Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2022 | Mar. 22, 2023 | Jun. 30, 2022 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2022 | ||
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 Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
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 | ||
ICFR Auditor Attestation Flag | false | ||
Entity Shell Company | false | ||
Entity Public Float | $ 27,814,183 | ||
Entity Common Stock, Shares Outstanding | 48,113,561 | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Location | New York, New York | ||
Auditor Firm ID | 42 | ||
Entity Central Index Key | 0001697532 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Fiscal Year Focus | 2022 | ||
Document Fiscal Period Focus | FY | ||
Amendment Flag | false |
Balance Sheets
Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 16,657 | $ 53,888 |
Investments | 13,923 | 26,935 |
Prepaid expenses and other current assets | 6,728 | 7,571 |
Total current assets | 37,308 | 88,394 |
Operating lease right-of-use asset | 857 | 1,298 |
Security deposits and leasehold improvements | 198 | 200 |
TOTAL ASSETS | 38,363 | 89,892 |
CURRENT LIABILITIES: | ||
Current portion of operating lease liabilities | 477 | 442 |
Accounts payable | 4,534 | 9,461 |
Accrued expenses and other current liabilities | 14,756 | 16,559 |
Warrant liability | 13,657 | |
Total current liabilities | 33,424 | 26,462 |
NONCURRENT LIABILITIES: | ||
Noncurrent portion of operating lease liabilities | 414 | 891 |
Clinical holdback - long-term portion | 464 | |
Total noncurrent liabilities | 878 | 891 |
Total liabilities | 34,302 | 27,353 |
STOCKHOLDERS' EQUITY: | ||
Common stock, $0.0001 par value; 200,000,000 shares authorized as of December 31, 2022 and 100,000,000 shares authorized as of December 31, 2021; 48,063,358 shares issued and outstanding as of December 31, 2022 and 26,215,514 shares issued and outstanding as of December 31, 2021 | 5 | 3 |
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of December 31, 2022 and December 31, 2021; 0 shares issued and outstanding as of December 31, 2022 and December 31, 2021 | ||
Additional paid-in capital | 352,828 | 328,958 |
Accumulated other comprehensive gain/(loss) | 51 | (107) |
Accumulated deficit | (348,823) | (266,315) |
Total stockholders' equity | 4,061 | 62,539 |
TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY | $ 38,363 | $ 89,892 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2022 | Dec. 31, 2021 |
Balance Sheets | ||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common Stock, authorized (in shares) | 200,000,000 | 100,000,000 |
Common Stock, issued (in shares) | 48,063,358 | 26,215,514 |
Common stock, outstanding (in shares) | 48,063,358 | 26,215,514 |
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 |
Statements of Operations
Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
OPERATING EXPENSES: | ||
Research and development | $ 55,634 | $ 62,570 |
General and administrative | 27,316 | 43,048 |
Total operating expenses | 82,950 | 105,618 |
LOSS FROM OPERATIONS | (82,950) | (105,618) |
OTHER INCOME (EXPENSE), NET: | ||
Interest income | 685 | 555 |
Change in fair value of warrant liabilities | (66) | |
Other expense | (177) | (521) |
Total other income, net | 442 | 34 |
Net loss | (82,508) | (105,584) |
Net loss attributable to common stockholders-basic | (82,508) | (105,584) |
Net loss attributable to common stockholders-diluted | $ (82,508) | $ (105,584) |
Net loss per share attributable to common stockholders-basic (in dollars per share) | $ (2.18) | $ (4.12) |
Net loss per share attributable to common stockholders-diluted (in dollars per share) | $ (2.18) | $ (4.12) |
Weighted-average common stock outstanding-basic (in shares) | 37,825,431 | 25,598,181 |
Weighted-average common stock outstanding-diluted (in shares) | 37,825,431 | 25,598,181 |
Statements of Comprehensive Inc
Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Statements of Comprehensive Income (Loss) | ||
Net Loss | $ (82,508) | $ (105,584) |
Other comprehensive income (loss) | ||
Unrealized gain (loss) on marketable securities | 158 | 5 |
Other comprehensive gain (loss), net of tax | 158 | 5 |
Comprehensive income (loss), net of tax | $ (82,350) | $ (105,579) |
Statements of Stockholders' Equ
Statements of Stockholders' Equity - USD ($) $ in Thousands | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) | Total |
Balance at Dec. 31, 2020 | $ 2 | $ 242,780 | $ (160,731) | $ (112) | $ 81,939 |
Balance (in shares) at Dec. 31, 2020 | 22,493,661 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock upon secondary public offering, net of issuance costs | $ 1 | 74,421 | 74,422 | ||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 3,450,000 | ||||
Exercise of options for common stock issued under Equity Incentive Plan | 511 | 511 | |||
Exercise of options for common stock issued under Equity Incentive Plan (in shares) | 191,436 | ||||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 52,562 | ||||
Exercise of warrants for common stock | 69 | 69 | |||
Exercise of warrants for common stock (in shares) | 27,855 | ||||
Stock-based compensation expense | 11,177 | 11,177 | |||
Net loss | (105,584) | (105,584) | |||
Other comprehensive income (loss) | 5 | 5 | |||
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 | |||||
Issuance of common stock, pre-funded warrants, and common warrants sold for cash, net of issuance costs | $ 2 | 12,801 | 12,803 | ||
Issuance of common stock, pre-funded warrants, and common warrants 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 prefunded warrants for common stock (in shares) | 1,750,000 | ||||
Exercise of options for common stock issued under Equity Incentive Plan | 49 | $ 49 | |||
Exercise of options for common stock issued under Equity Incentive Plan (in shares) | 47,602 | 47,602 | |||
Restricted Stock Units released for common stock issued under Equity Incentive Plan (in shares) | 50,242 | ||||
Stock-based compensation expense | 9,162 | $ 9,162 | |||
Issuance of options in-lieu of bonus | 441 | 441 | |||
Net loss | (82,508) | (82,508) | |||
Other comprehensive income (loss) | 158 | 158 | |||
Balance at Dec. 31, 2022 | $ 5 | $ 352,828 | $ (348,823) | $ 51 | $ 4,061 |
Balance (in shares) at Dec. 31, 2022 | 48,063,358 |
Statements of Stockholders' E_2
Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Increase (Decrease) in Stockholders' Equity | ||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Stock issuance costs | $ 96 | $ 167 |
Common Stock | ||
Increase (Decrease) in Stockholders' Equity | ||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Statements of Cash Flows
Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
OPERATING ACTIVITIES: | ||
Net loss | $ (82,508) | $ (105,584) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 9,162 | 11,177 |
Issuance of options in-lieu of bonus | 441 | |
Amortization of insurance premium | 3,589 | 4,282 |
Amortization of operating lease right-of-use assets | 441 | 415 |
Amortization of leasehold improvements | 2 | |
Change in operating lease liability | (442) | (404) |
Change in fair value of warrant liability | 66 | |
Changes in operating assets and liabilities: | ||
Financed insurance premium | (3,105) | (4,435) |
Prepaid expenses | 359 | (1,654) |
Accounts payable | (4,927) | 8,821 |
Accrued expenses and other current liabilities | (1,635) | (3,346) |
Other liabilities | 464 | |
Net cash used in operating activities | (78,093) | (90,728) |
INVESTING ACTIVITIES: | ||
Purchase of available-for-sale securities | (64,218) | (121,589) |
Proceeds from sale of available-for-sale securities | 5,538 | |
Proceeds from maturities of available-for-sale securities | 77,388 | 128,484 |
Net cash provided by investing activities | 13,170 | 12,433 |
FINANCING ACTIVITIES: | ||
Proceeds from June Offering issuance of shares and pre-funded warrants | 27,811 | |
Proceeds from February Offering, net of cash issuance costs of $169 | 74,421 | |
Proceeds from financed insurance premium | 3,105 | 4,434 |
Repayments of short-term borrowings | (3,273) | (4,718) |
Exercise of stock options for common stock under Equity Incentive Plan | 49 | 511 |
Exercise of Warrants | 69 | |
Net cash provided by financing activities | 27,692 | 74,717 |
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS | (37,231) | (3,578) |
Cash and cash equivalents at beginning of period | 53,888 | 57,466 |
Cash and cash equivalents at end of period | 16,657 | 53,888 |
SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION: | ||
Initial measurement of warrant liability | 21,835 | |
Conversion of warrant liability to equity for warrant exercises | 1,417 | |
Reclassification of prefunded warrant liability to equity | 6,827 | |
Unrealized gain (loss) on marketable securities | $ 158 | $ 5 |
Statements of Cash Flows (Paren
Statements of Cash Flows (Parenthetical) $ in Thousands | 12 Months Ended |
Dec. 31, 2022 USD ($) | |
Statements of Cash Flows | |
Cash issuance costs, February Offering | $ 169 |
ORGANIZATION AND SUMMARY OF SIG
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 12 Months Ended |
Dec. 31, 2022 | |
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 CNS rare diseases and diabetic complications. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York. On January 28, 2020, the Company completed its secondary public offering (the “Secondary Public Offering”), pursuant to which it issued and sold 2,741,489 shares of common stock at a public offering price of $45.50 per share, with an additional 411,223 shares sold pursuant to the underwriters’ full exercise of their option to purchase additional shares. The aggregate net proceeds received by the Company from the offering, after deducting underwriting discounts and commissions and offering costs, were $134.1 million. On June 4, 2020, the Company filed a shelf registration statement on Form S-3 (the “Shelf Registration Statement”) under which the Company may, from time to time, sell securities in one or more offerings having an aggregate offering price of up to $300.0 million. The Shelf Registration Statement was declared effective as of June 15, 2020. As of the filing date of this Annual Report, the Company has sold 3,450,000 shares of common stock under the Shelf Registration. On June 12, 2020, the Company entered into an equity distribution agreement (the “Goldman Equity Distribution Agreement”) with Goldman Sachs & Co. LLC (“Goldman”), 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 million. Goldman may act as an agent on the Company’s behalf or purchase shares of the Company’s common stock as a principal. As of December 31, 2021, the Company had On February 17, 2021, the Company completed an underwritten public offering (the “February Offering”) of 3,450,000 shares of common stock, including the exercise in full of the underwriters’ option to purchase 450,000 additional shares of common stock, which option closed on February 19, 2021. The shares were offered at a price to the public of per share, resulting in aggregate net proceeds of approximately , after deducting underwriting discounts and commissions and offering expenses. On January 26, 2022, the Company entered into an equity distribution agreement (the “Cowen 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 Cowen 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 Cowen 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 Cowen 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. As of December 31, 2022, the Company has not sold any shares of common stock pursuant to the Cowen Equity Distribution Agreement. 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. 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 revenue, and it does not expect to generate 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. We are actively pursuing several potential financing options. While we continue to explore opportunities to raise additional equity capital in the public markets, this has proven to be challenging in the biotech sector recently. Other options for structured finance which we continue to explore include a PIPE (private investment in public equity), debt, convertible debt, and synthetic royalty financing. Synthetic royalty financing, in particular, has become a favorable option for many companies for funding ongoing clinical development in late-stage and pre-approval programs. We have engaged an investment bank and we are specifically exploring this option in the near term. Additionally, we are in active dialogue with several potential partners regarding business development opportunities related to one or more of our programs. There can be no assurances that our 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 $82.5 million for the year ended December 31, 2022 and has an accumulated deficit of $348.8 million as of December 31, 2022. 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 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. 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 liability 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 Fair Value Measurements Certain assets and liabilities are reported on a recurring basis at fair value. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: Level 1—Quoted prices in active markets for identical assets or liabilities. Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. Level 3—Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. Cash and Cash Equivalents The Company considers all short-term, highly liquid investments, with an original maturity of three months or less, to be cash equivalents. The Company maintains its cash in bank deposit accounts which, at times, may exceed federally insured limits. The Company has not experienced any losses in these accounts and does not believe it is exposed to any significant credit risk on cash and cash equivalents. Investments The Company has investments in marketable debt securities. The Company determines the appropriate classification of its investments at the date of purchase and reevaluates the classifications at the balance sheet date. Marketable debt securities with maturities of 12 months or less are classified as short-term. Marketable debt securities with maturities greater than 12 months are classified as long-term. The Company’s marketable securities are accounted for as available for sale (“AFS”). AFS securities are reported at fair value. Unrealized gains and losses, after applicable income taxes, are reported in accumulated other comprehensive income/(loss). Realized gains or losses on the sale of marketable securities are determined using the specific identification method and are recorded as a component of other income (expense), net. The Company conducts an other-than-temporary impairment (“OTTI”) analysis on a quarterly basis or more often if a potential loss-triggering event occurs. The Company considers factors such as the duration, severity and the reason for the decline in value, the potential recovery period and whether the Company intends to sell. For AFS securities, the Company also considers whether (i) it is more likely than not that the Company will be required to sell the debt securities before recovery of their amortized cost basis and (ii) the amortized cost basis cannot be recovered as a result of credit losses. Leases At the inception of an arrangement, the Company determines if an arrangement is, or contains, a lease based on the unique facts and circumstances present in that arrangement. Lease classification, recognition, and measurement are then determined at the lease commencement date. For arrangements that contain a lease the Company (i) identifies lease and non-lease components, (ii) determines the consideration in the contract, (iii) determines whether the lease is an operating or financing lease; and (iv) recognizes lease right-of-use (“ROU”) assets and liabilities. Lease liabilities and their corresponding ROU assets are recorded based on the present value of lease payments over the expected lease term. The interest rate implicit in lease contracts is typically not readily determinable and as such, the Company uses its incremental borrowing rate based on the information available at the lease commencement date, which represents an internally developed rate that would be incurred to borrow, on a collateralized basis, over a similar term, an amount equal to the lease payments in a similar economic environment. Most leases include options to renew and, or, terminate the lease, which can impact the lease term. The exercise of these options is at the Company’s discretion and the Company does not include any of these options within the expected lease term as the Company is not reasonably certain it will exercise these options. The Company has elected to combine lease components (for example fixed payments including rent) with non-lease components (for example, non-dedicated parking and common-area maintenance costs) on our real estate asset classes. Fixed, or in substance fixed, lease payments on operating leases are recognized over the expected term of the lease on a straight-line basis. Fixed lease expense on operating leases is recognized within operating expenses within the statements of operations. The Company has operating leases for our corporate offices. The Company has elected the short-term lease exemption and, therefore, do not recognize a ROU asset or corresponding liability for lease arrangements with an original term of 12 months or less. Leasehold improvements and assets under financing lease arrangements are amortized over the lesser of the asset’s estimated useful life or the term of the respective lease. Maintenance costs are expensed as incurred. Operating leases are included in operating lease right-of-use asset, current portion of operating lease liabilities, and noncurrent portion of operating lease liabilities in the balance sheet as of December 31, 2022 and 2021. 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 year ended December 31, 2022: (in thousands) Balance as of December 31,2021 $ — Clinical holdback retained 464 Clinical holdback paid — Balance as of December 31,2022 $ 464 Less: clinical holdback current portion — Clinical holdback - long-term portion $ 464 There was no clinical holdback during the year ended December 31, 2021. Deferred Offering Costs The Company capitalizes certain legal, professional accounting and other third party fees that are directly associated with in-process equity financings as deferred offering costs until such financings are consummated. After consummation of the equity financing, these costs are recorded in the statement of stockholders’ (deficit) equity as a reduction of proceeds generated as a result of the offering. Should a planned equity financing be abandoned, the deferred offering costs would be expensed immediately as a charge to operating expenses in the statement of operations. As of December 31, 2022 and 2021, the Company did not have any deferred offering costs recorded in prepaid and other current assets. Research and Development The Company expenses all costs incurred in performing research and development activities. Research and development expenses include salaries and other related costs, materials and supplies, preclinical expenses, manufacturing expenses, contract services and other outside expenses. As part of the process of preparing the financial statements, the Company is required to estimate their accrued research and development expenses. The Company makes estimates of the accrued expenses as of each balance sheet date in the financial statements based on facts and circumstances known at that time. In addition, there may be instances in which payments made to the Company’s vendors will exceed the level of services provided and result in a prepayment of the expense in which case such amounts are reflected as prepaid expenses and other current assets. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual or the amount of prepaid expenses accordingly. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized in prepaid expenses and other current assets. The capitalized amounts are expensed as the related goods are delivered or the services are performed. Research and development costs also include costs incurred in connection with certain licensing arrangements. Before a compound receives regulatory approval, the Company records upfront and milestone payments made to third parties under licensing arrangements as expense. Upfront payments are recorded when incurred, and milestone payments are recorded when the specific milestone has been achieved. Once a compound receives regulatory approval, the Company will record any milestone payments in identifiable intangible assets, less accumulated amortization and, unless the asset is determined to have an indefinite life, the Company will amortize the payments on a straight-line basis over the remaining agreement term or the expected product life cycle, whichever is shorter. General and Administrative General and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, for personnel in the Company’s executive and finance functions. General and administrative expenses also include professional fees for legal, accounting, auditing, tax and consulting services; travel expenses; and facility-related expenses, which include allocated expenses for rent and maintenance of facilities and other operating costs. Commercial expenses consist of payroll expense for commercial personnel, as well as marketing, market research, market access, and other focused investments to support launch of drug candidates, generate evidence of commercial potential and value proposition, and maximize potential business development deal leverage. Commercial expenses are included in general and administrative expenses. Stock-Based Compensation The Company accounts for its stock-based compensation as expense in the statements of operations based on the awards’ grant date fair values. The Company accounts for forfeitures as they occur by reversing any expense recognized for unvested awards. The Company estimates the fair value of options granted using the Black-Scholes option pricing model. The Black-Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the award, (c) the risk-free interest rate and (d) expected dividends. Due to the lack of a public market for the Company’s common stock and a lack of company-specific historical and implied volatility data, the Company has based its estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded. The historical volatility is calculated based on a period of time commensurate with the expected term assumption. The computation of expected volatility is based on the historical volatility of a representative group of companies with similar characteristics to the Company, including stage of product development and life science industry focus. The Company uses the simplified method as allowed by the Securities and Exchange Commission (“SEC”) Staff Accounting Bulletin (“SAB”) No. 107, Share-Based Payment, to calculate the expected term for options granted to employees as it does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. The risk-free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. The expected dividend yield is assumed to be zero as the Company has never paid dividends and has no current plans to pay any dividends on its common stock. The fair value of stock-based payments is recognized as expense over the requisite service period which is generally the vesting period. Stock-Based Compensation–Restricted Stock Units The Company accounts for restricted stock units in accordance with the authoritative guidance for stock-based compensation. The fair value of restricted stock units is measured at the grant date based on the closing market price of the Company’s common stock on the date of grant, and is recognized as expense on a straight-line basis over the period of vesting. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur. Income Taxes The Company uses the asset and liability method of accounting for deferred income taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities at currently enacted tax rates. These temporary differences primarily relate to net operating loss carryforwards available to offset future taxable income. Valuation allowances are established, if necessary, to reduce a deferred tax asset to the amount that will more likely than not be realized. The Company recognizes tax liabilities from an uncertain tax position only if it is more likely than not that the tax position will not be sustained upon examination by the taxing authorities, based on the technical merits of the tax position. There are no uncertain tax positions that have been recognized in the accompanying financial statements. The Company is required to file tax returns in the U.S. federal jurisdiction and in the states of New York, California, Massachusetts, New Jersey, and New York City. The Company’s policy is to recognize interest and penalties related to uncertain tax benefits, if any, as part of income tax expense. No such interest and penalties have been accrued as of December 31, 2022 and 2021. In December 2019, the FASB issued ASU 2019-12, Income Taxes: Simplifying the Accounting for Income Taxes. Net Loss per Share Basic net loss per share is calculated by dividing net loss available to common stockholders by the weighted-average common stock outstanding. Diluted net loss per share is calculated similarly, except that it includes the dilutive effect of the assumed exercise of securities, including outstanding warrants and the effect of shares issuable under the Company’s stock-based compensation plan, if such effect is dilutive. Segment Information Operating segments are defined as components of an enterprise for which separate discrete information is available for evaluation by the chief operating decision-maker in deciding how to allocate resources and assess performance. The Company and the Company’s chief operating decision-maker, the Company’s chief executive officer, views the Company’s operations and manages its business as a single operating segment, which is the business of discovering and developing its product candidates. 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 | 12 Months Ended |
Dec. 31, 2022 | |
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 years ended December 31, 2022 and 2021, the Company recorded $0 and $0 in research and development expense, respectively, and $0.2 million and $0.2 million, respectively, in general and administrative expense related to the Columbia Agreements. In aggregate, the Company has incurred $2.7 million in expense from the execution of the Columbia Agreements through December 31, 2022. As of December 31, 2022, the Company had $0.1 million due to Columbia University included in accrued expenses and $0 included in accounts payable. As of December 31, 2021, the Company had $12,000 due to Columbia University included in accrued expenses and $0.1 million included in accounts payable. 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. The Company recorded $0.1 million and $0 in research and development expense and general and administrative expense, respectively, during the year-ended December 31, 2022. As of December 31, 2022, the Company had $0.3 million in accrued expenses. The Company recorded $0.03 million and $0 in research and development expense and general and administrative expense, respectively, during the year-ended December 31, 2021. As of December 31, 2021, the Company had $0.3 million in accrued expenses. 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 was from December 14, 2020 through December 30, 2021, and was extended through August 31, 2022. The total consideration for the 2020 Miami Research Agreement was $0.3 million. During the years ended December 31, 2022 and 2021, the Company recorded $48,000 and $0.2 million in research and development expenses, respectively, in relation to the 2020 Miami Research Agreement. As of December 31, 2022 and 2021, the Company had $1.0 million and $0.2 million, respectively, in accrued expenses. 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 | 12 Months Ended |
Dec. 31, 2022 | |
FAIR VALUE MEASUREMENTS | |
FAIR VALUE MEASUREMENTS | 3. FAIR VALUE MEASUREMENTS The following tables summarize, as of December 31, 2022 and 2021, 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 section. 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 As of December 31, 2021 (in thousands) Level 1 Level 2 Level 3 Total Cash $ 12,671 $ — $ — $ 12,671 Money market funds 41,217 — — 41,217 Total cash and cash equivalents $ 53,888 $ — $ — $ 53,888 U.S. government agency debt securities — 26,935 — 26,935 Total marketable securities $ — $ 26,935 $ — $ 26,935 Total financial assets measured at fair value on a recurring basis $ 53,888 $ 26,935 $ — $ 80,823 Investments in certificate of deposits, corporate bonds, and U.S. government agency debt securities Fair values of corporate bonds and 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 These Common Warrant liabilities were measured at fair value at inception and are then subsequently measured 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 consolidated statement of operations. The Common and Pre-Funded Warrants were initially valued and remeasured using a Black-Scholes option pricing model with a range of assumptions as follows: Expected term (in years) 3.7 Volatility 91.53 % Risk-free interest rate 4.11 % Dividend yield 0.00 % 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% probability of change in control was used for each of the five years in the term of the agreements. The following table provides a roll forward of the aggregate fair values of the Company’s warrant liability, for which fair value is determined using Level 3 inputs (in thousands): Warrant Liability Common Warrant Pre-Funded Warrant Balance as of January 1, 2022 $ — $ — Initial fair value of Warrant Liability 13,734 8,101 Warrants exercised — (1,417) Change in fair value (77) 143 Reclassification of pre-funded warrant liability to equity (6,827) Balance as of December 31, 2022 $ 13,657 $ — The inputs utilized by management to value the warrant liability for Common and Pre-Funded Warrants are highly subjective. The assumptions used in calculating the fair value of the warrant liability for Common and Pre-Funded 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 warrant liability for Common Warrants may be materially different in the future. |
INVESTMENTS
INVESTMENTS | 12 Months Ended |
Dec. 31, 2022 | |
INVESTMENTS | |
INVESTMENTS | 4. INVESTMENTS Marketable Securities Marketable securities, which the Company classifies as available-for-sale securities, primarily consist of high quality commercial paper, corporate bonds, and 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 (in thousands): As of December 31, 2022 As of December 31, 2021 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 $ 27,042 $ 1 $ (108) $ 26,935 Total $ 13,873 $ 50 $ — $ 13,923 $ 27,042 $ 1 $ (108) $ 26,935 As of December 31, 2022, the Company’s investment portfolio reported no unrealized loss. Based on its evaluations, the Company determined that a credit loss allowance is not required since the decline was not related to underlying credit issues of the counterparties. The counterparties to these investments have high credit quality with investment grade ratings of at least AA+ or above, along with a history of no defaults. No single investment in the portfolio had an individually material unrealized loss and in the aggregate. In addition, the Company does not intend to sell these investments and it is not more likely than not that the Company will be required to sell these investments before recovery of their amortized cost bases. Accordingly, based on the foregoing evaluation, the Company did not record any credit losses during the year ended December 31, 2022 and 2021. Unrealized gains are also reflected, net of tax, as other comprehensive income (loss) in the Statements of Comprehensive Loss. Contractual maturities of the Company’s marketable securities are summarized as follows (in thousands): As of December 31, 2022 As of December 31, 2021 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 $ 27,042 $ 1 $ (108) $ 26,935 Due in one through two years — — — — — — — — Total $ 13,873 $ 50 $ — $ 13,923 $ 27,042 $ 1 $ (108) $ 26,935 At December 31, 2022, the Company had $50,000 of gross unrealized gains and $0 of gross unrealized losses primarily due to fluctuations in the fair value of certain U.S. government agency debt securities. During the year ended December 31, 2022, the Company recorded gross realized losses of $0.2 million and gross realized gains of $16,000 from the redemption of marketable securities. The 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 December 31, 2022 are as follows: As of December 31, 2022 Securities in an unrealized loss position less than 12 months Securities in an unrealized loss position greater than 12 months Total Unrealized Estimated Unrealized Estimated Unrealized Estimated (in thousands) Losses Fair Value Losses Fair Value Losses Fair Value US government agency debt security $ — $ — $ — $ — $ — $ — Total $ — $ — $ — $ — $ — $ — The 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 December 31, 2021 are as follows: As of December 31, 2021 Securities in an unrealized loss position less than 12 months Securities in an unrealized loss position greater than 12 months Total Unrealized Estimated Unrealized Estimated Unrealized Estimated (in thousands) Losses Fair Value Losses Fair Value Losses Fair Value US government agency debt security $ (108) $ 17,696 $ — $ — $ (108) $ 17,696 Total $ (108) $ 17,696 $ — $ — $ (108) $ 17,696 |
PREPAID EXPENSES AND OTHER CURR
PREPAID EXPENSES AND OTHER CURRENT ASSETS | 12 Months Ended |
Dec. 31, 2022 | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | 5. PREPAID EXPENSE AND OTHER CURRENT ASSETS Prepaid expenses and other current assets consisted of the following (in thousands): December 31, December 31, (in thousands) 2022 2021 Prepaid research and development expenses $ 4,272 $ 4,483 Insurance premium asset 1,131 1,616 Prepaid rent expenses 99 117 Prepaid insurance expenses 71 105 Prepaid commercial and patient advocacy 206 254 Research and development tax credit receivable 252 502 Interest receivable 23 23 Other prepaid expenses and current assets 674 471 Total prepaid expenses & other current assets $ 6,728 $ 7,571 |
ACCRUED EXPENSES AND OTHER CURR
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | 12 Months Ended |
Dec. 31, 2022 | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | 6. ACCRUED EXPENSE AND OTHER CURRENT LIABILITIES Accrued expenses and other current liabilities consisted of the following (in thousands): December 31, December 31, (in thousands) 2022 2021 Accrued pre-clinical and clinical expenses $ 8,877 $ 9,912 Short-term insurance financing note 622 789 Accrued professional fees 1,218 419 Accrued compensation and benefits 2,301 2,779 Accrued commercial expenses 896 1,394 Accrued patent expenses 361 279 Other 481 987 Total accrued expenses & other current liabilities $ 14,756 $ 16,559 |
STOCK BASED COMPENSATION
STOCK BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2022 | |
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 is 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 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 December 31, 2022, there were 1,096,493 shares of common stock available for issuance under the 2019 Plan. On January 1, 2023, there were 2,403,167 shares that became issuable for future grants subject to the adjustments provided in the 2019 Plan. Stock-Based Compensation Expense Total stock-based compensation expense recorded for employees, directors and non-employees (in thousands): Year Ended December 31, (in thousands) 2022 2021 Research and development $ 3,619 $ 2,759 General and administrative 5,543 8,418 Total stock-based compensation expense $ 9,162 $ 11,177 Stock Option Activity During the year ended December 31, 2022, and 2021 the Company granted options to purchase 767,913 and 406,752 shares of common stock, respectively. For the years ended December 31, 2022, and 2021, amortization of stock compensation of options amounted to $7.8 million and $9.3 million, respectively. As of December 31, 2022, the total unrecognized stock-based compensation balance for unvested options was $8.1 million, which is expected to be recognized over 2.2 years. The weighted-average fair value of options granted during the years ended December 31, 2022, and December 31, 2021, were $1.79 per share and $8.43 per share, respectively. The following table summarizes the information about options outstanding at December 31, 2022: 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, 2021 4,704,888 $ 13.29 7.7 $ 10,305 Options granted 767,913 1.39 Options exercised (47,602) 1.04 21 Forfeited (377,378) 19.90 Expired (173,774) 26.82 Outstanding at December 31, 2022 4,874,047 $ 1.97 6.9 $ — Exercisable at December 31, 2022 3,859,880 $ 2.21 6.6 $ — Nonvested at December 31, 2022 1,014,167 $ 1.05 8.3 $ — 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 years ended December 31, 2022 and 2021. Year Ended December 31, 2022 2021 Expected term (in years) 5.7 5.8 Volatility 75.83 % 71.92 % Risk-free interest rate 2.13 % 1.08 % Dividend yield — % — % Restricted Stock Unit Activity During the year ended December 31, 2022, the Company granted 605,600 restricted stock units (“RSUs”). For the years ended December 31, 2022 and December 31, 20201 amortization of stock compensation of RSUs amounted to $1.3 million and $1.9 million, respectively. As of December 31, 2022, the unamortized compensation costs associated with non-vested restricted stock awards was $3.5 million with a weighted-average remaining amortization period of 3.2 years. The following table summarizes the information about restricted stock units outstanding at December 31, 2022: Weighted-Average Grant Date Aggregate (in thousands, except for share data) Shares Fair Value Intrinsic Value Outstanding at December 31, 2021 469,485 $ 18.05 $ 4,202 Awarded 605,600 2.33 Released (50,242) 24.57 Forfeited (209,334) 18.10 Outstanding at December 31, 2022 815,509 $ 5.96 $ 620 Nonvested at December 31, 2022 815,509 $ 5.73 $ 582 Weighted Average Remaining Recognition Period (in years) 3.2 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 December 31, 2022, 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 | 12 Months Ended |
Dec. 31, 2022 | |
STOCKHOLDERS' EQUITY | |
STOCKHOLDERS' EQUITY | 8. STOCKHOLDERS’ EQUITY As of December 31, 2022, the authorized capital stock of the Company consists 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. As of December 31, 2021, the authorized capital stock of the Company consists of 100,000,000 shares of common stock, par value $0.0001 per share and 10,000,000 shares of preferred stock, par value $0.0001 per share. Common Stock Voting The holders of the common stock are entitled to one vote for each share of common stock held at all meetings of the stockholders. There is no cumulative voting. Goldman Equity Distribution Agreement In June 2020, the Company entered into the Goldman 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. 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 Cowen 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 Cowen 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 Cowen 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 Cowen 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. As of December 31, 2022, the Company has not sold any shares of common stock pursuant to the Cowen Equity Distribution Agreement. 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. |
WARRANTS
WARRANTS | 12 Months Ended |
Dec. 31, 2022 | |
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 is 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 % On December 5, 2019, an optionholder exercised 20,000 options in a cashless exercise at net, and the Company issued 17,026 shares of common stock. On February 21, 2020, two warrantholders exercised 185,634 options in a cashless exercise at net, and the Company issued 176,092 shares of common stock. On February 24, 2020, a warrantholder exercised 72,818 options in a cashless exercise at net, and the Company issued 69,094 shares of common stock. On February 5, 2021, a warrantholder exercised 27,855 warrants on a cash basis and received 27,855 shares of common stock. The Company received $69,000 in cash proceeds for the exercise of these warrants. 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 the Company’s 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 2018 Notes Warrants vested immediately upon issuance and have an exercise price of $6.59 per share and expire on November 4, 2028. On February 24, 2020, a warrantholder exercised 386 options in a cashless exercise at net, and the Company issued 333 shares of common stock. On June 24, 2020, a warrantholder exercised 20,331 options in a cashless exercise at net, and the Company issued 17,369 shares of common stock. 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 warrants vest immediately upon issuance, have an exercise price of $8.24 per share and expire 10 years from the date of issuance. The fair value of the 2018 Warrants is 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 warrants vest immediately upon issuance, have an exercise price of $8.24 per share and expire 10 years from the date of issuance. 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. On June 28, 2022, a warrantholder 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. As of December 31, 2022, the Company had 8,250,000 Pre-Funded Warrants outstanding with a weighted average exercise price of $0.0001 per share and an average contractual life of 5 years . As of December 31, 2022, the Company had 30,000,000 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 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. 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 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. At December 31, 2022, the Pre-funded Warrants are recorded as equity, using their fair value as of the amendment date. The fair value of the Common and Pre-Funded Warrants were estimated using the Black-Scholes option pricing model with the following assumptions: Expected term (in years) 3.7 Volatility 91.53 % Risk-free interest rate 4.11 % Dividend yield 0.00 % A summary of the Company’s outstanding common stock warrants as of December 31, 2022 is as follows: Warrants Outstanding as of December 31, 2021 125,618 Warrants granted and issued 40,000,000 Warrants exercised (1,750,000) Warrants exchanged — Outstanding as of December 31, 2022 38,375,618 |
LEASES
LEASES | 12 Months Ended |
Dec. 31, 2022 | |
LEASES | |
LEASES | 10. LEASES The following table summarizes our lease assets and liabilities as of December 31, 2022 and 2021: Operating December 31, December 31, ROU Assets and Liabilities Balance Sheet Location 2022 2021 ROU - Asset Right-of-use assets $ 857 $ 1,298 Lease liabilities (current) Operating lease liabilities, current 477 442 Lease liabilities (non-current) Operating lease liabilities, non-current 414 891 The following table summarizes our lease related costs for the twelve months ended December 31, 2022 and 2021: Operating (in thousands) Lease Cost Statement of Operations Location 2022 2021 Operating Lease Cost General and administrative $ 489 $ 505 Total Lease Cost $ 489 $ 505 Average lease terms and discount rates for the Company’s operating leases were as follows: Year Ended December 31, December 31, Other Information 2022 2021 Weighted-average remaining lease term Operating leases 1.8 years 2.8 years Weighted-average discount rate Operating leases 5.69% 5.69% The following table summarizes the maturities of lease liabilities as of December 31, 2022: Operating Year (in thousands) 2023 $ 515 2024 425 Thereafter — Total lease payments 940 Less: interest 49 Total lease liabilities $ 891 |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2022 | |
INCOME TAXES | |
INCOME TAXES | 11. INCOME TAXES The Company’s current tax provision for the years ended December 31, 2022 and 2021 is $0 and $0, respectively. The Company’s deferred tax provision for the years ended December 31, 2022 and 2021 is $0 and $0, respectively. A reconciliation of the Company's deferred taxes is as follows (in thousands): Year ended December 31, 2022 2021 Deferred Tax Assets Operating Lease Liability $ 282 $ 420 Stock-based compensation 8,131 6,606 License fee 554 594 Net operating loss carryforwards 88,090 79,429 Research and development tax credits 21,397 14,940 Capitalized R&D expense 14,910 — Other 1,085 1,352 Total Deferred Tax Assets $ 134,449 $ 103,341 Valuation Allowance $ (134,177) $ (102,932) Net Deferred Tax Assets $ 272 $ 409 Deferred Tax Liabilities Right of Use Asset (272) (409) Net Deferred Tax Liabilities $ (272) $ (409) Net deferred tax asset (liability) $ — $ — Deferred tax assets result primarily from unutilized net operating losses, research tax credits, stock-based compensation, operating lease liability, and timing differences as a result of the Company reporting its income tax returns. As of December 31, 2022, the Company had approximately As of December 31, 2022, the Company had federal research tax credits (R&D) of approximately $4.9 million which may be used to offset future tax liabilities. Additionally, the Company had a federal orphan drug credit (“ODC”) related to qualifying research of $16.5 million. These tax credit carryforwards will begin to expire at various times beginning in 2037 for federal purposes. The NOL, R&D and ODC credits carry forwards are subject to review and possible adjustment by the U.S. and state tax authorities. NOL carry forwards and credit carry forwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders, as defined under Sections 382 and 383 Internal Revenue Code. This could limit the amount of NOLs and credits that the Company can utilize annually to offset future taxable income or tax liabilities. As of December 31, 2022, the Company has not performed such an analysis. Subsequent ownership changes and proposed future changes to tax rules in respect of the utilization of losses carried forward may further affect the limitation in future years. In assessing the realizability of the Company’s deferred tax assets, management considers whether or not it is more likely than not that some portion or all of the deferred tax assets will not be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income. The Company’s assessment is based on the weight of available evidence, including cumulative losses since inception and expected future losses and, as such, the Company does not believe it is more likely than not that the deferred tax assets will be realized. Accordingly, a full valuation allowance has been established and no deferred tax assets and related tax benefit have been recognized in the accompanying financial statements. At December 31, 2022 and 2021, the Company recorded valuation allowances of $134.2 million and $102.9 million, respectively. The increase in valuation allowance is primarily driven by additional net operating loss. The Company files its income tax returns in the United States, New York State, New York City, California, Massachusetts, and New Jersey. All years remain subject to examination for federal and state purpose. The U.S. federal statutory corporate tax rate reconciles to the Company’s effective tax rate for the years ended December 31, 2022 and 2021: Year Ended December 31, 2022 2021 Federal statutory rate 21.0 % 21.0 % State and local taxes net of federal tax benefit 10.2 10.7 Credits 7.8 5.6 Permanent Differences (0.1) 0.5 Change in valuation allowance (37.9) (37.7) Stock Compensation (1.1) — Other 0.1 (0.1) Total 0.0 % 0.0 % |
BENEFIT PLANS
BENEFIT PLANS | 12 Months Ended |
Dec. 31, 2022 | |
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. The Company made approximately $0.4 million and $0.2 million in matching contributions to the plan during the year ended December 31, 2022 and 2021, respectively. |
NET LOSS PER COMMON SHARE
NET LOSS PER COMMON SHARE | 12 Months Ended |
Dec. 31, 2022 | |
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 years ended December 2022 and 2021, as the inclusion of all potential common shares outstanding would have been anti-dilutive. The following table sets forth the computation of basic and diluted net loss per common share (in thousands, except share and per share data): Years Ended December 31, (in thousands, except for share data) 2022 2021 Numerator: Net loss $ (82,508) $ (105,584) Denominator: Weighted-average common stock outstanding 37,825,431 25,598,181 Net loss per share attributable to common stockholders - basic and diluted $ (2.18) $ (4.12) 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 at December 31, 2022 and 2021, 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 December 31, 2022 2021 Options to purchase common stock 4,874,047 4,704,888 Restricted stock units 815,509 469,485 Warrants to purchase common stock 30,125,618 125,618 |
RELATED PARTIES
RELATED PARTIES | 12 Months Ended |
Dec. 31, 2022 | |
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 years ended December 31, 2022 and 2021, the Company made payments to Alexandria LaunchLabs of approximately $0.1 million and $0.1 million, respectively under the LaunchLabs Agreement, which was recognized in research and development expenses. As of December 31, 2022, there were no amounts due to Alexandria LaunchLabs under the LaunchLabs Agreement. |
SUBSEQUENT EVENTS
SUBSEQUENT EVENTS | 12 Months Ended |
Dec. 31, 2022 | |
SUBSEQUENT EVENTS | |
SUBSEQUENT EVENTS | 15. SUBSEQUENT EVENTS On January 3, 2023, the “Company entered into an Exclusive License and Supply Agreement (the “Agreement”) with Mercury Pharma Group Limited (trading as Advanz Pharma Holdings), a company organized and existing under the laws of England and Wales (“ADVANZ PHARMA”), a UK headquartered global pharmaceutical company with a strategic focus on specialty, hospital, and rare disease medicines in Europe. Pursuant to the Agreement, the Company granted ADVANZ PHARMA the exclusive right and license to commercialize drug products containing AT-007 (also known as govorestat), the Company’s proprietary Aldose Reductase Inhibitor (ARI) (the “Licensed Product”), for use in treatment of sorbitol dehydrogenase deficiency (“SORD”) and Galactosemia in humans (each, a “Licensed Indication”) in the European Economic Area, Switzerland and the United Kingdom (the “Territory”). The Company also grants 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 the Company 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 Agreement, ADVANZ PHARMA agrees to pay the Company (i) an upfront payment of EUR 10 million (approx. 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 (approx. 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 Agreement is terminated in various circumstances in accordance with its terms. Certain of the patents licensed to ADVANZ PHARMA under the Agreement are sub-licensed from the University of Miami and Columbia University, and thus remain subject to certain obligations of the Company (including royalty obligations) to such institutions. Under the Agreement, the Company remains responsible for development of the Licensed Product, and must conduct such development through grant of marketing authorizations in the Licensed Indications in the Territory and as otherwise required under such marketing authorization, in accordance with any timeframe required by regulatory authorities. The Company retains sole responsibility for the conduct of all clinical trials (subject in some circumstances to cost-sharing with ADVANZ PHARMA), unless the Company provides ADVANZ PHARMA prior consent to conduct certain studies following marketing authorization, or ADVANZ PHARMA exercises certain step-in rights (as described below). The Company also agrees to manufacture and supply the Licensed Product in bulk form to ADVANZ PHARMA. ADVANZ PHARMA is responsible for secondary packaging and release for the Territory. The Agreement includes indemnification obligations on the part of both parties for third-party claims arising out of, among other things, a breach of the Agreement; an election by the other party not to initiate a recall; gross negligence or willful misconduct; and violation of applicable laws. In addition, both parties have agreed to indemnification obligations for third party liability product liability claims and certain exclusions from liability disclaimers, such as for breaches of confidentiality, death or personal injury caused by negligence or willful default. In certain circumstances, including in the event of specified supply shortages, bankruptcy and certain other financial events, a force majeure event lasting more than three months, or termination as a result of the Company’s gross negligence or willful misconduct, ADVANZ PHARMA may exercise certain step-in rights. Such step-in rights include the ability for ADVANZ PHARMA to perform its own supply arrangements, and in some cases, specified development rights in the Licensed Indications in the Territory and assignment of certain contract rights. In all such circumstances, ADVANZ PHARMA must continue to pay royalties and milestone payments. ADVANZ PHARMA may recoup certain of its manufacturing and development establishment costs, and deduct such costs from royalties. The Company may terminate the Agreement upon certain specified events, including ADVANZ PHARMA’s failure to launch or achieve certain sales threshold for the Licensed Product in major markets within a certain timeframe, or if ADVANZ PHARMA challenges a licensed patent, and either party may terminate the Agreement upon the other party’s material breach or insolvency, certain material safety issues or a force majeure event of the other party lasting longer than six months. ADVANZ PHARMA may also terminate the agreement if the Licensed Product does not receive marketing authorization for use in a Licensed Indication in any country in the Territory by a specified date, or in the event of the Company’s gross negligence or willful misconduct (subject to a cure period). |
ORGANIZATION AND SUMMARY OF S_2
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 12 Months Ended |
Dec. 31, 2022 | |
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 CNS rare diseases and diabetic complications. The Company was incorporated in Delaware on January 20, 2016 and is headquartered in New York, New York. On January 28, 2020, the Company completed its secondary public offering (the “Secondary Public Offering”), pursuant to which it issued and sold 2,741,489 shares of common stock at a public offering price of $45.50 per share, with an additional 411,223 shares sold pursuant to the underwriters’ full exercise of their option to purchase additional shares. The aggregate net proceeds received by the Company from the offering, after deducting underwriting discounts and commissions and offering costs, were $134.1 million. On June 4, 2020, the Company filed a shelf registration statement on Form S-3 (the “Shelf Registration Statement”) under which the Company may, from time to time, sell securities in one or more offerings having an aggregate offering price of up to $300.0 million. The Shelf Registration Statement was declared effective as of June 15, 2020. As of the filing date of this Annual Report, the Company has sold 3,450,000 shares of common stock under the Shelf Registration. On June 12, 2020, the Company entered into an equity distribution agreement (the “Goldman Equity Distribution Agreement”) with Goldman Sachs & Co. LLC (“Goldman”), 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 million. Goldman may act as an agent on the Company’s behalf or purchase shares of the Company’s common stock as a principal. As of December 31, 2021, the Company had On February 17, 2021, the Company completed an underwritten public offering (the “February Offering”) of 3,450,000 shares of common stock, including the exercise in full of the underwriters’ option to purchase 450,000 additional shares of common stock, which option closed on February 19, 2021. The shares were offered at a price to the public of per share, resulting in aggregate net proceeds of approximately , after deducting underwriting discounts and commissions and offering expenses. On January 26, 2022, the Company entered into an equity distribution agreement (the “Cowen 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 Cowen 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 Cowen 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 Cowen 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. As of December 31, 2022, the Company has not sold any shares of common stock pursuant to the Cowen Equity Distribution Agreement. 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. |
Liquidity and Going Concern | 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 revenue, and it does not expect to generate 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. We are actively pursuing several potential financing options. While we continue to explore opportunities to raise additional equity capital in the public markets, this has proven to be challenging in the biotech sector recently. Other options for structured finance which we continue to explore include a PIPE (private investment in public equity), debt, convertible debt, and synthetic royalty financing. Synthetic royalty financing, in particular, has become a favorable option for many companies for funding ongoing clinical development in late-stage and pre-approval programs. We have engaged an investment bank and we are specifically exploring this option in the near term. Additionally, we are in active dialogue with several potential partners regarding business development opportunities related to one or more of our programs. There can be no assurances that our 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 $82.5 million for the year ended December 31, 2022 and has an accumulated deficit of $348.8 million as of December 31, 2022. 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 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. |
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 liability 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. |
Fair Value Measurements | Fair Value Measurements Certain assets and liabilities are reported on a recurring basis at fair value. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: Level 1—Quoted prices in active markets for identical assets or liabilities. Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. Level 3—Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all short-term, highly liquid investments, with an original maturity of three months or less, to be cash equivalents. The Company maintains its cash in bank deposit accounts which, at times, may exceed federally insured limits. The Company has not experienced any losses in these accounts and does not believe it is exposed to any significant credit risk on cash and cash equivalents. |
Investments | Investments The Company has investments in marketable debt securities. The Company determines the appropriate classification of its investments at the date of purchase and reevaluates the classifications at the balance sheet date. Marketable debt securities with maturities of 12 months or less are classified as short-term. Marketable debt securities with maturities greater than 12 months are classified as long-term. The Company’s marketable securities are accounted for as available for sale (“AFS”). AFS securities are reported at fair value. Unrealized gains and losses, after applicable income taxes, are reported in accumulated other comprehensive income/(loss). Realized gains or losses on the sale of marketable securities are determined using the specific identification method and are recorded as a component of other income (expense), net. The Company conducts an other-than-temporary impairment (“OTTI”) analysis on a quarterly basis or more often if a potential loss-triggering event occurs. The Company considers factors such as the duration, severity and the reason for the decline in value, the potential recovery period and whether the Company intends to sell. For AFS securities, the Company also considers whether (i) it is more likely than not that the Company will be required to sell the debt securities before recovery of their amortized cost basis and (ii) the amortized cost basis cannot be recovered as a result of credit losses. |
Leases | Leases At the inception of an arrangement, the Company determines if an arrangement is, or contains, a lease based on the unique facts and circumstances present in that arrangement. Lease classification, recognition, and measurement are then determined at the lease commencement date. For arrangements that contain a lease the Company (i) identifies lease and non-lease components, (ii) determines the consideration in the contract, (iii) determines whether the lease is an operating or financing lease; and (iv) recognizes lease right-of-use (“ROU”) assets and liabilities. Lease liabilities and their corresponding ROU assets are recorded based on the present value of lease payments over the expected lease term. The interest rate implicit in lease contracts is typically not readily determinable and as such, the Company uses its incremental borrowing rate based on the information available at the lease commencement date, which represents an internally developed rate that would be incurred to borrow, on a collateralized basis, over a similar term, an amount equal to the lease payments in a similar economic environment. Most leases include options to renew and, or, terminate the lease, which can impact the lease term. The exercise of these options is at the Company’s discretion and the Company does not include any of these options within the expected lease term as the Company is not reasonably certain it will exercise these options. The Company has elected to combine lease components (for example fixed payments including rent) with non-lease components (for example, non-dedicated parking and common-area maintenance costs) on our real estate asset classes. Fixed, or in substance fixed, lease payments on operating leases are recognized over the expected term of the lease on a straight-line basis. Fixed lease expense on operating leases is recognized within operating expenses within the statements of operations. The Company has operating leases for our corporate offices. The Company has elected the short-term lease exemption and, therefore, do not recognize a ROU asset or corresponding liability for lease arrangements with an original term of 12 months or less. Leasehold improvements and assets under financing lease arrangements are amortized over the lesser of the asset’s estimated useful life or the term of the respective lease. Maintenance costs are expensed as incurred. Operating leases are included in operating lease right-of-use asset, current portion of operating lease liabilities, and noncurrent portion of operating lease liabilities in the balance sheet as of December 31, 2022 and 2021. |
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 year ended December 31, 2022: (in thousands) Balance as of December 31,2021 $ — Clinical holdback retained 464 Clinical holdback paid — Balance as of December 31,2022 $ 464 Less: clinical holdback current portion — Clinical holdback - long-term portion $ 464 There was no clinical holdback during the year ended December 31, 2021. |
Deferred Offering Costs | Deferred Offering Costs The Company capitalizes certain legal, professional accounting and other third party fees that are directly associated with in-process equity financings as deferred offering costs until such financings are consummated. After consummation of the equity financing, these costs are recorded in the statement of stockholders’ (deficit) equity as a reduction of proceeds generated as a result of the offering. Should a planned equity financing be abandoned, the deferred offering costs would be expensed immediately as a charge to operating expenses in the statement of operations. As of December 31, 2022 and 2021, the Company did not have any deferred offering costs recorded in prepaid and other current assets. |
Research and Development | Research and Development The Company expenses all costs incurred in performing research and development activities. Research and development expenses include salaries and other related costs, materials and supplies, preclinical expenses, manufacturing expenses, contract services and other outside expenses. As part of the process of preparing the financial statements, the Company is required to estimate their accrued research and development expenses. The Company makes estimates of the accrued expenses as of each balance sheet date in the financial statements based on facts and circumstances known at that time. In addition, there may be instances in which payments made to the Company’s vendors will exceed the level of services provided and result in a prepayment of the expense in which case such amounts are reflected as prepaid expenses and other current assets. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual or the amount of prepaid expenses accordingly. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized in prepaid expenses and other current assets. The capitalized amounts are expensed as the related goods are delivered or the services are performed. Research and development costs also include costs incurred in connection with certain licensing arrangements. Before a compound receives regulatory approval, the Company records upfront and milestone payments made to third parties under licensing arrangements as expense. Upfront payments are recorded when incurred, and milestone payments are recorded when the specific milestone has been achieved. Once a compound receives regulatory approval, the Company will record any milestone payments in identifiable intangible assets, less accumulated amortization and, unless the asset is determined to have an indefinite life, the Company will amortize the payments on a straight-line basis over the remaining agreement term or the expected product life cycle, whichever is shorter. |
General and Administrative | General and Administrative General and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, for personnel in the Company’s executive and finance functions. General and administrative expenses also include professional fees for legal, accounting, auditing, tax and consulting services; travel expenses; and facility-related expenses, which include allocated expenses for rent and maintenance of facilities and other operating costs. Commercial expenses consist of payroll expense for commercial personnel, as well as marketing, market research, market access, and other focused investments to support launch of drug candidates, generate evidence of commercial potential and value proposition, and maximize potential business development deal leverage. Commercial expenses are included in general and administrative expenses. |
Stock Based Compensation and Stock-Based Compensation-Restricted Stock Units | Stock-Based Compensation The Company accounts for its stock-based compensation as expense in the statements of operations based on the awards’ grant date fair values. The Company accounts for forfeitures as they occur by reversing any expense recognized for unvested awards. The Company estimates the fair value of options granted using the Black-Scholes option pricing model. The Black-Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the award, (c) the risk-free interest rate and (d) expected dividends. Due to the lack of a public market for the Company’s common stock and a lack of company-specific historical and implied volatility data, the Company has based its estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded. The historical volatility is calculated based on a period of time commensurate with the expected term assumption. The computation of expected volatility is based on the historical volatility of a representative group of companies with similar characteristics to the Company, including stage of product development and life science industry focus. The Company uses the simplified method as allowed by the Securities and Exchange Commission (“SEC”) Staff Accounting Bulletin (“SAB”) No. 107, Share-Based Payment, to calculate the expected term for options granted to employees as it does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. The risk-free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. The expected dividend yield is assumed to be zero as the Company has never paid dividends and has no current plans to pay any dividends on its common stock. The fair value of stock-based payments is recognized as expense over the requisite service period which is generally the vesting period. Stock-Based Compensation–Restricted Stock Units The Company accounts for restricted stock units in accordance with the authoritative guidance for stock-based compensation. The fair value of restricted stock units is measured at the grant date based on the closing market price of the Company’s common stock on the date of grant, and is recognized as expense on a straight-line basis over the period of vesting. Forfeitures are recognized as a reduction of stock-based compensation expense as they occur. |
Income Taxes | Income Taxes The Company uses the asset and liability method of accounting for deferred income taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities at currently enacted tax rates. These temporary differences primarily relate to net operating loss carryforwards available to offset future taxable income. Valuation allowances are established, if necessary, to reduce a deferred tax asset to the amount that will more likely than not be realized. The Company recognizes tax liabilities from an uncertain tax position only if it is more likely than not that the tax position will not be sustained upon examination by the taxing authorities, based on the technical merits of the tax position. There are no uncertain tax positions that have been recognized in the accompanying financial statements. The Company is required to file tax returns in the U.S. federal jurisdiction and in the states of New York, California, Massachusetts, New Jersey, and New York City. The Company’s policy is to recognize interest and penalties related to uncertain tax benefits, if any, as part of income tax expense. No such interest and penalties have been accrued as of December 31, 2022 and 2021. In December 2019, the FASB issued ASU 2019-12, Income Taxes: Simplifying the Accounting for Income Taxes. |
Net Loss per Share | Net Loss per Share Basic net loss per share is calculated by dividing net loss available to common stockholders by the weighted-average common stock outstanding. Diluted net loss per share is calculated similarly, except that it includes the dilutive effect of the assumed exercise of securities, including outstanding warrants and the effect of shares issuable under the Company’s stock-based compensation plan, if such effect is dilutive. |
Segment Information | Segment Information Operating segments are defined as components of an enterprise for which separate discrete information is available for evaluation by the chief operating decision-maker in deciding how to allocate resources and assess performance. The Company and the Company’s chief operating decision-maker, the Company’s chief executive officer, views the Company’s operations and manages its business as a single operating segment, which is the business of discovering and developing its product candidates. |
Recent Accounting Pronouncements | 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) | 12 Months Ended |
Dec. 31, 2022 | |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Schedule of activity within the clinical holdback liability accounts | (in thousands) Balance as of December 31,2021 $ — Clinical holdback retained 464 Clinical holdback paid — Balance as of December 31,2022 $ 464 Less: clinical holdback current portion — Clinical holdback - long-term portion $ 464 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Fair value | |
Schedule of financial assets or liabilities 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 As of December 31, 2021 (in thousands) Level 1 Level 2 Level 3 Total Cash $ 12,671 $ — $ — $ 12,671 Money market funds 41,217 — — 41,217 Total cash and cash equivalents $ 53,888 $ — $ — $ 53,888 U.S. government agency debt securities — 26,935 — 26,935 Total marketable securities $ — $ 26,935 $ — $ 26,935 Total financial assets measured at fair value on a recurring basis $ 53,888 $ 26,935 $ — $ 80,823 |
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 liability, for which fair value is determined using Level 3 inputs (in thousands): Warrant Liability Common Warrant Pre-Funded Warrant Balance as of January 1, 2022 $ — $ — Initial fair value of Warrant Liability 13,734 8,101 Warrants exercised — (1,417) Change in fair value (77) 143 Reclassification of pre-funded warrant liability to equity (6,827) Balance as of December 31, 2022 $ 13,657 $ — |
Common And Pre-Funded Warrants | |
Fair value | |
Schedule of fair value of warrants using the Black Scholes option pricing model | Expected term (in years) 3.7 Volatility 91.53 % Risk-free interest rate 4.11 % Dividend yield 0.00 % |
INVESTMENTS (Tables)
INVESTMENTS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
INVESTMENTS | |
Schedule of marketable securities by security type | The following tables provide the Company’s marketable securities by security type (in thousands): As of December 31, 2022 As of December 31, 2021 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 $ 27,042 $ 1 $ (108) $ 26,935 Total $ 13,873 $ 50 $ — $ 13,923 $ 27,042 $ 1 $ (108) $ 26,935 |
Schedule of contractual maturities | Contractual maturities of the Company’s marketable securities are summarized as follows (in thousands): As of December 31, 2022 As of December 31, 2021 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 $ 27,042 $ 1 $ (108) $ 26,935 Due in one through two years — — — — — — — — Total $ 13,873 $ 50 $ — $ 13,923 $ 27,042 $ 1 $ (108) $ 26,935 |
Unrealized Gain (Loss) on Investments | As of December 31, 2022 Securities in an unrealized loss position less than 12 months Securities in an unrealized loss position greater than 12 months Total Unrealized Estimated Unrealized Estimated Unrealized Estimated (in thousands) Losses Fair Value Losses Fair Value Losses Fair Value US government agency debt security $ — $ — $ — $ — $ — $ — Total $ — $ — $ — $ — $ — $ — As of December 31, 2021 Securities in an unrealized loss position less than 12 months Securities in an unrealized loss position greater than 12 months Total Unrealized Estimated Unrealized Estimated Unrealized Estimated (in thousands) Losses Fair Value Losses Fair Value Losses Fair Value US government agency debt security $ (108) $ 17,696 $ — $ — $ (108) $ 17,696 Total $ (108) $ 17,696 $ — $ — $ (108) $ 17,696 |
PREPAID EXPENSES AND OTHER CU_2
PREPAID EXPENSES AND OTHER CURRENT ASSETS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | |
Summary of prepaid expenses and other current assets | Prepaid expenses and other current assets consisted of the following (in thousands): December 31, December 31, (in thousands) 2022 2021 Prepaid research and development expenses $ 4,272 $ 4,483 Insurance premium asset 1,131 1,616 Prepaid rent expenses 99 117 Prepaid insurance expenses 71 105 Prepaid commercial and patient advocacy 206 254 Research and development tax credit receivable 252 502 Interest receivable 23 23 Other prepaid expenses and current assets 674 471 Total prepaid expenses & other current assets $ 6,728 $ 7,571 |
ACCRUED EXPENSES AND OTHER CU_2
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | |
Summary of accrued expenses and other current liabilities | Accrued expenses and other current liabilities consisted of the following (in thousands): December 31, December 31, (in thousands) 2022 2021 Accrued pre-clinical and clinical expenses $ 8,877 $ 9,912 Short-term insurance financing note 622 789 Accrued professional fees 1,218 419 Accrued compensation and benefits 2,301 2,779 Accrued commercial expenses 896 1,394 Accrued patent expenses 361 279 Other 481 987 Total accrued expenses & other current liabilities $ 14,756 $ 16,559 |
STOCK BASED COMPENSATION (Table
STOCK BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Stock-based compensation | |
Schedule of stock-based compensation expenses | Total stock-based compensation expense recorded for employees, directors and non-employees (in thousands): Year Ended December 31, (in thousands) 2022 2021 Research and development $ 3,619 $ 2,759 General and administrative 5,543 8,418 Total stock-based compensation expense $ 9,162 $ 11,177 |
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, 2021 4,704,888 $ 13.29 7.7 $ 10,305 Options granted 767,913 1.39 Options exercised (47,602) 1.04 21 Forfeited (377,378) 19.90 Expired (173,774) 26.82 Outstanding at December 31, 2022 4,874,047 $ 1.97 6.9 $ — Exercisable at December 31, 2022 3,859,880 $ 2.21 6.6 $ — Nonvested at December 31, 2022 1,014,167 $ 1.05 8.3 $ — |
Schedule of restricted stock units outstanding | Weighted-Average Grant Date Aggregate (in thousands, except for share data) Shares Fair Value Intrinsic Value Outstanding at December 31, 2021 469,485 $ 18.05 $ 4,202 Awarded 605,600 2.33 Released (50,242) 24.57 Forfeited (209,334) 18.10 Outstanding at December 31, 2022 815,509 $ 5.96 $ 620 Nonvested at December 31, 2022 815,509 $ 5.73 $ 582 Weighted Average Remaining Recognition Period (in years) 3.2 |
Stock options - Service conditions only | |
Stock-based compensation | |
Schedule of valuation assumptions | Year Ended December 31, 2022 2021 Expected term (in years) 5.7 5.8 Volatility 75.83 % 71.92 % Risk-free interest rate 2.13 % 1.08 % Dividend yield — % — % |
WARRANTS (Tables)
WARRANTS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Warrants | |
Schedule of warrants outstanding | Warrants Outstanding as of December 31, 2021 125,618 Warrants granted and issued 40,000,000 Warrants exercised (1,750,000) Warrants exchanged — Outstanding as of December 31, 2022 38,375,618 |
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 | Expected term (in years) 3.7 Volatility 91.53 % Risk-free interest rate 4.11 % Dividend yield 0.00 % |
LEASES (Tables)
LEASES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
LEASES | |
Summary of lease assets and liabilities | Operating December 31, December 31, ROU Assets and Liabilities Balance Sheet Location 2022 2021 ROU - Asset Right-of-use assets $ 857 $ 1,298 Lease liabilities (current) Operating lease liabilities, current 477 442 Lease liabilities (non-current) Operating lease liabilities, non-current 414 891 |
Summary of lease related costs | Operating (in thousands) Lease Cost Statement of Operations Location 2022 2021 Operating Lease Cost General and administrative $ 489 $ 505 Total Lease Cost $ 489 $ 505 |
Summary of lease terms and discount rates | Year Ended December 31, December 31, Other Information 2022 2021 Weighted-average remaining lease term Operating leases 1.8 years 2.8 years Weighted-average discount rate Operating leases 5.69% 5.69% |
Summary of maturities of lease liabilities | Operating Year (in thousands) 2023 $ 515 2024 425 Thereafter — Total lease payments 940 Less: interest 49 Total lease liabilities $ 891 |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
INCOME TAXES | |
Schedule of reconciliation of deferred taxes | A reconciliation of the Company's deferred taxes is as follows (in thousands): Year ended December 31, 2022 2021 Deferred Tax Assets Operating Lease Liability $ 282 $ 420 Stock-based compensation 8,131 6,606 License fee 554 594 Net operating loss carryforwards 88,090 79,429 Research and development tax credits 21,397 14,940 Capitalized R&D expense 14,910 — Other 1,085 1,352 Total Deferred Tax Assets $ 134,449 $ 103,341 Valuation Allowance $ (134,177) $ (102,932) Net Deferred Tax Assets $ 272 $ 409 Deferred Tax Liabilities Right of Use Asset (272) (409) Net Deferred Tax Liabilities $ (272) $ (409) Net deferred tax asset (liability) $ — $ — |
Schedule of the U.S. federal statutory corporate tax rate reconciles to the Company's effective tax rate | Year Ended December 31, 2022 2021 Federal statutory rate 21.0 % 21.0 % State and local taxes net of federal tax benefit 10.2 10.7 Credits 7.8 5.6 Permanent Differences (0.1) 0.5 Change in valuation allowance (37.9) (37.7) Stock Compensation (1.1) — Other 0.1 (0.1) Total 0.0 % 0.0 % |
NET LOSS PER COMMON SHARE (Tabl
NET LOSS PER COMMON SHARE (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
NET LOSS PER COMMON SHARE | |
Schedule of computation of basic and diluted net loss per common share | The following table sets forth the computation of basic and diluted net loss per common share (in thousands, except share and per share data): Years Ended December 31, (in thousands, except for share data) 2022 2021 Numerator: Net loss $ (82,508) $ (105,584) Denominator: Weighted-average common stock outstanding 37,825,431 25,598,181 Net loss per share attributable to common stockholders - basic and diluted $ (2.18) $ (4.12) |
Schedule of potential dilutive securities excluded from the computation of diluted net loss per share attributable to common stockholders | As of December 31, 2022 2021 Options to purchase common stock 4,874,047 4,704,888 Restricted stock units 815,509 469,485 Warrants to purchase common stock 30,125,618 125,618 |
ORGANIZATION AND SUMMARY OF S_4
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Stock issued (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | 31 Months Ended | |||||||
Jun. 27, 2022 | Jan. 26, 2022 | Feb. 19, 2021 | Jun. 12, 2020 | Jun. 04, 2020 | Jan. 28, 2020 | Jun. 30, 2020 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2022 | |
Sale of stock | ||||||||||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | $ 0.0001 | |||||||
Proceeds from stock issuance, net of cash issuance costs | $ 74,421 | |||||||||
Common And Pre-Funded Warrants | ||||||||||
Sale of stock | ||||||||||
Warrants term | 5 years | |||||||||
Secondary Public Offering, 2020 | ||||||||||
Sale of stock | ||||||||||
Offering price (in dollars per share) | $ 45.50 | |||||||||
Proceeds from stock issuance, net of cash issuance costs | $ 134,100 | |||||||||
Secondary Public Offering, Excluding Underwriters' Option | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 2,741,489 | |||||||||
Secondary Public Offering, Underwriters' Option | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 411,223 | |||||||||
Shelf Registration Statement | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 3,450,000 | |||||||||
Maximum value of shares authorized to be sold in stock offering | $ 300,000 | |||||||||
Goldman Equity Distribution Agreement | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 0 | |||||||||
Maximum value of shares authorized to be sold in stock offering | $ 100,000 | $ 100,000 | ||||||||
Maximum amount to be paid to sales agent (as a percent) | 3% | |||||||||
February Offering, 2021 | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 3,450,000 | |||||||||
Offering price (in dollars per share) | $ 23 | |||||||||
Proceeds from stock issuance, net of cash issuance costs | $ 74,400 | |||||||||
February Offering Underwriters' Option | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 450,000 | |||||||||
Cowen Equity Distribution Agreement | ||||||||||
Sale of stock | ||||||||||
Maximum value of shares authorized to be sold in stock offering | $ 100,000 | |||||||||
Maximum amount to be paid to sales agent (as a percent) | 3% | |||||||||
June Offering | ||||||||||
Sale of stock | ||||||||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 20,000,000 | |||||||||
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 pre-funded warrants, net | $ 27,800 | |||||||||
June 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 Offering | Pre-Funded Warrants | ||||||||||
Sale of stock | ||||||||||
Warrants to purchase common stock (in shares) | 10,000,000 | |||||||||
June Offering | Common Warrants | ||||||||||
Sale of stock | ||||||||||
Warrants to purchase common stock (in shares) | 30,000,000 | 30,000,000 | 30,000,000 |
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 | 12 Months Ended | |||
Jan. 03, 2023 USD ($) | Jan. 03, 2023 EUR (€) | Jan. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Cash and cash equivalents | |||||
Net loss | $ (82,508) | $ (105,584) | |||
Accumulated deficit | (348,823) | $ (266,315) | |||
Cash and cash equivalents and investments | $ 30,600 | ||||
Substantial Doubt about Going Concern, within One Year | true | ||||
Subsequent Event. | ADVANZ PHARMA | Exclusive License and Supply Agreement | |||||
Cash and cash equivalents | |||||
Upfront payment received | $ 10,700 | € 10 | $ 10,700 |
ORGANIZATION AND SUMMARY OF S_6
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Clinical hold-back, long-term (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Agreements | ||
Clinical holdback retained | $ 464 | $ 0 |
Clinical holdback, Balance as of end of period | 464 | |
Clinical holdback - long-term portion | $ 464 | |
Minimum | ||
Agreements | ||
Clinical holdback, Total invoiced costs withheld from payment (as a percent) | 5% | |
Maximum | ||
Agreements | ||
Clinical holdback, Total invoiced costs withheld from payment (as a percent) | 10% |
ORGANIZATION AND SUMMARY OF S_7
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Deferred Offering Costs (Details) - USD ($) $ in Millions | Dec. 31, 2021 | Dec. 31, 2020 |
Deferred offering cost | ||
Deferred offering costs | $ 0 | $ 0 |
ORGANIZATION AND SUMMARY OF S_8
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Stock-based compensation (Details) | 12 Months Ended |
Dec. 31, 2022 | |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |
Dividend yield (as a percent) | 0% |
ORGANIZATION AND SUMMARY OF S_9
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Income Taxes (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
ORGANIZATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | ||
Uncertain tax positions | $ 0 | $ 0 |
Accrued interest and penalties | $ 0 | $ 0 |
LICENSE AGREEMENT - Columbia (D
LICENSE AGREEMENT - Columbia (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | 75 Months Ended | ||||
Oct. 03, 2019 | Mar. 31, 2019 | Jan. 31, 2019 | Oct. 31, 2016 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2022 | |
Agreements | |||||||
Research and development | $ 55,634 | $ 62,570 | |||||
General and administrative | 27,316 | 43,048 | |||||
Operating expenses | 82,950 | 105,618 | |||||
Accrued expenses and other current liabilities | 14,756 | 16,559 | $ 14,756 | ||||
Accounts payable | 4,534 | 9,461 | 4,534 | ||||
Columbia University | |||||||
Agreements | |||||||
Research and development | 0 | 0 | |||||
General and administrative | 200 | 200 | |||||
Operating expenses | 2,700 | ||||||
Accrued expenses and other current liabilities | 100 | 12 | 100 | ||||
Accounts payable | $ 0 | $ 100 | $ 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 ($) | 12 Months Ended | ||||
Dec. 14, 2020 | Oct. 28, 2020 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Agreements | |||||
Research and development | $ 55,634,000 | $ 62,570,000 | |||
General and administrative | 27,316,000 | 43,048,000 | |||
Operating expenses | 82,950,000 | 105,618,000 | |||
Accrued expenses and other current liabilities | 14,756,000 | 16,559,000 | |||
2020 license agreement | University of Miami | |||||
Agreements | |||||
Amount of first payment to be made for license issue fee | $ 1,100,000 | ||||
Amount of second payment to be made for license issue fee, due on first anniversary of the date of license | 500,000 | ||||
Payment to be made on achievement of specified patenting and development milestones | 2,200,000 | ||||
Payment to be made on achievement of late stage regulatory milestones | $ 4,100,000 | ||||
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 | 100,000 | 30,000 | |||
General and administrative | 0 | 0 | |||
Accrued expenses and other current liabilities | $ 300,000 | 300,000 | |||
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 | $ 300,000 | ||||
Research and development | 48,000 | $ 200,000 | |||
Accrued expenses and other current liabilities | $ 1,000,000 | $ 200,000 |
FAIR VALUE MEASUREMENTS - FV hi
FAIR VALUE MEASUREMENTS - FV hierarchy (Details) - Fair Value, Recurring - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | $ 16,657 | $ 53,888 |
Marketable securities | 13,923 | 26,935 |
Total financial assets measured at fair value on a recurring basis | 30,580 | 80,823 |
Warrant liabilities - Common Warrants | 13,657 | |
Total financial liabilities measured at fair value on a recurring basis | 13,657 | |
Level 1 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 16,657 | 53,888 |
Total financial assets measured at fair value on a recurring basis | 16,657 | 53,888 |
Level 2 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Marketable securities | 13,923 | 26,935 |
Total financial assets measured at fair value on a recurring basis | 13,923 | 26,935 |
Level 3 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Warrant liabilities - Common Warrants | 13,657 | |
Total financial liabilities measured at fair value on a recurring basis | 13,657 | |
Cash | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 1,337 | 12,671 |
Cash | Level 1 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 1,337 | 12,671 |
Money market funds | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 15,320 | 41,217 |
Money market funds | Level 1 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Cash and cash equivalents | 15,320 | 41,217 |
U.S. government agency debt securities | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Marketable securities | 13,923 | 26,935 |
U.S. government agency debt securities | Level 2 | ||
Financial assets or liabilities measured at fair value on a recurring basis | ||
Marketable securities | $ 13,923 | $ 26,935 |
FAIR VALUE MEASUREMENTS - Trans
FAIR VALUE MEASUREMENTS - Transfers (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
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) - June Offering - shares | Dec. 31, 2022 | Jun. 27, 2022 |
Common Warrants | ||
Warrants | ||
Warrants to purchase common stock (in shares) | 30,000,000 | 30,000,000 |
Pre-Funded Warrants | ||
Warrants | ||
Warrants to purchase common stock (in shares) | 10,000,000 |
FAIR VALUE MEASUREMENTS - War_2
FAIR VALUE MEASUREMENTS - Warrants Valuation (Details) - Common And Pre-Funded Warrants | 12 Months Ended |
Dec. 31, 2022 Y | |
Fair value | |
Warrants term | 5 years |
Expected term (in years) | |
Fair value | |
Measurement input | 3.7 |
Volatility | |
Fair value | |
Measurement input | 0.9153 |
Risk-free interest rate | |
Fair value | |
Measurement input | 0.0411 |
Dividend yield | |
Fair value | |
Measurement input | 0 |
Probability of change in control | |
Fair value | |
Measurement input | 0.10 |
FAIR VALUE MEASUREMENTS - Fair
FAIR VALUE MEASUREMENTS - Fair Values of Company's Warrant Liability (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2022 USD ($) | |
Common Warrants | |
Fair Value | |
Initial fair value of Warrant Liability | $ 13,734 |
Change in fair value | (77) |
Ending Balance | 13,657 |
Pre-Funded Warrants | |
Fair Value | |
Initial fair value of Warrant Liability | 8,101 |
Warrants exercised | (1,417) |
Change in fair value | 143 |
Reclassification of warrant liability to equity | $ (6,827) |
INVESTMENTS - By type (Details)
INVESTMENTS - By type (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Marketable securities by type | ||
Cost | $ 13,873 | $ 27,042 |
Gross Unrealized Gains | 50 | 1 |
Gross Unrealized Losses | 0 | (108) |
Estimated Fair Value | 13,923 | 26,935 |
U.S. government agency debt securities | ||
Marketable securities by type | ||
Cost | 13,873 | 27,042 |
Gross Unrealized Gains | 50 | 1 |
Gross Unrealized Losses | (108) | |
Estimated Fair Value | $ 13,923 | $ 26,935 |
INVESTMENTS - Additional Inform
INVESTMENTS - Additional Information (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
INVESTMENTS | ||
Gross unrealized loss | $ 0 | $ 108 |
INVESTMENTS - By contractual ma
INVESTMENTS - By contractual maturity (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
INVESTMENTS | ||
Due in one year or less - Cost | $ 13,873 | $ 27,042 |
Due in on year or less - Gross Unrealized Gains | 50 | 1 |
Due in one year or less - Gross Unrealized Losses | (108) | |
Due in one year or less - Estimated Fair Value | 13,923 | 26,935 |
Cost | 13,873 | 27,042 |
Gross Unrealized Gains | 50 | 1 |
Gross Unrealized Losses | 0 | (108) |
Estimated Fair Value | $ 13,923 | $ 26,935 |
INVESTMENTS - Realized gains an
INVESTMENTS - Realized gains and losses (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2022 USD ($) | |
Marketable securities realized gain (loss) recorded | |
Gross realized losses from redemption of marketable securities | $ 200 |
Gross realized gains from redemption of marketable securities | $ 16 |
INVESTMENTS - Unrealized losses
INVESTMENTS - Unrealized losses and fair values of available-for-sale securities (Details) $ in Thousands | Dec. 31, 2021 USD ($) |
Securities in an unrealized loss position less than 12 months | |
Unrealized Losses Less Than 12 months | $ (108) |
Estimated Fair Value Less Than 12 Months | 17,696 |
Total Unrealized Losses | (108) |
Total Estimated Fair Value | 17,696 |
U.S. government agency debt securities | |
Securities in an unrealized loss position less than 12 months | |
Unrealized Losses Less Than 12 months | (108) |
Estimated Fair Value Less Than 12 Months | 17,696 |
Total Unrealized Losses | (108) |
Total Estimated Fair Value | $ 17,696 |
PREPAID EXPENSES AND OTHER CU_3
PREPAID EXPENSES AND OTHER CURRENT ASSETS (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | ||
Prepaid research and development expenses | $ 4,272 | $ 4,483 |
Insurance premium asset | 1,131 | 1,616 |
Prepaid rent expenses | 99 | 117 |
Prepaid insurance expenses | 71 | 105 |
Prepaid commercial and patient advocacy | 206 | 254 |
Research and development tax credit receivable | 252 | 502 |
Interest receivable | 23 | 23 |
Other prepaid expenses and current assets | 674 | 471 |
Total prepaid expenses & other current assets | $ 6,728 | $ 7,571 |
ACCRUED EXPENSES AND OTHER CU_3
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | ||
Accrued pre-clinical and clinical expenses | $ 8,877 | $ 9,912 |
Short-term insurance financing note | 622 | 789 |
Accrued professional fees | 1,218 | 419 |
Accrued compensation and benefits | 2,301 | 2,779 |
Accrued commercial expenses | 896 | 1,394 |
Accrued patent expenses | 361 | 279 |
Other | 481 | 987 |
Total accrued expenses & other current liabilities | $ 14,756 | $ 16,559 |
STOCK BASED COMPENSATION - Plan
STOCK BASED COMPENSATION - Plans (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 12 Months Ended | |||
Aug. 02, 2022 | May 31, 2019 | Dec. 31, 2022 | Jan. 01, 2023 | Aug. 01, 2022 | |
Options | |||||
Stock-based compensation | |||||
Weighted Average Remaining Recognition Period (in years) | 2 years 2 months 12 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 | 1,096,493 | ||||
2019 Plan | Subsequent Event. | |||||
Stock-based compensation | |||||
Number of shares available for future issuance | 2,403,167 | ||||
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 | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Stock-based compensation | ||
Total stock-based compensation expense | $ 9,162 | $ 11,177 |
Research and development | ||
Stock-based compensation | ||
Total stock-based compensation expense | 3,619 | 2,759 |
General and administrative | ||
Stock-based compensation | ||
Total stock-based compensation expense | $ 5,543 | $ 8,418 |
STOCK BASED COMPENSATION - Opti
STOCK BASED COMPENSATION - Options (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Stock-based compensation | ||
Options granted (in shares) | 767,913 | 406,752 |
Stock-based compensation expense | $ 9,162 | $ 11,177 |
Unrecognized stock-based compensation balance for unvested options | $ 8,100 | |
Weighted-average fair value of options granted (in dollars per share) | $ 1.79 | $ 8.43 |
Options | ||
Stock-based compensation | ||
Stock-based compensation expense | $ 7,800 | $ 9,300 |
Weighted Average Remaining Recognition Period (in years) | 2 years 2 months 12 days |
STOCK BASED COMPENSATION - Op_2
STOCK BASED COMPENSATION - Options activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Options Outstanding | ||
Outstanding at beginning of period (in shares) | 4,704,888 | |
Options granted (in shares) | 767,913 | 406,752 |
Options exercised (in shares) | (47,602) | |
Forfeited (in shares) | (377,378) | |
Expired (in shares) | (173,774) | |
Outstanding at end of period (in shares) | 4,874,047 | 4,704,888 |
Exercisable at end of period (in shares) | 3,859,880 | |
Nonvested at end of period (in shares) | 1,014,167 | |
Weighted-Average Exercise Price | ||
Outstanding at beginning of period (in dollars per share) | $ 13.29 | |
Options granted (in dollars per share) | 1.39 | |
Options exercised (in dollars per share) | 1.04 | |
Options forfeited (in dollars per share) | 19.90 | |
Options expired (in dollars per share) | 26.82 | |
Outstanding at end of period (in dollars per share) | 1.97 | $ 13.29 |
Exercisable at end of period (in dollars per share) | 2.21 | |
Nonvested at end of period (in dollars per share) | $ 1.05 | |
Weighted-Average Remaining Contractual Term-in years | ||
Outstanding | 6 years 10 months 24 days | 7 years 8 months 12 days |
Exercisable | 6 years 7 months 6 days | |
Nonvested | 8 years 3 months 18 days | |
Aggregate Intrinsic Value | ||
Options exercised (in dollars) | $ 21 | |
Outstanding (in dollars) | $ 10,305 |
STOCK BASED COMPENSATION - Valu
STOCK BASED COMPENSATION - Valuation assumptions (Details) | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Valuation Assumptions | ||
Dividend yield (as a percent) | 0% | |
Stock options - Service conditions only | ||
Valuation Assumptions | ||
Expected term (in years) | 5 years 8 months 12 days | 5 years 9 months 18 days |
Volatility (as a percent) | 75.83% | 71.92% |
Risk-free interest rate (as a percent) | 2.13% | 1.08% |
STOCK BASED COMPENSATION - RSUs
STOCK BASED COMPENSATION - RSUs (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Stock-based compensation | ||
Stock-based compensation expense | $ 9,162 | $ 11,177 |
Restricted Stock Units (RSUs) | ||
Stock-based compensation | ||
Stock-based compensation expense | 1,300 | $ 1,900 |
Unamortized stock-based compensation costs for unvested awards | $ 3,500 | |
Summary of restricted stock units outstanding | ||
Outstanding at beginning of period ( in shares) | 469,485 | |
Awarded (in shares) | 605,600 | |
Released (in shares) | (50,242) | |
Forfeited (in shares) | (209,334) | |
Outstanding at end of period ( in shares) | 815,509 | 469,485 |
Nonvested at end of period (in shares) | 815,509 | |
Weighted-Average Grant Date Fair Value | ||
Outstanding at beginning of period (in dollars per share) | $ 18.05 | |
Awarded (in dollars per share) | 2.33 | |
Released (in dollars per share) | 24.57 | |
Forfeited (in dollars per share) | 18.10 | |
Outstanding at end of period (in dollars per share) | 5.96 | $ 18.05 |
Nonvested at end of period (in dollars per share) | $ 5.73 | |
Aggregate Intrinsic Value | ||
Outstanding at end of period (in dollars) | $ 620 | $ 4,202 |
Nonvested at end of period (in dollars) | $ 582 | |
Weighted Average Remaining Recognition Period (in years) | 3 years 2 months 12 days |
STOCK BASED COMPENSATION - 2019
STOCK BASED COMPENSATION - 2019 Employee Stock Purchase Plan (Details) - 2019 ESPP - shares | 1 Months Ended | 12 Months Ended |
May 31, 2019 | Dec. 31, 2022 | |
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 | Dec. 31, 2022 | Dec. 31, 2021 |
STOCKHOLDERS' EQUITY | ||
Common Stock, authorized (in shares) | 200,000,000 | 100,000,000 |
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 - Common S
STOCKHOLDERS' EQUITY - Common Stock and Equity Distribution Agreement (Details) $ in Millions | 1 Months Ended | 12 Months Ended | |||
Jan. 26, 2022 USD ($) | Jun. 12, 2020 USD ($) | Jun. 30, 2020 USD ($) | Dec. 31, 2022 Vote | Dec. 31, 2021 shares | |
STOCKHOLDERS' EQUITY | |||||
Number of votes per common stock | Vote | 1 | ||||
Goldman Equity Distribution Agreement | |||||
STOCKHOLDERS' EQUITY | |||||
Maximum value of shares authorized to be sold in stock offering. | $ 100 | $ 100 | |||
Maximum amount to be paid to sales agent (as a percent) | 3% | ||||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | shares | 0 | ||||
Cowen 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 | 12 Months Ended | ||
Jun. 27, 2022 | Dec. 31, 2022 | Dec. 31, 2021 | |
Equity | |||
Common Stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | |
Common And Pre-Funded Warrants | |||
Equity | |||
Warrants term | 5 years | ||
June Offering | |||
Equity | |||
Issuance of common stock upon secondary public offering, net of issuance costs (in shares) | 20,000,000 | ||
Common Stock, par value (in dollars per share) | $ 0.0001 | ||
Proceeds from issuance of shares and pre-funded warrants, net | $ 27.8 | ||
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 | ||
June 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 Offering | Pre-Funded Warrants | |||
Equity | |||
Warrants to purchase common stock (in shares) | 10,000,000 | ||
June Offering | Common Warrants | |||
Equity | |||
Warrants to purchase common stock (in shares) | 30,000,000 | 30,000,000 |
WARRANTS - Issued with Series A
WARRANTS - Issued with Series A Preferred Stock (Details) $ / shares in Units, $ in Thousands | 12 Months Ended | ||||||
Feb. 05, 2021 USD ($) shares | Feb. 24, 2020 shares | Feb. 21, 2020 item shares | Dec. 05, 2019 shares | Jan. 26, 2017 USD ($) $ / shares shares | Dec. 31, 2022 Rate Y shares | Dec. 31, 2021 USD ($) | |
Warrants | |||||||
Warrants exercised (in shares) | 1,750,000 | ||||||
Proceeds from exercise of warrants | $ | $ 69 | ||||||
2017 Warrants | Warrants Issued with Series A Preferred Stock | |||||||
Warrants | |||||||
Warrants to purchase common stock (in shares) | 309,389 | ||||||
Warrants to purchase common stock (in dollars) | $ | $ 200 | ||||||
Exercise price (in dollars per share) | $ / shares | $ 2.49 | ||||||
Number of warrant holders that exercised options | item | 2 | ||||||
Warrants exercised (in shares) | 27,855 | 72,818 | 185,634 | 20,000 | |||
Stock issued in exercise of warrants (in shares) | 27,855 | 69,094 | 176,092 | 17,026 | |||
Proceeds from exercise of warrants | $ | $ 69 | ||||||
2017 Warrants | Warrants Issued with Series A Preferred Stock | Contractual term (in years) | |||||||
Warrants | |||||||
Measurement input | Y | 10 | ||||||
2017 Warrants | Warrants Issued with Series A Preferred Stock | Volatility | |||||||
Warrants | |||||||
Measurement input | Rate | 0.7448 | ||||||
2017 Warrants | Warrants Issued with Series A Preferred Stock | Risk-free interest rate | |||||||
Warrants | |||||||
Measurement input | Rate | 0.0320 | ||||||
2017 Warrants | Warrants Issued with Series A Preferred Stock | Dividend yield | |||||||
Warrants | |||||||
Measurement input | Rate | 0 |
WARRANTS - Issued with the 2018
WARRANTS - Issued with the 2018 Notes (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||||
Jun. 24, 2020 | Feb. 24, 2020 | Nov. 05, 2018 | Dec. 31, 2022 | Jan. 18, 2018 | |
Warrants | |||||
Warrants exercised (in shares) | 1,750,000 | ||||
Warrants Issued with the 2018 Notes | |||||
Warrants | |||||
Initial fair value recorded as debt discount (in dollars) | $ 0.1 | ||||
Warrants to purchase common stock (in shares) | 76,847 | ||||
Warrants to purchase common stock (in dollars) | $ 0.3 | ||||
Exercise price (in dollars per share) | $ 6.59 | ||||
Warrants exercised (in shares) | 20,331 | 386 | |||
Stock issued in exercise of warrants (in shares) | 17,369 | 333 |
WARRANTS - Issued with Series B
WARRANTS - Issued with Series B Preferred Stock (Details) $ / shares in Units, $ in Millions | 1 Months Ended | 2 Months Ended | 12 Months Ended |
Feb. 28, 2019 USD ($) $ / shares shares | Dec. 31, 2018 USD ($) $ / shares shares | Dec. 31, 2022 Rate Y shares | |
Warrants | |||
Warrants exercised (in shares) | shares | 1,750,000 | ||
Warrants Issued with Series B Preferred Stock | 2018 Warrants | |||
Warrants | |||
Warrants to purchase common stock (in shares) | shares | 72,261 | ||
Warrants to purchase common stock (in dollars) | $ | $ 0.2 | ||
Exercise price (in dollars per share) | $ / shares | $ 8.24 | ||
Expiration term | 10 years | ||
Warrants Issued with Series B Preferred Stock | 2018 Warrants | Contractual term (in years) | |||
Warrants | |||
Measurement input | Y | 10 | ||
Warrants Issued with Series B Preferred Stock | 2018 Warrants | Volatility | |||
Warrants | |||
Measurement input | Rate | 0.7322 | ||
Warrants Issued with Series B Preferred Stock | 2018 Warrants | Risk-free interest rate | |||
Warrants | |||
Measurement input | Rate | 0.0270 | ||
Warrants Issued with Series B Preferred Stock | 2018 Warrants | Dividend yield | |||
Warrants | |||
Measurement input | Rate | 0 | ||
Warrants Issued with Series B Preferred Stock | 2019 Warrants | |||
Warrants | |||
Warrants to purchase common stock (in shares) | shares | 23,867 | ||
Warrants to purchase common stock (in dollars) | $ | $ 0.1 | ||
Exercise price (in dollars per share) | $ / shares | $ 8.24 | ||
Expiration term | 10 years | ||
Warrants Issued with Series B Preferred Stock | 2019 Warrants | Contractual term (in years) | |||
Warrants | |||
Measurement input | Y | 10 | ||
Warrants Issued with Series B Preferred Stock | 2019 Warrants | Volatility | |||
Warrants | |||
Measurement input | 0.7322 | ||
Warrants Issued with Series B Preferred Stock | 2019 Warrants | Risk-free interest rate | |||
Warrants | |||
Measurement input | 0.0270 | ||
Warrants Issued with Series B Preferred Stock | 2019 Warrants | Dividend yield | |||
Warrants | |||
Measurement input | 0 |
WARRANTS - Issued with June 202
WARRANTS - Issued with June 2022 Offering (Details) | 12 Months Ended | |||
Jun. 28, 2022 USD ($) shares | Jun. 27, 2022 $ / shares shares | Dec. 31, 2022 Y $ / shares shares | Dec. 31, 2021 USD ($) shares | |
Warrants | ||||
Warrants exercised (in shares) | 1,750,000 | |||
Proceeds from exercise of warrants | $ | $ 69,000 | |||
Warrants outstanding (in shares) | 38,375,618 | 125,618 | ||
Common And Pre-Funded Warrants | ||||
Warrants | ||||
Warrants term | 5 years | |||
Common And Pre-Funded Warrants | Contractual 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 | |||
June 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 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 Offering | Pre-Funded Warrants | ||||
Warrants | ||||
Warrants to purchase common stock (in shares) | 10,000,000 | |||
Exercise price (in dollars per share) | $ / shares | $ 0.0001 | |||
Warrants exercised (in shares) | 1,750,000 | |||
Exercise of prefunded warrants for common stock (in shares) | 1,750,000 | |||
Proceeds from exercise of warrants | $ | $ 175 | |||
Warrants outstanding (in shares) | 8,250,000 | |||
June Offering | Pre-Funded Warrants | Weighted average | ||||
Warrants | ||||
Exercise price (in dollars per share) | $ / shares | $ 0.0001 | |||
Warrants term | 5 years | |||
June Offering | Common Warrants | ||||
Warrants | ||||
Warrants to purchase common stock (in shares) | 30,000,000 | 30,000,000 | ||
Exercise price (in dollars per share) | $ / shares | $ 1 | |||
June Offering | Common Warrants | Weighted average | ||||
Warrants | ||||
Exercise price (in dollars per share) | $ / shares | $ 1 | |||
Warrants term | 5 years |
WARRANTS - Warrant Activity (De
WARRANTS - Warrant Activity (Details) | 12 Months Ended |
Dec. 31, 2022 shares | |
WARRANTS | |
Outstanding as of beginning of period | 125,618 |
Warrants granted and issued | 40,000,000 |
Warrants exercised | (1,750,000) |
Outstanding as of end of period | 38,375,618 |
LEASES - ROU Assets and Liabili
LEASES - ROU Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
ROU Assets and Liabilities | ||
ROU - Asset | $ 857 | $ 1,298 |
Lease liabilities (current) | 477 | 442 |
Lease liabilities (non-current) | $ 414 | $ 891 |
LEASES - Lease Cost (Details)
LEASES - Lease Cost (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Leases | ||
Operating Lease Cost | $ 489 | $ 505 |
General and administrative | ||
Leases | ||
Operating Lease Cost | $ 489 | $ 505 |
LEASES - Lease terms and discou
LEASES - Lease terms and discount rates (Details) | Dec. 31, 2022 | Dec. 31, 2021 |
LEASES | ||
Weighted-average remaining lease term - operating leases | 1 year 9 months 18 days | 2 years 9 months 18 days |
Weighted-average discount rate - operating leases | 5.69% | 5.69% |
LEASES - Maturities of Lease Li
LEASES - Maturities of Lease Liabilities (Details) $ in Thousands | Dec. 31, 2022 USD ($) |
LEASES | |
2023 | $ 515 |
2024 | 425 |
Total lease payments | 940 |
Less: interest | 49 |
Total lease liabilities | $ 891 |
Operating Lease, Liability, Statement of Financial Position | Current portion of operating lease liabilities, Noncurrent portion of operating lease liabilities |
INCOME TAXES - Provision (Detai
INCOME TAXES - Provision (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
INCOME TAXES | ||
Current tax provision | $ 0 | $ 0 |
Deferred tax provision | $ 0 | $ 0 |
INCOME TAXES - Deferred taxes (
INCOME TAXES - Deferred taxes (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Deferred Tax Assets | ||
Operating lease liability | $ 282 | $ 420 |
Stock-based compensation | 8,131 | 6,606 |
License fee | 554 | 594 |
Net operating loss carryforwards | 88,090 | 79,429 |
Research and development tax credits | 21,397 | 14,940 |
Capitalized R&D expense | 14,910 | |
Other | 1,085 | 1,352 |
Total Deferred Tax Assets | 134,449 | 103,341 |
Valuation Allowance | (134,177) | (102,932) |
Net Deferred Tax Assets | 272 | 409 |
Deferred Tax Liabilities | ||
Right of Use Asset | (272) | (409) |
Net Deferred Tax Liabilities | (272) | (409) |
Net deferred tax asset (liability) | $ 0 | $ 0 |
INCOME TAXES - NOLs (Details)
INCOME TAXES - NOLs (Details) $ in Millions | Dec. 31, 2022 USD ($) |
Federal | |
Income taxes | |
NOLs | $ 280.6 |
NOLs expiring through 2037 | 3.3 |
NOLs not subject expiration | 277.3 |
State | |
Income taxes | |
NOLs | $ 572.8 |
INCOME TAXES - Tax credit carry
INCOME TAXES - Tax credit carryforward (Details) $ in Millions | Dec. 31, 2022 USD ($) |
Research tax credit carryforward | |
Tax credit carryforward | |
Tax credit carryforward | $ 4.9 |
Orphan drug credit carryforward | |
Tax credit carryforward | |
Tax credit carryforward | $ 16.5 |
INCOME TAXES - Valuation allowa
INCOME TAXES - Valuation allowance (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
INCOME TAXES | ||
Net deferred tax asset (liability) | $ 0 | $ 0 |
Valuation Allowance | $ (134,177) | $ (102,932) |
INCOME TAXES - Income tax rate
INCOME TAXES - Income tax rate reconciliation (Details) | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Tax rate reconciliation | ||
Federal statutory rate (as a percent) | 21% | 21% |
State and local taxes net of federal tax benefit (as a percent) | 10.20% | 10.70% |
Credits (as a percent) | 7.80% | 5.60% |
Permanent Differences (as a percent) | (0.10%) | 0.50% |
Change in valuation allowance (as a percent) | (37.90%) | (37.70%) |
Stock Compensation (as a percent) | (1.10%) | |
Other (as a percent) | 0.10% | (0.10%) |
Total (as a percent) | 0% | 0% |
BENEFIT PLANS (Details)
BENEFIT PLANS (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
BENEFIT PLANS | ||
Employer matching contributions to the plan | $ 0.4 | $ 0.2 |
NET LOSS PER COMMON SHARE - EPS
NET LOSS PER COMMON SHARE - EPS (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Numerator: | ||
Net loss attributable to common stockholders-basic | $ (82,508) | $ (105,584) |
Net loss attributable to common stockholders-diluted | $ (82,508) | $ (105,584) |
Denominator: | ||
Weighted-average common stock outstanding-basic (in shares) | 37,825,431 | 25,598,181 |
Weighted-average common stock outstanding-diluted (in shares) | 37,825,431 | 25,598,181 |
Net loss per share attributable to common stockholders-basic (in dollars per share) | $ (2.18) | $ (4.12) |
Net loss per share attributable to common stockholders-diluted (in dollars per share) | $ (2.18) | $ (4.12) |
NET LOSS PER COMMON SHARE - Ant
NET LOSS PER COMMON SHARE - Anti-dilutive securities (Details) - shares | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Options 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 | 4,874,047 | 4,704,888 |
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 | 815,509 | 469,485 |
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 | 30,125,618 | 125,618 |
RELATED PARTIES (Details)
RELATED PARTIES (Details) - LaunchLabs Agreement - USD ($) | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Related Party Transactions | ||
Payments to related party | $ 100,000 | $ 100,000 |
Due to related parties | $ 0 |
SUBSEQUENT EVENTS (Details)
SUBSEQUENT EVENTS (Details) - Subsequent Event. - ADVANZ PHARMA - Exclusive License and Supply Agreement € in Millions, $ in Millions | 1 Months Ended | |||
Jan. 03, 2023 USD ($) | Jan. 03, 2023 EUR (€) | Jan. 31, 2023 USD ($) | Jan. 03, 2023 EUR (€) | |
Subsequent event | ||||
Upfront payment received | $ 10.7 | € 10 | $ 10.7 | |
Potential milestone payments to be received | $ 142.2 | € 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% | ||
Threshold period in which counterparty may exercise certain step-in rights following a force majeure event | 3 months | 3 months | ||
Threshold period in which entity may terminate contract following a force majeure even | 6 months | 6 months |