Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2023 | Nov. 08, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2023 | |
Document Transition Report | false | |
Entity File Number | 001-38898 | |
Entity Registrant Name | Applied Therapeutics, Inc. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 81-3405262 | |
Entity Address, Address Line One | 545 Fifth Avenue | |
Entity Address, Address Line Two | Suite 1400 | |
Entity Address, City or Town | New York | |
Entity Address, State or Province | NY | |
Entity Address, Postal Zip Code | 10017 | |
City Area Code | 212 | |
Local Phone Number | 220-9226 | |
Title of 12(b) Security | Common Stock | |
Trading Symbol | APLT | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | true | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 77,229,207 | |
Entity Central Index Key | 0001697532 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q3 | |
Amendment Flag | false |
Condensed Balance Sheets
Condensed Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 37,457 | $ 16,657 |
Investments | 13,923 | |
Prepaid expenses and other current assets | 7,031 | 6,728 |
Total current assets | 44,488 | 37,308 |
Operating lease right-of-use asset | 510 | 857 |
Security deposits and leasehold improvements | 197 | 198 |
TOTAL ASSETS | 45,195 | 38,363 |
CURRENT LIABILITIES: | ||
Current portion of operating lease liabilities | 491 | 477 |
Accounts payable | 6,005 | 4,534 |
Accrued expenses and other current liabilities | 12,245 | 14,756 |
Warrant liabilities | 36,763 | 13,657 |
Total current liabilities | 55,504 | 33,424 |
NONCURRENT LIABILITIES: | ||
Noncurrent portion of operating lease liabilities | 44 | 414 |
Clinical holdback - long-term portion | 691 | 464 |
Total noncurrent liabilities | 735 | 878 |
Total liabilities | 56,239 | 34,302 |
STOCKHOLDERS' (DEFICIT)/EQUITY: | ||
Common stock, $0.0001 par value; 200,000,000 shares authorized as of September 30, 2023 and December 31, 2022; 77,133,516 shares issued and outstanding as of September 30, 2023 and 48,063,358 shares issued and outstanding as of December 31, 2022 | 7 | 5 |
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of September 30, 2023 and December 31, 2022; 0 shares issued and outstanding as of September 30, 2023 and December 31, 2022 | ||
Additional paid-in capital | 419,856 | 352,828 |
Accumulated other comprehensive gain | 51 | |
Accumulated deficit | (430,907) | (348,823) |
Total stockholders' (deficit)/equity | (11,044) | 4,061 |
TOTAL LIABILITIES AND STOCKHOLDERS' (DEFICIT)/EQUITY | $ 45,195 | $ 38,363 |
Condensed Balance Sheets (Paren
Condensed Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2023 | Dec. 31, 2022 |
Condensed Balance Sheets | ||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common Stock, authorized (in shares) | 200,000,000 | 200,000,000 |
Common Stock, issued (in shares) | 77,133,516 | 48,063,358 |
Common stock, outstanding (in shares) | 77,133,516 | 48,063,358 |
Preferred Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred Stock, authorized (in shares) | 10,000,000 | 10,000,000 |
Preferred Stock, issued (in shares) | 0 | 0 |
Preferred Stock, outstanding (in shares) | 0 | 0 |
Condensed Statements of Operati
Condensed Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
REVENUE: | ||||
License Revenue | $ 10,660 | |||
Revenue from Contract with Customer, Product and Service | us-gaap:LicenseMember | |||
Total Revenue | $ 10,660 | |||
OPERATING EXPENSES: | ||||
Research and development | $ 10,785 | $ 13,116 | 38,602 | $ 43,542 |
General and administrative | 4,710 | 6,240 | 15,585 | 20,436 |
Total operating expenses | 15,495 | 19,356 | 54,187 | 63,978 |
LOSS FROM OPERATIONS | (15,495) | (19,356) | (43,527) | (63,978) |
OTHER INCOME (EXPENSE), NET: | ||||
Interest income | 392 | 227 | 1,020 | 414 |
Change in fair value of warrant liabilities | (27,277) | 36 | (39,611) | (4,321) |
Other income (expense): | 10 | (8) | 34 | (194) |
Total other income (expense), net | (26,875) | 255 | (38,557) | (4,101) |
Net loss | (42,370) | (19,101) | (82,084) | (68,079) |
Net loss attributable to common stockholders-basic | (42,370) | (19,101) | (82,084) | (68,079) |
Net loss attributable to common stockholders-diluted | $ (42,370) | $ (19,101) | $ (82,084) | $ (68,079) |
Net loss per share attributable to common stockholders-basic (in dollars per share) | $ (0.47) | $ (0.40) | $ (1.09) | $ (2.02) |
Net loss per share attributable to common stockholders-diluted (in dollars per share) | $ (0.47) | $ (0.40) | $ (1.09) | $ (2.02) |
Weighted-average common stock outstanding-basic (in shares) | 90,669,969 | 48,000,183 | 75,482,234 | 33,785,386 |
Weighted-average common stock outstanding-diluted (in shares) | 90,669,969 | 48,000,183 | 75,482,234 | 33,785,386 |
Condensed Statements of Compreh
Condensed Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Condensed Statements of Comprehensive Income (Loss) | ||||
Net Loss | $ (42,370) | $ (19,101) | $ (82,084) | $ (68,079) |
Other comprehensive income (loss) | ||||
Unrealized gain (loss) on marketable securities | 17 | (51) | 130 | |
Other comprehensive gain (loss), net of tax | 17 | (51) | 130 | |
Comprehensive loss, net of tax | $ (42,370) | $ (19,084) | $ (82,135) | $ (67,949) |
Condensed Statements of Stockho
Condensed Statements of Stockholders' (Deficit)/Equity - USD ($) $ in Thousands | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) | Total |
Balance at Dec. 31, 2021 | $ 3 | $ 328,958 | $ (266,315) | $ (107) | $ 62,539 |
Balance (in shares) at Dec. 31, 2021 | 26,215,514 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 21,417 | ||||
Restricted Stock Units released for common stock not yet issued (in shares) | (21,417) | ||||
Stock-based compensation expense | 2,518 | 2,518 | |||
Net loss | (23,121) | (23,121) | |||
Other comprehensive income (loss) | 27 | 27 | |||
Balance at Mar. 31, 2022 | $ 3 | 331,476 | (289,436) | (80) | 41,963 |
Balance (in shares) at Mar. 31, 2022 | 26,215,514 | ||||
Balance at Dec. 31, 2021 | $ 3 | 328,958 | (266,315) | (107) | 62,539 |
Balance (in shares) at Dec. 31, 2021 | 26,215,514 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | (68,079) | ||||
Balance at Sep. 30, 2022 | $ 5 | 343,995 | (334,394) | 23 | 9,629 |
Balance (in shares) at Sep. 30, 2022 | 48,058,956 | ||||
Balance at Mar. 31, 2022 | $ 3 | 331,476 | (289,436) | (80) | 41,963 |
Balance (in shares) at Mar. 31, 2022 | 26,215,514 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock sold for cash, net of issuance costs | $ 2 | 5,966 | 5,968 | ||
Issuance of common stock sold for cash, net of issuance costs (in shares) | 20,000,000 | ||||
Exercise of prefunded warrants for common stock | 1,417 | 1,417 | |||
Exercise of pre-funded warrants for common stock (in shares) | 1,750,000 | ||||
Exercise of prefunded warrants for common stock not yet issued (in shares) | (1,750,000) | ||||
Issuance of common stock for Restricted Stock released in prior periods under Equity Incentive Plan (in shares) | 21,417 | ||||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 16,593 | ||||
Stock-based compensation expense | 2,231 | 2,231 | |||
Net loss | (25,857) | (25,857) | |||
Other comprehensive income (loss) | 86 | 86 | |||
Balance at Jun. 30, 2022 | $ 5 | 341,090 | (315,293) | 6 | 25,808 |
Balance (in shares) at Jun. 30, 2022 | 46,253,524 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock for pre-funded warrants exercised in prior periods (in shares) | 1,750,000 | ||||
Exercise of stock options | 49 | 49 | |||
Exercise of stock options (in shares) | 47,602 | ||||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 7,830 | ||||
Stock-based compensation expense | 2,856 | 2,856 | |||
Net loss | (19,101) | (19,101) | |||
Other comprehensive income (loss) | 17 | 17 | |||
Balance at Sep. 30, 2022 | $ 5 | 343,995 | (334,394) | 23 | 9,629 |
Balance (in shares) at Sep. 30, 2022 | 48,058,956 | ||||
Balance at Dec. 31, 2022 | $ 5 | 352,828 | (348,823) | 51 | 4,061 |
Balance (in shares) at Dec. 31, 2022 | 48,063,358 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 50,203 | ||||
Stock-based compensation expense | 2,055 | 2,055 | |||
Net loss | (10,137) | (10,137) | |||
Other comprehensive income (loss) | (51) | (51) | |||
Balance at Mar. 31, 2023 | $ 5 | 354,883 | (358,960) | (4,072) | |
Balance (in shares) at Mar. 31, 2023 | 48,113,561 | ||||
Balance at Dec. 31, 2022 | $ 5 | 352,828 | (348,823) | $ 51 | $ 4,061 |
Balance (in shares) at Dec. 31, 2022 | 48,063,358 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Exercise of stock options (in shares) | 85,619 | ||||
Net loss | $ (82,084) | ||||
Balance at Sep. 30, 2023 | $ 7 | 419,856 | (430,907) | (11,044) | |
Balance (in shares) at Sep. 30, 2023 | 77,133,516 | ||||
Balance at Mar. 31, 2023 | $ 5 | 354,883 | (358,960) | (4,072) | |
Balance (in shares) at Mar. 31, 2023 | 48,113,561 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock and pre-funded warrants, net of issuance costs | $ 1 | 27,449 | 27,450 | ||
Issuance of common stock and pre-funded warrants, net of issuance costs (in shares) | 9,735,731 | ||||
Exercise of pre-funded warrants for common stock (in shares) | 4,250,000 | ||||
Exercise of stock options | 22 | 22 | |||
Exercise of stock options (in shares) | 20,174 | ||||
Stock-based compensation expense | 1,843 | 1,843 | |||
Net loss | (29,577) | (29,577) | |||
Balance at Jun. 30, 2023 | $ 6 | 384,197 | (388,537) | (4,334) | |
Balance (in shares) at Jun. 30, 2023 | 62,119,466 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock sold for cash, net of issuance costs | 7,004 | 7,004 | |||
Issuance of common stock sold for cash, net of issuance costs (in shares) | 4,284,344 | ||||
Exercise of common warrants | $ 1 | 26,754 | 26,755 | ||
Exercise of common warrants (in shares) | 10,250,000 | ||||
Exercise of stock options | 65 | 65 | |||
Exercise of stock options (in shares) | 65,445 | ||||
Exercise of stock options, not yet issued (in shares) | (3,804) | ||||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 418,065 | ||||
Stock-based compensation expense | 1,836 | 1,836 | |||
Net loss | (42,370) | (42,370) | |||
Balance at Sep. 30, 2023 | $ 7 | $ 419,856 | $ (430,907) | $ (11,044) | |
Balance (in shares) at Sep. 30, 2023 | 77,133,516 |
Condensed Statements of Stock_2
Condensed Statements of Stockholders' (Deficit)/Equity (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | ||
Sep. 30, 2023 | Jun. 30, 2023 | Jun. 30, 2022 | |
Increase (Decrease) in Stockholders' Equity | |||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||
Common Stock | |||
Increase (Decrease) in Stockholders' Equity | |||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||
Stock issuance costs | $ 400 | $ 2,500 | $ 96 |
Condensed Statements of Cash Fl
Condensed Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
OPERATING ACTIVITIES: | ||
Net loss | $ (82,084) | $ (68,079) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 5,734 | 7,605 |
Amortization of insurance premium | 1,723 | 2,806 |
Amortization of operating lease right-of-use assets | 347 | 328 |
Amortization of leasehold improvements | 1 | 1 |
Change in operating lease liability | (356) | (327) |
Change in fair value of warrant liabilities | 39,611 | 4,321 |
Changes in operating assets and liabilities: | ||
Financed insurance premium | (1,546) | (3,105) |
Prepaid expenses | (480) | (262) |
Accounts payable | 1,471 | (2,488) |
Accrued expenses and other current liabilities | (2,816) | (3,000) |
Other liabilities | 227 | |
Net cash used in operating activities | (38,168) | (62,200) |
INVESTING ACTIVITIES: | ||
Purchase of available-for-sale securities | (36,911) | |
Proceeds from sale of available-for-sale securities | 4,944 | |
Proceeds from maturities of available-for-sale securities | 8,928 | 56,986 |
Net cash provided by investing activities | 13,872 | 20,075 |
FINANCING ACTIVITIES: | ||
Proceeds from issuance of common stock and pre-funded warrants | 34,606 | 27,811 |
Proceeds from financed insurance premium | 1,546 | 3,105 |
Repayments of short-term borrowings | (1,393) | (2,340) |
Exercise of stock options for common stock under Equity Incentive Plan | 87 | 49 |
Exercise of Warrants | 10,250 | |
Net cash provided by financing activities | 45,096 | 28,625 |
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS | 20,800 | (13,500) |
Cash and cash equivalents at beginning of period | 16,657 | 53,888 |
Cash and cash equivalents at end of period | 37,457 | 40,388 |
SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION: | ||
Initial measurement of warrant liabilities | 21,835 | |
Conversion of warrant liabilities to equity for warrant exercises | 16,505 | 1,417 |
Unrealized gain (loss) on marketable securities | (51) | 130 |
Offering costs still in accrued expense | $ 152 | $ 8 |
ORGANIZATION AND SUMMARY OF SIG
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 9 Months Ended |
Sep. 30, 2023 | |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 1. ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Operations and Business Applied Therapeutics, Inc. (the “Company”) is a clinical-stage biopharmaceutical company developing a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need. In particular, the Company is currently targeting treatments for rare metabolic diseases such as Galactosemia, Sorbitol Dehydrogenase (“SORD”) deficiency, and diabetic complications including diabetic cardiomyopathy. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York. On January 26, 2022, the Company entered into an equity distribution agreement (the “Equity Distribution Agreement) with Cowen and Company, LLC (“Cowen”), as a sales agent, to sell shares of the Company’s common stock, from time to time, having an aggregate offering price of up to $100.0 million. Pursuant to the Equity Distribution Agreement shares of our common stock may be offered and sold through the sales agent in sales deemed “at-the-market” offerings under the Securities Act of 1933, as amended, or the Securities Act. Under the Equity Distribution Agreement, the sales agent will be entitled to compensation of up to 3% of the gross offering proceeds of all shares of our common stock sold through it pursuant to the Equity Distribution Agreement. In connection with the sale of shares of our common stock on our behalf, the sales agent may be deemed to be “underwriters” within the meaning of the Securities Act, and the compensation paid to the sales agent may be deemed to be underwriting commissions or discounts. On June 27, 2022, the Company completed an underwritten public offering (the “June Offering”) of 20,000,000 shares of common stock, par value $0.0001 per share, 10,000,000 pre-funded warrants to purchase shares of common stock (the “Pre-Funded Warrants”), and accompanying warrants to purchase up to 30,000,000 shares of common stock (the “Common Warrants”). The shares and accompanying Common Warrants were offered at a price to the public of $1.00 per share and warrant, and the Pre-Funded Warrants and accompanying Common Warrants were offered at a price to the public of $0.9999 , resulting in aggregate net proceeds of approximately $27.8 million, after deducting underwriting discounts and commissions and offering expenses. The Pre-Funded Warrants and the Common Warrants are immediately exercisable and will expire five years from the date of issuance. Holders may not exercise any Pre-Funded Warrants or Common Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. Holders of the Pre-Funded Warrants and/or Common Warrants (together with affiliates) who immediately prior to June 27, 2022 beneficially owned more than 9.99% of the Company’s outstanding common stock may not exercise any portion of their Pre-Funded Warrants or Common Warrants if the holder (together with affiliates) would beneficially own more than 19.99% of the Company’s outstanding common stock after exercise. The Pre-Funded Warrants and Common Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants and/or Common Warrants will be entitled to receive, upon exercise, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants and/or Common Warrants immediately prior to such transaction. The Pre-Funded Warrants and Common Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which the Company’s stockholders are entitled. The Company intends to use the net proceeds from the June Offering for general corporate purposes, which may include research and development costs, including the conduct of clinical trials and process development and manufacturing of the Company’s product candidates, expansion of the Company’s research and development capabilities, working capital and capital expenditures. On April 26, 2023, the Company completed its sale of a total of 9,735,731 shares of the Company’s common stock, par value $0.0001 (the “Shares”), at a purchase price of $0.946 per Share, and 22,000,000 pre-funded warrants to purchase common stock (the “Pre-Funded Warrants” and together with the Shares, the “Securities”), at a purchase price of $0.946 per Pre-Funded Warrant, in a private placement (the “Private Placement”) to a select group of accredited investors (the “Purchasers”), pursuant to a Securities Purchase Agreement (the “Securities Purchase Agreement”), dated as of April 23, 2023, by and between the Company and the Purchasers. The Private Placement underwriting discounts, commissions and offering expenses The Securities have the benefit of the Registration Rights Agreement (the “Registration Rights Agreement”), dated as of April 23, 2023, by and among the Company and the Purchasers, requiring the Company to prepare and file a registration statement with the SEC as soon as reasonably practicable, but in no event later than May 26, 2023 (the “Filing Deadline”), and to use commercially reasonable efforts to have the registration statement declared effective within 30 days of the Filing Deadline, subject to extension under the terms of the Registration Rights Agreement. The Company satisfied its obligations under the Registration Rights Agreement by filing a registration statement on Form S-3 on May 12, 2023. The Securities were issued to the Purchasers pursuant to an exemption from registration under Rule 506 of Regulation D, which is promulgated under the Securities Act. The Company relied on this exemption from registration based in part on representations made by the Purchasers. The sale of the Securities pursuant to the Securities Purchase Agreement has not been registered under the Securities Act or any state securities laws. The Securities may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements. On August 11, 2023, the Company entered into a sales agreement (the “Leerink ATM Agreement”) with Leerink Partners LLC, pursuant to which the Company may offer and sell, from time to time, shares of common stock having an aggregate offering price of up to $75.0 million through Leerink Partners LLC as sales agent. Under the Leerink ATM Agreement, the sales agent may sell shares of common stock by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended. The Company will pay the sales agent a commission rate of up to 3% of the gross offering proceeds of any shares sold and has agreed to provide the sales agent with indemnification and contribution against certain liabilities. The Leerink ATM Agreement contains customary representations and warranties. As of September 30, 2023, the Company has sold an aggregate of 4,284,344 shares of the company’s common stock, par value $0.0001, pursuant to the Leerink ATM Agreement with an average sale price of $1.75 per share, resulting in net proceeds of $7.0 million, after deducting underwriting discounts, commissions and offering expenses. On October 13, 2023, the Company completed the sale of an additional 5,750,000 shares of the company’s common stock, par value $0.0001, pursuant to the Leerink ATM Agreement with an average sale price of $2.28 per share, resulting in net proceeds of $12.7 million, after deducting underwriting discounts, commissions and offering expenses. The accompanying unaudited condensed financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. These condensed financial statements should be read in conjunction with the Company’s audited financial statements and the notes thereto for the year ended December 31, 2022 included in the Annual Report, filed with the SEC on March 23, 2023 (the “Annual Report”). The unaudited condensed financial statements have been prepared on the same basis as the audited financial statements. In the opinion of management, the accompanying unaudited condensed financial statements contain all adjustments which are necessary for a fair presentation of the Company’s financial position as of September 30, 2023, results of operations for the three and nine months ended September 30, 2023 and 2022 and cash flows for the nine months ended September 30, 2023 and 2022. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2023, are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2023. Certain reclassifications have been made to prior period financial statements to conform to the current period presentation. Liquidity and Going Concern The Company has incurred, and expects to continue to incur, significant operating losses for the foreseeable future as it continues to develop its drug candidates. To date, the Company has not generated any product revenue, and it does not expect to generate product revenue unless and until it successfully completes development and obtains regulatory approval for one of its product candidates. Under ASC Topic 205-40, Presentation of Financial Statements - Going Concern, management is required at each reporting period to evaluate whether there are conditions and events, considered in the aggregate, that raise substantial doubt about an entity's ability to continue as a going concern within one year after the date that the financial statements are issued. As of September 30, 2023, financing through the Company’s private placement, Leerink ATM Agreement and warrant exercises has resulted in net proceeds of $44.7 million, after deducting placement agent commissions and other offering expenses. Additionally, on October 13, 2023, the Company issued additional shares under the Leerink ATM Agreement that resulted in net proceeds of $12.7 million, after deducting placement agent commissions and other offering expenses (see Note 16). The Company continues to actively pursue several potential long-term financing options, including equity capital, debt, convertible debt, and synthetic royalty financing. Additionally, the Company is in active dialogue with several potential partners regarding business development opportunities related to one or more of its programs. There can be no assurances that the Company’s discussions with any of the current counterparties will be successful, and the Company expects to continue to pursue additional opportunities. As reflected in the accompanying financial statements, the Company incurred a net loss of $82.1 million for the nine months ended September 30, 2023 and has an accumulated deficit of $430.9 million as of September 30, 2023. The exclusive licensing agreement with Advanz Pharma for commercialization rights to AT-007 in Europe provides a source of capital to the Company based on clinical and regulatory milestones. We received a $10.7 million upfront payment from Advanz Pharma in January 2023 in conjunction with signing the agreement. If actualization of these milestones aligns with the projected timelines, and product approvals are received in the timeframes expected, this source of capital may be sufficient to cover operating expenses through expected product approvals and potential revenues. However, there are no guarantees that this will materialize timely or at all, and delays or unexpected data could disrupt this potential liquidity. Broadly, the Company has not yet established an ongoing source of revenues sufficient to cover its operating costs and is dependent on debt and equity financing to fund its operations. As of September 30, 2023, our cash and cash equivalents totaled $37.5 million. Given our planned expenditures for the next twelve months, we have concluded there is substantial doubt Risks and Uncertainties The Company is subject to risks common to companies in the biotechnology industry, including, but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for any product candidate that it may identify and develop, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations and reliance on third-party manufacturers. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities and the Company's ability to continue as a going concern as of the date of the financial statements and the reported amounts of expenses during the reporting period. In preparing the financial statements, management used estimates in the following areas, among others: prepaid and accrued expenses; warrant liabilities valuation; stock-based compensation expense; and the evaluation of the existence of conditions and events that raise substantial doubt regarding the Company’s ability to continue as a going concern. Significant Accounting Policies The significant accounting policies and estimates used in preparation of the condensed financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2022, and the notes thereto, which are included in the Annual Report. There have been no material changes to the Company’s significant accounting policies during the three and nine months ended September 30, 2023. Clinical hold-back, long-term As part of the regulatory approval process for taking its products to market, the Company enters into certain Clinical Trial Agreements (CTAs) which include, among other things, the compensation and payment schedule the participating medical institutions and physicians will receive for all costs in connection with the clinical trial (or study) under the terms of the CTA. As individual patients are enrolled in the study by the participating medical institution or physician, the Company pays certain per study fees according to the CTA for the duration of the trial. As invoices are received by the Company from the medical institution or physician, the Company retains an agreed upon percentage of total invoiced costs, generally ranging between 5% - 10%, that is withheld from payment until the end of the study. These retained amounts are recorded as clinical holdback, a liability, on the accompanying balance sheets, and all expenses incurred in connection with these CTA activities are expensed as services are provided, which are included as research and development expenses on the accompanying statements of operations. The following table shows the activity within the clinical holdback liability accounts for the nine months ended September 30, 2023: (in thousands) Balance as of December 31, 2022 $ 464 Clinical holdback retained 227 Clinical holdback paid — Balance as of September 30, 2023 $ 691 Recent Accounting Pronouncements Any recent pronouncements issued by the FASB or other authoritative standards groups with future effective dates are either not applicable or are not expected to be significant to the financial statements of the Company. |
LICENSE AGREEMENT
LICENSE AGREEMENT | 9 Months Ended |
Sep. 30, 2023 | |
LICENSE AGREEMENT | |
LICENSE AGREEMENT | 2. LICENSE AGREEMENT Columbia University In October 2016, the Company entered into a license agreement (the “2016 Columbia Agreement”) with the Trustees of Columbia University (“Columbia University”) to obtain an exclusive royalty-bearing sublicensable license in respect to certain patents. As part of the consideration for entering into the 2016 Columbia Agreement, the Company issued to Columbia University shares equal to 5% of its outstanding common stock on a fully diluted basis at the time of issue. The common stock had a fair value of $0.5 million at the time of issuance. The Company will be required to make further payments to Columbia University of up to an aggregate of $1.3 million for the achievement of specified development and regulatory milestones, and up to an aggregate of $1.0 million for the achievement of a specified level of aggregate annual net sales, in each case in connection with products covered by the 2016 Columbia Agreement. The Company will also be required to pay tiered royalties to Columbia University in the low- to mid-single digit percentages on the Company’s, its affiliates’ and its sublicensees’ net sales of licensed products, subject to specified offsets and reductions. In addition, the Company is required to make specified annual minimum royalty payments to Columbia University, which is contingent upon the approval of the licensed products, in the mid-six figures beginning on the 10th anniversary of the effective date of the 2016 Columbia Agreement. When we grant sublicenses under the 2016 Columbia Agreement we are required to pay Columbia University a portion of the net sublicensing revenue received from such third parties, at percentages between 10% and 20%, depending on the stage of development at the time such revenue is received from such third parties. The Advanz Agreement includes a sublicense under the 2016 Columbia Agreement. The 2016 Columbia Agreement will terminate upon the expiration of all the Company’s royalty payment obligations in all countries. The Company may terminate the 2016 Columbia Agreement for convenience upon 90 days’ written notice to Columbia University. At its election, Columbia University may terminate the 2016 Columbia Agreement, or convert the licenses granted to the Company into non-exclusive, non-sublicensable licenses, in the case of (a) the Company’s uncured material breach upon 30 days’ written notice (which shall be extended to 90 days if the Company is diligently attempting to cure such material breach), (b) the Company’s failure to achieve the specified development and funding milestone events, or (c) the Company’s insolvency. In January 2019, the Company entered into a second license agreement with Columbia University (the “2019 Columbia Agreement”). Pursuant to the 2019 Columbia Agreement, Columbia University granted the Company a royalty-bearing, sublicensable license that is exclusive with respect to certain patents, and non-exclusive with respect to certain know-how, in each case to develop, manufacture and commercialize PI3k inhibitor products. The license grant is worldwide. Under the 2019 Columbia Agreement, the Company is obligated to use commercially reasonable efforts to research, discover, develop and market licensed products for commercial sale in the licensed territory, and to comply with certain obligations to meet specified development and funding milestones within defined time periods. Columbia University retains the right to conduct, and grant third parties the right to conduct, non-clinical academic research using the licensed technology; provided that such research is not funded by a commercial entity or for-profit entity or results in rights granted to a commercial or for-profit entity. As consideration for entering into the 2019 Columbia Agreement, the Company made a nominal upfront payment to Columbia University. The Company will be required to make further payments to Columbia University of up to an aggregate of $1.3 million for the achievement of specified development and regulatory milestones, and up to an aggregate of $1.0 million for the achievement of a specified level of aggregate annual net sales, in each case in connection with products covered by the 2019 Columbia Agreement. The Company will also be required to pay tiered royalties to Columbia University in the low- to mid-single digit percentages on the Company’s, its affiliates’ and its sublicensees’ net sales of licensed products, subject to specified offsets and reductions. In addition, the Company is required to make specified annual minimum royalty payments to Columbia University, which is contingent upon the approval of the licensed products, in the mid-six figures beginning on the tenth anniversary of the effective date of the 2019 Columbia Agreement. In July 2022, following regulatory changes impacting development of the class of PI3k inhibitors and the Company’s decision to discontinue its early stage preclinical PI3k program, the Company and Columbia entered into an agreement terminating the 2019 Columbia Agreement (the “2022 Columbia Termination Agreement”) as of July 25, 2022. Under the terms of the 2022 Columbia Termination Agreement, the Company assigned certain regulatory documents regarding the preclinical PI3k inhibitor AT-104 to Columbia and granted Columbia a non-exclusive royalty free license (with rights to sublicense any future Columbia licensee) under certain know-how, technical information and data relating to AT-104 that was developed by the Company during the term of the 2019 Columbia Agreement . In March 2019, and in connection with the 2016 Columbia Agreement, the Company entered into a research services agreement (the “2019 Columbia Research Agreement”) with Columbia University with the purpose of analyzing structural and functional changes in brain tissue in an animal model of Galactosemia, and the effects of certain compounds whose intellectual property rights were licensed to the Company as part of the 2016 Columbia Agreement on any such structural and functional changes. The 2019 Columbia Research Agreement had a term of from its effective date and expired in accordance with its terms. On October 3, 2019, and in connection with the 2019 Columbia Agreement, the Company entered into a research services agreement (the “PI3k Columbia Research Agreement” and collectively with the 2016 Columbia Agreement, 2019 Columbia Agreement and 2019 Columbia Research Agreement, the “Columbia Agreements”) with Columbia University with the purpose of analyzing PI3k inhibitors for the treatment of lymphoid malignancies. The PI3k Columbia Research Agreement had a term of 18 months from its effective date and expired in accordance with its terms. During the three and nine months ended September 30, 2023, the Company recorded no research and development expense; and $0 and $0.1 million in general and administrative expense, respectively, related to the Columbia Agreements. During the three and nine months ended September 30, 2022, the Company recorded no research and development expense and $0.1 and $0.2 million in general and administrative expense related to the Columbia Agreements. In aggregate, the Company has incurred $2.8 million in expense from the execution of the Columbia Agreements through September 30, 2023. As of September 30, 2023, the Company had $0.1 million due to Columbia University included in accrued expenses. As of December 31, 2022, the Company had $0.1 million due to Columbia University included in accrued expenses. The Company recorded no accounts payable for the period ended September 30, 2023 and December 31, 2022 related to the Columbia Agreements. University of Miami 2020 Miami License Agreement On October 28, 2020, the Company entered into a license agreement with the University of Miami (the “2020 Miami License Agreement”) relating to certain technology that is co-owned by the University of Miami (UM), the University of Rochester (UR) and University College London (UCL). UM was granted an exclusive agency from UR and UCL to license each of their rights in the technology. Pursuant to the 2020 Miami License Agreement, UM, on behalf of itself and UR and UCL, granted the Company a royalty-bearing, sublicensable license that is exclusive with respect to certain patent applications and patents that may grant from the applications, and non-exclusive with respect to certain know-how, in each case to research, develop, make, have made, use, sell and import products for use in treating and/or detecting certain inherited neuropathies, in particular those caused by mutation in the sorbitol dehydrogenase (SORD) gene. The license grant is worldwide. Under the 2020 Miami License Agreement, the Company is obligated to use commercially reasonable efforts to develop, manufacture, market and sell licensed products in the licensed territory, and to comply with certain obligations to meet specified development milestones within defined time periods. UM retains for itself, UR, and UCL the right to use the licensed patent rights and licensed technology for their internal non-commercial educational, research and clinical patient care purposes, including in sponsored research and collaboration with commercial entities. Under the terms of the 2020 Miami License Agreement, the Company was obligated to pay UM an up-front non-refundable license fee of $1.1 million, and a second non-refundable license fee of $0.5 million due on the first anniversary of the date of the license. The Company will be required to make further payments to UM of up to an aggregate $2.2 million for the achievement of specified patenting and development milestones, and up to an aggregate of $4.1 million for achievement of late stage regulatory milestones. The Company will also be required to pay royalties ranging from 0.88% - 5% on the Company’s, the Company’s affiliates’ and the Company’s sublicensees’ net sales of licensed products. When the Company sublicenses the rights granted under the 2020 Miami License Agreement to one or more third parties, the Company will be required to pay to UM a portion of the non-royalty sublicensing revenue received from such third parties ranging from 15% – 25%. The Advanz Agreement includes a sublicense under the 2020 Miami License Agreement. The 2020 Miami License Agreement terminates upon the expiration of all issued patents and filed patent applications or 10 years after the first commercial sale of the last product or process for which a royalty is due, unless earlier terminated. In addition, the 2020 Miami License Agreement may be terminated by the Company at any time upon 60 days prior written notice to UM, and may be terminated by either the Company or UM upon material breach of an obligation if action to cure the breach is not initiated within 60 days of receipt of written notice. During the three and nine months ended September 30, 2023 the company recorded $25,000 and $0.1 million in research and development expense related to the Miami License Agreement. During the three and nine months ended September 30, 2023, there were no general and administrative expense related to 2020 Miami License Agreement. During the three and nine months ended September 30, 2022 the Company recorded $25,000 and $0.1 million in research and development expense related to the 2020 Miami License Agreement. During the three and nine months ended September 30, 2022, the company recorded $0 and $2,000 in general and administrative expense, respectively, related to the 2020 Miami License Agreement. In aggregate, the Company has incurred $2.5 million in expense from execution of the 2020 Miami License Agreement through September 30, 2023. As of September 30, 2023 and December 31, 2022, the Company had $0.3 million due to UM included in accrued expenses relating to the 2020 Miami License Agreement. 2020 Miami Option Agreement On October 28, 2020, the Company entered into an option agreement with the University of Miami (the “2020 Miami Option Agreement”) concerning certain research activities and technology relating to SORD neuropathy that may be pursued and developed by UM. Under the 2020 Miami Option Agreement, if UM conducts such research activities, then UM is obligated to grant us certain option rights to access and use the research results and to obtain licenses to any associated patent rights upon us making specified payments to UM within specified time limits. If the Company elects to obtain option rights the Company will be required to make payments to UM in the low-six figures to the low-seven figures, depending upon the rights the Company elects to obtain, and the Company will be obligated to make certain milestone payments in the high-six figures to mid-seven figures if UM conducts and completes certain research activities within specified time periods and the Company elects to receive rights to use the results of that research. 2020 Miami Sponsored Research Agreement On December 14, 2020, the Company entered into a research agreement with the University of Miami (the “2020 Miami Research Agreement”), under which the University of Miami will conduct a research study relating to SORD neuropathy and deliver a final report on the study to the Company. The term of the research agreement is from December 14, 2020 through December 30, 2021, and was extended through August 31, 2022, whereby the research study was completed. The total consideration for the 2020 Miami Research Agreement was During the three and nine months ended September 30, 2023, the Company recorded no research and development expense in relation to the 2020 Miami Research Agreement. During the three and nine months ended September 30, 2022, the Company recorded $0 and $0.1 million, in research and development expense in relation to the 2020 Miami Research Agreement. As of September 30, 2023, and December 31, 2022, the Company had $0.1 million in accrued expenses relating to the 2020 Miami Research Agreement. Bayh-Dole Act Some of the intellectual property rights the Company has licensed, including certain rights licensed in the agreements described above, may have been generated through the use of U.S. government funding. As a result, the U.S. government may have certain rights to intellectual property embodied in the Company’s current or future product candidates under the Bayh-Dole Act of 1980, or Bayh-Dole Act, including the grant to the government of a non-exclusive, worldwide, freedom to operate license under any patents, and the requirement, absent a waiver, to manufacture products substantially in the United States. To the extent any of the Company’s current or future intellectual property is generated through the use of U.S. government funding, the provisions of the Bayh-Dole Act may similarly apply. |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 9 Months Ended |
Sep. 30, 2023 | |
FAIR VALUE MEASUREMENTS | |
FAIR VALUE MEASUREMENTS | 3. FAIR VALUE MEASUREMENTS The following tables summarize, as of September 30, 2023, the Company’s financial assets and liabilities that are measured at fair value on a recurring basis, according to the fair value hierarchy described in the significant accounting policies in the Company’s audited financial statements as of and for the year ended December 31, 2022, and the notes thereto, which are included in the Annual Report. As of September 30, 2023 (in thousands) Level 1 Level 2 Level 3 Total Cash $ 8,507 $ — $ — $ 8,507 Money market funds 28,950 — — 28,950 Total cash and cash equivalents $ 37,457 $ — $ — $ 37,457 U.S. government agency debt securities — — — — Total marketable securities $ — $ — $ — $ — Total financial assets measured at fair value on a recurring basis $ 37,457 $ — $ — $ 37,457 Warrant liabilities - Common Warrants — — 36,763 36,763 Total financial liabilities measured at fair value on a recurring basis $ — $ — $ 36,763 $ 36,763 The following tables summarize, as of December 31, 2022, the Company’s financial assets and liabilities that are measured at fair value on a recurring basis. As of December 31, 2022 (in thousands) Level 1 Level 2 Level 3 Total Cash $ 1,337 $ — $ — $ 1,337 Money market funds 15,320 — — 15,320 Total cash and cash equivalents $ 16,657 $ — $ — $ 16,657 U.S. government agency debt securities — 13,923 — 13,923 Total marketable securities $ — $ 13,923 $ — $ 13,923 Total financial assets measured at fair value on a recurring basis $ 16,657 $ 13,923 $ — $ 30,580 Warrant liabilities - Common Warrants — — 13,657 13,657 Total financial liabilities measured at fair value on a recurring basis $ — $ — $ 13,657 $ 13,657 Investments in U.S. government agency debt securities Fair values of U.S. government agency debt securities On June 27, 2022 the Company issued Common Warrants exercisable for 30,000,000 shares of common stock and Pre-Funded Warrants exercisable for 10,000,000 shares of common stock in connection with the June Offering (see note 1 and note 8 for more information on the June Offering). The Common Warrants were accounted for as liabilities under ASC 815-40, Derivatives and Hedging, Contracts in Entity’s Own Equity (“ASC 815-40”), as these warrants provide for a settlement provision that does not meet the requirements of the indexation guidance under ASC 815-40. The Pre-Funded Warrants were initially recorded at fair value as a liability as the Company could be required to settle the Pre-Funded Warrants in cash under certain circumstances. In December 2022, the Company amended the Pre-Funded Warrants to remove the potential requirement that they could be settled in cash under certain circumstances. Upon the amendment to the Pre-Funded Warrants, the Pre-funded Warrants liability was reclassified to equity, using their fair value as of the amendment date. These Common Warrant liabilities were measured at fair value at inception and are then subsequently measured at fair value on a recurring basis, with changes in fair value recognized in other income (expense) within the Company’s statement of operations. The Company uses a Black-Scholes option pricing model to estimate the fair value of the Common and Pre-Funded Warrants, which utilizes certain unobservable inputs and is therefore considered a Level 3 fair value measurement. Certain inputs used in this Black-Scholes pricing model may fluctuate in future periods based upon factors that are outside of the Company’s control, including a potential change in control outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liabilities, which could also result in material non-cash gains or losses being reported in the Company’s condensed statement of operations. The Common Warrants were remeasured using a Black-Scholes option pricing model with a range of assumptions included below as of September 30, 2023 and December 31, 2022. The Pre-Funded warrants were remeasured and reclassified to equity using the Black-Scholes option pricing model with a range of assumptions included below as of December 31, 2022: September 30, December 31, 2023 2022 Expected term (in years) 2.7 3.7 Volatility 89.8 % 91.53 % Risk-free interest rate 4.92 % 4.11 % Dividend yield 0.00 % 0.00 % As of September 30, 2023, the Company utilized a probability-weighted approach that considered the probability of a change in control at the Company in the Black-Scholes option pricing model, whereby a 20% probability of change in control was used for years two and three and a 5% probability was used for years four and five in the term of the agreements. As of December 31, 2022, the Company utilized a probability-weighted approach that considered the probability of a change in control at the Company in the Black-Scholes option pricing model, whereby a 10.0% probability of change in control was used for each of the remaining years in the term of the agreements. The following table provides a roll forward of the aggregate fair values of the Company’s warrant liabilities, for which fair value is determined using Level 3 inputs (in thousands): Warrant Liabilities Balance as of January 1, 2023 $ 13,657 Warrants exercised (16,505) Change in fair value 39,611 Balance as of September 30, 2023 $ 36,763 The inputs utilized by management to value the warrant liabilities are highly subjective. The assumptions used in calculating the fair value of the warrant liabilities represent the Company’s best estimates, but these estimates involve inherent uncertainties and the application of management judgment. As a result, if factors change and the Company uses different assumptions, the fair value of the warrant liabilities may be materially different in the future. |
INVESTMENTS
INVESTMENTS | 9 Months Ended |
Sep. 30, 2023 | |
INVESTMENTS | |
INVESTMENTS | 4. INVESTMENTS Marketable Securities Marketable securities, which the Company classifies as available-for-sale securities, primarily consist of U.S. government debt obligations. Marketable securities with remaining effective maturities of twelve months or less from the balance sheet date are classified as short-term; otherwise, they are classified as long-term on the balance sheets. The following tables provide the Company’s marketable securities by security type: As of September 30, 2023 As of December 31, 2022 Gross Gross Gross Gross Unrealized Unrealized Estimated Unrealized Unrealized Estimated (in thousands) Cost Gains Losses Fair Value Cost Gains Losses Fair Value US government agency debt security $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 Total $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 As of September 30, 2023, the Company had $0 in its investment portfolio. Contractual maturities of the Company’s marketable securities are summarized as follows: As of September 30, 2023 As of December 31, 2022 Gross Gross Gross Gross Unrealized Unrealized Estimated Unrealized Unrealized Estimated (in thousands) Cost Gains Losses Fair Value Cost Gains Losses Fair Value Due in one year or less $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 Due in one through two years — — — — — — — — Total $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 During the three and nine months ended September 30, 2023, the Company had no gross unrealized gains and gross unrealized losses. During the three months ended September 30, 2023, the Company recorded no gross realized gains and no gross realized losses from the sale of marketable securities. During the nine months ended September 30, 2023, the Company recorded gross realized gains and losses from the sale of marketable securities, which were deemed immaterial. There were no unrealized losses and fair values of available-for-sale securities that have been in an unrealized loss position for a period of less than and greater than 12 months as of September 30, 2023 or December 31, 2022. |
PREPAID EXPENSES AND OTHER CURR
PREPAID EXPENSES AND OTHER CURRENT ASSETS | 9 Months Ended |
Sep. 30, 2023 | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | 5. PREPAID EXPENSES AND OTHER CURRENT ASSETS Prepaid expenses and other current assets consisted of the following: September 30, December 31, (in thousands) 2023 2022 Prepaid research and development expenses $ 4,263 $ 4,272 Insurance premium asset 955 1,131 Prepaid rent expenses 233 99 Prepaid insurance expenses 112 71 Prepaid commercial and patient advocacy 346 206 Research and development tax credit receivable 262 252 Interest receivable 23 23 Other prepaid expenses and current assets 837 674 Total prepaid expenses & other current assets $ 7,031 $ 6,728 |
ACCRUED EXPENSES AND OTHER CURR
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | 9 Months Ended |
Sep. 30, 2023 | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | 6. ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES Accrued expenses and other current liabilities consisted of the following: September 30, December 31, (in thousands) 2023 2022 Accrued pre-clinical and clinical expenses $ 8,354 $ 8,877 Short-term insurance financing note 775 622 Accrued professional fees 1,032 1,218 Accrued compensation and benefits 1,196 2,301 Accrued commercial expenses 59 896 Accrued patent expenses 425 361 Other 404 481 Total accrued expenses & other current liabilities $ 12,245 $ 14,756 |
STOCK BASED COMPENSATION
STOCK BASED COMPENSATION | 9 Months Ended |
Sep. 30, 2023 | |
STOCK BASED COMPENSATION | |
STOCK BASED COMPENSATION | 7. STOCK-BASED COMPENSATION Equity Incentive Plans In May 2019, the Company’s board of directors (the “Board”) adopted its 2019 Equity Incentive Plan (“2019 Plan”), which was subsequently approved by its stockholders and became effective on May 13, 2019. As a result, no additional awards under the Company’s 2016 Equity Incentive Plan, as amended (the “2016 Plan”) will be granted and all outstanding stock awards granted under the 2016 Plan that are repurchased, forfeited, expired, or are cancelled will become available for grant under the 2019 Plan in accordance with its terms. The 2016 Plan will continue to govern outstanding equity awards granted thereunder. The 2019 Plan provides for the issuance of incentive stock options (“ISOs”) to employees, and for the grant of nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock unit awards, performance stock awards, performance cash awards and other forms of stock awards to the Company’s employees, officers, and directors, as well as non- employees, consultants, and affiliates to the Company. Under the terms of the 2019 Plan, stock options may not be granted at an exercise price less than fair market value of the Company’s common stock on the date of the grant. The 2019 Plan is administered by the Compensation Committee of the Company’s Board. Initially, subject to adjustments as provided in the 2019 Plan, the maximum number of the Company’s common stock that may be issued under the 2019 Plan was 4,530,000 shares, which is the sum of (i) 1,618,841 new shares, plus (ii) the number of shares (not to exceed 2,911,159 shares) that remained available for the issuance of awards under the 2016 Plan, at the time the 2019 Plan became effective, and (iii) any shares subject to outstanding stock options or other stock awards granted under the 2016 Plan that are forfeited, expired, or reacquired. The 2019 Plan provides that the number of shares reserved and available for issuance under the 2019 Plan will automatically increase each January 1, beginning on January 1, 2020, by 5% of the outstanding number of shares of common stock on the immediately preceding December 31 or such lesser number of shares as determined by the Board. Subject to certain changes in capitalization of the Company, the aggregate maximum number of shares of common stock that may be issued pursuant to the exercise of ISOs shall be equal to 13,000,000 shares of common stock. Stock options awarded under the 2019 Plan expire 10 years after grant and typically vest over four years. On August 2, 2022, the Board took action in accordance with its authority under the terms of the 2019 Plan to reset the per-share exercise price of all stock options previously granted under the 2019 Plan to $1.05 per share (the "Options Repricing"), which is equal to the closing price of a share of the Company’s common stock on August 1, 2022. The Options Repricing was deemed to be a Type I modification event under ASC 718, Compensation-Stock Compensation . No other terms of the repriced stock options were modified, and the repriced stock options will continue to vest according to their original vesting schedules and will retain their original expiration dates. As a result of the Options Repricing, 1,797,517 vested and 1,380,917 unvested stock options outstanding as of August 2, 2022, with original exercise prices ranging from $1.22 to $49.60 , were repriced. The Options Repricing resulted in incremental stock-based compensation expense of $1.4 million, of which $0.9 million related to vested stock option awards and was expensed on the repricing date, and $0.5 million of which related to unvested stock option awards and is being amortized on a ratable basis over the remaining weighted-average vesting period of those awards being approximately 2.4 years. As of September 30, 2023, there were 615,885 shares of common stock available for issuance under the 2019 Plan. Stock-Based Compensation Expense Total stock-based compensation expense recorded for employees, directors and non-employees: Three Months Ended Nine Months Ended September 30, September 30, (in thousands) 2023 2022 2023 2022 Research and development $ 764 $ 1,178 $ 2,378 $ 2,822 General and administrative 1,072 1,678 3,356 4,342 Total stock-based compensation expense $ 1,836 $ 2,856 $ 5,734 $ 7,164 Stock Option Activity During the nine months ended September 30, 2023, the Company did not grant any stock options. For the three months ended September 30, 2023 and 2022, amortization of stock compensation of options amounted to $1.1 million and $2.5 million, respectively, and for the nine months ended September 30, 2023 and 2022, amortization of stock compensation of options amounted to $3.9 million and $6.2 million, respectively. The following table summarizes the information about stock options outstanding at September 30, 2023: Weighted-Average Weighted- Remaining Aggregate Options Average Contractual Intrinsic (in thousands, except for share data) Outstanding Exercise Price Term (in years) Value Outstanding at December 31, 2022 4,874,047 $ 1.97 6.9 $ — Options granted Options exercised (85,619) 1.05 Forfeited (84,639) 1.05 Expired (8,170) 1.05 Outstanding at September 30, 2023 4,695,619 $ 2.01 6.1 $ 4,837 Exercisable at September 30, 2023 4,247,076 $ 2.11 6.0 $ 4,192 Nonvested at September 30, 2023 448,543 $ 1.05 7.8 $ 645 Valuation of Stock Options Granted to Employees that Contain Service Conditions Only The fair value of each option award granted with service-based vesting is estimated on the date of the grant using the Black-Scholes option valuation model based on the weighted average assumptions noted in the table below for those options granted in the three and nine months ended September 30, 2023 and 2022. Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Expected term (in years) — — — 5.7 Volatility — % — % — % 75.83 % Risk-free interest rate — % — % — % 2.13 % Dividend yield — % — % — % — % Restricted Stock Unit Activity During the nine months ended September 30, 2023, the Company granted 3,164,362 restricted stock units (“RSUs”). For the three months ended September 30, 2023 and 2022, amortization of stock compensation of RSUs amounted to $0.7 million and $0.4 million, respectively. For the nine months ended September 30, 2023 and 2022, amortization of stock compensation of RSUs amounted to $1.8 million and $0.9 million, respectively. As of September 30, 2023 and 2022, the unamortized compensation costs associated with non-vested restricted stock awards were The following table summarizes the information about restricted stock units outstanding at September 30, 2023: Weighted-Average Grant Date (in thousands, except for share data) Shares Fair Value Outstanding at December 31, 2022 815,509 $ 5.96 Awarded 3,164,362 1.04 Released (468,268) 4.75 Forfeited (185,267) 2.81 Outstanding at September 30, 2023 3,326,336 $ 1.63 Nonvested at September 30, 2023 3,326,336 $ 1.63 Weighted Average Remaining Recognition Period (in years) 3.1 2019 Employee Stock Purchase Plan In May 2019, the Company’s Board and its stockholders approved the 2019 Employee Stock Purchase Plan (the “ESPP”), which became effective as of May 13, 2019. The ESPP is intended to qualify as an “employee stock purchase plan” within the meaning of Section 423 of the U.S. Internal Revenue Code of 1986, as amended. The number of shares of common stock initially reserved for issuance under the ESPP was 180,000 shares. The ESPP provides for an annual increase on the first day of each year beginning in 2020 and ending in 2029, in each case subject to the approval of the Board, equal to the lesser of (i) 1% of the shares of common stock outstanding on the last day of the calendar month before the date of the automatic increase and (ii) 360,000 shares; provided that prior to the date of any such increase, the Board may determine that such increase will be less than the amount set forth in clauses (i) and (ii). As of September 30, 2023, no shares of common stock had been issued under the ESPP. The first offering period has not yet been decided by the Board. |
STOCKHOLDERS' EQUITY
STOCKHOLDERS' EQUITY | 9 Months Ended |
Sep. 30, 2023 | |
STOCKHOLDERS' EQUITY | |
STOCKHOLDERS' EQUITY | 8. STOCKHOLDERS’ EQUITY As of September 30, 2023, and December 31, 2022, the authorized capital stock of the Company consisted of 200,000,000 shares of common stock, par value $0.0001 per share and 10,000,000 shares of preferred stock, par value $0.0001 per share, respectively. Common Stock Goldman Equity Distribution Agreement In June 2020, the Company entered into an equity distribution agreement (the “Goldman Equity Distribution Agreement”) with Goldman Sachs & Co. LLC (“Goldman”) to sell shares of the Company’s common stock, from time to time, having an aggregate offering price of up to $100.0 million. The issuance and sale of shares of common stock by the Company pursuant to the Goldman Equity Distribution Agreement is deemed an “at-the-market” offering under the Securities Act of 1933, as amended, or the Securities Act. Goldman is entitled to compensation for its services equal to up to of the gross offering proceeds of all shares of the Company’s common stock sold through it as a sales agent pursuant to the Goldman Equity Distribution Agreement. The Goldman Equity Distribution Agreement was terminated as of January 24, 2022. Cowen Equity Distribution Agreement On January 26, 2022, the Company entered into the Equity Distribution Agreement to sell shares of the Company’s common stock, from time to time, having an aggregate offering price of up to $100.0 million. Pursuant to the Equity Distribution Agreement shares of our common stock may be offered and sold through the sales agent in sales deemed “at-the-market” offerings under the Securities Act of 1933, as amended, or the Securities Act. Under the Equity Distribution Agreement, the sales agent will be entitled to compensation of up to 3% of the gross offering proceeds of all shares of our common stock sold through it pursuant to the Equity Distribution Agreement. In connection with the sale of shares of our common stock on our behalf, the sales agent may be deemed to be “underwriters” within the meaning of the Securities Act, and the compensation paid to the sales agent may be deemed to be underwriting commissions or discounts. The Equity Distribution Agreement was terminated as of May 12, 2023. June 2022 Offering On June 27, 2022, the Company completed the June Offering, an underwritten public offering of 20,000,000 shares of common stock, par value $0.0001 per share, 10,000,000 Pre-Funded Warrants, and accompanying Common Warrants to purchase up to 30,000,000 shares of common stock. The shares and accompanying Common Warrants were offered at a price to the public of $1.00 per share and warrant, and the Pre-Funded Warrants and accompanying Common Warrants were offered at a price to the public of $0.9999 , resulting in aggregate net proceeds of approximately $27.8 million, after deducting underwriting discounts and commissions and offering expenses. The Pre-Funded Warrants and the Common Warrants are immediately exercisable and will expire five years from the date of issuance. Holders may not exercise any Pre-Funded Warrants or Common Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. Holders of the Pre-Funded Warrants and/or Common Warrants (together with affiliates) who immediately prior to June 27, 2022 beneficially owned more than 9.99% of the Company’s outstanding common stock may not exercise any portion of their Pre-Funded Warrants or Common Warrants if the holder (together with affiliates) would beneficially own more than 19.99% of the Company’s outstanding common stock after exercise. The Pre-Funded Warrants and Common Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants and/or Common Warrants will be entitled to receive, upon exercise, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants and/or Common Warrants immediately prior to such transaction. The Pre-Funded Warrants and Common Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which the Company’s stockholders are entitled. The Company intends to use the net proceeds from the June Offering for general corporate purposes, which may include research and development costs, including the conduct of clinical trials and process development and manufacturing of the Company’s product candidates, expansion of the Company’s research and development capabilities, working capital and capital expenditures. April 2023 Offering On April 26, 2023, the Company completed its sale of a total of 9,735,731 shares of the Company’s common stock, par value $0.0001, at a purchase price of $0.946 per Share, and 22,000,000 pre-funded warrants to purchase common stock, at a purchase price of $0.946 per Pre-Funded Warrant, in a private placement to a select group of accredited investors, pursuant to the Securities Purchase Agreement, dated as of April 23, 2023, by and between the Company and the Purchasers. The Private Placement resulted in net proceeds to the Company of approximately $27.5 million, after deducting underwriting discounts, commissions and offering expenses. The Pre-Funded Warrants are immediately exercisable from the date of issuance and do not have an expiration date. They have an exercise price of $0.001. Holders may not exercise any Pre-Funded Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. The Pre-Funded Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants will be entitled to receive, upon exercise of the Pre-Funded Warrants, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants immediately prior to such transaction. The Pre-Funded Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which holders of common stock are entitled. The Company intends to use the net proceeds to fund research and development and registration of its pipeline candidates, and for working capital and general corporate purposes. ATM Offering On August 11, 2023, the Company entered into the Leerink ATM Agreement with Leerink Partners LLC, pursuant to which the Company may offer and sell, from time to time, shares of common stock having an aggregate offering price of up to $75.0 million through Leerink Partners LLC as sales agent. Under the Leerink ATM Agreement, the sales agents may sell shares of common stock by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended. The Company will pay the sales agent a commission rate of up to 3% of the gross offering proceeds of any shares sold and has agreed to provide the sales agent with indemnification and contribution against certain liabilities. The Leerink ATM Agreement contains customary representations and warranties. As of September 30, 2023, the Company has sold an aggregate of 4,284,344 shares of the company’s common stock, par value $0.0001, pursuant to the Leerink ATM Agreement with an average sale price of $1.75 per share, resulting in net proceeds of $7.0 million, after deducting underwriting discounts, commissions and offering expenses. On October 13, 2023, the Company completed the sale of an additional 5,750,000 shares of the company’s common stock, par value $0.0001, pursuant to the Leerink ATM Agreement with an average sale price of $2.28 per share, resulting in net proceeds of $12.7 million, after deducting underwriting discounts, commissions and offering expenses. The Company intends to use the net proceeds from the Leerink ATM to fund research and development and registration of its pipeline candidates, and for working capital and general corporate purposes. |
WARRANTS
WARRANTS | 9 Months Ended |
Sep. 30, 2023 | |
WARRANTS | |
WARRANTS | 9. WARRANTS Warrants Issued with Series A Preferred Stock On January 26, 2017, in connection with the sale and issuance of the Series A Preferred Stock, the Company issued equity-classified warrants to purchase 309,389 shares of common stock (the “2017 Warrants”), valued at $0.2 million, and included in the issuance costs of the Series A Preferred Stock. The warrants vested immediately and have an exercise price of $2.49 per share and expire on March 13, 2027. The fair value of warrants issued was estimated using the Black-Scholes option pricing model with the following assumptions for the 2017 Warrants. Contractual term (in years) 10.0 Volatility 74.48 % Risk-free interest rate 3.20 % Dividend yield 0.00 % Warrants Issued with the 2018 Notes On January 18, 2018, the Company entered into a placement agent agreement through which it became obligated to issue common stock warrants in connection with the issuance of convertible promissory notes, issued on February 5, 2018 (the “2018 Notes”). The obligation to issue the 2018 Notes Warrants was recorded as a liability at its fair value, (see Note 3), which was initially $0.1 million, and was included in the issuance costs of the 2018 Notes. On November 5, 2018, in connection with the extinguishment of the 2018 Notes into shares of Series B Preferred Stock, the Company issued the 2018 Notes Warrants, which were equity-classified warrants upon issuance, to purchase 76,847 shares of common stock, valued at $0.3 million. The exercise price of the 2018 Notes Warrants resets each time the Company issues common stock with an issue price less than the exercise price of the Warrants. The 2018 Notes Warrants vested immediately upon issuance and expire on November 4, 2028. The exercise price of the 2018 Notes Warrants was $3.99 and $6.59 per share as of September 30, 2023 and December 31, 2022, respectively. Warrants Issued with Series B Preferred Stock In November and December 2018, in connection with the sale and issuance of the Series B Preferred Stock, the Company was obligated to issue equity-classified warrants to purchase 72,261 shares of common stock (collectively the “2018 Warrants”), valued in the aggregate at $0.2 million, which was included in the issuance costs for the Series B Preferred Stock. The exercise price of the warrants resets each time the Company issues common stock with an issue price less than the exercise price of the warrants. The warrants vested immediately upon issuance and expire 10 years from the date of issuance. The exercise price of the warrants was $4.86 and $8.24 per share as of September 30, 2023 and December 31, 2022, respectively. The fair value of the 2018 Warrants was estimated using the Black-Scholes option pricing model with the following assumptions: Contractual term (in years) 10.0 Volatility 73.22 % Risk-free interest rate 2.70 % Dividend yield 0.00 % In February 2019, in connection with the sale and issuance of the Series B Preferred Stock, the Company was obligated to issue warrants to purchase 23,867 shares of common stock (collectively the “2019 Warrants”), valued in the aggregate at $0.1 million, which was included in the issuance costs for the Series B Preferred Stock. The exercise price of the warrants resets each time the Company issues common stock with an issue price less than the exercise price of the warrants. The warrants vested immediately upon issuance and expire 10 years from the date of issuance. The exercise price of the warrants was $4.86 and $8.24 per share as of September 30, 2023 and December 31, 2022, respectively. The fair value of the 2019 Warrants was estimated using the Black-Scholes option pricing model with the following assumptions: Contractual term (in years) 10.0 Volatility 73.22 % Risk-free interest rate 2.70 % Dividend yield 0.00 % The inputs utilized by management to value the warrants are highly subjective. The assumptions used in calculating the fair value of the warrants represent the Company’s best estimates, but these estimates involve inherent uncertainties and the application of management judgment. As a result, if factors change and the Company uses different assumptions, the fair value of the warrants may be materially different in the future. Warrants Issued with June 2022 Offering On June 27, 2022, in connection with the sale and issuance common stock as part of the June Offering, the Company issued 10,000,000 Pre-Funded Warrants at an exercise price of $0.0001 per share, and 30,000,000 accompanying Common Warrants at an exercise price of $1.00 per share. Each share of common stock and accompanying Common Warrant was sold at a public offering price of $1.00 , less underwriting discounts and commissions, and each Pre-Funded Warrant and accompanying Common Warrant was sold at a public offering price of $0.9999 , less underwriting discounts and commissions, as described in the prospectus supplement, dated June 22, 2022, filed with the Securities and Exchange Commission on June 24, 2022. The Pre-Funded Warrants and the Common Warrants are immediately exercisable and will expire five years from the date of issuance. Holders may not exercise any Pre-Funded Warrants or Common Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. Holders of the Pre-Funded Warrants and/or Common Warrants (together with affiliates) who immediately prior to June 27, 2022 beneficially owned more than 9.99% of the Company’s outstanding common stock may not exercise any portion of their Pre-Funded Warrants or Common Warrants if the holder (together with affiliates) would beneficially own more than 19.99% of the Company’s outstanding common stock after exercise. The Pre-Funded Warrants and Common Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants and/or Common Warrants will be entitled to receive, upon exercise, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants and/or Common Warrants immediately prior to such transaction. The Pre-Funded Warrants and Common Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which the Company’s stockholders are entitled. The June 2022 Pre-Funded Warrants were initially recorded at fair value as a liability as the Company could be required to settle the Pre-Funded Warrants in cash in the event of an acquisition of the Company under certain circumstances. In December 2022, the Company amended the Pre-Funded Warrants to remove the potential requirement that they could be settled in cash under certain circumstances. Beginning December 31, 2022, the Pre-funded Warrants are recorded as equity, using their fair value as of the amendment date. On June 28, 2022, a warrant holder exercised 1,750,000 Pre-Funded Warrants on a cash basis and received 1,750,000 shares of common stock. The Company received $175 in cash proceeds for the exercise of these Pre-Funded Warrants. On June 8, 2023, a warrant holder exercised 4,250,000 Pre-Funded Warrants on a cash basis and received 4,250,000 shares of common stock. The Company received $425 in cash proceeds for the exercise of these Pre-Funded Warrants. As of September 30, 2023, the Company had 4,000,000 Pre-Funded Warrants from the June 2022 offering outstanding with a weighted average exercise price of $0.0001 per share and an average contractual life of 5 years . The Common Warrants were accounted for as liabilities under ASC 815-40, as these warrants provide for a settlement provision that does not meet the requirements of the indexation guidance under ASC 815-40. These warrant liabilities are measured at fair value at inception and on a recurring basis, with changes in fair value presented within the statement of operations. On August 1, 2023, a warrant holder exercised 1,000,000 Common Warrants on a cash basis and received 1,000,000 shares of common stock. The Company received $1.0 million in cash proceeds for the exercise of these Common Warrants. On September 11, 2023, a warrant holder exercised 2,500,000 Common Warrants on a cash basis and received 2,500,000 shares of common stock. The Company received $2.5 million in cash proceeds for the exercise of these Common Warrants. On September 14, 2023, a warrant holder exercised 750,000 Common Warrants on a cash basis and received 750,000 shares of common stock. The Company received $0.8 million in cash proceeds for the exercise of these Common Warrants. On September 19, 2023, a warrant holder exercised 6,000,000 Common Warrants on a cash basis and received 6,000,000 shares of common stock. The Company received $6.0 million in cash proceeds for the exercise of these Common Warrants. As of September 30, 2023, the Company had 19,750,000 Common Warrants outstanding with a weighted average exercise price of $1.00 per share and an average contractual life of 5 years . The Common Warrants were remeasured using a Black-Scholes option pricing model with a range of assumptions included below as of September 30, 2023 and December 31, 2022. The Pre-Funded warrants were remeasured and reclassified to equity using the Black-Scholes option pricing model with a range of assumptions included below as of December 31, 2022: September 30, December 31, 2023 2022 Expected term (in years) 2.7 3.7 Volatility 89.8 % 91.53 % Risk-free interest rate 4.92 % 4.11 % Dividend yield 0.00 % 0.00 % Warrants Issued with April 2023 Offering On April 23, 2023, in connection with the sale and issuance of common stock as part of the April Offering, the Company issued 22,000,000 Pre-Funded Warrants at an exercise price of $0.001 per share. Each share of common stock was sold at a public offering price of $0.946 , less underwriting discounts and commissions, and each Pre-Funded Warrant was sold at a public offering price of $0.946 , less underwriting discounts and commissions, pursuant to a Securities Purchase Agreement, dated as of April 23, 2023, by and between the Company and the Purchasers. The Pre-Funded Warrants are immediately exercisable from the date of issuance and do not have an expiration date. They have an exercise price of $0.001. Holders may not exercise any Pre-Funded Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. The Pre-Funded Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants will be entitled to receive, upon exercise of the Pre-Funded Warrants, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants immediately prior to such transaction. The Pre-Funded Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which holders of common stock are entitled. The Company intends to use the net proceeds to fund research and development and registration of its pipeline candidates, and for working capital and general corporate purposes. The April 2023 pre-funded warrants were classified as equity. As of September 30, 2023, the Company had 22,000,000 Pre-Funded Warrants from the April 2023 offering outstanding with a weighted average exercise price of $0.001 per share. The pre-funded warrants do not have an expiration date. A summary of the Company’s outstanding pre-funded and common stock warrants as of September 30, 2023 is as follows: Equity Classified Liability Classified Total Warrants Warrants Warrants Outstanding as of January 1, 2023 8,375,618 30,000,000 38,375,618 Warrants granted and issued 22,000,000 — 22,000,000 Warrants exercised (4,250,000) (10,250,000) (14,500,000) Warrants exchanged — — — Outstanding as of September 30, 2023 26,125,618 19,750,000 45,875,618 |
LEASES
LEASES | 9 Months Ended |
Sep. 30, 2023 | |
LEASES | |
LEASES | 10. LEASES The following table summarizes the Company’s lease related costs for the three and nine months ended September 30, 2023 and 2022: Three Months Ended Nine Months Ended (in thousands) September 30, September 30, Lease Cost Statement of Operations Location 2023 2022 2023 2022 Operating Lease Cost General and administrative $ 126 $ 126 $ 378 $ 378 Total Lease Cost $ 126 $ 126 $ 378 $ 378 Average lease terms and discount rates for the Company’s operating leases were as follows: Nine Months Ended September 30, September 30, Other Information 2023 2022 Weighted-average remaining lease term Operating leases 1.1 years 2.1 years Weighted-average discount rate Operating leases 5.69% 5.69% The following table summarizes the maturities of lease liabilities as of September 30, 2023: Operating Year (in thousands) 2023 $ 126 2024 426 Thereafter — Total lease payments 552 Less: interest 17 Total lease liabilities $ 535 |
INCOME TAXES
INCOME TAXES | 9 Months Ended |
Sep. 30, 2023 | |
INCOME TAXES | |
INCOME TAXES | 11. INCOME TAXES During the nine months ended September 30, 2023 and the year ended December 31, 2022, the Company recorded a full valuation allowance on federal and state deferred tax assets since management does not forecast the Company to be in a profitable position in the near future. |
BENEFIT PLANS
BENEFIT PLANS | 9 Months Ended |
Sep. 30, 2023 | |
BENEFIT PLANS | |
BENEFIT PLANS | 12. BENEFIT PLANS The Company established a defined contribution savings plan under Section 401(k) of the Internal Revenue Code in 2018. This plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Matching contributions to the plan may be made at the discretion of the Company’s board of directors. The Company made approximately $ 0.1 million and $ 0.3 million in matching contributions to the plan during the three and nine months ended September 30, 2023, respectively. The Company made approximately $ 0.1 million and $ 0.4 million in matching contributions to the plan during the three and nine months ended September 30, 2022, respectively. |
NET LOSS PER COMMON SHARE
NET LOSS PER COMMON SHARE | 9 Months Ended |
Sep. 30, 2023 | |
NET LOSS PER COMMON SHARE | |
NET LOSS PER COMMON SHARE | 13. NET LOSS PER COMMON SHARE Basic net loss per common share is computed by dividing the net loss available to common stockholders by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per common share is computed by giving the effect of all potential shares of common stock, including stock options, preferred shares, warrants and instruments convertible into common stock, to the extent dilutive. Basic and diluted net loss per common share was the same for the three and nine months ended September 30, 2023 and 2022, as the inclusion of all potential common shares outstanding would have been anti-dilutive. The following tables set forth the computation of basic and diluted net loss per common share for the three and nine months ended September 30, 2023 and 2022: Three Months Ended September 30, (in thousands, except for share data) 2023 2022 Numerator: Net loss $ (42,370) $ (19,101) Denominator: Weighted-average common stock outstanding 90,669,969 48,000,183 Net loss per share attributable to common stockholders - basic and diluted $ (0.47) $ (0.40) Nine Months Ended September 30, (in thousands, except for share data) 2023 2022 Numerator: Net loss $ (82,084) $ (68,079) Denominator: Weighted-average common stock outstanding 75,482,234 33,785,386 Net loss per share attributable to common stockholders - basic and diluted $ (1.09) $ (2.02) The Company’s potentially dilutive securities, which include restricted stock units, stock options, and common warrants, have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted-average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the following potential common shares, presented based on amounts outstanding for the nine months ended September 30, 2023 and 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect: As of September 30, 2023 2022 Options to purchase common stock 4,695,619 4,925,678 Restricted stock units 3,326,336 533,114 Warrants to purchase common stock 19,875,618 38,375,618 |
RELATED PARTIES
RELATED PARTIES | 9 Months Ended |
Sep. 30, 2023 | |
RELATED PARTIES | |
RELATED PARTIES | 14. RELATED PARTIES In December 2018, the Company entered into an agreement (the “LaunchLabs Agreement”) with ARE-LaunchLabs NYC LLC (“Alexandria LaunchLabs”), a subsidiary of Alexandria Real Estate Equities, Inc. for use of specified premises within the Alexandria LaunchLabs space on a month-to-month basis. A member of the Company’s board of directors is the founder and executive chairman of Alexandria Real Estate Equities, Inc. During the three and nine months ended September 30, 2023, the Company made payments to Alexandria LaunchLabs of approximately $24,000 and $0.1 million, respectively. During the three and nine months ended September 30, 2022, the Company made payments of approximately $23,000 and $70,000, respectively, under the LaunchLabs Agreement, which was recognized in research and development expenses. As of September 30, 2023, there was $7,000 due to Alexandria LaunchLabs under the LaunchLabs Agreement. As of December 31, 2022, there were no amounts due to Alexandria LaunchLabs under the LaunchLabs Agreement. |
REVENUE
REVENUE | 9 Months Ended |
Sep. 30, 2023 | |
REVENUE | |
REVENUE | 15. REVENUE License Agreement with Advanz Pharma On January 3, 2023, the Company entered into an Exclusive License and Supply Agreement (the “Advanz Agreement”) with Mercury Pharma Group Limited (trading as Advanz Pharma Holdings). Pursuant to the Advanz Agreement, the Company granted Advanz Pharma the exclusive right and license to commercialize drug products containing AT-007 (also known as govorestat), our proprietary Aldose Reductase Inhibitor (ARI) (the “Licensed Product”), for use in treatment of Sorbitol Dehydrogenase Deficiency (“SORD”) and Galactosemia (each a “Licensed Indication”) in the European Economic Area, Switzerland and the United Kingdom (the “Territory”). The Company also granted Advanz Pharma a right of negotiation and “most-favored nation” rights with respect to acquiring the European commercialization rights for any additional indications for which the Licensed Product may be developed in the future (or any other products we may develop solely to the extent used for the Licensed Indications). Advanz Pharma is required to use commercially reasonable efforts to launch and commercialize the Licensed Products in the major markets in the Territory in each Licensed Indication following, and subject to, receipt of marketing authorization therein. Under the Advanz Agreement, Advanz Pharma agreed to pay the Company (i) an upfront payment of EUR 10 million (approximately USD $10.7 million), and certain development milestone payments upon clinical trial completions and receipt of marketing authorization in the territory, as well as certain commercial milestone payments, totaling EUR 134 million (approximately USD $142.2 million) in the aggregate, and (ii) royalties of 20% of net sales of the Licensed Product. Such royalty rate will be payable on a country-by-country basis until the later of (i) the expiration of the licensed patents covering the composition of matter of AT-007, or (ii) 10 years after the European Medicines Agency’s grant of marketing authorization for the Licensed Product. The royalties are subject to certain deductions, including certain secondary finishing costs, certain step-in establishment costs and a portion of fees for any potential third party patent licenses if applicable in the future. Following the initial term of the license, as described above, the royalty rate shall be reduced to 10% and shall continue in perpetuity unless the Advanz Agreement is terminated in various circumstances in accordance with its terms. In accordance with the Company's ASC 606 assessment, Advanz Pharma is considered to be a customer. The Company identified two performance obligations, the exclusive license to commercialize the Licensed Product, that was satisfied on the date of the execution of the Advanz Agreement when control of the license was transferred, and the obligation to manufacture and supply Advanz with the Product. The Company determined that the upfront license fee of EUR 10 million (approximately USD $10.7 million) is the transaction price. The performance-based milestone payments, sales-based milestone payments, sales-based royalties, and manufacture and supply of Product are each determined to be variable consideration that are constrained due to the uncertainty of achievement. The Company determined that the EUR 10 million represented the point at which the licensee was able to use and benefit from the license and recognized revenue from upfront license fees when the license was transferred to Advanz upon execution of the Advanz Agreement. The Company recognized the upfront fee as license revenue on its condensed statement of operations for the nine months ended September 30, 2023. |
SUBSEQUENT EVENTS
SUBSEQUENT EVENTS | 9 Months Ended |
Sep. 30, 2023 | |
SUBSEQUENT EVENTS | |
SUBSEQUENT EVENTS | 16. SUBSEQUENT EVENTS Warrant Exchange On October 12, 2023, the Company entered into an exchange agreement (the “Exchange Agreement”) with entities affiliated with Venrock Healthcare Capital Partners (the “Exchanging Stockholders”), pursuant to which the Company exchanged an aggregate of 5,658,034 shares of common stock, owned by the Exchanging Stockholders for pre-funded warrants to purchase an aggregate of 5,658,034 shares of common stock (subject to adjustment in the event of stock splits, recapitalizations and other similar events affecting common stock), with an exercise price of $0.001 per share. ATM Offering On October 13, 2023, the Company sold an additional 5,750,000 shares of the company’s common stock pursuant to the Leerink ATM Agreement with an average sale price of $2.28 per share, resulting in net proceeds of $12.7 million, after deducting underwriting discounts, commissions and offering expenses. |
ORGANIZATION AND SUMMARY OF S_2
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 9 Months Ended |
Sep. 30, 2023 | |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Operations and Business | Operations and Business Applied Therapeutics, Inc. (the “Company”) is a clinical-stage biopharmaceutical company developing a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need. In particular, the Company is currently targeting treatments for rare metabolic diseases such as Galactosemia, Sorbitol Dehydrogenase (“SORD”) deficiency, and diabetic complications including diabetic cardiomyopathy. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York. On January 26, 2022, the Company entered into an equity distribution agreement (the “Equity Distribution Agreement) with Cowen and Company, LLC (“Cowen”), as a sales agent, to sell shares of the Company’s common stock, from time to time, having an aggregate offering price of up to $100.0 million. Pursuant to the Equity Distribution Agreement shares of our common stock may be offered and sold through the sales agent in sales deemed “at-the-market” offerings under the Securities Act of 1933, as amended, or the Securities Act. Under the Equity Distribution Agreement, the sales agent will be entitled to compensation of up to 3% of the gross offering proceeds of all shares of our common stock sold through it pursuant to the Equity Distribution Agreement. In connection with the sale of shares of our common stock on our behalf, the sales agent may be deemed to be “underwriters” within the meaning of the Securities Act, and the compensation paid to the sales agent may be deemed to be underwriting commissions or discounts. On June 27, 2022, the Company completed an underwritten public offering (the “June Offering”) of 20,000,000 shares of common stock, par value $0.0001 per share, 10,000,000 pre-funded warrants to purchase shares of common stock (the “Pre-Funded Warrants”), and accompanying warrants to purchase up to 30,000,000 shares of common stock (the “Common Warrants”). The shares and accompanying Common Warrants were offered at a price to the public of $1.00 per share and warrant, and the Pre-Funded Warrants and accompanying Common Warrants were offered at a price to the public of $0.9999 , resulting in aggregate net proceeds of approximately $27.8 million, after deducting underwriting discounts and commissions and offering expenses. The Pre-Funded Warrants and the Common Warrants are immediately exercisable and will expire five years from the date of issuance. Holders may not exercise any Pre-Funded Warrants or Common Warrants that would cause the aggregate number of shares of common stock beneficially owned by the holder to exceed 9.99% of the Company’s outstanding common stock immediately after exercise. Holders of the Pre-Funded Warrants and/or Common Warrants (together with affiliates) who immediately prior to June 27, 2022 beneficially owned more than 9.99% of the Company’s outstanding common stock may not exercise any portion of their Pre-Funded Warrants or Common Warrants if the holder (together with affiliates) would beneficially own more than 19.99% of the Company’s outstanding common stock after exercise. The Pre-Funded Warrants and Common Warrants are subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the common stock and also upon any distributions for no consideration of assets to the Company’s stockholders. In the event of certain corporate transactions, the holders of the Pre-Funded Warrants and/or Common Warrants will be entitled to receive, upon exercise, the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants and/or Common Warrants immediately prior to such transaction. The Pre-Funded Warrants and Common Warrants do not entitle the holders thereof to any voting rights or any of the other rights or privileges to which the Company’s stockholders are entitled. The Company intends to use the net proceeds from the June Offering for general corporate purposes, which may include research and development costs, including the conduct of clinical trials and process development and manufacturing of the Company’s product candidates, expansion of the Company’s research and development capabilities, working capital and capital expenditures. On April 26, 2023, the Company completed its sale of a total of 9,735,731 shares of the Company’s common stock, par value $0.0001 (the “Shares”), at a purchase price of $0.946 per Share, and 22,000,000 pre-funded warrants to purchase common stock (the “Pre-Funded Warrants” and together with the Shares, the “Securities”), at a purchase price of $0.946 per Pre-Funded Warrant, in a private placement (the “Private Placement”) to a select group of accredited investors (the “Purchasers”), pursuant to a Securities Purchase Agreement (the “Securities Purchase Agreement”), dated as of April 23, 2023, by and between the Company and the Purchasers. The Private Placement underwriting discounts, commissions and offering expenses The Securities have the benefit of the Registration Rights Agreement (the “Registration Rights Agreement”), dated as of April 23, 2023, by and among the Company and the Purchasers, requiring the Company to prepare and file a registration statement with the SEC as soon as reasonably practicable, but in no event later than May 26, 2023 (the “Filing Deadline”), and to use commercially reasonable efforts to have the registration statement declared effective within 30 days of the Filing Deadline, subject to extension under the terms of the Registration Rights Agreement. The Company satisfied its obligations under the Registration Rights Agreement by filing a registration statement on Form S-3 on May 12, 2023. The Securities were issued to the Purchasers pursuant to an exemption from registration under Rule 506 of Regulation D, which is promulgated under the Securities Act. The Company relied on this exemption from registration based in part on representations made by the Purchasers. The sale of the Securities pursuant to the Securities Purchase Agreement has not been registered under the Securities Act or any state securities laws. The Securities may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements. On August 11, 2023, the Company entered into a sales agreement (the “Leerink ATM Agreement”) with Leerink Partners LLC, pursuant to which the Company may offer and sell, from time to time, shares of common stock having an aggregate offering price of up to $75.0 million through Leerink Partners LLC as sales agent. Under the Leerink ATM Agreement, the sales agent may sell shares of common stock by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended. The Company will pay the sales agent a commission rate of up to 3% of the gross offering proceeds of any shares sold and has agreed to provide the sales agent with indemnification and contribution against certain liabilities. The Leerink ATM Agreement contains customary representations and warranties. As of September 30, 2023, the Company has sold an aggregate of 4,284,344 shares of the company’s common stock, par value $0.0001, pursuant to the Leerink ATM Agreement with an average sale price of $1.75 per share, resulting in net proceeds of $7.0 million, after deducting underwriting discounts, commissions and offering expenses. On October 13, 2023, the Company completed the sale of an additional 5,750,000 shares of the company’s common stock, par value $0.0001, pursuant to the Leerink ATM Agreement with an average sale price of $2.28 per share, resulting in net proceeds of $12.7 million, after deducting underwriting discounts, commissions and offering expenses. The accompanying unaudited condensed financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. These condensed financial statements should be read in conjunction with the Company’s audited financial statements and the notes thereto for the year ended December 31, 2022 included in the Annual Report, filed with the SEC on March 23, 2023 (the “Annual Report”). The unaudited condensed financial statements have been prepared on the same basis as the audited financial statements. In the opinion of management, the accompanying unaudited condensed financial statements contain all adjustments which are necessary for a fair presentation of the Company’s financial position as of September 30, 2023, results of operations for the three and nine months ended September 30, 2023 and 2022 and cash flows for the nine months ended September 30, 2023 and 2022. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2023, are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2023. Certain reclassifications have been made to prior period financial statements to conform to the current period presentation. |
Reclassifications | Certain reclassifications have been made to prior period financial statements to conform to the current period presentation. |
Liquidity and Going Concern | The Company has incurred, and expects to continue to incur, significant operating losses for the foreseeable future as it continues to develop its drug candidates. To date, the Company has not generated any product revenue, and it does not expect to generate product revenue unless and until it successfully completes development and obtains regulatory approval for one of its product candidates. Under ASC Topic 205-40, Presentation of Financial Statements - Going Concern, management is required at each reporting period to evaluate whether there are conditions and events, considered in the aggregate, that raise substantial doubt about an entity's ability to continue as a going concern within one year after the date that the financial statements are issued. As of September 30, 2023, financing through the Company’s private placement, Leerink ATM Agreement and warrant exercises has resulted in net proceeds of $44.7 million, after deducting placement agent commissions and other offering expenses. Additionally, on October 13, 2023, the Company issued additional shares under the Leerink ATM Agreement that resulted in net proceeds of $12.7 million, after deducting placement agent commissions and other offering expenses (see Note 16). The Company continues to actively pursue several potential long-term financing options, including equity capital, debt, convertible debt, and synthetic royalty financing. Additionally, the Company is in active dialogue with several potential partners regarding business development opportunities related to one or more of its programs. There can be no assurances that the Company’s discussions with any of the current counterparties will be successful, and the Company expects to continue to pursue additional opportunities. As reflected in the accompanying financial statements, the Company incurred a net loss of $82.1 million for the nine months ended September 30, 2023 and has an accumulated deficit of $430.9 million as of September 30, 2023. The exclusive licensing agreement with Advanz Pharma for commercialization rights to AT-007 in Europe provides a source of capital to the Company based on clinical and regulatory milestones. We received a $10.7 million upfront payment from Advanz Pharma in January 2023 in conjunction with signing the agreement. If actualization of these milestones aligns with the projected timelines, and product approvals are received in the timeframes expected, this source of capital may be sufficient to cover operating expenses through expected product approvals and potential revenues. However, there are no guarantees that this will materialize timely or at all, and delays or unexpected data could disrupt this potential liquidity. Broadly, the Company has not yet established an ongoing source of revenues sufficient to cover its operating costs and is dependent on debt and equity financing to fund its operations. As of September 30, 2023, our cash and cash equivalents totaled $37.5 million. Given our planned expenditures for the next twelve months, we have concluded there is substantial doubt |
Risk and Uncertainties | Risks and Uncertainties The Company is subject to risks common to companies in the biotechnology industry, including, but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for any product candidate that it may identify and develop, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations and reliance on third-party manufacturers. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities and the Company's ability to continue as a going concern as of the date of the financial statements and the reported amounts of expenses during the reporting period. In preparing the financial statements, management used estimates in the following areas, among others: prepaid and accrued expenses; warrant liabilities valuation; stock-based compensation expense; and the evaluation of the existence of conditions and events that raise substantial doubt regarding the Company’s ability to continue as a going concern. |
Clinical hold-back, long-term | Clinical hold-back, long-term As part of the regulatory approval process for taking its products to market, the Company enters into certain Clinical Trial Agreements (CTAs) which include, among other things, the compensation and payment schedule the participating medical institutions and physicians will receive for all costs in connection with the clinical trial (or study) under the terms of the CTA. As individual patients are enrolled in the study by the participating medical institution or physician, the Company pays certain per study fees according to the CTA for the duration of the trial. As invoices are received by the Company from the medical institution or physician, the Company retains an agreed upon percentage of total invoiced costs, generally ranging between 5% - 10%, that is withheld from payment until the end of the study. These retained amounts are recorded as clinical holdback, a liability, on the accompanying balance sheets, and all expenses incurred in connection with these CTA activities are expensed as services are provided, which are included as research and development expenses on the accompanying statements of operations. The following table shows the activity within the clinical holdback liability accounts for the nine months ended September 30, 2023: (in thousands) Balance as of December 31, 2022 $ 464 Clinical holdback retained 227 Clinical holdback paid — Balance as of September 30, 2023 $ 691 |
Recent Accounting Pronouncements | Any recent pronouncements issued by the FASB or other authoritative standards groups with future effective dates are either not applicable or are not expected to be significant to the financial statements of the Company. |
ORGANIZATION AND SUMMARY OF S_3
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Schedule of activity within the clinical holdback liability accounts | (in thousands) Balance as of December 31, 2022 $ 464 Clinical holdback retained 227 Clinical holdback paid — Balance as of September 30, 2023 $ 691 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Fair value | |
Schedule of financial assets or liabilities measured at fair value on a recurring basis | As of September 30, 2023 (in thousands) Level 1 Level 2 Level 3 Total Cash $ 8,507 $ — $ — $ 8,507 Money market funds 28,950 — — 28,950 Total cash and cash equivalents $ 37,457 $ — $ — $ 37,457 U.S. government agency debt securities — — — — Total marketable securities $ — $ — $ — $ — Total financial assets measured at fair value on a recurring basis $ 37,457 $ — $ — $ 37,457 Warrant liabilities - Common Warrants — — 36,763 36,763 Total financial liabilities measured at fair value on a recurring basis $ — $ — $ 36,763 $ 36,763 As of December 31, 2022 (in thousands) Level 1 Level 2 Level 3 Total Cash $ 1,337 $ — $ — $ 1,337 Money market funds 15,320 — — 15,320 Total cash and cash equivalents $ 16,657 $ — $ — $ 16,657 U.S. government agency debt securities — 13,923 — 13,923 Total marketable securities $ — $ 13,923 $ — $ 13,923 Total financial assets measured at fair value on a recurring basis $ 16,657 $ 13,923 $ — $ 30,580 Warrant liabilities - Common Warrants — — 13,657 13,657 Total financial liabilities measured at fair value on a recurring basis $ — $ — $ 13,657 $ 13,657 |
Schedule of aggregate fair values of the Company's warrant liability | The following table provides a roll forward of the aggregate fair values of the Company’s warrant liabilities, for which fair value is determined using Level 3 inputs (in thousands): Warrant Liabilities Balance as of January 1, 2023 $ 13,657 Warrants exercised (16,505) Change in fair value 39,611 Balance as of September 30, 2023 $ 36,763 |
Common And Pre-Funded Warrants | |
Fair value | |
Schedule of fair value of warrants using the Black Scholes option pricing model | September 30, December 31, 2023 2022 Expected term (in years) 2.7 3.7 Volatility 89.8 % 91.53 % Risk-free interest rate 4.92 % 4.11 % Dividend yield 0.00 % 0.00 % |
INVESTMENTS (Tables)
INVESTMENTS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
INVESTMENTS | |
Schedule of marketable securities by security type | As of September 30, 2023 As of December 31, 2022 Gross Gross Gross Gross Unrealized Unrealized Estimated Unrealized Unrealized Estimated (in thousands) Cost Gains Losses Fair Value Cost Gains Losses Fair Value US government agency debt security $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 Total $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 |
Schedule of contractual maturities | As of September 30, 2023 As of December 31, 2022 Gross Gross Gross Gross Unrealized Unrealized Estimated Unrealized Unrealized Estimated (in thousands) Cost Gains Losses Fair Value Cost Gains Losses Fair Value Due in one year or less $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 Due in one through two years — — — — — — — — Total $ — $ — $ — $ — $ 13,873 $ 50 $ — $ 13,923 |
PREPAID EXPENSES AND OTHER CU_2
PREPAID EXPENSES AND OTHER CURRENT ASSETS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | |
Summary of prepaid expenses and other current assets | September 30, December 31, (in thousands) 2023 2022 Prepaid research and development expenses $ 4,263 $ 4,272 Insurance premium asset 955 1,131 Prepaid rent expenses 233 99 Prepaid insurance expenses 112 71 Prepaid commercial and patient advocacy 346 206 Research and development tax credit receivable 262 252 Interest receivable 23 23 Other prepaid expenses and current assets 837 674 Total prepaid expenses & other current assets $ 7,031 $ 6,728 |
ACCRUED EXPENSES AND OTHER CU_2
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | |
Summary of accrued expenses and other current liabilities | September 30, December 31, (in thousands) 2023 2022 Accrued pre-clinical and clinical expenses $ 8,354 $ 8,877 Short-term insurance financing note 775 622 Accrued professional fees 1,032 1,218 Accrued compensation and benefits 1,196 2,301 Accrued commercial expenses 59 896 Accrued patent expenses 425 361 Other 404 481 Total accrued expenses & other current liabilities $ 12,245 $ 14,756 |
STOCK BASED COMPENSATION (Table
STOCK BASED COMPENSATION (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Stock-based compensation | |
Schedule of stock-based compensation expenses | Three Months Ended Nine Months Ended September 30, September 30, (in thousands) 2023 2022 2023 2022 Research and development $ 764 $ 1,178 $ 2,378 $ 2,822 General and administrative 1,072 1,678 3,356 4,342 Total stock-based compensation expense $ 1,836 $ 2,856 $ 5,734 $ 7,164 |
Schedule of options outstanding | Weighted-Average Weighted- Remaining Aggregate Options Average Contractual Intrinsic (in thousands, except for share data) Outstanding Exercise Price Term (in years) Value Outstanding at December 31, 2022 4,874,047 $ 1.97 6.9 $ — Options granted Options exercised (85,619) 1.05 Forfeited (84,639) 1.05 Expired (8,170) 1.05 Outstanding at September 30, 2023 4,695,619 $ 2.01 6.1 $ 4,837 Exercisable at September 30, 2023 4,247,076 $ 2.11 6.0 $ 4,192 Nonvested at September 30, 2023 448,543 $ 1.05 7.8 $ 645 |
Schedule of restricted stock units outstanding | Weighted-Average Grant Date (in thousands, except for share data) Shares Fair Value Outstanding at December 31, 2022 815,509 $ 5.96 Awarded 3,164,362 1.04 Released (468,268) 4.75 Forfeited (185,267) 2.81 Outstanding at September 30, 2023 3,326,336 $ 1.63 Nonvested at September 30, 2023 3,326,336 $ 1.63 Weighted Average Remaining Recognition Period (in years) 3.1 |
Stock options - Service conditions only | |
Stock-based compensation | |
Schedule of valuation assumptions | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Expected term (in years) — — — 5.7 Volatility — % — % — % 75.83 % Risk-free interest rate — % — % — % 2.13 % Dividend yield — % — % — % — % |
WARRANTS (Tables)
WARRANTS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Warrants Issued with Series A Preferred Stock | 2017 Warrants | |
Warrants | |
Schedule of fair value of warrants using the Black Scholes option pricing model | Contractual term (in years) 10.0 Volatility 74.48 % Risk-free interest rate 3.20 % Dividend yield 0.00 % |
Warrants Issued with Series B Preferred Stock | 2018 Warrants | |
Warrants | |
Schedule of fair value of warrants using the Black Scholes option pricing model | Contractual term (in years) 10.0 Volatility 73.22 % Risk-free interest rate 2.70 % Dividend yield 0.00 % |
Warrants Issued with Series B Preferred Stock | 2019 Warrants | |
Warrants | |
Schedule of fair value of warrants using the Black Scholes option pricing model | Contractual term (in years) 10.0 Volatility 73.22 % Risk-free interest rate 2.70 % Dividend yield 0.00 % |
Common And Pre-Funded Warrants | |
Warrants | |
Schedule of fair value of warrants using the Black Scholes option pricing model | September 30, December 31, 2023 2022 Expected term (in years) 2.7 3.7 Volatility 89.8 % 91.53 % Risk-free interest rate 4.92 % 4.11 % Dividend yield 0.00 % 0.00 % |
Schedule of warrants outstanding | Equity Classified Liability Classified Total Warrants Warrants Warrants Outstanding as of January 1, 2023 8,375,618 30,000,000 38,375,618 Warrants granted and issued 22,000,000 — 22,000,000 Warrants exercised (4,250,000) (10,250,000) (14,500,000) Warrants exchanged — — — Outstanding as of September 30, 2023 26,125,618 19,750,000 45,875,618 |
LEASES (Tables)
LEASES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
LEASES | |
Summary of lease related costs | Three Months Ended Nine Months Ended (in thousands) September 30, September 30, Lease Cost Statement of Operations Location 2023 2022 2023 2022 Operating Lease Cost General and administrative $ 126 $ 126 $ 378 $ 378 Total Lease Cost $ 126 $ 126 $ 378 $ 378 |
Summary of lease terms and discount rates | Nine Months Ended September 30, September 30, Other Information 2023 2022 Weighted-average remaining lease term Operating leases 1.1 years 2.1 years Weighted-average discount rate Operating leases 5.69% 5.69% |
Summary of maturities of lease liabilities | Operating Year (in thousands) 2023 $ 126 2024 426 Thereafter — Total lease payments 552 Less: interest 17 Total lease liabilities $ 535 |
NET LOSS PER COMMON SHARE (Tabl
NET LOSS PER COMMON SHARE (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
NET LOSS PER COMMON SHARE | |
Schedule of computation of basic and diluted net loss per common share | Three Months Ended September 30, (in thousands, except for share data) 2023 2022 Numerator: Net loss $ (42,370) $ (19,101) Denominator: Weighted-average common stock outstanding 90,669,969 48,000,183 Net loss per share attributable to common stockholders - basic and diluted $ (0.47) $ (0.40) Nine Months Ended September 30, (in thousands, except for share data) 2023 2022 Numerator: Net loss $ (82,084) $ (68,079) Denominator: Weighted-average common stock outstanding 75,482,234 33,785,386 Net loss per share attributable to common stockholders - basic and diluted $ (1.09) $ (2.02) |
Schedule of potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders | As of September 30, 2023 2022 Options to purchase common stock 4,695,619 4,925,678 Restricted stock units 3,326,336 533,114 Warrants to purchase common stock 19,875,618 38,375,618 |
ORGANIZATION AND SUMMARY OF S_4
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Operations and Business (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | 9 Months Ended | |||||||||
Oct. 13, 2023 | Aug. 11, 2023 | Apr. 26, 2023 | Jun. 27, 2022 | Jan. 26, 2022 | Sep. 30, 2023 | Jun. 30, 2022 | Sep. 30, 2023 | Oct. 12, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | |
Sale of stock | |||||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||||||||
Subsequent Event | |||||||||||
Sale of stock | |||||||||||
Common Stock, par value (in dollars per share) | $ 0.001 | ||||||||||
Common And Pre-Funded Warrants | |||||||||||
Sale of stock | |||||||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||||||
Pre-Funded Warrants | |||||||||||
Sale of stock | |||||||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||||||
Common Stock | |||||||||||
Sale of stock | |||||||||||
Issuance of common stock (in shares) | 4,284,344 | 20,000,000 | |||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||||||||
Equity Distribution Agreement | |||||||||||
Sale of stock | |||||||||||
Maximum value of shares authorized to be sold in stock offering | $ 100 | ||||||||||
Maximum amount to be paid to sales agent (as a percent) | 3% | ||||||||||
June 2022 Offering | |||||||||||
Sale of stock | |||||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||||||||||
Offering price per share and accompanying warrant (in dollars per share) | 1 | ||||||||||
Offering price per pre-funded warrant and accompanying warrant (in dollars per share) | $ 0.9999 | ||||||||||
Proceeds from issuance of shares and warrants, net | $ 27.8 | ||||||||||
June 2022 Offering | Common And Pre-Funded Warrants | |||||||||||
Sale of stock | |||||||||||
Warrants term | 5 years | ||||||||||
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants | 9.99% | ||||||||||
Threshold percentage of aggregate beneficial ownership of holder (together with affiliates) for exercising warrants | 19.99% | ||||||||||
June 2022 Offering | Pre-Funded Warrants | |||||||||||
Sale of stock | |||||||||||
Issuance of warrants (in shares) | 10,000,000 | ||||||||||
Exercise price (in dollars per share) | $ 0.0001 | ||||||||||
June 2022 Offering | Common Warrants | |||||||||||
Sale of stock | |||||||||||
Issuance of warrants (in shares) | 30,000,000 | ||||||||||
Exercise price (in dollars per share) | $ 1 | ||||||||||
June 2022 Offering | Common Stock | |||||||||||
Sale of stock | |||||||||||
Issuance of common stock (in shares) | 20,000,000 | ||||||||||
April 2023 Offering, Private Placement | |||||||||||
Sale of stock | |||||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||||||||||
Proceeds from issuance of shares and warrants, net | $ 27.5 | ||||||||||
Registration statement, period to be declared effective | 30 days | ||||||||||
April 2023 Offering, Private Placement | Pre-Funded Warrants | |||||||||||
Sale of stock | |||||||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||||||
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants | 9.99% | ||||||||||
Purchase price (in dollars per share) | $ 0.946 | ||||||||||
Exercise price (in dollars per share) | $ 0.001 | ||||||||||
April 2023 Offering, Private Placement | Common Stock | |||||||||||
Sale of stock | |||||||||||
Issuance of common stock (in shares) | 9,735,731 | ||||||||||
Purchase price (in dollars per share) | $ 0.946 | ||||||||||
April 2023 Offering, Private Placement | Common Stock | Pre-Funded Warrants | |||||||||||
Sale of stock | |||||||||||
Purchase price (in dollars per share) | $ 0.946 | ||||||||||
Leerink ATM Agreement | |||||||||||
Sale of stock | |||||||||||
Maximum value of shares authorized to be sold in stock offering | $ 75 | ||||||||||
Maximum amount to be paid to sales agent (as a percent) | 3% | ||||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | 0.0001 | |||||||||
Proceeds from stock issuance, net of cash issuance costs | $ 7 | ||||||||||
Leerink ATM Agreement | Subsequent Event | |||||||||||
Sale of stock | |||||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||||||||||
Proceeds from stock issuance, net of cash issuance costs | $ 12.7 | ||||||||||
Leerink ATM Agreement | Common Stock | |||||||||||
Sale of stock | |||||||||||
Issuance of common stock (in shares) | 4,284,344 | ||||||||||
Purchase price (in dollars per share) | $ 1.75 | $ 1.75 | |||||||||
Leerink ATM Agreement | Common Stock | Subsequent Event | |||||||||||
Sale of stock | |||||||||||
Issuance of common stock (in shares) | 5,750,000 | ||||||||||
Purchase price (in dollars per share) | $ 2.28 |
ORGANIZATION AND SUMMARY OF S_5
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Liquidity and Going Concern (Details) $ in Thousands, € in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended | |||||
Oct. 13, 2023 USD ($) | Jan. 03, 2023 USD ($) | Jan. 03, 2023 EUR (€) | Jan. 31, 2023 USD ($) | Sep. 30, 2023 USD ($) | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Dec. 31, 2022 USD ($) | |
Cash and cash equivalents | ||||||||
Net loss | $ (82,084) | $ (68,079) | ||||||
Accumulated deficit | $ (430,907) | (430,907) | $ (348,823) | |||||
Cash and cash equivalents | 37,457 | $ 37,457 | $ 16,657 | |||||
Substantial Doubt about Going Concern, within One Year | true | |||||||
ADVANZ PHARMA | Exclusive License and Supply Agreement | ||||||||
Cash and cash equivalents | ||||||||
Upfront payment received | $ 10,700 | € 10 | $ 10,700 | |||||
Leerink ATM Agreement | ||||||||
Cash and cash equivalents | ||||||||
Proceeds from issuance of shares and warrants and warrant exercises, net | 44,700 | |||||||
Proceeds from stock issuance, net of cash issuance costs | $ 7,000 | |||||||
Leerink ATM Agreement | Subsequent Event | ||||||||
Cash and cash equivalents | ||||||||
Proceeds from stock issuance, net of cash issuance costs | $ 12,700 |
ORGANIZATION AND SUMMARY OF S_6
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Clinical hold-back, long-term (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Agreements | |
Clinical holdback, Balance as of beginning of period | $ 464 |
Clinical holdback retained | 227 |
Clinical holdback, Balance as of end of period | $ 691 |
Minimum | |
Agreements | |
Clinical holdback, Invoiced costs withheld from payment (as a percent) | 5% |
Maximum | |
Agreements | |
Clinical holdback, Invoiced costs withheld from payment (as a percent) | 10% |
LICENSE AGREEMENT - Columbia (D
LICENSE AGREEMENT - Columbia (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 84 Months Ended | ||||||
Oct. 03, 2019 | Mar. 31, 2019 | Jan. 31, 2019 | Oct. 31, 2016 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Dec. 31, 2022 | |
Agreements | ||||||||||
Research and development | $ 10,785 | $ 13,116 | $ 38,602 | $ 43,542 | ||||||
General and administrative | 4,710 | 6,240 | 15,585 | 20,436 | ||||||
Operating expenses | 15,495 | 19,356 | 54,187 | 63,978 | ||||||
Accrued expenses and other current liabilities | 12,245 | 12,245 | $ 12,245 | $ 14,756 | ||||||
Accounts payable | 6,005 | 6,005 | 6,005 | 4,534 | ||||||
License Agreements and Research Services Agreements | Columbia University | ||||||||||
Agreements | ||||||||||
Research and development | 0 | 0 | 0 | 0 | ||||||
General and administrative | 0 | $ 100 | 100 | $ 200 | ||||||
Operating expenses | 2,800 | |||||||||
Accrued expenses and other current liabilities | 100 | 100 | 100 | 100 | ||||||
Accounts payable | $ 0 | $ 0 | $ 0 | $ 0 | ||||||
2016 license agreement | Columbia University | ||||||||||
Agreements | ||||||||||
Percentage of outstanding common stock issued | 5% | |||||||||
Fair value of stock issued | $ 500 | |||||||||
Written notice period for termination of agreement | 90 days | |||||||||
Written notice period for termination of agreement, uncured material breach | 30 days | |||||||||
Extended written notice period for termination of agreement, attempt to cure material breach | 90 days | |||||||||
2016 license agreement | Columbia University | Minimum | ||||||||||
Agreements | ||||||||||
Percentage of sublicensing revenue, if sublicenses rights granted | 10% | |||||||||
2016 license agreement | Columbia University | Maximum | ||||||||||
Agreements | ||||||||||
Payment on achievement of specified development and regulatory milestones | $ 1,300 | |||||||||
Payment on achievement of specified level of aggregate annual net sales | $ 1,000 | |||||||||
Percentage of sublicensing revenue, if sublicenses rights granted | 20% | |||||||||
2019 license agreement | Columbia University | Maximum | ||||||||||
Agreements | ||||||||||
Payment on achievement of specified development and regulatory milestones | $ 1,300 | |||||||||
Payment on achievement of specified level of aggregate annual net sales | $ 1,000 | |||||||||
2019 research agreement | Columbia University | ||||||||||
Agreements | ||||||||||
Agreement term | 12 months | |||||||||
PI3k Research agreement | Columbia University | ||||||||||
Agreements | ||||||||||
Agreement term | 18 months |
LICENSE AGREEMENT - Miami Unive
LICENSE AGREEMENT - Miami University (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | 35 Months Ended | |||||
Dec. 14, 2020 | Oct. 28, 2020 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Dec. 31, 2022 | |
Agreements | ||||||||
Research and development | $ 10,785 | $ 13,116 | $ 38,602 | $ 43,542 | ||||
General and administrative | 4,710 | 6,240 | 15,585 | 20,436 | ||||
Operating expenses | 15,495 | 19,356 | 54,187 | 63,978 | ||||
Accrued expenses and other current liabilities | 12,245 | 12,245 | $ 12,245 | $ 14,756 | ||||
2020 license agreement | University of Miami | ||||||||
Agreements | ||||||||
Amount of first payment to be made for license issue fee | $ 1,100 | |||||||
Amount of second payment to be made for license issue fee, due on first anniversary of the date of license | 500 | |||||||
Payment to be made on achievement of specified patenting and development milestones | 2,200 | |||||||
Payment to be made on achievement of late stage regulatory milestones | $ 4,100 | |||||||
Agreement term | 10 years | |||||||
Written notice period for termination of agreement | 60 days | |||||||
Written notice period of material breach leading to termination | 60 days | |||||||
Research and development | 25 | 25 | 100 | 100 | ||||
General and administrative | 0 | 0 | 0 | 2 | ||||
Operating expenses | 2,500 | |||||||
Accrued expenses and other current liabilities | 300 | 300 | 300 | $ 300 | ||||
2020 license agreement | University of Miami | Minimum | ||||||||
Agreements | ||||||||
Royalties payment (as a percent) | 0.88% | |||||||
Non-royalty sublicensing revenue payment (as a percent) | 15% | |||||||
2020 license agreement | University of Miami | Maximum | ||||||||
Agreements | ||||||||
Royalties payment (as a percent) | 5% | |||||||
Non-royalty sublicensing revenue payment (as a percent) | 25% | |||||||
2020 research agreement and amendment | University of Miami | ||||||||
Agreements | ||||||||
Consideration | $ 300 | |||||||
Research and development | 0 | $ 0 | 0 | $ 100 | ||||
Accrued expenses and other current liabilities | $ 100 | $ 100 | $ 100 |
FAIR VALUE MEASUREMENTS - FV hi
FAIR VALUE MEASUREMENTS - FV hierarchy (Details) - Fair Value, Recurring - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | $ 37,457 | $ 16,657 |
Marketable securities | 13,923 | |
Total financial assets measured at fair value on a recurring basis | 37,457 | 30,580 |
Warrant liabilities - Common Warrants | 36,763 | 13,657 |
Total financial liabilities measured at fair value on a recurring basis | 36,763 | 13,657 |
Level 1 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 37,457 | 16,657 |
Total financial assets measured at fair value on a recurring basis | 37,457 | 16,657 |
Level 2 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Marketable securities | 13,923 | |
Total financial assets measured at fair value on a recurring basis | 13,923 | |
Level 3 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Warrant liabilities - Common Warrants | 36,763 | 13,657 |
Total financial liabilities measured at fair value on a recurring basis | 36,763 | 13,657 |
Cash | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 8,507 | 1,337 |
Cash | Level 1 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 8,507 | 1,337 |
Money market funds | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 28,950 | 15,320 |
Money market funds | Level 1 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | $ 28,950 | 15,320 |
U.S. government agency debt securities | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Marketable securities | 13,923 | |
U.S. government agency debt securities | Level 2 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Marketable securities | $ 13,923 |
FAIR VALUE MEASUREMENTS - Trans
FAIR VALUE MEASUREMENTS - Transfers (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
FAIR VALUE MEASUREMENTS | ||
Assets, transfer from level 1 to level 2 | $ 0 | $ 0 |
Assets, transfer from level 2 to level 1 | $ 0 | $ 0 |
FAIR VALUE MEASUREMENTS - Warra
FAIR VALUE MEASUREMENTS - Warrants (Details) - shares | 9 Months Ended | |
Jun. 27, 2022 | Sep. 30, 2023 | |
Common And Pre-Funded Warrants | ||
Warrants | ||
Issuance of warrants (in shares) | 22,000,000 | |
Pre-Funded Warrants | ||
Warrants | ||
Issuance of warrants (in shares) | 22,000,000 | |
June 2022 Offering | Common Warrants | ||
Warrants | ||
Issuance of warrants (in shares) | 30,000,000 | |
June 2022 Offering | Pre-Funded Warrants | ||
Warrants | ||
Issuance of warrants (in shares) | 10,000,000 |
FAIR VALUE MEASUREMENTS - War_2
FAIR VALUE MEASUREMENTS - Warrants Valuation (Details) | Sep. 30, 2023 Y | Dec. 31, 2022 Y |
Expected term (in years) | Common And Pre-Funded Warrants | ||
Fair value | ||
Measurement input | 3.7 | |
Expected term (in years) | Common Warrants | ||
Fair value | ||
Measurement input | 2.7 | |
Volatility | Common And Pre-Funded Warrants | ||
Fair value | ||
Measurement input | 0.9153 | |
Volatility | Common Warrants | ||
Fair value | ||
Measurement input | 0.898 | |
Risk-free interest rate | Common And Pre-Funded Warrants | ||
Fair value | ||
Measurement input | 0.0411 | |
Risk-free interest rate | Common Warrants | ||
Fair value | ||
Measurement input | 0.0492 | |
Dividend yield | Common And Pre-Funded Warrants | ||
Fair value | ||
Measurement input | 0 | |
Dividend yield | Common Warrants | ||
Fair value | ||
Measurement input | 0 | |
Probability of change in control | Common And Pre-Funded Warrants | ||
Fair value | ||
Measurement input | 0.100 | |
Probability of change in control | Common Warrants | Valuation input period, years two and three | ||
Fair value | ||
Measurement input | 0.20 | |
Probability of change in control | Common Warrants | Valuation input period, years four and five | ||
Fair value | ||
Measurement input | 0.05 |
FAIR VALUE MEASUREMENTS - Fair
FAIR VALUE MEASUREMENTS - Fair Values of Company's Warrant Liability (Details) - Common Warrants $ in Thousands | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Fair Value | |
Beginning Balance | $ 13,657 |
Warrants exercised | (16,505) |
Change in fair value | 39,611 |
Ending Balance | $ 36,763 |
INVESTMENTS - By type (Details)
INVESTMENTS - By type (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Marketable securities by type | ||
Cost | $ 13,873 | |
Gross Unrealized Gains | $ 0 | 50 |
Gross Unrealized Losses | 0 | |
Estimated Fair Value | $ 0 | 13,923 |
U.S. government agency debt securities | ||
Marketable securities by type | ||
Cost | 13,873 | |
Gross Unrealized Gains | 50 | |
Estimated Fair Value | $ 13,923 |
INVESTMENTS - By contractual ma
INVESTMENTS - By contractual maturity (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
INVESTMENTS | ||
Due in one year or less - Cost | $ 13,873 | |
Due in on year or less - Gross Unrealized Gains | 50 | |
Due in one year or less - Estimated Fair Value | 13,923 | |
Cost | 13,873 | |
Gross Unrealized Gains | $ 0 | 50 |
Gross Unrealized Losses | 0 | |
Estimated Fair Value | $ 0 | $ 13,923 |
INVESTMENTS - Realized gains an
INVESTMENTS - Realized gains and losses (Details) $ in Millions | 3 Months Ended |
Sep. 30, 2023 USD ($) | |
Marketable securities realized gain (loss) recorded | |
Gross realized gains from sale of marketable securities | $ 0 |
Gross realized losses from sale of marketable securities | $ 0 |
INVESTMENTS - Unrealized losses
INVESTMENTS - Unrealized losses and fair values of available-for-sale securities (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Securities in an unrealized loss position greater than 12 months | ||
Unrealized Losses Greater Than 12 months | $ 0 | $ 0 |
PREPAID EXPENSES AND OTHER CU_3
PREPAID EXPENSES AND OTHER CURRENT ASSETS (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | ||
Prepaid research and development expenses | $ 4,263 | $ 4,272 |
Insurance premium asset | 955 | 1,131 |
Prepaid rent expenses | 233 | 99 |
Prepaid insurance expenses | 112 | 71 |
Prepaid commercial and patient advocacy | 346 | 206 |
Research and development tax credit receivable | 262 | 252 |
Interest receivable | 23 | 23 |
Other prepaid expenses and current assets | 837 | 674 |
Total prepaid expenses & other current assets | $ 7,031 | $ 6,728 |
ACCRUED EXPENSES AND OTHER CU_3
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | ||
Accrued pre-clinical and clinical expenses | $ 8,354 | $ 8,877 |
Short-term insurance financing note | 775 | 622 |
Accrued professional fees | 1,032 | 1,218 |
Accrued compensation and benefits | 1,196 | 2,301 |
Accrued commercial expenses | 59 | 896 |
Accrued patent expenses | 425 | 361 |
Other | 404 | 481 |
Total accrued expenses & other current liabilities | $ 12,245 | $ 14,756 |
STOCK BASED COMPENSATION - Plan
STOCK BASED COMPENSATION - Plans (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 9 Months Ended | |||
Aug. 02, 2022 | May 31, 2019 | Sep. 30, 2023 | Sep. 30, 2022 | Aug. 01, 2022 | |
Options | |||||
Stock-based compensation | |||||
Weighted Average Remaining Recognition Period (in years) | 1 year 9 months 18 days | 2 years 3 months 18 days | |||
2019 Plan | |||||
Stock-based compensation | |||||
Shares reserved (in shares) | 4,530,000 | ||||
New shares reserved (in shares) | 1,618,841 | ||||
Reserved shares available from 2016 Plan (in shares) | 2,911,159 | ||||
Increase in number of shares reserved and available for issuance (as a percent) | 5% | ||||
Option exercise price upon option repricing (in dollars per share) | $ 1.05 | ||||
Options vested upon option repricing (in Shares) | 1,797,517 | ||||
Options non-vested upon option repricing (in Shares) | 1,380,917 | ||||
Incremental stock based compensation expense | $ 1.4 | ||||
Weighted Average Remaining Recognition Period (in years) | 2 years 4 months 24 days | ||||
Number of shares available for future issuance | 615,885 | ||||
2019 Plan | Minimum | |||||
Stock-based compensation | |||||
Original exercise price (in dollars per share) | $ 1.22 | ||||
2019 Plan | Maximum | |||||
Stock-based compensation | |||||
Original exercise price (in dollars per share) | $ 49.60 | ||||
2019 Plan | Options | |||||
Stock-based compensation | |||||
Maximum number of shares authorized for issuance | 13,000,000 | ||||
Expiration period | 10 years | ||||
Vesting period | 4 years | ||||
2019 Plan | Vested stock option | |||||
Stock-based compensation | |||||
Incremental stock based compensation expense | $ 0.9 | ||||
2019 Plan | Non-vested stock option | |||||
Stock-based compensation | |||||
Incremental stock based compensation expense | $ 0.5 |
STOCK BASED COMPENSATION - Stoc
STOCK BASED COMPENSATION - Stock-based compensation expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Stock-based compensation | ||||
Total stock-based compensation expense | $ 1,836 | $ 2,856 | $ 5,734 | $ 7,164 |
Research and development | ||||
Stock-based compensation | ||||
Total stock-based compensation expense | 764 | 1,178 | 2,378 | 2,822 |
General and administrative | ||||
Stock-based compensation | ||||
Total stock-based compensation expense | $ 1,072 | $ 1,678 | $ 3,356 | $ 4,342 |
STOCK BASED COMPENSATION - Opti
STOCK BASED COMPENSATION - Options (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Stock-based compensation | ||||
Options granted (in shares) | 0 | |||
Stock-based compensation expense | $ 1,836 | $ 2,856 | $ 5,734 | $ 7,164 |
Unrecognized stock-based compensation balance for unvested options | 3,700 | 9,900 | $ 3,700 | $ 9,900 |
Weighted-average fair value of options granted (in dollars per share) | $ 0 | $ 1.79 | ||
Options | ||||
Stock-based compensation | ||||
Stock-based compensation expense | $ 1,100 | $ 2,500 | $ 3,900 | $ 6,200 |
Weighted Average Remaining Recognition Period (in years) | 1 year 9 months 18 days | 2 years 3 months 18 days |
STOCK BASED COMPENSATION - Op_2
STOCK BASED COMPENSATION - Options activity (Details) $ / shares in Units, $ in Thousands | 9 Months Ended | 12 Months Ended |
Sep. 30, 2023 USD ($) $ / shares shares | Dec. 31, 2022 $ / shares shares | |
Options Outstanding | ||
Outstanding at beginning of period (in shares) | 4,874,047 | |
Options granted (in shares) | 0 | |
Options exercised (in shares) | (85,619) | |
Forfeited (in shares) | (84,639) | |
Expired (in shares) | (8,170) | |
Outstanding at end of period (in shares) | 4,695,619 | 4,874,047 |
Exercisable at end of period (in shares) | 4,247,076 | |
Nonvested at end of period (in shares) | 448,543 | |
Weighted-Average Exercise Price | ||
Outstanding at beginning of period (in dollars per share) | $ / shares | $ 1.97 | |
Options exercised (in dollars per share) | $ / shares | 1.05 | |
Options forfeited (in dollars per share) | $ / shares | 1.05 | |
Options expired (in dollars per share) | $ / shares | 1.05 | |
Outstanding at end of period (in dollars per share) | $ / shares | 2.01 | $ 1.97 |
Exercisable at end of period (in dollars per share) | $ / shares | 2.11 | |
Nonvested at end of period (in dollars per share) | $ / shares | $ 1.05 | |
Weighted-Average Remaining Contractual Term-in years | ||
Outstanding | 6 years 1 month 6 days | 6 years 10 months 24 days |
Exercisable | 6 years | |
Nonvested | 7 years 9 months 18 days | |
Aggregate Intrinsic Value | ||
Outstanding (in dollars) | $ | $ 4,837 | |
Exercisable (in dollars) | $ | 4,192 | |
Nonvested (in dollars) | $ | $ 645 |
STOCK BASED COMPENSATION - Valu
STOCK BASED COMPENSATION - Valuation assumptions (Details) - Stock options - Service conditions only | 9 Months Ended |
Sep. 30, 2022 | |
Valuation Assumptions | |
Expected term (in years) | 5 years 8 months 12 days |
Volatility (as a percent) | 75.83% |
Risk-free interest rate (as a percent) | 2.13% |
STOCK BASED COMPENSATION - RSUs
STOCK BASED COMPENSATION - RSUs (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Stock-based compensation | ||||
Stock-based compensation expense | $ 1,836 | $ 2,856 | $ 5,734 | $ 7,164 |
Restricted stock units | ||||
Stock-based compensation | ||||
Stock-based compensation expense | 700 | 400 | 1,800 | 900 |
Unamortized stock-based compensation costs for unvested awards | $ 4,500 | $ 3,900 | $ 4,500 | $ 3,900 |
Summary of restricted stock units outstanding | ||||
Outstanding at beginning of period ( in shares) | 815,509 | |||
Awarded (in shares) | 3,164,362 | |||
Released (in shares) | (468,268) | |||
Forfeited (in shares) | (185,267) | |||
Outstanding at end of period ( in shares) | 3,326,336 | 3,326,336 | ||
Nonvested at end of period (in shares) | 3,326,336 | 3,326,336 | ||
Weighted-Average Grant Date Fair Value | ||||
Outstanding at beginning of period (in dollars per share) | $ 5.96 | |||
Awarded (in dollars per share) | 1.04 | |||
Released (in dollars per share) | 4.75 | |||
Forfeited (in dollars per share) | 2.81 | |||
Outstanding at end of period (in dollars per share) | $ 1.63 | 1.63 | ||
Nonvested at end of period (in dollars per share) | $ 1.63 | $ 1.63 | ||
Weighted Average Remaining Recognition Period (in years) | 3 years 1 month 6 days |
STOCK BASED COMPENSATION - 2019
STOCK BASED COMPENSATION - 2019 Employee Stock Purchase Plan (Details) - 2019 ESPP - shares | 1 Months Ended | 9 Months Ended |
May 31, 2019 | Sep. 30, 2023 | |
Stock-based compensation | ||
Shares authorized (in shares) | 180,000 | |
Shares issued under ESPP | 0 | |
Maximum | ||
Stock-based compensation | ||
Increase in number of shares reserved and available for issuance (as a percent) | 1% | |
Increase in number of shares reserved and available for issuance (in shares) | 360,000 |
STOCKHOLDERS' EQUITY - Authoriz
STOCKHOLDERS' EQUITY - Authorized stock (Details) - $ / shares | Sep. 30, 2023 | Dec. 31, 2022 |
STOCKHOLDERS' EQUITY | ||
Common Stock, authorized (in shares) | 200,000,000 | 200,000,000 |
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred Stock, authorized (in shares) | 10,000,000 | 10,000,000 |
Preferred Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
STOCKHOLDERS' EQUITY - Equity D
STOCKHOLDERS' EQUITY - Equity Distribution Agreements (Details) - USD ($) $ in Millions | 1 Months Ended | |
Jan. 26, 2022 | Jun. 30, 2020 | |
Goldman Equity Distribution Agreement | ||
STOCKHOLDERS' EQUITY | ||
Maximum value of shares authorized to be sold in stock offering. | $ 100 | |
Maximum amount to be paid to sales agent (as a percent) | 3% | |
Equity Distribution Agreement | ||
STOCKHOLDERS' EQUITY | ||
Maximum value of shares authorized to be sold in stock offering. | $ 100 | |
Maximum amount to be paid to sales agent (as a percent) | 3% |
STOCKHOLDERS' EQUITY - June 202
STOCKHOLDERS' EQUITY - June 2022 Offering (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | 9 Months Ended | ||||
Jun. 27, 2022 | Sep. 30, 2023 | Jun. 30, 2022 | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | |
Equity | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | |||
Common And Pre-Funded Warrants | ||||||
Equity | ||||||
Issuance of warrants (in shares) | 22,000,000 | |||||
Pre-Funded Warrants | ||||||
Equity | ||||||
Issuance of warrants (in shares) | 22,000,000 | |||||
Common Stock | ||||||
Equity | ||||||
Issuance of common stock (in shares) | 4,284,344 | 20,000,000 | ||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | |||
June 2022 Offering | ||||||
Equity | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | |||||
Offering price per share and accompanying warrant (in dollars per share) | 1 | |||||
Offering price per pre-funded warrant and accompanying warrant (in dollars per share) | $ 0.9999 | |||||
Proceeds from issuance of shares and warrants, net | $ 27.8 | |||||
June 2022 Offering | Common And Pre-Funded Warrants | ||||||
Equity | ||||||
Warrants term | 5 years | |||||
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants | 9.99% | |||||
Threshold percentage of aggregate beneficial ownership of holder (together with affiliates) for exercising warrants | 19.99% | |||||
June 2022 Offering | Pre-Funded Warrants | ||||||
Equity | ||||||
Issuance of warrants (in shares) | 10,000,000 | |||||
June 2022 Offering | Common Warrants | ||||||
Equity | ||||||
Issuance of warrants (in shares) | 30,000,000 | |||||
June 2022 Offering | Common Stock | ||||||
Equity | ||||||
Issuance of common stock (in shares) | 20,000,000 |
STOCKHOLDERS' EQUITY - April 20
STOCKHOLDERS' EQUITY - April 2023 Offering (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | 9 Months Ended | |||||
Apr. 26, 2023 | Sep. 30, 2023 | Jun. 30, 2023 | Jun. 30, 2022 | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | |
Equity | |||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||||
Common And Pre-Funded Warrants | |||||||
Equity | |||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||
Pre-Funded Warrants | |||||||
Equity | |||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||
Common Stock | |||||||
Equity | |||||||
Issuance of common stock (in shares) | 4,284,344 | 20,000,000 | |||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||||
Issuance of common stock and pre-funded warrants, net of issuance costs (in shares) | 9,735,731 | ||||||
April 2023 Offering, Private Placement | |||||||
Equity | |||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||||||
Proceeds from issuance of shares and warrants, net | $ 27.5 | ||||||
April 2023 Offering, Private Placement | Pre-Funded Warrants | |||||||
Equity | |||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||
Purchase price (in dollars per share) | $ 0.946 | ||||||
Exercise price (in dollars per share) | $ 0.001 | ||||||
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants | 9.99% | ||||||
April 2023 Offering, Private Placement | Common Stock | |||||||
Equity | |||||||
Issuance of common stock (in shares) | 9,735,731 | ||||||
Purchase price (in dollars per share) | $ 0.946 | ||||||
April 2023 Offering, Private Placement | Common Stock | Pre-Funded Warrants | |||||||
Equity | |||||||
Purchase price (in dollars per share) | $ 0.946 |
STOCKHOLDERS' EQUITY - ATM Offe
STOCKHOLDERS' EQUITY - ATM Offering (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | ||||||
Oct. 13, 2023 | Aug. 11, 2023 | Sep. 30, 2023 | Jun. 30, 2022 | Oct. 12, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | |
Equity | |||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | |||||
Subsequent Event | |||||||
Equity | |||||||
Common Stock, par value (in dollars per share) | $ 0.001 | ||||||
Common Stock | |||||||
Equity | |||||||
Issuance of common stock (in shares) | 4,284,344 | 20,000,000 | |||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | |||||
Leerink ATM Agreement | |||||||
Equity | |||||||
Maximum value of shares authorized to be sold in stock offering | $ 75 | ||||||
Maximum amount to be paid to sales agent (as a percent) | 3% | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||||||
Proceeds from stock issuance, net of cash issuance costs | $ 7 | ||||||
Leerink ATM Agreement | Subsequent Event | |||||||
Equity | |||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||||||
Proceeds from stock issuance, net of cash issuance costs | $ 12.7 | ||||||
Leerink ATM Agreement | Common Stock | |||||||
Equity | |||||||
Issuance of common stock (in shares) | 4,284,344 | ||||||
Purchase price (in dollars per share) | $ 1.75 | ||||||
Leerink ATM Agreement | Common Stock | Subsequent Event | |||||||
Equity | |||||||
Issuance of common stock (in shares) | 5,750,000 | ||||||
Purchase price (in dollars per share) | $ 2.28 |
WARRANTS - Issued with Series A
WARRANTS - Issued with Series A Preferred Stock (Details) - 2017 Warrants - Warrants Issued with Series A Preferred Stock $ / shares in Units, $ in Millions | Jan. 26, 2017 USD ($) $ / shares shares | Sep. 30, 2023 Y |
Warrants | ||
Issuance of warrants (in shares) | shares | 309,389 | |
Warrants to purchase common stock (in dollars) | $ | $ 0.2 | |
Exercise price (in dollars per share) | $ / shares | $ 2.49 | |
Expected term (in years) | ||
Warrants | ||
Measurement input | Y | 10 | |
Volatility | ||
Warrants | ||
Measurement input | 0.7448 | |
Risk-free interest rate | ||
Warrants | ||
Measurement input | 0.0320 | |
Dividend yield | ||
Warrants | ||
Measurement input | 0 |
WARRANTS - Issued with the 2018
WARRANTS - Issued with the 2018 Notes (Details) - Warrants Issued with the 2018 Notes - USD ($) $ / shares in Units, $ in Millions | Nov. 05, 2018 | Sep. 30, 2023 | Dec. 31, 2022 | Jan. 18, 2018 |
Warrants | ||||
Initial fair value recorded as debt discount (in dollars) | $ 0.1 | |||
Issuance of warrants (in shares) | 76,847 | |||
Warrants to purchase common stock (in dollars) | $ 0.3 | |||
Exercise price (in dollars per share) | $ 3.99 | $ 6.59 |
WARRANTS - Issued with Series B
WARRANTS - Issued with Series B Preferred Stock (Details) - Warrants Issued with Series B Preferred Stock $ / shares in Units, $ in Millions | 1 Months Ended | 2 Months Ended | ||
Feb. 28, 2019 USD ($) shares | Dec. 31, 2018 USD ($) shares | Sep. 30, 2023 Y $ / shares | Dec. 31, 2022 $ / shares | |
2018 Warrants | ||||
Warrants | ||||
Number of warrants entity became obligated to issue (in shares) | shares | 72,261 | |||
Warrants to purchase common stock (in dollars) | $ | $ 0.2 | |||
Expiration term | 10 years | |||
Exercise price (in dollars per share) | $ / shares | $ 4.86 | $ 8.24 | ||
2018 Warrants | Expected term (in years) | ||||
Warrants | ||||
Measurement input | Y | 10 | |||
2018 Warrants | Volatility | ||||
Warrants | ||||
Measurement input | 0.7322 | |||
2018 Warrants | Risk-free interest rate | ||||
Warrants | ||||
Measurement input | 0.0270 | |||
2018 Warrants | Dividend yield | ||||
Warrants | ||||
Measurement input | 0 | |||
2019 Warrants | ||||
Warrants | ||||
Number of warrants entity became obligated to issue (in shares) | shares | 23,867 | |||
Warrants to purchase common stock (in dollars) | $ | $ 0.1 | |||
Expiration term | 10 years | |||
Exercise price (in dollars per share) | $ / shares | $ 4.86 | $ 8.24 | ||
2019 Warrants | Expected term (in years) | ||||
Warrants | ||||
Measurement input | Y | 10 | |||
2019 Warrants | Volatility | ||||
Warrants | ||||
Measurement input | 0.7322 | |||
2019 Warrants | Risk-free interest rate | ||||
Warrants | ||||
Measurement input | 0.0270 | |||
2019 Warrants | Dividend yield | ||||
Warrants | ||||
Measurement input | 0 |
WARRANTS - Issued with June 202
WARRANTS - Issued with June 2022 Offering (Details) | 3 Months Ended | 9 Months Ended | |||||||||||
Sep. 30, 2023 Y $ / shares shares | Sep. 19, 2023 USD ($) shares | Sep. 14, 2023 USD ($) shares | Sep. 11, 2023 USD ($) shares | Aug. 01, 2023 USD ($) shares | Jun. 08, 2023 USD ($) shares | Jun. 28, 2022 USD ($) shares | Jun. 27, 2022 $ / shares shares | Sep. 30, 2023 USD ($) Y $ / shares shares | Jun. 30, 2023 shares | Jun. 30, 2022 shares | Sep. 30, 2023 USD ($) Y $ / shares shares | Dec. 31, 2022 Y shares | |
Warrants | |||||||||||||
Exercise of common warrants | $ | $ 26,755,000 | ||||||||||||
Proceeds from exercise of warrants | $ | $ 10,250,000 | ||||||||||||
Common Stock | |||||||||||||
Warrants | |||||||||||||
Exercise of pre-funded warrants for common stock (in shares) | 4,250,000 | 1,750,000 | |||||||||||
Exercise of common warrants | $ | $ 1,000 | ||||||||||||
Exercise of common warrants (in shares) | 10,250,000 | ||||||||||||
Common And Pre-Funded Warrants | |||||||||||||
Warrants | |||||||||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||||||||
Warrants exercised (in shares) | 14,500,000 | ||||||||||||
Warrants outstanding (in shares) | 45,875,618 | 45,875,618 | 45,875,618 | 38,375,618 | |||||||||
Common And Pre-Funded Warrants | Expected term (in years) | |||||||||||||
Warrants | |||||||||||||
Measurement input | Y | 3.7 | ||||||||||||
Common And Pre-Funded Warrants | Volatility | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.9153 | ||||||||||||
Common And Pre-Funded Warrants | Risk-free interest rate | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.0411 | ||||||||||||
Common And Pre-Funded Warrants | Dividend yield | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0 | ||||||||||||
Pre-Funded Warrants | |||||||||||||
Warrants | |||||||||||||
Issuance of warrants (in shares) | 22,000,000 | ||||||||||||
Warrants exercised (in shares) | 4,250,000 | ||||||||||||
Warrants outstanding (in shares) | 26,125,618 | 26,125,618 | 26,125,618 | 8,375,618 | |||||||||
Common Warrants | |||||||||||||
Warrants | |||||||||||||
Warrants exercised (in shares) | 10,250,000 | ||||||||||||
Warrants outstanding (in shares) | 19,750,000 | 19,750,000 | 19,750,000 | 30,000,000 | |||||||||
Common Warrants | Expected term (in years) | |||||||||||||
Warrants | |||||||||||||
Measurement input | Y | 2.7 | 2.7 | 2.7 | ||||||||||
Common Warrants | Volatility | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.898 | 0.898 | 0.898 | ||||||||||
Common Warrants | Risk-free interest rate | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.0492 | 0.0492 | 0.0492 | ||||||||||
Common Warrants | Dividend yield | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0 | 0 | 0 | ||||||||||
June 2022 Offering | |||||||||||||
Warrants | |||||||||||||
Offering price per share and accompanying warrant (in dollars per share) | $ / shares | $ 1 | ||||||||||||
Offering price per pre-funded warrant and accompanying warrant (in dollars per share) | $ / shares | $ 0.9999 | ||||||||||||
June 2022 Offering | Common Stock | |||||||||||||
Warrants | |||||||||||||
Exercise of pre-funded warrants for common stock (in shares) | 4,250,000 | 1,750,000 | |||||||||||
Exercise of common warrants (in shares) | 6,000,000 | 750,000 | 2,500,000 | 1,000,000 | |||||||||
June 2022 Offering | Common And Pre-Funded Warrants | |||||||||||||
Warrants | |||||||||||||
Warrants term | 5 years | ||||||||||||
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants | 9.99% | ||||||||||||
Threshold percentage of aggregate beneficial ownership of holder (together with affiliates) for exercising warrants | 19.99% | ||||||||||||
June 2022 Offering | Common And Pre-Funded Warrants | Expected term (in years) | |||||||||||||
Warrants | |||||||||||||
Measurement input | Y | 3.7 | ||||||||||||
June 2022 Offering | Common And Pre-Funded Warrants | Volatility | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.9153 | ||||||||||||
June 2022 Offering | Common And Pre-Funded Warrants | Risk-free interest rate | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.0411 | ||||||||||||
June 2022 Offering | Common And Pre-Funded Warrants | Dividend yield | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0 | ||||||||||||
June 2022 Offering | Pre-Funded Warrants | |||||||||||||
Warrants | |||||||||||||
Issuance of warrants (in shares) | 10,000,000 | ||||||||||||
Exercise price (in dollars per share) | $ / shares | $ 0.0001 | ||||||||||||
Warrants exercised (in shares) | 4,250,000 | 1,750,000 | |||||||||||
Proceeds from exercise of warrants | $ | $ 425 | $ 175 | |||||||||||
Warrants outstanding (in shares) | 4,000,000 | 4,000,000 | 4,000,000 | ||||||||||
June 2022 Offering | Pre-Funded Warrants | Weighted average | |||||||||||||
Warrants | |||||||||||||
Exercise price (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||||||||||
June 2022 Offering | Pre-Funded Warrants | Average | |||||||||||||
Warrants | |||||||||||||
Warrants term | 5 years | ||||||||||||
June 2022 Offering | Common Warrants | |||||||||||||
Warrants | |||||||||||||
Issuance of warrants (in shares) | 30,000,000 | ||||||||||||
Exercise price (in dollars per share) | $ / shares | $ 1 | ||||||||||||
Warrants exercised (in shares) | 6,000,000 | 750,000 | 2,500,000 | 1,000,000 | |||||||||
Proceeds from exercise of warrants | $ | $ 6,000,000 | $ 800,000 | $ 2,500,000 | $ 1,000,000 | |||||||||
Warrants outstanding (in shares) | 19,750,000 | 19,750,000 | 19,750,000 | ||||||||||
June 2022 Offering | Common Warrants | Expected term (in years) | |||||||||||||
Warrants | |||||||||||||
Measurement input | Y | 2.7 | 2.7 | 2.7 | ||||||||||
June 2022 Offering | Common Warrants | Volatility | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.898 | 0.898 | 0.898 | ||||||||||
June 2022 Offering | Common Warrants | Risk-free interest rate | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0.0492 | 0.0492 | 0.0492 | ||||||||||
June 2022 Offering | Common Warrants | Dividend yield | |||||||||||||
Warrants | |||||||||||||
Measurement input | 0 | 0 | 0 | ||||||||||
June 2022 Offering | Common Warrants | Weighted average | |||||||||||||
Warrants | |||||||||||||
Exercise price (in dollars per share) | $ / shares | $ 1 | $ 1 | $ 1 | ||||||||||
June 2022 Offering | Common Warrants | Average | |||||||||||||
Warrants | |||||||||||||
Warrants term | 5 years |
WARRANTS - Issued with April 20
WARRANTS - Issued with April 2023 Offering (Details) - $ / shares | 9 Months Ended | ||
Apr. 26, 2023 | Sep. 30, 2023 | Dec. 31, 2022 | |
Common And Pre-Funded Warrants | |||
Warrants | |||
Issuance of warrants (in shares) | 22,000,000 | ||
Warrants outstanding (in shares) | 45,875,618 | 38,375,618 | |
Pre-Funded Warrants | |||
Warrants | |||
Issuance of warrants (in shares) | 22,000,000 | ||
Warrants outstanding (in shares) | 26,125,618 | 8,375,618 | |
Common Warrants | |||
Warrants | |||
Warrants outstanding (in shares) | 19,750,000 | 30,000,000 | |
April 2023 Offering, Private Placement | Pre-Funded Warrants | |||
Warrants | |||
Issuance of warrants (in shares) | 22,000,000 | ||
Exercise price (in dollars per share) | $ 0.001 | ||
Offering price (in dollars per share) | $ 0.946 | ||
Threshold percentage of aggregate beneficial ownership of holder for exercising warrants | 9.99% | ||
Warrants outstanding (in shares) | 22,000,000 | ||
April 2023 Offering, Private Placement | Pre-Funded Warrants | Weighted average | |||
Warrants | |||
Exercise price (in dollars per share) | $ 0.001 |
WARRANTS - Warrant Activity (De
WARRANTS - Warrant Activity (Details) | 9 Months Ended |
Sep. 30, 2023 shares | |
Common And Pre-Funded Warrants | |
Warrants | |
Outstanding as of beginning of period (in shares) | 38,375,618 |
Warrants granted and issued (in shares) | 22,000,000 |
Warrants exercised (in shares) | (14,500,000) |
Outstanding as of end of period (in shares) | 45,875,618 |
Pre-Funded Warrants | |
Warrants | |
Outstanding as of beginning of period (in shares) | 8,375,618 |
Warrants granted and issued (in shares) | 22,000,000 |
Warrants exercised (in shares) | (4,250,000) |
Outstanding as of end of period (in shares) | 26,125,618 |
Common Warrants | |
Warrants | |
Outstanding as of beginning of period (in shares) | 30,000,000 |
Warrants exercised (in shares) | (10,250,000) |
Outstanding as of end of period (in shares) | 19,750,000 |
LEASES - Lease Cost (Details)
LEASES - Lease Cost (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Leases | ||||
Operating Lease Cost | $ 126 | $ 126 | $ 378 | $ 378 |
General and administrative | ||||
Leases | ||||
Operating Lease Cost | $ 126 | $ 126 | $ 378 | $ 378 |
LEASES - Lease terms and discou
LEASES - Lease terms and discount rates (Details) | Sep. 30, 2023 | Sep. 30, 2022 |
LEASES | ||
Weighted-average remaining lease term - operating leases | 1 year 1 month 6 days | 2 years 1 month 6 days |
Weighted-average discount rate - operating leases | 5.69% | 5.69% |
LEASES - Maturities of Lease Li
LEASES - Maturities of Lease Liabilities (Details) $ in Thousands | Sep. 30, 2023 USD ($) |
LEASES | |
2023 | $ 126 |
2024 | 426 |
Total lease payments | 552 |
Less: interest | 17 |
Total lease liabilities | $ 535 |
Operating Lease, Liability, Statement of Financial Position | Current portion of operating lease liabilities, Noncurrent portion of operating lease liabilities |
BENEFIT PLANS (Details)
BENEFIT PLANS (Details) - USD ($) $ in Millions | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
BENEFIT PLANS | ||||
Employer matching contributions to the plan | $ 0.1 | $ 0.1 | $ 0.3 | $ 0.4 |
NET LOSS PER COMMON SHARE - EPS
NET LOSS PER COMMON SHARE - EPS (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Numerator: | ||||
Net loss attributable to common stockholders-basic | $ (42,370) | $ (19,101) | $ (82,084) | $ (68,079) |
Net loss attributable to common stockholders-diluted | $ (42,370) | $ (19,101) | $ (82,084) | $ (68,079) |
Denominator: | ||||
Weighted-average common stock outstanding-basic (in shares) | 90,669,969 | 48,000,183 | 75,482,234 | 33,785,386 |
Weighted-average common stock outstanding-diluted (in shares) | 90,669,969 | 48,000,183 | 75,482,234 | 33,785,386 |
Net loss per share attributable to common stockholders-basic (in dollars per share) | $ (0.47) | $ (0.40) | $ (1.09) | $ (2.02) |
Net loss per share attributable to common stockholders-diluted (in dollars per share) | $ (0.47) | $ (0.40) | $ (1.09) | $ (2.02) |
NET LOSS PER COMMON SHARE - Ant
NET LOSS PER COMMON SHARE - Anti-dilutive securities (Details) - shares | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Options | ||
Antidilutive securities excluded from computation of loss per share | ||
Potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders | 4,695,619 | 4,925,678 |
Restricted stock units | ||
Antidilutive securities excluded from computation of loss per share | ||
Potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders | 3,326,336 | 533,114 |
Warrants to purchase common stock | ||
Antidilutive securities excluded from computation of loss per share | ||
Potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders | 19,875,618 | 38,375,618 |
RELATED PARTIES (Details)
RELATED PARTIES (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Related Party Transactions | |||||
Research and development | $ 10,785 | $ 13,116 | $ 38,602 | $ 43,542 | |
Due to related parties | 56,239 | 56,239 | $ 34,302 | ||
LaunchLabs Agreement | Subsidiary whose founder and executive chairman of parent is a member of the Company's board of directors | Alexandria LaunchLabs | |||||
Related Party Transactions | |||||
Research and development | 24 | $ 23 | 100 | $ 70 | |
Due to related parties | $ 7 | $ 7 | $ 0 |
REVENUE (Details)
REVENUE (Details) $ in Thousands, € in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended | ||||
Jan. 03, 2023 USD ($) item | Jan. 03, 2023 EUR (€) item | Jan. 31, 2023 USD ($) | Sep. 30, 2023 EUR (€) | Sep. 30, 2023 USD ($) | Sep. 30, 2023 EUR (€) | Jan. 03, 2023 EUR (€) | |
Revenue | |||||||
License Revenue | $ | $ 10,660 | ||||||
Exclusive License and Supply Agreement | ADVANZ PHARMA | |||||||
Revenue | |||||||
Upfront payment received | $ 10,700 | € 10 | $ 10,700 | ||||
Potential milestone payments to be received | $ 142,200 | € 134 | |||||
Royalties as a percent of sales | 20% | 20% | |||||
Initial term | 10 years | 10 years | |||||
Royalties as a percent of sales following initial term | 10% | 10% | |||||
Number of performance obligations | item | 2 | 2 | |||||
License Revenue | € | € 10 | € 10 |
SUBSEQUENT EVENTS (Details)
SUBSEQUENT EVENTS (Details) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | |||||
Oct. 13, 2023 | Oct. 12, 2023 | Sep. 30, 2023 | Jun. 30, 2022 | Dec. 31, 2022 | Sep. 30, 2022 | |
Subsequent event | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | ||||
Common Stock | ||||||
Subsequent event | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | ||||
Issuance of common stock (in shares) | 4,284,344 | 20,000,000 | ||||
Leerink ATM Agreement | ||||||
Subsequent event | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | |||||
Proceeds from stock issuance, net of cash issuance costs | $ 7 | |||||
Leerink ATM Agreement | Common Stock | ||||||
Subsequent event | ||||||
Issuance of common stock (in shares) | 4,284,344 | |||||
Purchase price (in dollars per share) | $ 1.75 | |||||
Subsequent Event | ||||||
Subsequent event | ||||||
Common Stock, par value (in dollars per share) | $ 0.001 | |||||
Subsequent Event | Pre-Funded Warrants | ||||||
Subsequent event | ||||||
Warrants exchanged for shares of common stock (in shares) | 5,658,034 | |||||
Subsequent Event | Common Stock | ||||||
Subsequent event | ||||||
Stock received in exchange for warrants (in shares) | 5,658,034 | |||||
Subsequent Event | Leerink ATM Agreement | ||||||
Subsequent event | ||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | |||||
Proceeds from stock issuance, net of cash issuance costs | $ 12.7 | |||||
Subsequent Event | Leerink ATM Agreement | Common Stock | ||||||
Subsequent event | ||||||
Issuance of common stock (in shares) | 5,750,000 | |||||
Purchase price (in dollars per share) | $ 2.28 |