ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) | |
(Address of principal executive offices) |
(Zip Code) |
Title of each class |
Trading symbol(s) |
Name of each exchange on which registered | ||
Large accelerated filer |
☐ |
☒ | ||||
Non-accelerated filer |
☐ |
Smaller reporting company |
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Emerging Growth Company |
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97 |
• | primary biliary cholangitis (PBC), an autoimmune disease that causes progressive destruction of the bile ducts in the liver resulting in impaired bile flow (cholestasis) and inflammation; |
• | nonalcoholic steatohepatitis (NASH), a prevalent and serious chronic liver disease caused by excessive fat accumulation in the liver that results in inflammation and cellular injury that can progress to fibrosis and cirrhosis, and potentially liver failure and death; and |
• | primary sclerosing cholangitis (PSC), a rare, chronic cholestatic liver disease characterized by diffuse inflammation and fibrosis of the intrahepatic and extrahepatic bile ducts. |
Product Candidates |
Disease/condition |
Status |
Description | |||
Seladelpar |
Primary Biliary Cholangitis (PBC) |
Phase 3 (terminated) |
52-week study to evaluate seladelpar in PBC patients with inadequate response or intolerance to ursodeoxycholic acid (UDCA) (NCT03602560)† | |||
Seladelpar |
Nonalcoholic Steatohepatitis (NASH) |
Phase 2 (terminated) |
52-week study to evaluate safety, tolerability, and effect of seladelpar in patients with NASH (NCT03551522)† | |||
Seladelpar |
Primary Sclerosing Cholangitis (PSC) |
Phase 2 (terminated) |
A 24-week study to evaluate the safety, tolerability, and efficacy of Seladelpar in patients with PSC(NCT04024813) † | |||
MBX-2982* (GPR 119 agonist) |
Gut/Liver |
Pre-IND |
Undisclosed indication(s) | |||
CB-001 (GPR 120 agonist) |
Gut/Liver |
Preclinical |
Undisclosed indication(s) |
* | Phase 2 (discontinued) in type 2 diabetes supported safety and pharmacokinetic profile, currently being explored for other indication(s). |
• | Significant reductions in markers of cholestasis, such as alkaline phosphatase (ALP) and gamma-glutamyl transferase (GGT), |
• | Decreases in high-sensitivity C-reactive protein (hs-CRP), a marker of inflammation, |
• | Lowered LDL-C and raised high-density-lipoprotein (HDL-C), and |
• | Decreased triglycerides and free fatty acids. |
• | Completion of preclinical laboratory tests, animal studies and formulation studies according to Good Laboratory Practices (GLP) or other applicable regulations; |
• | Submission to the FDA of an Investigational New Drug (IND) application, which must become effective before human clinical studies may begin; |
• | Performance of adequate and well-controlled human clinical studies according to the FDA’s current Good Clinical Practices (GCP), to establish the safety and efficacy of the proposed pharmaceutical product for its intended use; |
• | Submission to the FDA of a New Drug Application (NDA) for a new pharmaceutical product; |
• | Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced to assess compliance with the FDA’s current Good Manufacturing Practice standards (cGMP), to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; |
• | Potential FDA audit of selected preclinical and clinical study sites that generated the data in support of the NDA; and |
• | FDA review and approval of the NDA. |
• | Phase 1. The pharmaceutical product is initially introduced into healthy human subjects and tested for safety, dosage tolerance, absorption, metabolism, distribution and excretion. |
• | Phase 2. The pharmaceutical product is evaluated in a limited patient population to identify possible adverse effects and safety risks, to preliminarily evaluate the efficacy of the product for specific targeted diseases, to determine dosage tolerance, optimal dosage and dosing schedule and to identify patient populations with specific characteristics where the pharmaceutical product may be more effective. |
• | Phase 3. Clinical studies are undertaken to further evaluate dosage, clinical efficacy and safety in an expanded patient population at geographically dispersed clinical study sites. These clinical studies are intended to establish the overall risk/benefit ratio of the product and provide an adequate basis for product labeling. The studies must be well-controlled and usually include a control arm for comparison. One or two Phase 3 studies are required by the FDA for an NDA approval, depending on the disease severity and other available treatment options. |
• | Post-approval studies, or Phase 4 clinical studies, may be conducted after initial marketing approval. These studies are used to gain additional experience from the treatment of patients in the intended therapeutic indication. |
• | an annual, nondeductible fee on any entity that manufactures or imports certain branded prescription drugs and biologic agents, apportioned among these entities according to their market share in certain government healthcare programs; |
• | an increase in the rebates a manufacturer must pay under the Medicaid Drug Rebate Program to 23.1% and 13% of the average manufacturer price for branded and generic drugs, respectively; |
• | a new Medicare Part D coverage gap discount program, in which manufacturers must now agree to offer 70% point-of-sale discounts to negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D; |
• | extension of manufacturers’ Medicaid rebate liability to covered drugs dispensed to individuals who are enrolled in Medicaid managed care organizations; |
• | expansion of eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to additional individuals and by adding new mandatory eligibility categories for certain individuals with income at or below 133% of the Federal Poverty Level, thereby potentially increasing manufacturers’ Medicaid rebate liability; |
• | expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program; |
• | new transparency reporting requirements under the federal Physician Payments Sunshine Act, created under Section 6002 of the PPACA; |
• | a requirement to annually report drug samples that manufacturers and distributors provide to physicians; |
• | expansion of health care fraud and abuse laws, including the False Claims Act and the Anti-Kickback Statute, new government investigative powers, and enhanced penalties for noncompliance; |
• | a licensure framework for follow-on biologic products; |
• | a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research; and |
• | establishment of a Center for Medicare & Medicaid Innovation at the Centers for Medicare & Medicaid Services (CMS) to test innovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescription drug spending. |
Name |
Age |
Position Held With CymaBay | ||||
Executive Officers |
||||||
Sujal Shah |
46 |
President & Chief Executive Officer | ||||
Charles A. McWherter, Ph.D. |
64 |
Chief Scientific Officer | ||||
Klara Dickinson |
52 |
Chief Regulatory and Compliance Officer | ||||
Daniel Menold |
50 |
Vice President, Finance | ||||
Key Other Officers |
||||||
Robert L. Martin, Ph D |
57 |
Senior Vice President, Manufacturing and Nonclinical Development | ||||
Patrick J. O’Mara |
58 |
Senior Vice President, Business Development |
• | the rate of progress and cost of our clinical studies; |
• | the need for additional or expanded clinical studies; |
• | the rate of progress and cost of our Chemistry, Manufacturing and Control development, registration, validation and commercial programs; |
• | the timing, economic and other terms of any licensing, collaboration or other similar arrangement into which we may enter; |
• | the costs and timing of seeking and obtaining U.S. Food and Drug Administration (FDA) and other regulatory approvals; |
• | the extent of our other development activities; |
• | the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; and |
• | the effect of competing products and market developments. |
• | successful enrollment and completion of clinical trials; |
• | receipt of marketing approvals from the FDA and regulatory authorities outside the United States for the product candidate; |
• | establishing commercial manufacturing capabilities by making arrangements with third-party manufacturers; |
• | launching commercial sales of the product, whether alone or in collaboration with others; |
• | acceptance of the product by patients, the medical community and third-party payors; |
• | effectively competing with other therapies; |
• | a continued acceptable safety profile of the product following marketing approval; and |
• | obtaining, maintaining, enforcing and defending intellectual property rights and claims. |
• | regulators or institutional review boards may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site; |
• | the clinical study protocol may require one or more amendments delaying study completion; |
• | clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs; |
• | the number of subjects required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be insufficient or slower than we anticipate, we may have to compete with other clinical trials to enroll eligible subjects, or subjects may drop out of these clinical trials at a higher rate than we anticipate; |
• | clinical investigators or study subjects fail to comply with clinical study protocols; |
• | trial conduct and data analysis errors may occur, including, but not limited to, data entry and/or labeling errors; |
• | our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all; |
• | we might have to suspend or terminate clinical trials of our product candidates for various reasons, including a finding that the subjects are being exposed to unacceptable health risks; |
• | regulators or institutional review boards may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements; |
• | the cost of clinical trials of our product candidates may be greater than we anticipate; |
• | the supply or quality of our clinical trial materials or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; and |
• | our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators to suspend or terminate the trials. |
• | inability to raise funding necessary to initiate or continue a trial; |
• | delays in obtaining regulatory approval to commence a trial; |
• | delays in reaching agreement with the FDA or other regulatory authorities on final trial design; |
• | imposition of a clinical hold following a reported safety event; |
• | an inspection of our clinical trial operations or trial sites by the FDA or other regulatory authorities; |
• | delays in reaching agreement on acceptable terms with prospective contract research organizations (CROs) and clinical trial sites; |
• | delays in obtaining required institutional review board (IRB) approval at each site; |
• | delays in recruiting suitable patients to participate in a trial; |
• | delays in having subjects complete participation in a trial or return for post-treatment follow-up; |
• | delays caused by subjects dropping out of a trial due to side effects or otherwise; |
• | changes to treatment guidelines or the introduction of a new standard of care; |
• | delays caused by clinical sites dropping out of a trial; |
• | time required to add new clinical sites; |
• | delays by our contract manufacturers to produce and deliver sufficient supply of clinical trial materials; and |
• | delays in importing clinical trial materials into foreign countries where our clinical trials are being conducted. |
• | regulatory authorities may withdraw their approval of the product or impose restrictions on its distribution in a form of a risk evaluation and mitigation strategy (REMS) plan; |
• | regulatory authorities may require the addition of labeling statements, such as black box or other warnings or contraindications that could diminish the usage of the product or otherwise limit the commercial success of the affected product; |
• | we may be required to change the way the product is administered or to conduct additional clinical studies; |
• | we may choose to discontinue sale of the product; |
• | we could be sued and held liable for harm caused to patients; or |
• | our reputation may suffer. |
• | the efficacy and safety, as demonstrated in clinical studies; |
• | the risk/benefit profile of our product candidates; |
• | the prevalence and severity of any side effects; |
• | the clinical indications for which the product is approved; |
• | acceptance of the product by physicians, other health care providers and patients as a safe and effective treatment; |
• | the potential and perceived advantages of product candidates over alternative treatments; |
• | the safety of product candidates seen in a broader patient group, including if physicians prescribe our products for uses outside the approved indications; |
• | the cost of treatment in relation to alternative treatments; |
• | the timing of market introduction of competitive products; |
• | the availability of coverage and adequate reimbursement by third party payors and government authorities; |
• | relative convenience and ease of administration; and |
• | the effectiveness of our or our partners’ sales, marketing and distribution efforts. |
• | we may be unable to demonstrate to the satisfaction of regulatory authorities that a product candidate is safe and effective for any indication; |
• | regulatory authorities may not find the data from nonclinical studies and clinical studies sufficient or may differ in the interpretation of the data; |
• | regulatory authorities may require additional nonclinical or clinical studies; |
• | the FDA or foreign regulatory authority might not approve our third party manufacturers’ processes or facilities for clinical or commercial product; |
• | the FDA or foreign regulatory authority may change its approval policies or adopt new regulations; |
• | the FDA or foreign regulatory authority may disagree with the design or implementation of our clinical studies; |
• | the FDA or foreign regulatory authority may not accept clinical data from studies that are conducted in countries where the standard of care is potentially different from that in the United States; |
• | the results of clinical studies may not meet the level of statistical significance required by the FDA or foreign regulatory authorities for approval; |
• | we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks; and |
• | the data collection from clinical studies of our product candidates may not be sufficient to support the submission of a new drug application (NDA), marketing authorization or other equivalent submission, or to obtain regulatory approval in the United States or elsewhere. |
• | issue an untitled or warning letter asserting violation of the law; |
• | seek an injunction or impose civil or criminal penalties up to and including imprisonment or monetary fines; |
• | suspend or withdraw regulatory approval; |
• | suspend any ongoing clinical trials; |
• | refuse to approve a pending NDA or supplements to an NDA; or |
• | request recall and/or seize product. |
• | the inability to meet our product specifications, including product formulation, and quality requirements consistently; |
• | a delay or inability to procure or expand sufficient manufacturing capacity; |
• | manufacturing and product quality issues, including those related to scale-up of manufacturing; |
• | costs and validation of new equipment and facilities required for scale-up; |
• | a failure to comply with cGMP and similar quality standards; |
• | the inability to negotiate manufacturing agreements with third parties under commercially reasonable terms; |
• | termination or nonrenewal of manufacturing agreements with third parties in a manner or at a time that is costly or damaging to us; |
• | the reliance on a limited number of sources, and in some cases, single sources for key materials, such that if we are unable to secure a sufficient supply of these key materials, we will be unable to manufacture and sell our product candidates in a timely fashion, in sufficient quantities or under acceptable terms; |
• | the lack of qualified backup suppliers for those materials that are currently purchased from a sole or single source supplier; |
• | operations of our third-party manufacturers or suppliers could be disrupted by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or supplier; |
• | disruption of the distribution of chemical supplies between the U.K. and E.U. due to Brexit; |
• | carrier disruptions or increased costs that are beyond our control; and |
• | the failure to deliver our products under specified storage conditions and in a timely manner. |
• | demonstration of clinical safety and efficacy in our clinical trials; |
• | the risk/benefit profile of our product candidates; |
• | the relative convenience, ease of administration and acceptance by physicians, patients and health care payors; |
• | the prevalence and severity of any side effects; |
• | the safety of product candidates seen in a broader patient group, including its use outside the approved indications; |
• | limitations or warnings contained in the FDA and other regulatory authorities approved label for the relevant product candidate; |
• | acceptance of the product by physicians, other health care providers and patients as a safe and effective treatment; |
• | the potential and perceived advantages of product candidates over alternative treatments; |
• | the timing of market introduction of competitive products; |
• | pricing and cost-effectiveness; |
• | the effectiveness of our or any future collaborators’ sales and marketing strategies; |
• | our ability to obtain formulary approval; |
• | our ability to obtain and maintain sufficient third-party coverage or reimbursement, which may vary from country to country; and |
• | the effectiveness of our or any future collaborators’ sales, marketing and distribution efforts. |
• | different regulatory requirements for drug approvals in foreign countries; |
• | reduced protection for intellectual property rights; |
• | unexpected changes in tariffs, trade barriers and regulatory requirements; |
• | differing payor reimbursement regimes, governmental payors or patient self-pay systems and price controls; |
• | economic weakness, including inflation, or political instability in particular foreign economies and markets; |
• | compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; |
• | foreign taxes, including withholding of payroll taxes; |
• | foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country; |
• | workforce uncertainty in countries where labor unrest is more common than in the United States; |
• | production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and |
• | business interruptions resulting from geopolitical actions, including war and terrorism, pandemics, or natural disasters including earthquakes, typhoons, volcanic eruptions, floods and fires. |
• | research and development resources, including personnel and technology; |
• | regulatory experience; |
• | experience in pharmaceutical development and commercialization; |
• | ability to negotiate competitive pricing and reimbursement with third-party payors; |
• | experience and expertise in the exploitation of intellectual property rights; and |
• | capital resources. |
• | decreased demand for our product candidates; |
• | impairment to our business reputation; |
• | withdrawal of clinical study participants; |
• | distraction of management’s attention from our primary business; |
• | substantial monetary awards to patients or other claimants; |
• | the inability to commercialize our product candidates; and |
• | loss of revenues. |
• | adverse or inconclusive results or delays in preclinical testing or clinical trials; |
• | inability to obtain additional funding; |
• | any delay in filing an Investigational New Drug (IND) application or NDA for any of our future product candidates and any adverse development or perceived adverse development with respect to the FDA’s review of an IND or NDA; |
• | failure to maintain our existing collaborations or enter into new collaborations; |
• | failure of our collaboration partners to elect to develop or commercialize product candidates under our collaboration agreements or the termination of any programs under our collaboration agreements; |
• | failure by us or our licensors and collaboration partners to prosecute, maintain or enforce our intellectual property rights; |
• | failure to successfully develop and commercialize our future product candidates; |
• | changes in laws or regulations applicable to future products; |
• | changes in the structure of payment systems; |
• | inability to obtain adequate product supply for our future product candidates or the inability to do so at acceptable prices; |
• | adverse regulatory decisions; |
• | introduction of new products, services or technologies by our competitors; |
• | failure to meet or exceed financial projections we may provide to the public; |
• | failure to meet or exceed the estimates and projections of the investment community; |
• | the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; |
• | announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us, our collaboration partners or our competitors; |
• | announcements of significant or potential equity or debt sales by us; |
• | announcements of clinical trial plans or results by us; |
• | disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies; |
• | additions or departures of key scientific or management personnel; |
• | significant lawsuits, including patent or stockholder litigation; |
• | changes in the market valuations of similar companies; |
• | sales of our common stock by us or our stockholders in the future; and |
• | trading volume of our common stock. |
• | primary biliary cholangitis (PBC), an autoimmune disease that causes progressive destruction of the bile ducts in the liver resulting in impaired bile flow (cholestasis) and inflammation |
• | nonalcoholic steatohepatitis (NASH), a prevalent and serious chronic liver disease caused by excessive fat accumulation in the liver that results in inflammation and cellular injury that can progress to fibrosis and cirrhosis, and potentially liver failure and death |
• | primary sclerosing cholangitis (PSC), a rare, chronic cholestatic liver disease characterized by diffuse inflammation and fibrosis of the intrahepatic and extrahepatic bile ducts. |
• | contract research organizations and other service providers in connection with clinical studies; |
• | contract manufacturers in connection with the production of clinical trial materials; and |
• | vendors in connection with preclinical development activities. |
Year Ended |
||||||||||||
December 31, |
Change |
|||||||||||
2019 |
2018 |
2019 vs. 2018 |
||||||||||
Operating expenses: |
||||||||||||
Research and development |
$ | 83,837 |
$ | 58,124 |
25,713 |
|||||||
General and administrative |
19,238 |
14,381 |
4,857 |
|||||||||
Restructuring charges |
5,075 |
— |
5,075 |
|||||||||
Total operating expenses |
108,150 |
72,505 |
35,645 |
|||||||||
Loss from operations |
(108,150 |
) | (72,505 |
) | (35,645 |
) | ||||||
Other income (expense): |
||||||||||||
Interest income, net |
5,342 |
3,652 |
1,690 |
|||||||||
Loss on extinguishment of debt |
— |
(407 |
) | 407 |
||||||||
Other expense, net |
— |
(3,288 |
) | 3,288 |
||||||||
Net loss |
$ | (102,808 |
) | $ | (72,548 |
) | (30,260 |
) | ||||
Year Ended |
||||||||||||
December 31, |
Change |
|||||||||||
2019 |
2018 |
2019 vs. 2018 |
||||||||||
Operating expenses: |
||||||||||||
Research and development |
$ | 83,837 |
$ | 58,124 |
25,713 |
|||||||
General and administrative |
19,238 |
14,381 |
4,857 |
|||||||||
Restructuring charges |
5,075 |
— |
5,075 |
|||||||||
Total operating expenses |
$ | 108,150 |
$ | 72,505 |
35,645 |
|||||||
Year Ended December 31, |
Change |
|||||||||||
2019 |
2018 |
2019 vs. 2018 |
||||||||||
Project costs: |
||||||||||||
Seladelpar PBC clinical studies |
$ | 37,907 |
$ | 21,009 |
16,898 |
|||||||
Seladelpar NASH clinical studies |
10,445 |
15,614 |
(5,169 |
) | ||||||||
Seladelpar PSC clinical studies |
4,189 |
— |
4,189 |
|||||||||
Seladelpar drug manufacturing & development |
9,235 |
5,759 |
3,476 |
|||||||||
Seladelpar other studies |
2,442 |
1,181 |
1,261 |
|||||||||
Non-seladelpar studies |
361 |
184 |
177 |
|||||||||
Total project costs |
64,579 |
43,747 |
20,832 |
|||||||||
Internal research and development costs |
19,258 |
14,377 |
4,881 |
|||||||||
Total research and development |
$ | 83,837 |
$ | 58,124 |
25,713 |
|||||||
• | expenses incurred under agreements with contract research organizations, investigative sites and consultants that conduct our clinical trials and a substantial portion of our preclinical activities; |
• | the cost of acquiring and manufacturing clinical trial and other materials; and |
• | other costs associated with development activities, including additional studies. |
Year Ended |
||||||||||||
December 31, |
Change |
|||||||||||
2019 |
2018 |
2019 vs. 2018 |
||||||||||
Other income (expense): |
||||||||||||
Interest income, net |
$ | 5,342 |
$ | 3,652 |
1,690 |
|||||||
Loss on extinguishment of debt |
— |
(407 |
) | 407 |
||||||||
Other expense, net |
— |
(3,288 |
) | 3,288 |
||||||||
Total other income (expense) |
$ | 5,342 |
$ | (43 |
) | 5,385 |
||||||
Year Ended December 31, |
||||||||
2019 |
2018 |
|||||||
Net cash used in operating activities |
$ | (97,911 |
) | $ | (54,936 |
) | ||
Net cash used in investing activities |
(34,347 |
) | (54,111 |
) | ||||
Net cash provided by financing activities |
108,132 |
134,988 |
||||||
Net (decrease) increase in cash and cash equivalents |
$ | (24,126 |
) | $ | 25,941 |
|||
(a) | Documents filed as part of this report |
Page |
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70 |
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71 |
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72 |
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73 |
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74 |
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75 |
Exhibit No. |
Description of Document | |||
3.1 |
||||
3.2 |
||||
4.1 |
||||
4.2 |
||||
10.1* |
||||
10.2* |
||||
10.3* |
||||
10.4* |
Exhibit No. |
Description of Document | |||
10.5* |
||||
10.6* |
||||
10.7 |
||||
10.8# |
||||
10.9 |
||||
10.10 |
||||
10.11* |
||||
10.12* |
||||
10.13* |
||||
10.14* |
||||
10.15* |
||||
10.16* |
||||
10.17* |
||||
10.18* |
||||
21.1 |
||||
23.1 |
Exhibit No. |
Description of Document | |||
24.1 |
||||
31.1 |
||||
31.2 |
||||
32.1 |
||||
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 |
Inline XBRL Taxonomy Extension Presentation Document | |||
104 |
Cover Page Interactive Data File (formatted as inline XBRL and contained in exhibit 101) |
* | Indicates management contract or compensatory plan. |
# | Portions of this exhibit have been omitted pursuant to a grant of confidential treatment, which portions were omitted and filed separately with the Securities and Exchange Commission. |
Page |
||||
70 |
||||
71 |
||||
72 |
||||
73 |
||||
74 |
||||
75 |
December 31, |
||||||||
2019 |
2018 |
|||||||
Assets |
||||||||
Current assets: |
||||||||
Cash and cash equivalents |
$ | |
$ | |
||||
Marketable securities |
|
|
||||||
Accrued interest receivable |
|
|
||||||
Prepaid research and development expenses |
|
|
||||||
Other prepaid expenses |
|
|
||||||
Total current assets |
|
|
||||||
Property and equipment, net |
|
|
||||||
Operating lease right-of-use assets |
|
— |
||||||
Other assets |
|
|
||||||
Total assets |
$ | |
$ | |
||||
Liabilities and stockholders’ equity |
||||||||
Current liabilities: |
||||||||
Accounts payable |
$ | |
$ | |
||||
Accrued research and development expenses |
|
|
||||||
Accrued restructuring |
|
|
|
|
|
|
— |
|
Other accrued liabilities |
|
|
||||||
Total current liabilities |
|
|
||||||
Long-term portion of operating lease liability |
|
— |
||||||
Other liabilities |
— |
|
||||||
Total liabilities |
|
|
||||||
Commitments and contingencies |
||||||||
Stockholders’ equity: |
||||||||
Preferred stock, $ |
|
|
||||||
Common stock, $ |
|
|
||||||
Additional paid-in capital |
|
|
||||||
Accumulated other comprehensive income (loss) |
|
( |
) | |||||
Accumulated deficit |
( |
) | ( |
) | ||||
Total stockholders’ equity |
|
|
||||||
Total liabilities and stockholders’ equity |
$ | |
$ | |
||||
Year Ended |
||||||||
December 31, |
||||||||
2019 |
2018 |
|||||||
Operating expenses: |
||||||||
Research and development |
$ |
|
$ |
|
||||
General and administrative |
|
|
||||||
Restructuring charges |
|
— |
||||||
Total operating expenses |
|
|
||||||
Loss from operations |
( |
) | ( |
) | ||||
Other income (expense): |
||||||||
Interest income |
|
|
||||||
Interest expense |
— |
( |
) | |||||
Loss on extinguishment of debt |
— |
( |
) | |||||
Other expense, net |
— |
( |
) | |||||
Total other income (expense) |
|
( |
) | |||||
Net loss |
$ | ( |
) | $ | ( |
) | ||
Other comprehensive income (loss): |
||||||||
Unrealized gain (loss) on marketable securities |
|
( |
) | |||||
Other comprehensive income (loss) |
|
( |
) | |||||
Comprehensive loss |
$ | ( |
) | $ | ( |
) | ||
Basic net loss per common share |
$ | ( |
) | $ | ( |
) | ||
Diluted net loss per common share |
$ |
( |
) | $ | ( |
) | ||
Weighted average common shares outstanding used to calculate basic net loss per common share |
|
|
||||||
Weighted average common shares outstanding used to calculate diluted net loss per common share |
|
|
|
|
|
Additional Paid-in Capital |
Accumulated Other Comprehensive Income (Loss) |
Accumulated Deficit |
Total Stockholders’ Equity |
||||||||||||||||||
|
|
|
||||||||||||||||||||||
|
Common Stock |
|||||||||||||||||||||||
|
Shares |
Amount |
||||||||||||||||||||||
Balances as of December 31, 2017 |
|
$ |
|
$ |
|
$ |
( |
) | $ |
( |
) | $ |
|
|||||||||||
Issuance of common stock upon exercise of warrants |
|
— |
|
— |
— |
|
||||||||||||||||||
Issuance of common stock upon exercise of stock options |
|
— |
|
— |
— |
|
||||||||||||||||||
Stock-based compensation expense |
— |
— |
|
— |
— |
|
||||||||||||||||||
Issuance of common stock, net of $ issuance costs |
|
|
|
— |
— |
|
||||||||||||||||||
Net loss |
— |
— |
— |
— |
( |
) | ( |
) | ||||||||||||||||
Net unrealized loss on marketable securities |
— |
— |
— |
( |
) | — |
( |
) | ||||||||||||||||
Balances as of December 31, 2018 |
|
$ | |
$ | |
$ | ( |
) | $ | ( |
) | $ | |
|||||||||||
Issuance of common stock upon exercise of stock options |
|
— |
|
— |
— |
|
||||||||||||||||||
Stock-based compensation expense |
— |
— |
|
— |
— |
|
||||||||||||||||||
Issuance of common stock, net of $ issuance costs |
|
|
|
— |
— |
|
||||||||||||||||||
Net loss |
— |
— |
— |
— |
( |
) | ( |
) | ||||||||||||||||
Net unrealized gain on marketable securities |
— |
— |
— |
|
— |
|
||||||||||||||||||
Balances as of December 31, 2019 |
|
$ | |
$ | |
$ | |
$ | ( |
) | $ | |
||||||||||||
Year Ended December 31, |
||||||||
2019 |
2018 |
|||||||
Operating activities |
||||||||
Net loss |
$ |
( |
) | $ |
( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
||||||||
Depreciation and amortization |
|
|
||||||
Stock-based compensation expense |
|
|
||||||
Accelerated vesting of stock-based compensation expense due to restructuring |
|
— |
||||||
Net accretion and amortization of investments in marketable securities |
( |
) | ( |
) | ||||
Non-cash interest associated with debt discount accretion |
— |
|
||||||
Loss on extinguishment of debt |
— |
|
||||||
Change in fair value of warrant liability |
— |
|
||||||
Gain on extinguishment of warrant liability |
— |
( |
) | |||||
Accretion of tenant improvement allowance |
— |
( |
) | |||||
Changes in assets and liabilities: |
||||||||
Receivable from collaboration |
— |
|
||||||
Interest receivable and other current assets |
( |
) | ( |
) | ||||
Prepaid research and development and other prepaid expenses |
( |
) | ( |
) | ||||
Other assets |
|
( |
) | |||||
Accounts payable |
|
|
||||||
Accrued restructuring |
|
|
|
|
|
|
— |
|
Accrued liabilities |
( |
) | |
|||||
Accrued interest payable |
— |
( |
) | |||||
Net cash used in operating activities |
( |
) | ( |
) | ||||
|
|
|
|
|
|
|
|
|
Investing activities |
||||||||
Purchases of property and equipment |
( |
) | ( |
) | ||||
Purchases of marketable securities |
( |
) | ( |
) | ||||
Proceeds from maturities of marketable securities |
|
|
||||||
Proceeds from sale of marketable securities |
|
— |
||||||
Net cash used in investing activities |
( |
) | ( |
) | ||||
|
|
|
|
|
|
|
|
|
Financing activities |
||||||||
Proceeds from issuance of common stock, net of issuance costs |
|
|
||||||
Proceeds from issuance of common stock pursuant to equity award plans |
|
|
||||||
Proceeds from issuance of common stock upon exercise of warrants |
— |
|
||||||
Repayment of facility loan principal |
— |
( |
) | |||||
Payment of fees to extinguish facility loan |
— |
( |
) | |||||
Net cash provided by financing activities |
|
|
||||||
Net (decrease) increase in cash and cash equivalents |
( |
) | |
|||||
Cash and cash equivalents at beginning of period |
|
|
||||||
Cash and cash equivalents at end of period |
$ | |
$ | |
||||
|
|
|
|
|
|
|
|
|
Supplemental disclosures |
||||||||
Cash paid for amounts included in the measurement of lease liabilities |
$ | |
$ | — |
||||
Cash paid for interest |
— |
|
||||||
|
|
|
|
|
|
| ||
Supplemental non-cash investing and financing activities |
||||||||
Issuance of common stock upon warrant exercises |
$ | — |
$ | |
||||
Operating lease right-of-use assets obtained in exchange for lease liabilities |
|
|
|
|
|
— |
| |
Lessor funded lease incentives included in property and equipment |
— |
|
||||||
Accrued property and equipment |
— |
|
As of December 31, 2019 |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
|
Total |
||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ |
$ |
— |
$ |
— |
$ |
||||||||||
Total cash equivalents |
— |
— |
||||||||||||||
Short-term investments: |
||||||||||||||||
U.S. and foreign commercial paper |
— |
— |
||||||||||||||
U.S. and foreign corporate debt securities |
— |
— |
||||||||||||||
Asset-backed securities |
— |
— |
||||||||||||||
U.S. treasury securities |
— |
— |
||||||||||||||
Total short-term investments |
— |
— |
||||||||||||||
Total assets measured at fair value |
$ | |
$ | $ | — |
$ | ||||||||||
As of December 31, 2018 |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ |
$ |
— |
$ |
— |
$ |
||||||||||
U.S. and foreign commercial paper |
— |
— |
||||||||||||||
Total cash equivalents |
— |
|||||||||||||||
Short-term investments: |
||||||||||||||||
U.S. and foreign commercial paper |
— |
— |
||||||||||||||
U.S. and foreign corporate debt securities |
— |
— |
||||||||||||||
Asset-backed securities |
— |
— |
||||||||||||||
U.S. treasury securities |
— |
— |
||||||||||||||
Total short-term investments |
— |
— |
||||||||||||||
Total assets measured at fair value |
$ | $ | $ | — |
$ | |||||||||||
Year Ended December 31, |
||||||||
2019 |
2018 |
|||||||
Balance, beginning of period |
$ | — |
$ | |||||
Change in fair value |
— |
|||||||
Settlement of financial instruments |
— |
( |
) | |||||
Extinguishment of financial instruments |
— |
( |
) | |||||
Balance, end of period |
$ | — |
$ | — |
||||
Year Ended December 31, |
||||||||
2019 |
2018 |
|||||||
Numerator: |
||||||||
Net loss allocated to common stock—basic |
$ |
( |
) |
$ |
( |
) | ||
Adjustment for revaluation and extinguishment of common stock warrants |
— |
( |
) | |||||
Net loss allocated to common stock—diluted |
$ |
( |
) |
$ |
( |
) | ||
Denominator: |
||||||||
Weighted average number of common stock shares outstanding—basic |
||||||||
Dilutive securities: |
||||||||
Common stock warrants |
— |
|||||||
Weighted average number of common stock shares outstanding—diluted |
||||||||
Net loss per share—basic |
$ |
( |
) |
$ |
( |
) | ||
Net loss per share—diluted |
$ |
( |
) |
$ |
( |
) |
Year Ended December 31, |
||||||||
2019 |
2018 |
|||||||
Common stock options |
||||||||
Incentive awards |
||||||||
Total |
||||||||
Amortized Cost |
Gross Unrealized Gains |
Gross Unrealized Losses |
Estimated Fair Value |
|||||||||||||
As of December 31, 2019: |
||||||||||||||||
Cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Money market funds |
|
$ |
|
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
|
U.S. and foreign commercial paper |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
Total cash equivalents |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
|
Short-term investments: |
|
|
|
|
|
|
|
|
|
|
|
| ||||
U.S. and foreign commercial paper |
|
— |
— |
|
||||||||||||
U.S. and foreign corporate debt securities |
|
|
— |
|
||||||||||||
Asset-backed securities |
|
|
— |
|
||||||||||||
U.S. treasury securities |
|
|
— |
|
||||||||||||
Total short-term investments |
|
|
|
|
|
|
|
|
|
|
— |
|
|
|
|
|
Total marketable securities |
$ | |
$ | |
$ | — |
$ | |
||||||||
Amortized Cost |
Gross Unrealized Gains |
Gross Unrealized Losses |
Estimated Fair Value |
|||||||||||||
As of December 31, 2018: |
||||||||||||||||
Cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Money market funds |
|
$ |
|
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
|
U.S. and foreign commercial paper |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
|
Total cash equivalents |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
|
Short-term investments: |
|
|
|
|
|
|
|
|
|
|
|
| ||||
U.S. and foreign commercial paper |
|
— |
— |
|
||||||||||||
U.S. and foreign corporate debt securities |
|
— |
( |
) | |
|||||||||||
Asset-backed securities |
|
— |
( |
) | |
|||||||||||
U.S. treasury securities |
|
— |
( |
) | |
|||||||||||
Total short-term investments |
|
|
|
|
|
|
— |
|
|
|
( |
) |
|
|
|
|
Total marketable securities |
$ | |
$ | — |
$ | ( |
) | $ | |
|||||||
December 31, |
||||||||
2019 |
2018 |
|||||||
Leasehold improvements |
$ | |
$ | |
||||
Office and computer equipment |
|
|
||||||
Purchased software |
|
|
||||||
Furniture and fixtures |
|
|
||||||
Total |
|
|
||||||
Less accumulated depreciation and amortization |
( |
) | ( |
) | ||||
Property and equipment, net |
$ | |
$ | |
||||
December 31, |
||||||||
2019 |
2018 |
|||||||
Accrued compensation |
$ | |
$ | |
||||
Operating lease liability |
|
— |
||||||
Accrued professional fees and other |
|
|
||||||
Deferred rent |
— |
|
||||||
Other accr u ed liabiliti es |
$ | |
$ | |
||||
Operating Leases |
||||
Year ending December 31, |
||||
2020 |
|
|||
2021 |
|
|||
2022 |
|
|||
2023 |
|
|||
2024 |
|
|||
Total undiscounted future minimum lease payments |
$ | |
||
Less: Imputed interest |
|
|||
Total operating lease liability |
$ | |
||
Less: Current portion of operating lease liability (included in other accrued liabilities) |
|
|||
Long-term portion of operating lease liability |
$ | |
||
December 31, |
||||||||
2019 |
2018 |
|||||||
Equity incentive plan |
|
|
||||||
Total reserved shares of common stock |
|
|
||||||
Shares Subject to Outstanding Options |
Weighted Average Exercise Price of Options |
Weighted Average Remaining Contractual Term (Years) |
Aggregate Intrinsic Value (in thousands) |
|||||||||||||
Outstanding as of December 31, 2018 |
|
$ | |
|||||||||||||
Options granted |
|
|
||||||||||||||
Options exercised |
( |
) | |
|||||||||||||
Options forfeited |
( |
) | |
|||||||||||||
Options expired |
( |
) | |
|||||||||||||
Outstanding as of December 31, 2019 |
|
$ | |
$ | |
$ | |
|||||||||
Vested and expected to vest as of December 31, 2019 |
|
|
|
$ | |
|||||||||||
Exercisable as of December 31, 2019 |
|
$ | |
$ | |
$ | |
|||||||||
Year Ended |
||||||||
December 31, |
||||||||
2019 |
2018 |
|||||||
Research and development |
$ | |
$ | |
||||
General and administrative |
|
|
||||||
Restructuring charges |
|
— |
||||||
Total stock-based compensation expense |
$ | |
$ | |
||||
Year Ended December 31, |
||||||||
2019 |
2018 |
|||||||
Expected term (in years) |
|
|
||||||
Expected volatility |
|
% | |
% | ||||
Risk-free interest rate |
|
% | |
% | ||||
Expected dividend yield |
|
% |
— |
% | ||||
Weighted-average grant date fair value per share |
$ | |
$ | |
December 31 |
||||||||
2019 |
2018 |
|||||||
Deferred tax assets: |
||||||||
Federal and state net operating loss carryforwards |
$ | |
$ | |
||||
Federal and state research and development tax credit carryforwards |
|
|
||||||
Capitalized research and development |
|
|
||||||
Stock based compensation |
|
|
||||||
Other |
|
|
||||||
Total deferred tax assets |
|
|
||||||
Deferred tax liabilities: |
||||||||
Depreciation and amortization |
( |
) | ( |
) | ||||
Other |
|
|
( |
) |
|
|
— |
|
Total deferred tax liabilities |
( |
) | ( |
) | ||||
Valuation allowance |
( |
) | ( |
) | ||||
Net deferred tax assets |
$ | |
$ | |
||||
December 31 |
||||||||
2019 |
2018 |
|||||||
Income tax benefit at federal statutory tax rate |
$ | ( |
) | $ | ( |
) | ||
Change in valuation allowance |
|
|
||||||
State income taxes, net of federal benefit |
|
( |
) | |||||
Research credits |
( |
) | ( |
) | ||||
Other |
|
|
|
|
|
|
|
|
Income tax (benefit) expense |
$ | |
$ | — |
||||
Total |
||||
Balances as of December 31, 2017 |
$ | |
||
Increases related to prior year tax positions |
|
|||
Increases related to 2018 tax positions |
|
|||
Balances as of December 31, 2018 |
$ | |
||
Increases related to prior year tax positions |
|
|||
Increases related to 2019 tax positions |
|
|||
Balances as of December 31, 2019 |
$ | |
||
Termination Benefits |
Contract Termination Costs |
Total |
|||||||||||
Balances as of January 1, 2019 |
$ | |
$ | |
$ | |
|||||||
Restructuring charges |
|
|
|
||||||||||
Reductions for cash payments |
( |
) | — |
( |
) | ||||||||
Balances as of December 31, 2019 |
$ | |
$ | |
$ | |
|||||||
CymaBay Therapeutics, Inc. | ||||||
Registrant | ||||||
March 16, 2020 |
/s/ Sujal Shah | |||||
Date |
Sujal Shah President and Chief Executive Officer |
Name and Signature |
Title |
Date | ||
/s/ Sujal Shah Sujal Shah |
President, Chief Executive Officer and Director (Principal Executive Officer) |
March 16, 2020 | ||
/s/ Daniel Menold Daniel Menold |
Vice President, Finance (Principal Financial Officer) |
March 16, 2020 | ||
/s/ Robert J. Wills Robert J. Wills, Ph.D. |
Director |
March 16, 2020 | ||
/s/ Kurt von Emster Kurt von Emster, CFA |
Director |
March 16, 2020 | ||
/s/ Caroline Loewy Caroline Loewy |
Director |
March 16, 2020 | ||
/s/ Paul F. Truex Paul F. Truex |
Director |
March 16, 2020 |