United States
Securities and Exchange Commission
Washington, D.C. 20549
FORM
(Mark One)
| QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For
the quarterly period ended
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
(Exact name of registrant as specified in its charter)
(State or other jurisdiction of incorporation or organization) |
(IRS Employer Identification No.) |
Securities registered pursuant to Section 12(b) of the Exchange Act:
| Title of Each Class | Trading Symbol(s) | Name of each exchange on which registered | ||
| The
|
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.
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 during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or emerging growth company. See definition 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 | ☐ |
| ☐ | 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 ☐
The number of outstanding shares of the registrant’s common stock as of November 1, 2022 was .
PROCESSA PHARMACEUTICALS, INC.
TABLE OF CONTENTS
| 2 |
PART 1: FINANCIAL INFORMATION
ITEM 1: FINANCIAL STATEMENTS
Processa Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(Unaudited)
| September 30, 2022 | December 31, 2021 | |||||||
| ASSETS | ||||||||
| Current Assets | ||||||||
| Cash and cash equivalents | $ | $ | ||||||
| Due from tax agencies | ||||||||
| Prepaid expenses and other | ||||||||
| Total Current Assets | ||||||||
| Property and Equipment, net | ||||||||
| Other Assets | ||||||||
| Operating lease right-of-use assets, net of accumulated amortization | ||||||||
| Intangible assets, net of accumulated amortization | ||||||||
| Security deposit | ||||||||
| Total Other Assets | ||||||||
| Total Assets | $ | $ | ||||||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||||
| Current Liabilities | ||||||||
| Current maturities of operating lease liability | $ | $ | ||||||
| Accounts payable | ||||||||
| Due to licensor | ||||||||
| Due to related parties | ||||||||
| Accrued expenses | ||||||||
| Total Current Liabilities | ||||||||
| Non-current Liabilities | ||||||||
| Non-current operating lease liability | ||||||||
| Total Liabilities | ||||||||
| Commitments and Contingencies | ||||||||
| Stockholders’ Equity | ||||||||
| Common stock, par value $, shares authorized: issued and outstanding at September 30, 2022, and issued and outstanding at December 31, 2021 | ||||||||
| Additional paid-in capital | ||||||||
| Treasury stock at cost — shares at September 30, 2022 | ( | ) | ||||||
| Accumulated deficit | ( | ) | ( | ) | ||||
| Total Stockholders’ Equity | ||||||||
| Total Liabilities and Stockholders’ Equity | $ | $ | ||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
| 3 |
Processa Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(Unaudited)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Operating Expenses | ||||||||||||||||
| Research and development expenses | $ | $ | $ | $ | ||||||||||||
| Acquisition of in-process research and development | ||||||||||||||||
| General and administrative expenses | ||||||||||||||||
| Operating Loss | ( | ) | ( | ) | ( | ) | ( | ) | ||||||||
| Other Income (Expense) | ||||||||||||||||
| Forgiveness of PPP loan and related accrued interest | ||||||||||||||||
| Interest income, net | ||||||||||||||||
| Net Operating Loss Before Income Tax Benefit | ( | ) | ( | ) | ( | ) | ( | ) | ||||||||
| Income Tax Benefit | ||||||||||||||||
| Net Loss | $ | ( | ) | $ | ( | ) | $ | ( | ) | $ | ( | ) | ||||
| Net Loss per Common Share - Basic and Diluted | $ | ( | ) | $ | ( | ) | $ | ( | ) | $ | ( | ) | ||||
| Weighted Average Common Shares Used to Compute Net Loss Applicable to Common Shares - Basic and Diluted | ||||||||||||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
| 4 |
Processa Pharmaceuticals, Inc.
Condensed Consolidated Statements of Changes in Stockholders’ Equity
Three and Nine Months Ended September 30, 2021 and 2022
(Unaudited)
| Additional | ||||||||||||||||||||||||||||
| Common Stock | Paid-In | Treasury Stock | Accumulated | |||||||||||||||||||||||||
| Shares | Amount | Capital | Shares | Amount | Deficit | Total | ||||||||||||||||||||||
| Balance at January 1, 2021 | $ | $ | $ | $ | $ | ( | ) | $ | ||||||||||||||||||||
| Stock-based compensation | ||||||||||||||||||||||||||||
| Shares issued in private placement, net of transaction costs | ||||||||||||||||||||||||||||
| Net loss | - | - | ( | ) | ( | ) | ||||||||||||||||||||||
| Balance, March 31, 2021 | ( | ) | ||||||||||||||||||||||||||
| Stock-based compensation | ||||||||||||||||||||||||||||
| Shares issued in connection with license agreement | ||||||||||||||||||||||||||||
| Net loss | - | - | ( | ) | ( | ) | ||||||||||||||||||||||
| Balance, June 30, 2021 | ( | ) | ||||||||||||||||||||||||||
| Stock-based compensation | ( | ) | ||||||||||||||||||||||||||
| Shares withheld to pay income taxes on stock-based compensation | ( | ) | ( | ) | ( | ) | ||||||||||||||||||||||
| Net loss | - | - | ( | ) | ( | ) | ||||||||||||||||||||||
| Balance, September 30, 2021 | $ | $ | $ | $ | ( | ) | $ | |||||||||||||||||||||
| Additional | ||||||||||||||||||||||||||||
| Common Stock | Paid-In | Treasury Stock | Accumulated | |||||||||||||||||||||||||
| Shares | Amount | Capital | Shares | Amount | Deficit | Total | ||||||||||||||||||||||
| Balance at January 1, 2022 | $ | $ | $ | $ | ( | ) | $ | |||||||||||||||||||||
| Stock-based compensation | - | |||||||||||||||||||||||||||
| Acquisition of treasury stock | - | ( | ) | ( | ) | ( | ) | |||||||||||||||||||||
| Shares issued in connection with the Purchase Agreement with Lincoln Park | - | |||||||||||||||||||||||||||
| Net loss | - | - | ( | ) | ( | ) | ||||||||||||||||||||||
| Balance, March 31, 2022 | ( | ) | ( | ) | ( | ) | ||||||||||||||||||||||
| Stock-based compensation and forfeitures | ( | ) | ( | ) | ||||||||||||||||||||||||
| Net loss | - | - | ( | ) | ( | ) | ||||||||||||||||||||||
| Balance, June 30, 2022 | ( | ) | ( | ) | ( | ) | ||||||||||||||||||||||
| Stock-based compensation and forfeitures | ||||||||||||||||||||||||||||
| Shares withheld to pay income taxes on stock-based compensation | ( | ) | ( | ) | ( | ) | ( | ) | ||||||||||||||||||||
| Net loss | - | - | ( | ) | ( | ) | ||||||||||||||||||||||
| Balance, September 30, 2022 | $ | $ | ( | ) | $ | ( | ) | $ | ( | ) | $ | |||||||||||||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
| 5 |
Processa Pharmaceuticals, Inc.
Condensed Consolidated Statements of Cash Flows
(Unaudited)
| Nine Months Ended September 30, | ||||||||
| 2022 | 2021 | |||||||
| Cash Flows From Operating Activities | ||||||||
| Net loss | $ | ( | ) | $ | ( | ) | ||
| Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||
| Depreciation | ||||||||
| Lease expense for right-of-use assets | ||||||||
| Milestone expense in connection with license agreement | ||||||||
| Acquisition of in-process research and development | ||||||||
| Amortization of issuance costs | ||||||||
| Amortization of intangible asset | ||||||||
| Deferred income tax benefit | ( | ) | ||||||
| Stock-based compensation | ||||||||
| Forgiveness of PPP loan and related accrued interest | ( | ) | ||||||
| Net changes in operating assets and liabilities: | ||||||||
| Prepaid expenses and other | ( | ) | ||||||
| Operating lease liability | ( | ) | ( | ) | ||||
| Accrued interest | ||||||||
| Accounts payable | ||||||||
| Due (from) to related parties | ( | ) | ||||||
| Other receivables | ||||||||
| Accrued expenses | ( | ) | ||||||
| Net Cash Used In Operating Activities | ( | ) | ( | ) | ||||
| Cash Flows From Financing Activities | ||||||||
| Net proceeds from issuance of common stock | ||||||||
| Shares withheld to pay taxes on stock-based compensation | ( | ) | ( | ) | ||||
| Acquisition of treasury stock | ( | ) | ||||||
| Other | ( | ) | ||||||
| Net Cash (Used In) Provided By Financing Activities | ( | ) | ||||||
| Net Increase (Decrease) in Cash | ( | ) | ||||||
| Cash and Cash Equivalents – Beginning of Period | ||||||||
| Cash and Cash Equivalents – End of Period | $ | $ | ||||||
| Non-Cash Financing Activities | ||||||||
| Issuance of shares of common stock in connection with the Purchase Agreement with Lincoln Park | $ | $ | ||||||
| Right-of-use asset obtained in exchange for operating lease liability | $ | ( | ) | |||||
| Operating lease liability | ||||||||
| Net | ||||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
| 6 |
Processa Pharmaceuticals, Inc.
Notes to Condensed Consolidated Financial Statements
(Unaudited)
Note 1 – Organization and Summary of Significant Accounting Policies
Business Activities and Organization
We are a diversified clinical-stage biopharmaceutical company focused on the development of drug products that are intended to provide treatment for patients who have a high unmet medical need condition that affects survival or the patient’s quality of life and for which few or no treatment options currently exist. We currently have five drugs in our pipeline: four in various stages of clinical development (Next Generation Capecitabine previously identified as PCS6422, PCS12852, PCS499, and PCS3117) and one in nonclinical development (PCS11T). A summary of each drug is provided below.
| ● | Next Generation Capecitabine (NGC), previously identified as PCS6422, is a combination of PCS6422 and the FDA-approved cancer drug capecitabine. PCS6422 is an orally administered irreversible inhibitor of the enzyme dihydropyrimidine dehydrogenase (DPD). DPD metabolizes 5-Fluorouracil (5-FU), the major metabolite of capecitabine and widely used itself as an intravenous chemotherapeutic agent in many types of cancer, to multiple metabolites classified as catabolites. These catabolites do not have any cancer-killing properties but do cause dose-limiting side effects that may require dose adjustments or discontinuation of therapy.
When combining capecitabine with PCS6422 in NGC, capecitabine becomes a more potent cancer chemotherapy agent than current FDA-approved capecitabine requiring significantly lower doses. On August 2, 2021, we enrolled the first patient in our Phase 1B dose-escalation maximum tolerated dose trial in patients with advanced refractory gastrointestinal (GI) tract tumors. Our interim analysis of Cohorts 1 and 2A found no dose-limiting toxicities (DLTs), no drug-related adverse events greater than Grade 1, and no hand-foot syndrome, a major side effect found with the catabolites of 5-FU. In this Phase 1B trial it was demonstrated that the irreversible inhibition of DPD by PCS6422 could alter the elimination of 5-FU making NGC significantly more potent (greater than 50 times more potent) and potentially lead to higher levels of the group of metabolites called anabolites which can kill replicating cancer and normal cells. By administering NGC to cancer patients, the balance between anabolites and catabolites changes depending on the dosage regimens of PCS6422 and capecitabine used, making the efficacy-safety profile of NGC different than that of FDA-approved capecitabine and requiring further evaluation of the PCS6422 and capecitabine regimens to determine the optimal NGC regimens for patients.
In order for NGC to provide a safer and more efficacious profile for cancer patients compared to existing chemotherapy, understanding how the different regimens of PCS6422 and capecitabine may affect the systemic and tumor exposure to the anabolites, as well as the systemic exposure to the catabolites, is required. This can be achieved by following the timeline of DPD irreversible inhibition and the formation of new DPD using the plasma concentrations of 5-FU and its catabolites.
In an effort to better estimate the timeline of DP inhibition and formation of new DPD, we modified the protocol for the Phase 1B trial and began enrolling patients in the amended Phase 1B trial in April 2022. On November 1, 2022, we announced that data from the Phase 1B trial identified multiple dosage regimens with potentially better safety and efficacy profiles than currently existing chemotherapy regimens. Since 5-FU exposure is dependent on both the PCS6422 regimen and the capecitabine regimen, safe regimens were identified as well as regimens that cause DLTs. One regimen in the Phase 1B trial did cause DLTs in 2 patients, one of whom died. The Phase 1B study is continuing to enroll patients and is expected to complete enrollment in the first half of 2023. The next study will be a Phase 2B trial to determine which regimens provide an improved efficacy-safety profile over present therapy using the principles of the FDA’s Oncology Project Optimus initiative to help guide the design of the trial. This FDA initiative requires us to consider NGC regimens that are not at the maximum tolerated dose or exposure level. In 2023, we plan to meet with FDA to discuss the design of our Phase 2B trial and initiate the trial. | |
| ● |
PCS12852 is a highly specific and potent 5HT4 agonist which has already been evaluated in clinical studies in South Korea for gastric emptying and gastrointestinal motility in healthy volunteers and volunteers with a history of constipation. In October 2021, the FDA cleared our IND application to proceed with a Phase 2A trial for the treatment of gastroparesis. We enrolled our first patient on April 5, 2022, and completed enrollment of the trial on September 2, 2022. Early results from this Phase 2A study which included 25 patients with moderate to severe gastroparesis, demonstrated improvements in gastric emptying in patients receiving 0.5 mg of PCS12852 as compared to placebo, and statistical significance was shown at p < 0.10 even though there were only a total of 14 patients in the 0.5 mg and placebo groups of this study. The mean (±SD) t50 change from baseline was decreased for 0.5 mg of PCS12852 compared with placebo by -31.90 ± 50.53 min vs -9.36 ±42.43 min, respectively. Differences were not observed between the placebo and the 0.1 mg dose. Adverse events associated with the administration of PCS12852 were generally mild to moderate as expected, occurred within the first few days after administration, and quickly resolved without any sequelae. There were no clinically significant cardiovascular safety events or serious adverse events (SAEs) reported during the study. Additional data from the study will be available at the end of 2022 and we plan to initiate a Phase 2B study in 2023. |
| 7 |
| ● | Our pipeline drug to treat unmet medical need conditions caused by multiple pathophysiological changes in a patient is PCS499. PCS499 is an oral tablet of the deuterated analog of one of the major metabolites of pentoxifylline (PTX or Trental®). We completed a Phase 2A trial for PCS499 in patients with ulcerative and non-ulcerative necrobiosis lipoidica (NL) in late 2020, and in May 2021 we enrolled the first patient in our Phase 2B trial for the treatment of ulcerative NL. Based on our current enrollment, we expect to complete our interim analysis and complete enrollment of the Phase 2B trial during the first half of 2023; and, depending on the results, begin a pivotal Phase 3 trial in late 2023 or 2024. We have experienced delays in the enrollment of patients for this trial due in part to COVID-19 and the ultra-rare nature of this disease. As a result, we have initiated a number of recruitment programs to increase the enrollment of patients in this study and continue to evaluate additional options to meet our overall trial enrollment goals. |
| ● | PCS3117 is a cytosine analog that is similar to gemcitabine (Gemzar®), but different enough in chemical structure that some patients are more likely to respond to PCS3117 than gemcitabine. The difference in response occurs because PCS3117 is metabolized to its active metabolite through a different enzyme system gemcitabine. We continue to evaluate the potential use of PCS3117 in patients with pancreatic cancer and to evaluate ways to identify patients who are more likely to respond to PCS3117 than gemcitabine. We anticipate initiating a Phase 2B trial or adaptive designed Phase 3 trial in 2023 subject to meeting with FDA and obtaining additional funding. | |
| ● | PCS11T is an analog of SN38 (SN38 being the active metabolite of irinotecan) and a next generation irinotecan drug for multiple types of cancers. The manufacturing process and sites for drug substance and drug product are presently being evaluated. In addition, we are defining the potential paths to approval, which include defining the targeted patient population and the type of cancer. We hope to submit an IND in 2024, followed by a Phase 1B maximum tolerated dose trial. |
Impact of COVID-19
The COVID-19 pandemic continues to create uncertainties in expected timelines by causing delays in clinical trials and disruptions in the supply chain for raw materials used in clinical trial work. We have experienced delays in the enrollment of patients in our PCS499 Phase 2B trial due to COVID-19 and the rareness of the disease. Some potential patients died from COVID-19 prior to screening and others continue to be reluctant to travel to our testing sites for fear of contracting COVID-19. Delays in enrollment lengthen the time of studies and increase their costs, which could materially impact our business in future periods. While we are hopeful the infection rate of COVID-19 will continue to decline, we cannot predict the future impact COVID-19 will have on our current and future clinical trials and continued delays could materially impact our business in future periods and further extend our timelines. For more information on the risks associated with COVID-19, refer to Part I, Item 1A, “Risk Factors” included in our Annual Report on Form 10-K for the year ended December 31, 2021.
Basis of Presentation
The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) for interim financial information and with the instructions of the Securities and Exchange Commission (“SEC”) on Form 10-Q and Article 8 of Regulation S-X.
Accordingly, they do not include all the information and disclosures required by U.S. GAAP for complete financial statements. All material intercompany accounts and transactions have been eliminated in consolidation. In the opinion of management, the accompanying unaudited condensed consolidated financial statements include all adjustments necessary, which are of a normal and recurring nature, for the fair presentation of our financial position and of the results of operations and cash flows for the periods presented. These condensed consolidated financial statements should be read in conjunction with the audited financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the SEC. The results of operations for the interim periods shown in this report are not necessarily indicative of the results that may be expected for any other interim period or for the full year.
| 8 |
Liquidity
We
have incurred losses since inception, devoting substantially all of our efforts toward moving the drugs in our pipeline through the
regulatory process and other research and development activities, and have an accumulated deficit of approximately $
We
had
On August 20, 2021, we entered into an equity distribution agreement (the “Sales Agreement”) with Oppenheimer & Co. Inc. under which we may issue and sell in a registered “at-the-market” offering shares of our common stock having an aggregate offering price of up to $ million from time to time through or to our Sales Agent (the “ATM Offering”). We also expect to use net proceeds from any future ATM Offering as a source for working capital and general corporate purposes. The shares under the ATM Offering will be sold and issued pursuant to our S-3 shelf registration statement (Registration No. 333-257588) filed on July 30, 2021. See Note 3 for additional details concerning the ATM Offering.
On
March 23, 2022, we entered into a Purchase Agreement (the “Purchase Agreement”) with Lincoln Park Capital Fund, LLC (“Lincoln
Park”) under which we have the right, but not the obligation, to sell to Lincoln Park, and Lincoln Park is obligated to purchase
up to $
Use of Estimates
In preparing our condensed consolidated financial statements and related disclosures in conformity with GAAP and pursuant to the rules and regulations of the SEC, we make estimates and judgments that affect the amounts reported in the condensed consolidated financial statements and accompanying notes. Estimates are used for, but not limited to preclinical and clinical trial expenses, stock-based compensation, intangible assets, future milestone payments and income taxes. These estimates and assumptions are continuously evaluated and are based on management’s experience and knowledge of the relevant facts and circumstances. While we believe the estimates to be reasonable, actual results could differ materially from those estimates and could impact future results of operations and cash flows.
Income Taxes
We
account for income taxes in accordance with ASC Topic 740, Income Taxes. Deferred income taxes are recorded for the expected
tax consequences of temporary differences between the basis of assets and liabilities for financial reporting purposes and amounts
recognized for income tax purposes. As of September 30, 2022 and December 31, 2021, we recorded a valuation allowance equal to the
full recorded amount of our net deferred tax assets since it is more-likely-than-not that benefits from our deferred tax assets will
not be realized. The valuation allowance is reviewed quarterly and is maintained until sufficient positive evidence exists to
support its reversal. As part of an evaluation of our tax attributes, we recharacterized approximately
$
Under ACS 740-270 Income Taxes – Interim Reporting, we are required to project our annual federal and state effective income tax rate and apply it to the year-to-date ordinary operating tax basis loss before income taxes. Based on the projection, we recognized the tax benefit from our projected ordinary tax loss, which was used to offset the deferred tax liabilities related to the intangible asset and resulted in the recognition of a deferred tax benefit shown in the condensed consolidated statements of operations for three and nine months ended September 30, 2021. No current income tax benefit or expense was recorded or is expected for 2022 or in the foreseeable future since the deferred tax liability was offset completely as of December 31, 2021 and we expect to generate future taxable net operating losses.
| 9 |
Concentration of Credit Risk
Financial instruments that potentially subject us to significant concentration of credit risk consist primarily of our cash and cash equivalents. We utilize only well-established banks and financial institutions with high credit ratings. Balances on deposit are insured by the Federal Deposit Insurance Corporation (FDIC) up to specified limits. Total cash held by our banks at September 30, 2022 exceeded FDIC limits.
Recent Accounting Pronouncements
From time to time, the Financial Accounting Standards Board (“FASB”) or other standard setting bodies issue new accounting pronouncements. Updates to the FASB Accounting Standards Codification are communicated through issuance of an Accounting Standards Update (“ASU”). We have implemented all new accounting pronouncements that are in effect and that may impact our condensed consolidated financial statements. We have evaluated recently issued accounting pronouncements and determined that there is no material impact on our condensed consolidated financial position or results of operations.
Note 2 – Intangible Assets
Intangible assets at September 30, 2022 and December 31, 2021 consisted of the following:
| September 30, 2022 | December 31, 2021 | |||||||
| Gross intangible assets | $ | $ | ||||||
| Less: accumulated amortization | ( | ) | ( | ) | ||||
| Total intangible assets, net | $ | $ | ||||||
Gross
intangible assets consist primarily of costs we capitalized when we acquired the license rights to PCS499 in 2018. Amortization expense
was $
Note 3 – Stockholders’ Equity
Preferred Stock
There were issued or outstanding shares of preferred stock at either September 30, 2022 or December 31, 2021.
Common Stock
Increase in Our Authorized Number of Shares
On January 1, 2022, we amended our Certificate of Incorporation to increase the number of authorized shares of our common stock from to .
ATM Offering
On August 20, 2021, we entered into the Sales Agreement with the Sales Agent under which we may issue and sell up to $ million of our common stock from time to time under the ATM Offering. We expect to use net proceeds from the ATM Offering over time as a source for working capital and general corporate purposes. We did not sell any shares during the nine months ended September 30, 2022.
| 10 |
Lincoln Park Capital Fund, LLC Purchase Agreement
On
March 23, 2022, we entered into the Purchase Agreement with Lincoln Park, pursuant to which Lincoln Park has committed to purchase up
to $
We have the right to present Lincoln Park with a purchase notice (a “Regular Purchase Notice”), directing
Lincoln Park to purchase up to Purchase Shares (the “Regular Purchase Amount”) provided that the closing sale price
of the common stock on the purchase date is not below a threshold price set forth in the Purchase Agreement (a “Regular Purchase”).
The Regular Purchase Amount may be increased to up to shares if the closing sale price of our common stock on the applicable purchase
date equals or exceeds certain higher threshold prices set forth in the Purchase Agreement. We and Lincoln Park may mutually agree to
increase the Regular Purchase Amount with respect to any Regular Purchase under the Purchase Agreement, provided that Lincoln Park’s
maximum committed purchase obligation under any single Regular Purchase shall not exceed $
The
aggregate number of shares that we can issue to Lincoln Park under the Purchase Agreement may not exceed shares (subject to
proportional adjustments for stock splits, reverse stock splits and similar events as described above), which is equal to
We may terminate the Purchase Agreement at any time, at our sole discretion, without any cost or penalty, by giving one business day notice to Lincoln Park to terminate the Purchase Agreement. Lincoln Park has covenanted not to cause or engage in any manner whatsoever, any direct or indirect short selling or hedging of the common stock.
There are no limitations on use of proceeds, financial or business covenants, restrictions on future financings (other than restrictions on our ability to enter into variable rate transactions described in the Purchase Agreement), rights of first refusal, participation rights, penalties or liquidated damages in the Purchase Agreement. We may deliver Purchase Notices under the Purchase Agreement, subject to market conditions, and in light of our capital needs from time to time and under the limitations contained in the Purchase Agreement. Any proceeds that we receive under the Purchase Agreement are expected to be used for working capital and general corporate purposes.
Repurchase of Shares from Aposense, Ltd.
On
March 29, 2022, we purchased shares of our common stock from Aposense Ltd. for $
| 11 |
The Processa Pharmaceuticals Inc. 2019 Omnibus Equity Incentive Plan (the “2019 Plan”) allows us to make grants of stock options, restricted and unrestricted stock and other stock-based awards to employees, including our executive officers, consultants and directors. The 2019 Plan originally provided for the aggregate issuance of shares of our common stock. On July 11, 2022, our shareholders approved an increase in the aggregate number of shares of our common stock available for issuance under our 2019 plan by shares to shares in total. As of September 30, 2022, shares were available for future grants.
Stock Compensation Expense
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Research and development | $ | $ | $ | $ | ||||||||||||
| General and administrative | ||||||||||||||||
| Total | $ | $ | $ | $ | ||||||||||||
No tax benefits were attributed to the stock-based compensation expense because a valuation allowance was maintained for all net deferred tax assets relating to this expense.
Stock Options
stock options were granted, cancelled or forfeited during the nine months ended September 30, 2022. As of September 30, 2022, we had fully vested outstanding options for the purchase of shares with a weighted average exercise price of $ and a weighted average remaining contractual life of years.
Restricted Stock Awards
| Number of shares | Weighted- average grant-date fair value per share | |||||||
| Outstanding at January 1, 2022 | $ | |||||||
| Awarded | ||||||||
| Forfeited | ( | ) | ||||||
| Issued | ( | ) | ||||||
| Outstanding at September 30, 2022 | $ | |||||||
As of September 30, 2022, unrecognized stock-based compensation expense of approximately $ for RSAs is expected to be fully recognized over years.
During
the nine months ended September 30, 2022, we granted and issued RSAs for shares of our common
stock to consultants for services to be provided in 2022. These RSAs had a cumulative fair market value of $on the dates of grant
and were expensed as stock-based compensation. We also granted RSAs for shares of our common
stock to our directors for their 2022 service which had a fair market value of $on the dates of grant.
Additionally, on March 31, 2022, we issued shares of our common
stock to our directors in satisfaction of the $
We did not cancel any RSAs during the nine months ended September 30, 2022. Unvested RSAs representing shares are expected to vest in the fourth quarter of 2022, shares are expected to vest in 2023, and the remainder are expected to vest in 2024.
| 12 |
Restricted Stock Units
| Number of shares | Weighted- average grant-date fair value per share | |||||||
| Outstanding at January 1, 2022 | $ | |||||||
| Awarded | ||||||||
| Forfeited | ( | ) | ||||||
| Issued | ( | ) | ||||||
| Outstanding at September 30, 2022 | ||||||||
| Vested and unissued | ( | ) | ||||||
| Unvested at September 30, 2022 | $ | |||||||
As of September 30, 2022, unrecognized stock-based compensation expense of approximately $ million for RSUs is expected to be fully recognized over a weighted average period of years. The unrecognized expense excludes approximately $ of expense related to certain RSUs with a performance milestone that is not currently probable of occurring.
During the nine months ended September 30, 2022, we granted RSUs related to the future issuance of shares of our common stock pursuant to agreements with our Executive team and certain other employees where a portion of their base compensation is paid in RSUs. The value of an RSU award is based on the average share price of the month services were provided. These RSUs vest quarterly, but must meet distribution requirements before any shares of common stock are issued. Effective July 1, 2022, we established a $ per share floor for the computation of the number of shares our Executive team members could earn under this program.
On April 1, 2022, we also granted RSUs for shares of our common stock, of which RSUs related to the future issuance of shares were subject to shareholder approval, which was received on July 11, 2022. These RSUs vest on January 1, 2023 and are subject to distribution requirements before any shares of common stock are issued. The RSUs that were not subject to shareholder approval had a fair value on April 1, 2022 of $million and the RSUs that were subject to shareholder approval had a fair value on July 11, 2022 of $million. The $million is being recognized ratably over the period from April 1, 2022 through December 31, 2022 and the $million is being recognized ratably over the period from July 2022 through December 31, 2022.
Holders of our vested RSUs will be issued shares of our common stock upon the satisfaction of the distribution restrictions contained in their Restricted Stock Unit Award Agreement. The distribution restrictions are typically different (longer) than the vesting schedule, imposing an additional restriction on the holder. Unlike RSAs, while employees may hold fully vested RSUs, the individual does not hold any shares or have any rights of a shareholder until the distribution restrictions are met. Upon distribution to the employee, each RSU converts into one share of our common stock. The RSUs contain dividend equivalent rights.
Warrants
stock purchase warrants were granted
and expired during the nine months ended September 30, 2022. At September 30, 2022, we had outstanding and vested stock
purchase warrants for the purchase of
Effective
May 3, 2022, we extended the expiration date of a warrant held by a consultant by one year in connection with the extension of their
service agreement. No other terms of the warrant, including the exercise price of $
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Net Loss Per Share
Basic net loss per share is computed by dividing our net loss available to common shareholders by the weighted average number of shares of common stock outstanding (which excludes unvested RSAs and includes vested RSUs) during the period. Diluted loss per share is computed by dividing our net loss available to common shareholders by the diluted weighted average number of shares of common stock (which includes the potentially dilutive effect of stock options, unvested RSAs, unvested RSUs and warrants) during the period. Since we experienced a net loss for both periods presented, basic and diluted net loss per share are the same. As such, diluted loss per share for the three and nine month periods ended September 30, 2022 and 2021 excludes the impact of potentially dilutive common shares since those shares would have an anti-dilutive effect on net loss per share.
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Basic and diluted net loss per share: | ||||||||||||||||
| Net loss available to common stockholders | $ | ( | ) | $ | ( | ) | $ | ( | ) | $ | ( | ) | ||||
| Weighted average number of common shares-basic and diluted | ||||||||||||||||
| Basic and diluted net loss per share | $ | ( | ) | $ | ( | ) | $ | ( | ) | $ | ( | ) | ||||
Our diluted net loss per share for the three and nine month periods ended September 30, 2022 and 2021 excluded and of potentially dilutive common shares, respectively, related to outstanding stock options, stock purchase warrants and unvested restricted stock since those shares would have had an anti-dilutive effect on net loss per share during the periods then ended.
Note 6 – Operating Leases
We
lease our office space under an operating lease agreement, which was renewed on October 3, 2022 and its effects were recorded as of September 30, 2022. This lease does not have
significant rent escalation, concessions, leasehold improvement incentives, or other build-out clauses. Further, the lease does not
contain contingent rent provisions. Our office space lease includes both lease (e.g., fixed payments including rent, taxes, and
insurance costs) and non-lease components (e.g., common-area or other maintenance costs), which are accounted for as a single lease
component as we have elected the practical expedient to group lease and non-lease components for all leases. We also lease office
equipment under an operating lease. Our leases do not provide an implicit rate and, as such, we have used our incremental borrowing
rate of
Lease
costs included in our condensed consolidated statements of operations totaled $
| Remaining lease term (years) for our office lease | * | |||
| Remaining lease term (years) for our equipment lease | ||||
| Weighted average remaining lease term (years) for our facility and equipment leases | ||||
| Weighted average discount rate for our facility and equipment leases | % |
| * |
Annual lease liabilities for all operating leases were as follows as of September 30, 2022, including the remaining costs associated with the office lease renewal executed on October 3, 2022:
| 2022 | $ | |||
| 2023 | ||||
| 2024 | ||||
| 2025 | ||||
| Total lease payments | ||||
| Less: Interest | ( | ) | ||
| Present value of lease liabilities | ||||
| Less: current maturities | ( | ) | ||
| Non-current lease liability | $ |
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Note 7 – Related Party Transactions
CorLyst,
LLC (“CorLyst”) reimburses us for shared costs related to payroll, health insurance and rent based on actual costs incurred,
which are recognized as a reduction of our general and administrative operating expenses in our condensed consolidated
statement of operations. We recorded $
Note 8 – Elion License Agreement
On
May 17, 2022, we amended the third Milestone Event of Section 6.4 of our License Agreement with Elion Oncology, Inc. changing the third
Milestone Event from “1st Patient in Dose Confirmation Study” to (a) determination of the maximum tolerated dose
(MTD) or (b) determination of the recommended Phase 2 Dose. Prior to this amendment, the third milestone was not considered probable
since it was unknown when, or if a dose confirmation study was going to be conducted. As a result of the modification, we consider it
probable that the recommended Phase 2 dosage regimen could be determined in connection with our current Phase 1B trial for PCS6422. We
recorded an expense and related liability of $
Note 9 – Commitments and Contingencies
Purchase Obligations
We enter into contracts in the normal course of business with contract research organizations and subcontractors to further develop our products. The contracts are cancellable, with varying provisions regarding termination. If we terminated a cancellable contract with a specific vendor, we would only be obligated for products or services that we received as of the effective date of the termination and any applicable cancellation fees. As of September 30, 2022, we are contractually obligated to pay up to approximately $4.4 million of future services under the agreements with the CROs, but our actual contractual obligations will vary depending on the progress and results of the clinical trials.
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operation
Forward Looking Statements
This Quarterly Report on Form 10-Q contains “forward-looking statements” that reflect, when made, the Company’s expectations or beliefs concerning future events that involve risks and uncertainties. Forward-looking statements frequently are identified by the words “believe,” “anticipate,” “expect,” “estimate,” “intend,” “project,” “will be,” “will continue,” “will likely result,” or other similar words and phrases. Similarly, statements herein that describe the Company’s objectives, plans or goals also are forward-looking statements. Actual results could differ materially from those projected, implied or anticipated by the Company’s forward-looking statements. Some of the factors that could cause actual results to differ include: our limited operating history, limited cash and history of losses; our ability to achieve profitability; our ability to obtain adequate financing to fund our business operations in the future; the impact of the global pandemic caused by COVID-19, including its impact on our ability to obtain financing or complete clinical trials; our ability to secure required FDA or other governmental approvals for our product candidates and the breadth of the indication sought; the impact of competitive or alternative products, technologies and pricing; whether we are successful in developing and commercializing our technology, including through licensing; the adequacy of protections afforded to us and/or our licensor by the anticipated patents that we own or license and the cost to us of maintaining, enforcing and defending those patents; our and our licensor’s ability to protect non-patented intellectual property rights; our exposure to and ability to defend third-party claims and challenges to our and our licensor’s anticipated patents and other intellectual property rights; and our ability to continue as a going concern. For a discussion of these and all other known risks and uncertainties that could cause actual results to differ from those contained in the forward-looking statements, see “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, which is available on the SEC’s website at www.sec.gov. All forward-looking statements are qualified in their entirety by this cautionary statement, and the Company undertakes no obligation to revise or update this Quarterly Report on Form 10-Q to reflect events or circumstances after the date hereof.
For purposes of this Management’s Discussion and Analysis of Financial Condition and Results of Operations, references to the “Company,” “we,” “us” or “our” refer to the operations of Processa Pharmaceuticals, Inc. and its direct and indirect subsidiaries for the periods described herein.
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Overview
Our mission is to develop drug products that improve the survival and/or quality of life for patients with high unmet medical need conditions for which few or no treatment options currently exist. We are a diversified clinical-stage development company, not a discovery company, that seeks to identify and develop drugs for patients who need better treatment options. To increase the probability of development success, our pipeline only includes drugs which have previously demonstrated some efficacy in the targeted population or a drug with very similar pharmacological properties that has been shown to be effective in the population.
Our screening criteria for identifying and selecting new candidates include:
| ● | addressing an unmet or underserved clinical need, | |
| ● | having demonstrated evidence of efficacy in humans, and | |
| ● | leveraging our regulatory science approach to improve the probability of approval. |
In many instances, these clinical candidates have significant pre-clinical and clinical data that we may leverage to high value inflection points while de-risking the programs and adding in optionality to potential future indications. Our regulatory science approach developed by our team over decades of work with regulatory authorities attempts to balance the “benefit/risk” equation to identify a regulatory path with higher clinical benefit and/or lower clinical risk with shorter timelines to deliver better treatment options to patients, physicians, and caregivers.
Our pipeline includes drugs that (i) already have clinical proof-of-concept data demonstrating the desired pharmacological activity in humans or, minimally, clinical evidence in the form of case studies or clinical experience demonstrating the drug or a similar drug pharmacologically can successfully treat patients with the targeted indication; (ii) target indications for which a single positive pivotal study demonstrating efficacy might provide enough evidence that the clinical benefits of the drug and its approval outweighs the risks associated with the drug or the present standard of care (e.g., some orphan indications, many serious life-threatening conditions, some serious quality of life conditions); and/or (iii) target indications where the prevalence of the condition and the likelihood of patients enrolling in a study meet the desired timeframe to demonstrate that the drug can, at some level, treat or potentially treat patients with the condition.
To advance our mission, we have assembled an experienced and successful development team with a track record of drug approvals and successful exits. Our team is experienced in developing drug products through all principal regulatory tiers from IND enabling studies to NDA submission. Throughout their careers, the combined scientific, development and regulatory experience of our team members have resulted in more than 30 drug approvals by the FDA, over 100 meetings with the FDA, and involvement with more than 50 drug development programs, including drug products targeted to patients who have an unmet medical need. Although we believe that the skills and experience of our team members in drug development and commercialization is an important indicator of our future success, the past successes of our team members in developing and commercializing pharmaceutical products do not guarantee that they will successfully develop and commercialize drugs in our current pipeline. In addition, the growth in revenues of companies in which our executive officers and directors served was due to many factors and does not guarantee that they will successfully operate or manage us or that we will experience similar growth in revenues, even if they continue to serve as executive officers and/or directors.
Our ability to generate meaningful revenue from any products depends on our ability to out-license the drugs before or after we obtain FDA NDA approval. Even if our products are authorized and approved by the FDA, it should be noted that the products must still meet the challenges of successful marketing, distribution, and consumer acceptance.
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Our Strategy
Our strategy is to obtain and develop drugs that will not only treat patients with unmet medical need conditions, but, with our regulatory science approach, also have the potential to be more efficiently developed with a greater probability of development success than what typically occurs in the biotech-pharma industry, as well as a better return on investment given lower development costs, more efficient development, and high commercial value. Given the prior successes of our regulatory science approach, we have selected drugs for our portfolio which may have a greater chance for approval in a population of patients who desperately need better treatment options. We have applied rigorous standards to identify drugs for our portfolio, namely:
| i. | The drug must represent a treatment option to patients with a high unmet medical need condition by improving survival and/or quality of life for these patients, | |
| ii. | The drug or its metabolite or a drug with similar pharmacological properties must have demonstrated some evidence of efficacy in the target population, and | |
| iii. | The drug presents opportunities to be developed such that within 2-4 years, critical value-added clinical milestones can be achieved while advancing the drug closer to commercialization and adding to the potential for a high return on investment. |
To add significant value to our in-licensed drugs within 2 to 4 years, the drugs must be in the clinical development stage and not in discovery stage, and during those 2 to 4 years we must be able to obtain clinical data to support the added value. The additional clinical data could range from clinical proof-of-concept data which further demonstrate that the proposed pharmacology occurs clinically in the targeted patient population to a pivotal well-designed randomized controlled trial.
Recent Developments
On March 23, 2022, we entered into a purchase agreement (the “Purchase Agreement”) with Lincoln Park Capital Fund, LLC (“Lincoln Park”), pursuant to which Lincoln Park has committed to purchase up to $15.0 million of shares (the “Purchase Shares”) of our common stock, subject to the terms and conditions in the Purchase Agreement. We issued 123,609 shares of common stock (valued at $450,000) to Lincoln Park as a commitment fee in connection with entering into the Purchase Agreement and agreed to reimburse Lincoln Park $25,000 for fees incurred in connection with the Purchase Agreement. Concurrent with entering into the Purchase Agreement, we also entered into a registration rights agreement with Lincoln Park (the “Registration Rights Agreement”), pursuant to which we agreed to take certain actions relating to the registration under the Securities Act of 1933, as amended, of the offer and sale of the shares of common stock available for issuance under the Purchase Agreement. See Note 3 to the Condensed Consolidated Financial Statements for additional details concerning the Purchase Agreement. We put this facility in place to ensure that financing will not hinder the continued development of our pipeline should we run into unexpected circumstances in the future.
Impact of COVID-19
The COVID-19 pandemic continues to create uncertainties in expected timelines by causing delays in clinical trials and disruptions in the supply chain for raw materials used in clinical trial work. We have experienced delays in the enrollment of patients in our PCS499 Phase 2B trial due to COVID-19 and the rareness of the disease. Some potential patients died from COVID-19 prior to screening and others continue to be reluctant to travel to our testing sites for fear of contracting COVID-19. Delays in enrollment lengthen the time of studies and increase their costs, which could materially impact our business in future periods. While we are hopeful the infection rate of COVID-19 will continue to decline, we cannot predict the future impact COVID-19 will have on our current and future clinical trials. Continued delays could materially impact our business in future periods and further extend our timelines.
Our Drug Pipeline
We currently have five drugs: four in various stages of clinical development (Next Generation Capecitabine previously identified as PCS6422, PCS12852, PCS499, and PCS3117) and one in nonclinical development (PCS11T). A summary of each drug is provided below:
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PCS6422 (“Next Generation Capecitabine” when administered with Capecitabine)
On August 23, 2020, we in-licensed PCS6422 from Elion Oncology, Inc. (“Elion”), pursuant to which we acquired an exclusive license to develop, manufacture and commercialize PCS6422 globally.
PCS6422 is an oral, potent, selective, and irreversible inhibitor of dihydropyrimidine dehydrogenase (DPD), the enzyme that rapidly metabolizes a common chemotherapy drug known as 5-Fluorouracil (5-FU), into inactive metabolites, such as α-fluoro-β-alanine (F-Bal). F-Bal is a metabolite that has no anti-cancer activity but causes unwanted side effects, which notably leads to dose interruptions and significantly affects a patient’s quality of life. F-Bal is thought to cause Hand–Foot Syndrome (HFS) and other side effects associated with 5-FU, and greater formation of F-Bal appears to be associated with a decrease in the antitumor activity of 5-FU. HFS can affect activities of daily living, quality of life, and may require dose interruptions/adjustments and even therapy discontinuation resulting in suboptimal tumor effects. We believe that the inhibition of DPD by PCS6422 will significantly reduce 5-FU side effects related to a decrease in F-Bal, although the timeframe and magnitude for DPD inhibition have been shown to vary, ranging from 2-14 days depending on the de novo formation of DPD within a patient and the dosage regimen of PCS6422. With the inhibition of DPD, the level of the 5-FU anti-cancer metabolites could also be higher within cancer and normal cells leading to an improved efficacy profile and/or increased side effects associated with these anabolites such as neutropenia. By combining capecitabine (an oral pro-drug form of 5-FU) with PCS6422, the change in 5-FU metabolism should result in an increase in the systemic exposure of 5-FU based on the 5-FU Area Under the Plasma Concentration Curve (AUC) per mg of capecitabine dosed. This results in needing less capecitabine to kill cancer cells and treat each patient, making the combination of PCS6422 and capecitabine (the “Next Generation Capecitabine”) more potent than current FDA approved capecitabine.
Fluoropyrimidines (e.g., 5-FU, capecitabine) remain the cornerstone of treatment for many types of cancers, either as monotherapy or in combination with other chemotherapy agents by an estimated two million patients annually. Xeloda®, the brand name of capecitabine, is an oral pro-drug of 5-FU and approved as first-line therapy for metastatic colorectal and breast cancer. However, its use is limited by adverse effects such as the development of HFS in up to 60% of patients.
Elion evaluated the potential for the combination of PCS6422 with capecitabine as a treatment of advanced gastrointestinal (GI) tumors. Nonclinical efficacy data indicated that in colorectal cancer models, pretreatment with PCS6422 enhanced the antitumor activity of capecitabine. PCS6422 dramatically increased the antitumor potency of capecitabine without increasing the toxicity. The antitumor efficacy of the combination of PCS6422 and capecitabine was tested in several xenograft animal models with human breast, pancreatic and colorectal cancer cells. These preclinical xenograft models demonstrate that PCS6422 potentiates the antitumor activity of capecitabine and significantly reduces the dose of capecitabine required to be efficacious.
Other DPD enzyme inhibitors (e.g., Gimeracil used in Teysuno® approved only outside the US) act as competitive reversible inhibitors. These agents must be present when 5-FU or capecitabine are administered to inhibit 5-FU breakdown by DPD in order to improve the efficacy and safety profiles of 5-FU. Given the reversible nature of their effect on DPD, over time 5-FU metabolism to F-Bal will return if the reversible inhibitor is not present, decreasing the amount of 5-FU in the cancer cells and decreasing the potential cytotoxicity on the cancer cells. There is also evidence that administering large amounts of DPD inhibitors directly with 5-FU may also decrease the antitumor effect of the 5-FU. Because PCS6422 is an irreversible inactivator of DPD, it is dosed the day before capecitabine administration and its effect on DPD can last longer than the reversible DPD inhibitors and beyond the time 5-FU exists in the cancer cell, even after PCS6422 has been completely eliminated out of the body. We believe this can optimize the potential cytotoxic effect of the 5-FU nucleotide metabolites and minimize the catabolism of 5-FU to F-Bal.
Prior to Elion’s involvement, two multicenter Phase 3 studies were conducted in patients with colorectal cancer with PCS6422 administered in 10-fold excess to 5-FU and administered with the 5-FU. Unfortunately, we believe the dose of PCS6422 during these trials was not optimal and that PCS6422 was not administered early enough to irreversibly affect the DPD enzyme, thus the regimen tended to produce less antitumor benefit than the control arm with the standard regimen of 5-FU/leucovorin (LV) without PCS6422. In addition, later preclinical work suggested that when PCS6422 was present in excess of 5-FU and at the same time, it diminished the antitumor activity of 5-FU, which we believe supports the rationale of dosing PCS6422 such that the exposure at the time of 5-FU or capecitabine administration is as low as possible, yet continues to inhibit DPD activity to less than 10% of normal activity.
Elion met with the FDA in 2019 and agreed upon the clinical development program required for the combination of PCS6422 and capecitabine as first-line therapy for metastatic colorectal cancer when treatment with fluoropyrimidine therapy alone is preferred. On May 17, 2020, an IND for the Phase 1B study was granted safe to proceed by the FDA. This Phase 1B study was designed to evaluate: (i) the safety and tolerability of PCS6422 and several doses of capecitabine in advanced GI tumor patients; (ii) the pharmacokinetics of PCS6422, capecitabine, 5-FU and selected metabolites; (iii) the activity of DPD over time after PCS6422 administration; and (iv) the maximum tolerated dose in up to 30 patients over multiple cycles.
On August 2, 2021, we enrolled the first patient in our Phase 1B dose-escalation maximum tolerated dose trial in patients with advanced refractory gastrointestinal (GI) tract tumors. Our interim analysis of Cohorts 1 and 2A found no dose-limiting toxicities (DLTs), no drug related adverse events greater than Grade 1, and no HFS. In the Phase 1B study, it was demonstrated that the irreversible inhibition of DPD by PCS6422 could alter the elimination of 5-FU making NGC significantly more potent (greater than 50 times more potent) and potentially lead to higher levels of the anabolites which can kill replicating cancer and normal cells. By administering NGC to cancer patients, the balance between anabolites and catabolites changes depending on the dosage regimens of PCS6422 and capecitabine used, making the efficacy-safety profile of NGC different than that of FDA-approved capecitabine and requiring further evaluation of the PCS6422 and capecitabine regimens to determine the optimal NGC regimens for patients. In order for NGC to provide a safer and more efficacious profile for cancer patients compared to existing chemotherapy, understanding how the different regimens of PCS6422 and capecitabine may affect the systemic and tumor exposure to the anabolites, as well as the systemic exposure to the catabolites, is required. This can be achieved by following the timeline of DPD irreversible inhibition and the formation of new DPD using the plasma concentrations of 5-FU and its catabolites.
In an effort to better estimate the timeline of DP inhibition and formation of new DPD, we modified the protocol for the Phase 1B trial. We began enrolling patients in the amended Phase 1B trial in April 2022. On November 1, 2022, we announced that data from the Phase 1B trial identified multiple dosage regimens with potentially better safety and efficacy profiles than currently existing chemotherapy regimens. Since 5-FU exposure is dependent on both the PCS6422 regimen and the capecitabine regimen, safe regimens were identified as well as regimens that cause DLTs. One regimen in the Phase 1B trial did cause DLTs in 2 patients, one of whom died. The Phase 1B study is continuing to enroll patients and is expected to complete enrollment in the first half of 2023. The next study will be a Phase 2B trial to determine which regimens provide an improved efficacy-safety profile over present therapy using the principles of the FDA’s Oncology Project Optimus initiative to help guide the design of the trial. This FDA initiative requires us to consider NGC regimens that are not at the maximum tolerated dose or exposure level. In 2023, we plan to meet with FDA to discuss the design of our Phase 2B trial and initiate the trial.
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PCS12852
On August 19, 2020, we in-licensed PCS12852 from Yuhan Corporation (“Yuhan”), pursuant to which we acquired an exclusive license to develop, manufacture and commercialize PCS12852 globally, excluding South Korea.
PCS12852 is a novel, potent, and highly selective 5-hydroxytryptamine 4 (5-HT4) receptor agonist. Other 5-HT receptor agonists with less 5-HT4 selectivity have been shown to successfully treat gastrointestinal (GI) motility disorders such as gastroparesis, chronic constipation, constipation-predominant irritable bowel syndrome, and functional dyspepsia. Less selective 5-HT4 agonists, such as cisapride, have been either removed from the market or not approved because of the cardiovascular side effects associated with the drugs binding to other receptors, especially receptors other than 5-HT4. PCS12852 has been shown in nonclinical studies to have a cardiovascular side effect only at concentrations greater than 1,000 times the maximum concentration seen in humans.
Two clinical studies, both of which have demonstrated the effectiveness of PCS12852 on GI motility, were previously conducted by Yuhan. In a Phase 1 trial (Protocol YH12852-101), the initial safety and tolerability of PCS12852 were evaluated after single and multiple oral doses in healthy subjects. PCS12852 was shown to increase GI motility in this study, increasing stool frequency with faster onset when compared to prucalopride, a less specific 5-HT4 agonist FDA-approved drug for the treatment of chronic idiopathic constipation. Based on an increase of ≥1 spontaneous bowel movement (SBM)/week from baseline during 7-day multiple dosing, the PCS12852 dose group had a higher percentage of patients with an increase than the prucalopride group. All doses of PCS12852 were safe and well tolerated and no SAEs occurred during the study. The most frequently reported AEs were headache, nausea and diarrhea which were temporal, manageable and reversible within 24 hours. There were no clinically significant changes in platelet aggregation and ECG parameters including a change in QTc prolongation in the study. In a Phase 1/2A clinical trial (Protocol YH12852-102), the safety, tolerability, gastric emptying rate and pharmacokinetics of multiple doses of a PCS12852 immediate release (IR) formulation and a delayed release (DR) formulation were evaluated. PCS12852 was safe and well tolerated after single and multiple administrations. The most frequent AEs for both the IR and DR formulations of PCS12852 were headache, nausea and diarrhea, but the incidences of these AEs were comparable with those of the 2mg prucalopride group. These AEs, which were transient and mostly mild in severity, are also commonly observed with other 5-HT4 agonists. Both formulations of PCS12852 also increased the gastric emptying rate and increased GI motility.
Yuhan had also conducted extensive toxicological studies for the product that demonstrated that the product is safe for use and can be moved into Phase 2 studies.
We received guidance from the FDA in the first half of 2021 and in October 2021 we received notice of safe to proceed for PCS12852 evaluation in a Phase 2A randomized, placebo-controlled study in patients with gastroparesis. We enrolled our first patient on April 5, 2022 and completed enrollment on September 2, 2022. The purpose of the Phase 2A trial was to evaluate the safety, efficacy, and pharmacokinetics of two different dosing regimens for PCS12852. Data obtained from this study will be used to better design the Phase 2B efficacy study. Since patients with gastroparesis have an abnormal pattern of upper GI motility in the absence of mechanical obstruction, the Phase 2A study was designed to evaluate the change in gastric emptying in patients with gastroparesis from two different dosing regimens of PCS12852 compared to placebo. The only FDA-approved drug to treat gastroparesis is metoclopramide, a dopamine D2 receptor antagonist that has documented serious side effects which limit dosing to no more than 12 weeks. Other 5-HT4 drugs have been used clinically but the side effects, mainly caused by binding to other receptors, has resulted in these drugs not being a viable option to treat patients with gastroparesis.
Early results from this Phase 2A study which included 25 patients with moderate to severe gastroparesis, demonstrated improvements in gastric emptying in patients receiving 0.5 mg of PCS12852 as compared to placebo, and statistical significance was shown at p < 0.10 even though there were only a total of 14 patients in the 0.5 mg and placebo groups of this study. The mean (±SD) t50 change from baseline was decreased for 0.5 mg of PCS12852 compared with placebo by -31.90 ± 50.53 min vs -9.36 ±42.43 min, respectively. Differences were not observed between the placebo and the 0.1 mg dose. Adverse events associated with the administration of PCS12852 were generally mild to moderate as expected, occurred within the first few days after administration, and quickly resolved without any sequelae. There were no clinically significant cardiovascular safety events or serious adverse events (SAEs) reported during the study. Additional data from the study will be available at the end of 2022 and we plan to initiate a Phase 2B study in 2023.
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PCS499
PCS499, an oral tablet of a deuterated analog of one of the major metabolites of pentoxifylline (PTX or Trental®), is classified by FDA as a new molecular entity. PCS499 and its metabolites act on multiple pharmacological targets that are important in a variety of conditions. We have targeted ulcerative Necrobiosis Lipoidica (uNL) as our lead indication for PCS499. NL is a chronic, disfiguring condition affecting the skin and tissue under the skin typically on the lower extremities with no currently approved FDA treatments. NL presents more commonly in women than in men and occurs more often in people with diabetes. Ulceration has been reported to occur in up to 30% of NL patients, which can lead to more severe complications, such as deep tissue infections and osteonecrosis threatening the life of the limb.
The degeneration of tissue occurring at the NL lesion site may be caused by a number of pathophysiological changes, which makes it extremely difficult to develop effective treatments for this condition. Because PCS499 and its metabolites appear to affect most of the biological pathways that contribute to the pathophysiology associated with NL, PCS499 may provide a novel treatment solution for NL.
On June 18, 2018, the FDA granted orphan-drug designation for PCS499 for the treatment of NL. On September 28, 2018, the IND for PCS499 in NL became effective, such that we initiated and completed a Phase 2A multicenter, open-label prospective trial designed to determine the safety and tolerability of PCS499 in patients with NL. The study initially had a nine-month treatment phase and a nine-month optional extension phase. In December 2019, we informed patients and sites that the study would conclude after the treatment phase and there would no longer be an extension phase. The first NL patient enrolled in this Phase 2A clinical trial was dosed on January 29, 2019, and the study completed enrollment on August 23, 2019. The last patient visit took place in February 2020.
The primary objective of the Phase 2A trial was to evaluate the safety and tolerability of PCS499 in patients with ulcerated and non-ulcerated NL and to use the safety and efficacy data to design future clinical trials. Based on toxicology studies and healthy human volunteer studies, we and the FDA agreed that a PCS499 dose of 1.8 grams/day would be the highest dose administered to NL patients in this Phase 2A trial. As anticipated, the PCS499 dose of 1.8 grams/day, 50% greater than the maximum tolerated dose of PTX, appeared to be well tolerated with no serious adverse events (SAEs) reported. All adverse events (AEs) reported in the study were mild in severity. As expected, gastrointestinal symptoms were the most frequent AEs and were reported in four patients, all of which resolved within 1-2 weeks of starting dosing.
Two of the twelve patients in the study presented with uNL and had ulcers for more than two months prior to dosing. At baseline, the reference ulcer in one of the two patients measured 3.5 cm2 and had completely closed by Month 2 of treatment. The second patient had a baseline reference ulcer of 1.2 cm2 which completely closed by Month 9 during the patient’s treatment extension period. In addition, while in the trial, both patients also developed small ulcers at other sites, possibly related to contact trauma, and these ulcers resolved within one month. The other ten patients, presenting with mild to moderate non-ulcerated NL, had more limited improvement of the NL lesions during treatment. Historically, 13 - 20% of all the patients with NL naturally progress to complete healing over many years after presenting with NL. Although the natural healing of the uNL patients has not been evaluated independently, medical experts who treat NL patients suggest that the natural progression of an open ulcerated wound to complete closure may be significantly less than 13% over 1-2 years and likely close to 0% in patients with the larger ulcers.
On March 25, 2020, we met with the FDA and discussed the clinical program, as well as the nonclinical and clinical pharmacology plans to ultimately support the submission of the PCS499 New Drug Application (NDA) in the U.S. for the treatment of ulcers in NL patients. With input from the FDA, we designed the next trial as a randomized, placebo-controlled Phase 2B study to evaluate the ability of PCS499 to completely close ulcers in patients with uNL and better understand the potential response of uNL patients on drug and on placebo. We currently have selected nine clinical trial sites in the United States and are evaluating additional sites to add to our study. We had four sites in Europe, but these sites were unable to recruit patients timely, largely due to COVID-19, so we decided to close them and concentrate our efforts on recruiting patients within the United States.
We began recruiting for the clinical trial in the first half of 2021. On May 19, 2021, we dosed our first patient in the randomized, placebo-controlled trial and are planning to complete an interim analysis of the data from this trial during the first half of 2023. We have experienced delays in the enrollment of patients in the PCS499 Phase 2B trial due to COVID-19. Many potential patients also have co-morbidities, such as diabetes, and have been unwilling to travel to the testing sites for fear of contracting COVID-19. Potential patients in this trial have died from COVID-19 prior to screening. We have initiated a number of programs to hopefully increase the enrollment rate in this study, including paying for travel to study sites and informing physicians who might be treating these patients that a trial is occurring.
After obtaining the results from this Phase 2B study, we expect to have an end of Phase 2 meeting with the FDA to agree on the design of the Phase 3 study, with the intent to define a Special Protocol Assessment for the Phase 3 study and to agree on the next steps to obtain approval.
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PCS3117
On June 16, 2021, we executed a License Agreement with Ocuphire Pharma, Inc. (“Ocuphire Agreement”) under which we received a license to research, develop and commercialize PCS3117 globally, excluding Republic of Singapore, China, Hong Kong, Macau, and Taiwan.
PCS3117 is a novel, investigational, oral small molecule nucleoside compound. PCS3117 is an analog of the endogenous nucleoside, cytidine, and an analog of the cancer drug gemcitabine. Once intracellularly activated (phosphorylated) by the enzyme UCK2, it is incorporated into the DNA or RNA of cells and inhibits both DNA and RNA synthesis, which induces apoptotic cell death of tumor cells. PCS3117 has received orphan drug designation from the FDA and the European Commission for the treatment of patients with pancreatic cancer.
Gemcitabine is usually used as second line therapy for metastatic pancreatic cancer and non-small cell lung cancer, as well as used as second line therapy for other types of cancer. The difference between PCS3117 and gemcitabine is how they are activated to cancer killing nucleotides. PCS3117 also has additional pharmacological pathways which will result in cancer cell apoptosis. Since 45% - 85% of pancreatic cancer and non-small cell lung cancer patients are inherently resistant or acquire resistance to gemcitabine, the differences between PCS3117 and gemcitabine could potentially provide a therapeutic alternative to patients who do not or will not respond to gemcitabine.
Resistance to gemcitabine or PCS3117 is likely caused by:
| ● | an increase in the cytidine deaminase (CDA) enzyme which breaks down gemcitabine and PCS3117, | |
| ● | a deficiency in transportation of gemcitabine or PCS3117 across the cell membrane, | |
| ● | down regulation of the activation enzyme (dCK for gemcitabine, UCK2 for PCS3117), | |
| ● | a change in ribonucleotide reductase activity, and | |
| ● | non-genetic influences that alter gene expression. |
PCS3117 has shown broad spectrum anti-tumor activity against over 100 different human cancer cell lines and efficacy in 17 different mouse xenograft models. In preclinical trials, PCS3117 retained its anti-tumor activity in human cancer cell lines made resistant to the anti-tumor effects of gemcitabine. In August 2012, the completion of an exploratory Phase 1 clinical trial of PCS3117 in cancer patients to investigate the oral bioavailability, safety and tolerability of the compound was reported. In that study, oral administration of a 50 mg dose of PCS3117 indicated an oral bioavailability of 56% and a plasma half-life (T1/2) of 14 hours. In addition, PCS3117 appeared to be well tolerated in all subjects throughout the dose range evaluated.
Final results from a Phase 1B clinical trial of PCS3117 were presented in June 2016 showing evidence of single agent activity. Patients in the study had generally received four or more cancer therapies prior to enrollment. In this study, 12 patients experienced stable disease persisting for up to 276 days and three patients showed evidence of tumor burden reduction. A maximum tolerated dose of 700 mg was identified in the study. At the doses evaluated, PCS3117 appeared to be well tolerated with a predictable pharmacokinetic profile following oral administration.
In March 2016, a multi-center Phase 2A clinical trial of PCS3117 in patients with relapsed or refractory pancreatic cancer was initiated to further evaluate safety and efficacy. The study was designed as a two-stage study with 10 patients in stage 1 and an additional 40 patients in stage 2. According to pre-set criteria, if greater than 20% of the patients had an increase in progression free survival of more than four months, or an objective clinical response rate and reduction in tumor size, additional pancreatic cancer patients would be enrolled into stage 2. Secondary endpoints included time to disease progression, overall response rate and duration of response, as well as pharmacokinetic assessments and safety parameters. In January 2018, the final data from this trial showed evidence of tumor shrinkage in some patients with metastatic pancreatic cancer that was resistant to gemcitabine and who had failed on multiple prior treatments was presented. In this study, 31% of patients experienced progression free survival for two months or more and five patients, or 12%, had disease stabilization for greater than four months. Although the pre-set criteria of 20% of the patients having an increase in progression free survival for four months was not met, some of the gemcitabine refractory patients did respond to PCS3117. However, an evaluation of why patients were resistant to PCS3117 was not undertaken within the study.
In November 2017, a Phase 2A trial of PCS3117 in combination with ABRAXANE in patients newly diagnosed with metastatic pancreatic cancer was initiated. The multicenter, single-arm, open-label study was designed to evaluate PCS3117 in combination with ABRAXANE in first line metastatic pancreatic cancer patients. In February 2019, the target enrollment of 40 evaluable patients in this trial was reached. An overall response rate of 23% had been observed in 40 patients that had at least one scan on treatment. Preliminary and unaudited data indicated that the median progression free survival for patients in the study was approximately 5.6 months. The most commonly reported related adverse events were nausea, diarrhea, fatigue, alopecia, decreased appetite, rash, vomiting, and anemia. Again, an evaluation of the cause of treatment resistance to PCS3117 was not undertaken.
We are currently defining the potential paths to approval, which include an evaluation of which population of pancreatic cancer patients to target, FDA acceptable primary endpoints for the population, and other ways (e.g., biomarkers) to identify patients likely to respond to PCS3117 over gemcitabine.
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PCS11T
On May 24, 2020, we in-licensed PCS11T from Aposense, Ltd. (“Aposense”), pursuant to which we were granted Aposense’s patent rights and Know-How to develop and commercialize their next generation irinotecan cancer drug, PCS11T.
PCS11T is a novel lipophilic anti-cancer pro-drug that is being developed for the treatment of the same solid tumors as prescribed for irinotecan. This pro-drug is a conjugate of a specific proprietary Aposense molecule connected to SN-38, the active metabolite of irinotecan. The proprietary molecule in PCS11T has been designed to allow PCS11T to bind to cell membranes to form an inactive pro-drug depot on the cell with SN-38 preferentially accumulating in the membrane of tumors cells and the tumor core. This unique characteristic may make the therapeutic window of PCS11T wider than other irinotecan products such that the antitumor effect of PCS11T could occur at a much lower dose with a milder adverse effect profile than irinotecan. Despite the widespread use of commercially marketed irinotecan products in the treatment of metastatic colorectal cancer and other cancers resulting in peak annual sales of approximately $1.1 billion, irinotecan has a narrow therapeutic window and includes an FDA “Black Box” warning for both neutropenia and severe diarrhea. Therefore, there is a substantial unmet need to overcome the limitations of the current commercially marketed irinotecan products, improving efficacy and reducing the severity of treatment emergent AEs. We believe the potential wider therapeutic window of PCS11T will likely lead to more patients responding with less side effects when on PCS11T compared to other irinotecan products.
Pre-clinical studies conducted to date showed that PCS11T demonstrated tumor eradication at much lower doses than irinotecan across various tumor xenograft models. PCS11T does not affect acetylcholine esterase (AChE) activity in human and rat plasma in vitro, which would suggest that PCS11T will show an improved safety profile, compared to irinotecan, which is known for its cholinergic-related side effects.
We are currently evaluating the manufacturing process and sites for drug substance and drug product. In addition, we are defining the potential paths to approval, which include defining the targeted patient population and type of cancer. We hope to submit an IND in 2024, followed by a Phase 1B trial in oncology patients with solid tumors.
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Results of Operations
Comparison of the three and nine months ended September 30, 2022 and 2021
The following table summarizes our operations during the periods indicated:
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||||||||||
| 2022 | 2021 | Change | 2022 | 2021 | Change | |||||||||||||||||||
| Operating Expenses | ||||||||||||||||||||||||
| Research and development expenses | $ | 3,136,838 | $ | 1,722,364 | $ | 1,414,474 | $ | 8,319,907 | $ | 4,806,845 | $ | 3,513,062 | ||||||||||||
| Acquisition of in-process research and development | - | 50,953 | (50,953 | ) | - | 566,583 | (566,583 | ) | ||||||||||||||||
| General and administrative expenses | 2,920,280 | 1,338,113 | 1,582,167 | 6,137,674 | 3,391,105 | 2,746,569 | ||||||||||||||||||
| Operating Loss | (6,057,118 | ) | (3,111,430 | ) | (14,457,581 | ) | (8,764,533 | ) | ||||||||||||||||
| Other Income (Expense) | ||||||||||||||||||||||||
| Forgiveness of PPP loan and related accrued interest | - | - | - | - | 163,771 | (163,771 | ) | |||||||||||||||||
| Interest income, net | 36,708 | 1,771 | 34,937 | 45,672 | 8,163 | 37,509 | ||||||||||||||||||
| Net Operating Loss Before Income Tax Benefit | (6,020,410 | ) | (3,109,659 | ) | (2,910,751 | ) | (14,411,909 | ) | (8,592,599 | ) | (5,819,310 | ) | ||||||||||||
| Income Tax Benefit | - | 122,442 | (122,442 | ) | - | 348,859 | (348,859 | ) | ||||||||||||||||
| Net Loss | $ | (6,020,410 | ) | $ | (2,987,217 | ) | $ | (14,411,909 | ) | $ | (8,243,740 | ) | ||||||||||||
Revenues.
We do not currently have any revenue under contract or any immediate sales prospects.
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Research and Development Expenses.
Our research and development costs are expensed as incurred. Research and development expenses include: (i) amortization of the exclusive PCS499 license intangible asset used in research and development activities; (ii) internal research and development staff related salaries and other payroll costs including stock-based compensation, payroll taxes and employee benefits; and (iii) program and testing related expenses, including external consulting and professional fees related to the product testing and our development activities. Non-refundable advance payments for goods and services to be used in future research and development activities are recorded as prepaid expenses and expensed when the research and development activities are performed.
Our research and development expenses increased during both the three and nine month periods ended September 30, 2022 when compared to the same periods in 2021 as shown below.
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Amortization of intangible assets | $ | 197,124 | $ | 195,700 | $ | 591,372 | $ | 593,365 | ||||||||
| Research and development salaries and benefits | 1,502,065 | 546,415 | 3,068,241 | 1,246,887 | ||||||||||||
| Preclinical, clinical trial and other costs | 1,437,649 | 980,249 | 4,660,294 | 2,966,593 | ||||||||||||
| Total | $ | 3,136,838 | $ | 1,722,364 | $ | 8,319,907 | $ | 4,806,845 | ||||||||
During the three and nine months ended September 30, 2022, when compared to the same periods in 2021, we experienced increases in payroll and related costs of approximately $162,000 and $393,000, respectively, and increases in stock-based compensation of approximately $793,000 and $1.4 million, respectively, as a result of expanding our development team, and increasing salary rates and stock-based compensation.
We also experienced increases in preclinical, clinical trial and other costs from activities in our three active clinical trials. Expenses include costs we paid contract research organizations, regulatory filing and maintenance fees, drug product testing and stability, consulting, and other clinical fees. Additionally, on May 17, 2022, we amended the third Milestone Event of our License Agreement with Elion changing the third Milestone Event as described in Note 8 to our condensed consolidated financial statements. As a result, we recorded research and development expense and a related liability of $189,000, which represents the value of the shares we anticipate issuing to Elion upon satisfaction of the third Milestone Event (as modified) at the fair value on the date of modification. During the same period in 2021, we began our Phase 2B clinical trial for PCS499, our Phase 1B clinical trial for PCS6422 and were conducting pre-IND tasks for PCS12852.
The funding necessary to bring a drug candidate to market is subject to numerous uncertainties. Once a drug candidate is identified, the further development of that drug candidate may be halted or abandoned at any time due to a number of factors. These factors include, but are not limited to, funding constraints, safety or a change in market demand. For each of our drug candidate programs, we periodically assess the scientific progress and merits of the programs to determine if continued research and development is economically viable. Some programs may be terminated due to the lack of scientific progress and lack of prospects for ultimate commercialization. We anticipate our research and development costs to increase in the future as we finalize our three current clinical trials; plan/conduct future clinical trials, including the cost of having drug product manufactured; continue our evaluation of the remaining drugs in our portfolio; and expand our development team.
Our clinical trial cost accruals are based on estimates of patient enrollment and related costs at clinical investigator sites, as well as estimates for the services received and efforts expended pursuant to contracts with multiple research institutions and CROs that conduct and manage clinical trials on our behalf.
In accruing service fees, we estimate the time period over which services will be performed and the level of patient enrollment and activity expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, we will adjust the accrual accordingly. Payments made to third parties under these arrangements in advance of the receipt of the related series are recorded as prepaid expenses until the services are rendered. At September 30, 2022, we recorded $1.2 million in prepaid expenses for advanced payments made to our CROs related to our three current clinical trials.
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General and Administrative Expenses.
Our general and administrative expenses for the three months ended September 30, 2022 increased by approximately $1.6 million to $2.9 million from $1.3 million for the three months ended September 30, 2021. The majority of the increase was due to an increase in employee stock-based compensation of approximately $1.3 million. We also experienced net increases in other expenses including professional fees, travel, salaries and other payroll-related costs of approximately $255,000. Reimbursements from CorLyst totaled approximately $31,000 for rent and other costs during the three months ended September 30, 2022 and 2021.
During the nine months ended September 30, 2022, our general and administrative expenses increased by approximately $2.7 million to $6.1 million from $3.4 million for the nine months ended September 30, 2021. The majority of the increase was due to an increase in employee stock-based compensation of approximately $2.3 million. We also experienced net increases in other expenses including professional fees, travel, salaries and other payroll-related costs of approximately $439,000. Reimbursements from CorLyst totaled approximately $95,000 for rent and other costs during the nine months ended September 30, 2022, approximately $2,000 more than reimbursements for the same period in 2021.
We expect the general and administrative expenses to continue to increase as we add staff to support our growing research and development activities and the administration required to operate as a public company.
Other Income
Net other income was approximately $37,000 and $1,800 for the three months ended September 30, 2022 and 2021, respectively, and approximately $46,000 and $172,000 for the nine months ended September 30, 2022 and 2021, respectively. During the nine months ended September 30, 2021, we recognized $163,771 as other income for the principal amount and accrued interest related to the forgiveness of our Paycheck Protection Program (PPP) loan. Interest income represents interest earned on money market funds. Interest expense in 2021 was related to our PPP loan.
Income Tax Benefit.
We did not recognize any income tax benefit for the three or nine months ended September 30, 2022 compared to the $122,442 and $348,859 income tax benefit we recognized for the three and nine months ended September 30, 2021, respectively, as a result of our recording and amortizing the deferred tax liability created in connection with our acquisition of CoNCERT’s license and “Know-How” in exchange for Processa stock that had been issued in the Internal Revenue Code Section 351 transaction on March 19, 2018. The Section 351 transaction treated the acquisition of the Know-How for stock as a tax-free exchange. As a result, under ASC 740-10-25-51 Income Taxes, we recorded a deferred tax liability of $3,037,147 for the acquired temporary difference between the financial reporting basis of $11,038,929 and the tax basis of $1,782. The deferred tax liability has been reduced for the effect of the non-deductibility of the amortization of the intangible asset and has been offset by the deferred tax assets resulting from net operating tax losses and was fully offset at December 31, 2021. This offset resulted in the recognition of a deferred tax benefit shown in the consolidated statements of operations in 2021 and prior periods.
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Cash Flows
The following table sets forth our sources and uses of cash and cash equivalents for the nine months ended September 30, 2022 and 2021:
| Nine months ended | ||||||||
| September 30, | ||||||||
| 2022 | 2021 | |||||||
| Net cash (used in) provided by: | ||||||||
| Operating activities | $ | (7,107,758 | ) | $ | (5,990,321 | ) | ||
| Financing activities | (335,247 | ) | 9,667,285 | |||||
| Net (decrease) increase in cash | $ | (7,443,005 | ) | $ | 3,676,964 | |||
Net cash used in operating activities
We used net cash in our operating activities of $7,107,758 and $5,990,321 during the nine months ended September 30, 2022 and 2021, respectively. The increase in cash used in operating activities during the nine months ended September 30, 2022 compared to the same period in 2021 was primarily related to cash payments made to conduct our three clinical trials and increased salaries to our employees.
At September 30, 2022, our prepaid expense and other balance consisted primarily of $1.2 million for advanced payments we made to our CROs that have not yet been applied to our clinical trials, representing a $100,000 reduction from the prepaid amount recorded at December 31, 2021 of $1.3 million. Our prepaid expense and other at September 30, 2022 also included the unamortized non-cash commitment fee we paid to Lincoln Park of approximately $372,000 and the unamortized ATM offering related costs of $186,000. The remainder of the change in prepaid expenses and other was the result of several other operating activities, none of which, individually or in the aggregate, were considered significant by management.
As we continue our three clinical trials and evaluate the other drugs in our portfolio, we anticipate our research and development efforts and ongoing general and administrative costs will continue to generate negative cash flows from operating activities for the foreseeable future. We expect these amounts to increase in the future as the drugs in our pipeline continue to advance and if new or additional clinical trials begin.
Net cash (used in) provided by financing activities
We used net cash in financing activities during the nine months ended September 30, 2022 of $300,000 to purchase 100,000 shares of our common stock from a licensee and $35,247 to pay income taxes owed on vested stock-based compensation. During the nine months ended September 30, 2021, we closed a private offering, raising net proceeds of $9.9 million, paid approximately $28,000 for income taxes owed on vested stock-based compensation and incurred $180,285 in expenses related to our ATM Offering.
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Liquidity
At September 30, 2022, we had $9.1 million in cash and cash equivalents.
On March 23, 2022, we entered into the Purchase Agreement with Lincoln Park under which we have the right, but not the obligation, to sell to Lincoln Park, and Lincoln Park is obligated to purchase up to $15.0 million of our shares of common stock, subject to the terms and conditions in the Purchase Agreement, as more fully described in Note 3 to the Consolidated Financial Statements. We did not sell any shares to Lincoln Park under the Purchase Agreement during the nine months ended September 30, 2022. In addition, on August 20, 2021, we entered into the Sales Agreement with the Sales Agent under which we may issue and sell up to $30.0 million from time to time under the ATM Offering. We expect to use net proceeds from the ATM Offering over time as a source for working capital and general corporate purposes. During the year ended December 31, 2021, we sold 21,597 shares of our common stock under the ATM Offering at an average price of approximately $8.33 per share for aggregate gross proceeds of approximately $180,000 prior to deducting sales commissions. We did not sell any shares during the nine months ended September 30, 2022.
We have incurred losses and net cash used in our operating activities during the nine months ended September 30, 2022, which we expect to continue for the foreseeable future. We have incurred losses since our inception, devoting substantially all of our efforts toward research and development, and have an accumulated deficit of approximately $51.2 million at September 30, 2022. During the nine months ended September 30, 2022, we generated a net loss of approximately $14.4 million, of which $7.0 million are non-cash expenses. Based on our current plans, we believe our current cash balance along with amounts available from the Purchase Agreement with Lincoln Park will be adequate for at least the next twelve months. Our ability to execute our longer-term operating plans, including unplanned future clinical trials for our portfolio of drugs depend on our ability to obtain additional funding from the sale of equity and/or debt securities, a strategic transaction or other funding transactions. We plan to continue to actively pursue financing alternatives, but there can be no assurance that we will obtain the necessary funding in the future when needed.
Our estimate of future cash needs is based on assumptions that may prove to be wrong, and we could utilize our available cash sooner than we currently expect. Our ultimate success depends on the outcome of our planned clinical trials and our research and development activities, as disclosed above. We expect to incur additional losses in the future, and we will need to raise additional capital to fully implement our business plan if the costs of our clinical trials are greater than we expect or they take longer than anticipated. We also expect to incur increased general and administrative expenses in the future. In addition, there may be costs we incur as we develop these drug products that we do not currently anticipate, requiring us to need additional capital sooner than currently expected.
Our future capital requirements will depend on many factors, including:
| ● | the cost of clinical trials for PCS499, PCS6422 and PCS12852, and the cost of third-party manufacturing; | |
| ● | the delays in patient enrollment due to the COVID-19 pandemic; | |
| ● | the initiation, progress, timing, costs and results of drug manufacturing, pre-clinical studies, and clinical trials of PCS3117 and PCS11T, as well as any other future product candidates; | |
| ● | the number and characteristics of product candidates that we pursue; | |
| ● | the outcome, timing, and costs of seeking regulatory approvals; | |
| ● | the costs associated with hiring additional personnel and consultants as our pre-clinical and clinical activities increase; | |
| ● | the emergence of competing therapies and other adverse market developments; | |
| ● | the costs involved in preparing, filing, prosecuting, maintaining, expanding, defending, and enforcing patent claims, including litigation costs and the outcome of such litigation; | |
| ● | the extent to which we in-license or acquire other products and technologies; and | |
| ● | the costs of operating as a public company. |
Until such time as we can generate substantial product revenues to support our capital requirements, if ever, we expect to finance our cash needs through a combination of public or private equity offerings, debt financings, collaborations and licensing arrangements or other capital sources. We currently have an effective S-3 shelf registration statement on file with the SEC, which provides us flexibility and optionality to raise capital, including pursuant to the ATM Offering and the Purchase Agreement with Lincoln Park, but there can be no assurance that capital will continue to be available to us on acceptable terms or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders.
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Contractual Obligations and Commitments
On October 3, 2022, we renewed our office lease. See Note 6 to the Consolidated Condensed Financial Statements for more details. There have been no other significant changes to the contractual obligations reported in our Annual Report on Form 10-K for the fiscal year ended December 31, 2021.
Off Balance Sheet Arrangements
At September 30, 2022, we did not have any off-balance sheet arrangements.
Critical Accounting Policies and Use of Estimates
Our discussion and analysis of our financial condition and results of operations are based upon our Unaudited Condensed Consolidated Financial Statements, which have been prepared in accordance with U.S. GAAP. The preparation of these financial statements requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, revenues and expenses and related disclosure of contingent assets and liabilities.
We believe that the estimates, assumptions, and judgments involved in the accounting policies described in the “Management’s Discussion and Analysis of Financial Condition and Results of Operations” section of our most recent Annual Report on Form 10-K have the greatest potential impact on our financial statements, so we consider these to be our critical accounting policies. Actual results could differ from the estimates we use in applying our critical accounting policies. We are not currently aware of any reasonably likely events or circumstances that would result in materially different amounts being reported.
There have been no changes in our critical accounting policies since our most recent Annual Report on Form 10-K.
Recently Issued Accounting Pronouncements
We have evaluated recently issued accounting pronouncements and determined that there is no material impact on our financial position or results of operations.
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Item 3. Quantitative and Qualitative Disclosures About Market Risk
Item 3 is not applicable to us as a smaller reporting company and has been omitted.
Item 4. Controls and Procedures
As of September 30, 2022, management, with the participation of the Chief Executive Officer and Chief Financial Officer, conducted an evaluation of the effectiveness of the design and operation of our disclosure controls and procedures, as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act. Based on the evaluation of its disclosure controls and procedures, the Chief Executive Officer and Chief Financial Officer concluded that our disclosure controls and procedures were effective as of September 30, 2022 to provide reasonable assurance that information required to be disclosed in our reports under the Exchange Act is (i) recorded, processed, summarized and reported within the time periods specified in the SEC’s rules and forms and (ii) accumulated and communicated to our management, as appropriate, to allow timely decisions regarding required disclosure. In designing and evaluating our disclosure controls and procedures, management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the benefits of possible controls and procedures relative to their costs.
Changes in Internal Control over Financial Reporting
There were no changes in our internal control over financial reporting during the quarter ended September 30, 2022 that have materially affected or are reasonably likely to materially affect our internal control over financial reporting.
PART II. OTHER INFORMATION
Item 1. Legal Proceedings
We are currently not a party to any material legal proceedings.
Item 1A. Risk Factors
There have been no material changes to our risk factors as described in Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2021.
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds
(a) Recent Sale of Unregistered Securities
There were no sales of unregistered securities during the three months ended September 30, 2022.
(b) Use of Proceeds from Public Offering of Common Stock
None.
(c) Issuer Purchases of Equity Securities
None.
Item 3. Defaults Upon Senior Securities
None.
Item 4. Mine Safety Disclosures
Not applicable.
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Item 5. Other Information
On July 25, 2022, we changed our transfer agent from Equiniti Trust Ltd. to Continental Stock Transfer and Trust Company.
Item 6. Exhibits
| SEC Ref. No. | Title of Document | |
| 10.1* | First Amendment to Lease Agreement for Processa Pharmaceuticals, Inc. | |
| 31.1* | Rule 13a-14(a)/15d-14(a) Certification of Chief Executive Officer | |
| 31.2* | Rule 13a-14(a)/15d-14(a) Certification of Chief Financial Officer | |
| 32.1*++ | Section 1350 Certification of Principal Executive Officer and Principal Financial Officer | |
| 99.1 | XBRL Files | |
| 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 Linkbase Document | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |
* Filed herewith.
++ This certification is being furnished solely to accompany this Quarterly Report pursuant to 18 U.S.C. Section 1350 and are not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and are not to be incorporated by reference into any filing of the Company, whether made before or after the date hereof, regardless of any general incorporation language in such filing herewith.
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
| PROCESSA PHARMACEUTICALS, INC. | ||
| By: | /s/ David Young | |
| David Young | ||
| Chief Executive Officer | ||
| (Principal Executive Officer) | ||
| Dated: | November 8, 2022 | |
| By: | /s/ James Stanker | |
| James Stanker | ||
| Chief Financial Officer | ||
| (Principal Financial and Accounting Officer) | ||
| Dated: | November 8, 2022 | |
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