cash unless we elected to settle conversions solely in shares of our common stock.

We may not have enough available cash or be able to obtain financing at the time we are required to repurchase the 2025 Notes and 2029 Notes or pay the cash amounts due upon conversion of the 2025 Notes and 2029 Notes. In addition, applicable law, regulatory authorities and the agreements governing our future indebtedness may restrict our ability to repurchase the 2025 Notes and 2029 Notes or pay the cash amounts due upon conversion of the 2025 Notes and 2029 Notes. Our failure to repurchase the 2025 Notes and 2029 Notes or to pay the cash amounts due upon conversion of the 2025 Notes and 2029 Notes when required will constitute a default under the base and supplemental indentures that govern the 2025 Notes and 2029 Notes, which we refer to collectively as the “indenture.” We may not have sufficient funds to satisfy all amounts due under the other indebtedness and the 2025 Notes and 2029 Notes.

A default under the 2025 Notes or 2029 Notes may have a material adverse effect on our financial condition.

If an event of default under the 2025 Notes or 2029 Notes occurs, the principal amount of the 2025 Notes or the 2029 Notes, as applicable, plus accrued and unpaid interest (including additional interest, if any) may be declared immediately due and payable, subject to certain conditions set forth in the indenture governing such notes. Events of default include, but are not limited to:

•failure to pay (for more than 30 days) interest when due;

•failure to pay principal when due;

•failure to deliver shares of common stock upon conversion of a 2025 Note or 2029 Note;

•failure to provide notice of a fundamental change;

•acceleration on our other indebtedness in excess of $10 million (other than indebtedness that is non-recourse to us); or

•certain types of bankruptcy or insolvency involving us.

Accordingly, the occurrence of a default under the 2025 Notes or 2029 Notes, unless cured or waived, may have a material adverse effect on our results of operations.

Provisions of the 2025 Notes and 2029 Notes could discourage an acquisition of us by a third party.

Certain provisions of the 2025 Notes and 2029 Notes could make it more difficult or more expensive for a third party to acquire us. Upon the occurrence of certain transactions constituting a fundamental change, holders of the 2025 Notes and 2029 Notes will have the right, at their option, to require us to repurchase all of their 2025 Notes and 2029 Notes or any portion of the principal amount of such Notes in integral multiples of $1,000. We may also be required to increase the conversion rate for conversions in connection with certain fundamental changes.

Conversion of the Notes may dilute the ownership interest of existing stockholders, including holders who had previously converted their 2025 Notes or 2029 Notes.

To the extent we issue shares of common stock upon conversion of the 2025 Notes or 2029 Notes, the conversion of some or all of the 2025 Notes or 2029 Notes will dilute the ownership interests of existing stockholders. Any sales in the public market of shares of the common stock issuable upon such conversion could adversely affect prevailing market prices of shares of our common stock. In addition, the existence of the 2025 Notes and 2029 Notes may encourage short selling by market participants because the conversion of the 2025 Notes and 2029 Notes could depress the price of shares of our common stock.

General Risk Factors

Unstable market, economic and geopolitical conditions may have serious adverse consequences on our business, financial condition and stock price.

The global credit and financial markets have experienced extreme volatility and disruptions, including as a result of inflation and rising interest rates, bank failures, COVID-19, Russia's invasion of Ukraine and global geopolitical tension, and may experience disruptions in the future. These disruptions can result in severely diminished liquidity and credit availability, increase in inflation, declines in consumer confidence, declines in economic growth, increases in unemployment rates and uncertainty about economic stability. There can be no assurance that further deterioration in credit and financial markets and confidence in economic conditions will not occur. Our general business strategy may be adversely affected by any such economic downturn, volatile business environment, higher inflation, or continued unpredictable and unstable market conditions. If the current equity and credit markets deteriorate, it may make any necessary debt or equity financing more difficult, more costly and more dilutive. Failure to secure any necessary financing in a timely manner and on favorable terms could have a material adverse effect on our operations, growth strategy, financial performance and stock price and could require us to delay or abandon clinical development plans. In addition, there is a risk that one or more of our current service providers, manufacturers and other partners may not survive an economic downturn or rising inflation, which could directly affect our ability to attain our operating goals on schedule and on budget.

Other international and geopolitical events could also have a serious adverse impact on our business. For instance, in February 2022, Russia initiated military action against Ukraine. In response, the United States and certain other countries imposed significant sanctions and trade actions against Russia and could

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impose further sanctions, trade restrictions, and other retaliatory actions. While we cannot predict the broader consequences, the conflict and retaliatory and counter-retaliatory actions could materially adversely affect global trade, currency exchange rates, inflation, regional economies, and the global economy, which in turn may increase our costs, disrupt our supply chain, impair our ability to raise or access additional capital when needed on acceptable terms, if at all, or otherwise adversely affect our business, financial condition, and results of operations.

Item 2.  Unregistered Sales of Equity Securities and Use of Proceeds

None.

Item 3.  Defaults Upon Senior Securities

None. 

Item 4.  Mine Safety Disclosures

Not applicable.

Item 5.  Other Information

Trading Arrangements

None of the Company’s directors or officers adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the quarter ended June 30, 2023.

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Item 6.  Exhibits

(a) Exhibits

3.1	Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.1 to Amendment No. 2 to the Company’s General Form for Registration of Securities on Form 10-12G, filed with the SEC on October 28, 2010).
3.2	Certificate of Amendment of Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K, filed with the SEC on June 11, 2015).
3.3	Certificate of Amendment of Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.2 to the Company's Current Report on Form 8-K, filed with the SEC on November 16, 2020).
3.4	Certificate of Amendment of Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K, filed with the SEC on May 18, 2021).
3.5	Amended and Restated Bylaws of the Company (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K, filed with the SEC on November 16, 2020).
3.6	Certificate of Amendment of Bylaws of the Company, effective June 9, 2021 (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K, filed with the SEC on June 10, 2021).
10.1†	The Company’s 2018 Equity Incentive Plan, as amended (incorporated by reference to Exhibit 99.1 to the Company’s Current Report on Form 8-K, filed with the SEC on May 22, 2023.
10.2*	Asset Purchase Agreement, dated January 10, 2015, by and between the Company and Asklepion Pharmaceuticals, LLC.
10.3*†	Asset Purchase Agreement, dated July 16, 2023, by and between Mirum Therapeutics, Inc. and the Company (incorporated by reference to Exhibit 10.1 to the Company’s Current Report on Form 8-K, filed with the SEC on July 17, 2023).
31.1	Chief Executive Officer's Certification pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
31.2	Chief Financial Officer's Certification pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
32.1	Chief Executive Officer’s Certification pursuant to Section 906 of the Sarbanes Oxley Act of 2002
32.2	Chief Financial Officer’s Certification pursuant to Section 906 of the Sarbanes Oxley Act of 2002
101.INS	Inline XBRL Instance Document
101.SCH	Inline XBRL Taxonomy Extension Schema Document
101.CAL	Inline XBRL Taxonomy Extension Calculation Linkbase Document
101.DEF	Inline XBRL Taxonomy Extension Definition Linkbase Document
101.LAB	Inline XBRL Taxonomy Extension Label Linkbase Document
101.PRE	Taxonomy Extension Presentation Linkbase Document
104	The cover page to this Quarterly Report on Form 10-Q has been formatted in Inline XBRL

*	Certain confidential information contained in this Exhibit, marked in brackets, has been omitted, because it is both not material and of the type of information that the registrant treats as private or confidential.
†	Schedules have been omitted pursuant to Item 601(a)(5) of Regulation S-K. The Company undertakes to furnish supplemental copies of any of the omitted schedules upon request by the SEC.

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SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.

Date: August 3, 2023	TRAVERE THERAPEUTICS, INC.
	By:	/s/ Eric M. Dube
		Name:	Eric M. Dube
		Title:	Chief Executive Officer
	By:	/s/ Christopher Cline
		Name:	Christopher Cline
		Title:	Chief Financial Officer

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EX-10.2

EXHIBIT 10.2

CERTAIN CONFIDENTIAL INFORMATION CONTAINED IN THIS DOCUMENT, MARKED BY [***], HAS BEEN OMITTED BECAUSE IT IS BOTH (I) NOT MATERIAL AND (II) IS THE TYPE OF INFORMATION THAT THE REGISTRANT TREATS AS PRIVATE OR CONFIDENTIAL.

ASSET PURCHASE AGREEMENT

THIS ASSET PURCHASE AGREEMENT (this “Agreement”) is made as of January 10, 2015 (the “Effective Date”), by and between Retrophin, Inc., a Delaware corporation (“Retrophin” or “Buyer”)) and Asklepion Pharmaceuticals, LLC, a Delaware limited liability company (“Asklepion” or “Seller”).  Buyer and Seller may be referred to herein collectively as the “Parties” and individually as a “Party.”

RECITALS

WHEREAS, Asklepion desires to sell, assign and convey all of its rights, interests and obligations in and to certain of its assets related to its Cholic Acid business, and Retrophin desires to purchase, assume and accept from Asklepion such rights, interests and obligations, all on the terms and subject to the conditions set forth in this Agreement.

NOW, THEREFORE, in consideration of these premises, the respective covenants of Buyer and Seller set forth below and other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, the parties agree as follows:

ARTICLE 1
DEFINITIONS

Definitions.  In addition to the other capitalized terms defined herein, the following capitalized terms shall have the following respective meanings:

1.1            “Act” means the United States Food, Drug and Cosmetic Act, as amended from time to time and the regulations promulgated thereunder.

1.2       “Affiliate” means, with respect to any Party, any Person that, directly or indirectly, controls, is controlled by, or is under common control with such Party at any time during the period for which the determination of affiliation is being made.  For the purposes of this definition, “control” (with correlative meanings for the terms “controlled by” and “under common control with”) means the possession by the applicable Person, directly or indirectly, of the power to direct or cause the direction of the management, policies and business affairs of a Person, whether through ownership of voting securities or general partnership or managing member interests, by contract or otherwise.

1.3          “Agency” means any governmental or regulatory authority having jurisdiction over the subject activities, products, and/or services.

1.4         “Anti-Corruption Laws” means the U.S. Foreign Corrupt Practices Act, as amended, the UK Bribery Act 2010, as amended, and any other applicable anti-corruption laws.

1.5         “Applicable Laws” means (i) all applicable laws, rules and regulations, including any rules, regulations, guidelines or other requirements of all Agencies, that may be in effect with respect to the subject activities, products and services, including the Act, and the PDMA, (ii) in the U.S., the American Medical Association Guidelines on Gifts to Physicians from

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Industry, and, outside the U.S., any foreign counterparts, (iii) in the U.S., the PhRMA Code on Interactions with Healthcare Professionals, and, outside the U.S., any foreign counterparts, and (iv) any requirement of action as directed by court order.

1.6           “Assigned Contracts” means the contracts between Asklepion and Third Parties for the continued development and commercialization of the Cholic Acid Product as set forth on “Assigned Contracts Schedule”.

1.7           “Assignment and Assumption Agreement” means the Assignment and Assumption Agreement between Buyer and Seller in the form to be mutually agreed upon by Buyer and Seller.

1.8          “Bill of Sale” means the Bill of Sale by Seller to Buyer in the form to be mutually agreed upon by Buyer and Seller.

1.9         “Business Day” means any day other than a Saturday, Sunday, or a day on which banking institutions in the State of New York are authorized or obligated by law or executive order to close.

1.10       “Chenodal” means chenodeoxycholic acid human pharmaceutical product sold by Retrophin or its licensee in the United States.

1.11         “Cholic Acid” means the primary bile acid 3α,7α,12α-trihydroxy-5β-cholan-24-oic acid as defined by the United States Adopted Names Council entry BC-106 and CAS Number 81-25-4.

1.12          “Cholic Acid Product” means (a) the pharmaceutical product being developed as of the Effective Date by Seller that has Cholic Acid as an “active pharmaceutical ingredient”, (b) any other product […***…] using Cholic Acid as an active ingredient or as one of two or more active ingredients of its “active pharmaceutical ingredients”, in each case (with respect to clauses (a) and (b)), including any additional indications and other product extensions, and (c) any […***…] in connection with the products described in the foregoing clauses (a) or (b), if such […***…] is based, in whole or in part, or used Cholic Acid Product IP or Cholic Acid Product Data Assets.

1.13        “Cholic AcidProduct Data Assets” means (a) any and all pre-clinical, clinical, chemical synthesis, manufacturing and testing data, protocols and other information, including chemical, pharmacological, toxicological, pharmaceutical, physical, analytical, safety, efficacy, bioequivalency, quality assurance, quality control and clinical data for the development and commercialization of the Cholic Acid Product, including its registration, formulation, manufacture, use, storage, transport, importation, sale, offer for sale, promotion and distribution; and, (b) all files, correspondence, records, laboratory notebooks, photographs, vendor and other audits, reports, documentation and other tangible embodiments (whether in writing, electronically stored or otherwise) related to the matters described in clause (i) above.

1.14         “Cholic Acid Product Inventory” means any and all inventory of the Cholic Acid Product, including work in process inventory and finished product of Cholic Acid Product as set forth on the “Cholic Acid Product Inventory Schedule”.

1.15          “Cholic Acid Product IP” means the Intellectual Property for the commercialization of the Cholic Acid Product (including, to the extent applicable, its registration, formulation, manufacture, use, storage, transport, offer for sale, sale, importation, promotion and distribution) and includes Copyrights and Cholic Acid Product Know-How, in each case, as set forth on the “Cholic Acid Product IP Schedule”.

1.16        “Cholic Acid Product Know-How” means any and all Know-How for the development and commercialization of the Cholic Acid Product (including, to the extent applicable, its registration, formulation, manufacture, use, storage, transport, offer for sale, sale, importation, promotion and distribution).

***Certain Confidential Information Omitted

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1.17         “Cholic Acid Product MSA” means thecentralized EU marketing authorization held by Seller’s wholly-owned subsidiary, ASK Pharmaceuticals GmbH Heimeranstraße 35, D-80339 München, Deutschland, relating to the product named “Kolbam” (previously Cholic Acid FGK) for the therapeutic areas of “Metabolism, Inborn Errors” and therapeutic indication of the treatment of inborn errors in primary bile acid synthesis due to Sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency, 2- (or a-) methylacyl-CoA racemase (AMACR) deficiency or Cholesterol 7a-hydroxylase (CYP7A1) deficiency in infants, children and adolescents aged 1 month to 18 years and adults, with agency product number EMEA/H/C/002081 -T/0004 and the date of issue of marketing authorization valid throughout the European Union:  04/04/2014.

1.18         “Cholic Acid Product NDA” means NDA 205-570 filed by Asklepion with the FDA.

1.19       “Cholic Acid Product Regulatory Assets” means the Cholic Acid Product NDA, the Cholic Acid Product MSA, the Orphan Drug Designations, the Seller’s rights to the Other Marketing Authorization Applications and, in each case, all supporting documents, files and data.

1.20        “Commercially Reasonable Efforts” means, with respect to a Party, that level of effort and resources required to carry out a particular task or obligation in an active and sustained manner consistent with the general practices applied in the research-based pharmaceutical industry in the development and commercialization of products of similar market potential to the Cholic Acid Product at a similar stage in development or product life, taking into account issues of orphan drug or other exclusivity, safety, and other relevant factors, including technical, legal, scientific, medical, operational and commercial factors, and taking into account profitability exclusive of applicable royalties, milestone, and any other similar payments.

1.21        “Competition Period” means, on a jurisdiction by jurisdiction basis, the longer of (a) […***…], or (b) any marketing exclusivity period in respect of a Product under Applicable Law, in each case, measured from the time of approval in the applicable jurisdiction.

1.22         “Competitive Product” means a product that is […***…].

1.23     “Confidential Information” means any information that (i) in any way relates to products, business, Know-How, business strategies and technology of a Party or Affiliate thereof, (ii) is furnished or disclosed to the other Party in connection with this Agreement, and (iii) is identified as “confidential” (or words of similar import) upon such disclosure; provided, however, that the term “Confidential Information” shall not include any specific information that:

  (a)          at the time of disclosure, is generally available to the public;

  (b)          after disclosure hereunder, becomes generally available to the public, except as a result of a breach of this Agreement by the recipient of such information;

  (c)          becomes available to the recipient of such information from a Third Party that is not legally or contractually prohibited by the disclosing Party from disclosing such Confidential Information; or

  (d)          the recipient of which can demonstrate by clear and convincing evidence was developed by or for such recipient without the use of any of the Confidential Information of the disclosing Party or its Affiliates hereunder.  

1.24       “Contingent Payments” means the contingent payments contemplated under Section 3.4.

***Certain Confidential Information Omitted

3

1.25         “Copyrights” means all of Seller’s (a) U.S. and foreign copyrights, whether statutory or arising under common law, (b) all copyright applications and registrations, and certificates of copyright pertaining thereto, including but not limited to, copyright registrations in each case relating to Cholic Acid Product.

1.26         “Distributor Receipts” means, with respect to a Product, all amounts paid or payable to Retrophin and/or its Affiliates for sales anywhere in the world of such Product to a Third Party (including, without limitation, licensees, sublicensees and distributors, which includes, without limitation, the Initial Distributors) to whom Retrophin or its Affiliates (or their respective successors or assigns) sells Product for resale by such Third Party.  Notwithstanding the foregoing, […***…] prior to the expiration of the initial term specified in such […***…], including, but not limited to, […***…], then, for the remaining period of the initial term ([…***…]), the amounts paid or payable to Retrophin and/or its Affiliates attributable to such […***…] that has been terminated shall be […***…][…***…].  A sample calculation of such discount to amounts paid or payable to Retrophin and/or its Affiliates is set forth on Schedule 1.26.  Distributor Receipts with respect to a Product shall also include […***…].

1.27     “Electronic Data Room” means the documents relating to Asklepion and its subsidiaries provided electronically on the share file site by Asklepion to Retrophin or its advisors as of the Effective Date and, solely taking into account the addition of (a) the manufacturing batch records for finished goods from Patheon in respect of the period prior to the Effective Date (which Asklepion may provide electronically on the share file site subsequent to the Effective Date and will be deemed to have been provided by Asklepion as of the Effective Date), and (b) such documents to the share file site required or permitted in accordance with Sections 2.2 and 6.4, as of the Closing Date.

1.28         “EMA” means the European Medicines Agency or any successor agency thereto.

1.29         “Escrow Agent” means Wilmington Trust, N.A.

1.30      “Escrow Agreement” means the Escrow Agreement between Buyer, Seller and the Escrow Agent governing the deposit, holding and release of the Escrowed Assets between the Closing Date and the date that is seven (7) days after the due date for the FDA Approval Milestone for the Bile Acid Indications as provided in Section 3.3(a), in the form to be mutually agreed upon by Buyer, Seller and the Escrow Agent.

1.31        “FDA” means the United States Food and Drug Administration or any successor agency thereto.

1.32         “First Commercial Sale” means the first sale for transfer for cash or some equivalent to which value can be assigned of a Cholic Acid Product in the United States after FDA approval of the Cholic Acid Product NDA. A sale on a cost reimbursement basis for use in a clinical trial will not constitute a First Commercial Sale.

1.33          “Initial Distributors” means […***…].

1.34     “Initial Distribution Agreements” means (i) that certain Distribution Agreement between Asklepion Pharmaceuticals LLC and […***…], as amended by the First Amendment to Distribution Agreement between Asklepion Pharmaceuticals LLC and […***…], (ii) that certain Distribution Agreement between Asklepion Pharmaceuticals LLC and […***…], as amended by the First Amendment to Distribution Agreement between Asklepion Pharmaceuticals LLC and […***…], and (iii) that certain Distribution Agreement between Asklepion Pharmaceuticals LLC […***…], as amended or supplemented by Addendum to Distribution Agreement between […***…] and Asklepion Pharmaceuticals, Addendum II to Distribution Agreement between […***…] and Asklepion Pharmaceuticals, LLC dated 22 October 2012, dated January 7, 2015, and the Second Amendment to Distribution Agreement between Asklepion Pharmaceuticals LLC and […***…].

***Certain Confidential Information Omitted

4

1.35         “Intellectual Property” means any and all rights in and to Copyrights, Know-How, trademarks, service marks, service names, trade names, internet domain names, e-mail addresses, applications or registration for any of the foregoing, and any similar type of rights and interests and intangible assets, in each case, recognized under any laws as intellectual property to which rights of ownership accrue pursuant to such laws or conventions or under any applicable license or contract, whether now existing or hereafter created, together with all modifications, enhancements and improvements thereto.

1.36        “Know-How” means any and all know-how, trade secrets, inventions (other than inventions covered by a patent), data, processes, photographs, techniques, procedures, drawings, compositions, devices, methods, formulas, algorithms, protocols and information, whether or not patentable, which are not generally publicly known, including, all chemical, pharmacological, toxicological, pharmaceutical, physical, analytical, safety, efficacy, bioequivalency, quality assurance, quality control, clinical data, and scientific research information and data relating to a product.

1.37        “Knowledge of Seller” or “Seller’s Knowledge” means (a) for purposes of this Agreement other than the representations and warranties contained in Section 4.11(a), the actual knowledge of the executive officers of Seller, […***…], after exercising their duties in good faith, and (b) for purposes of the representations and warranties contained in Section 4.11(a), is based solely on the written representations and/or statements made to Seller by […***…].

1.38        “Liens” means any mortgages, security interests, liens, options, pledges, equities, claims, charges, restrictions, conditions, conditional sale contracts and any other adverse interests or other encumbrances of any kind whatsoever.  Notwithstanding the foregoing, the term “Liens” shall not include liens as set forth on the “Permitted Liens Schedule”.

1.39         “[…***…] Matter” means all claims or rights of Seller’s wholly-owned subsidiary, ASK Pharmaceuticals GmbH Heimeranstraße 35, D-80339 München, Deutschland, […***…], as more fully described on Schedule 4.5.

1.40         “Net Revenues” means the sum of (i) Net Sales of the Cholic Acid Product, and (ii) the Distributor Receipts.

1.41         “Net Sales” means, with respect to a Product, the […***…], less:

  (a)          […***…];

  (b)          […***…];

  (c)          […***…]; and

  (d)          […***…].

For the avoidance of doubt, Net Sales shall not include any payments among Retrophin and its Affiliates.  Net Sales shall be determined in accordance with generally accepted accounting principles, consistently applied.  

***Certain Confidential Information Omitted

5

1.42         “Orphan Drug Designations” means (i) the Orphan Product Designation from the Department of Health and Human Services, Food and Drug Administration Office of Orphan Products Development for Cholic Acid, designated as of 07-18-2003, under the name “Cholbam” as a “drug for a rare disease or condition” for the treatment of inborn errors of cholesterol and bile acid synthesis and metabolism under the Orphan Drug Act, as amended, and implementing regulations at 21 C.F.R. Part 316, and (ii) the designation of Cholic Acid as an orphan medicinal product under Regulation (EC) No 141/2000 of the European Parliament and of the Council by the Commission of the European Communities (EU orphan designation number: EU/3/09/683) to Seller’s wholly-owned subsidiary, ASK Pharmaceuticals GmbH Heimeranstraße 35, D-80339 München, Deutschland, for the treatment of inborn errors of primary bile acid synthesis responsive to treatment with Cholic Acid.

1.43         “Other Marketing Authorization Applications” means the applications for marketing authorization or similar approvals with the applicable regulatory bodies for the commercialization of the Cholic Acid Product made by […***…][…***…].

1.44        “PDMA” means the Prescription Drug Marketing Act of 1987, as amended, and the rules, regulations and guidelines promulgated thereunder and in effect from time to time, and any foreign counterpart thereto.

1.45         “Person” means any individual, partnership, association, corporation, limited liability company, trust, or other legal Person or entity.

1.46         “Product” means Cholic Acid Product, any Combination Product or […***…], as the case may be.

1.47         “Royalties” means the royalties on Net Revenues of Cholic Acid Product and Net Sales of […***…] payable to Asklepion pursuant to Section 3.5.

1.48         “Security Agreement” means the Security Agreement to be executed by the Buyer for the benefit of the Seller at the Closing in order to grant Seller a first-priority security interest and lien in and to the Asset as security for the Buyer’s obligations to pay the FDA Milestone Payments in accordance with the terms of Section 3.3 of this Agreement in the form to be mutually agreed upon by Buyer and Seller.

1.49         “Third Party” means any Person other than a Party and such Party’s Affiliates.

1.50        “U.S. Commercialization Plan” means the general marketing and promotional plans for the Cholic Acid Product in the United States, in a consistent with Retrophin’s plans generally and pharmaceutical industry standards, for each calendar year.

1.51       “Voucher” means a Paediatric Rare Disease Priority Review Voucher, if and only if, granted to Asklepion by the FDA in respect of the Cholic Acid Product.  

1.52         Interpretation.  Unless the context of this Agreement otherwise requires (a) words of any gender include each other gender, (b) words using the singular or plural number also include the plural or singular number, respectively, (c) the terms “hereof,” “herein,” “hereby,” and derivative or similar words refer to this entire Agreement, (d) the terms “Article,” “Section,” and “Exhibit” refer to the specified Article, Section and Exhibit of this Agreement and (e) the terms “include,” “includes,” or “including,” shall be deemed to be followed by the words “without limitation” unless otherwise indicated.  Whenever this Agreement refers to a number of days, unless otherwise specified, such number shall refer to calendar days.  The headings in this Agreement are for reference purposes only and shall not affect in any way the meaning or interpretation of this Agreement.

***Certain Confidential Information Omitted

6

ARTICLE 2
SALE AND PURCHASE OF ASSETS

2.1         Conveyance; Assets; Escrowed Assets.  

(a)          Assets.  Upon the terms and subject to the conditions of this Agreement, on the Closing Date, the Seller shall and, if owned or held by its Affiliates, shall cause its Affiliates to, irrevocably sell, assign, transfer, convey and deliver to Buyer or its Affiliates (as directed by Buyer in writing), and Buyer shall and, if and to the extent directed by Buyer, shall cause its Affiliates to, purchase, acquire, assume and accept, free and clear of any and all Liens, all right, title and interest of Seller and its Affiliates in and to the following assets related to the Cholic Acid Product (the “Assets”):

(i)          the Assigned Contracts;

(ii)         the Cholic Acid Product Data Assets;

(iii)        the Cholic Acid Product IP;

(iv)        the Cholic Acid Product Regulatory Assets;

(v)         to the extent assignable, all claims, judgments, cases in action or rights related to the Cholic Acid Product, including, for past, present or future infringement of the Cholic Acid Product IP;

(vi)        copies of other books, records (including computer records), correspondence (including email communications) of the Seller relating to the Cholic Acid Product and/or the Assets;

(vii)       to the extent assignable, all representations, warranties, guarantees, indemnities, undertakings, covenants not to compete and covenants not to sue benefitting the Assets, certificates, covenants, agreements and all security therefor received by the Seller on the purchase, license or other acquisition of any part of the Assets; and

(viii)      to the extent granted, if and when granted (if at all), to Asklepion, any Voucher; and

(ix)         all claims or rights related to the […***…] Matter to be assigned by Seller’s wholly-owned subsidiary, ASK Pharmaceuticals GmbH Heimeranstraße 35, D-80339 München, Deutschland.

(b)          Conveyance of Assets; Escrowed Assets; Security Interest.  No right, title or interest in or to the Assets or Assumed Liabilities (as herein defined) shall be sold, assigned, transferred, conveyed or delivered to Buyer until the Closing Date.  

(i)          At the Closing, Buyer shall deposit with the Escrow Agent to hold and release in accordance with the terms of the Escrow Agreement an assignment and assumption agreement assigning and transferring from Buyer to Seller all of the Assigned Contracts, a bill of sale and assignment assigning and transferring from Buyer to Seller all of the Assets, together with any other applicable instruments of assignment or transfer relating to the transfer of the Cholic Acid Product Regulatory Assets or necessary or appropriate to effect the transfer and assignment of the Assets from Buyer to Seller (collectively, the “Escrowed Transfer Documents”), for release to (x) Retrophin upon receipt of a joint instruction letter from Asklepion and Retrophin confirming Retrophin’s payment in full to Asklepion of the FDA Approval Milestone for the Bile Acid Indications on the FDA Approval Milestone Payment Date (or within the 7 day cure period thereafter), or (y) Asklepion if no such instruction letter shall have been received by the Escrow Agent on or prior to the tenth day following the FDA Approval Milestone Payment Date.  All of the Escrowed Transfer Documents shall provide that all the Assets shall be transferred to Seller, free and clear of all Liens.

***Certain Confidential Information Omitted

7

(ii)         At the Closing, Buyer’s obligation to pay the FDA Approval Milestone for the Bile Acid Indications and any other accrued and unpaid payments under Article 3, will be secured by a second-priority, perfected security interest and lien in the Assets, on the terms and conditions set forth in the Security Agreement, which security interest and lien will be released upon payment in full to Asklepion of the FDA Approval Milestone for the Bile Acid Indications on the FDA Approval Milestone Payment Date (or within the 7 day cure period thereafter) in accordance with the terms of the Security Agreement.  Additional rights of the Seller will be set forth in the Security Agreement and any applicable subordination or intercreditor agreement.  

(iii)        At the Closing, and for so long as the Escrowed Transfer Documents are held in escrow, Buyer shall not, directly or indirectly through its Affiliates, subsidiaries or otherwise, mortgage, pledge, hypothecate, encumber or subject to any Lien any of the Assets, other than to U.S. Bank National Association, as administrative agent, under Buyer’s Credit Agreement, dated as of June 30, 2014, as amended.  In addition, for so long as the Escrowed Transfer Documents are in escrow, Buyer will use Commercially Reasonable Efforts to preserve intact the Assets and the business-related thereto, including the commercialization, development, promotion, marketing and sales of the Cholic Acid Product, to maintain the rights and franchises associated with or related to the Cholic Acid Product and the other Assets, and preserve the relationships with customers, distributors, and others having business dealings with respect to the Cholic Acid Product or other Assets.

(c)          Pre-Closing Sales.  From the Effective Date until the Closing Date, Asklepion will book all Net Sales of Cholic Acid Products outside the United States (“OUS”).

2.2         Cholic Acid Product NDA.  Asklepion, as the sponsor, will use Commercially Reasonable Efforts to obtain the Cholic Acid Product NDA during the period from the Effective Date until FDA approval, including, but not limited to, correspondence, reports and filings with FDA and responsibility for all clinical trials and data generated therefrom, and will use reasonable efforts to keep Retrophin informed of FDA correspondence and calls and, to the extent reasonably practicable, to participate in such calls with the FDA; provided, however, that notwithstanding the foregoing, Asklepion makes no representation, warranty or guaranty concerning the receipt (if at all) of the Cholic Acid Product NDA or the Voucher or the indications for which the Cholic Acid Product NDA may be granted (if at all).  Until the Closing Date, Asklepion shall promptly within three (3) days after the delivery or receipt thereof, make available to Retrophin in the Electronic Data Room (or by other reasonable means mutually agreeable to Asklepion and Retrophin) such correspondence with the FDA required to be made available to Retrophin by Asklepion in the Electronic Data Room in order for the representation and warranty contained in Section 4.16(b) to be true and correct in all material respects at and as of the Closing Date.  In connection with the Closing and any return of the Assets to Asklepion pursuant to Section 2.1(b)(i) and 9.3, Retrophin shall take all reasonable steps to cooperate in an orderly transfer of the Cholic Acid Product NDA (and associated IND) in accordance with all applicable FDA regulations.  After the Closing, Retrophin will have full ownership, control of and responsibility for the Cholic Acid Product NDA (and associated IND) and will have sole responsibility for, and control of, all subsequent FDA and other regulatory filings in respect of the Cholic Acid Product NDA (and associated IND).  Notwithstanding anything to the contrary in this Section 2.2 or elsewhere in this Agreement, until at or immediately prior to the Closing, in no event shall Asklepion be required to disclose or make available to Retrophin any information or materials, including, but not be limited to, correspondence, reports and filings with FDA and clinical trials and data generated therefrom, with respect to the CTX Indication, which Retrophin acknowledges and agrees are of a commercially sensitive and competitive nature.

2.3         Assumed Liabilities.  Upon the terms and subject to the conditions of this Agreement, on the Closing Date, Buyer shall assume, be responsible for and pay, perform and discharge when due to assume, any and all of the liabilities of Seller to the extent relating to the Assigned Contracts, the Cholic Acid Product, the Assets or the Cholic Acid Product Inventory, each of which are expressly assumed by Buyer and accrue from and after the Closing Date (the “Assumed Liabilities”).  

2.4         Excluded Liabilities.  Except for the Assumed Liabilities, but without limiting the terms or conditions of this Agreement, Buyer shall not assume or be liable for any liabilities of Seller or their respective Affiliates (fixed, contingent or otherwise, and whether or not accrued) relating to the Assigned

8

Contracts, the Cholic Acid Product, the Assets or the Cholic Acid Product Inventory in respect of the period prior to the Closing Date (the “Excluded Liabilities”).

2.5         Excluded Assets.  Notwithstanding anything to the contrary contained in Article 2 or elsewhere in this Agreement, all assets of Seller (collectively, the “Excluded Assets”) that are not part of the Assets or the Cholic Acid Product Inventory, are excluded from the transactions contemplated by this Agreement and shall remain the property of Seller after the Closing Date.

2.6        Cholic Acid Product Inventory. Concurrently with the Closing, upon the terms and subject to the conditions of this Agreement, on the Closing Date, the Seller, on behalf of itself and its Affiliates, shall irrevocably sell, assign, transfer, convey and deliver to Buyer, and Buyer shall purchase, acquire and accept, free and clear of any and all Liens, all right, title and interest of Seller and its Affiliates in and to the Cholic Acid Product Inventory, at cost to Asklepion determined in accordance with Section 3.7.

2.7         Transfer Taxes and Fees.  Any and all sales, excise, use, value-added and similar taxes, fees or duties assessed or incurred by reason of the sale by Seller and the purchaseby Buyer of the Purchased  Assets hereunder shall be shared equally between the Seller and Buyer, regardless of which Party such taxes, fees or duties are assessed against.

ARTICLE 3
CONSIDERATION

3.1         Consideration.  Subject to the terms and conditions of this Agreement, the consideration (the “Consideration”) for the transfer and conveyance of the Assets to Buyer in accordance with Article 2 shall be paid by Buyer by delivery of the following to Seller.

3.2       Effective Date Payment.  No later than January 13, 2015, Retrophin will pay to Asklepion five million dollars ($5,000,000).  

3.3          FDA Approval Milestones. Within forty five (45) days of FDA approval of the Cholic Acid Product NDA, Retrophin will pay one-time FDA approval milestones (each, and “FDA Approval Milestone”) as follows:

(a)           Bile Acid Indications. Retrophin will pay Asklepion an FDA Approval Milestone no later than the latest of (such latest date, the “FDA Approval Milestone Payment Date”) (i) March 31, 2015, or (ii) 45 days after product approval of FDA approval of the Cholic Acid Product NDA, provided the approved labeling includes indication statements for each of the following bile acid indications:

(i)          treatment of children and adults with an inborn error of primary bile acid synthesis due to deficiency in 3β-hydroxy-C27-steroid oxidoreductase deficiency; and

(ii)         treatment of children and adults with an inborn error of primary bile acid synthesis due to deficiency in Δ4-3-oxosteroid 5β-reductase deficiency (collectively, clauses (i) and (ii), the “Bile Acid Indications”).

9

The FDA Approval Milestone for the Bile Acid Indications will be equal to (i) twenty-seven million dollars ($27,000,000) if achieved on or before December 31, 2015, (ii) […***…] ($[…***…]) if achieved after December 31, 2015 but on or before December 31, 2016, (iii) […***…] ($[…***…]) if achieved after December 31, 2016 but on or before December 31, 2017.  If the FDA Approval Milestone for the Bile Acid Indications has not been achieved by December 31, 2017, the parties will mutually agree to either (i) further reductions in the FDA Approval Milestone for the Bile Acid Indications for later approvals or (ii) termination of this Agreement.  If Buyer fails to deliver to Seller the full FDA Approval Milestone for the Bile Acid Indication on or before the FDA Approval Milestone Payment Date, Buyer shall have an additional seven (7) days in which cure such payment, during which time the late charges contemplated by Section 3.6(g) shall accrue and become payable to Seller.

(b)            CTX Indication.  Retrophin will pay Asklepion an additional FDA Approval Milestone of nine million dollars ($9,000,000) on the applicable FDA Approval Milestone Payment Date provided the approved labeling includes an indication statement for the treatment of children and adults for Cerebrotendindous Xanthomatosis (the “CTX Indication”).  The FDA Approval Milestone for the CTX Indication shall be paid in shares of Retrophin common stock, calculated based on the last reported sale price regular way on the trading day immediately preceding the Effective Date or, in case no such reported sale takes place on such day, the average of the last closing bid and ask prices regular way, in either case, on the NASDAQ Global Market, and any such shares that may be so issued shall not be subject to any Liens or restrictions on transferability other than such restrictions contained under Rule 144 promulgated under the Securities Act of 1933, as amended (the “Securities Act”).  

3.4         Contingent Payments.  Following the First Commercial Sale of Cholic Acid Product, Retrophin will, within forty five (45) days of the end of the first calendar quarter in which cumulative Cholic Acid Product Net Revenues meet the thresholds below, make the following Contingent Payments to Asklepion.  The Contingent Payments will be paid one-time only upon the first achievement of cumulative Net Revenues for Cholic Acid Products.  The Contingent Payments will be paid in cash or, upon the mutual agreement of the Parties, in shares of Retrophin common stock calculated based on the last reported sale price regular way on the last trading day of the applicable calendar quarter or, in case no such reported sale takes place on such day, the average of the last closing bid and ask prices regular way, in either case, on the NASDAQ Global Market, and any such shares that may be so issued shall not be subject to any Liens or restrictions on transferability other than such restrictions contained under Rule 144 promulgated under the Securities Act, or a combination of cash and shares of Retrophin common stock.

Cumulative Cholic Acid Product Net Revenues Threshold		Contingent Payment
$	[…***…]	$	[…***…]
$	[…***…]	$	[…***…]
$	[…***…]	$	[…***…]
$	[…***…]	$	[…***…]

***Certain Confidential Information Omitted

10

3.5           Product Royalties.

(a)          Cholic Acid Product.  In addition to the above payments, Retrophin will, no later than forty five (45) days following the close of each calendar quarter, pay Asklepion tiered Royalties based on annual Net Revenues of Cholic Acid Product as set forth below:

Annual Net Revenues of Cholic Acid Product		Royalty Rate Percent Net Revenues
$	[…***…]	$	[…***…]	%
>$	[…***…]	$	[…***…]	%
>$	[…***…]	$	[…***…]	%

(b)          […***…]. In the event that the FDA approves the Cholic Acid Product NDA for the […***…], Retrophin will, thereafter pay to Asklepion no later than forty five (45) days following the close of each calendar quarter, Royalties equal to […***…] of Net Sales of Chenodal in the United States.

3.6         Payment Terms.

(a)          Manner of Payment.  All payments to be made by Retrophin under this Article 3 will be made in U.S. dollars by wire transfer to such bank account as Asklepion may designate.

(b)          Records and Audits. Retrophin shall keep, and shall cause each of its Affiliates and licensees, to keep adequate books and records of accounting for the purpose of calculating all Contingent Payments and Royalties payable to Asklepion under Sections 3.4 and 3.5.  For the seven (7) years next following the end of the calendar year to which each shall pertain, such books and records of accounting (including those of Retrophin’s Affiliates and licensees) shall be kept at each of their principal place of business and shall be open for inspection at reasonable times and upon reasonable notice by an independent certified accountant selected by Asklepion, and which is reasonably acceptable to Retrophin, for the sole purpose of inspecting the Contingent Payments and Royalties due to Asklepion under this Agreement.  In no event shall such inspections be conducted hereunder more frequently than once every twelve (12) months.  Such accountant must have executed and delivered to Retrophin and its Affiliates or licensees, a confidentiality agreement as reasonably requested by Retrophin, which shall include provisions limiting such accountant’s disclosure to Asklepion to only the results and basis for such results of such inspection.  The results of such inspection, if any, shall be binding on both Parties.  Any underpayments shall be paid by Retrophin within thirty (30) days of notification of the results of such inspection.  Any overpayments shall be fully creditable against amounts payable in subsequent payment periods.  Asklepion shall pay for such inspections, except that in the event there is any upward adjustment in aggregate Contingent Payments and/or Royalties payable for any calendar year shown by such inspection of more than […***…] of the amount paid, Retrophin shall reimburse Asklepion for any reasonable out-of-pocket costs of such accountant.

***Certain Confidential Information Omitted

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(c)          Reports and Royalty and Contingent Payments.  For as long as Contingent Payments or Royalties are due under Sections 3.4 or 3.5, Retrophin shall furnish to Asklepion a written report (each, a “Report”) for each calendar quarter, showing the amount of Net Sales of Products and Net Revenues in respect of Products and, as applicable, any Contingent Payment or Royalty due for such calendar quarter under Sections 3.4 or 3.5.  Reports shall be provided within thirty (30) days of the end of the quarter for Net Sales and Net Revenues generated by Retrophin and its Affiliates, and within forty-five (45) days of the end of the quarter for Net Sales and Net Revenues generated by licensees.  Royalty for each calendar quarter shall be due at the same time as such written Report for the calendar quarter.  The Report shall include, at a minimum, the following information for the calendar quarter, each listed by Product and region:  (i) the number of units of Products sold by Retrophin and its Affiliates and licensees on which Contingent Payments or Royalties are owed to Asklepion hereunder; (ii) the gross amount received for such sales; (iii) deductions taken from Net Sales as specified in the definition thereof; (iv) Net Sales and (v) Net Revenues.  All Reports shall be treated as Confidential Information of Retrophin.

(d)          Disputed Reports. Each Report shall be final, binding and conclusive, unless Seller or its designee notifies Buyer in writing of any disagreement therewith (an “Objection Notice”) within thirty (30) after its receipt thereof, specifying (a) those items as to which there is disagreement and (b) a reasonably detailed description of the basis, nature, dollar amount and extent of the dispute or disagreement. If Seller delivers an Objection Notice within such period, then for a period of twenty (20) business days from the date of delivery of the Objection Notice, Buyer shall afford Seller and its agents or other representatives with reasonable access during normal business hours to the books and records of Buyer and its licensees so as to enable its review of the Report and the information contained therein. Buyer and Seller shall attempt in good faith to resolve such dispute, and any resolution by them as to any disputed amounts shall be final, binding and conclusive. If Buyer and Seller are unable to resolve all disputes reflected in the Objection Notice within twenty (20) business days after the date of delivery of the Objection Notice (or such longer period as Buyer and Seller may mutually agree upon), then Buyer and Seller shall request Ernst & Young (the “Independent Accounting Firm”) to resolve any remaining disagreements. Buyer and Seller shall use their commercially reasonable efforts to cause the Independent Accounting Firm to make its determination within thirty (30) days of accepting its selection. The determination by the Independent Accounting Firm shall be final, binding and conclusive on Buyer and Seller and shall not be appealable. Buyer and Seller shall deliver to the Independent Accounting Firm all work papers and back-up materials relating to the unresolved disputes requested by the Independent Accounting Firm to the extent available to Buyer, Seller and their respective agents or other representatives. Buyer and Seller shall be afforded the opportunity to present to the Independent Accounting Firm any material related to the unresolved disputes and to discuss the issues with the Independent Accounting Firm; provided, however, that no such presentation or discussion shall occur without the presence of agents or other representatives of the Buyer and Seller. The determination of the Independent Accounting Firm shall be limited to the disagreements submitted to the Independent Accounting Firm.  Upon resolution by the Independent Accounting Firm to its satisfaction of all such disputed matters, the Independent Accounting Firm shall cause to be prepared and shall deliver to Buyer and Seller a final Report setting forth the Net Sales and Net Revenues for the Products in respect of the calendar quarter at issue in the disputed Reported, and the date of such delivery by the Independent Accounting Firm shall be deemed the date on which the Report and the Net Sales and Net Revenues for the Products in respect of the calendar quarter at issue in the disputed Reported shall become final, binding and conclusive.  The fees and expenses of the Independent Accounting Firm shall be borne equally by Seller and Buyer.

(e)          Marketing and Sale of Cholic Acid Product.  From and after the Closing Date, Buyer shall, and shall cause its Affiliates and its and its Affiliates’ successors and assigns to:

(i)          keep complete, true and accurate books and records of all Net Sales and Net Revenues and deliver to Seller or its Affiliates, successors or assigns, the U.S. Commercialization Plan on an annual basis;

(ii)         use Commercially Reasonable Efforts to commercialize, including development to support commercialization as commercially reasonable, the Cholic Acid Product in the United States in a manner consistent with the U.S. Commercialization Plan and in the rest of the world;

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(iii)        if, at any time, Buyer, its Affiliates, or any of their respective successors or assigns shall (A) consolidate with or merge with or merge into any other Person, or (B) sell, assign, convey, transfer, license, sublicense, lease or sublease all or any portion of the Assets, give a written notice to Asklepion or its designee (or their respective successors or assigns) setting forth the name and address of any such Person with which Buyer, its Affiliates or their respective successors or assigns engaged in such transaction described in clauses (A) and/or (B), together with the name, telephone number, facsimile number and email address of an individual contact at such Person and will provide a copy of such notice to such Person; provided, however, that if any such Person with which Buyer, its Affiliates or any of their respective successors or permitted assigns engages in a transaction contemplated by clauses (A) and/or (B) owns, holds or commercializes a […***…], then the assignment of this Agreement in connection with or pursuant to such transaction shall be permitted if, following the approval of Seller or its successors or permitted assigns (which approval shall not be unreasonably withheld), such Person shall affirmatively undertake to continue to use Commercially Reasonable Efforts with respect to the Cholic Acid Product […***…] owned, held or commercialized by such Person in an amount reasonably sufficient to compensate Seller or its successors or permitted assigns for […***…].

(iv)        promptly furnish to Seller written notice of (A) the termination of any material contract with respect to the Cholic Acid Product, including, but not limited to, any Material Supplier Contract or Initial Distributor Agreement, that could have a material adverse effect on the business, financial condition or results of operation of the Buyer, taken as a whole, or (B) any material breach by any party to any Material Supplier Agreement or Initial Distributor Agreement;

(v)         comply with all Applicable Laws with respect to the marketing, promotion and commercialization of the Cholic Acid Product, except where the failure to comply would not have a material adverse effect on the business, financial condition or results of operation of the Buyer, taken as a whole;

(vi)        Retrophin shall perform its obligations under this Agreement and shall conduct its business in compliance with Applicable Law where the failure to comply would not have a material adverse effect on the business, financial condition or results of operation of the Buyer, taken as a whole.  Without limiting the generality of the foregoing: (a) Retrophin shall ensure that all of its employees and consultants comply with Applicable Law, and shall implement and maintain policies and procedures to ensure such compliance, including maintaining a corporate compliance program that will include compliance monitoring focused on specific risk areas, including off-label promotion, fraud and abuse, and false claims, for the purpose of assessing whether Retrophin’s policies and procedures are being followed; and (b) Retrophin shall, and shall ensure that all of its employees and consultants, comply the Anti-Corruption Laws;

(vii)       for the Competition Period not, directly or indirectly, (A) manufacture, produce, market, commercialize or supply any Competitive Product, without the prior written consent of Seller, or (B) acquire, own an interest in, manage, operate, join, control, lend money or render financial or other assistance to or participate in or be connected with, as an officer, employee, partner, stockholder, consultant or otherwise, any Person that competes in manufacturing, producing, marketing or supplying any Competitive Product; provided, that the provisions of this Section 3.6(e)(vii) shall not apply to […***…].

***Certain Confidential Information Omitted

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The Parties agree that the covenant set forth in Section 3.6(e)(vii) is reasonable with respect to duration and scope and necessary to protect the legitimate interests of Seller and its Affiliates, and that any violation thereof would cause irreparable injuries.  Therefore, Buyer, on behalf of itself and its Affiliates and their respective successors and assigns, acknowledges and agrees that, in the event of a violation by Buyer or its Affiliates of any of the restrictions contained in Section 3.6(e)(vii), Seller or its Affiliates or their respective successor or assigns shall be entitled to obtain from any court of competent jurisdiction temporary, preliminary and permanent injunctive relief, in addition to any other rights Seller or its Affiliates or their respective successor or assigns may be entitled.  In addition, if the final judgment of any such court declares that any term or provision of Section 3.6(e)(vii) is invalid or unenforceable, the Parties agree that the court making the determination of invalidity or unenforceability shall have the power to reduce the scope, duration, or area of the term or provision, to delete specific words or phrases, or to replace any invalid or unenforceable term or provision with a term or provision that is valid and enforceable and that comes closest to expressing the intention of the invalid or unenforceable term or provision, and Section 3.6(e)(vii) shall be enforceable as so modified.

(f)          Taxes. Retrophin may withhold from payments due to Asklepion amounts for payment of any withholding tax that is required by law to be paid to any taxing authority with respect to such payments.  Retrophin will give proper evidence from time to time as to the payment of any such tax.

(g)          Late Charge.  If Retrophin fails to make any payment under this Agreement or any Ancillary Agreement timely when due, Retrophin shall pay to Asklepion, in addition to any other sum due Asklepion under this Agreement or any Ancillary Agreement, a late charge equal to […***…] of such past due payment, compounding monthly (the “Late Charge”), which Late Charge is a reasonable estimate of the losses that may be sustained by Asklepion due to the failure of Retrophin to make timely payments.  The Late Charge shall be due whether or not Asklepion declares a breach of this Agreement or otherwise demands immediate payment of the sums due under this Article 3.  The right to impose the Late Charge shall not constitute a grace period or provide any right of Retrophin to make a payment other than on its due date.  It is hereby expressly agreed that such Late Charge is to compensate Asklepion for costs incurred in connection with the administration of such late payment, and does not constitute a penalty.  The Late Charge is in addition to, and not in any way in limitation of, any other money due by Retrophin under this Agreement or any Ancillary Agreement by reason of such late payment or otherwise.

3.7         Cholic Acid Product Inventory Payment.  

(a)          No more than five (5) days after the Closing Date, Retrophin will pay to Asklepion an amount in cash by wire transfer of immediately available funds equal to the cost (the “Pre-Closing Inventory Cost”) of the Cholic Acid Product Inventory, determined based upon a physical inventory of the Cholic Acid Product Inventory completed no earlier than three Business Days prior to the Closing Date.  

(b)          Within three Business Days after the Closing Date (the “Physical Inventory Date”), Retrophin shall be permitted to conduct a physical inventory of the Cholic Acid Product Inventory or to request from Asklepion its work paper or other supporting documentation with respect to the cost (the “Closing Inventory Cost”) of the Cholic Acid Product Inventory as of the Closing Date, and shall deliver to Asklepion a notice (the “Inventory Notice”) setting forth the Closing Inventory Cost, including the supporting detail thereof based on the physical inventory or work papers and supporting documentation, within five days after the Physical Inventory Date; provided that, if Retrophin shall not deliver an Inventory Notice to Asklepion on or prior to the Physical Inventory Date, then Buyer shall be deemed to have accepted Pre-Closing Inventory Cost and the Cholic Acid Product Inventory and cost thereof shall be determined to be final and binding on Retrophin and Asklepion.  

***Certain Confidential Information Omitted

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(c)          Asklepion shall have five days to review any Inventory Notice and to notify Retrophin in writing of any objections thereto.  Within three days after Retrophin’s receipt of objections (if any) to the Inventory Notice from Asklepion, Retrophin shall notify Asklepion in writing if it accepts or rejects all or any portion of such objections; provided that, if Retrophin shall not deliver any response to the Seller in writing on or prior to the expiration of such 5-day period, then Retrophin shall be deemed to have accepted Asklepion’s objections to the Inventory Notice and Asklepion’s determination of the Closing Inventory Cost, which shall be determined to be final and binding on Retrophin and Asklepion.  If Buyer does not accept all of Asklepion’s objections to the Inventory Notice, then Retrophin and Asklepion shall attempt to resolve any objections in good faith for a period of ten days.  If the Parties shall be unable to resolve any objections to the Inventory Notice or the determination of the Closing Inventory Cost, then either Retrophin or Asklepion shall be permitted, at its sole cost or expense, to submit any remaining objections to the Independent Accounting Firm for resolution within ten days after engagement.  The Independent Accounting Firm shall issue a written statement as to its resolutions of any outstanding objections to the Inventory Notice or the determination of the Closing Inventory Cost, which determination and the Inventory Notice shall be determined to be final and binding on Retrophin and Asklepion.  The Independent Accounting Firm shall consider only those items and amounts that were set forth in the written notices of Retrophin and Asklepion and that remain unresolved.  In resolving any Item of dispute, the Independent Accounting Firm may not assign a value to any item greater than the greatest value for such item claimed by either Party or less than the smallest value for such item claimed by either Party.  

(d)          If, following the final determination of the Closing Inventory Cost pursuant to this Section 3.7, the Closing Inventory Cost exceeds the Pre-Closing Inventory Cost, then Asklepion shall pay Retrophin by check or wire transfer of immediately available funds to an account designated by Retrophin in writing an amount equal to such excess within three (3) Business Days after date on which the Closing Inventory Cost is determined to be final.  If, however, the Pre-Closing Inventory Cost exceeds the Closing Inventory Cost, then Retrophin shall pay Asklepion an amount equal to such excess within three (3) Business Days after date on which the Closing Inventory Cost is determined to be final.

ARTICLE 4
REPRESENTATIONS AND WARRANTIES OF SELLER

Except as set forth on the Seller’s disclosure schedule attached hereto and incorporated herein, comprising schedules numbered according to the sections of this Article 4 and as specifically set forth herein (the “Seller’s Disclosure Schedule”), with each exception set forth in the Seller’s Disclosure Schedule deemed to qualify (a) the corresponding representation and warranty set forth in this Agreement that is specifically identified (by cross-reference or otherwise) in the Seller’s Disclosure Schedule and (b) all other representations and warranties to the extent the relevance of such exception to such other representation and warranty is reasonably clear, Seller hereby represents and warrants to Buyer as of the Effective Date and as of the Closing Date (except if another date is specified in the representation or warranty) as follows:

4.1         Organization; Subsidiary.Selleris a business entity duly organized, validly existing and in good standing under the laws of Delaware.  Seller has the requisite power and authority to own, lease and operate the properties now owned, leased and operated by it and to carry on its business as currently conducted.  Seller is duly qualified to do business as a foreign entity in each jurisdiction in which the nature of its business or the character of its properties makes such qualification necessary, except where the failure to do so would not have a material adverse effect on the Seller or any of the Assets, taken as a whole.  

4.2         Authority and Enforceability.Sellerhas the requisite power and authority to enter into this Agreement and each of the Bill of Sale, Assignment and Assumption Agreement, the Escrow Agreement and the Security Agreement, in each case, to which it is a party (collectively the “Ancillary Agreements”), and to perform its obligations hereunder and thereunder.  Seller has taken all necessary action on its part to authorize the execution and delivery of this Agreement and each Ancillary Agreement to which it is a party, and the performance of its obligations hereunder and thereunder.  This Agreement has been, and each Ancillary Agreement to which it is a party will be, duly and validly executed and delivered by Seller and this Agreement is, and each Ancillary Agreement to which it is a party will be, the legal, valid and binding obligation of Seller, enforceable against Seller in accordance with its terms,

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except that such enforceability may be limited by bankruptcy, insolvency, moratorium or other similar laws affecting or relating to creditors’ rights generally, and is subject to general principles of equity.

4.3         No Violation, Etc.The execution and delivery of this Agreement and the performance of the Seller’s obligations hereunder does not, and the execution and delivery of each Ancillary Agreement to which it is a party and the performance of the Seller’s obligations thereunder will not, (a) violate or conflict with any provision of the certificate of formation or limited liability company agreement of Seller, (b) violate, or conflict with, or result in a breach of any provision of, or constitute a default or give rise to any right of termination, cancellation or acceleration (with the passage of time, notice or both) under any Assigned Contract, (c) violate any Applicable Law which Seller or any of the Assets are subject or (d) result in any Lien on the Assets.   Without limiting the foregoing, Seller has not granted any right to any Third Party which would conflict with the conveyance of the Assets to Buyer.  

4.4         No Consents and Approvals.Except for the consents to assign the Assigned Contracts, no permit, consent, approval or authorization of, or notice, declaration, filing or registration with, any governmental authority or Third Party is or will be necessary in connection with the execution and delivery by Seller of this Agreement and each Ancillary Agreement to which it is a party or the performance by Seller of its obligations hereunder and thereunder.  

4.5          Litigation.There is no litigation, proceeding, arbitration, or, to the Seller’s Knowledge, investigation pending against the Seller or its Affiliates, or to Seller’s Knowledge, threatened with respect to the Assets or the transactions contemplated herein.

4.6       Compliance with Law.  Seller has complied with the Applicable Laws and has conducted, and, to Seller’s Knowledge, each of Seller’s contractors or consultants have conducted, all development and commercialization activities related to the Cholic Acid Product in accordance with the Applicable Laws, except, in each case, where the failure to comply would not have a material adverse effect on development and/or commercialization of Cholic Acid Product.

4.7         Assets.Seller has, and on the Closing Date will convey and transfer to Buyer, legal, equitable and valid title to, or a valid lease or license to use, each and all of the Assets, free and clear of any and all Liens.  The Assets constitute all assets (tangible and intangible) of Seller relating to the development, manufacture and commercialization of the Cholic Acid Product as currently held by Seller as of the Effective Date.

4.8         Product Data Assets. Seller has made available to Buyer in the Electronic Data Room true, correct and complete copies of all tangible embodiments in Seller’s possession or control of the Cholic Acid Product Data Assets.  The Product Data Assets constitute all information in the possession or control of Seller or its Affiliates in the development, manufacture and commercialization of the Cholic Acid Product, including efficacy, side effects, injury, toxicity or sensitivity, reaction and incidents or severity thereof, associated with any clinical use, studies, investigations or tests with such Cholic Acid Product (animal or human), whether or not determined to be attributable to such Cholic Acid Product.  Neither Seller nor its Affiliates have employed, or, to Seller’s Knowledge, used a contractor or a consultant that employs, any individual or entity debarred by the FDA, or any individual who or entity which is the subject of any FDA debarment investigation or proceeding.  

4.9       Assigned Contracts.The Assigned Contracts Schedule lists all material Seller contracts relating to the Cholic Acid Product.  Seller has made available to Buyer in the Electronic Data Room true, correct and complete copies of the Assigned Contracts (including amendments thereto).  The Assigned Contracts are valid and binding obligation of the parties thereto, enforceable in accordance with their terms, except as enforceability may be limited by bankruptcy, insolvency, moratorium or other similar laws affecting or relating to creditors’ rights generally, or general principles of equity.  As applicable, Seller has duly performed all of its obligations under the Assigned Contracts to the extent that such obligations to perform have accrued; and no breach or default by Seller, alleged breach or default by Seller, or event which would (with the passage of time, notice or both) constitute a breach or default by Seller thereunder has occurred.  Seller has not received written notice of default or breach under the Assigned Contracts.  Assuming the receipt of all consents to assign the Assigned Contracts, the execution, delivery and performance of this Agreement or any Ancillary Agreement and the consummation of the transactions contemplated hereby and thereby will not result in a breach of or default under any Assigned

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Contract, will not terminate any rights of, or accelerate any obligation of, Seller under any Assigned Contract and do not require any consent, approval, waiver or other action by any party to any Assigned Contract.  

4.10      Intellectual Property.

(a)          Seller is the sole and exclusive owner of all right, title and interest in the Cholic Acid Product IP.  

(b)          Seller has sufficient right to transfer and convey and is not obligated to pay, and immediately following the Closing Date, Buyer will not be obligated to pay, any Person any royalty, fee or other consideration with respect to the use of the Cholic Acid Product IP, other than the Consideration payable to Seller pursuant to Article 3.  Without limiting the generality of the last sentence of Section 4.3, Seller has not previously granted any rights to any Third Party that conflict with or are otherwise inconsistent with conveyance of the Cholic Acid Product IP to Buyer as provided herein and further represent and warrant that, except as set forth in this Agreement and the Ancillary Agreements, the Seller has not entered into any agreement pursuant to which it has assigned or otherwise disposed of any interest it has in, to, or under any Cholic Acid Product IP, or has agreed to do any of the foregoing in the future.

(c)          No written claim has been received by Seller or, to Seller’s Knowledge are there any facts or circumstances which would result in receipt of a claim against Seller, nor has Seller received written notice of any threatened claim with respect to any Cholic Acid Product IP that alleges that such Intellectual Property, or the use or exploitation thereof, infringes or misappropriates the Intellectual Property rights of any Third Party, and Seller has not threatened or initiated any claim against any Third Party alleging that such Third Party infringes or has misappropriated any Cholic Acid Product IP.

(d)          To the Knowledge of Seller, Seller has taken reasonable measures to protect and preserve the confidentiality of any trade secrets included in the Cholic Acid Product Know-How.

(e)          None of the Cholic Acid Product IP (i) is the product or subject of any joint development activity or agreement with any Third Party; (ii) is the subject of any consortia agreement or cross-license; and/or (iii) has been financed in whole or in part by any Third Party.  To the Knowledge of Seller, Seller has not used any Intellectual Property in connection with the commercialization of the Cholic Acid Product  that Seller does not own and that Buyer is not free to use without liability, subject to the terms of this Agreement.

(f)          To the Knowledge of Seller, no invention included in the Cholic Acid Product IP, including the manufacture or use thereof, infringes or misappropriates any Intellectual Property right of any Third Party.

4.11       Cholic Acid Product Inventory.  

(a)          To the Knowledge of Seller, all of the Cholic Acid Product Inventory (i) meets the specifications therefor, and (ii) is free from known defects and damage and is usable in the ordinary course.  

(b)          The Cholic Acid Product Inventory Schedule sets forth a true and complete listing of the Cholic Acid Product Inventory held by Seller as of December 30, 2014 by work in process inventory and finished product.

4.12       Solvency.  Upon and immediately following the Closing Date, after giving effect to all of the transactions contemplated by and in this Agreement (including the payment of the Effective Date Payment and the assumption by Buyer of the Assumed Liabilities in accordance herewith), to Seller’s Knowledge, Seller will not be insolvent and will have sufficient capital to continue in business and pay its debts as they become due.

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4.13       Absence of Certain Practices.  To Seller’s Knowledge, no director, manager, officer or employee of Seller or other Person acting on Seller’s behalf, directly or indirectly, has given, made or agreed to give or make any material or illegal commission, payment, gratuity, gift, political contribution or other similar benefit to any employee or official of any governmental entity or any other Person who is or may be in a position to help or hinder such Seller or assist such Seller in connection with any proposed transaction.

4.14     Brokers, Finders, Etc.  Seller has not entered into any brokerage or other agreement contemplating commissions or other payments payable upon sale or conveyance of the Assets as provided herein or otherwise upon consummation of the transactions contemplated hereby.  All negotiations relating to this Agreement and the transactions contemplated hereby have been carried on without the intervention of any Person acting on behalf of Seller in such manner as to give rise to any valid claim against Buyer for any brokerage or finder’s commission, fee, or similar compensation.

4.15       Reliance.  Seller recognizes and agrees that, notwithstanding any investigation by Buyer, Buyer is relying upon the representations and warranties made by Seller in this Article 4.

4.16       No Filing Misrepresentations; Cholic Acid Product Approvals and Commitments in the US.  

(a)          To the Seller’s Knowledge, Seller has not, with respect to the Cholic Acid Product:  (a) made any untrue statement of material fact or fraudulent statement to the FDA, EMA, or any other equivalent foreign agency; (b) failed to timely disclose a material fact required to be disclosed to the FDA, EMA, or any other or any equivalent foreign agency; or (c) committed an act, made a statement, or failed to make a statement that would reasonably be expected to provide the basis for the FDA to invoke its policy respecting “Fraud, Untrue Statements of Material Facts, Bribery, and Illegal Gratuities,” as set forth in 56 Fed. Reg. 46191 (September 10, 1991).

(b)          Except for any information or materials with respect to the CTX Indication, Seller has made available to Buyer in the Electronic Data Room all material correspondence between Seller and the FDA (including submission cover sheets) relating to the Seller’s submission for approval and approval of the Cholic Acid Product NDA in the United States since January 1, 2012, as well as such additional materials contemplated by such correspondence as were reasonably requested by Buyer, in each case, that relate to the indications for product approval for the Cholic Acid Product NDA, the likelihood of approval of the Cholic Acid Product NDA, the timing of approval of the Cholic Acid Product NDA, the timing of approval of the Cholic Acid Product NDA and any post-approval obligations within the United States.

(c)          Schedule 4.16(c) sets forth a true and complete description or listing of (i) postmarketing requirements (“PMRs”) for studies and clinical trials that sponsors are required to conduct under Applicable Law, and (ii) postmarketing commitments (“PMCs”) for studies or clinical trials that a sponsor has agreed to conduct, but that are not required by Applicable Law.

4.17       Development Plans.  Seller is not developing, and, to the Seller’s Knowledge, Seller’s principle investigators, […***…], are not […***…].

4.18       Exclusive Representations and Warranties.         Other than the representations and warranties set forth in this Article 4 of this Agreement or in any Ancillary Agreement, Seller is not making any representations or warranties, express or implied.

***Certain Confidential Information Omitted

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ARTICLE 5
REPRESENTATIONS AND WARRANTIES OF BUYER

Buyer hereby represents and warrants to Seller as of the Effective Date and as of the Closing Date as follows:

5.1         Organization.Buyer is organized, validly existing and in good standing under the laws of state of Delaware.  Buyer has the requisite power and authority to own, lease and operate the properties now owned, leased and operated by it and to carry on its businesses as currently conducted.  Buyer is duly qualified to do business as a foreign entity in each jurisdiction in which the nature of its business or the character of its properties makes such qualification necessary, except where the failure to do so would not have a material adverse effect on Buyer.

5.2         Authority and Enforceability.Buyer has the requisite power and authority to enter into this Agreement and each Ancillary Agreement to which it is a party and to perform its obligations hereunder and thereunder.  Buyer (including its board of directors) has taken all necessary action on its part to authorize the execution and delivery of this Agreement and each Ancillary Agreement to which it is a party, and the performance of its obligations hereunder and thereunder.  No vote of Buyer’s stockholders is needed to approve this Agreement, each Ancillary Agreement to which Buyer is a party or the transactions contemplated hereby, including the issuance of any shares of common stock to Seller.  This Agreement and each Ancillary Agreement to which it is a party has been duly and validly executed and delivered by Buyer, and is the legal, valid and binding obligation of Buyer, enforceable against Buyer in accordance with its terms.

5.3        No Violation, Etc.The execution and delivery of this Agreement and each Ancillary Agreement to which it is a party and the performance of the obligations hereunder and thereunder by Buyer does not and will not (a) violate or conflict with any provision of the charter documents of Buyer, (b) violate, or conflict with, or result in a breach of any provision of, or constitute a default or give rise to any right of termination, cancellation or acceleration (with the passage of time, notice or both) under any agreement, lease, instrument, obligation, understanding or arrangement, oral or written, to which Buyer or its Affiliate is a party or by which any of Buyer’s properties or assets is subject, or (c) violate any Applicable Law which Buyer or any of its properties or assets are subject.

5.4        No Consents and Approvals.No permit, consent, approval or authorization of, or notice, declaration, filing or registration with, any governmental authority or Third Party is or will be necessary in connection with the execution and delivery by Buyer of this Agreement and each Ancillary Agreement to which it is a party or the performance by Buyer of its obligations hereunder and thereunder.

5.5         Brokers, Finders, Etc.  Buyer has not entered into any brokerage or other agreement contemplating commissions or other payments payable upon sale or conveyance of the Assets as provided herein or otherwise upon consummation of the transactions contemplated hereby.  All negotiations relating to this Agreement and the transactions contemplated hereby have been carried on without the intervention of any Person acting on behalf of Buyer in such manner as to give rise to any valid claim against Seller for any brokerage or finder’s commission, fee, or similar compensation.

5.6         SEC Reporting.  Buyer has timely filed all reports, schedules, forms, statements and other documents required to be filed by Buyer (hereinafter “SEC Reports”) under the Securities Act of 1933, as amended, and the rules and regulation promulgated thereunder (the “Securities Act”) and the Exchange Act of 1934, as amended, and the rules and regulations promulgated thereunder (the “Exchange Act”).  As of their respective dates, the SEC Reports complied in all material respects with the requirements of the Exchange Act, or the Securities Act, as the case may be, and the rules and regulations of the U.S. Securities and Exchange Commission promulgated thereunder. None of the SEC Reports, including any financial statements or schedules included or incorporated by reference therein (the “Financial Statements”), at the time filed or, if amended or superseded by a subsequent filing, as of the date of the last such amendment or superseding filing, contained any untrue statement of a material fact or omitted to state a material fact required to be stated therein or necessary in order to make the statements therein, in light of the circumstances under which they were made, not misleading.  The Financial Statements and the related notes have been prepared in accordance with accounting principles

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generally accepted in the United States, consistently applied, during the periods involved (except (i) as may be otherwise indicated in the Financial Statements or the notes thereto, or (ii) in the case of unaudited interim statements, to the extent they may not include footnotes, may be condensed or summary statements or may conform to the SEC’s rules and instructions for Quarterly Reports on Form 10-Q) and fairly present in all material respects the consolidated financial position of Buyer and its subsidiaries as of the dates thereof and the consolidated results of its operations and cash flows for the periods then ended (subject, in the case of unaudited statements, to normal year-end audit adjustments).

5.7        Financing.  Buyer has, and will on the Closing Date have, sufficient funds to consummate the transactions contemplated by this Agreement, and Buyer understands that under the terms of this Agreement, Buyer’s consummation of the transactions contemplated hereby is not in any way contingent upon or otherwise subject to (a) Buyer’s consummation of any financial arrangements or Buyer’s obtaining of any financing or (b) the availability, grant, provision or extension of any financing to Buyer.  Buyer has and reasonably expects that Buyer, its Affiliates and/or their respective successors and assigns will maintain for so long as it commercializes the Cholic Acid Product, appropriate financing or sources of liquidity to commercialize the Cholic Acid Product in the United States and the rest of the world consistent with the provisions of this Section 3.6(e);

5.8         Compliance with Law.  Buyer has complied with the Applicable Laws with respect to the development, promotion, marketing and sales of its products, and has conducted, and, to Buyer’s knowledge, each of Buyer’s contractors or consultants have conducted, all development and commercialization activities related to the development, promotion, marketing and sales of its products with the Applicable Laws, except, in each case, where the failure to comply would not have a material adverse effect on the business, financial condition or results of operation of the Buyer, taken as a whole.

5.9         Reliance.  Buyer recognizes and agrees that, notwithstanding any investigation by Seller, Seller are relying upon the representations and warranties made by Buyer in this Article 5. Without limiting the representations or warranties of Seller set forth in Article 4, Buyer or its representatives have inspected and conducted such reasonable review and analysis of the Assets and the Cholic Acid Product Inventory and the Assumed Liabilities as desired by Buyer.  The purchase of the Assets and Cholic Acid Product Inventory and the assumption of the Assumed Liabilities by Buyer and the consummation of the transactions contemplated hereunder by Buyer are not done in reliance upon any warranty or representation by, or information from, Seller or its Affiliates or their respective representatives of any sort, oral or written, except the warranties and representations specifically set forth in this Agreement (including the schedules and exhibits hereto).  

5.10       Exclusive Representations and Warranties.  Other than the representations and warranties set forth in this Article 5 of this Agreement or in any Ancillary Agreement, Buyer is not making any representations or warranties, express or implied.

5.11       No Knowledge of Breach.  To the Buyer’s knowledge, there exists no fact, circumstance or matter which may constitute a breach of any representation or warranty contained in this Agreement by Seller, including any schedule attached hereto.

5.12       Disclaimer.  Buyer acknowledges that Seller makes no representation, warranty or guaranty under this Agreement, including pursuant to the representations and warranties contained in Article 4, and expressly disclaims all warranties of any kind, concerning the receipt (if at all) of the Cholic Acid Product NDA or the Voucher or the indications for which the Cholic Acid Product NDA may be granted (if at all).

ARTICLE 6
COVENANTS AND AGREEMENTS

6.1         Additional Deliveries.  For no additional consideration, from time to time, on and after the Closing Date, at Buyer’s reasonable request, Seller shall, and shall cause its Affiliates to, execute and deliver such additional or confirmatory instruments, documents of conveyance, endorsements, assignments and acknowledgments as are reasonably necessary to evidence or vest in Buyer sole and exclusive title in and to the Assets.

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6.2         Additional Assistance.  For no additional consideration, from time to time, on and after the Closing Date, at Buyer’s request, Seller and its Affiliates shall provide reasonable assistance and cooperation to Buyer in connection with the conveyance of the Assets and enforcing and defending statutory protections in and to any Cholic Acid Product IP, and Seller hereby irrevocably designates and appoints Buyer as its agent and attorney-in-fact, coupled with an interest, to act for and on Seller’s behalf to execute and file any document and to do all other lawfully permitted acts to further the foregoing with the same legal force and effect as if executed by Seller.

6.3         Noncompetition.  During the Competition Period, the Seller and its Affiliates shall not, without the prior written consent of Buyer, directly or indirectly, not, (A) manufacture, produce, market, commercialize or supply any Competitive Product, or (B) acquire, own an interest in, manage, operate, join, control, lend money or render financial or other assistance to or participate in or be connected with, as an officer, employee, partner, stockholder, consultant or otherwise, any Person that competes in manufacturing, producing, marketing or supplying any Competitive Product.  The Parties hereto agree that the covenant set forth in this Section 6.3 is reasonable with respect to its duration and scope and necessary to protect the legitimate interests of Buyer, and that any violation thereof would cause irreparable injuries.  Therefore, Seller, on behalf of itself and its Affiliates, acknowledges and agrees that, in the event of a violation by Seller or its Affiliates of any of the restrictions contained in this Section 6.3, Buyer shall be entitled to obtain from any court of competent jurisdiction temporary, preliminary and permanent injunctive relief, in addition to any other rights Buyer may be entitled.  If the final judgment of any such court declares that any term or provision of this Section 6.3 is invalid or unenforceable, the Parties agree that the court making the determination of invalidity or unenforceability shall have the power to reduce the scope, duration, or area of the term or provision, to delete specific words or phrases, or to replace any invalid or unenforceable term or provision with a term or provision that is valid and enforceable and that comes closest to expressing the intention of the invalid or unenforceable term or provision, and this Section 6.3 shall be enforceable as so modified.

6.4         Supplemental Disclosure.  Seller may until the Closing Date promptly supplement or amend the Seller’s Disclosure Schedule with respect to any matter hereafter arising or discovered that, if existing or known at the date of this Agreement, would have been required to be set forth or described in the Seller’s Disclosure Schedule.  In the event that such supplemented or amended Seller’s Disclosure Schedule reflects any event, condition or circumstance occurring or arising that is not otherwise prohibited pursuant to Sections 7.1 or 7.2 and which does not have a Material Adverse Effect on the Assets, then prior to the Closing, the specified representations and warranties made by Seller will be deemed automatically modified to reflect such event as of the date that such event occurs or arises.  The delivery of any such supplemented or amended Seller’s Disclosure Schedule pursuant to this Section 6.4 will be deemed to have cured any misrepresentation or breach of warranty that otherwise might have existed hereunder by reason of such event, condition or circumstance and Buyer will not be entitled to terminate this Agreement nor will any Indemnitee of Buyer have any claim to indemnification or reimbursement for any such event.

6.5        Cooperation with Financials.  For 90 days after the Closing Date, Seller shall cooperate, at the sole cost and expense of Buyer, with all reasonable requests of Buyer in connection with preparation of any financial statements for the Assets as may be required by Buyer in connection with disclosure obligations under the U.S. Securities Exchange Act of 1934, as amended, or the rules or regulations promulgated thereunder or any applicable stock exchange rules, including such requests necessary in order (a) to determine whether financial statements for the Buyer with respect to the Assets are required to be prepared under Rule 3-05 of Regulation S-X under the Securities Exchange Act of 1934 (“Post-Closing Financials”), and (b) if Post-Closing Financials statements are required, to prepare and file such financial statements, including assistance in obtaining audited financials of Seller and the associated consent of any auditors of the Seller; provided, however, that the Seller shall have no responsibility or liability for (and Buyer shall indemnify, defend and hold harmless the Seller in respect of) any and all Losses that may be suffered or incurred as a result of such financial statements.

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6.6         Material Supplier Contracts.  Between the Effective Date and the Closing Date, Seller shall not, and shall cause its subsidiaries not to, enter into or amend any of the following contracts without the prior written consent of Buyer (which consent shall not be unreasonably withheld, conditioned or delayed by Buyer) (the “Material Supplier Contracts”):

(a)          […***…]

(b)          […***…]

(c)          […***…]

Buyer shall be deemed for purposes of this Section 6.6 to have consented to Seller’s entry into or amendment of any Material Supplier Contract if Buyer shall fail to notify Seller in writing of any objections to such entry into or amendment of any Material Supplier Contract within five days after Seller requests such consent in writing from Buyer.  Between the Effective Date and the Closing Date, Seller will keep Buyer reasonably informed of the status and occurrence of material negotiations with respect to any Material Supplier Contract or any amendment thereto, as well as material drafts or material changes in the drafts of any Material Supplier Contract or any amendment thereto.

6.7         Allocation of Expenses and Receivables.  Following the Closing:

(a)          Allocable Expenses.  All accounts payable and expenses (including, without limitation, accounts payables to the Suppliers identified under the arrangements described under the heading “Cholic Acid Commercial Supply Chainas of December 30, 2014” and the expenses related to the […***…] Matter)  arising out of or relating to the Assets (such accounts payable and expenses, the “Allocable Expenses”) in respect of the periods prior to and following the Closing shall be prorated and apportioned as follows:  (A) to Asklepion for all periods prior to (but not including) the Closing Date, and (B) to Retrophin for all periods from and after the Closing Date (which shall include the Closing Date).  The payment of any Allocable Expenses subject to proration pursuant to this Section 6.7(a) shall be the responsibility of the Party required to pay such Allocable Expense pursuant to this Section 6.7(a).  

(b)          Allocable Receivables.  All accounts receivable arising out of or relating to the Assets (such accounts receivable, the “Allocable Receivables”) in respect of the periods prior to and following the Closing shall be prorated and apportioned as follows:  (A) to Asklepion for all periods prior to (but not including) the Closing Date, and (B) to Retrophin for all periods from and after the Closing Date (which shall include the Closing Date).  The collection of any Allocable Receivables subject to proration pursuant to this Section 6.7(b) shall be the responsibility of the Party entitled to be paid such Allocable Receivable pursuant to this Section 6.7(b).  If Asklepion collects or receives any Allocable Receivable to which Retrophin is entitled to be paid pursuant to this Section 6.7(b), then Asklepion shall promptly remit the same to Retrophin, at no cost or expense to Retrophin.  Additionally, if Retrophin collects or receives any Allocable Receivable to which Asklepion is entitled to be paid pursuant to this Section 6.7(b), then Retrophin shall promptly remit such Allocable Receivable to Asklepion, at no cost or expense to Asklepion.

ARTICLE 7
CONDITIONS PRECENDENT; CLOSING DATE

7.1    Conditions Precedent of Buyer and Seller.  Each of the Party’s obligations to consummate the transactions contemplated by this Agreement are subject to the satisfaction or waiver, at or prior to the Closing Date, of each of the following conditions precedent:

(a)          No Injunctions or Restraints.  No temporary restraining order, preliminary or permanent injunction or other material legal restraint or prohibition issued or promulgated by a governmental authority preventing the consummation of the transactions contemplated by this Agreement shall be in effect, and there shall not be any Applicable Law that makes consummation of the transactions contemplated by this Agreement illegal.

***Certain Confidential Information Omitted

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(b)          No Governmental Litigation.  There shall not be any litigation, proceeding, arbitration, or known investigation commenced by a governmental authority seeking to prohibit, limit, delay, or otherwise restrain the consummation of this Agreement and the transactions contemplated by this Agreement.

7.2       Buyer’s Conditions Precedent.  The obligations of Buyer to consummate the transactions contemplated by this Agreement are subject to the satisfaction or waiver, at or prior to the Closing Date, of each of the following conditions precedent:

(a)          Accuracy of Representations.  Each of the representations and warranties made by Seller in this Agreement shall be true and correct in all material respects at and as of the Closing Date (or, if made as of a specified period or date, as of such period or date), except to the extent that such representations and warranties are qualified by the term “material”, or words of similar import, in which case such representations and warranties (as so written, including the terms “material”, or words of similar import) shall be true and correct in all respects at and as of the Closing Date (or, if made as of a specified period or date, as of such period or date), and the Seller shall have delivered to Buyer a certificate certifying to the effect of the foregoing.  

(b)          Performance of Covenants.  All of the covenants and obligations that Seller is required to comply with or to perform at or prior to the Closing Date shall have been complied with and performed in all material respects, and the Seller shall have delivered to Buyer a certificate certifying to the effect of the foregoing has been satisfied.

(c)          Transaction Documents.  Seller shall have executed and delivered to Buyer all Ancillary Agreements to which it is a party.

(d)          Required Consents.  Seller shall have obtained and delivered to Buyer all consents, approvals, or waivers, if any, listed on Schedule 7.2(d) of the Seller’s Disclosure Schedules.

(e)          FDA approval of the Cholic Acid Product NDA  Seller shall have obtained FDA approval of the Cholic Acid Product NDA for the Bile Acid Indications.  

(f)          Materials relating to the CTX Indication.  Seller shall have made available to Buyer at or immediately prior to the Closing, information or materials, including, but not limited to, correspondence, reports and filings with FDA and clinical trials and data generated therefrom, with respect to the CTX Indication.

7.3        Seller’s Conditions Precedent.  The obligations of Seller to consummate the transactions contemplated by this Agreement are subject to the satisfaction or waiver, at or prior to the Closing Date, of each of the following conditions precedent:

(a)          Accuracy of Representations.  Each of the representations and warranties made by Buyer in this Agreement shall be true and correct in all material respects at and as of the Closing Date (or, if made as of a specified period or date, as of such period or date), except to the extent that such representations and warranties are qualified by the term “material”, or words of similar import, in which case such representations and warranties (as so written, including the terms “material”, or words of similar import) shall be true and correct in all respects at and as of the Closing Date (or, if made as of a specified period or date, as of such period or date), and the Buyer shall have delivered to Seller a certificate certifying to the effect of the foregoing.

(b)          Performance of Covenants.  All of the covenants and obligations that Buyer is required to comply with or to perform at or prior to the Closing Date shall have been complied with and performed in all material respects, and the Buyer shall have delivered to Seller a certificate certifying to the effect of the foregoing has been satisfied.

(c)          Transaction Documents.  Buyer shall have executed and delivered to Seller all Ancillary Agreements to which it is a party.

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(d)          Effective Date Payment.  Buyer shall have made the Effective Date Payment in accordance with Section 3.2.

(e)          U.S. Commercialization Plan.  Buyer shall have delivered to Seller at or immediately prior to Closing, the U.S. Commercialization Plan for calendar year 2015.

7.4         Closing Date.  The consummation of the transactions contemplated by this Agreement (the “Closing”) shall be conducted telephonically and/or via email, facsimile transfer or other similar means of correspondence on such date to be mutually agreed upon by Buyer and Seller, which date shall be no later than the third business day after all of the conditions set forth in Sections 7.1, 7.2 and 7.3 of this Agreement have been satisfied or waived (other than those conditions which, by their terms, are intended to be satisfied at the Closing), or at such other time and place as Buyer and Seller shall mutually agree.  The date on which the Closing actually takes place is referred to in this Agreement as the “Closing Date.”  

ARTICLE 8
INDEMNIFICATION

8.1         By Seller. From and after the Closing Date, to the extent provided in, and subject to the limitations set forth in, this Article 8, Seller shall indemnify, defend and hold harmless Buyer and its Affiliates and their respective officers, directors, employees, agents, successors and assigns (the “Buyer Indemnitee Group”) from and against any Third Party claims, suits or proceedings and any damages and/or liabilities therefrom or settlement thereof (including reasonable fees of attorneys and court costs) (collectively, “Losses”) to the extent arising out of or related to (a) any breach of any representation, warranty made by Seller contained in herein, (b) any breach in the performance of any covenant or agreement of Seller contained in this Agreement, (c) any payment obligations under any “bulk transfer” law or similar Applicable Law applicable to the transfer of the Assets to Buyer, and (d) any Excluded Liability.  

8.2        By Buyer.  From and after the Closing Date, to the extent provided in this Article 8 , Buyer shall indemnify, defend and hold harmless Seller and its Affiliates and their respective officers, directors, employees, agents, successors and assigns (the “Seller Indemnitee Group” and together with the Buyer Indemnitee Group, the “Indemnitee Groups” and each, and “Indemnitee Group”) from and against any Losses to the extent arising out of or related to (a) any breach of any representation, warranty made Buyer contained in this Agreement, (b) any breach in the performance of any covenant or agreement of Buyer contained in this Agreement, (c) any Losses indemnifiable under Section 6.5, and (d) any Assumed Liability.

8.3     Indemnification Procedures.  An Party (the “Indemnitee”) that intends to claim indemnification under this Article 8 shall promptly notify the other Party (the “Indemnitor”) in writing of any action, claim or liability in respect to which the Indemnitee or any member of its Indemnitee Group intends to claim such indemnification.  The Indemnitee shall permit and shall cause its employees and agents to permit, the Indemnitor, at its discretion, to settle any such action, claim or liability and agrees to the complete control of such defense or settlement by the Indemnitor; provided, however, that such settlement does not materially and adversely affect the Indemnitee’s rights hereunder or impose an injunction or equitable relief against the Indemnitee or to compel the Indemnitee to take any action.  No such action, claim or liability shall be settled by the Indemnitee without the prior written consent of the Indemnitor (which consent shall not be unreasonably withheld, delayed or conditioned), and the Indemnitor shall not be responsible for any fees or other costs incurred other than as provided herein.  The Indemnitee, its employees, agents and Affiliates shall cooperate fully with the Indemnitor and its legal representatives in the investigation and defense of any action, claim or liability covered by this indemnification.  The Indemnitee shall have the right, but not the obligation to be represented by counsel of its own selection at its own expense.

8.4         Limitations on Indemnification.

(a)          The representations, warranties and covenants of the Parties in this Agreement shall survive the Closing Date and continue in full force and effect for a period of twelve (12) months thereafter; provided that (i) claims related to breaches by Seller of the representations and warranties

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contained in Section 4.16(a) […***…], (ii) claims related to fraud or willful or intentional misconduct shall survive the Closing Date until the expiration of the date on which the statute of limitations otherwise applicable to such claims has expired, and (iii) any covenants or agreements contained in this Agreement that by their terms are to be performed after the Closing Date shall survive until fully discharged.  For the avoidance of doubt, Retrophin’s obligations to make any Contingent Payment or Royalty payment contemplated by the covenants set forth in Sections 3.3 or 3.4, respectively, shall survive the Closing Date for so long as Retrophin has Net Revenues or Product is otherwise sold.  

(b)          The Seller shall not be obligated to provide indemnification for Losses in respect of claims made under Section 8.1 unless and until the aggregate of the Losses exceeds […***…] ($[…***…]) (the “Basket”), after which point Seller shall be liable for all such Losses dollar for dollar in excess of the Basket, but only to the extent that Losses do not exceed […***…] ($[…***…]) (the “Cap Amount”); provided, however, that the Basket and Cap Amount shall not apply, and all Losses of the Buyer Indemnitee Group shall be immediately subject to indemnification, in respect of any Loss (but shall not exceed […***…] ($[…***…]) in the aggregate with respect to (i) claims related to any breach of any representation and warranty contained in Sections 4.2, 4.7, 4.10(b), 4.11(b), 4.16(b) and 4.16(c), and 4.16(a) (but solely to the extent that breaches by Seller of the representations and warranties contained in Section 4.16(a) […***…], such that any and all other breaches by Seller of the representations and warranties contained in Section 4.16(a) shall be subject to the Basket and Cap Amount) (ii) claims related to fraud or willful or intentional misconduct, or (iii) claims made under Section 8.1(c) or (d); provided, further, that any and all such Losses of the Buyer Indemnitee Group described in the foregoing proviso shall be applied against the Cap for purposes of calculating the Seller’s aggregate liabilities under this Section 8.4(b).  In no event shall the Seller be liable for Losses under this Agreement in an aggregate amount greater than […***…] ($[…***…]) in the aggregate.

(c)          The amount of any and all Losses will be determined net of any amounts recovered by the Buyer Indemnitee Group under insurance policies (net of any deductible or self-insurance retention amounts and any increases in premiums resulting therefrom) and any indemnity, contribution or similar payment actually recovered by the Buyer Indemnitee Group thereof from any Third Party with respect to such Losses.  Each Indemnitee Group shall use commercially reasonable efforts to mitigate all Losses suffered by it which are subject to indemnification hereunder.

(d)          No Indemnity Group shall be entitled to indemnification pursuant this Article 8 for punitive damages, lost profits, consequential, exemplary or special damages.  No Indemnitee Group shall be entitled to any duplicative recovery for the same Loss under this Article 8 to the extent that any such member of such Indemnitee Group has been expressly compensated for such Loss.

(e)          All indemnification payments made pursuant to this Article 8 shall be treated for tax purposes as adjustments to the Consideration unless otherwise required by Applicable Law.

(f)          Buyer acknowledges and agrees that any and all Losses of the Buyer Indemnitee Group in respect of any breach by Seller of the representations and warranties contained in Section 4.11(a) will be recoverable by Buyer solely from […***…].

8.5         Exclusive Remedy.  The Parties acknowledge and agree that, except with respect to claims based on fraud or intentional or willful misrepresentation, claims involving specific performance or other equitable remedies or relief permitted under this Agreement or the Ancillary Agreements, claims involving Buyer’s failure to make any payment when due under Article 3, claims involving a breach of Buyer’s obligations pursuant to Section 3.6(e), or claims involving a breach of Seller’s obligations pursuant to Section 6.3 hereof, the foregoing indemnification provisions in this Article 8 shall be the exclusive remedy for any breach of this Agreement or the Ancillary Agreements and any claims with respect to the transactions contemplated hereby.

***Certain Confidential Information Omitted

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ARTICLE 9
TERMINATION

9.1         Termination Prior to Closing Date.  Notwithstanding any contrary provisions of this Agreement, the respective obligations of the Parties hereto to consummate the transactions contemplated by this Agreement may be terminated and abandoned at any time at or before the Closing Date only as follows:

(a)          At any time, without liability of any Party to the others, upon the mutual written consent of the Buyer and Seller

(b)          At any time, upon the mutual written consent of the Buyer and Seller in accordance with Section 3.3(a); or

(c)          By either Buyer or Seller, if Seller, on the one hand, or Buyer, on the other hand, has materially breached any representation, warranty, covenant or agreement contained herein (provided that such breach is not the result of any breach of any covenant, representation or warranty by the terminating Party), which breach has not been cured within 30 calendar days following written notice of such breach by the terminating Party, and such breach renders the conditions precedent to the terminating Party’s obligation to consummate the transactions contemplated by this Agreement, set forth in Article 7 incapable of being satisfied.

9.2      Termination After Closing Date.  In addition, following the Closing Date, this Agreement may be terminated by the Seller if the FDA Approval Milestone for the Bile Acid Indications is not paid by Buyer in full on or prior to the FDA Approval Milestone Payment Date (after expiration of the 7 day cure period).

9.3          Effect of Termination.  In the event of the termination of this Agreement as provided in Sections 9.1 or 9.2, written notice thereof shall forthwith be given to the other party hereto specifying the provision hereof pursuant to which such termination is made, and this Agreement shall forthwith become null and void (except for the provisions of this Section 9.3, the payment made pursuant to Section 3.2, which Seller shall be entitled to retain, Article 10 and Article 11, which shall survive any such termination and, in the case of termination of this Agreement pursuant to Section 9.2, the additional reversion by Buyer to Seller of all right, title and interest and to the Assets to Seller) and there shall be no liability on the part of Buyer or Seller, except for (a) in the case of termination of this Agreement pursuant to Section 9.2, any rights of Seller to reversion by Buyer to Seller of all right, title and interest and to the Assets to Seller, or (b) damages resulting from any breach of this Agreement or any Ancillary Agreement by Buyer or Seller.

ARTICLE 10
DISPUTE RESOLUTION

10.1       Consent to Jurisdiction; Venue; Service of Process.  Each Party hereto, by its execution hereof, (i) hereby irrevocably submits to the exclusive jurisdiction of any New York federal court sitting in the Borough of Manhattan of The City of New York for the purpose of any claim, action, suit, or proceeding among the Parties arising in whole or in part under or in connection with this Agreement (a “Dispute”); provided, however, that if such federal court does not have jurisdiction over such Dispute, such Dispute shall be heard and determined exclusively in any New York state court sitting in the Borough of Manhattan of The City of New York, (ii) hereby waives to the extent not prohibited by Applicable Law, and agrees not to assert, by way of motion, as a defense or otherwise, in any such Dispute, any claim that it is not subject personally to the jurisdiction of the above-named courts, that its property is exempt or immune from attachment or execution, that any such Dispute brought in one of the above-named courts should be dismissed on grounds of forum non conveniens, should be transferred or removed to any court other than one of the above-named courts, or should be stayed by reason of the pendency of some other proceeding in any other court other than one of the above-named courts, or that this Agreement or any of the other Ancillary Agreements or the subject matter hereof and thereof may not be enforced in or by such court, and (iii) hereby agrees to commence any such Dispute only before one of the above-named courts.  Notwithstanding the immediately preceding sentence, a party may commence

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any Dispute in a court other than the above-named courts solely for the purpose of enforcing an order or judgment issued by one of the above-named courts.

10.2       Waiver of Jury Trial.  TO THE EXTENT NOT PROHIBITED BY APPLICABLE LAW THAT CANNOT BE WAIVED, THE PARTIES HERETO HEREBY WAIVE, AND COVENANT THAT THEY WILL NOT ASSERT (WHETHER AS PLAINTIFF, DEFENDANT OR OTHERWISE), ANY RIGHT TO TRIAL BY JURY IN ANY DISPUTE ARISING IN WHOLE OR IN PART UNDER OR IN CONNECTION WITH THIS AGREEMENT, THE OTHER ANCILLARY AGREEMENTS OR ANY OF THE CONTEMPLATED TRANSACTIONS, WHETHER NOW EXISTING OR HEREAFTER ARISING, AND WHETHER SOUNDING IN CONTRACT, TORT OR OTHERWISE. THE PARTIES HERETO AGREE THAT ANY OF THEM MAY FILE A COPY OF THIS PARAGRAPH WITH ANY COURT AS WRITTEN EVIDENCE OF THE KNOWING, VOLUNTARY AND BARGAINED-FOR AGREEMENT AMONG THE PARTIES IRREVOCABLY TO WAIVE ITS RIGHT TO TRIAL BY JURY. ANY DISPUTE WHATSOEVER AMONG THEM RELATING TO THIS AGREEMENT, THE OTHER ANCILLARY AGREEMENTS OR ANY OF THE CONTEMPLATED TRANSACTIONS SHALL INSTEAD BE TRIED IN A COURT OF COMPETENT JURISDICTION BY A JUDGE SITTING WITHOUT A JURY.

10.3       Consent to Service of Process.  Each Party hereto hereby agrees that service of any process, summons, notice or document by U.S. registered mail, return receipt requested, at its address specified pursuant to Section 11.8 shall constitute good and valid service of process in any Dispute among the Parties hereto arising in whole or in part under or in connection with this Agreement or any other Ancillary Agree, and each Party hereto hereby waives and agrees not to assert (by way of motion, as a defense, or otherwise) in any such Dispute any claim that service of process made in accordance with this Section 10.3 does not constitute good and valid service of process.

ARTICLE 11
MISCELLANEOUS

11.1      Confidentiality.  

(a)          Each Party will treat as confidential the Confidential Information of the other Party, and will take all necessary precautions to assure the confidentiality of such Confidential Information.  Each Party agrees to return to the other Party upon the expiration or termination of this Agreement all Confidential Information acquired from such other Party, except as to such information it may be required to retain under Applicable Laws, and except for one copy of such information to be retained by such Party solely to enable it to assess its compliance with the confidentiality provisions of this Section 11.1.  From and after the Effective Date through the period ending […***…] after the Effective Date, neither Party shall, without the other Party’s express prior written consent, use or disclose any such Confidential Information for any purpose other than to carry out its obligations hereunder.  Each Party, prior to disclosure of Confidential Information of the other Party to any employee, consultant or advisor shall ensure that such Person is bound in writing to observe the confidentiality such Party’s Confidential Information on terms no less restrictive than those contained herein.  The obligations of confidentiality shall not apply to Confidential Information that the receiving Party is required by law or regulation to disclose, provided however that the receiving Party shall so notify the disclosing Party of its intent and cooperate with the disclosing Party on reasonable measures to protect the confidentiality of the Confidential Information.  For the avoidance of doubt, either Party may disclose the terms of this Agreement to the extent required, in the reasonable opinion of such Party’s legal counsel, to comply with applicable laws, including, without limitation, the rules and regulations promulgated by the United States Securities and Exchange Commission. Notwithstanding the foregoing, before disclosing this Agreement or any of the terms hereof pursuant to this Section 11.1(a), such Party so required to disclose the terms of this Agreement will consult with the other on the terms of this Agreement to be redacted in making any such disclosure.  If such disclosing Party discloses this Agreement or any of the terms hereof in accordance with this Section 11.1(a), such disclosing Party agrees, at its own expense, to seek confidential treatment of portions of this Agreement or such terms, as may be reasonably requested by the other.  Seller hereby acknowledges and agrees that any Confidential Information of Seller on or before the Closing Date included in the Assets shall be Buyer’s Confidential Information after the Closing Date.

***Certain Confidential Information Omitted

27

(b)          No public announcement, news release, statement, publication, or presentation relating to the existence of this Agreement, the subject matter hereof, or either Party’s performance hereunder will be made without the other Party’s prior written approval, which approval shall not be unreasonably withheld or delayed.  The Parties shall not make any joint announcement, news release, statement, publication, or presentation relating to the existence of this Agreement, the subject matter hereof, or either Party’s performance hereunder, which such announcements, news releases, statements, publications, or presentations shall solely be made separately.  If a Party desires to announce or make any news release, statement, publication, or presentation relating to the existence of this Agreement, the subject matter hereof, or either Party’s performance hereunder and such public announcement, news release, statement, publication, or presentation contains Confidential Information of the other Party, then at least five days in advance of making any such public announcement, news release, statement, publication, or presentation, such Party shall provide a complete copy thereof to the other for its review and prior written approval, which approval shall not be unreasonably withheld, conditioned or delayed.  If the other Party fails to object in writing to all or any portion of such public announcement, news release, statement, publication, or presentation containing Confidential Information of the other Party within five days after being requested to consent thereto, then such Party shall be deemed to have consented to such public announcement, news release, statement, publication, or presentation containing such Confidential Information in whole upon expiration of such 5-day period.

11.2       Counterparts.  This Agreement may be executed in counterparts, each of which shall be deemed an original and all of which shall constitute a single document.

11.3       Entire Agreement.  This Agreement, and the Exhibits and Schedules referenced herein, the Ancillary Agreements and the other specific agreements contemplated herein or thereby, contain the entire agreement between the Parties with respect to the subject matter hereof and supersede all previous agreements, negotiations, discussions, writings, understandings, commitments and conversations with respect to such subject matter.

11.4     Exhibits and Schedules.  The Exhibits and Schedules referenced herein and attached hereto are incorporated into this Agreement by reference.

11.5       Governing Law.  This Agreement shall be governed by and construed and interpreted in accordance with the laws of the State of New York irrespective of the choice of laws principles of the State of New York.

11.6     Assignability.  This Agreement shall be binding upon and inure to the benefit of the Parties and their respective successors and permitted assigns.  No Party may assign its respective rights or delegate its respective obligations under this Agreement without the express prior written consent of the other Party; provided that either Party may assign or transfer this Agreement, to an Affiliate (provided the assigning Party remains liable hereunder), or to any Third Party in connection with the sale or transfer of the business to which this Agreement relates.  Without limiting the foregoing, and for the avoidance of doubt, Buyer may assign or transfer this Agreement, in whole or in part to any Third Party in connection with the sale, license or transfer of any of Buyer’s rights in the Product.  

11.7       Third Party Beneficiaries.  Nothing in this Agreement shall be deemed to create any third party beneficiary rights in or on behalf of any other Person.

11.8       Notices.  All notices required to be given hereunder shall be in writing and shall be given by Personal delivery, by an internationally recognized overnight carrier or by registered or certified mail, postage prepaid with return receipt requested or by email or facsimile transmission.  All notices hereunder shall be addressed as follows:

28

If to Buyer, to:	Retrophin, Inc.
	12255 El Camino Real
	Suite 250
	San Diego, CA 92130
	Attention:  General Counsel
If to Seller, to:	Asklepion Pharmaceuticals, LLC
	729 East Pratt St
	Suite 360
	Maryland 21202, USA
	Attn:  Gary R. Pasternack – Chief Executive Officer

Any Party may, by notice to the other Parties given in the form specified in this Section 11.8, change the address to which such notices are to be given.  Notices delivered Personally shall be deemed communicated as of actual receipt; notices sent via overnight courier shall be deemed received three Business days following sending; notices mailed shall be deemed communicated as of seven (7) business days after mailing; and notices transmitted by email or facsimile transmission shall be deemed received upon return email or electronic facsimile acknowledgement of receipt.  

11.9       Severability.  If any provision of this Agreement shall be held invalid, illegal or unenforceable, the validity, legality or unenforceability of the other provisions of this Agreement shall not be affected thereby, and there shall be deemed substituted for the provision at issue a valid, legal and enforceable provision as similar as possible to the provision at issue.

11.10      Survival.  Except as expressly set forth herein, the covenants, representations and warranties contained in this Agreement, and liability for the breach of any obligations contained herein, shall survive the Closing Date and shall remain in full force and effect.

11.11         No Implied Waiver.  No failure or delay on the part of the Parties hereto to exercise any right, power or privilege hereunder or under any instrument executed pursuant hereto shall operate as a waiver; nor shall any single or partial exercise of any right, power or privilege preclude any other or further exercise thereof or the exercise of any other right, power or privilege.

11.12         Amendments.  Any amendment or modification of this Agreement shall only be valid if made in writing and signed by the Parties hereto.

11.13        Independent Contractors.  The relationship between Seller on the one hand and Buyer on the other had is that of independent contractors and nothing herein shall be deemed to constitute the relationship of partners, joint venturers nor of principal and agent between Seller on the one hand and Buyer on the other hand.

11.14          Expenses.  Except as expressly set forth herein, each Party shall pay all of its own fees and expenses (including all legal, accounting and other advisory fees) incurred in connection with the negotiation and execution of this Agreement and the arrangements contemplated hereby.

11.15         Representation By Counsel; Interpretation.  Seller and Buyer each acknowledge that it has been represented by its own legal counsel in connection with this Agreement and the transactions contemplated by this Agreement.  Accordingly, any rule of law, or any legal decision that would require interpretation of any claimed ambiguities in this Agreement against the Party that drafted it, has no application and is expressly waived.  The provisions of this Agreement shall be interpreted in a reasonable manner to effect the intent of Seller and Buyer.

(SIGNATURE PAGE FOLLOWS)

29

IN WITNESS WHEREOF, the Parties, intending to be bound hereby, have executed this Agreement as of the date first written above.

    “Buyer”

    RETROPHIN, INC.

    By:    /s/ Steve Aselage

    Title:    Chief Executive Officer

    “Seller”

    ASKLEPION PHARMACEUTICALS, LLC

    By:    /s/ Kevin Jackson

    Title:    Chairman Board of Managers 

30

EX-31.1

Exhibit 31.1

CERTIFICATION OF CHIEF EXECUTIVE OFFICER

PURSUANT TO EXCHANGE ACT RULE 13a-14(a) OR 15d-14(a)

I, Eric M. Dube, certify that:

1.    I have reviewed this Quarterly Report on Form 10-Q of Travere Therapeutics, Inc.;

2.    Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.    Based on my knowledge, the financial statements and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.    The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

(a)    Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

(b)    Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

(c)    Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

(d)    Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5.    The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

(a)    All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

(b)    Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.  

Date: August 3, 2023
	/s/ Eric M. Dube
	Eric M. Dube
	Chief Executive Officer
	(Principal Executive Officer)

EX-31.2

Exhibit 31.2

CERTIFICATION OF CHIEF FINANCIAL OFFICER

PURSUANT TO EXCHANGE ACT RULE 13a-14(a) OR 15d-14(a)

I, Christopher Cline, certify that:

1.    I have reviewed this Quarterly Report on Form 10-Q of Travere Therapeutics, Inc.;

2.    Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.    Based on my knowledge, the financial statements and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.    The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

(a)    Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

(b)    Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

(c)    Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

(d)    Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5.    The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

(a)    All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

(b)    Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: August 3, 2023
	/s/ Christopher Cline
	Christopher Cline
	Chief Financial Officer
	(Principle Financial Officer)

EX-32.1

Exhibit 32.1

CERTIFICATION OF

CHIEF EXECUTIVE OFFICER

PURSUANT TO 18 U.S.C. SECTION 1350

AS ADOPTED PURSUANT TO

SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002

In connection with the accompanying Quarterly Report on Form 10-Q of Travere Therapeutics, Inc. (the “Company”), for the period ending June 30, 2023 (the “Report”), the undersigned officer of the Company hereby certifies pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that, to such officer’s knowledge:

1. The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and

2. The information contained in the Report, fairly presents, in all material respects, the financial condition and results of operations of the Company.

Date: August 3, 2023
	/s/ Eric M. Dube
	Eric M. Dube
	Chief Executive Officer
	(Principal Executive Officer)

EX-32.2

Exhibit 32.2

CERTIFICATION OF

CHIEF FINANCIAL OFFICER

PURSUANT TO 18 U.S.C. SECTION 1350

AS ADOPTED PURSUANT TO

SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002

In connection with the accompanying Quarterly Report on Form 10-Q of Travere Therapeutics, Inc. (the “Company”), for the period ending June 30, 2023 (the “Report”), the undersigned officer of the Company hereby certifies pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that, to such officer’s knowledge:

1. The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and

2. The information contained in the Report, fairly presents, in all material respects, the financial condition and results of operations of the Company.

Date: August 3, 2023
	/s/ Christopher Cline
	Christopher Cline
	Chief Financial Officer
	(Principal Financial Officer)

XML

Cover - shares	6 Months Ended
	Jun. 30, 2023	Aug. 01, 2023
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2023
Document Transition Report	false
Entity File Number	001-36257
Entity Registrant Name	TRAVERE THERAPEUTICS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	27-4842691
Entity Address, Address Line One	3611 Valley Centre Drive
Entity Address, Address Line Two	Suite 300
Entity Address, City or Town	San Diego
Entity Address, State or Province	CA
Entity Address, Postal Zip Code	92130
City Area Code	888
Local Phone Number	969-7879
Title of 12(b) Security	Common Stock, par value $0.0001 per share
Trading Symbol	TVTX
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		75,010,865
Entity Central Index Key	0001438533
Current Fiscal Year End Date	--12-31
Amendment Flag	false
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q2

XML

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 70,874	$ 61,688
Marketable debt securities, at fair value	420,463	388,557
Accounts receivable, net	20,397	16,646
Inventory, net	18,765	6,922
Prepaid expenses and other current assets	11,556	12,624
Total current assets	542,055	486,437
Property and equipment, net	8,570	9,049
Operating lease right of use assets	19,559	21,000
Intangible assets, net	154,456	145,038
Other assets	11,789	11,061
Total assets	736,429	672,585
Current liabilities:
Accounts payable	19,915	17,290
Accrued expenses	88,749	95,742
Deferred revenue, current portion	10,244	11,976
Business combination-related contingent consideration, current portion	6,900	7,000
Operating lease liabilities, current portion	4,663	4,433
Other current liabilities	5,240	5,722
Total current liabilities	135,711	142,163
Convertible debt	376,403	375,545
Deferred revenue, less current portion	6,788	10,931
Business combination-related contingent consideration, less current portion	67,200	64,200
Operating lease liabilities, less current portion	25,106	27,510
Other non-current liabilities	8,736	9,385
Total liabilities	619,944	629,734
Commitments and Contingencies (See Note 13)
Stockholders' Equity:
Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of June 30, 2023 and December 31, 2022	0	0
Common stock $0.0001 par value; 200,000,000 shares authorized; 74,971,807, and 64,290,570 issued and outstanding as of June 30, 2023 and December 31, 2022, respectively	7	6
Additional paid-in capital	1,306,517	1,059,975
Accumulated deficit	(1,186,184)	(1,014,223)
Accumulated other comprehensive loss	(3,855)	(2,907)
Total stockholders' equity	116,485	42,851
Total liabilities and stockholders' equity	$ 736,429	$ 672,585

XML

CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Jun. 30, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred stock, shares authorized (in shares)	20,000,000	20,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common stock par value (in dollars per share)	$ 0.0001	$ 0.0001
Common stock shares authorized (in shares)	200,000,000	200,000,000
Common stock shares issued (in shares)	74,971,807	64,290,570
Common stock shares outstanding (in shares)	74,971,807	64,290,570

XML

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Total revenue	$ 59,697	$ 54,167	$ 116,690	$ 102,654
Operating expenses:
Cost of goods sold	1,990	2,051	7,115	4,189
Research and development	69,411	59,681	129,324	116,292
Selling, general and administrative	74,037	52,979	146,282	99,767
Change in fair value of contingent consideration	1,840	4,907	8,596	13,987
Total operating expenses	147,278	119,618	291,317	234,235
Operating loss	(87,581)	(65,451)	(174,627)	(131,581)
Other income (expenses), net:
Interest income	5,128	782	8,774	1,060
Interest expense	(2,911)	(2,972)	(5,851)	(5,487)
Other (expense) income, net	(201)	662	(114)	688
Loss on extinguishment of debt	0	0	0	(7,578)
Total other income (expense), net	2,016	(1,528)	2,809	(11,317)
Loss before income tax provision	(85,565)	(66,979)	(171,818)	(142,898)
Income tax provision	(65)	(53)	(143)	(105)
Net loss	$ (85,630)	$ (67,032)	$ (171,961)	$ (143,003)
Basic net loss per common share (in dollars per share)	$ (1.13)	$ (1.05)	$ (2.38)	$ (2.26)
Diluted net loss per common share (in dollars per share)	$ (1.13)	$ (1.05)	$ (2.38)	$ (2.26)
Basic weighted average common shares outstanding (in shares)	76,001,801	63,638,385	72,109,573	63,387,009
Diluted weighted average common shares outstanding (in shares)	76,001,801	63,638,385	72,109,573	63,387,009
Comprehensive loss:
Net loss	$ (85,630)	$ (67,032)	$ (171,961)	$ (143,003)
Foreign currency translation (loss) gain	(170)	1,416	(736)	1,487
Unrealized loss on marketable debt securities	(1,509)	(803)	(212)	(2,007)
Comprehensive loss	(87,309)	(66,419)	(172,909)	(143,523)
Net product sales
Total revenue	57,012	50,950	107,295	97,393
Operating expenses:
Cost of goods sold	1,983	2,051	4,071	4,189
License and collaboration revenue
Total revenue	2,685	3,217	9,395	5,261
Operating expenses:
Cost of goods sold	$ 7	$ 0	$ 3,044	$ 0

XML

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Total	Underwritten Equity Offering	At-The-Market Offering	Cumulative Effect, Period of Adoption, Adjustment	Common Stock	Common Stock Underwritten Equity Offering	Common Stock At-The-Market Offering	Additional Paid in Capital	Additional Paid in Capital Underwritten Equity Offering	Additional Paid in Capital At-The-Market Offering	Additional Paid in Capital Cumulative Effect, Period of Adoption, Adjustment	Accumulated Other Comprehensive Loss	Accumulated Deficit	Accumulated Deficit Cumulative Effect, Period of Adoption, Adjustment
Beginning balance (in shares) at Dec. 31, 2021					62,491,498
Beginning balance at Dec. 31, 2021	$ 302,112			$ (44,720)	$ 6			$ 1,068,634			$ (74,945)	$ (562)	$ (765,966)	$ 30,225
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	20,287							20,287
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)					567,777
Issuance of common stock under the equity incentive plan and proceeds from exercise	947							947
Employee stock purchase program purchase and expense (in shares)					77,175
Employee stock purchase program purchase and expense	2,065							2,065
Issuance of common stock/equity offering, net of issuance costs (in shares)							701,600
Issuance of common stock/equity offering, net of issuance costs			$ 19,545							$ 19,545
Foreign currency translation adjustments	1,487											1,487
Unrealized loss on marketable debt securities	(2,007)											(2,007)
Net loss	(143,003)												(143,003)
Ending balance (in shares) at Jun. 30, 2022					63,838,050
Ending balance at Jun. 30, 2022	156,713				$ 6			1,036,533				(1,082)	(878,744)
Beginning balance (in shares) at Mar. 31, 2022					63,510,277
Beginning balance at Mar. 31, 2022	208,141				$ 6			1,021,542				(1,695)	(811,712)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	12,352							12,352
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)					250,598
Issuance of common stock under the equity incentive plan and proceeds from exercise	824							824
Employee stock purchase program purchase and expense (in shares)					77,175
Employee stock purchase program purchase and expense	1,815							1,815
Foreign currency translation adjustments	1,416											1,416
Unrealized loss on marketable debt securities	(803)											(803)
Net loss	(67,032)												(67,032)
Ending balance (in shares) at Jun. 30, 2022					63,838,050
Ending balance at Jun. 30, 2022	156,713				$ 6			1,036,533				(1,082)	(878,744)
Beginning balance (in shares) at Dec. 31, 2022					64,290,570
Beginning balance at Dec. 31, 2022	42,851				$ 6			1,059,975				(2,907)	(1,014,223)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	24,497							24,497
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)					820,947
Issuance of common stock under the equity incentive plan and proceeds from exercise	3,089							3,089
Employee stock purchase program purchase and expense (in shares)					156,540
Employee stock purchase program purchase and expense	3,128							3,128
Issuance of common stock/equity offering, net of issuance costs (in shares)						9,703,750
Issuance of common stock/equity offering, net of issuance costs		$ 191,199				$ 1			$ 191,198
Issuance of pre-funded common stock warrants, net of issuance costs of $1.6 million	24,630							24,630
Foreign currency translation adjustments	(736)											(736)
Unrealized loss on marketable debt securities	(212)											(212)
Net loss	(171,961)												(171,961)
Ending balance (in shares) at Jun. 30, 2023					74,971,807
Ending balance at Jun. 30, 2023	116,485				$ 7			1,306,517				(3,855)	(1,186,184)
Beginning balance (in shares) at Mar. 31, 2023					74,586,806
Beginning balance at Mar. 31, 2023	189,140				$ 7			1,291,863				(2,176)	(1,100,554)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	11,172							11,172
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)					228,461
Issuance of common stock under the equity incentive plan and proceeds from exercise	793							793
Employee stock purchase program purchase and expense (in shares)					156,540
Employee stock purchase program purchase and expense	2,689							2,689
Foreign currency translation adjustments	(170)											(170)
Unrealized loss on marketable debt securities	(1,509)											(1,509)
Net loss	(85,630)												(85,630)
Ending balance (in shares) at Jun. 30, 2023					74,971,807
Ending balance at Jun. 30, 2023	$ 116,485				$ 7			$ 1,306,517				$ (3,855)	$ (1,186,184)

XML

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Parenthetical) - USD ($) $ in Millions	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Pre-funded common stock warrants, net of issuance costs	$ 1.6
Underwritten Equity Offering
Issuance costs	$ 12.6
At-The-Market Offering
Issuance costs		$ 0.6

XML

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Cash Flows From Operating Activities:
Net loss	$ (171,961)	$ (143,003)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	23,449	15,200
Share based compensation	25,368	20,823
Change in estimated fair value of contingent consideration	8,596	13,987
Payments from change in fair value of contingent consideration	(4,890)	(4,247)
Amortization of (discounts) premiums on investments	(3,329)	895
Loss on extinguishment of debt	0	7,578
Other	1,367	3,980
Changes in operating assets and liabilities:
Accounts receivable	(3,735)	(777)
Inventory	(13,022)	(2,060)
Prepaid expenses and other current and non-current assets	933	(2,336)
Change in lease assets and liabilities, net	(492)	(492)
Accounts payable	2,512	(2,917)
Accrued expenses	(7,904)	9,070
Deferred revenue, current and non-current	(6,823)	(5,700)
Other current and non-current liabilities	(483)	(2,127)
Net cash used in operating activities	(150,414)	(92,126)
Cash Flows From Investing Activities:
Proceeds from the sale/maturity of marketable debt securities	196,871	217,325
Purchase of marketable debt securities	(225,660)	(206,529)
Purchase of intangible assets	(31,170)	(16,579)
Other	(633)	(148)
Net cash used in by investing activities	(60,592)	(5,931)
Cash Flows From Financing Activities:
Payment of guaranteed minimum royalty	(1,050)	(1,050)
Payment of business combination-related contingent consideration	(863)	(1,271)
Proceeds from issuances of 2029 convertible senior notes	0	316,250
Payment of debt issuance costs	0	(9,882)
Repurchase of 2025 convertible senior notes including premium	0	(211,324)
Proceeds from the issuance of pre-funded warrants, net of issuance costs	24,630	0
Proceeds from exercise of stock options	3,089	947
Proceeds from issuances under the employee stock purchase plan	2,258	1,529
Net cash provided by financing activities	219,262	114,744
Effect of exchange rate changes on cash	930	(2,681)
Net increase in cash and cash equivalents	9,186	14,006
Cash and cash equivalents, beginning of year	61,688	165,753
Cash and cash equivalents, end of period	70,874	179,759
Underwritten Equity Offering
Cash Flows From Financing Activities:
Proceeds from the issuance of common stock, net of issuance costs	191,198	0
At-The-Market Offering
Cash Flows From Financing Activities:
Proceeds from the issuance of common stock, net of issuance costs	$ 0	$ 19,545

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DESCRIPTION OF BUSINESS

6 Months Ended

Jun. 30, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

DESCRIPTION OF BUSINESS

DESCRIPTION OF BUSINESS

Organization and Description of Business

Travere Therapeutics, Inc. (“we”, “our”, “us”, “Travere” and the “Company”) refers to Travere Therapeutics, Inc., a Delaware corporation, as well as its subsidiaries. Travere is a fully integrated biopharmaceutical company headquartered in San Diego, California focused on identifying, developing and delivering life-changing therapies to people living with rare kidney, liver, and metabolic diseases. The Company regularly evaluates and, where appropriate, acts on opportunities to expand its product pipeline through licenses and acquisitions of products in areas that will serve patients with serious unmet medical need and that the Company believes offer attractive growth characteristics.

FILSPARI™ (sparsentan)

On February 17, 2023, the U.S. Food and Drug Administration (the "FDA") granted accelerated approval of FILSPARI™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally at UPCR ≥1.5 gram/gram. FILSPARI, a once-daily, oral medication is designed to selectively target two critical pathways (endothelin 1 and angiotensin-II) in the disease progression of IgAN.

Clinical-Stage Programs:

The continued approval of FILSPARI for IgAN may be contingent upon confirmation of a clinical benefit in the Company's ongoing Phase 3 clinical trial of sparsentan for the treatment of IgAN (the "PROTECT Study"), which is designed to demonstrate whether FILSPARI slows kidney function decline. Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in the fourth quarter of 2023 and are intended to support traditional approval of FILSPARI.

Sparsentan remains a novel investigational product candidate which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis (FSGS) in the U.S. and Europe. The double-blind portion of the Phase 3 study of sparsentan for FSGS has recently concluded and, following release of the top-line data from the study which showed that the study did not meet its primary endpoint, the Company is conducting further analyses of the data and is preparing to engage with regulators to explore a potential path forward toward a potential regulatory submission in FSGS.

Pegtibatinase (TVT-058) is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. Pegtibatinase is currently being evaluated in the Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. In May 2023, the Company announced positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study. The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited.

Chenodal (chenodeoxycholic acid or CDCA) is a naturally occurring bile acid that is approved for the treatment of people with radiolucent stones in the gallbladder. In September 2022, the Company was granted Fast Track Designation by the FDA for the investigation of Chenodal for cerebrotendinous xanthomatosis (CTX). In January 2020, the Company randomized the first patients in its Phase 3 RESTORE Study to evaluate the effects of Chenodal in adult and pediatric patients with CTX, and the study enrollment remains open. The pivotal study is intended to support an NDA submission for marketing authorization of Chenodal for CTX in the United States. In July 2023, the Company entered into an Asset Purchase Agreement to sell substantially all of the Company’s assets that are primarily related to the Company’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam. See Note 18.

Preclinical Programs:

The Company is a participant in a Cooperative Research and Development Agreement ("CRADA"), which forms a multi-stakeholder approach to pool resources with leading experts, and incorporate the patient perspective early in the therapeutic identification and development process. The Company is partnering with the National Institutes of Health’s National Center for Advancing Translational Sciences ("NCATS") and a leading patient advocacy organization, Alagille Syndrome Alliance, aimed at the identification of potential small molecule therapeutics for Alagille syndrome ("ALGS"). There are no treatment options currently approved for ALGS.

The Company is party to a collaboration agreement with PharmaKrysto Limited and their early-stage cystinuria discovery program, whereby the Company is responsible for funding all research and development expenses for the pre-clinical activities associated with the cystinuria program.

Other Commercial Products:

•Thiola® and Thiola EC® (tiopronin tablets) are approved in the United States for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria.

•Cholbam® (cholic acid capsules) is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders.

•Chenodal (chenodiol tablets) is approved in the United States for the treatment of patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age.

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BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES

6 Months Ended

Jun. 30, 2023

Accounting Policies [Abstract]

BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES

BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES

The accompanying unaudited consolidated financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 23, 2023. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial information, the instructions for Form 10-Q and the rules and regulations of the SEC. Accordingly, since they are interim statements, the accompanying consolidated financial statements do not include all of the information and notes required by GAAP for annual financial statements, but reflect all adjustments consisting of normal, recurring adjustments, that are necessary for a fair statement of the financial position, results of operations and cash flows for the interim periods presented. Interim results are not necessarily indicative of the results that may be expected for any future periods. The December 31, 2022 balance sheet information was derived from the audited financial statements as of that date. Certain reclassifications have been made to the prior period consolidated financial statements to conform to the current period presentation.

A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows:

Principles of Consolidation

The unaudited consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation. See Note 6 for further discussion of variable interest entities (“VIE”) that the Company consolidates.

Revenue Recognition

The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Accounting Standards Codification ("ASC") 606, Revenue from Contracts with Customers ("ASC 606"), the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only recognizes revenue from contracts when it is probable that the entity will collect substantially all the consideration it is entitled to in exchange for the goods or services it transfers to the customer. See Note 3 and Note 4 for further discussion.

Payments received under collaboration and licensing agreements may include non-refundable fees at the inception of the arrangements, milestone payments for specific achievements and royalties on the sale of products. At the inception of arrangements that include milestone payments, the Company uses judgement to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, and is recorded in license and collaboration revenue.

The Company utilizes significant judgement to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgement in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin.

If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly.

The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary.

Cost of goods sold

Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping and handling costs, and provisions for excess and obsolete inventory. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partner, at cost plus a margin.

The following table summarizes cost of goods sold for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Cost of goods sold - product sales	$	1,983	$	2,051	$	4,071	$	4,189
Cost of goods sold - license and collaboration	7		—		3,044		—
Total cost of goods sold	$	1,990	$	2,051	$	7,115	$	4,189

Capitalization of Inventory Costs

Prior to the regulatory approval of the Company's drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. The Company capitalizes inventory costs associated with its products after regulatory approval, when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Until the date at which regulatory approval has been received, costs related to the production of inventory are recorded as research and development expenses as incurred. Any eventual sale of previously expensed ("zero-cost") inventories may impact future margins, for any periods in which those inventories are sold.

Sales of FILSPARI for the three and six months ended June 30, 2023 primarily consisted of zero-cost inventories, which favorably impacted gross margin for related sales. Prior to the February 2023 FDA approval of FILSPARI (sparsentan), the Company recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredients to support the commercial launch of FILSPARI. Had these costs been included, total cost of goods sold would have increased by less than $0.1 million and by approximately $0.1 million for the three and six months ended June 30, 2023, respectively. The Company expects to continue to benefit from the sale of previously expensed inventories through at least 2024.

Research and Development Expenses

Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, develop drug materials and delivery devices, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.

Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered.

Clinical Trial Expenses

The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process.

Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain.

The Company currently has one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter.

Intangible Assets with Cost Accumulation Model

In 2014, the Company entered into a license agreement with Mission Pharmacal in which the Company obtained the exclusive right to license the trademark of Thiola. The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the asset under the cost accumulation model ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining useful life.

In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows, resulting in incremental expense of $3.7 million for the three and six months ended June 30, 2023, recorded in selling, general, and administrative. The change in estimated useful life has been accounted for as a change in accounting estimate and the remaining carrying amounts of the Thiola Intangible will be amortized prospectively over the new useful life.

Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at June 30, 2023 as such royalties are not yet probable and estimable.

Variable Interest Entity

The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgements regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made.

Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.

Recently Adopted Accounting Pronouncements

In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU will require entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The ASU is effective for fiscal years beginning after December 15, 2021, including interim periods within those fiscal years. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 10, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million.

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption.

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REVENUE RECOGNITION

6 Months Ended

Jun. 30, 2023

Revenue from Contract with Customer [Abstract]

REVENUE RECOGNITION

REVENUE RECOGNITION

Product Sales, Net

Product sales consist of FILSPARI, bile acid products (Chenodal and Cholbam) and tiopronin products (Thiola and Thiola EC). The Company sells its products to specialty pharmacies and through direct-to-patient distributors worldwide, with the United States and Canada representing approximately 98% and 1% of net product sales, respectively, and rest of world representing less than 1% of net product sales, based on the product shipment destination.

The Company sells FILSPARI to three direct-to-patient specialty pharmacies. The Company sells its other products to patients and pharmacies, with distribution facilitated through a single direct-to-patient distributor. Revenues from product sales are recognized in satisfaction of a single performance obligation when the customer obtains control of the Company’s product. For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's other products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its other products, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient. Contracts do not contain significant financing components based on the typical period of time between performance of services and collection of consideration.

Deductions from Revenue

Revenues from product sales are recorded at the net sales price, which includes provisions resulting from discounts, rebates and co-pay assistance that are offered to customers, payers and other indirect customers relating to the Company’s sales of its products. These provisions are based on the estimates of the amounts earned or to be claimed on the related sales. These amounts are treated as variable consideration, estimated and recognized as a reduction of the transaction price at the time of the sale, using the most likely amount method, and are classified as a reduction of accounts receivable (if the amount is payable to a customer) or as a current liability (if the amount is payable to a party other than a customer). The Company includes these estimated amounts in the transaction price to the extent it is probable that a significant reversal of cumulative revenue recognized for such transactions will not occur. Where appropriate, these reserves take into consideration the Company’s historical experience, current contractual and statutory requirements and specific known market events and trends. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contract. If actual results in the future vary from the Company’s provisions, the Company will adjust the estimate, which would affect net product revenue and earnings in the period such variances become known. For the six months ended June 30, 2023 and 2022, adjustments to net product revenue related to performance obligations satisfied in previous periods, were immaterial.

Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Commercial Rebates: The Company calculates the rebates it incurs according to any contracts with certain commercial payers and deducts these amounts from its gross product sales at the time the revenues are recognized. Allowances for commercial rebates are established based on actual payer information, which is reasonably estimated at the time of delivery for applicable products. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale.

Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. Historically, returns have been immaterial.

Co-pay Assistance: The Company offers a co-pay assistance program, which is intended to provide financial assistance to qualified commercially insured patients with prescription drug co-payments required by payers. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue.

The following table summarizes net product sales for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Bile acid products	$	27,501	$	25,534	$	53,606	$	50,609
Tiopronin products	26,050		25,416		47,224		46,784
FILSPARI	3,461		—		6,465		—
Total net product sales	$	57,012	$	50,950	$	107,295	$	97,393

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COLLABORATION AND LICENSE AGREEMENTS

6 Months Ended

Jun. 30, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

COLLABORATION AND LICENSE AGREEMENTS

COLLABORATION AND LICENSE AGREEMENTS

On September 15, 2021, the Company entered into a license and collaboration agreement (“License Agreement”) with Vifor (International) Ltd. (“CSL Vifor”), pursuant to which the Company granted an exclusive license to CSL Vifor for the commercialization of sparsentan in Europe, Australia and New Zealand ("Licensed Territories"). CSL Vifor also has first right of negotiation to expand the licensed territories into Canada, China, Brazil and/ or Mexico. Under the terms of the License Agreement, the Company received an upfront payment of $55.0 million and will be eligible for up to $135.0 million in aggregate regulatory and market access related milestone payments and up to $655.0 million in aggregate sales-based milestone payments for a total potential value of up to $845.0 million. The Company is also entitled to receive tiered double-digit royalties of up to 40 percent of annual net sales of sparsentan in the Licensed Territories.

Under the License Agreement, CSL Vifor will be responsible for all commercialization activities in the Licensed Territories. The Company remains responsible for the worldwide clinical development of sparsentan through regulatory approval as defined and will retain all rights to sparsentan in the United States and rest of world outside of the Licensed Territories. Development costs for any post regulatory approval development activities, subject to approval by both parties, will be borne by the Company and CSL Vifor as defined, respectively. The License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the licensed territories. Each party has the right to terminate the License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the License Agreement by the other party is extended due to a force majeure event that continues for six months or more.

The Company assessed the License Agreement and determined that it meets both criteria to be considered a collaborative agreement within the Scope of ASC 808, Collaborative Arrangements of active participation by both parties and exposures to significant risks and rewards dependent on the commercial success of the activities. Both parties participate on joint steering and other committees overseeing the collaboration activities. Also, both parties are exposed to significant risks and rewards based on the economic outcomes of regulatory approvals and commercialization of sparsentan.

The Company determined the transaction price under the License Agreement totaled $55.0 million, consisting of the fixed non-refundable upfront payment. The variable regulatory and access related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the transaction price and will be recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated have been satisfied.

The Company concluded that CSL Vifor represented a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct from the promise to provide clinical development services resulting in two performance obligations. As a result, the Company allocated $12.0 million of the transaction price, based on the performance obligations' relative standalone selling prices, to the license, which was recognized in full in 2021. The remaining $43.0 million of the transaction price was allocated to the clinical development activities and recorded as deferred revenue, which will be recognized over the development period based upon the ratio of costs incurred to date to the total estimated costs.

For the three months ended June 30, 2023, the Company recognized $2.7 million in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the six months ended June 30, 2023, the Company recognized $9.4 million in license and collaboration revenue, which consisted of $3.3 million from the sale of active pharmaceutical ingredients to CSL Vifor at cost plus a margin, and $6.1 million for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the three and six months ended June 30, 2022, the Company recognized $3.2 million and $5.3 million, respectively, in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs.

Deferred revenue related to the clinical development activities as of June 30, 2023 was $17.0 million. Of this amount, $10.2 million was classified as current as of June 30, 2023, based upon amounts expected to be realized within the next year.

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MARKETABLE DEBT SECURITIES

6 Months Ended

Jun. 30, 2023

Investments, Debt and Equity Securities [Abstract]

MARKETABLE DEBT SECURITIES

MARKETABLE DEBT SECURITIES

The Company's marketable debt securities as of June 30, 2023 and December 31, 2022 were composed of available-for-sale commercial paper and corporate and government debt securities. The primary objective of the Company’s investment portfolio is to preserve capital and liquidity while enhancing overall returns. The Company’s investment policy limits interest-bearing security investments to certain types of instruments issued by institutions with primarily investment grade credit ratings and places restrictions on maturities and concentration by asset class and issuer.

Marketable debt securities consisted of the following (in thousands):

	June 30, 2023		December 31, 2022
Marketable debt securities:
Commercial paper	$	97,201	$	123,647
Corporate debt securities	235,631		224,055
Securities of government sponsored entities	87,631		40,855
Total available-for-sale marketable debt securities	$	420,463	$	388,557

The following is a summary of short-term marketable debt securities classified as available-for-sale as of June 30, 2023 (in thousands):

	Remaining Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	97,362	$	—	$	(161)	$	97,201
Corporate debt securities	Less than 1	97,682		8		(847)		96,843
Securities of government-sponsored entities	Less than 1	37,294		—		(229)		37,065
Total maturity less than 1 year		232,338		8		(1,237)		231,109
Corporate debt securities	1 to 2	140,014		33		(1,259)		138,788
Securities of government-sponsored entities	1 to 2	51,277		—		(711)		50,566
Total maturity 1 to 2 years		191,291		33		(1,970)		189,354
Total available-for-sale marketable debt securities		$	423,629	$	41	$	(3,207)	$	420,463

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 (in thousands):

	Remaining Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	124,301	$	2	$	(656)	$	123,647
Corporate debt securities	Less than 1	155,841		—		(1,355)		154,486
Securities of government-sponsored entities	Less than 1	7,473		—		(80)		7,393
Total maturity less than 1 year		287,615		2		(2,091)		285,526
Corporate debt securities	1 to 2	70,195		33		(659)		69,569
Securities of government-sponsored entities	1 to 2	33,702		6		(246)		33,462
Total maturity 1 to 2 years		103,897		39		(905)		103,031
Total available-for-sale securities		$	391,512	$	41	$	(2,996)	$	388,557

During the six months ended June 30, 2023, investment activity for the Company included $196.9 million in maturities and $225.7 million in purchases, all relating to debt-based marketable securities. During the six months ended June 30, 2022, investment activity for the Company included $217.3 million in maturities and $206.5 million in purchases, all relating to debt-based marketable securities. As of June 30, 2023 and December 31, 2022, the accrued interest receivable related to the Company's marketable debt securities was $2.5 million and $1.9 million, respectively, and was recorded in prepaid expenses and other current assets on the Consolidated Balance Sheets.

The Company reviews the available-for-sale marketable debt securities for declines in fair value below the cost basis each quarter. For any security whose fair value is below its amortized cost basis, the Company first evaluates whether it intends to sell the impaired security, or will otherwise be more likely than not required to sell the security before recovery. If either are true, the amortized cost basis of the security is written down to its fair value at the reporting date. If neither circumstance holds true, the Company assesses whether any portion of the unrealized loss is a result of a credit loss. Any amount deemed to be attributable to credit loss is recognized in the income statement, with the amount of the loss limited to the difference between fair value and amortized cost and recorded as an allowance for credit losses. The portion of the unrealized loss related to factors other than credit losses is recognized in other comprehensive income (loss).

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of June 30, 2023 (in thousands):

	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	97,201	$	161	$	—	$	—	$	97,201	$	161
Corporate debt securities	168,000		1,680		43,621		426		211,621		2,106
Securities of government-sponsored entities	86,632		936		999		4		87,631		940
Total	$	351,833	$	2,777	$	44,620	$	430	$	396,453	$	3,207

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 (in thousands):

	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	117,853	$	656	$	—	$	—	$	117,853	$	656
Corporate debt securities	99,066		1,041		107,964		973		207,030		2,014
Securities of government-sponsored entities	31,402		263		4,456		63		35,858		326
Total	$	248,321	$	1,960	$	112,420	$	1,036	$	360,741	$	2,996

As of June 30, 2023 and December 31, 2022, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $399.7 million and $363.7 million, respectively.

As of June 30, 2023 and December 31, 2022, the Company does not intend to sell these investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis. The increase in unrealized losses for the six months ended June 30, 2023 was primarily due to fluctuations in short-term interest rates. The Company does not believe the unrealized losses incurred during the period are due to credit-related factors. The credit ratings of the securities held remain of the highest quality. Moreover, the Company continues to receive payments of interest and principal as they become due, and our expectation is that those payments will continue to be received timely. Factors unknown to us at this time may cause actual results to differ and require adjustments to the Company’s estimates and assumptions in the future.

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VARIABLE INTEREST ENTITIES

6 Months Ended

Jun. 30, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

VARIABLE INTEREST ENTITIES

VARIABLE INTEREST ENTITIES

On March 8, 2022, the Company entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company related to PharmaKrysto's early-stage cystinuria discovery program, and concurrently therewith entered into a Stock Purchase Agreement with PharmaKrysto (together, the "Agreements"). Pursuant to the terms of the Agreements, the Company paid PharmaKrysto's shareholders $0.6 million in cash to purchase 5% of the outstanding common shares of PharmaKrysto and $0.4 million to PharmaKrysto as a one-time signing fee. Under the Collaboration Agreement, the Company will fund all research and development expenses for the pre-clinical activities associated with the cystinuria program, which are estimated to be approximately $5.0 million. The Agreements require the Company to purchase an additional 5% of the outstanding common shares for $1.0 million upon the occurrence of a specified pre-clinical milestone, and grant an option to the Company to purchase all of the remaining outstanding shares of PharmaKrysto for $5.0 million upon the occurrence of a subsequent pre-clinical milestone prior to expiration of the option on March 8, 2025. If the Company elects to exercise the option, it would be required to perform commercially reasonable clinical diligence obligations. In addition, it would be required to make cash milestone payments totaling up to an aggregate $16.0 million upon the achievement of certain development and regulatory milestones, plus tiered royalty payments of less than 4% on future net sales of a product, if approved. The Company has the right to terminate the Agreements and return the shares for a nominal price at any time upon 60 days’ notice, subject to survival of contingent obligations, if any.

The Company determined that PharmaKrysto is a VIE because it lacks the resources to conduct the cystinuria clinical program and the limitation on the residual returns through the Company's option to purchase the remaining outstanding shares. The Company further concluded that it is the primary beneficiary of the VIE due to the Company's ultimate control over the research and development program, and its obligation, subject to continuation of the collaboration, to fund 100% of research and development costs of the program pursuant to the terms of the Collaboration Agreement.

The upfront payments were expensed to research and development and other income (expense), net upon initial consolidation. The consolidated assets and liabilities as of June 30, 2023 and December 31, 2022 were immaterial. The results of operations were not significant for the three and six months ended June 30, 2023 and 2022. The Company is not required to provide additional funding other than the contractually required amounts disclosed above. The creditors and beneficial holders of PharmaKrysto have no recourse to the general credit or assets of the Company.

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LEASES

6 Months Ended

Jun. 30, 2023

Leases [Abstract]

LEASES

LEASES

As of June 30, 2023, the Company had two operating leases, including one operating lease with Kilroy Realty, L.P. (the "Landlord") for office space located in San Diego, California, which was entered into in April 2019 and subsequently amended in May 2020. Coinciding with the Company's ability to direct the use of the office space, which occurred in phases over 2020, and utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established ROU assets totaling $34.6 million and lease liabilities totaling $34.5 million. The total ROU asset and lease liability at measurement were each offset by lease incentives associated with tenant improvement allowances totaling $7.9 million.

The initial term of the office lease ends in August 2028, and the Landlord has granted the Company an option to extend the term of the lease by a period of 5 years. At lease inception, it was not reasonably certain that the Company will extend the term of the lease and therefore the renewal period has been excluded from the aforementioned ROU asset and lease liability measurements. The measurement of the lease term occurs from the February 2021 occupancy date of the office space delivered in September 2020.

The Company has one operating lease with Esprit Investments Limited for office space located in Dublin, Ireland, which was entered into in October 2022. The initial term of the office lease ends in September 2027. The lease provides the option to extend the term of the lease by a period of 5 years, although at lease inception, it was not reasonably certain that the Company would elect this option and therefore the renewal period was excluded from the initial lease

measurement. The aggregate base rent due over the initial term of the lease is approximately $0.5 million. Utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established an ROU asset and corresponding lease liability of $0.4 million.

Following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of June 30, 2023 (in thousands):

	June 30, 2023
2023 (remaining six months)	$	3,174
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	35,082
Present value discount	(5,313)
Total lease liability	29,769
Unamortized lease incentives	(5,083)
Cash payments in excess of straight-line lease expense	(5,127)
Total ROU asset	$	19,559

The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:

	June 30, 2023		December 31, 2022
Weighted-average remaining lease term in years	5.2		5.7
Weighted-average discount rate	6.48	%	6.48	%

For the three and six months ended June 30, 2023, the Company recorded $1.2 million and $2.5 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances. For the three and six months ended June 30, 2022, the Company recorded $1.3 million and $2.5 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances.

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FAIR VALUE MEASUREMENTS

6 Months Ended

Jun. 30, 2023

Fair Value Disclosures [Abstract]

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS

The Company utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy are described below:

Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and

Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.

The valuation techniques used to measure the fair value of the Company’s debt securities and all other financial instruments, all of which have counter-parties with high credit ratings, were valued based on quoted market prices or model driven valuations using significant inputs derived from or corroborated by observable market data. Based on the fair value hierarchy, the Company classified marketable debt securities within Level 2.

Financial instruments with carrying values approximating fair value include cash and cash equivalents, accounts receivable, and accounts payable, due to their short-term nature. As of June 30, 2023, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $65.4 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $284.9 million. As of December 31, 2022, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $62.9 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $283.0 million. The fair values were estimated utilizing market quotations and are considered Level 2.

The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of June 30, 2023 (in thousands):

	As of June 30, 2023
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	$	70,874	$	70,874	$	—	$	—
Marketable debt securities, available-for-sale	420,463		—		420,463		—
Total	$	491,337	$	70,874	$	420,463	$	—
Liabilities:
Business combination-related contingent consideration	$	74,100	$	—	$	—	$	74,100
Total	$	74,100	$	—	$	—	$	74,100

The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2022 (in thousands):

	As of December 31, 2022
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	$	61,688	$	61,688	$	—	$	—
Marketable debt securities, available-for-sale	388,557		—		388,557		—
Total	$	450,245	$	61,688	$	388,557	$	—
Liabilities:
Business combination-related contingent consideration	$	71,200	$	—	$	—	$	71,200
Total	$	71,200	$	—	$	—	$	71,200

The Company acquired two businesses, related to the Cholbam and Chenodal products, whose purchase price included potential future payments that are contingent on the achievement of certain milestones and percentages of future net sales derived from the products acquired. The Company recorded contingent consideration liabilities at their fair value on the acquisition date and revalues them at the end of each reporting period. In estimating the fair value of the Company’s contingent consideration, the Company uses a Monte Carlo Simulation. The determination of the contingent consideration liabilities requires significant judgements including the appropriateness of the valuation model and reasonableness of estimates and assumptions included in the forecasts of future net sales and the discount rates applied to such forecasts. Changes in these estimates and assumptions could have a significant impact on the fair value of the contingent consideration liabilities.

Discount rates used to determine the fair value at June 30, 2023 and December 31, 2022 are as follows:

	Revenue Discount		Payment Discount
	Cholbam	Chenodal
June 30, 2023	7.50%	7.50%	6.90%
December 31, 2022	7.75%	8.00%	8.10%

Based on the fair value hierarchy, the Company classified the fair value measurement of contingent consideration within Level 3 because valuation inputs are based on projected revenues discounted to a present value.

The following table sets forth a summary of changes in the estimated fair value of the Company's Level 3 business combination-related contingent consideration for the six months ended June 30, 2023 and 2022 (in thousands):

	Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3)
	2023		2022
Balance at January 1	$	71,200	$	67,100
Changes in the fair value of contingent consideration	8,596		13,987
Contractual payments disbursed	(2,756)		(2,685)
Contractual payments included in accrued liabilities at June 30	(2,940)		(2,702)
Balance at June 30	$	74,100	$	75,700

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INTANGIBLE ASSETS

6 Months Ended

Jun. 30, 2023

Goodwill and Intangible Assets Disclosure [Abstract]

INTANGIBLE ASSETS

INTANGIBLE ASSETS

Ligand License Agreement

In 2012, the Company entered into an agreement with Ligand Pharmaceuticals, Inc. ("Ligand") for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). As consideration for the license, the Company is required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million. In March 2023, the Company capitalized a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) that was triggered upon the accelerated approval of FILSPARI in February 2023. Pursuant to the Ligand License Agreement, the Company is obligated to pay to Ligand (and BMS) an escalating royalty between 15% and 17% of net sales of sparsentan, with payments due quarterly. The Company began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the three and six months ended June 30, 2023, the Company capitalized $0.5 million and $1.0 million, respectively, to intangible assets for royalties owed on net sales of FILSPARI. The cost of the $23.0 million milestone payment and royalty payments are being amortized to selling, general and administration on a straight-line basis through April 30, 2033.

The following table sets forth amortizable intangible assets as of June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Finite-lived intangible assets	$	334,685	$	302,935
Less: accumulated amortization	(181,165)		(158,833)
Net carrying value	$	153,520	$	144,102

As of June 30, 2023 and December 31, 2022, the Company had goodwill of $0.9 million.

The following table summarizes amortization expense for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Research and development	$	2,420	$	1,625	$	4,814	$	1,911
Selling, general and administrative	10,925		5,946		17,518		12,216
Total amortization expense	$	13,345	$	7,571	$	22,332	$	14,127

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CONVERTIBLE NOTES PAYABLE

6 Months Ended

Jun. 30, 2023

Debt Disclosure [Abstract]

CONVERTIBLE NOTES PAYABLE

CONVERTIBLE NOTES PAYABLE

The composition of the Company’s convertible senior notes are as follows (in thousands):

	June 30, 2023		December 31, 2022
2.25% convertible senior notes due 2029	$	316,250	$	316,250
2.50% convertible senior notes due 2025	68,904		68,904
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029	(8,050)		(8,750)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025	(701)		(859)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$	376,403	$	375,545

Convertible Senior Notes Due 2029

On March 11, 2022, the Company completed a registered underwritten public offering of $316.3 million aggregate principal amount of 2.25% Convertible Senior Notes due 2029 (“2029 Notes”), which includes $41.3 million aggregate principal amount of 2029 Notes sold pursuant to the full exercise of the underwriters’ option to purchase additional 2029 Notes. The Company issued the 2029 Notes under an indenture, dated as of September 10, 2018, as supplemented by the second supplemental indenture, dated as of March 11, 2022 (collectively, the “2029 Indenture”). The 2029 Notes will mature on March 1, 2029, unless earlier repurchased, redeemed, or converted. The 2029 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.25%, payable semi-annually in arrears on March 1 and September 1 of each year, beginning on September 1, 2022.

The Company received net proceeds from the issuance of the 2029 Notes of $306.4 million, after deducting commissions and offering expenses of $9.9 million. At June 30, 2023, accrued interest on the 2029 Notes of $2.4 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2029 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2029 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.

Holders may convert their 2029 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on June 30, 2022 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days

immediately after any 10 consecutive trading day period (such 10 consecutive trading day period, the “measurement period”) if the trading price per $1,000 principal amount of 2029 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions of the Company’s common stock; (4) if the Company calls the 2029 Notes for redemption; and (5) at any time from, and including, December 1, 2028 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate. The initial conversion rate for the 2029 Notes is 31.3740 shares of the Company’s common stock per $1,000 principal amount of 2029 Notes, which represents an initial conversion price of approximately $31.87 per share. If a “make-whole fundamental change” (as defined in the 2029 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.

The 2029 Notes will be redeemable, in whole or in part at the Company’s option at any time, and from time to time, on or after March 2, 2026 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2029 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on (1) each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice; and (2) the trading day immediately before the date the Company sends such notice. However, the Company may not redeem less than all of the outstanding 2029 Notes unless at least $100.0 million aggregate principal amount of 2029 Notes are outstanding and not called for redemption as of the time the Company sends the related redemption notice. In addition, calling any 2029 Note for redemption will constitute a make-whole fundamental change with respect to that 2029 Note, in which case the conversion rate applicable to the conversion of that 2029 Note will be increased in certain circumstances if it is converted after it is called for redemption. If a fundamental change (as defined in the 2029 Indenture) occurs, then, except as described in the 2029 Indentures, holders may require the Company to repurchase their 2029 Notes at a cash repurchase price equal to the principal amount of the 2029 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2029 Notes will be paid pursuant to the terms of the 2029 Indenture. In the event that all of the 2029 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2029 Notes for redemption will constitute a “make-whole fundamental change."

The Company incurred approximately $9.9 million of debt issuance costs relating to the issuance of the 2029 Notes, which were recorded as a reduction to the 2029 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2029 Notes using the effective interest method. We determined the expected life of the debt is equal to the seven-year term of the 2029 Notes. The effective interest rate on the 2029 Notes is 2.74%.

Convertible Senior Notes Due 2025

On September 10, 2018, the Company completed a registered underwritten public offering of $276.0 million aggregate principal amount of 2.50% Convertible Senior Notes due 2025 ("2025 Notes"), and entered into a base indenture and supplemental indenture agreement (collectively, the "2025 Indenture") with respect to the 2025 Notes. The 2025 Notes will mature on September 15, 2025, unless earlier repurchased, redeemed, or converted. The 2025 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.50%, payable semi-annually in arrears on March 15 and September 15 of each year, beginning on March 15, 2019.

The net proceeds from the issuance of the 2025 Notes were approximately $267.2 million, after deducting commissions and the offering expenses of $8.8 million payable by the Company. At June 30, 2023, accrued interest of $0.5 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2025 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2025 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.

Holders may convert their 2025 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on December 31, 2018 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days immediately after any 10 consecutive trading day period (“measurement period”) if the trading price per $1,000 principal amount of 2025 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions on the Company’s common stock; (4) if the Company calls the 2025 Notes for redemption; and (5) at any time from, and including, May 15, 2025 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate.

The initial conversion rate for the 2025 Notes is 25.7739 shares of the Company’s common stock per $1,000 principal amount of 2025 Notes, which represents an initial conversion price of approximately $38.80 per share. If a “make-whole fundamental change” (as defined in the 2025 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.

The 2025 Notes will be redeemable, in whole or in part, at the Company’s option at any time, and from time to time, on or after September 15, 2022 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2025 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date, but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on each of at least 20 trading days during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice. If a fundamental change (as

defined in the 2025 Indenture) occurs, then, subject to certain exceptions, holders may require the Company to repurchase their 2025 Notes at a cash repurchase price equal to the principal amount of the 2025 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2025 Notes will be paid pursuant to the terms of the 2025 Indenture. In the event that all of the 2025 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2025 Notes for redemption will constitute a “make-whole fundamental change."

The Company incurred approximately $8.8 million of debt issuance costs relating to the issuance of the 2025 Notes, which were recorded as a reduction to the 2025 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2025 Notes using the effective interest method. The Company determined the expected life of the debt is equal to the seven-year term of the 2025 Notes. The effective interest rate on the 2025 Notes is 2.98%.

On March 11, 2022, the Company completed its repurchase of $207.1 million aggregate principal amount of 2025 Notes for cash, including accrued and unpaid interest, for a total of $213.8 million. This transaction involved a contemporaneous exchange of cash between the Company and holders of the 2025 Notes participating in the issuance of the 2029 Notes. Accordingly, we evaluated the transaction for modification or extinguishment accounting in accordance with ASC 470-50, Debt – Modifications and Extinguishments on a creditor-by creditor basis depending on whether the exchange was determined to have substantially different terms. The repurchase of the 2025 Notes and issuance of the 2029 Notes were deemed to have substantially different terms based on the present value of the cash flows or significant difference between the value of the conversion option immediately prior to and after the exchange. Therefore, the repurchase of the 2025 Notes was accounted for as a debt extinguishment. The Company recorded a $7.6 million loss on extinguishment of debt on its Consolidated Statements of Operations for the six months ended June 30, 2022, which includes the write-off of related deferred financing costs of $3.4 million. After giving effect to the repurchase, the total remaining principal amount outstanding under the 2025 Notes as of June 30, 2023 was $68.9 million.

The 2025 and 2029 Notes are accounted for in accordance with ASC 470-20, Debt with conversion and Other Options (“ASC 470-20”) and ASC 815-40, Contracts in Entity’s Own Equity (“ASC 815-40”). Under ASC 815-40, to qualify for equity classification (or nonbifurcation, if embedded) the instrument (or embedded feature) must be both (1) indexed to the issuer’s stock and (2) meet the requirements of equity classification guidance. Based upon the Company’s analysis, it was determined that the 2025 Notes and the 2029 Notes do not contain embedded features requiring recognition as derivatives and bifurcation, and therefore are measured at amortized cost and recorded as liabilities on the Consolidated Balance Sheets.

The 2025 and 2029 Notes do not contain any financial or operating covenants or any restrictions on the payment of dividends, the issuance of other indebtedness or the issuance or repurchase of securities by the Company. There were no events of default for the 2025 Notes or 2029 Notes at June 30, 2023.

The 2025 and 2029 Notes are classified on the Company's Consolidated Balance Sheets at June 30, 2023 as long-tern convertible debt.

The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Contractual interest expense	$	2,210	$	2,210	$	4,419	$	4,053
Amortization of debt issuance costs	429		429		858		766
Total interest expense for the 2025 and 2029 Notes	$	2,639	$	2,639	$	5,277	$	4,819

Total interest expense recognized for the three and six months ended June 30, 2023 was $2.9 million and $5.9 million, respectively. Total interest expense recognized for the three and six months ended June 30, 2022 was $3.0 million and $5.5 million, respectively.

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ACCRUED EXPENSES

6 Months Ended

Jun. 30, 2023

Payables and Accruals [Abstract]

ACCRUED EXPENSES

ACCRUED EXPENSES

Accrued expenses at June 30, 2023 and December 31, 2022 consisted of the following (in thousands):

	June 30, 2023		December 31, 2022
Research and development	$	28,254	$	26,070
Compensation related costs	21,489		35,267
Sales discounts, rebates, and allowances	14,376		13,486
Selling, general and administrative	11,252		8,791
Accrued royalties	8,467		7,755
Miscellaneous accrued expenses	4,911		4,373
Total accrued expenses	$	88,749	$	95,742

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NET LOSS PER COMMON SHARE

6 Months Ended

Jun. 30, 2023

Earnings Per Share [Abstract]

NET LOSS PER COMMON SHARE

NET LOSS PER COMMON SHARE

Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period.

As discussed in Note 17, as part of its February 2023 underwritten public offering, the Company issued and sold pre-funded warrants to purchase 1.25 million shares of its common stock at a price to the public of $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. Since the $0.0001 price per share represents little consideration and is non-substantive in relation to the $20.9999 price per pre-funded warrant and the $21.00 price per share of the common stock offered to the public, and as the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them, the shares underlying the warrants are therefore included in the calculation of basic net loss per common share.

The Company’s potentially dilutive shares, which include outstanding stock options, restricted stock units, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents and are not included in the calculation of diluted net loss per share because their effect is anti-dilutive.

Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):

	Three Months Ended June 30,
	2023					2022
	Shares	Net Loss		Loss per common share		Shares	Net Loss		Loss per common share
Basic and diluted loss per share	76,001,801	$	(85,630)	$	(1.13)	63,638,385	$	(67,032)	$	(1.05)
	Six Months Ended June 30,
	2023					2022
	Shares	Net Loss		Loss per common share		Shares	Net Loss		Loss per common share
Basic and diluted loss per share	72,109,573	$	(171,961)	$	(2.38)	63,387,009	$	(143,003)	$	(2.26)

The following common stock equivalents have been excluded because they were anti-dilutive:

	Three Months Ended June 30,		Six Months Ended June 30,
	2023	2022	2023	2022
Convertible debt	11,697,952	11,697,952	11,697,952	10,026,309
Options	10,719,869	10,313,152	10,633,802	10,090,259
Restricted stock	3,626,814	2,160,842	3,453,767	2,031,149
Total anti-dilutive shares	26,044,635	24,171,946	25,785,521	22,147,717

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COMMITMENTS AND CONTINGENCIES

6 Months Ended

Jun. 30, 2023

Commitments and Contingencies Disclosure [Abstract]

COMMITMENTS AND CONTINGENCIES

COMMITMENTS AND CONTINGENCIES

Commitments

Certain of the Company's contractual arrangements with contract manufacturing organizations ("CMOs") require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's financial statements. As of June 30, 2023, we have commitments to purchase $22.1 million in active pharmaceutical ingredients, to be delivered in 2023, which is planned to support commercial sales of FILSPARI.

Contingencies

In October 2021, our Kolbam distributor in France notified us that the French authorities were seeking reimbursement for a portion of Kolbam sales in France during the periods from 2015-2020. During this period, the Company had aggregate revenues from sales of Kolbam in France of approximately $8.0 million. At this time, the Company is not able to estimate the potential liability that may be incurred, if any.

Legal Proceedings

From time to time in the normal course of business, the Company is subject to various legal matters such as threatened or pending claims or litigation. Although the results of claims and litigation cannot be predicted with certainty, the Company does not believe it is a party to any claim or litigation the outcome of which, if determined adversely to it, would individually or in the aggregate be reasonably expected to have a material adverse effect on its results of operations or financial condition.

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SHARE-BASED COMPENSATION

6 Months Ended

Jun. 30, 2023

Equity [Abstract]

SHARE-BASED COMPENSATION

SHARE-BASED COMPENSATION

Stock Options

The following table summarizes stock option activity during the six months ended June 30, 2023:

	Shares Underlying Options	Weighted Average Exercise Price		Weighted Average Remaining Contractual Life (years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658
Granted	1,157,750	21.69		—	—
Exercised	(211,663)	14.59		—	1,341
Forfeited/canceled	(292,208)	25.72		—	—
Outstanding at June 30, 2023	10,586,301	$	21.60	5.82	$	3,677
Vested and expected to vest at June 30, 2023	10,586,301	$	21.60	5.82	$	3,677

At June 30, 2023, unamortized stock compensation for stock options was $35.0 million, with a weighted-average recognition period of 2.6 years.

At June 30, 2023, outstanding options to purchase 7.5 million shares of common stock were exercisable with a weighted-average exercise price per share of $20.71.

In connection with the retirement of the Company's former Chief Financial Officer, the Board of Directors approved a modification to extend the deadline to exercise each stock option held to the earlier of three months following the last vesting date or the original expiration date of the option, and to continue vesting on the original schedule of any underlying unvested stock options and restricted stock units. The modification resulted in incremental compensation cost of $2.6 million for the three and six months ended June 30, 2023.

Restricted Stock Units

Service Based Restricted Stock Units

The following table summarizes the Company’s service based restricted stock unit activity during the six months ended June 30, 2023:

	Number of Restricted Stock Units	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	2,343,709	$	24.65
Granted	1,773,692	21.97
Vested	(609,284)	23.33
Forfeited/canceled	(117,837)	23.83
Unvested at June 30, 2023	3,390,280	$	23.52

At June 30, 2023, unamortized stock compensation for service based restricted stock units was $67.8 million, with a weighted-average recognition period of 3.0 years.

Performance Based Restricted Stock Units

The following table summarizes the Company’s performance based restricted stock unit activity during the six months ended June 30, 2023:

	Number of Restricted Stock Units	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	157,048	$	25.24
Granted	66,250	22.40
Vested	—	—
Forfeited/canceled	(22,840)	24.86
Unvested at June 30, 2023	200,458	$	24.34

At June 30, 2023, unamortized stock compensation for performance based restricted stock units was $2.0 million, with a weighted-average recognition period of 0.9 years.

Share-Based Compensation

The following table sets forth total share-based compensation for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Research and development	$	4,615	$	3,684	$	9,096	$	6,852
Selling, general and administrative	6,988		8,953		16,271		13,971
Total share-based compensation	$	11,603	$	12,637	$	25,367	$	20,823

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INVENTORY

6 Months Ended

Jun. 30, 2023

Inventory Disclosure [Abstract]

INVENTORY

INVENTORY

Inventory consisted of the following at June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Raw materials	$	14,652	$	3,627
Finished goods	4,113		3,295
Total inventory	$	18,765	$	6,922

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ACCOUNTS RECEIVABLE	6 Months Ended
	Jun. 30, 2023
Receivables [Abstract]
ACCOUNTS RECEIVABLE	ACCOUNTS RECEIVABLEAccounts receivable, net of reserves for prompt pay discounts and expected credit losses, was $20.4 million and $16.6 million at June 30, 2023 and December 31, 2022, respectively. The total reserves for both periods were immaterial.The Company's evaluation and accounting for credit losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses.

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EQUITY OFFERINGS

6 Months Ended

Jun. 30, 2023

Equity [Abstract]

EQUITY OFFERINGS

EQUITY OFFERINGS

Underwritten Public Offering of Common Stock

In February 2023, the Company sold an aggregate of approximately 9.7 million shares of its common stock and pre-funded warrants to purchase 1.25 million shares of its common stock in an underwritten public offering, at a price to the public of $21.00 per share of common stock and $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately, subject to certain beneficial ownership limitations which can be modified by the respective holders with at least 61 days' notice, and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. The net proceeds to the Company from the offering, after deducting the underwriting discounts and offering expenses, were approximately $215.8 million.

The pre-funded warrants were classified as a component of permanent stockholders' equity within additional paid-in capital and were recorded at the issuance date using a relative fair value allocation method. The pre-funded warrants are equity classified because they (i) are freestanding financial instruments that are legally detachable and separately exercisable from the equity instruments, (ii) are immediately exercisable, (iii) do not embody an obligation for the Company to repurchase its shares, (iv) permit the holders to receive a fixed number of shares of common stock upon exercise, (v) are indexed to the Company's common stock and (vi) meet the equity classification criteria. In addition, such pre-funded warrants do not provide any guarantee of value or return. The Company valued the pre-funded warrants at issuance, concluding that their sale price approximated their fair value, and allocated the aggregate net proceeds from the sale proportionately to the common stock and prefunded warrants, including approximately $24.6 million allocated to the pre-funded warrants and recorded as a component of additional paid-in capital.

At-the-Market Equity Offering

In February 2020, the Company entered into an Open Market Sale Agreement ("ATM Agreement") with Jefferies LLC, as agent (“Jefferies”), pursuant to which the Company may offer and sell, from time to time through Jefferies, shares of its common stock having an aggregate offering price of up to $100.0 million. Of the $100.0 million originally authorized for sale under the ATM Agreement, approximately $28.6 million were sold under the Company’s prior registration statement on Form S-3 (Registration No. 333-227182). An additional $51.9 million were sold under the Company's effective registration statement on Form S-3 (Registration Statement No. 333-259311), which included $20.1 million in the year ended December 31, 2022. The Company did not sell any shares under the ATM Agreement during the six months ended June 30, 2023. As of June 30, 2023, an aggregate of $19.5 million remained eligible for sale under the ATM Agreement.

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SUBSEQUENT EVENTS

6 Months Ended

Jun. 30, 2023

Subsequent Events [Abstract]

SUBSEQUENT EVENTS

SUBSEQUENT EVENTS

On July 16, 2023, the Company executed an Asset Purchase Agreement (the “Purchase Agreement”) with Mirum Pharmaceuticals, Inc., a Delaware corporation (“Mirum”). Subject to the terms and conditions of the Purchase Agreement, at the closing (the “Closing”) of the transactions contemplated by the Purchase Agreement (the “Asset Purchase”), Mirum will purchase from the Company substantially all of the assets that are primarily related to the Company’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Products”) for an aggregate purchase price, subject to certain adjustments pursuant to the terms of the Purchase Agreement, of up to $445.0 million in cash, with $210.0 million due at the Closing and up to $235.0 million after the Closing, upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products (the “Milestone Events”).

Mirum has agreed to use certain specified resources and efforts during stipulated time-periods to obtain regulatory approval and to cause the Milestone Events to be achieved. The Company and Mirum have also entered into a transition services agreement pursuant to which the Company has agreed to perform certain services for a period of time following the Closing, with respect to Mirum’s use and operation of the assets purchased in the Asset Purchase.

The Company and Mirum are permitted under certain circumstances to terminate the Agreement, including in the event that the Closing has not occurred by October 16, 2023, which period will be automatically extended to April 16, 2024 if any condition remaining to be satisfied relates to regulatory approval.

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Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Pay vs Performance Disclosure
Net loss	$ (85,630)	$ (67,032)	$ (171,961)	$ (143,003)

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Insider Trading Arrangements	3 Months Ended
	Jun. 30, 2023
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

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BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES (Policies)

6 Months Ended

Jun. 30, 2023

Accounting Policies [Abstract]

Principles of Consolidation

Principles of ConsolidationThe unaudited consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation.

Revenue Recognition and Deductions from Revenue

Revenue RecognitionThe Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Accounting Standards Codification ("ASC") 606, Revenue from Contracts with Customers ("ASC 606"), the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only recognizes revenue from contracts when it is probable that the entity will collect substantially all the consideration it is entitled to in exchange for the goods or services it transfers to the customer.

Deductions from Revenue

Revenues from product sales are recorded at the net sales price, which includes provisions resulting from discounts, rebates and co-pay assistance that are offered to customers, payers and other indirect customers relating to the Company’s sales of its products. These provisions are based on the estimates of the amounts earned or to be claimed on the related sales. These amounts are treated as variable consideration, estimated and recognized as a reduction of the transaction price at the time of the sale, using the most likely amount method, and are classified as a reduction of accounts receivable (if the amount is payable to a customer) or as a current liability (if the amount is payable to a party other than a customer). The Company includes these estimated amounts in the transaction price to the extent it is probable that a significant reversal of cumulative revenue recognized for such transactions will not occur. Where appropriate, these reserves take into consideration the Company’s historical experience, current contractual and statutory requirements and specific known market events and trends. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contract. If actual results in the future vary from the Company’s provisions, the Company will adjust the estimate, which would affect net product revenue and earnings in the period such variances become known. For the six months ended June 30, 2023 and 2022, adjustments to net product revenue related to performance obligations satisfied in previous periods, were immaterial.

Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Commercial Rebates: The Company calculates the rebates it incurs according to any contracts with certain commercial payers and deducts these amounts from its gross product sales at the time the revenues are recognized. Allowances for commercial rebates are established based on actual payer information, which is reasonably estimated at the time of delivery for applicable products. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale.

Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. Historically, returns have been immaterial.

Co-pay Assistance: The Company offers a co-pay assistance program, which is intended to provide financial assistance to qualified commercially insured patients with prescription drug co-payments required by payers. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue.

Collaborative Arrangements

Payments received under collaboration and licensing agreements may include non-refundable fees at the inception of the arrangements, milestone payments for specific achievements and royalties on the sale of products. At the inception of arrangements that include milestone payments, the Company uses judgement to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, and is recorded in license and collaboration revenue.

The Company utilizes significant judgement to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgement in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin.

If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly.

The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary.

Cost of Goods Sold

Cost of goods sold

Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping and handling costs, and provisions for excess and obsolete inventory. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partner, at cost plus a margin.

Capitalization of Inventory Costs

Capitalization of Inventory Costs

Prior to the regulatory approval of the Company's drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. The Company capitalizes inventory costs associated with its products after regulatory approval, when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Until the date at which regulatory approval has been received, costs related to the production of inventory are recorded as research and development expenses as incurred. Any eventual sale of previously expensed ("zero-cost") inventories may impact future margins, for any periods in which those inventories are sold.

Sales of FILSPARI for the three and six months ended June 30, 2023 primarily consisted of zero-cost inventories, which favorably impacted gross margin for related sales. Prior to the February 2023 FDA approval of FILSPARI (sparsentan), the Company recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredients to support the commercial launch of FILSPARI. Had these costs been included, total cost of goods sold would have increased by less than $0.1 million and by approximately $0.1 million for the three and six months ended June 30, 2023, respectively. The Company expects to continue to benefit from the sale of previously expensed inventories through at least 2024.

Research and Development Expense

Research and Development Expenses

Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, develop drug materials and delivery devices, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.

Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered.

Clinical Trial Expenses

Clinical Trial Expenses

The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process.

Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain.

The Company currently has one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter.

Intangible Assets with Cost Accumulation Model

Intangible Assets with Cost Accumulation Model

In 2014, the Company entered into a license agreement with Mission Pharmacal in which the Company obtained the exclusive right to license the trademark of Thiola. The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the asset under the cost accumulation model ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining useful life.

In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows, resulting in incremental expense of $3.7 million for the three and six months ended June 30, 2023, recorded in selling, general, and administrative. The change in estimated useful life has been accounted for as a change in accounting estimate and the remaining carrying amounts of the Thiola Intangible will be amortized prospectively over the new useful life.

Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at June 30, 2023 as such royalties are not yet probable and estimable.

Variable Interest Entity

Variable Interest Entity

The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgements regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made.

Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.

Recently Adopted and Issued Accounting Pronouncements

Recently Adopted Accounting Pronouncements

In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU will require entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The ASU is effective for fiscal years beginning after December 15, 2021, including interim periods within those fiscal years. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 10, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million.

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption.

Fair Value Measurements

The Company utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy are described below:

Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and

Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.

The valuation techniques used to measure the fair value of the Company’s debt securities and all other financial instruments, all of which have counter-parties with high credit ratings, were valued based on quoted market prices or model driven valuations using significant inputs derived from or corroborated by observable market data. Based on the fair value hierarchy, the Company classified marketable debt securities within Level 2.

Net Loss Per Common Share

Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period.

As discussed in Note 17, as part of its February 2023 underwritten public offering, the Company issued and sold pre-funded warrants to purchase 1.25 million shares of its common stock at a price to the public of $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. Since the $0.0001 price per share represents little consideration and is non-substantive in relation to the $20.9999 price per pre-funded warrant and the $21.00 price per share of the common stock offered to the public, and as the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them, the shares underlying the warrants are therefore included in the calculation of basic net loss per common share.

The Company’s potentially dilutive shares, which include outstanding stock options, restricted stock units, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents and are not included in the calculation of diluted net loss per share because their effect is anti-dilutive.

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BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES (Tables)

6 Months Ended

Jun. 30, 2023

Accounting Policies [Abstract]

Schedule of Cost and Goods Sold

The following table summarizes cost of goods sold for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Cost of goods sold - product sales	$	1,983	$	2,051	$	4,071	$	4,189
Cost of goods sold - license and collaboration	7		—		3,044		—
Total cost of goods sold	$	1,990	$	2,051	$	7,115	$	4,189

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REVENUE RECOGNITION (Tables)

6 Months Ended

Jun. 30, 2023

Revenue from Contract with Customer [Abstract]

Schedule of Net Product Revenue

The following table summarizes net product sales for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Bile acid products	$	27,501	$	25,534	$	53,606	$	50,609
Tiopronin products	26,050		25,416		47,224		46,784
FILSPARI	3,461		—		6,465		—
Total net product sales	$	57,012	$	50,950	$	107,295	$	97,393

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MARKETABLE DEBT SECURITIES (Tables)

6 Months Ended

Jun. 30, 2023

Investments, Debt and Equity Securities [Abstract]

Schedule of Marketable Debt Securities

Marketable debt securities consisted of the following (in thousands):

	June 30, 2023		December 31, 2022
Marketable debt securities:
Commercial paper	$	97,201	$	123,647
Corporate debt securities	235,631		224,055
Securities of government sponsored entities	87,631		40,855
Total available-for-sale marketable debt securities	$	420,463	$	388,557

Schedule of Available for Sale Securities

The following is a summary of short-term marketable debt securities classified as available-for-sale as of June 30, 2023 (in thousands):

	Remaining Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	97,362	$	—	$	(161)	$	97,201
Corporate debt securities	Less than 1	97,682		8		(847)		96,843
Securities of government-sponsored entities	Less than 1	37,294		—		(229)		37,065
Total maturity less than 1 year		232,338		8		(1,237)		231,109
Corporate debt securities	1 to 2	140,014		33		(1,259)		138,788
Securities of government-sponsored entities	1 to 2	51,277		—		(711)		50,566
Total maturity 1 to 2 years		191,291		33		(1,970)		189,354
Total available-for-sale marketable debt securities		$	423,629	$	41	$	(3,207)	$	420,463

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 (in thousands):

	Remaining Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	124,301	$	2	$	(656)	$	123,647
Corporate debt securities	Less than 1	155,841		—		(1,355)		154,486
Securities of government-sponsored entities	Less than 1	7,473		—		(80)		7,393
Total maturity less than 1 year		287,615		2		(2,091)		285,526
Corporate debt securities	1 to 2	70,195		33		(659)		69,569
Securities of government-sponsored entities	1 to 2	33,702		6		(246)		33,462
Total maturity 1 to 2 years		103,897		39		(905)		103,031
Total available-for-sale securities		$	391,512	$	41	$	(2,996)	$	388,557

Schedule of Marketable Debt Securities in an Unrealized Loss Position

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of June 30, 2023 (in thousands):

	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	97,201	$	161	$	—	$	—	$	97,201	$	161
Corporate debt securities	168,000		1,680		43,621		426		211,621		2,106
Securities of government-sponsored entities	86,632		936		999		4		87,631		940
Total	$	351,833	$	2,777	$	44,620	$	430	$	396,453	$	3,207

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 (in thousands):

	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	117,853	$	656	$	—	$	—	$	117,853	$	656
Corporate debt securities	99,066		1,041		107,964		973		207,030		2,014
Securities of government-sponsored entities	31,402		263		4,456		63		35,858		326
Total	$	248,321	$	1,960	$	112,420	$	1,036	$	360,741	$	2,996

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LEASES (Tables)

6 Months Ended

Jun. 30, 2023

Leases [Abstract]

Schedule of Future Minimum Rent Commitments

Following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of June 30, 2023 (in thousands):

	June 30, 2023
2023 (remaining six months)	$	3,174
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	35,082
Present value discount	(5,313)
Total lease liability	29,769
Unamortized lease incentives	(5,083)
Cash payments in excess of straight-line lease expense	(5,127)
Total ROU asset	$	19,559

Schedule of Weighted-average Remaining Lease Term and Discount Rate

The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:

	June 30, 2023		December 31, 2022
Weighted-average remaining lease term in years	5.2		5.7
Weighted-average discount rate	6.48	%	6.48	%

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FAIR VALUE MEASUREMENTS (Tables)

6 Months Ended

Jun. 30, 2023

Fair Value Disclosures [Abstract]

Schedule of Fair Value on a Recurring Basis

The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of June 30, 2023 (in thousands):

	As of June 30, 2023
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	$	70,874	$	70,874	$	—	$	—
Marketable debt securities, available-for-sale	420,463		—		420,463		—
Total	$	491,337	$	70,874	$	420,463	$	—
Liabilities:
Business combination-related contingent consideration	$	74,100	$	—	$	—	$	74,100
Total	$	74,100	$	—	$	—	$	74,100

The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2022 (in thousands):

	As of December 31, 2022
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	$	61,688	$	61,688	$	—	$	—
Marketable debt securities, available-for-sale	388,557		—		388,557		—
Total	$	450,245	$	61,688	$	388,557	$	—
Liabilities:
Business combination-related contingent consideration	$	71,200	$	—	$	—	$	71,200
Total	$	71,200	$	—	$	—	$	71,200

Schedule of Discount Rates Used

Discount rates used to determine the fair value at June 30, 2023 and December 31, 2022 are as follows:

	Revenue Discount		Payment Discount
	Cholbam	Chenodal
June 30, 2023	7.50%	7.50%	6.90%
December 31, 2022	7.75%	8.00%	8.10%

Schedule of Fair Value Measurements of Acquisition-related Contingent Consideration

The following table sets forth a summary of changes in the estimated fair value of the Company's Level 3 business combination-related contingent consideration for the six months ended June 30, 2023 and 2022 (in thousands):

	Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3)
	2023		2022
Balance at January 1	$	71,200	$	67,100
Changes in the fair value of contingent consideration	8,596		13,987
Contractual payments disbursed	(2,756)		(2,685)
Contractual payments included in accrued liabilities at June 30	(2,940)		(2,702)
Balance at June 30	$	74,100	$	75,700

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INTANGIBLE ASSETS (Tables)

6 Months Ended

Jun. 30, 2023

Goodwill and Intangible Assets Disclosure [Abstract]

Schedule of Finite-lived Amortizable Intangible Assets

The following table sets forth amortizable intangible assets as of June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Finite-lived intangible assets	$	334,685	$	302,935
Less: accumulated amortization	(181,165)		(158,833)
Net carrying value	$	153,520	$	144,102

Schedule of Amortization Expense

The following table summarizes amortization expense for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Research and development	$	2,420	$	1,625	$	4,814	$	1,911
Selling, general and administrative	10,925		5,946		17,518		12,216
Total amortization expense	$	13,345	$	7,571	$	22,332	$	14,127

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CONVERTIBLE NOTES PAYABLE (Tables)

6 Months Ended

Jun. 30, 2023

Debt Disclosure [Abstract]

Schedule of Long-term Debt Instruments

The composition of the Company’s convertible senior notes are as follows (in thousands):

	June 30, 2023		December 31, 2022
2.25% convertible senior notes due 2029	$	316,250	$	316,250
2.50% convertible senior notes due 2025	68,904		68,904
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029	(8,050)		(8,750)
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025	(701)		(859)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$	376,403	$	375,545

The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Contractual interest expense	$	2,210	$	2,210	$	4,419	$	4,053
Amortization of debt issuance costs	429		429		858		766
Total interest expense for the 2025 and 2029 Notes	$	2,639	$	2,639	$	5,277	$	4,819

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ACCRUED EXPENSES (Tables)

6 Months Ended

Jun. 30, 2023

Payables and Accruals [Abstract]

Schedule of Accrued Expenses

Accrued expenses at June 30, 2023 and December 31, 2022 consisted of the following (in thousands):

	June 30, 2023		December 31, 2022
Research and development	$	28,254	$	26,070
Compensation related costs	21,489		35,267
Sales discounts, rebates, and allowances	14,376		13,486
Selling, general and administrative	11,252		8,791
Accrued royalties	8,467		7,755
Miscellaneous accrued expenses	4,911		4,373
Total accrued expenses	$	88,749	$	95,742

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NET LOSS PER COMMON SHARE (Tables)

6 Months Ended

Jun. 30, 2023

Earnings Per Share [Abstract]

Schedule of Basic and Diluted Net Loss Per Share

Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):

	Three Months Ended June 30,
	2023					2022
	Shares	Net Loss		Loss per common share		Shares	Net Loss		Loss per common share
Basic and diluted loss per share	76,001,801	$	(85,630)	$	(1.13)	63,638,385	$	(67,032)	$	(1.05)
	Six Months Ended June 30,
	2023					2022
	Shares	Net Loss		Loss per common share		Shares	Net Loss		Loss per common share
Basic and diluted loss per share	72,109,573	$	(171,961)	$	(2.38)	63,387,009	$	(143,003)	$	(2.26)

Schedule of Common Stock Options, Convertible Debt and Restricted Stock Units Anti-dilutive

The following common stock equivalents have been excluded because they were anti-dilutive:

	Three Months Ended June 30,		Six Months Ended June 30,
	2023	2022	2023	2022
Convertible debt	11,697,952	11,697,952	11,697,952	10,026,309
Options	10,719,869	10,313,152	10,633,802	10,090,259
Restricted stock	3,626,814	2,160,842	3,453,767	2,031,149
Total anti-dilutive shares	26,044,635	24,171,946	25,785,521	22,147,717

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SHARE-BASED COMPENSATION (Tables)

6 Months Ended

Jun. 30, 2023

Equity [Abstract]

Schedule of Stock Option Issuances and Balances Outstanding

The following table summarizes stock option activity during the six months ended June 30, 2023:

	Shares Underlying Options	Weighted Average Exercise Price		Weighted Average Remaining Contractual Life (years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658
Granted	1,157,750	21.69		—	—
Exercised	(211,663)	14.59		—	1,341
Forfeited/canceled	(292,208)	25.72		—	—
Outstanding at June 30, 2023	10,586,301	$	21.60	5.82	$	3,677
Vested and expected to vest at June 30, 2023	10,586,301	$	21.60	5.82	$	3,677

Schedule of Service Based Restricted Stock Activity

The following table summarizes the Company’s service based restricted stock unit activity during the six months ended June 30, 2023:

	Number of Restricted Stock Units	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	2,343,709	$	24.65
Granted	1,773,692	21.97
Vested	(609,284)	23.33
Forfeited/canceled	(117,837)	23.83
Unvested at June 30, 2023	3,390,280	$	23.52

Schedule of Performance Based Restricted Stock Activity

The following table summarizes the Company’s performance based restricted stock unit activity during the six months ended June 30, 2023:

	Number of Restricted Stock Units	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	157,048	$	25.24
Granted	66,250	22.40
Vested	—	—
Forfeited/canceled	(22,840)	24.86
Unvested at June 30, 2023	200,458	$	24.34

Schedule of Share Based Compensation Expenses

The following table sets forth total share-based compensation for the three and six months ended June 30, 2023 and 2022 (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2023		2022		2023		2022
Research and development	$	4,615	$	3,684	$	9,096	$	6,852
Selling, general and administrative	6,988		8,953		16,271		13,971
Total share-based compensation	$	11,603	$	12,637	$	25,367	$	20,823

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INVENTORY (Tables)

6 Months Ended

Jun. 30, 2023

Inventory Disclosure [Abstract]

Schedule of Inventory

Inventory consisted of the following at June 30, 2023 and December 31, 2022 (in thousands):

	June 30, 2023		December 31, 2022
Raw materials	$	14,652	$	3,627
Finished goods	4,113		3,295
Total inventory	$	18,765	$	6,922

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BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Cost of Goods Sold (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Cost of goods sold	$ 1,990	$ 2,051	$ 7,115	$ 4,189
Net product sales
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Cost of goods sold	1,983	2,051	4,071	4,189
License and collaboration revenue
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Cost of goods sold	$ 7	$ 0	$ 3,044	$ 0

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BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Additional Information (Details) $ in Thousands	3 Months Ended	6 Months Ended
	Jun. 30, 2023 USD ($) clinicalTrial	Jun. 30, 2023 USD ($) clinicalTrial	Dec. 31, 2022 USD ($)	Jan. 01, 2022 USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Research and development		$ 7,500
Cost of goods sold, potential increase from included costs	$ 100	$ 100
Number of Phase1/2 clinical trials in process | clinicalTrial		1
Number of Phase 3 clinical trials in process | clinicalTrial	3	3
Intangible asset impairment	$ 3,700	$ 3,700
Reduction in additional paid-in capital	(1,306,517)	(1,306,517)	$ (1,059,975)
Reduction in accumulated deficit	$ (1,186,184)	$ (1,186,184)	$ (1,014,223)
Cumulative Effect, Period of Adoption, Adjustment | Accounting Standards Update 2020-06
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Convertible notes payable				$ 44,700
Reduction in additional paid-in capital				74,900
Reduction in accumulated deficit				$ 30,200

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REVENUE RECOGNITION - Additional Information (Details)	6 Months Ended
	Jun. 30, 2023
Disaggregation of Revenue [Line Items]
Revenue, performance obligation, description of timing	For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's other products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its other products, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient.
Geographic Concentration Risk | Revenue Benchmark | United States
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	98.00%
Geographic Concentration Risk | Revenue Benchmark | Canada
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	1.00%
Geographic Concentration Risk | Revenue Benchmark | Rest of World
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	1.00%

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REVENUE RECOGNITION - Net Product Revenue (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Disaggregation of Revenue [Line Items]
Total revenue	$ 59,697	$ 54,167	$ 116,690	$ 102,654
Net product sales
Disaggregation of Revenue [Line Items]
Total revenue	57,012	50,950	107,295	97,393
Bile acid products
Disaggregation of Revenue [Line Items]
Total revenue	27,501	25,534	53,606	50,609
Tiopronin products
Disaggregation of Revenue [Line Items]
Total revenue	26,050	25,416	47,224	46,784
FILSPARI
Disaggregation of Revenue [Line Items]
Total revenue	$ 3,461	$ 0	$ 6,465	$ 0

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COLLABORATION AND LICENSE AGREEMENTS (Details) $ in Thousands		3 Months Ended		6 Months Ended		12 Months Ended
	Sep. 15, 2021 USD ($) performance_obligation	Jun. 30, 2023 USD ($)	Jun. 30, 2022 USD ($)	Jun. 30, 2023 USD ($)	Jun. 30, 2022 USD ($)	Dec. 31, 2021 USD ($)	Dec. 31, 2022 USD ($)
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue		$ 59,697	$ 54,167	$ 116,690	$ 102,654
Deferred revenue, current portion		10,244		10,244			$ 11,976
License
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue		2,685	$ 3,217	9,395	$ 5,261
Sale of Active Pharmaceutical Ingredient
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue				3,300
Clinical Development Activity
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue				6,100
Collaborative Arrangement | Vifor Pharma
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Upfront payment	$ 55,000
Maximum milestone payments	$ 845,000
Percentage of royalty on net sales receives up to	40.00%
Force majeure event period threshold	6 months
Revenue	$ 55,000
Number of performance obligations | performance_obligation	2
Deferred revenue	$ 43,000	17,000		17,000
Deferred revenue, current portion		$ 10,200		$ 10,200
Collaborative Arrangement | Vifor Pharma | Regulatory and Market Access Milestone
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments	135,000
Collaborative Arrangement | Vifor Pharma | Sales-based Milestone Payments
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments	$ 655,000
Collaborative Arrangement | Vifor Pharma | License
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue						$ 12,000

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MARKETABLE DEBT SECURITIES - Marketable Securities (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	$ 420,463	$ 388,557
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	97,201	123,647
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	235,631	224,055
Securities of government sponsored entities
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	$ 87,631	$ 40,855

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MARKETABLE DEBT SECURITIES - Available for Sale Securities (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	$ 232,338	$ 287,615
Marketable debt securities, available for sale, unrealized gain, current	8	2
Marketable debt securities, available for sale, unrealized loss, current	(1,237)	(2,091)
Marketable debt securities, available-for-sale, current	231,109	285,526
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	191,291	103,897
Marketable debt securities, available for sale, unrealized gain, noncurrent	33	39
Marketable debt securities, available for sale, unrealized loss, noncurrent	(1,970)	(905)
Marketable debt securities, available-for-sale, noncurrent	189,354	103,031
Marketable debt securities, available-for-sale, amortized cost	423,629	391,512
Marketable debt securities, available for sale, unrealized gains	41	41
Marketable debt securities, available-for-sale, unrealized loss	(3,207)	(2,996)
Total available-for-sale marketable debt securities	420,463	388,557
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	97,362	124,301
Marketable debt securities, available for sale, unrealized gain, current	0	2
Marketable debt securities, available for sale, unrealized loss, current	(161)	(656)
Marketable debt securities, available-for-sale, current	97,201	123,647
Total available-for-sale marketable debt securities	97,201	123,647
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	97,682	155,841
Marketable debt securities, available for sale, unrealized gain, current	8	0
Marketable debt securities, available for sale, unrealized loss, current	(847)	(1,355)
Marketable debt securities, available-for-sale, current	96,843	154,486
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	140,014	70,195
Marketable debt securities, available for sale, unrealized gain, noncurrent	33	33
Marketable debt securities, available for sale, unrealized loss, noncurrent	(1,259)	(659)
Marketable debt securities, available-for-sale, noncurrent	138,788	69,569
Total available-for-sale marketable debt securities	235,631	224,055
Securities of government sponsored entities
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	37,294	7,473
Marketable debt securities, available for sale, unrealized gain, current	0	0
Marketable debt securities, available for sale, unrealized loss, current	(229)	(80)
Marketable debt securities, available-for-sale, current	37,065	7,393
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	51,277	33,702
Marketable debt securities, available for sale, unrealized gain, noncurrent	0	6
Marketable debt securities, available for sale, unrealized loss, noncurrent	(711)	(246)
Marketable debt securities, available-for-sale, noncurrent	50,566	33,462
Total available-for-sale marketable debt securities	$ 87,631	$ 40,855

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MARKETABLE DEBT SECURITIES - Additional Information (Details) - USD ($) $ in Millions	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Dec. 31, 2022
Investments, Debt and Equity Securities [Abstract]
Proceeds from sale of debt securities	$ 196.9	$ 217.3
Purchase of debt securities	225.7	$ 206.5
Accrued interest receivable	2.5		$ 1.9
Available-for-sale marketable debt securities in an unrealized loss position	$ 399.7		$ 363.7

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MARKETABLE DEBT SECURITIES - Securities in an Unrealized Loss Position (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	$ 351,833	$ 248,321
Less than 12 months, unrealized losses	2,777	1,960
12 months or greater, fair value	44,620	112,420
12 months or greater, unrealized losses	430	1,036
Total, fair value	396,453	360,741
Total, unrealized losses	3,207	2,996
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	97,201	117,853
Less than 12 months, unrealized losses	161	656
12 months or greater, fair value	0	0
12 months or greater, unrealized losses	0	0
Total, fair value	97,201	117,853
Total, unrealized losses	161	656
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	168,000	99,066
Less than 12 months, unrealized losses	1,680	1,041
12 months or greater, fair value	43,621	107,964
12 months or greater, unrealized losses	426	973
Total, fair value	211,621	207,030
Total, unrealized losses	2,106	2,014
Securities of government-sponsored entities
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	86,632	31,402
Less than 12 months, unrealized losses	936	263
12 months or greater, fair value	999	4,456
12 months or greater, unrealized losses	4	63
Total, fair value	87,631	35,858
Total, unrealized losses	$ 940	$ 326

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VARIABLE INTEREST ENTITIES (Details) $ in Millions	Mar. 08, 2022 USD ($)
Collaborative Arrangement | PharmaKrysto, LTD
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration agreement payment	$ 0.4
Research and development expenses, estimated	5.0
Collaborative arrangement, option to purchase additional shares of VIE, amount	1.0
Collaborative arrangement, option to purchase remaining shares of VIE, amount	5.0
Milestone payments contingently due	$ 16.0
Royalty payments, percentage (less than)	4.00%
Agreement termination notice period	60 days
Percentage of research and development to be funded by the company	100.00%
PharmaKrysto, LTD
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Payment to purchase interest in VIE	$ 0.6
Percentage ownership purchased	5.00%
PharmaKrysto, LTD | Collaborative Arrangement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Additional ownership interest to be purchased upon achievement of certain milestones	5.00%

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LEASES - Additional Information (Details) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023 USD ($) lease	Jun. 30, 2022 USD ($)	Jun. 30, 2023 USD ($) lease	Jun. 30, 2022 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2020 USD ($)
Lessee, Lease, Description [Line Items]
Operating lease right of use assets	$ 19,559		$ 19,559		$ 21,000
Lease liability	$ 29,769		$ 29,769
Operating lease extension term	5 years		5 years
Operating lease expense	$ 1,200	$ 1,300	$ 2,500	$ 2,500
Office Lease 2020
Lessee, Lease, Description [Line Items]
Operating lease right of use assets						$ 34,600
Lease liability						34,500
Lease incentive, tenant improvements						$ 7,900
Kilroy Realty, L.P.
Lessee, Lease, Description [Line Items]
Number of operating leases | lease	2		2
Esprit Investments Limited
Lessee, Lease, Description [Line Items]
Number of operating leases | lease	1		1
Operating lease right of use assets	$ 400		$ 400
Lease liability	$ 400		$ 400
Operating lease extension term	5 years		5 years
Base rent due over the initial term of the lease			$ 500

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LEASES - Future Minimum Rent Commitments (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Leases [Abstract]
2023 (remaining six months)	$ 3,174
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	35,082
Present value discount	(5,313)
Total lease liability	29,769
Unamortized lease incentives	(5,083)
Cash payments in excess of straight-line lease expense	(5,127)
Total ROU asset	$ 19,559	$ 21,000

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LEASES - Weighted-Average Remaining Lease Term and Discount Rate (Details)	Jun. 30, 2023	Dec. 31, 2022
Leases [Abstract]
Weighted-average remaining lease term in years	5 years 2 months 12 days	5 years 8 months 12 days
Weighted-average discount rate	6.48%	6.48%

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FAIR VALUE MEASUREMENTS - Additional Information (Details) $ in Millions	6 Months Ended
	Jun. 30, 2023 USD ($) business	Dec. 31, 2022 USD ($)
Business Acquisition [Line Items]
Number of businesses acquired | business	2
Senior Notes Due 2025 | Senior Notes
Business Acquisition [Line Items]
Interest rate percentage	2.50%	2.50%
Fair value of convertible debt	$ 65.4	$ 62.9
Senior Notes Due 2029 | Senior Notes
Business Acquisition [Line Items]
Interest rate percentage	2.25%	2.25%
Fair value of convertible debt	$ 284.9	$ 283.0

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FAIR VALUE MEASUREMENTS - Assets and Liabilities (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Assets:
Cash and cash equivalents	$ 70,874	$ 61,688
Marketable debt securities, available-for-sale	420,463	388,557
Total	491,337	450,245
Liabilities:
Business combination-related contingent consideration	74,100	71,200
Total	74,100	71,200
Quoted prices in active markets (Level 1)
Assets:
Cash and cash equivalents	70,874	61,688
Marketable debt securities, available-for-sale	0	0
Total	70,874	61,688
Liabilities:
Business combination-related contingent consideration	0	0
Total	0	0
Significant other observable inputs (Level 2)
Assets:
Cash and cash equivalents	0	0
Marketable debt securities, available-for-sale	420,463	388,557
Total	420,463	388,557
Liabilities:
Business combination-related contingent consideration	0	0
Total	0	0
Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	0	0
Marketable debt securities, available-for-sale	0	0
Total	0	0
Liabilities:
Business combination-related contingent consideration	74,100	71,200
Total	$ 74,100	$ 71,200

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FAIR VALUE MEASUREMENTS - Discount Rates Used (Details)	Jun. 30, 2023	Dec. 31, 2022
Payment Discount
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Measurement input	0.0690	0.0810
Cholbam | Revenue Discount
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Measurement input	0.0750	0.0775
Chenodal | Revenue Discount
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Measurement input	0.0750	0.0800

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FAIR VALUE MEASUREMENTS - Acquisition-related Contingent Consideration (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3)
Beginning balance	$ 71,200	$ 67,100
Changes in the fair value of contingent consideration	8,596	13,987
Contractual payments disbursed	(2,756)	(2,685)
Contractual payments included in accrued liabilities at June 30	(2,940)	(2,702)
Ending balance	$ 74,100	$ 75,700

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INTANGIBLE ASSETS - Additional Information (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended	6 Months Ended
	Mar. 31, 2023	Jun. 30, 2023	Jun. 30, 2023	Jun. 30, 2022	Dec. 31, 2022
Finite-Lived Intangible Assets [Line Items]
Payments to date under terms of agreement			$ 31,170	$ 16,579
Goodwill		$ 900	900		$ 900
Ligand License Agreement
Finite-Lived Intangible Assets [Line Items]
Substantial payments payable upon achievement of milestones		114,100	$ 114,100
Payments to date under terms of agreement	$ 23,000
Ligand License Agreement | Minimum
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage			15.00%
Ligand License Agreement | Maximum
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage			17.00%
Royalty Agreements
Finite-Lived Intangible Assets [Line Items]
Payments to date under terms of agreement		$ 500	$ 1,000

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INTANGIBLE ASSETS - Amortizable Intangible Assets (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Goodwill and Intangible Assets Disclosure [Abstract]
Finite-lived intangible assets	$ 334,685	$ 302,935
Less: accumulated amortization	(181,165)	(158,833)
Net carrying value	$ 153,520	$ 144,102

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INTANGIBLE ASSETS - Amortization (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Finite-Lived Intangible Assets [Line Items]
Total amortization expense	$ 13,345	$ 7,571	$ 22,332	$ 14,127
Research and development
Finite-Lived Intangible Assets [Line Items]
Total amortization expense	2,420	1,625	4,814	1,911
Selling, general and administrative
Finite-Lived Intangible Assets [Line Items]
Total amortization expense	$ 10,925	$ 5,946	$ 17,518	$ 12,216

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CONVERTIBLE NOTES PAYABLE - Schedule of Carrying Amount of Debt (Details) - Senior Notes - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Debt Instrument [Line Items]
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$ 376,403	$ 375,545
Senior Notes Due 2029
Debt Instrument [Line Items]
Interest rate percentage	2.25%	2.25%
Convertible senior notes	$ 316,250	$ 316,250
Unamortized debt issuance costs	$ (8,050)	$ (8,750)
Senior Notes Due 2025
Debt Instrument [Line Items]
Interest rate percentage	2.50%	2.50%
Convertible senior notes	$ 68,904	$ 68,904
Unamortized debt issuance costs	$ (701)	$ (859)

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CONVERTIBLE NOTES PAYABLE - Additional Information (Details) $ / shares in Units, $ in Thousands			3 Months Ended		6 Months Ended
	Mar. 11, 2022 USD ($)	Sep. 10, 2018 USD ($)	Jun. 30, 2023 USD ($) $ / shares	Jun. 30, 2022 USD ($)	Jun. 30, 2023 USD ($) day $ / shares	Jun. 30, 2022 USD ($)	Dec. 31, 2022
Debt Instrument [Line Items]
Debt instrument, repurchase amount Including accrued and unpaid interest	$ 213,800
Loss on extinguishment of debt			$ 0	$ 0	$ 0	$ 7,578
Write off of deferred debt financing costs						3,400
Interest expense recognized			$ 2,900	$ 3,000	$ 5,900	$ 5,500
Senior Notes | Senior Notes Due 2029
Debt Instrument [Line Items]
Long-term debt, excluding current maturities	316,300
Interest rate percentage			2.25%		2.25%		2.25%
Proceeds from issuance of debt	306,400
Debt issuance costs, net	9,900		$ 9,900		$ 9,900
Accrued interest			$ 2,400		$ 2,400
Debt instrument, convertible, threshold percentage of stock price trigger					130.00%
Conversion ratio					0.031374
Conversion price (in dollars per share) | $ / shares			$ 31.87		$ 31.87
Notes payable			$ 100,000		$ 100,000
Long-term debt, term			7 years		7 years
Effective interest percentage			2.74%		2.74%
Senior Notes | Senior Notes Due 2029 | Debt Conversion, Scenario One
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days | day					20
Debt instrument, convertible, threshold consecutive trading days | day					30
Debt instrument, convertible, threshold percentage of stock price trigger					130.00%
Senior Notes | Senior Notes Due 2029 | Debt Conversion, Scenario Two
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days | day					5
Debt instrument, convertible, threshold consecutive trading days | day					10
Debt instrument, convertible, threshold percentage of stock price trigger					98.00%
Senior Notes | Senior Notes Due 2029, Issued Pursuant to Underwriters Option
Debt Instrument [Line Items]
Long-term debt, excluding current maturities	41,300
Senior Notes | Senior Notes Due 2025
Debt Instrument [Line Items]
Long-term debt, excluding current maturities		$ 276,000
Interest rate percentage			2.50%		2.50%		2.50%
Proceeds from issuance of debt		267,200
Debt issuance costs, net		$ 8,800
Accrued interest			$ 500		$ 500
Debt instrument, convertible, threshold percentage of stock price trigger					130.00%
Conversion ratio					0.0257739
Conversion price (in dollars per share) | $ / shares			$ 38.80		$ 38.80
Long-term debt, term			7 years		7 years
Effective interest percentage			2.98%		2.98%
Debt instrument, repurchase amount	$ 207,100
Long-term debt, excluding current maturities, repaid if converted			$ 68,900		$ 68,900
Senior Notes | Senior Notes Due 2025 | Debt Conversion, Scenario One
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days | day					20
Debt instrument, convertible, threshold consecutive trading days | day					30
Debt instrument, convertible, threshold percentage of stock price trigger					130.00%
Senior Notes | Senior Notes Due 2025 | Debt Conversion, Scenario Two
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days | day					5
Debt instrument, convertible, threshold consecutive trading days | day					10
Debt instrument, convertible, threshold percentage of stock price trigger					98.00%

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CONVERTIBLE NOTES PAYABLE - Schedule of Interest Expense (Details) - Senior Notes Due 2025 and Senior Notes Due 2029 - Senior Notes - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Debt Instrument [Line Items]
Contractual interest expense	$ 2,210	$ 2,210	$ 4,419	$ 4,053
Amortization of debt issuance costs	429	429	858	766
Total interest expense for the 2025 and 2029 Notes	$ 2,639	$ 2,639	$ 5,277	$ 4,819

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ACCRUED EXPENSES (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Payables and Accruals [Abstract]
Research and development	$ 28,254	$ 26,070
Compensation related costs	21,489	35,267
Sales discounts, rebates, and allowances	14,376	13,486
Selling, general and administrative	11,252	8,791
Accrued royalties	8,467	7,755
Miscellaneous accrued expenses	4,911	4,373
Total accrued expenses	$ 88,749	$ 95,742

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NET LOSS PER COMMON SHARE - Additional Information (Details) - Underwritten Equity Offering	Feb. 28, 2023 $ / shares shares
Subsidiary, Sale of Stock [Line Items]
Sales price per share (in dollars per share)	$ 21.00
Common Stock
Subsidiary, Sale of Stock [Line Items]
Pre-funded warrants exercisable for share of common stock (in shares) | shares	1
Pre Funded Warrant
Subsidiary, Sale of Stock [Line Items]
Number of securities called by warrants or rights (in shares) | shares	1,250,000
Sales price per share (in dollars per share)	$ 20.9999
Exercise price of each pre-funded warrant (in dollars per share)	$ 0.0001

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NET LOSS PER COMMON SHARE - Basic and Diluted (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Earnings Per Share [Abstract]
Basic shares outstanding (in shares)	76,001,801	63,638,385	72,109,573	63,387,009
Diluted shares outstanding (in shares)	76,001,801	63,638,385	72,109,573	63,387,009
Net loss basic	$ (85,630)	$ (67,032)	$ (171,961)	$ (143,003)
Net loss diluted	$ (85,630)	$ (67,032)	$ (171,961)	$ (143,003)
Basic net loss per common share (in dollars per share)	$ (1.13)	$ (1.05)	$ (2.38)	$ (2.26)
Diluted net loss per common share (in dollars per share)	$ (1.13)	$ (1.05)	$ (2.38)	$ (2.26)

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NET LOSS PER COMMON SHARE - Antidilutive Shares (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	26,044,635	24,171,946	25,785,521	22,147,717
Convertible debt
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	11,697,952	11,697,952	11,697,952	10,026,309
Options
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	10,719,869	10,313,152	10,633,802	10,090,259
Restricted stock
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	3,626,814	2,160,842	3,453,767	2,031,149

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COMMITMENTS AND CONTINGENCIES (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended		6 Months Ended
	Oct. 31, 2021	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Loss Contingencies [Line Items]
Purchase commitment				$ 22,100
Total revenue		$ 59,697	$ 54,167	$ 116,690	$ 102,654
Kolbam | FRANCE
Loss Contingencies [Line Items]
Total revenue	$ 8,000

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SHARE-BASED COMPENSATION - Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended	6 Months Ended	12 Months Ended
	Jun. 30, 2023	Jun. 30, 2023	Dec. 31, 2022
Aggregate Intrinsic Value (in thousands)
Number of shares exercisable (in shares)	7,500,000	7,500,000
Weighted average exercise price (in dollars per share)	$ 20.71	$ 20.71
Stock Options
Shares Underlying Options
Beginning balance (in shares)		9,932,422
Granted (in shares)		1,157,750
Exercised (in shares)		(211,663)
Forfeited/canceled (in shares)		(292,208)
Ending balance (in shares)	10,586,301	10,586,301	9,932,422
Vested and expected to vest (in shares)	10,586,301	10,586,301
Weighted Average Exercise Price
Beginning balance (in dollars per share)		$ 21.56
Granted (in dollars per share)		21.69
Exercised (in dollars per share)		14.59
Forfeited/canceled (in dollars per share)		25.72
Ending balance (in dollars per share)	$ 21.60	21.60	$ 21.56
Vested and expected to vest (in dollars per share)	$ 21.60	$ 21.60
Weighted Average Remaining Contractual Life (years)		5 years 9 months 25 days	5 years 9 months 14 days
Vested and expected to vest		5 years 9 months 25 days
Aggregate Intrinsic Value (in thousands)
Aggregate intrinsic value, beginning balance		$ 24,658
Exercised		1,341
Aggregate intrinsic value, ending balance	$ 3,677	3,677	$ 24,658
Aggregate intrinsic value, vested and expected to vest	3,677	3,677
Unamortized stock compensation expense	35,000	$ 35,000
Weighted-average recognition period (in years)		2 years 7 months 6 days
Stock Options | Chief Executive Officer
Aggregate Intrinsic Value (in thousands)
Incremental compensation cost	$ 2,600	$ 2,600

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SHARE-BASED COMPENSATION - Service Based Restricted Stock Activity (Details) - Restricted stock units $ / shares in Units, $ in Millions	6 Months Ended
	Jun. 30, 2023 USD ($) $ / shares shares
Number of Restricted Stock Units
Beginning balance (in shares) | shares	2,343,709
Granted (in shares) | shares	1,773,692
Vested (in shares) | shares	(609,284)
Forfeited/canceled (in shares) | shares	(117,837)
Ending balance (in shares) | shares	3,390,280
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share) | $ / shares	$ 24.65
Granted (in dollars per share) | $ / shares	21.97
Vested (in dollars per share) | $ / shares	23.33
Forfeited/canceled (in dollars per share) | $ / shares	23.83
Ending balance (in dollars per share) | $ / shares	$ 23.52
Unamortized stock compensation expense | $	$ 67.8
Weighted-average recognition period (in years)	3 years

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SHARE-BASED COMPENSATION - Performance Based Restricted Stock Activity (Details) - Performance Shares $ / shares in Units, $ in Millions	6 Months Ended
	Jun. 30, 2023 USD ($) $ / shares shares
Number of Restricted Stock Units
Beginning balance (in shares) | shares	157,048
Granted (in shares) | shares	66,250
Vested (in shares) | shares	0
Forfeited/canceled (in shares) | shares	(22,840)
Ending balance (in shares) | shares	200,458
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share) | $ / shares	$ 25.24
Granted (in dollars per share) | $ / shares	22.40
Vested (in dollars per share) | $ / shares	0
Forfeited/canceled (in dollars per share) | $ / shares	24.86
Ending balance (in dollars per share) | $ / shares	$ 24.34
Unamortized stock compensation expense | $	$ 2.0
Weighted-average recognition period (in years)	10 months 24 days

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SHARE-BASED COMPENSATION - Stock based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total share-based compensation	$ 11,603	$ 12,637	$ 25,367	$ 20,823
Research and development
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total share-based compensation	4,615	3,684	9,096	6,852
Selling, general and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total share-based compensation	$ 6,988	$ 8,953	$ 16,271	$ 13,971

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INVENTORY - Schedule of Inventory (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Inventory Disclosure [Abstract]
Raw materials	$ 14,652	$ 3,627
Finished goods	4,113	3,295
Total inventory	$ 18,765	$ 6,922

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ACCOUNTS RECEIVABLE (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Receivables [Abstract]
Accounts receivable, net	$ 20,397	$ 16,646

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EQUITY OFFERINGS (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	6 Months Ended		12 Months Ended
	Feb. 28, 2023	Jun. 30, 2023	Jun. 30, 2022	Dec. 31, 2022	Feb. 29, 2020
Class of Stock [Line Items]
Proceeds from the issuance of pre-funded warrants, net of issuance costs		$ 24,630	$ 0
Underwritten Equity Offering
Class of Stock [Line Items]
Shares authorized in sale (in shares)	9,700,000
Sales price per share (in dollars per share)	$ 21.00
Proceeds from the issuance of common stock, net of issuance costs	$ 215,800
Underwritten Equity Offering | Common Stock
Class of Stock [Line Items]
Prefunded warrants, notice period for modifying limitations	61 days
Pre-funded warrants exercisable for share of common stock (in shares)	1
Underwritten Equity Offering | Pre Funded Warrant
Class of Stock [Line Items]
Number of securities called by warrants or rights (in shares)	1,250,000
Sales price per share (in dollars per share)	$ 20.9999
Exercise price of each pre-funded warrant (in dollars per share)	$ 0.0001
Proceeds from the issuance of pre-funded warrants, net of issuance costs	$ 24,600
At-The-Market Offering
Class of Stock [Line Items]
Shares authorized in sale (in shares)		0
Proceeds from the issuance of common stock, net of issuance costs				$ 20,100
Aggregate offering amount authorized					$ 100,000
Remaining offering amount authorized		$ 19,500
At-The-Market Offering Under Previous Registration Statement
Class of Stock [Line Items]
Amount sold to date		28,600
At-The-Market Offering Under Current Registration Statement
Class of Stock [Line Items]
Amount sold to date		$ 51,900

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SUBSEQUENT EVENTS (Details) - Mirum - Subsequent Event $ in Millions	Jul. 16, 2023 USD ($)
Subsequent Event [Line Items]
Asset acquisition, expected price of acquisition (up to)	$ 445.0
Asset acquisition, consideration due at closing	210.0
Asset acquisition, consideration due after closing	235.0
Minimum
Subsequent Event [Line Items]
Milestone payment based on specified amounts of annual net sales	125.0
Maximum
Subsequent Event [Line Items]
Milestone payment based on specified amounts of annual net sales	$ 500.0

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Label	Element	Value
Accounting Standards Update [Extensible Enumeration]	us-gaap_AccountingStandardsUpdateExtensibleList	Accounting Standards Update 2020-06 Cumulative Effect, Period of Adoption [Member]