Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Feb. 29, 2024 | Jun. 30, 2023 | |
Document and Entity Information | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2023 | ||
Document Transition Report | false | ||
Entity File Number | 001-36576 | ||
Entity Registrant Name | Marinus Pharmaceuticals, Inc. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 20-0198082 | ||
Entity Address, Address Line One | 5 Radnor Corporate Center, Suite 500 | ||
Entity Address, Address Line Two | 100 Matsonford Road | ||
Entity Address, City or Town | Radnor | ||
Entity Address, State or Province | PA | ||
Entity Address, Postal Zip Code | 19087 | ||
City Area Code | 484 | ||
Local Phone Number | 801-4670 | ||
Title of 12(b) Security | Common Stock, par value $0.001 per share | ||
Trading Symbol | MRNS | ||
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 | false | ||
ICFR Auditor Attestation Flag | false | ||
Document Financial Statement Error Correction [Flag] | false | ||
Entity Shell Company | false | ||
Entity Public Float | $ 545,158,729 | ||
Entity Common Stock, Shares Outstanding | 54,642,580 | ||
Auditor Firm ID | 42 | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Location | Philadelphia, Pennsylvania | ||
Entity Central Index Key | 0001267813 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Fiscal Year Focus | 2023 | ||
Document Fiscal Period Focus | FY | ||
Amendment Flag | false |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 120,572 | $ 240,551 |
Short-term investments | 29,716 | |
Accounts receivable, net | 3,799 | 6,348 |
Inventory | 2,413 | 77 |
Prepaid expenses and other current assets | 8,746 | 5,402 |
Total current assets | 165,246 | 252,378 |
Property and equipment, net | 3,843 | 4,236 |
Other assets | 1,819 | 2,904 |
Total assets | 170,908 | 259,518 |
Current liabilities: | ||
Accounts payable | 4,003 | 4,461 |
Current portion of notes payable | 11,551 | |
Current portion of revenue interest financing payable | 2,211 | 1,020 |
Accrued expenses | 22,859 | 19,536 |
Total current liabilities | 40,624 | 25,017 |
Notes payable, net of deferred financing costs | 61,423 | 71,018 |
Revenue interest financing payable, net of deferred financing costs | 33,766 | 29,857 |
Contract liabilities, net | 17,545 | 16,285 |
Other long-term liabilities | 785 | 1,341 |
Total liabilities | 154,143 | 143,518 |
Stockholders' equity: | ||
Series A convertible preferred stock, $0.001 par value; 25,000,000 shares authorized, no shares issued and outstanding at December 31, 2023; 4,300 shares issued and outstanding at December 31, 2022 | 4,043 | |
Common stock, $0.001 par value; 150,000,000 shares authorized, 54,585,428 issued and 54,578,121 outstanding at December 31, 2023 and 49,650,074 issued and 49,642,767 outstanding at December 31, 2022 | 55 | 50 |
Additional paid-in capital | 588,656 | 542,428 |
Treasury stock at cost, 7,307 shares at December 31, 2023 and December 31, 2022 | ||
Accumulated other comprehensive loss | (20) | |
Accumulated deficit | (571,926) | (430,521) |
Total stockholders' equity | 16,765 | 116,000 |
Total liabilities and stockholders' equity | $ 170,908 | $ 259,518 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 54,585,428 | 49,650,074 |
Common stock, shares outstanding | 54,578,121 | 49,642,767 |
Treasury stock, shares | 7,307 | 7,307 |
Series A Convertible Preferred Stock | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 25,000,000 | 25,000,000 |
Preferred stock, shares issued | 0 | 4,300 |
Preferred stock, shares outstanding | 0 | 4,300 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Revenue: | ||
Federal contract revenue | $ 11,374 | $ 6,935 |
Total revenue | 30,989 | 25,478 |
Expenses: | ||
Research and development | 99,388 | 79,912 |
Selling, general and administrative | 61,152 | 56,845 |
Cost of IP license fee | 1,169 | |
Total expenses | 162,474 | 138,266 |
Loss from operations | (131,485) | (112,788) |
Interest income | 8,113 | 2,354 |
Interest expense | (16,895) | (10,672) |
(Loss) gain from sale of priority review voucher, net | (4,000) | 107,375 |
Other income (expense), net | 1,324 | (2,696) |
Loss before income taxes | (142,943) | (16,427) |
Benefit (provision) for income taxes | 1,538 | (3,389) |
Net loss applicable to common shareholders | $ (141,405) | $ (19,816) |
Per share information: | ||
Net loss per share of common stock-basic | $ (2.63) | $ (0.51) |
Net loss per share of common stock-diluted | $ (2.63) | $ (0.51) |
Basic weighted average shares outstanding | 53,746,518 | 39,072,599 |
Diluted weighted average shares outstanding | 53,746,518 | 39,072,599 |
Other comprehensive loss: | ||
Unrealized loss on available-for-sale securities | $ (20) | |
Total comprehensive loss | (141,425) | $ (19,816) |
Product revenue, net | ||
Revenue: | ||
Revenue | 19,561 | 2,872 |
Expenses: | ||
Cost of revenue | 1,909 | 190 |
Collaboration revenue | ||
Revenue: | ||
Revenue | 54 | 15,671 |
Expenses: | ||
Cost of revenue | $ 25 | $ 150 |
CONSOLIDATED STATEMENTS OF STOC
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Series A Convertible Preferred Stock Preferred Stock | Common Stock | Additional Paid-in Capital | Treasury Stock | Accumulated Other Comprehensive Income | Accumulated Deficit | Total |
Balance Beginning at Dec. 31, 2021 | $ 4,302 | $ 37 | $ 459,852 | $ (410,705) | $ 53,486 | ||
Balance Beginning (in shares) at Dec. 31, 2021 | 4,575 | 36,790,254 | 7,307 | ||||
Increase (Decrease) in Stockholders' Equity (Deficit) | |||||||
Stock-based compensation expense | 14,890 | 14,890 | |||||
Issuance of common stock and pre-funded warrants | $ 13 | 64,464 | 64,477 | ||||
Issuance of common stock and pre-funded warrants (in shares) | 12,421,053 | ||||||
Exercise of stock options | 1,794 | 1,794 | |||||
Exercise of stock options (in shares) | 238,312 | ||||||
Net issuance of common stock in connection with the vesting of restricted stock (in shares) | 14,893 | ||||||
Issuance of stock related to IP license agreement with Ovid | 1,169 | $ 1,169 | |||||
Issuance of stock related to IP license agreement with Ovid (in shares) | 123,255 | ||||||
Conversion of convertible preferred stock into common | $ (259) | 259 | |||||
Conversion of convertible preferred stock into common stock (in shares) | (275) | 55,000 | |||||
Issuance of common stock in connection with at-the-market facility offering (average price of $7.17 per share), net of expenses of $529 (in shares) | 0 | ||||||
Net Income (Loss) | (19,816) | $ (19,816) | |||||
Balance Ending at Dec. 31, 2022 | $ 4,043 | $ 50 | 542,428 | (430,521) | 116,000 | ||
Balance Ending (in shares) at Dec. 31, 2022 | 4,300 | 49,642,767 | 7,307 | ||||
Increase (Decrease) in Stockholders' Equity (Deficit) | |||||||
Stock-based compensation expense | 15,563 | 15,563 | |||||
Exercise of stock options | 804 | 804 | |||||
Exercise of stock options (in shares) | 126,735 | ||||||
Net issuance of common stock in connection with the vesting of restricted stock (in shares) | 258,819 | ||||||
Conversion of convertible preferred stock into common | $ (4,043) | $ 1 | 4,042 | ||||
Conversion of convertible preferred stock into common stock (in shares) | (4,300) | 860,000 | |||||
Issuance of common stock in connection with at-the-market facility offering (average price of $7.17 per share), net of expenses of $529 | $ 4 | 25,819 | 25,823 | ||||
Issuance of common stock in connection with at-the-market facility offering (average price of $7.17 per share), net of expenses of $529 (in shares) | 3,689,800 | ||||||
Unrealized loss on short-term investments | $ (20) | (20) | |||||
Net Income (Loss) | (141,405) | (141,405) | |||||
Balance Ending at Dec. 31, 2023 | $ 55 | $ 588,656 | $ (20) | $ (571,926) | $ 16,765 | ||
Balance Ending (in shares) at Dec. 31, 2023 | 54,578,121 | 7,307 |
CONSOLIDATED STATEMENTS OF ST_2
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Share Price (in dollars per share) | $ 7.17 | |
Stock issuance costs | $ 529 | |
Follow-on Public Offering | ||
Share Price (in dollars per share) | $ 4.75 | |
Stock issuance costs | $ 4,521 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Cash flows from operating activities | ||
Net loss | $ (141,405) | $ (19,816) |
Adjustments to reconcile net loss income to net cash used in operating activities: | ||
Gain from sale of PRV, net of transaction costs | (107,375) | |
Depreciation and amortization | 551 | 418 |
Amortization of debt issuance costs | 2,248 | 1,663 |
Accretion of revenue interest financing debt | 4,806 | 956 |
Amortization of discount on short-term investments | (1,241) | |
Stock-based compensation expense | 15,563 | 14,890 |
Amortization of net contract asset/liability | (1,353) | (1,052) |
Noncash lease expense | 187 | 251 |
Noncash lease liability | 413 | 344 |
Write off of fixed assets | 61 | 828 |
Issuance of common stock for cost of license agreement | 1,169 | |
Unrealized loss on foreign currency transactions | 930 | |
Changes in operating assets and liabilities: | ||
Refund liability | (22,163) | |
Net contract asset/liability | 2,613 | 17,894 |
Prepaid expenses and other current assets, non-current assets, inventory and accounts receivable | (2,552) | (5,577) |
Accounts payable and accrued expenses | 2,108 | 3,754 |
Net cash used in operating activities | (118,001) | (112,886) |
Cash flows from investing activities | ||
Proceeds from sale of PRV, net of transaction costs | 107,375 | |
Proceeds from sale of property and equipment | 9 | 171 |
Maturities of short-term investments | 23,500 | |
Purchases of short-term investments | (51,995) | |
Purchases of property and equipment | (119) | (1,774) |
Net cash (used in) provided by investing activities | (28,605) | 105,772 |
Cash flows from financing activities | ||
Proceeds from exercise of stock options | 804 | 1,794 |
Proceeds from notes payable, net of fees | 28,588 | |
Proceeds from revenue interest financing agreement, net of issuance costs | 29,921 | |
Proceeds from equity offerings, net of offering costs | 25,823 | 64,477 |
Other cash flows from financing activities | (42) | |
Net cash provided by financing activities | 26,627 | 124,738 |
Net (decrease) increase in cash and cash equivalents | (119,979) | 117,624 |
Cash and cash equivalents-beginning of period | 240,551 | 122,927 |
Cash and cash equivalents-end of period | 120,572 | 240,551 |
Supplemental disclosure of cash flow information | ||
Increase in right-of-use asset and liability | 66 | |
Property and equipment in accrued expenses | 22 | |
Cash paid for interest during the year | 8,697 | 7,892 |
Cash paid for income taxes during the year | $ 903 | |
Property and equipment in deposits placed in service | $ 1,664 |
Organization and Description of
Organization and Description of the Business | 12 Months Ended |
Dec. 31, 2023 | |
Organization and Description of the Business | |
Organization and Description of the Business | 1. Organization and Description of the Business We are a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for the treatment of seizure disorders, including rare genetic epilepsies and status epilepticus, which includes the use of ZTALMY® (ganaxolone). On March 18, 2022, the U.S. Food and Drug Administration (FDA) approved our new drug application (NDA) for the use of ZTALMY oral suspension CV for the treatment of seizures associated with Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in patients two years of age and older. ZTALMY, our first FDA approved product, became available for commercial sale and shipment in the third quarter of 2022. , We are also developing ganaxolone for the treatment of other rare genetic epilepsies, including Tuberous Sclerosis Complex (TSC), and for the treatment of Refractory Status Epilepticus (RSE), which is defined as status epilepticus (SE) that is refractory to treatment with a first-line benzodiazepine and at least one second-line antiseizure medication. SE is a life-threatening condition characterized by continuous, prolonged seizures or rapidly recurring seizures without intervening recovery of consciousness. If SE is not treated urgently, permanent neuronal damage may occur, which contributes to high rates of morbidity and mortality. We are developing ganaxolone in formulations for two different routes of administration: intravenous (IV) and oral. The different formulations are intended to maximize potential therapeutic applications of ganaxolone for adult and pediatric patient populations, in both acute and chronic care. While the precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures is unknown, its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A (GABA A A COVID-19 COVID-19 affected our clinical operations and timelines. For example, our RAISE trial for RSE is conducted in hospitals, primarily intensive care units in academic medical centers, which experienced high rates of COVID-19 admissions. Several of these sites participating in the RAISE trial experienced COVID-related difficulties, including staff turnover and the need to devote significant resources to patients with COVID-19, which resulted in site initiation and enrollment delays for the RAISE trial. Given these COVID-19-related challenges and the interruption in drug supply in mid-2022, we previously adjusted our expectation for our top-line data readout for the RAISE trial. In May 2022, we resumed screening and recruitment for the RAISE trial. We reached the enrollment target for the interim analysis in the first quarter of 2024. Liquidity Since inception, we have incurred negative cash flows from our operations, and other than for the three months ended September 30, 2022 due to a one-time net gain from the sale of our Priority Review Voucher (PRV), we have incurred net losses. We incurred a net loss of $141.4 million for the year ended December 31, 2023. There is no assurance that profitable operations will be achieved in the future, and if achieved, could be sustained on a continuing basis. In addition, development activities, clinical and preclinical testing, and commercialization of ganaxolone (in indications other than CDD in the U.S.) will require significant additional financing. Our accumulated deficit as of December 31, 2023 was $571.9 million and we expect to incur substantial losses in future periods. We plan to finance our future operations with a combination of proceeds from the issuance of equity securities, the issuance of debt, government funding, collaborations, licensing transactions and other commercial transactions or other sources, and revenues from future product sales. We have not generated positive cash flows from operations, and there are no assurances that we will be successful in obtaining an adequate level of financing for the continued development and commercialization of ganaxolone. Management’s operating plan which underlies the analysis of our ability to continue as a going concern involves the estimation of the amount and timing of future cash inflows and outflows. Actual results could vary from the operating plan. We follow the provisions of Financial Accounting Standards Board (FASB) Accounting Standards Codification (ASC) Topic 205-40, Presentation of Financial Statements—Going Concern, which requires management to assess our ability to continue as a going concern within one year after the date the financial statements are issued. We had Cash and cash equivalents and Short-term investments of $150.3 million as of December 31, 2023. We believe that such amount is not sufficient to fund our operations for the one year period after the date the financial statements are issued. As a result, there is substantial doubt about our ability to continue as a going concern through the one-year period from the date these financial statements are issued. Management’s plans that are intended to mitigate this risk include securing additional funding in the future from one or more equity or debt financings, government funding, collaborations, licensing transactions, and other commercial or strategic transactions. However, there can be no assurance that we will be successful in raising additional capital or that such capital, if available, will be on terms acceptable to us. We have and will continue to evaluate alternatives to extend our operations beyond the one-year period after the date the financial statements are issued. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2023 | |
Summary of Significant Accounting Policies | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Principles of Consolidation The financial statements include the accounts of Marinus Pharmaceuticals, Inc. and its wholly-owned subsidiary as of December 31, 2023. In February 2021, a wholly-owned subsidiary was established in Ireland. All intercompany accounts and transactions have been eliminated. Use of Estimates The preparation of financial statements in conformity with generally accepted accounting principles (GAAP) requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from such estimates. Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. We view our operations and manage our business in one segment, which is the identification and development of innovative therapeutics to treat rare seizure disorders. Fair Value of Financial Instruments and Credit Risk Cash equivalents and Short-term investments subject us to concentrations of credit risk. However, we invest our cash in accordance with a policy objective that seeks to ensure both liquidity and safety of principal. The policy limits investments to instruments issued by the U.S. government, certain Securities and Exchange Commission (SEC)-registered money market funds that invest only in U.S. government obligations and various other low-risk liquid investment options, and places restrictions on portfolio maturity terms. Cash and Cash Equivalents We consider all highly liquid investments that have maturities of three months or less when acquired to be cash equivalents. As of December 31, 2023 and 2022, we invested a portion of our cash balances in money market investments, which we have included as Cash equivalents on our balance sheets. Additionally, as of December 31, 2023, we invested a portion of our cash balances in U.S Treasury securities and U.S Government Agency securities, which we have included as Short-term investments on our balance sheet. Short-Term Investments We classify our Short-term investments as available-for-sale securities, which include U.S. government agency debt securities and U.S. treasury debt securities with original maturities of greater than three months. These securities are carried at fair market value, with unrealized gains and losses reported in Other comprehensive loss and Accumulated other comprehensive loss within stockholders’ equity. All of our investments were short-term in nature as of December 31, 2023. We did not have any investments as of December 31, 2022. Accounts Receivable, net Net trade receivables related to ZTALMY sales, which are recorded in Accounts receivable, net on the consolidated balance sheets, were approximately $2.6 million and $1.3 million as of December 31, 2023 and December 31, 2022, respectively. We had no allowance for doubtful accounts as of December 31, 2023 or 2022. An allowance for doubtful accounts is determined based on our assessment of the credit worthiness and financial condition of our customers, aging of receivables, as well as the general economic environment. Any allowance would reduce the net receivables to the amount that is expected to be collected. We have three customers, one of which, Orsini Pharmaceutical Services, LLC (Orsini), a specialty pharmacy that dispenses ZTALMY directly to patients, represents approximately 99% of our ZTALMY revenue to date. Payment terms for Orsini are 30 days from the shipment date. Excluding net trade receivables, Accounts receivable, net represents amounts due to us under the BARDA contract for valid expenditures expected to be reimbursed to us under the terms of the BARDA contract and current amounts due to us from Orion Corporation (Orion) under the collaboration agreement (Note 11). Inventory Inventories are recorded using actual costs and may consist of raw materials (ganaxolone API), work in process and finished goods. We began capitalizing Inventory related to ZTALMY subsequent to the March 2022 FDA approval of ZTALMY, as the related costs were expected to be recoverable through the commercialization and subsequent sale of ZTALMY. Prior to FDA approval of ZTALMY, costs estimated at approximately $2 million for commercially saleable product and materials were incurred and included in Research and development expenses. As a result, Cost of product revenues related to ZTALMY will initially reflect a lower average per unit cost of materials into approximately the first half of 2024, as previously expensed inventory is utilized for commercial production and sold to customers. Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets generally represent payments made for goods or services to be received within one year, and are expensed as the related benefit is received. Property and Equipment, net Property and equipment, net consist of laboratory and office equipment and are recorded at cost. Property and equipment are depreciated on a straight-line basis over their estimated useful lives. We estimate a life of five years for office equipment and furniture, five to fifteen years for laboratory equipment, and the lesser of the lease term or useful life for leasehold improvements. When property and equipment are sold or otherwise disposed of, the cost and related accumulated depreciation are removed from the accounts and the resulting gain or loss is included in Other income (expense), net. Impairment of Long-Lived Assets We review long-lived assets, including property and equipment, for impairment whenever events or changes in business circumstances indicate that the carrying amount of an asset may not be fully recoverable. If the estimated undiscounted future cash flows expected to result from the use of the asset and its eventual disposition is less than its carrying amount an impairment loss would be recognized if the carrying value of the asset exceeded its fair value. Fair value is generally determined using discounted cash flows. Other Assets Income Taxes The Tax Cuts and Jobs Act passed in 2017 included a provision which would require taxpayers to capitalize and amortize U.S.-based research & experimentation (R&E) expenses over a period of five years and non-U.S. R&E expenses over 15 years effective for tax years beginning after December 31, 2021 pursuant to Internal Revenue Code Section 174. For the year ended December 31, 2023, we recorded a Benefit for income taxes due to the identification of a discrete item of tax determined upon preparation of our 2022 tax return. As a result of the capitalization of R&E expenses, income generated from the sale of the PRV, and limitations related to the utilization of state net operating losses, Debt Issuance Costs Contract Liabilities, net Liability Related to Revenue Interest Financing and Non-Cash Interest Expense Sagard Healthcare Royalty Partners, LP (Sagard) Debt Interest - Imputation of Interest. Product Revenue, net We recognize ZTALMY revenue in accordance with ASC 606 – Revenue from contracts with customers. Our revenue recognition analysis consists of the following steps: (i) identification of the promised goods in the contract; (ii) determination of whether the promised goods are performance obligations, including whether they are capable of being distinct; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue as we satisfy each performance obligation. Our first FDA approved product, ZTALMY, became available for commercial sale and shipment to patients in the third quarter of 2022. We have three customers, one of which, Orsini, a specialty pharmacy that dispenses ZTALMY directly to patients, is responsible for approximately 99% of our ZTALMY revenue to date. Our contract with Orsini has a single performance obligation to deliver ZTALMY upon receipt of a purchase order, which is satisfied when Orsini receives ZTALMY. We recognize ZTALMY revenue at the point in time when control of ZTALMY is transferred to Orsini, which is upon delivery to Orsini. The transaction price that we recognize for ZTALMY revenue includes an estimate of variable consideration. Shipping and handling costs to Orsini are recorded as Selling, general and administrative expenses. The components of variable consideration include: Trade Discounts and Allowances. Product Returns and Recall. Government Rebates. Patient Assistance . Federal Contract Revenue We recognize Federal contract revenue from the BARDA Contract in the period in which the allowable Research and development expenses are incurred, and receivables associated with this revenue are included within Accounts receivable, net on our balance sheets. This revenue is not within the scope of Accounting Standards Codification (ASC) 606 – Revenue from contracts with customers. Collaboration and Licensing Revenue We must develop estimates and assumptions that require judgment to determine the underlying stand-alone selling price for each performance obligation, which determines how the transaction price is allocated among the performance obligations. The estimation of the stand-alone selling price may include such estimates as forecasted revenues and costs, development timelines, discount rates and probabilities of regulatory and commercial success. We also apply significant judgment when evaluating whether contractual obligations represent distinct performance obligations, allocating transaction price to performance obligations within a contract, determining when performance obligations have been met, assessing the recognition and future reversal of variable consideration and determining and applying appropriate methods of measuring progress for performance obligations satisfied over time. Research and Development Research and development costs are expensed as incurred. Costs for certain development activities, such as clinical trials, are recognized based on an evaluation of the progress to completion of specific tasks using data such as subject enrollment, monitoring visits, clinical site activations, or information provided to us by our vendors with respect to their actual costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected in the financial statements as prepaid or accrued research and development expense, as the case may be. Clinical Trial Expenses As part of the process of preparing our financial statements, we are required to estimate our expenses resulting from our obligations under contracts with vendors, clinical research organizations and consultants and under clinical site agreements in connection with conducting clinical trials. The financial terms of these contracts are subject to negotiations, which vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided under such contracts. Our objective is to reflect the appropriate trial expenses in our financial statements by matching those expenses with the period in which services are performed and efforts are expended. We account for these expenses according to the progress of the trial as measured by patient progression and the timing of various aspects of the trial. We determine accrual estimates based on estimates of services received and efforts expended that take into account discussion with applicable personnel and outside service providers as to the progress or state of consummation of trials. During the course of a clinical trial, we adjust our clinical expense recognition if actual results differ from its estimates. We make estimates of our Accrued expenses as of each balance sheet date based on the facts and circumstances known at that time. Our clinical trial accruals are dependent upon the timely and accurate reporting of contract research organizations and other third-party vendors. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in reporting amounts that are too high or too low for any particular period. For the years ended December 31, 2023 and 2022 there were no material adjustments to our prior period estimates of accrued or prepaid expenses for clinical trials. For the years ended December 31, 2023 and 2022, our accrued clinical trial expenses were $4.7 million and $5.7 million, respectively. For the years ended December 31, 2023 and 2022, our prepaid clinical expenses were $5.2 million and $3.9 million, respectively. Stock-Based Compensation We account for stock-based compensation in accordance with the provisions of Accounting Standards Codification (ASC) Topic 718, Compensation—Stock Compensation Loss Per Share of Common Stock Basic loss per share is computed by dividing Net loss applicable to common stockholders by the Weighted average number of shares of common stock outstanding during each period. Diluted loss per share includes the effect, if any, from the potential exercise or conversion of securities, such as convertible preferred stock, stock options, and unvested restricted stock, which would result in the issuance of incremental shares of common stock. In computing the Basic and diluted net loss per share applicable to common stockholders for the years ended December 31, 2023 and 2022, the Weighted average number of shares remains the same for both calculations due to the fact that when a Net loss exists, dilutive shares are not included in the calculation. These potentially dilutive securities are more fully described in Note 7. The following table sets forth the computation of Basic and diluted net loss per share for the years ended December 31, 2023 and 2022 (in thousands, except share and per share amounts): Year ended December 31, 2023 2022 Basic and diluted net loss per share of common stock: Net loss applicable to common stockholders $ (141,405) $ (19,816) Weighted average shares of common stock outstanding 53,746,518 39,072,599 Net loss per share of common stock—basic and diluted $ (2.63) $ (0.51) The pre-funded warrants to purchase common shares issued in connection with the November 2022 offering are included in the calculation of Basic and diluted net loss per share as the exercise price of $0.001 per share is non-substantive and is virtually assured. The pre-funded warrants are more fully described in Note 7. The following potentially dilutive securities (common stock equivalents) have been excluded from the computation of diluted weighted average shares outstanding, as they would be antidilutive: Year Ended December 31, 2023 2022 Convertible preferred stock — 860,000 Restricted stock units 1,271,894 671,976 Stock options 7,164,981 5,730,219 8,436,875 7,262,195 The convertible preferred stock meets the definition of a participating security; however, the holders were not obligated to share in our losses. As of December 31, 2023 and 2022, we had no other potentially dilutive securities. |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Measurements | |
Fair Value Measurements | 3. Fair Value Measurements FASB accounting guidance defines fair value as the price that would be received to sell an asset or paid to transfer a liability (exit price) in an orderly transaction between market participants at the measurement date. The accounting guidance outlines a valuation framework and creates a fair value hierarchy in order to increase the consistency and comparability of fair value measurements and the related disclosures. In determining fair value, we use quoted prices and observable inputs. Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from independent sources. The fair value hierarchy is broken down into three levels based on the source of inputs as follows: ● Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities. ● Level 2—Valuations based on observable inputs and quoted prices in active markets for similar assets and liabilities. ● Level 3—Valuations based on inputs that are unobservable and models that are significant to the overall fair value measurement. If the inputs used to measure fair value fall within different levels of the hierarchy, the category level is based on the lowest priority level input that is significant to the fair value measurement of the instrument. As of December 31, 2023, all of our financial assets and liabilities were classified as Level 1 or Level 2 valuations. As of December 31, 2022, all of our financial assets and liabilities were classified as Level 1 valuations. We estimate the fair values of our financial instruments categorized as Level 2 in the fair value hierarchy, including U.S. Treasury securities and U.S. Government Agency securities, by taking into consideration valuations obtained from third party pricing services. The pricing services use industry standard valuation models, including both income- and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include reported trades of and broker/dealer quotes on the same or similar securities, benchmark yields, issuer credit spreads, benchmark securities, and other observable inputs. We obtain a single price for each financial instrument and do not adjust the prices obtained from the pricing service. The following fair value hierarchy table presents information about each major category of our financial assets and liabilities measured at fair value on a recurring basis (in thousands): Level 1 Level 2 Level 3 Total December 31, 2023 Assets Cash $ 1,255 $ — $ — $ 1,255 Money market funds (cash equivalents) 119,317 — — 119,317 U.S. Treasury securities — 26,835 — 26,835 U.S. Government Agency securities — 2,881 — 2,881 Total assets $ 120,572 $ 29,716 $ — $ 150,288 December 31, 2022 Assets Cash $ 10,569 $ — $ — $ 10,569 Money market funds (cash equivalents) 229,982 — — 229,982 Total assets $ 240,551 $ — $ — $ 240,551 The following table provides details regarding our portfolio of Short-term investments (in thousands) as of December 31, 2023: Amortized Cost Unrealized Gains Unrealized Losses Fair Value U.S. Treasury securities $ 26,852 $ 138 $ (155) $ 26,835 U.S. Government Agency securities 2,884 31 (34) 2,881 Total $ 29,736 $ 169 $ (189) $ 29,716 |
Property and Equipment
Property and Equipment | 12 Months Ended |
Dec. 31, 2023 | |
Property and Equipment | |
Property and Equipment | 4. Property and Equipment Property and equipment consisted of the following (in thousands): December 31, December 31, 2023 2022 Laboratory equipment $ 4,330 $ 4,277 Leasehold improvements 899 899 Office furniture and equipment 514 514 Total property and equipment 5,743 5,690 Less: accumulated depreciation (1,900) (1,454) Total property and equipment, net $ 3,843 $ 4,236 Depreciation expense was $0.5 million and $0.3 million for the years ended December 31, 2023 and 2022, respectively. In 2023 and 2022, we determined certain of our laboratory equipment was not required and held for sale or placed into storage, and as a result, write-downs of net equipment of approximately $0.1 million and $0.8 million, respectively, were recorded as a loss and included in Other income (expense), net on the consolidated statement of operations for the years ended December 31, 2023 and 2022. We did not have any assets held for sale as of December 31, 2023. We had assets held for sale of approximately $0.7 million as of December 31, 2022, which was included in Other long-term assets on our consolidated balance sheets. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2023 | |
Accrued Expenses | |
Accrued Expenses | 5. Accrued Expenses Accrued expenses consisted of the following (in thousands): December 31, December 31, 2023 2022 Payroll and related costs $ 7,746 $ 7,061 Clinical trials and drug development 4,701 5,725 Accrued license agreement payment 4,000 — Professional fees 1,236 1,417 Accrued tax provision — 2,445 Selling and commercial liabilities 3,901 1,880 Short-term lease liabilities 774 637 Other 501 371 Total accrued expenses $ 22,859 $ 19,536 |
Leases
Leases | 12 Months Ended |
Dec. 31, 2023 | |
Leases | |
Leases | 6. Leases We have entered into one operating lease for real estate and one operating lease for clinical site equipment. Our real estate operating lease has a term of 78 months and includes renewal terms that can extend the lease term by 60 months , which is included in the lease term when it is reasonably certain that we will exercise the option. As of December 31, 2023, our operating lease for real estate had a remaining term of 21 months . Our operating lease for clinical site equipment has a term of 18 months and includes renewal terms that can extend the lease term monthly at the end of the term. As of December 31, 2023, our operating lease for clinical site equipment had a remaining term of 17 months . We expect to enter into several similar operating leases for clinical site equipment for our RAISE II trial throughout the remainder of 2024. The right-of-use (ROU) assets are included in Other assets on our balance sheets as of December 31, 2023 and 2022, and represent our right to use the underlying assets for the lease term. Our obligations to make lease payments are included in Accrued expenses and Other long-term liabilities on our balance sheets as of December 31, 2023 and 2022. The ROU assets were initially measured at cost, which comprise the initial amount of the lease liability adjusted for lease payments made at or before the lease commencement date, plus any initial direct costs incurred, less any lease incentives received. The ROU assets are subsequently measured throughout the lease term at the carrying amount of the lease liability, plus initial direct costs, plus (minus) any prepaid (accrued) lease payments, less the unamortized balance of lease incentives received. As of December 31, 2023 and 2022, we had $1.0 million and $1.3 million, respectively, of ROU assets and our operating lease liabilities were $1.4 million and $2.0 million, respectively. We have entered into other various short-term operating leases, primarily for clinical trial equipment, with an initial term of twelve months or less. These leases are not recorded on our balance sheets. All operating lease expense is recognized on a straight-line basis over the lease term. During each of the years ended December 31, 2023 and 2022, we recognized an aggregate $0.6 million in total lease costs, which included less than $0.1 million in short-term lease costs related to short-term operating leases in each year. Because the rate implicit in the lease is not readily determinable, we use our incremental borrowing rate to determine the present value of the lease payments. The weighted average incremental borrowing rate used to determine the initial value of the ROU asset and lease liability related to the real estate operating lease was 11.0%. We have certain contracts for real estate which may contain lease and non-lease components which we have elected to treat as a single lease component. The borrowing rate used to determine the initial value of the ROU asset and lease liability related to our operating lease for clinical site equipment was approximately 7.0%. ROU assets for operating leases are periodically reduced by impairment losses. We use the long-lived assets impairment guidance in ASC Subtopic 360-10, Property, Plant, and Equipment – Overall, to determine whether an ROU asset is impaired, and if so, the amount of the impairment loss to recognize. As of December 31, 2023 and 2022, we have not recognized any impairment losses for our ROU assets. Maturities of operating lease liabilities as of December 31, 2023 were as follows (in thousands): 2024 $ 889 2025 662 1,551 Less: imputed interest (144) Total lease liabilities $ 1,407 Current operating lease liabilities $ 774 Non-current operating lease liabilities 633 Total lease liabilities $ 1,407 |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2023 | |
Stockholders' Equity | |
Stockholders' Equity | 7. Stockholders’ Equity In 2005, we adopted the 2005 Stock Option and Incentive Plan (2005 Plan) that authorizes us to grant options, restricted stock and other equity-based awards. As of December 31, 2023, 577 options to purchase shares of common stock were outstanding pursuant to grants in connection with the 2005 Plan. No additional shares are available for issuance under the 2005 Plan. The amount, terms of grants, and exercisability provisions are determined and set by our board of directors. Effective August 2014, we adopted our 2014 Equity Incentive Plan, as amended (2014 Plan) that authorizes us to grant options, restricted stock, and other equity-based awards, subject to adjustment in accordance with the 2014 Plan. As of December 31, 2023, . In accordance with the 2014 Plan, on January 1, 2024, the shares of common stock available for future grants under the 2014 Plan was increased to In addition, during the years ended December 31, 2023 and 2022, we granted 566,685 and 788,885 options, respectively, to purchase shares of common stock and 2,500 and 39,900 restricted stock units, respectively, outside of our 2014 Plan as inducement grants material to new employees entering into employment agreements with us pursuant to Nasdaq Listing Rule 5635(c)(4). The amount, terms of grants, and exercisability provisions of these grants are determined and set by our board of directors, and are largely consistent with the terms and exercisability provisions of grants under our 2014 Plan. Stock Options Options issued under both the 2005 Plan and 2014 occurs over a period of not greater than four years. A summary of stock option activity for the years ended December 31, 2023 and 2022 is presented below (in thousands, except share and per share amounts): Weighted ‑ Average Aggregate Exercise Price Intrinsic Shares Per Share Value Outstanding—December 31, 2021 4,738,855 $ 12.23 Granted 2,026,981 8.77 Exercised (238,312) 7.52 Forfeited (415,816) 10.49 Expired (381,489) 14.61 Outstanding—December 31, 2022 5,730,219 $ 10.87 Granted 2,117,451 6.43 Exercised (126,735) 6.35 Forfeited (267,239) 8.34 Expired (288,715) 13.34 Outstanding—December 31, 2023 7,164,981 $ 9.64 $ 17,854 Exercisable—December 31, 2023 4,620,748 $ 10.64 $ 9,292 The weighted average remaining contractual term of options outstanding and exercisable as of December 31, 2023 is 7.5 and 6.9 years, respectively. Intrinsic value in the table above was determined by calculating the difference between the market value of our common stock on the last trading day of 2023 of $10.87 per share and the exercise price for any in-the-money options. The weighted-average grant date fair value of options granted was $5.20 and $7.00 per share in 2023 and 2022, respectively, and was estimated at the date of grant using the Black-Scholes option-pricing model with the following ranges of weighted-average assumptions: 2023 2022 Expected stock price volatility 99 - 104 % 103 - 116 % Expected term of options 5.3 - 6.1 years 5.7 - 6.5 years Risk‑free interest rate 3.35 - 4.82 % 1.65 - 4.46 % Expected annual dividend yield 0 % 0 % The weighted-average valuation assumptions were determined as follows: ● Expected stock price volatility: The expected volatility is based on historical volatility of our stock price. ● Expected term of options: We estimated the expected term of our stock options with service-based vesting using the “simplified” method, as prescribed in SAB No. 107, whereby the expected life equals the average of the vesting tranches and the original contractual term of the option due to our lack of sufficient historical data. ● Risk-free interest rate: We base the risk-free interest rate on the interest rate payable on U.S. Treasury securities in effect at the time of grant for a period that is commensurate with the assumed expected option term. ● Expected annual dividend yield: The estimated annual dividend yield is 0% because we have not historically paid, and do not expect for the foreseeable future to pay, a dividend on our common stock. Restricted Stock and Restricted Stock Units All issued and outstanding restricted shares of common stock are time-based and generally become vested within two years after the grant date, pursuant to the 2014 Plan. Compensation expense is recorded ratably over the requisite service period. Compensation expense related to restricted stock is measured based on the fair value using the closing market price of our common stock on the date of the grant. As of December 31, 2023, we had During the year ended December 31, 2023, we granted 1,004,310 restricted stock units, which vest within four years of the grant date, pursuant to the 2014 Plan. Compensation expense related to restricted stock units is measured based on the fair value using the closing market price of our common stock on the date of the grant. As of December 31, 2023, we had 1,271,894 restricted stock units outstanding. A summary of activity for the years ended December 31, 2023 and 2022 is presented below: Weighted ‑ average Grant Date Shares Fair Value per Share Outstanding—December 31, 2021 26,025 $ 12.75 Granted 809,028 8.49 Vested (37,450) 11.19 Forfeited (125,627) 9.04 Outstanding—December 31, 2022 671,976 8.39 Granted 1,004,310 6.17 Vested (262,819) 8.26 Forfeited (141,573) 6.61 Outstanding—December 31, 2023 1,271,894 $ 6.86 Total compensation cost recognized for all stock options and restricted stock units in the statements of operations for the years ended December 31, 2023 and 2022 is as follows (in thousands): Year Ended December 31, 2023 2022 Research and development $ 5,806 $ 5,364 Selling, general and administrative 9,757 9,526 Total $ 15,563 $ 14,890 As of December 31, 2023, there was $15.6 million and $6.4 million of total unrecognized compensation expense related to unvested stock options and restricted stock units, respectively. That expense is expected to be recognized over the next four years as follows, in thousands: 2024 $ 12,690 2025 7,348 2026 1,549 2027 366 $ 21,953 Stock Issued in Connection with Ovid License Agreement On March 29, 2022, pursuant to an exclusive patent license agreement with Ovid Therapeutics Inc. (Ovid), we issued 123,255 shares of our common stock to Ovid. The shares were issued in reliance upon the exemption from the registration requirements of the Securities Act of 1933, as amended (Securities Act) provided by Section 4(a)(2) of the Securities Act and Regulation D thereunder as sales by an issuer not involving any public offering (see Part II, Item 2. Unregistered Sales of Equity Securities and Use of Proceeds ). The fair value of these shares is reflected in operating expenses for the year ended December 31, 2022. Underwritten Public Offering In connection with an underwritten public offering in November 2022 and the closing of the related exercise of the underwriters’ option in December 2022, we issued a total of 12,421,053 shares of common stock and 2,105,264 pre-funded warrants (the Pre-funded Warrants) resulting in aggregate net proceeds, after underwriting discounts and commissions in the public offering and fees, of $64.5 million . Sales Pursuant to Equity Distribution Agreement On July 9, 2020, we entered into an Equity Distribution Agreement (EDA) with JMP Securities LLC (JMP), as amended by the March 31, 2023 Amendment No. 1 to the EDA (Amended EDA), to create an at the market equity program under which we from time to time may offer and sell shares of our common stock without a specified maximum aggregate offering price. The Amended EDA was entered into in connection with our filing of a Registration Statement on Form S-3 (File No. 333-271041) with the SEC (the 2023 Registration Statement), which includes a prospectus supplement covering the offering, issuance and sale by us of up to $75,000,000 of shares of common stock that may be issued and sold under the Amended EDA. Subject to the terms and conditions of the Amended EDA, JMP will be entitled to a commission of up to 3.0% of the gross proceeds from each sale of shares of our common stock. In the year ended December 31, 2023, we sold approximately 3.7 million shares of our common stock pursuant to the Amended EDA, which resulted in net proceeds of approximately $25.8 million. We did no t sell any shares of our common stock during the year ended December 31, 2022 under the EDA. |
Convertible Preferred Stock
Convertible Preferred Stock | 12 Months Ended |
Dec. 31, 2023 | |
Convertible Preferred Stock. | |
Convertible Preferred Stock | 8. Convertible Preferred Stock Concurrent with a 2019 public offering, we entered into a Securities Purchase Agreement (Purchase Agreement), by and among us and the investors listed therein. Pursuant to the terms of the Purchase Agreement, we sold to the investors an aggregate of 30,000 shares of Series A Participating Convertible Preferred Stock, par value $0.001 per share (Series A Preferred Stock), at a per share price of $1,000 in a private placement, and received net proceeds of $28.2 million, after deducting underwriting discounts and commissions of $1.8 million. Each share of Series A Preferred Stock is convertible into 200 shares of common stock, reflecting a conversion price equal to $5.00 per share, subject to customary anti-dilution adjustments. During the year ended December 31, 2023, 4,300 shares of our Series A Preferred Stock converted into 860,000 shares of our common stock, pursuant to the terms of the P urchase A greement. During the year ended December 31, 2022, 275 shares of our Series A Preferred Stock converted into 55,000 shares of our Common stock, pursuant to the terms of the Purchase Agreement. As of December 31, 2023, no shares of our Series A Preferred Stock remain ed outstanding. |
Notes Payable
Notes Payable | 12 Months Ended |
Dec. 31, 2023 | |
Notes Payable | |
Notes Payable | 9. Notes Payable On May 11, 2021 (Closing Date) and as amended on May 17, 2021, May 23, 2022 and October 28, 2022 (Credit Agreement), we entered into the Credit Agreement with Oaktree Fund Administration, LLC as administrative agent (Oaktree) and the lenders party thereto (collectively, the Lenders) that provided for a five-year senior secured term loan facility in an aggregate original principal amount of up to $125.0 million that was available to us in five tranches (collectively, the Term Loans). Upon entering into the Credit Agreement in May 2021, we borrowed $15.0 million in term loans from the Lenders (Tranche A-1 Term Loans); upon receipt of written acceptance by the FDA of our NDA filing relating to the use of ganaxolone for CDD in September 2021, we borrowed $30.0 million of tranche A-2 term loans from the Lenders (Tranche A-2 Term Loans); and in March 2022, we borrowed $30.0 million in term loans from the Lenders that became available as a result of the approval by the FDA of ZTALMY oral suspension for the treatment of seizures associated with CDD in patients two years of age and older (Tranche B Term Loans). In May 2022, we entered into an amendment (the Credit Agreement Amendment) to extend the commitment date for the tranche C term loans (Tranche C Term Loans) commitment from June 30, 2023 to December 31, 2023, and to eliminate the commitment fees associated with the Tranche C Term Loans. Also in May 2022, we delivered to Oaktree a separate notice of commitment termination with respect to the tranche D term loans (Tranche D Term Loans) commitment. In October 2022, we entered into an amendment to, among other things, allow for the consummation of the Revenue Interest Financing Agreement with Sagard and the transactions thereunder. In addition, the Credit Agreement Amendment increases the exit fee due by us upon any repayment, whether as a prepayment or a scheduled repayment, of the principal of the loans under the Credit Agreement from 2.00% to 2.67%. In August 2023, we delivered to Oaktree a separate notice of commitment termination with respect to the $25.0 million of Tranche C Term Loans commitment. Following the termination of the Tranche C Term Loan Commitment, the loans under the Credit Agreement consist of $75.0 million of previously drawn Term Loans with no additional funds available thereunder. The Credit Agreement contains a minimum liquidity covenant that requires us to maintain Cash and cash equivalents of at least $15.0 million from the funding date of the tranche B term loans until the maturity of the Term Loans. The Term Loans will be guaranteed by certain of our future subsidiaries (Guarantors). Our obligations under the Credit Agreement are secured by a pledge of substantially all of our assets and will be secured by a pledge of substantially all of the assets of the Guarantors. The Term Loans mature on May 11, 2026 (Maturity Date). The Term Loans bear interest at a fixed per annum rate (subject to increase during an event of default) of 11.50%, and we are required to make quarterly interest payments until the Maturity Date. We are also required to make quarterly principal payments beginning on June 30, 2024 in an amount equal to 5.0% of the aggregate amount of the Term Loans outstanding on June 30, 2024, and continuing until the Maturity Date. On the Maturity Date, we are required to pay in full all outstanding Term Loans and other amounts owed under the Credit Agreement. At the time of borrowing any tranche of the Term Loans, we were required to pay an upfront fee of 2.0% of the aggregate principal amount borrowed at that time. In addition, a commitment fee of 75 basis points per annum began to accrue on each of the tranche B, C, and D commitments for the period beginning 120 days after the funding date of the tranche A-2 term loans and continued until the applicable tranche was either funded or terminated, at which time the related commitment fees were due. The Tranche A-2 Term Loans were funded on September 27, 2021, and as such, we began accruing the commitment fees for tranche B, C, and D Term Loans 120 days later, on January 25, 2022. We drew down the additional $30.0 million of Tranche B Term Loans in March 2022, and paid less than $0.1 million in commitment fees related to Tranche B Term Loans. The May 2022 amendment eliminated the commitment fees related to the Tranche C Term Loans, and separately, we terminated the Tranche D Term Loans in May 2022 and the Tranche C Term Loans in August 2023. We may prepay all or any portion of the Term Loans, and are required to make mandatory prepayments of the Term Loans from the proceeds of asset sales, casualty and condemnation events, and prohibited debt issuances, subject to certain exceptions. All mandatory and voluntary prepayments of the Term Loans are subject to prepayment premiums equal to (i) of the principal prepaid if prepayment occurs after May 11, 2024 but on or before May 11, 2025. If prepayment occurs after May 11, 2025, prepayment premium is due. In addition, we are required to pay an exit fee in an amount equal to 2.67% of all principal repaid, whether as a mandatory prepayment, voluntary prepayment, or a scheduled repayment. Prior to the October 28, 2022 amendment to the Credit Agreement, the exit fee was 2.0%. The increase in the exit fee resulted in an additional $0.5 million of debt issuance costs that are classified as a contra-liability on the consolidated balance sheets and is being recognized as interest expense over the term of the loan using the effective interest method. In addition, we paid $0.2 million in fees, which were recorded as debt issuance costs for the year ended December 31, 2022. In addition to the minimum liquidity covenant, we are subject to a number of affirmative and restrictive covenants under the Credit Agreement, including limitations on our ability and our subsidiaries’ abilities, among other things, to incur additional debt, grant or permit additional liens, make investments and acquisitions, merge or consolidate with others, dispose of assets, pay dividends and distributions, and enter into affiliate transactions, subject to certain exceptions. As of December 31, 2023, we were in compliance with all covenants. Upon the occurrence of certain events, including but not limited to our failure to satisfy our payment obligations under the Credit Agreement, the breach of certain of our other covenants under the Credit Agreement, the occurrence of cross defaults to other indebtedness, or defaults related to enforcement action by the FDA or other Regulatory Authority or recall of ganaxolone, Oaktree and the Lenders will have the right, among other remedies, to accelerate all amounts outstanding under the Term Loans and declare all principal, interest, and outstanding fees immediately due and payable. In March 2022, we borrowed $30.0 million upon the approval by the FDA of ZTALMY for CDD and incurred debt issuance costs of $1.8 million, including the exit fee of $0.6 million, that are classified as contra-liabilities on our consolidated balance sheets and are being recognized as Interest expense over the term of the loan using the effective-interest method. In September 2021, we borrowed $30.0 million upon receipt of written acceptance by the FDA of our NDA filing relating to the use of ganaxolone in the treatment of CDD and incurred debt issuance costs of $1.2 million, including the exit fee of $0.6 million, that are classified as contra-liability on our consolidated balance sheets and are being recognized as Interest expense over the term of the loan using the effective-interest method. In May 2021, we borrowed $15.0 million upon entering into the Credit Agreement and incurred debt issuance costs of $4.4 million, including the exit fee of $0.3 million, that are classified as a contra-liability on the consolidated balance sheet and are being recognized as Interest expense over the term of the loan using the effective-interest method. For the year ended December 31, 2023, we recognized Interest expense of $10.7 million, of which $8.7 million was cash interest paid on the Term Loans and $2.0 million was non-cash interest expense related to the amortization of debt issuance costs. For the year ended December 31, 2022, we recognized Interest expense of $9.5 million, of which $7.9 million was cash interest paid on the Term Loans and $1.6 million was non-cash Interest expense related to the amortization of debt issuance costs. The following table summarizes the composition of Notes payable as reflected on the consolidated balance sheet as of December 31, 2023 (in thousands): Gross proceeds $ 75,000 Contractual exit fee 2,003 Unamortized debt discount and issuance costs (4,029) Total note payable $ 72,974 Current portion of note payable 11,551 Non-current portion of note payable 61,423 Total note payable $ 72,974 The aggregate maturities of Notes payable as of December 31, 2023 are as follows (in thousands): 2024 $ 11,250 2025 15,000 2026 48,750 Total $ 75,000 |
Revenue Interest Financing Paya
Revenue Interest Financing Payable | 12 Months Ended |
Dec. 31, 2023 | |
Revenue Interest Financing Payable | |
Revenue Interest Financing Payable | 10. Revenue Interest Financing Payable On October 28, 2022 (Closing Date), we entered into a revenue interest financing agreement (Revenue Interest Financing Agreement) with Sagard Healthcare Royalty Partners, LP (Sagard) to provide funding for our development and commercialization of ganaxolone and related pharmaceutical products, including the commercial launch of ZTALMY, and for working capital and general administrative purposes. In exchange for the Investment Amount, we have agreed to make quarterly payments to Sagard (Payments) as follows: (i) for each calendar quarter from and after the Closing Date through and including the quarter ended June 30, 2026, an amount equal to 7.5% of (a) our U.S. net sales of ZTALMY and all other pharmaceutical products that contain ganaxolone (Net Sales), in each case with any dosage form, dosing regimen, or strength, or any improvements related thereto (collectively, Included Products) and (b) certain other payments received by us in connection with the manufacture, development and sale of the Included Products in the U.S. (Other Included Payments, and, together with Net Sales, Product Revenue); and (ii) for each calendar quarter following the calendar quarter ended June 30, 2026, an amount equal to (x) 15.0% of the first $100 million in annual Product Revenue of the Included Products and (y) 7.5% of annual Product Revenue of the Included Products in excess of $100 million. The Payments are subject to a hard cap equal to 190% of the Investment Amount (Hard Cap) or $61.8 million. Sagard’s right to receive payments will terminate when Sagard has received payments in respect of the Included Products, including any additional payments described below, equal to the Hard Cap. Further, we have the right to make voluntary prepayments to Sagard, and such payments will be credited against the Hard Cap. If Sagard has not received aggregate payments equaling at least 100% of the Investment Amount by December 31, 2027 or at least 190% of the Investment Amount by December 31, 2032 (each, a Minimum Amount), then we will be obligated to make a cash payment to Sagard in an amount sufficient to gross up Sagard up to the applicable Minimum Amount within a specified period of time after each reference date. The obligations under the Revenue Interest Financing Agreement, including the Payments, will be guaranteed by certain of our future subsidiaries that are required to become a party thereto as guarantors (Guarantors). Our obligations under the Revenue Interest Financing Agreement and the guarantee of such obligations are secured, subject to customary permitted liens and other agreed upon exceptions and subject to an intercreditor agreement with Oaktree as administrative agent for the lenders under our credit agreement (as described below, the Credit Agreement), by a pledge of substantially all of our and the Guarantors’ assets that relate to, or are used or held for use for, the development, manufacture, use and/or commercialization of ZTALMY and all other pharmaceutical products that contain ganaxolone in the U.S., including the Product Revenue, pursuant to the terms of the Security Agreement dated as of the Closing Date by and among us, the Guarantors from time to time party thereto, and Sagard (Security Agreement). At any time, we have the right, but not the obligation (Call Option), to repurchase all, but not less than all, of Sagard’s interest in the Payments at a repurchase price (Put/Call Price) equal to: (a) on or before the third anniversary of the Closing Date, 160% of the Investment Amount; (b) after the third anniversary but on or prior to the fourth anniversary of the Closing Date, 180% of the Investment Amount; and (c) after the fourth anniversary of the Closing Date, 190% of the Investment Amount, in each case, less the aggregate of all of our payments in respect of the Payments made to Sagard prior to such date. The Revenue Interest Financing Agreement contains certain restrictions on our and our subsidiaries’ abilities, among other things, to incur additional debt, grant or permit additional liens, make investments and acquisitions, dispose of assets, pay dividends and distributions and enter into affiliate transactions, in each case, subject to certain exceptions. In addition, the Revenue Interest Financing Agreement contains a financial covenant that requires us to maintain at all times Cash and cash equivalents in certain deposit accounts in an amount at least equal to (i) from the Closing Date until the repayment of the loans under the Credit Agreement, $15.0 million and (ii) thereafter, $10.0 million. In connection with the Revenue Interest Financing Agreement, on the Closing Date, we entered into the Credit Agreement Amendment with Oaktree which is fully described in Note 9. legal fees Revenue Interest Financing Agreement upon October 2022 closing $ 32,500 Issuance costs in the year ended December 31, 2022 (2,579) Non-cash interest expense in the year ended December 31, 2022 956 Amortization of debt discount in the year ended December 31, 2022 42 Payments made in the year ended December 31, 2022 (42) Revenue Interest Financing Balance at December 31, 2022 $ 30,877 Non-cash interest expense in the year ended December 31, 2023 5,950 Amortization of debt discount in the year ended December 31, 2023 294 Payments made in the year ended December 31, 2023 (1,144) Revenue Interest Financing Balance at December 31, 2023 $ 35,977 Current portion of revenue interest financing liability $ 2,211 Long-term portion of revenue interest financing liability 33,766 Revenue Interest Financing Balance at December 31, 2023 $ 35,977 |
License and Collaboration Reven
License and Collaboration Revenue | 12 Months Ended |
Dec. 31, 2023 | |
License and Collaboration Revenue | |
License and Collaboration Revenue | 11. License and Collaboration Revenue Orion Collaboration Agreement In July 2021, we entered into a collaboration agreement (Orion Collaboration Agreement) with Orion. The Orion Collaboration Agreement falls under the scope of ASC Topic 808, Collaborative Arrangements (ASC 808) as both parties are active participants in the arrangement that are exposed to significant risks and rewards. While this arrangement is in the scope of ASC 808, we analogize to ASC 606 for some aspects of this arrangement, including for the delivery of a good or service (i.e., a unit of account). Revenue recognized by analogizing to ASC 606 is recorded as Collaboration revenue on the consolidated statements of operations. Under the terms of the Orion Collaboration Agreement, we granted Orion an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights with respect to commercializing biopharmaceutical products incorporating our product candidate ganaxolone (Licensed Products) in the European Economic Area, the United Kingdom and Switzerland (collectively, the Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially in the indications of CDD, TSC and RSE. We will be responsible for the continued development of Licensed Products and regulatory interactions related thereto, including conducting and sponsoring all clinical trials, provided that Orion may conduct certain post-approval studies in the Territory. Orion will be responsible, at Orion’s sole cost and expense, for the commercialization of any Licensed Product in the Field in the Territory. Under the terms of the Orion Collaboration Agreement, we received a €25.0 million ( $29.6 million) upfront payment from Orion in July 2021. In connection with the upfront fee, we agreed to provide Orion with the results of a planned genotoxicity study on the M2 metabolite of ganaxolone, a “Combined Micronucleus & Comet study in vivo.” In May 2022, the final study report was received, which confirmed that no genotoxicity was found, as measured by formation of micronuclei in the bone marrow or comet morphology in the liver. In the event that the results of such study were positive, based on the criteria set forth in the study’s protocol, Orion would have had the right to terminate the Orion Collaboration Agreement within ninety ( 90 ) days after its receipt of the final report of such study, in which case we would have been required to refund Orion seventy-five percent ( 75% ) of the upfront fee. We are eligible to receive up to an additional €97 million in R&D reimbursement and cash milestone payments based on specific clinical and commercial achievements, as well as tiered royalty payments based on net sales ranging from the low double-digits to high teens for the oral programs and the low double-digits to low 20s for the IV program. Also, as part of the overall arrangement, we have agreed to supply the Licensed Products to Orion at an agreed upon price. The Orion Collaboration Agreement shall remain effective until the date of expiration of the last to expire Royalty Term, which is defined as the period beginning on the date of the first commercial sale Licensed Product in such country and ending on the latest to occur of (a) the tenth (10th) anniversary of the first commercial sale of Licensed Product in such country, (b) the expiration of the last-to-expire licensed patent covering the manufacture, use or sale of such Licensed Product in such country, and (c) the expiration of regulatory exclusivity period, if any, for such Licensed Product in such country. The Orion Collaboration Agreement has a term of at least ten ( 10 ) years since a commercial sale has yet to occur. The Orion Collaboration Agreement allows for termination in certain specific events, such as material breach, in the event Orion challenges the validity, enforceability or scope of the licensed patent rights, termination for forecast failure, insolvency and force majeure, none of which are probable at contract inception. In accordance with the guidance, we identified the following commitments under the arrangement: (i) exclusive rights to develop, use, sell, have sold, offer for sale and import any product comprised of Licensed Product (License) (ii) development and regulatory activities (Development and Regulatory Activities), and (iii) requirement to supply Orion with the Licensed Product at an agreed upon price (Supply of Licensed Product). We determined that these three commitments represent distinct performance obligations for purposes of recognizing revenue or reducing expense, which we will recognize such revenue or expense, as applicable, as we fulfill these performance obligations. At contract inception, we determined that the non-refundable portion of the upfront payment plus the research and development reimbursement constitutes the transaction price as of the outset of the Orion Collaboration Agreement. The refundable portion of the upfront payment and the future potential regulatory and development milestone payments were fully constrained at contract inception as the risk of significant revenue reversal related to these amounts had not yet been resolved. During 2022, the refundable portion of the upfront payment was determined to be included in the transaction price as the final genotoxicity study on the M2 metabolite of ganaxolone was received as described above and the remaining The transaction price was allocated to the three performance obligations based on the estimated stand-alone selling prices at contract inception. The stand-alone selling price of the License was based on a discounted cash flow approach and considered several factors including, but not limited to, discount rate, development timeline, regulatory risks, estimated market demand and future revenue potential using an adjusted market approach. The stand-alone selling price of the Development and Regulatory Activities and the Supply of Licensed Product was estimated using the expected cost-plus margin approach. As of December 31, 2022, we allocated the transaction price to the performance obligations as described below and recorded the remaining $12.7 million of the upfront payment as Collaboration revenue during the year ended December 31, 2022. During 2022, we amortized of $15.1 million was offset by the Contract asset of $5.1 million related to the reimbursement of research and development costs, resulting in a net Contract liability of $10.0 million at December 31, 2022. Transaction Price and Net Contract Asset at December 31, 2022 (in thousands): Cumulative Collaboration Transaction Revenue Recognized Contract Price as of December 31, 2022 Liability License $ 21,660 $ 21,660 $ - Development and Regulatory Services 6,717 1,158 5,559 Supply of Licensed Product 9,503 - 9,503 $ 37,880 $ 22,818 $ 15,062 Less Total Contract Asset 5,079 Net Contract Liability $ 9,983 During the year ended December 31, 2023, we amortized $1.4 million of the transaction price associated with the Development and Regulatory Services as a reduction of research and development costs. These reductions to the transaction price resulted in a total Contract liability of $13.7 million at December 31, 2023. In accordance with ASC 10-20, the Contract liability of $13.7 million is offset by the Contract asset of $2.9 million related to the reimbursement of research and development costs, resulting in a net Contract liability of $10.8 million at December 31, 2023. Transaction Price and Net Contract Asset at December 31, 2023 (in thousands): Cumulative Collaboration Transaction Revenue Recognized Contract Price as of December 31, 2023 Liability License $ 21,660 $ 21,660 $ - Development and Regulatory Services 6,717 2,511 4,206 Supply of Licensed Product 9,503 - 9,503 $ 37,880 $ 24,171 $ 13,709 Less Total Contract Asset 2,912 Net Contract Liability $ 10,797 We incurred $2.0 million of incremental costs in obtaining the Orion Collaboration Agreement. These contract acquisition costs were allocated consistent with the transaction price, resulting in $1.1 million of expense recorded to Selling, general and administrative expense commensurate with the recognition of the License performance obligation and $0.9 million recorded as capitalized contract costs, included in Prepaid expenses and other current assets and Other assets, which are being amortized as Development and Regulatory Services and Supply of License Product obligations are met. Tenacia Collaboration Agreement On November 16, 2022 (Effective Date), we entered into a Collaboration and Supply Agreement (Tenacia Collaboration Agreement) with Tenacia Biotechnology (Shanghai) Co., Ltd. (Tenacia). The Tenacia Collaboration Agreement falls under the scope of ASC Topic 808, Collaborative Arrangements (ASC 808) as both parties are active participants in the arrangement that are exposed to significant risks and rewards. While this arrangement is in the scope of ASC 808, we analogize to ASC 606 for some aspects of this arrangement, including for the delivery of a good or service (i.e., a unit of account). Revenue recognized by analogizing to ASC 606 is recorded as Collaboration revenue on the consolidated statements of operations. Under the terms of the Tenacia Collaboration Agreement, we granted Tenacia an exclusive, royalty-bearing, sublicensable license to certain of our intellectual property rights to develop, commercialize and otherwise exploit certain products incorporating certain oral and intravenous formulations of our product candidate ganaxolone (Licensed Products) in Mainland China, Hong Kong, Macau and Taiwan (collectively, Territory) for the diagnosis, prevention and treatment of certain human diseases, disorders or conditions (Field), initially for the treatment of cyclin-dependent kinase-like 5 deficiency disorder, tuberous sclerosis complex and SE (including refractory and established SE) (collectively, the Initial Indications). The collaboration can be expanded to include additional indications and formulations of ganaxolone pursuant to a right of first negotiation. Under the terms of the Tenacia Collaboration Agreement, Tenacia agreed to pay us an upfront cash payment of $10 million (the Upfront Fee) within forty-five ( 45 ) days after the Effective Date, which was paid in December 2022. In addition to the Upfront Fee, Tenacia has agreed to make cash payments to us upon the achievement of certain development, regulatory and sales-based milestones related to (i) the Initial Indications and (ii) the first new formulation or pro-drug of ganaxolone or any back-up compound of ganaxolone in a new indication (Selected Product) for which the parties amend the Tenacia Collaboration Agreement in connection with Tenacia’s exercise of its right of first negotiation and for which there is no other Licensed Product approved in China (for clarity, the milestone payments under this clause (ii) will only apply to one Selected Product), up to an aggregate amount of $256 million. Of the milestones, $15 million relates to regulatory approvals with separate milestones related to each of oral and intravenous formulations and the Selected Product, and an aggregate of $241 million of sales-based milestones are connected to annual revenue thresholds specific to each of the oral, intravenous and Selected Product formulations of ganaxolone. Tenacia has further agreed to pay us tiered royalty payments based on annual net sales of Licensed Products ranging from the low double digits to the mid-teens for each of the oral formulation, intravenous formulation and Selected Product formulation of Licensed Products. Tenacia’s obligations to pay royalties to us with respect to sales of a Licensed Product in each particular jurisdiction of the Territory will commence on the date of first commercial sale in such jurisdiction and expire upon the latest of (i) ten years following the first commercial sale of such Licensed Product in such jurisdiction, (ii) the expiration of the last-to-expire valid claim of any licensed patent rights that covers such Licensed Product in such jurisdiction and (iii) the expiration of all regulatory exclusivities for such Licensed Product in such jurisdiction. Royalty payments are subject to reduction in specified circumstances as set forth in the Tenacia Collaboration Agreement, including if net sales decrease by a certain percentage after the introduction of a generic product. Tenacia will be primarily responsible for the development of Licensed Products in the Territory and regulatory interactions related thereto, including conducting and sponsoring clinical studies in the Field in the Territory to support regulatory filings in the Territory. All regulatory approvals filed by Tenacia in the Territory will be in the name of and owned by us unless otherwise required by applicable law, in which case such regulatory approvals would be in the name of and owned by Tenacia for the benefit of us. We and Tenacia have agreed to enter into clinical and commercial supply agreements pursuant to which we will supply Tenacia with its requirements of Licensed Products necessary for Tenacia to develop and commercialize Licensed Products in the Field in the Territory. The parties entered into the clinical and commercial supply agreement in May 2023. The agreement contains pricing, delivery, acceptance, payment, termination, forecasting, and other terms consistent with the Tenacia Collaboration Agreement, as well as certain quality assurance, indemnification, liability and other standard industry terms. Tenacia will be responsible for, at Tenacia’s sole cost and expense, obtaining regulatory approval and commercializing the Licensed Product in the Field in Mainland China. Tenacia is enrolling patients in our Phase 3 randomized, double blind, placebo-controlled trial (TrustTSC trial) of adjunctive ganaxolone. The term of the Tenacia Collaboration Agreement extends for so long as royalties are payable anywhere in the Territory. Subject to the terms of the Tenacia Collaboration Agreement, (i) for a specified period of time after the Effective Date, Tenacia may terminate the Tenacia Collaboration Agreement in its entirety for any or no reason upon written notice to us, and (ii) either party may terminate the Tenacia Collaboration Agreement for the other party’s material breach following a cure period or insolvency. In accordance with the guidance, we identified the following commitments under the arrangement: (i) grant to Tenacia the exclusive rights to develop, commercialize and otherwise exploit Licensed Product in the Field in the Territory (License) and (ii) requirement to supply Tenacia with the Licensed Product at an agreed upon price (Supply of Licensed Product). We determined that these two commitments represent distinct performance obligations for purposes of recognizing revenue or reducing expense, which it will recognize such revenue or expense, as applicable, as it fulfills these performance obligations. The transaction price was allocated to the two performance obligations based on the estimated stand-alone selling prices at contract inception. The stand-alone selling price of the License was based on a discounted cash flow approach and considered several factors including, but not limited to, discount rate, development timeline, regulatory risks, estimated market demand and future revenue potential using an adjusted market approach. The stand-alone selling price of the Supply of Licensed Product was estimated using the expected cost-plus margin approach. As of December 31, 2022, we allocated the transaction price to the performance obligations as described below. There was no activity during the year ended December 31, 2023. The cumulative Collaboration revenue recognized as of December 31, 2023 is $3.0 million, which was the transaction price associated with the License as revenue for the year ended December 31, 2022. No license revenue was recorded during the year ending December 31, 2023. The total Contract liability of $7.0 million is offset by the Contract asset of $0.7 million, resulting in a net Contract liability of $6.3 million as of each December 31, 2023 and 2022. Transaction Price and Net Contract Liability at December 31, 2023 and 2022: Cumulative Collaboration Transaction Revenue Recognized Contract Price as of each December 31, 2023 and 2022 Liability License $ 2,998 $ 2,998 $ - Supply of Licensed Product 7,002 - 7,002 $ 10,000 $ 2,998 $ 7,002 Less Total Contract Asset 700 Net Contract Liability $ 6,302 We incurred $1.0 million of incremental costs in obtaining the Tenacia Collaboration Agreement. These contract acquisition costs were allocated consistent with the transaction price, resulting in $0.1 million of expense recorded to Selling, general and administrative expense and $0.2 million recorded to Cost of collaboration revenue in the period ended December 31, 2022, commensurate with the recognition of the License performance obligation, and $0.7 million recorded as capitalized contract costs, which will be amortized as Supply of License Product obligations are met. Biologix Distribution and Supply Agreement In May 2023, we entered into an exclusive distribution and supply agreement (Biologix Agreement) with Biologix FZCo (Biologix), whereby Biologix has the right to distribute and sell ganaxolone in Algeria, Bahrain, Egypt, Iraq, Jordan, Kingdom of Saudi Arabia, Kuwait, Lebanon, Libya, Morocco, Oman, Qatar, Tunisia and United Arab Emirates. In exchange for these rights, we will be the exclusive supplier of our products to Biologix on terms set forth in the respective agreements in exchange for a negotiated purchase price for the products. Upon execution of the Biologix Agreement, we received an upfront payment of $0.5 million which is to be recognized over the term of the Biologix agreement. We may be entitled to additional fees upon regulatory milestones. In the period ended December 31, 2023, we recorded $0.1 million of Collaboration revenue and Cost of collaboration revenue of less than $0.1 million related to the Biologix Agreement. There was a total Contract liability of $0.4 million at December 31, 2023. As the Biologix Agreement was entered into in May 2023, there was no Contract liability at December 31, 2022. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2023 | |
Commitments and Contingencies | |
Commitments and Contingencies | 12. Commitments and Contingencies Employee Benefit Plan We maintain a Section 401(k) retirement plan for all employees. The plan allows employees to make contributions up to a specified percentage of their compensation, subject to maximum amounts allowed under law, and we contribute 3% of compensation to each employee’s 401(k) retirement account. We contributed $1.1 million and $1.0 million for the years ended December 31, 2023 and 2022, respectively. We also can make discretionary profit sharing contributions, which would vest over a period of four years from each employee’s commencement of employment with us. We have not made any discretionary contributions. License Agreements We are obligated to pay royalties pursuant to a license agreement with Purdue Neuroscience Company (Purdue) as a percentage of net product sales for direct licensed products, such as ganaxolone. The obligation to pay royalties expires, on a country-by-country basis, 10 years from the first commercial sale of a licensed product in each country. The agreement also requires that we pay Purdue a percentage of the non-royalty consideration that we receive from a sublicensee and a percentage of milestone payments for indications other than seizure disorders and vascular migraine headaches not associated with mood disorders. Under the license agreement, we are committed to use commercially reasonable efforts to develop and commercialize at least one licensed product. ZTALMY, our first FDA approved product, became available for commercial sale and shipment in the third quarter of 2022. We recorded ZTALMY net Product revenue of $19.6 million and $2.9 million for the years ended December 31, 2023 and 2022, respectively. On July 28 , In connection with the November 2022 execution of the Tenacia Collaboration Agreement, we paid to Purdue a portion of the upfront payment we received from Tenacia pursuant to the Purdue license agreement, a portion of which was recorded as a Contract asset and a portion of which was recorded as Cost of collaboration revenue for the year ended December 31, 2022. Details of the Tenacia Collaboration Agreement are more fully described in Note 11. In March 2022, we entered into an exclusive patent license agreement (License Agreement) with Ovid Therapeutics Inc. (Ovid). Under the License Agreement, we have an exclusive, non-transferable (except as provided in the License Agreement), royalty-bearing, sublicensable license under certain of Ovid’s patent(s) and patent applications to develop, make, have made, commercialize, promote, distribute, sell, offer for sale and import, ganaxolone, including any analogues or derivatives, including its salts, and pharmaceutical formulations of the foregoing (Licensed Products), in the U.S., the member states of the EU, Iceland, Lichtenstein, Norway, the United Kingdom, and Switzerland (Territory) for the treatment of CDD in humans (Field). Under the License Agreement, we have the sole right and responsibility for, and control over, all development, manufacturing, and commercialization activities, including all regulatory activities, with respect to the Licensed Products in the Field in the Territory. In addition, all regulatory approvals and related filings with respect to the Licensed Products in the Field in the Territory will be in the name of, and be owned solely by, us. We were required, at Ovid’s option exercisable in accordance with the License Agreement, to (i) pay to Ovid the sum of $1.5 million in cash; or (ii) issue to Ovid 123,255 shares of our common stock, which option to obtain shares of our common stock was exercisable within the five -business day period following the filing of our Annual Report on Form 10-K for the year ended December 31, 2021 on March 24, 2022. On March 29, 2022, we issued In March 2017, we entered into a License Agreement and a Supply Agreement with CyDex Pharmaceuticals, Inc. (CyDex). Under the terms of the License Agreement, CyDex granted us an exclusive license to use CyDex’s sulfobutylether beta-cyclodextrin, or Captisol®, drug formulation system and related intellectual property in connection with the development and commercialization of ganaxolone in any and all therapeutic uses in humans, with some exceptions. As consideration for this license, we paid an upfront fee and are required to make additional payments in the future upon achievement of various specified clinical and regulatory milestones. We will also be required to pay royalties to CyDex on sales of ganaxolone, if successfully developed, in the low-to-mid single digits based on levels of annual net sales. As of March 5, 2024, we have achieved one milestone under the License Agreement, which occurred and was paid in the first quarter of 2021 . If approved, a second milestone will be due upon approval of an NDA by the FDA for our IV formulation of ganaxolone. Certain patents relating to Captisol®, including some that were licensed to us by CyDex, have expired, while other patents that are licensed to us remain in force. Under the terms of the Supply Agreement, we are required to purchase all of our requirements for Captisol with respect to ganaxolone from CyDex, and CyDex is required to supply us with Captisol for such purposes, subject to certain limitations. |
(Loss) Gain from Sale of Priori
(Loss) Gain from Sale of Priority Review Voucher (PRV) | 12 Months Ended |
Dec. 31, 2023 | |
(Loss) Gain from Sale of Priority Review Voucher (PRV) | |
(Loss) Gain from Sale of Priority Review Voucher (PRV) | 13. (Loss) Gain from Sale of Priority Review Voucher (PRV) On July 13, 2022, we entered into an asset purchase agreement (Purchase Agreement) with Novo Nordisk Inc. (Buyer), pursuant to which we agreed to sell our Priority Review Voucher (PRV) to Buyer. The United States Secretary of Health and Human Services, FDA, issued the PRV to us on March 18, 2022 in connection with the approval of the use of ZTALMY. Pursuant to the Purchase Agreement, Buyer agreed to pay us $110.0 million, payable in cash, upon the closing of the transaction which occurred in the third quarter of 2022. We recorded a one-time net gain from the sale of the PRV in the year ended December 31, 2022. We installments, the first on or before March 15, 2024, and the second on or before June 15, 2024. We recorded a one-time net loss of $4.0 million in the year ended December 31, 2023 related to this payment owed to Purdue in connection with the sale of the PRV. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2023 | |
Income Taxes | |
Income Taxes | 14. Income Taxes Year Ended December 31, 2023 2022 Current U.S. State $ (1,538) 2,445 Foreign — 944 Total current (1,538) 3,389 Deferred — — (Benefit) provision for income taxes $ (1,538) $ 3,389 Loss before income taxes is allocated as follows (in thousands): Year Ended December 31, 2023 2022 U.S. operations $ 142,943 $ 16,427 Foreign operations — — Loss before income taxes $ 142,943 $ 16,427 As of December 31, 2023 and 2022, we had approximately $292.2 million and $233.5 million respectively, of net operating loss (NOL) carry forwards available to offset future federal taxable income, $81.1 million of which will expire beginning in 2029, and the remaining amount can be carried forward indefinitely. As of December 31, 2023 and 2022, we had approximately $240.1 million and $204.6 million, respectively, of NOL carry forwards available to offset future state taxable income that will expire beginning in 2025. As of December 31, 2023, we also have federal research and development credit carryovers of approximately $39.2 million and state credit carryovers of approximately $0.4 million, which expire beginning in 2026. The NOL carry forwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. NOL and tax credit carry forwards are subject to an annual limitation due to certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, as well as similar state tax provisions. This limits the amount of NOLs that we can utilize annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to an ownership change. Subsequent ownership changes may further affect the limitation in future years. In addition, U.S. tax laws limit the time during which these carry forwards may be applied against future taxes, therefore, we may not be able to take full advantage of these carry forwards for federal income tax purposes. The components of the net deferred tax asset are as follows (in thousands): December 31, 2023 2022 Gross deferred tax assets: Net operating loss carryforwards $ 72,792 $ 58,056 Accrued expenses 1,754 124 Amortization of intangible 468 454 Stock‑based compensation 8,741 8,091 Research and development and other credits and other carryforwards 39,588 30,627 Lease liability 345 440 Capitalized research and development expenses 37,643 22,382 Royalty/ deferred revenue 4,298 3,781 Capital loss 74 94 Interest carryforward 2,152 — Other 152 64 Total gross deferred tax assets $ 168,007 $ 124,113 Gross deferred tax liabilities: ROU asset (237) (291) Depreciation (458) (224) Unrealized income (209) (192) Total gross deferred tax liabilities (904) (707) Net deferred tax assets 167,103 123,406 Less: valuation allowance (167,103) (123,406) Net deferred tax assets after valuation allowance $ — $ — In assessing the realizability of deferred tax assets, management considers whether it is more likely than not that some portion or all of the deferred income tax assets will not be realized. The ultimate realization of deferred income tax assets is dependent upon the generation of future taxable income during the periods in which those temporary differences become deductible. Management considers the scheduled reversal of deferred income tax liabilities, projected future taxable income, and tax planning strategies in making this assessment. Based on consideration of these items, management has determined that enough uncertainty exists relative to the realization of the deferred income tax asset balances to warrant the application of a full valuation allowance as of December 31, 2023 and 2022. The valuation allowance increased by $43.7 million and $7.7 million during the years ended December 31, 2023 and 2022, respectively. The increase for the year ended December 31, 2023 was due primarily to our increase in research and development credits and capitalized research and development expenses, as well as our net operating losses. The increase for the year ended December 31, 2022 was due primarily to our increase in research and development credits and capitalized research and development expenses, partially offset by a decrease in net operating losses. We did not have unrecognized tax benefits as of December 31, 2023 and 2022, and do not expect this to change significantly over the next twelve months. We recognize tax positions in the financial statements only when it is more likely than not that the position will be sustained on examination by the relevant taxing authority based on the technical merits of the position. A position that meets this standard is measured at the largest amount of benefit that will more likely than not be realized on settlement. A liability is established for differences between positions taken in a tax return and amounts recognized in the financial statements. Accrued interest and penalties, where appropriate, are recorded in income tax expense. We did not have uncertain tax positions as of December 31, 2023 and 2022. As of December 31, 2023 and 2022, we had not accrued interest or penalties related to any uncertain tax positions. The Tax Cuts and Jobs Act passed in 2017 included a provision which would require taxpayers to capitalize and amortize U.S.-based research & experimentation (R&E) expenses over a period of five years and non-U.S. R&E expenses over 15 years effective for tax years beginning after December 31, 2021 pursuant to Internal Revenue Code Section 174. We did not have a federal or state income tax liability for the year ended December 31, 2023 and the income tax benefit of $1.5 million is a result of return to accrual adjustments from the filing of the 2022 state tax returns. As a result of the capitalization of R&E expenses, income generated from the sale of the PRV, and limitations related to the utilization of state net operating losses, we had income tax expense of $2.4 million for the year ended December 31, 2022 attributable to state income taxes. We did not have a federal income tax liability for the year ended December 31, 2022 due to the utilization of our NOLs after taking into account Internal Revenue Code Section 382 limitations related to changes in ownership. Also included in our income tax expense for the year ended December 31, 2022 was $0.9 million of China withholding tax incurred in connection with the Tenacia Collaboration Agreement. A reconciliation of income tax (expense) benefit at the statutory federal income tax rate and income taxes as reflected in the financial statements is as follows: Year Ended December 31, 2023 2022 Federal income tax expense at statutory rate 21.0 % 21.0 % Permanent items (0.4) (2.6) State income tax, net of federal benefit 4.1 (9.0) R&D tax credits 6.3 88.1 Change in state apportionment 2.5 (59.5) Foreign withholding tax — (5.7) Change in valuation allowance (30.6) (47.0) Other (1.8) (5.9) Effective income tax rate 1.1 % (20.6) % For all years through December 31, 2023, we generated research and development credits but have not conducted a study to document the qualified activities. This study may result in an adjustment to our research and development credit carryforwards; however, until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position for these years. A full valuation allowance has been provided against our research and development credits and, if an adjustment is required, this adjustment to the deferred tax asset established for the research and development credit carryforwards would be offset by an adjustment to the valuation allowance. We file income tax returns in the U.S. and various state jurisdictions. The federal and state income tax returns are generally subject to tax examinations for the tax years ended December 31, 2020 through December 31, 2022. To the extent we have tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service or state tax authorities to the extent utilized in a future period. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
Summary of Significant Accounting Policies | |
Principles of Consolidation | Principles of Consolidation The financial statements include the accounts of Marinus Pharmaceuticals, Inc. and its wholly-owned subsidiary as of December 31, 2023. In February 2021, a wholly-owned subsidiary was established in Ireland. All intercompany accounts and transactions have been eliminated. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with generally accepted accounting principles (GAAP) requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from such estimates. |
Segment Information | Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. We view our operations and manage our business in one segment, which is the identification and development of innovative therapeutics to treat rare seizure disorders. |
Fair Value of Financial Instruments and Credit Risk | Fair Value of Financial Instruments and Credit Risk Cash equivalents and Short-term investments subject us to concentrations of credit risk. However, we invest our cash in accordance with a policy objective that seeks to ensure both liquidity and safety of principal. The policy limits investments to instruments issued by the U.S. government, certain Securities and Exchange Commission (SEC)-registered money market funds that invest only in U.S. government obligations and various other low-risk liquid investment options, and places restrictions on portfolio maturity terms. |
Cash and Cash Equivalents | Cash and Cash Equivalents We consider all highly liquid investments that have maturities of three months or less when acquired to be cash equivalents. As of December 31, 2023 and 2022, we invested a portion of our cash balances in money market investments, which we have included as Cash equivalents on our balance sheets. Additionally, as of December 31, 2023, we invested a portion of our cash balances in U.S Treasury securities and U.S Government Agency securities, which we have included as Short-term investments on our balance sheet. |
Short-Term Investments | Short-Term Investments We classify our Short-term investments as available-for-sale securities, which include U.S. government agency debt securities and U.S. treasury debt securities with original maturities of greater than three months. These securities are carried at fair market value, with unrealized gains and losses reported in Other comprehensive loss and Accumulated other comprehensive loss within stockholders’ equity. All of our investments were short-term in nature as of December 31, 2023. We did not have any investments as of December 31, 2022. |
Accounts Receivable, net | Accounts Receivable, net Net trade receivables related to ZTALMY sales, which are recorded in Accounts receivable, net on the consolidated balance sheets, were approximately $2.6 million and $1.3 million as of December 31, 2023 and December 31, 2022, respectively. We had no allowance for doubtful accounts as of December 31, 2023 or 2022. An allowance for doubtful accounts is determined based on our assessment of the credit worthiness and financial condition of our customers, aging of receivables, as well as the general economic environment. Any allowance would reduce the net receivables to the amount that is expected to be collected. We have three customers, one of which, Orsini Pharmaceutical Services, LLC (Orsini), a specialty pharmacy that dispenses ZTALMY directly to patients, represents approximately 99% of our ZTALMY revenue to date. Payment terms for Orsini are 30 days from the shipment date. Excluding net trade receivables, Accounts receivable, net represents amounts due to us under the BARDA contract for valid expenditures expected to be reimbursed to us under the terms of the BARDA contract and current amounts due to us from Orion Corporation (Orion) under the collaboration agreement (Note 11). |
Inventory | Inventory Inventories are recorded using actual costs and may consist of raw materials (ganaxolone API), work in process and finished goods. We began capitalizing Inventory related to ZTALMY subsequent to the March 2022 FDA approval of ZTALMY, as the related costs were expected to be recoverable through the commercialization and subsequent sale of ZTALMY. Prior to FDA approval of ZTALMY, costs estimated at approximately $2 million for commercially saleable product and materials were incurred and included in Research and development expenses. As a result, Cost of product revenues related to ZTALMY will initially reflect a lower average per unit cost of materials into approximately the first half of 2024, as previously expensed inventory is utilized for commercial production and sold to customers. |
Prepaid Expenses and Other Current Assets | Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets generally represent payments made for goods or services to be received within one year, and are expensed as the related benefit is received. |
Property and Equipment, net | Property and Equipment, net Property and equipment, net consist of laboratory and office equipment and are recorded at cost. Property and equipment are depreciated on a straight-line basis over their estimated useful lives. We estimate a life of five years for office equipment and furniture, five to fifteen years for laboratory equipment, and the lesser of the lease term or useful life for leasehold improvements. When property and equipment are sold or otherwise disposed of, the cost and related accumulated depreciation are removed from the accounts and the resulting gain or loss is included in Other income (expense), net. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets We review long-lived assets, including property and equipment, for impairment whenever events or changes in business circumstances indicate that the carrying amount of an asset may not be fully recoverable. If the estimated undiscounted future cash flows expected to result from the use of the asset and its eventual disposition is less than its carrying amount an impairment loss would be recognized if the carrying value of the asset exceeded its fair value. Fair value is generally determined using discounted cash flows. |
Other Assets | Other Assets |
Income Taxes | Income Taxes The Tax Cuts and Jobs Act passed in 2017 included a provision which would require taxpayers to capitalize and amortize U.S.-based research & experimentation (R&E) expenses over a period of five years and non-U.S. R&E expenses over 15 years effective for tax years beginning after December 31, 2021 pursuant to Internal Revenue Code Section 174. For the year ended December 31, 2023, we recorded a Benefit for income taxes due to the identification of a discrete item of tax determined upon preparation of our 2022 tax return. As a result of the capitalization of R&E expenses, income generated from the sale of the PRV, and limitations related to the utilization of state net operating losses, |
Debt Issuance Costs | Debt Issuance Costs |
Contract Liabilities, net | Contract Liabilities, net |
Liability Related to Revenue Interest Financing and Non-Cash Interest Expense | Liability Related to Revenue Interest Financing and Non-Cash Interest Expense Sagard Healthcare Royalty Partners, LP (Sagard) Debt Interest - Imputation of Interest. |
Product Revenue, net | Product Revenue, net We recognize ZTALMY revenue in accordance with ASC 606 – Revenue from contracts with customers. Our revenue recognition analysis consists of the following steps: (i) identification of the promised goods in the contract; (ii) determination of whether the promised goods are performance obligations, including whether they are capable of being distinct; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue as we satisfy each performance obligation. Our first FDA approved product, ZTALMY, became available for commercial sale and shipment to patients in the third quarter of 2022. We have three customers, one of which, Orsini, a specialty pharmacy that dispenses ZTALMY directly to patients, is responsible for approximately 99% of our ZTALMY revenue to date. Our contract with Orsini has a single performance obligation to deliver ZTALMY upon receipt of a purchase order, which is satisfied when Orsini receives ZTALMY. We recognize ZTALMY revenue at the point in time when control of ZTALMY is transferred to Orsini, which is upon delivery to Orsini. The transaction price that we recognize for ZTALMY revenue includes an estimate of variable consideration. Shipping and handling costs to Orsini are recorded as Selling, general and administrative expenses. The components of variable consideration include: Trade Discounts and Allowances. Product Returns and Recall. Government Rebates. Patient Assistance . |
Federal Contract Revenue | Federal Contract Revenue We recognize Federal contract revenue from the BARDA Contract in the period in which the allowable Research and development expenses are incurred, and receivables associated with this revenue are included within Accounts receivable, net on our balance sheets. This revenue is not within the scope of Accounting Standards Codification (ASC) 606 – Revenue from contracts with customers. |
Collaboration and Licensing Revenue | Collaboration and Licensing Revenue We must develop estimates and assumptions that require judgment to determine the underlying stand-alone selling price for each performance obligation, which determines how the transaction price is allocated among the performance obligations. The estimation of the stand-alone selling price may include such estimates as forecasted revenues and costs, development timelines, discount rates and probabilities of regulatory and commercial success. We also apply significant judgment when evaluating whether contractual obligations represent distinct performance obligations, allocating transaction price to performance obligations within a contract, determining when performance obligations have been met, assessing the recognition and future reversal of variable consideration and determining and applying appropriate methods of measuring progress for performance obligations satisfied over time. |
Research and Development | Research and Development Research and development costs are expensed as incurred. Costs for certain development activities, such as clinical trials, are recognized based on an evaluation of the progress to completion of specific tasks using data such as subject enrollment, monitoring visits, clinical site activations, or information provided to us by our vendors with respect to their actual costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected in the financial statements as prepaid or accrued research and development expense, as the case may be. |
Clinical Trial Expenses | Clinical Trial Expenses As part of the process of preparing our financial statements, we are required to estimate our expenses resulting from our obligations under contracts with vendors, clinical research organizations and consultants and under clinical site agreements in connection with conducting clinical trials. The financial terms of these contracts are subject to negotiations, which vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided under such contracts. Our objective is to reflect the appropriate trial expenses in our financial statements by matching those expenses with the period in which services are performed and efforts are expended. We account for these expenses according to the progress of the trial as measured by patient progression and the timing of various aspects of the trial. We determine accrual estimates based on estimates of services received and efforts expended that take into account discussion with applicable personnel and outside service providers as to the progress or state of consummation of trials. During the course of a clinical trial, we adjust our clinical expense recognition if actual results differ from its estimates. We make estimates of our Accrued expenses as of each balance sheet date based on the facts and circumstances known at that time. Our clinical trial accruals are dependent upon the timely and accurate reporting of contract research organizations and other third-party vendors. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in reporting amounts that are too high or too low for any particular period. For the years ended December 31, 2023 and 2022 there were no material adjustments to our prior period estimates of accrued or prepaid expenses for clinical trials. For the years ended December 31, 2023 and 2022, our accrued clinical trial expenses were $4.7 million and $5.7 million, respectively. For the years ended December 31, 2023 and 2022, our prepaid clinical expenses were $5.2 million and $3.9 million, respectively. |
Stock-Based Compensation | Stock-Based Compensation We account for stock-based compensation in accordance with the provisions of Accounting Standards Codification (ASC) Topic 718, Compensation—Stock Compensation |
Loss Per Share of Common Stock | Loss Per Share of Common Stock Basic loss per share is computed by dividing Net loss applicable to common stockholders by the Weighted average number of shares of common stock outstanding during each period. Diluted loss per share includes the effect, if any, from the potential exercise or conversion of securities, such as convertible preferred stock, stock options, and unvested restricted stock, which would result in the issuance of incremental shares of common stock. In computing the Basic and diluted net loss per share applicable to common stockholders for the years ended December 31, 2023 and 2022, the Weighted average number of shares remains the same for both calculations due to the fact that when a Net loss exists, dilutive shares are not included in the calculation. These potentially dilutive securities are more fully described in Note 7. The following table sets forth the computation of Basic and diluted net loss per share for the years ended December 31, 2023 and 2022 (in thousands, except share and per share amounts): Year ended December 31, 2023 2022 Basic and diluted net loss per share of common stock: Net loss applicable to common stockholders $ (141,405) $ (19,816) Weighted average shares of common stock outstanding 53,746,518 39,072,599 Net loss per share of common stock—basic and diluted $ (2.63) $ (0.51) The pre-funded warrants to purchase common shares issued in connection with the November 2022 offering are included in the calculation of Basic and diluted net loss per share as the exercise price of $0.001 per share is non-substantive and is virtually assured. The pre-funded warrants are more fully described in Note 7. The following potentially dilutive securities (common stock equivalents) have been excluded from the computation of diluted weighted average shares outstanding, as they would be antidilutive: Year Ended December 31, 2023 2022 Convertible preferred stock — 860,000 Restricted stock units 1,271,894 671,976 Stock options 7,164,981 5,730,219 8,436,875 7,262,195 The convertible preferred stock meets the definition of a participating security; however, the holders were not obligated to share in our losses. As of December 31, 2023 and 2022, we had no other potentially dilutive securities. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Summary of Significant Accounting Policies | |
Schedule of the computation of basic and diluted loss per share | The following table sets forth the computation of Basic and diluted net loss per share for the years ended December 31, 2023 and 2022 (in thousands, except share and per share amounts): Year ended December 31, 2023 2022 Basic and diluted net loss per share of common stock: Net loss applicable to common stockholders $ (141,405) $ (19,816) Weighted average shares of common stock outstanding 53,746,518 39,072,599 Net loss per share of common stock—basic and diluted $ (2.63) $ (0.51) |
Schedule of antidilutive securities excluded from the computation of diluted weighted average shares outstanding | Year Ended December 31, 2023 2022 Convertible preferred stock — 860,000 Restricted stock units 1,271,894 671,976 Stock options 7,164,981 5,730,219 8,436,875 7,262,195 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Measurements | |
Summary of major categories of financial assets and liabilities measured at fair value on a recurring basis | The following fair value hierarchy table presents information about each major category of our financial assets and liabilities measured at fair value on a recurring basis (in thousands): Level 1 Level 2 Level 3 Total December 31, 2023 Assets Cash $ 1,255 $ — $ — $ 1,255 Money market funds (cash equivalents) 119,317 — — 119,317 U.S. Treasury securities — 26,835 — 26,835 U.S. Government Agency securities — 2,881 — 2,881 Total assets $ 120,572 $ 29,716 $ — $ 150,288 December 31, 2022 Assets Cash $ 10,569 $ — $ — $ 10,569 Money market funds (cash equivalents) 229,982 — — 229,982 Total assets $ 240,551 $ — $ — $ 240,551 |
Schedule of short-term investments | The following table provides details regarding our portfolio of Short-term investments (in thousands) as of December 31, 2023: Amortized Cost Unrealized Gains Unrealized Losses Fair Value U.S. Treasury securities $ 26,852 $ 138 $ (155) $ 26,835 U.S. Government Agency securities 2,884 31 (34) 2,881 Total $ 29,736 $ 169 $ (189) $ 29,716 |
Property and Equipment (Tables)
Property and Equipment (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Property and Equipment | |
Schedule of property and equipment | Property and equipment consisted of the following (in thousands): December 31, December 31, 2023 2022 Laboratory equipment $ 4,330 $ 4,277 Leasehold improvements 899 899 Office furniture and equipment 514 514 Total property and equipment 5,743 5,690 Less: accumulated depreciation (1,900) (1,454) Total property and equipment, net $ 3,843 $ 4,236 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Accrued Expenses | |
Schedule of accrued expenses | Accrued expenses consisted of the following (in thousands): December 31, December 31, 2023 2022 Payroll and related costs $ 7,746 $ 7,061 Clinical trials and drug development 4,701 5,725 Accrued license agreement payment 4,000 — Professional fees 1,236 1,417 Accrued tax provision — 2,445 Selling and commercial liabilities 3,901 1,880 Short-term lease liabilities 774 637 Other 501 371 Total accrued expenses $ 22,859 $ 19,536 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Leases | |
Schedule of maturities of operating lease liabilities | Maturities of operating lease liabilities as of December 31, 2023 were as follows (in thousands): 2024 $ 889 2025 662 1,551 Less: imputed interest (144) Total lease liabilities $ 1,407 Current operating lease liabilities $ 774 Non-current operating lease liabilities 633 Total lease liabilities $ 1,407 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Schedule of total compensation cost recognized in the statement of operations | Total compensation cost recognized for all stock options and restricted stock units in the statements of operations for the years ended December 31, 2023 and 2022 is as follows (in thousands): Year Ended December 31, 2023 2022 Research and development $ 5,806 $ 5,364 Selling, general and administrative 9,757 9,526 Total $ 15,563 $ 14,890 |
Schedule of weighted-average assumptions estimated at the date of grant using the Black-Scholes option pricing model | 2023 2022 Expected stock price volatility 99 - 104 % 103 - 116 % Expected term of options 5.3 - 6.1 years 5.7 - 6.5 years Risk‑free interest rate 3.35 - 4.82 % 1.65 - 4.46 % Expected annual dividend yield 0 % 0 % |
Schedule of unrecognized compensation expense expected to be recognized in future years | 2024 $ 12,690 2025 7,348 2026 1,549 2027 366 $ 21,953 |
Employee Stock Option | |
Summary of activity for all options | A summary of stock option activity for the years ended December 31, 2023 and 2022 is presented below (in thousands, except share and per share amounts): Weighted ‑ Average Aggregate Exercise Price Intrinsic Shares Per Share Value Outstanding—December 31, 2021 4,738,855 $ 12.23 Granted 2,026,981 8.77 Exercised (238,312) 7.52 Forfeited (415,816) 10.49 Expired (381,489) 14.61 Outstanding—December 31, 2022 5,730,219 $ 10.87 Granted 2,117,451 6.43 Exercised (126,735) 6.35 Forfeited (267,239) 8.34 Expired (288,715) 13.34 Outstanding—December 31, 2023 7,164,981 $ 9.64 $ 17,854 Exercisable—December 31, 2023 4,620,748 $ 10.64 $ 9,292 |
Restricted Stock and Restricted Stock Units | |
Summary of activity for restricted stock and restricted stock units | Weighted ‑ average Grant Date Shares Fair Value per Share Outstanding—December 31, 2021 26,025 $ 12.75 Granted 809,028 8.49 Vested (37,450) 11.19 Forfeited (125,627) 9.04 Outstanding—December 31, 2022 671,976 8.39 Granted 1,004,310 6.17 Vested (262,819) 8.26 Forfeited (141,573) 6.61 Outstanding—December 31, 2023 1,271,894 $ 6.86 |
Notes Payable (Tables)
Notes Payable (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Notes Payable | |
Summary of composition of notes payable | The following table summarizes the composition of Notes payable as reflected on the consolidated balance sheet as of December 31, 2023 (in thousands): Gross proceeds $ 75,000 Contractual exit fee 2,003 Unamortized debt discount and issuance costs (4,029) Total note payable $ 72,974 Current portion of note payable 11,551 Non-current portion of note payable 61,423 Total note payable $ 72,974 |
Schedule of maturities of notes payable over the next five years | The aggregate maturities of Notes payable as of December 31, 2023 are as follows (in thousands): 2024 $ 11,250 2025 15,000 2026 48,750 Total $ 75,000 |
Revenue Interest Financing Pa_2
Revenue Interest Financing Payable (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Revenue Interest Financing Payable | |
Summary of the activity of revenue interest financing agreement | The following table summarizes the activity of the Revenue Interest Financing Agreement for the years ended December 31, 2023 and 2022 (in thousands): Revenue Interest Financing Agreement upon October 2022 closing $ 32,500 Issuance costs in the year ended December 31, 2022 (2,579) Non-cash interest expense in the year ended December 31, 2022 956 Amortization of debt discount in the year ended December 31, 2022 42 Payments made in the year ended December 31, 2022 (42) Revenue Interest Financing Balance at December 31, 2022 $ 30,877 Non-cash interest expense in the year ended December 31, 2023 5,950 Amortization of debt discount in the year ended December 31, 2023 294 Payments made in the year ended December 31, 2023 (1,144) Revenue Interest Financing Balance at December 31, 2023 $ 35,977 Current portion of revenue interest financing liability $ 2,211 Long-term portion of revenue interest financing liability 33,766 Revenue Interest Financing Balance at December 31, 2023 $ 35,977 |
License and Collaboration Rev_2
License and Collaboration Revenue (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
License and Collaboration Revenue | |
Schedule of allocation of the transaction price to the performance obligations | Cumulative Collaboration Transaction Revenue Recognized Contract Price as of December 31, 2022 Liability License $ 21,660 $ 21,660 $ - Development and Regulatory Services 6,717 1,158 5,559 Supply of Licensed Product 9,503 - 9,503 $ 37,880 $ 22,818 $ 15,062 Less Total Contract Asset 5,079 Net Contract Liability $ 9,983 Cumulative Collaboration Transaction Revenue Recognized Contract Price as of December 31, 2023 Liability License $ 21,660 $ 21,660 $ - Development and Regulatory Services 6,717 2,511 4,206 Supply of Licensed Product 9,503 - 9,503 $ 37,880 $ 24,171 $ 13,709 Less Total Contract Asset 2,912 Net Contract Liability $ 10,797 Cumulative Collaboration Transaction Revenue Recognized Contract Price as of each December 31, 2023 and 2022 Liability License $ 2,998 $ 2,998 $ - Supply of Licensed Product 7,002 - 7,002 $ 10,000 $ 2,998 $ 7,002 Less Total Contract Asset 700 Net Contract Liability $ 6,302 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Income Taxes | |
Summary of income tax expense by jurisdiction | Income tax (benefit) expense by jurisdiction as follows (in thousands): Year Ended December 31, 2023 2022 Current U.S. State $ (1,538) 2,445 Foreign — 944 Total current (1,538) 3,389 Deferred — — (Benefit) provision for income taxes $ (1,538) $ 3,389 |
Summary of loss before income taxes | Loss before income taxes is allocated as follows (in thousands): Year Ended December 31, 2023 2022 U.S. operations $ 142,943 $ 16,427 Foreign operations — — Loss before income taxes $ 142,943 $ 16,427 |
Schedule of components of the net deferred tax asset | The components of the net deferred tax asset are as follows (in thousands): December 31, 2023 2022 Gross deferred tax assets: Net operating loss carryforwards $ 72,792 $ 58,056 Accrued expenses 1,754 124 Amortization of intangible 468 454 Stock‑based compensation 8,741 8,091 Research and development and other credits and other carryforwards 39,588 30,627 Lease liability 345 440 Capitalized research and development expenses 37,643 22,382 Royalty/ deferred revenue 4,298 3,781 Capital loss 74 94 Interest carryforward 2,152 — Other 152 64 Total gross deferred tax assets $ 168,007 $ 124,113 Gross deferred tax liabilities: ROU asset (237) (291) Depreciation (458) (224) Unrealized income (209) (192) Total gross deferred tax liabilities (904) (707) Net deferred tax assets 167,103 123,406 Less: valuation allowance (167,103) (123,406) Net deferred tax assets after valuation allowance $ — $ — |
Schedule of reconciliation of income tax expense (benefit) at the statutory federal income tax rate and income taxes | Year Ended December 31, 2023 2022 Federal income tax expense at statutory rate 21.0 % 21.0 % Permanent items (0.4) (2.6) State income tax, net of federal benefit 4.1 (9.0) R&D tax credits 6.3 88.1 Change in state apportionment 2.5 (59.5) Foreign withholding tax — (5.7) Change in valuation allowance (30.6) (47.0) Other (1.8) (5.9) Effective income tax rate 1.1 % (20.6) % |
Organization and Description _2
Organization and Description of the Business (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Liquidity | ||
Net loss | $ 141,405 | $ 19,816 |
Accumulated deficit | 571,926 | $ 430,521 |
Cash, cash equivalents and investment balances | $ 150,300 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Other (Details) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 USD ($) customer segment | Dec. 31, 2022 USD ($) | |
Product Revenue, net | ||
Number of customers | customer | 3 | |
Trade Receivables, net | ||
Accounts receivable, net | $ 3,799 | $ 6,348 |
Allowance for doubtful accounts. | $ 0 | 0 |
Payment terms | 30 days | |
Segment Information | ||
Number of operating segments | segment | 1 | |
Accrued clinical trial expenses | $ 4,700 | 5,700 |
Prepaid clinical expenses | 5,200 | 3,900 |
ZTALMY | ||
Trade Receivables, net | ||
Accounts receivable, net | 2,600 | $ 1,300 |
Inventory | ||
Cost of product and materials included in research and development expenses prior to FDA approval | $ 2,000 | |
ZTALMY | Revenue Benchmark | Customer Concentration Risk | ||
Product Revenue, net | ||
Concentration risk, as a percent | 99% | |
Number of customers | customer | 1 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Property and Equipment (Details) | Dec. 31, 2023 |
Office equipment and furniture | |
Property and Equipment | |
Useful life | 5 years |
Minimum | Laboratory equipment | |
Property and Equipment | |
Useful life | 5 years |
Maximum | Laboratory equipment | |
Property and Equipment | |
Useful life | 15 years |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Loss Per Share of Common Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Nov. 30, 2022 | |
Basic and diluted net loss per share of common stock: | |||
Net loss applicable to common shareholders | $ (141,405) | $ (19,816) | |
Weighted average shares of common stock outstanding - Basic (in shares) | 53,746,518 | 39,072,599 | |
Weighted average shares of common stock outstanding - Diluted (in shares) | 53,746,518 | 39,072,599 | |
Net loss per share of common stock-basic | $ (2.63) | $ (0.51) | |
Net loss per share of common stock-diluted | $ (2.63) | $ (0.51) | |
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares) | 8,436,875 | 7,262,195 | |
Warrant exercise price (in dollars per share) | $ 0.001 | ||
Convertible preferred stock | |||
Basic and diluted net loss per share of common stock: | |||
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares) | 860,000 | ||
RSAs and RSUs | |||
Basic and diluted net loss per share of common stock: | |||
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares) | 1,271,894 | 671,976 | |
Employee Stock Option [Member] | |||
Basic and diluted net loss per share of common stock: | |||
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares) | 7,164,981 | 5,730,219 |
Fair Value Measurements - Fair
Fair Value Measurements - Fair Value Recurring Basis (Details) - Recurring basis - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Assets | ||
Total assets | $ 150,288 | $ 240,551 |
U.S. Treasury securities | ||
Assets | ||
Investments | 26,835 | |
U.S. Government Agency securities | ||
Assets | ||
Investments | 2,881 | |
Cash. | ||
Assets | ||
Cash and cash equivalents, fair value | 1,255 | 10,569 |
Money market funds | ||
Assets | ||
Cash and cash equivalents, fair value | 119,317 | 229,982 |
Level 1 | ||
Assets | ||
Total assets | 120,572 | 240,551 |
Level 1 | Cash. | ||
Assets | ||
Cash and cash equivalents, fair value | 1,255 | 10,569 |
Level 1 | Money market funds | ||
Assets | ||
Cash and cash equivalents, fair value | 119,317 | $ 229,982 |
Level 2 | ||
Assets | ||
Total assets | 29,716 | |
Level 2 | U.S. Treasury securities | ||
Assets | ||
Investments | 26,835 | |
Level 2 | U.S. Government Agency securities | ||
Assets | ||
Investments | $ 2,881 |
Fair Value Measurements - Short
Fair Value Measurements - Short-Term Investments (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Short-term Investments | |
Amortized Cost | $ 29,736 |
Unrealized Gains | 169 |
Unrealized Losses | (189) |
Fair Value | 29,716 |
U.S. Treasury securities | |
Short-term Investments | |
Amortized Cost | 26,852 |
Unrealized Gains | 138 |
Unrealized Losses | (155) |
Fair Value | 26,835 |
U.S. Government Agency securities | |
Short-term Investments | |
Amortized Cost | 2,884 |
Unrealized Gains | 31 |
Unrealized Losses | (34) |
Fair Value | $ 2,881 |
Property and Equipment (Details
Property and Equipment (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Property and equipment | ||
Total property and equipment | $ 5,743 | $ 5,690 |
Less: accumulated depreciation | (1,900) | (1,454) |
Total property and equipment, net | 3,843 | 4,236 |
Depreciation expense | 500 | 300 |
Equipment held for sale | 700 | |
Other expense | ||
Property and equipment | ||
Equipment held for sale | 100 | 800 |
Laboratory equipment | ||
Property and equipment | ||
Total property and equipment | 4,330 | 4,277 |
Leasehold improvements | ||
Property and equipment | ||
Total property and equipment | 899 | 899 |
Office furniture and equipment | ||
Property and equipment | ||
Total property and equipment | $ 514 | $ 514 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Accrued Expenses | ||
Payroll and related costs | $ 7,746 | $ 7,061 |
Clinical trials and drug development | 4,701 | 5,725 |
Accrued license agreement payment | 4,000 | |
Professional fees | 1,236 | 1,417 |
Accrued tax provision | 2,445 | |
Selling and commercial liabilities | 3,901 | 1,880 |
Short-term lease liabilities | 774 | 637 |
Other | 501 | 371 |
Total accrued expenses | $ 22,859 | $ 19,536 |
Leases - Other (Details)
Leases - Other (Details) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 USD ($) lease | Dec. 31, 2022 USD ($) | |
Leases | ||
Right-of-use assets | $ | $ 1,000 | $ 1,300 |
Operating Lease, Right-of-Use Asset, Statement of Financial Position [Extensible List] | Other Assets, Noncurrent | Other Assets, Noncurrent |
Operating lease liabilities | $ | $ 1,407 | $ 2,000 |
Real estate | ||
Leases | ||
Operating lease agreement term | 78 months | |
Operating lease renewal term | 60 months | |
Weighted average remaining lease term | 21 months | |
Number of leases entered | lease | 1 | |
Clinical site equipment | ||
Leases | ||
Operating lease agreement term | 18 months | |
Weighted average remaining lease term | 17 months | |
Number of leases entered | lease | 1 |
Leases - Costs (Details)
Leases - Costs (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Leases | ||
Total lease costs | $ 0.6 | $ 0.6 |
Impairment losses of ROU assets | $ 0 | 0 |
Clinical site equipment | ||
Leases | ||
Weighted-average incremental borrowing rate used to determine right-of-use assets and lease liabilities | 7% | |
Real estate | ||
Leases | ||
Weighted-average incremental borrowing rate used to determine right-of-use assets and lease liabilities | 11% | |
Maximum | ||
Leases | ||
Short-term operating lease costs | $ 0.1 | $ 0.1 |
Leases - Maturities (Details)
Leases - Maturities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Maturities of operating lease liabilities: | ||
2024 | $ 889 | |
2025 | 662 | |
Total lease payments | 1,551 | |
Less: imputed interest | (144) | |
Total lease liabilities | $ 1,407 | $ 2,000 |
Operating Lease, Liability, Statement of Financial Position [Extensible List] | Accrued Liabilities, Current, Other Accrued Liabilities, Noncurrent | Accrued Liabilities, Current, Other Accrued Liabilities, Noncurrent |
Current operating lease liabilities | $ 774 | |
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List] | Accrued Liabilities, Current | Accrued Liabilities, Current |
Non-current operating lease liabilities | $ 633 | |
Operating Lease, Liability, Noncurrent, Statement of Financial Position [Extensible List] | Other Accrued Liabilities, Noncurrent | Other Accrued Liabilities, Noncurrent |
Stockholders' Equity - Incentiv
Stockholders' Equity - Incentive Plans (Details) - shares | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Jan. 01, 2024 | Dec. 31, 2021 | |
Employee Stock Option | ||||
Stockholders' Equity | ||||
Options to purchase common stock (in shares) | 7,164,981 | 5,730,219 | 4,738,855 | |
Granted (in shares) | 2,117,451 | 2,026,981 | ||
Restricted Stock Units | ||||
Stockholders' Equity | ||||
Awards outstanding (in shares) | 1,271,894 | |||
2005 Plan | ||||
Stockholders' Equity | ||||
Common stock reserved for issuance (in shares) | 0 | |||
2005 Plan | Employee Stock Option | ||||
Stockholders' Equity | ||||
Options to purchase common stock (in shares) | 577 | |||
2014 Plan | ||||
Stockholders' Equity | ||||
Common stock reserved for issuance (in shares) | 907,095 | 3,090,220 | ||
2014 Plan | Employee Stock Option | ||||
Stockholders' Equity | ||||
Options to purchase common stock (in shares) | 4,924,969 | |||
2014 Plan | Restricted common stock | ||||
Stockholders' Equity | ||||
Awards outstanding (in shares) | 0 | |||
2014 Plan | Restricted Stock Units | ||||
Stockholders' Equity | ||||
Awards outstanding (in shares) | 1,257,019 | |||
Granted (in shares) | 1,004,310 | |||
Outside of 2014 Plan | Employee Stock Option | ||||
Stockholders' Equity | ||||
Granted (in shares) | 566,685 | 788,885 | ||
Outside of 2014 Plan | Restricted Stock Units | ||||
Stockholders' Equity | ||||
Granted (in shares) | 2,500 | 39,900 |
Stockholders' Equity - Stock Op
Stockholders' Equity - Stock Options, Restricted Stock, RSUs, , Offering (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Mar. 29, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | |
Stockholders' Equity | |||
Total compensation cost | $ 15,563 | $ 14,890 | |
Issuance of common stock (in shares) | 3,700,000 | ||
Proceeds from equity offerings, net of offering costs | $ 25,800 | ||
Maximum percentage of beneficial ownership for exercise of Pre-funded Warrants | 9.99% | ||
Maximum percentage increase or decrease of beneficial ownership for exercise of Pre-funded Warrants | 19.99% | ||
Additional Disclosures | |||
Weighted average remaining contractual term | 7 years 6 months | ||
Market value per share | $ 7.17 | ||
Unrecognized compensation expense | |||
Unrecognized compensation expense, unvested stock options | $ 15,600 | ||
Unrecognized compensation expense, restricted stock units | 6,400 | ||
Unrecognized compensation expense | $ 21,953 | ||
Common Stock | |||
Stock Options | |||
Exercised (in shares) | (126,735) | (238,312) | |
Public Offering. | |||
Stockholders' Equity | |||
Issuance of common stock (in shares) | 12,421,053 | ||
Number of pre-funded warrants (in shares) | 2,105,264 | ||
Proceeds from equity offerings, net of offering costs | $ 64,500 | ||
Ovid License Agreement | |||
Stockholders' Equity | |||
Issuance of common stock (in shares) | 123,255 | ||
Recognition in 2024 | |||
Unrecognized compensation expense | |||
Unrecognized compensation expense | $ 12,690 | ||
Recognition in 2025 | |||
Unrecognized compensation expense | |||
Unrecognized compensation expense | 7,348 | ||
Recognition in 2026 | |||
Unrecognized compensation expense | |||
Unrecognized compensation expense | 1,549 | ||
Recognition in 2027 | |||
Unrecognized compensation expense | |||
Unrecognized compensation expense | $ 366 | ||
Employee Stock Option | |||
Stock Options | |||
Outstanding at the beginning of the period (in shares) | 5,730,219 | 4,738,855 | |
Granted (in shares) | 2,117,451 | 2,026,981 | |
Exercised (in shares) | (126,735) | (238,312) | |
Forfeited (in shares) | (267,239) | (415,816) | |
Expired (in shares) | (288,715) | (381,489) | |
Outstanding at the end of the period (in shares) | 7,164,981 | 5,730,219 | |
Exercisable at the end of the period (in shares) | 4,620,748 | ||
Weighted-Average Exercise Price Per Share | |||
Outstanding at the beginning of the period (in dollars per share) | $ 10.87 | $ 12.23 | |
Granted (in dollars per share) | 6.43 | 8.77 | |
Exercised (in dollars per share) | 6.35 | 7.52 | |
Forfeited (in dollars per share) | 8.34 | 10.49 | |
Expired (in dollars per share) | 13.34 | 14.61 | |
Outstanding at the end of the period (in dollars per share) | 9.64 | 10.87 | |
Exercisable at the end of the period (in dollars per share) | $ 10.64 | ||
Aggregate Intrinsic Value | |||
Outstanding at the end of the period | $ 17,854 | ||
Exercisable at the end of the period | $ 9,292 | ||
Additional Disclosures | |||
Weighted average remaining contractual term exercisable | 6 years 10 months 24 days | ||
Market value per share | $ 10.87 | ||
Weighted average grant date fair value (in dollars per share) | $ 5.20 | $ 7 | |
Weighted-average assumptions | |||
Expected annual dividend yield (as a percent) | 0% | 0% | |
Employee Stock Option | Minimum | |||
Weighted-average assumptions | |||
Expected stock price volatility (as a percent) | 99% | 103% | |
Expected term of options | 5 years 3 months 18 days | 5 years 8 months 12 days | |
Risk-free interest rate (as a percent) | 3.35% | 1.65% | |
Employee Stock Option | Maximum | |||
Stockholders' Equity | |||
Vesting period | 4 years | ||
Weighted-average assumptions | |||
Expected stock price volatility (as a percent) | 104% | 116% | |
Expected term of options | 6 years 1 month 6 days | 6 years 6 months | |
Risk-free interest rate (as a percent) | 4.82% | 4.46% | |
Expected annual dividend yield (as a percent) | 0% | 0% | |
Restricted Stock and Restricted Stock Units | |||
Shares | |||
Restricted stock units outstanding at the beginning of the period (in shares) | 671,976 | 26,025 | |
Granted (in shares) | 1,004,310 | 809,028 | |
Vested (in shares) | (262,819) | (37,450) | |
Forfeited (in shares) | (141,573) | (125,627) | |
Restricted stock units outstanding at the end of the period (in shares | 1,271,894 | 671,976 | |
Weighted-average Grant Date Fair Value | |||
Restricted stock units outstanding at the beginning of the period (in dollars per shares) | $ 8.39 | $ 12.75 | |
Granted (in dollars per share) | 6.17 | 8.49 | |
Vested (in dollars per share) | 8.26 | 11.19 | |
Forfeited (in dollars per share) | 6.61 | 9.04 | |
Restricted stock units outstanding at the end of the period (in dollars per shares) | $ 6.86 | $ 8.39 | |
Restricted common stock | Maximum | |||
Stockholders' Equity | |||
Vesting period | 2 years | ||
Restricted Stock Units (RSUs) | |||
Shares | |||
Restricted stock units outstanding at the end of the period (in shares | 1,271,894 | ||
2005 Plan | Employee Stock Option | |||
Stock Options | |||
Outstanding at the end of the period (in shares) | 577 | ||
2005 Plan | Employee Stock Option | Maximum | |||
Stockholders' Equity | |||
Contractual life | 10 years | ||
2014 Plan | Employee Stock Option | |||
Stock Options | |||
Outstanding at the end of the period (in shares) | 4,924,969 | ||
2014 Plan | Employee Stock Option | Maximum | |||
Stockholders' Equity | |||
Contractual life | 10 years | ||
2014 Plan | Restricted common stock | |||
Shares | |||
Restricted stock units outstanding at the end of the period (in shares | 0 | ||
2014 Plan | Restricted Stock Units (RSUs) | |||
Stockholders' Equity | |||
Vesting period | 4 years | ||
Shares | |||
Granted (in shares) | 1,004,310 | ||
Restricted stock units outstanding at the end of the period (in shares | 1,257,019 | ||
Research and development. | |||
Stockholders' Equity | |||
Total compensation cost | $ 5,806 | $ 5,364 | |
Selling, general and administrative | |||
Stockholders' Equity | |||
Total compensation cost | $ 9,757 | $ 9,526 |
Stockholders' Equity - Equity D
Stockholders' Equity - Equity Distribution Agreement (Details) - USD ($) shares in Millions | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Stockholders' Equity | ||
Issuance of shares under equity distribution agreement (in shares) | 0 | |
Proceeds from equity offerings, net of offering costs | $ 25,823,000 | $ 64,477,000 |
Issuance of common stock (in shares) | 3.7 | |
Proceeds from issuance of common stock, net of issuance costs | $ 25,800,000 | |
Maximum | ||
Stockholders' Equity | ||
Maximum value of shares that may be issued and sold under Equity Distribution Agreement | $ 75,000,000 | |
Maximum commission rate, expressed as a percentage of the gross proceeds from each sale of shares of common stock, equity distribution agreement | 3% |
Convertible Preferred Stock (De
Convertible Preferred Stock (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2019 | |
Convertible Preferred Stock | |||
Share Price (in dollars per share) | $ 7.17 | ||
Common Stock | |||
Convertible Preferred Stock | |||
Conversion of convertible preferred stock to common stock (in shares) | 860,000 | 55,000 | |
Series A Convertible Preferred Stock | |||
Convertible Preferred Stock | |||
Conversion of stock, number of shares converted | 4,300 | 275 | |
Series A convertible preferred stock, shares outstanding | 0 | ||
Series A Convertible Preferred Stock | Common Stock | |||
Convertible Preferred Stock | |||
Number of common shares issued for each convertible Series A preferred stock | 200 | ||
Conversion price (in dollars per share) | $ 5 | ||
Private placement | Series A Convertible Preferred Stock | |||
Convertible Preferred Stock | |||
Issuance of Series A preferred stock (in shares) | 30,000 | ||
Series A convertible preferred stock, par value (in dollars per share) | $ 0.001 | ||
Share Price (in dollars per share) | $ 1,000 | ||
Net proceeds from issuance of Series A convertible preferred stock | $ 28.2 | ||
Underwriting discounts and commissions | $ 1.8 |
Notes Payable - Narratives (Det
Notes Payable - Narratives (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | |||||||
May 11, 2021 USD ($) tranche | Mar. 31, 2022 USD ($) | Sep. 30, 2021 USD ($) | May 31, 2021 USD ($) | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Aug. 31, 2023 USD ($) | Oct. 31, 2022 | Oct. 27, 2022 USD ($) | |
Notes Payable | |||||||||
Contractual exit fee | $ 2,003 | ||||||||
Interest expense | 16,895 | $ 10,672 | |||||||
Amortization of debt issuance costs | 2,248 | 1,663 | |||||||
Total debt commitments | 75,000 | ||||||||
Minimum liquidity covenant, cash and cash equivalents | 15,000 | ||||||||
Term Loan Tranche C | |||||||||
Notes Payable | |||||||||
Debt commitment, terminated | $ 25,000 | ||||||||
Oaktree Capital Management LP Credit Agreement | |||||||||
Notes Payable | |||||||||
Term of loan facility | 5 years | ||||||||
Maximum borrowing capacity available under the credit agreement | $ 125,000 | ||||||||
Number of tranches | tranche | 5 | ||||||||
Proceeds from line of credit | $ 30,000 | $ 30,000 | $ 15,000 | ||||||
Accrued interest rate (as a percent) | 11.50% | ||||||||
Percentage of periodic principal payment | 5% | ||||||||
Upfront fee (as a percent) | 2% | ||||||||
Commitment fee (as a percent) | 75% | ||||||||
Commitment fee accrual period | 120 days | ||||||||
Debt issuance costs | 200 | $ 500 | |||||||
Debt issuance costs | 1,800 | 1,200 | 4,400 | ||||||
Contractual exit fee | 600 | 600 | 300 | ||||||
Interest expense | 10,700 | 9,500 | |||||||
Interest on term loan | 8,700 | 7,900 | |||||||
Amortization of debt issuance costs | $ 2,000 | $ 1,600 | |||||||
Exit fee, percentage | 2.67% | 2% | |||||||
Oaktree Capital Management LP Credit Agreement | Maximum | |||||||||
Notes Payable | |||||||||
Exit fee, percentage | 2.67% | ||||||||
Oaktree Capital Management LP Credit Agreement | Minimum | |||||||||
Notes Payable | |||||||||
Exit fee, percentage | 2% | ||||||||
Oaktree Capital Management LP Credit Agreement | Scenario Of Prepayment Occurs Between May 11, 2023 To May 11, 2024 | |||||||||
Notes Payable | |||||||||
Prepayment premium (as a percent) | 4% | ||||||||
Oaktree Capital Management LP Credit Agreement | Scenario Of Prepayment Between May 11, 2024 To May 11, 2025 | |||||||||
Notes Payable | |||||||||
Prepayment premium (as a percent) | 2% | ||||||||
Oaktree Capital Management LP Credit Agreement | Scenario Of Prepayment After May 11, 2025 | |||||||||
Notes Payable | |||||||||
Prepayment premium (as a percent) | 0% | ||||||||
Oaktree Capital Management LP Credit Agreement | Term Loans Tranche A-1 | |||||||||
Notes Payable | |||||||||
Proceeds from line of credit | $ 15,000 | ||||||||
Oaktree Capital Management LP Credit Agreement | Term Loans Tranche A-2 | |||||||||
Notes Payable | |||||||||
Proceeds from line of credit | $ 30,000 | ||||||||
Oaktree Capital Management LP Credit Agreement | Term Loan Tranche B | |||||||||
Notes Payable | |||||||||
Proceeds from line of credit | 30,000 | ||||||||
Oaktree Capital Management LP Credit Agreement | Term Loan Tranche B | Maximum | |||||||||
Notes Payable | |||||||||
Commitment fees | $ 100 |
Notes Payable - Composition of
Notes Payable - Composition of debt (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Notes Payable | ||
Gross proceeds | $ 75,000 | |
Contractual exit fee | 2,003 | |
Unamortized debt discount and issuance costs | (4,029) | |
Total note payable | 72,974 | |
Current portion of note payable | 11,551 | |
Non-current portion of note payable | $ 61,423 | $ 71,018 |
Notes Payable - Debt maturities
Notes Payable - Debt maturities (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Notes Payable | |
2024 | $ 11,250 |
2025 | 15,000 |
2026 | 48,750 |
Total | $ 75,000 |
Revenue Interest Financing Pa_3
Revenue Interest Financing Payable - Terms (Details) - USD ($) $ in Thousands | 2 Months Ended | 12 Months Ended | ||
Oct. 28, 2022 | Dec. 31, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | |
Revenue Interest Financing Payable | ||||
Professional fees | $ 1,417 | $ 1,236 | $ 1,417 | |
Total issuance costs | 2,579 | $ 2,600 | ||
Estimated effective annual interest rate, percentage | 18% | 19% | ||
Each calendar quarter from and after the Closing Date through and including the quarter ended June 30, 2026 | Sagard | ||||
Revenue Interest Financing Payable | ||||
Quarterly payments expressed as a percentage of the U.S. net sales of Ztalmy and all other pharmaceutical products that contain ganaxolene, required under the revenue interest financing agreement. | 7.50% | |||
Revenue Interest Financing Agreement | Oaktree Capital Management LP Credit Agreement | ||||
Revenue Interest Financing Payable | ||||
Minimum cash and cash equivalents required to be maintained deposits until repayment | $ 15,000 | |||
Minimum cash and cash equivalents required to be maintained deposits after repayment | 10,000 | |||
Revenue Interest Financing Agreement | Sagard | ||||
Revenue Interest Financing Payable | ||||
Investment amount | $ 32,500 | |||
Hard Cap expressed as a percentage of the Investment Amount | 190% | |||
Hard cap amount | $ 61,800 | |||
Professional fees | $ 2,600 | $ 2,600 | ||
Revenue Interest Financing Agreement | Each calendar quarter from and after the Closing Date through and including the quarter ended June 30, 2026 | Sagard | ||||
Revenue Interest Financing Payable | ||||
Quarterly payments due, expressed as a percentage of the first $100 million in annual Product Revenue of the Included Products | 15% | |||
Quarterly payments due, expressed as a percentage of the annual Product Revenue of the Included Products in excess of $100 million | 7.50% | |||
Annual Product Revenue Threshold | $ 100,000 | |||
Revenue Interest Financing Agreement | Period Ending December 31, 2027 | Sagard | ||||
Revenue Interest Financing Payable | ||||
Minimum amount expressed as a percentage of the Investment Amount | 100% | |||
Revenue Interest Financing Agreement | Period Ending December 31, 2032 | Sagard | ||||
Revenue Interest Financing Payable | ||||
Minimum amount expressed as a percentage of the Investment Amount | 190% | |||
Revenue Interest Financing Agreement | On Or Before Third Anniversary | ||||
Revenue Interest Financing Payable | ||||
Payments at repurchase price investment percent | 160% | |||
Revenue Interest Financing Agreement | After Third Anniversary | ||||
Revenue Interest Financing Payable | ||||
Payments at repurchase price investment percent | 180% | |||
Royalty monetization agreement | After Fourth Anniversary | ||||
Revenue Interest Financing Payable | ||||
Payments at repurchase price investment percent | 190% |
Revenue Interest Financing Pa_4
Revenue Interest Financing Payable - Summary of Activity (Details) - USD ($) $ in Thousands | 2 Months Ended | 12 Months Ended |
Dec. 31, 2022 | Dec. 31, 2023 | |
Revenue Interest Financing Payable | ||
Revenue Interest Financing Starting Balance | $ 32,500 | $ 30,877 |
Issuance costs | (2,579) | (2,600) |
Non-cash interest expense | 956 | 5,950 |
Amortization of debt discount | 42 | 294 |
Payments | (42) | (1,144) |
Revenue Interest Financing Ending Balance | 30,877 | 35,977 |
Current portion of revenue interest financing liability | 1,020 | 2,211 |
Long-term portion of revenue interest financing liability | 29,857 | 33,766 |
Revenue Interest Financing Balance at December 31, 2023 | $ 30,877 | $ 35,977 |
License and Collaboration Rev_3
License and Collaboration Revenue - Narrative (Details) $ in Thousands, € in Millions | 1 Months Ended | 12 Months Ended | |||||
Nov. 26, 2022 USD ($) product | May 31, 2023 USD ($) | Jul. 31, 2021 USD ($) | Jul. 31, 2021 EUR (€) item | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Nov. 16, 2022 item | |
License and Collaboration Revenue | |||||||
Contract liability | $ 400 | $ 0 | |||||
Net Contract Liability | 17,545 | 16,285 | |||||
Collaboration revenue | |||||||
License and Collaboration Revenue | |||||||
Collaboration revenue | 54 | 15,671 | |||||
Cost of revenue | 25 | 150 | |||||
Collaboration Agreement with Orion | |||||||
License and Collaboration Revenue | |||||||
Upfront fee received | $ 29,600 | € 25 | |||||
Number of days from receipt of the final report, in which the collaboration agreement may be terminated | 90 days | 90 days | |||||
Percentage of upfront fee which must be refunded in the event of termination | 75% | ||||||
Amount of payment receivable upon achievement of specific clinical and commercial achievements | € | € 97 | ||||||
Term of collaboration agreement | 10 years | 10 years | |||||
Number of performance obligations | item | 3 | ||||||
Number of commitments represent distinct performance | item | 3 | ||||||
Collaboration revenue | 12,700 | ||||||
Transaction Price | 37,880 | 37,880 | |||||
Contract liability | 13,709 | 15,062 | |||||
Net Contract Liability | 10,797 | 10,000 | |||||
Offset by contract asset | 2,912 | 5,079 | |||||
Incremental costs incurred in obtaining the agreement | 2,000 | ||||||
Capitalized contract costs | 900 | ||||||
Collaboration Agreement with Orion | Selling, general and administrative | |||||||
License and Collaboration Revenue | |||||||
Contract acquisition costs included in selling, general and administrative expense | 1,100 | ||||||
Collaboration Agreement with Orion | License Revenue | |||||||
License and Collaboration Revenue | |||||||
Collaboration revenue | 12,700 | ||||||
Transaction Price | 21,660 | 21,660 | |||||
Collaboration Agreement with Orion | Development and Regulatory Services | |||||||
License and Collaboration Revenue | |||||||
Amortization of transaction price as a reduction of research and development costs | 1,400 | 1,100 | |||||
Transaction Price | 6,717 | 6,717 | |||||
Contract liability | 4,206 | 5,559 | |||||
Collaboration Agreement with Orion | Supply of License Product | |||||||
License and Collaboration Revenue | |||||||
Transaction Price | 9,503 | 9,503 | |||||
Contract liability | 9,503 | 9,503 | |||||
Collaboration Agreement with Tenacia | |||||||
License and Collaboration Revenue | |||||||
Upfront fee received | $ 10,000 | ||||||
Aggregate amount | $ 256,000 | ||||||
Milestone regulatory approvals | 15,000 | ||||||
Aggregative sales based milestone | $ 241,000 | ||||||
Term of collaboration agreement | 45 days | ||||||
Number of performance obligations | item | 2 | ||||||
Number of commitments represent distinct performance | item | 2 | ||||||
Transaction Price | 10,000 | ||||||
Contract liability | 7,002 | $ 7,000 | |||||
Net Contract Liability | 6,302 | ||||||
Offset by contract asset | 700 | 700 | |||||
Incremental costs incurred in obtaining the agreement | 1,000 | ||||||
Capitalized contract costs | 700 | ||||||
Cost of revenue | 200 | ||||||
Number of Selected Products to which milestone payments applicable | product | 1 | ||||||
Collaboration Agreement with Tenacia | Selling, general and administrative | |||||||
License and Collaboration Revenue | |||||||
Contract acquisition costs included in selling, general and administrative expense | $ 100 | ||||||
Collaboration Agreement with Tenacia | License Revenue | |||||||
License and Collaboration Revenue | |||||||
Collaboration revenue | 0 | ||||||
Transaction Price | 2,998 | ||||||
Collaboration Agreement with Tenacia | Supply of License Product | |||||||
License and Collaboration Revenue | |||||||
Transaction Price | 7,002 | ||||||
Contract liability | 7,002 | ||||||
Biologix Distribution and Supply Agreement | |||||||
License and Collaboration Revenue | |||||||
Upfront fee received | $ 500 | ||||||
Biologix Distribution and Supply Agreement | Maximum | |||||||
License and Collaboration Revenue | |||||||
Collaboration revenue | 100 | ||||||
Cost of revenue | $ 100 |
License and Collaboration Rev_4
License and Collaboration Revenue - Allocation of the transaction price to the performance obligations (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
License and Collaboration Revenue | ||
Contract Liability | $ 400 | $ 0 |
Net Contract Liability | 17,545 | 16,285 |
Collaboration Agreement with Orion | ||
License and Collaboration Revenue | ||
Transaction Price | 37,880 | 37,880 |
Cumulative Collaboration Revenue Recognized | 24,171 | 22,818 |
Contract Liability | 13,709 | 15,062 |
Less Total Contract Asset | 2,912 | 5,079 |
Net Contract Asset | 9,983 | |
Net Contract Liability | 10,797 | 10,000 |
Collaboration Agreement with Orion | License Revenue | ||
License and Collaboration Revenue | ||
Transaction Price | 21,660 | 21,660 |
Cumulative Collaboration Revenue Recognized | 21,660 | 21,660 |
Collaboration Agreement with Orion | Development and Regulatory Services | ||
License and Collaboration Revenue | ||
Transaction Price | 6,717 | 6,717 |
Cumulative Collaboration Revenue Recognized | 2,511 | 1,158 |
Contract Liability | 4,206 | 5,559 |
Collaboration Agreement with Orion | Supply of License Product | ||
License and Collaboration Revenue | ||
Transaction Price | 9,503 | 9,503 |
Contract Liability | 9,503 | 9,503 |
Collaboration Agreement with Tenacia | ||
License and Collaboration Revenue | ||
Transaction Price | 10,000 | |
Cumulative Collaboration Revenue Recognized | 2,998 | |
Contract Liability | 7,002 | 7,000 |
Less Total Contract Asset | 700 | $ 700 |
Net Contract Liability | 6,302 | |
Collaboration Agreement with Tenacia | License Revenue | ||
License and Collaboration Revenue | ||
Transaction Price | 2,998 | |
Cumulative Collaboration Revenue Recognized | 2,998 | |
Collaboration Agreement with Tenacia | Supply of License Product | ||
License and Collaboration Revenue | ||
Transaction Price | 7,002 | |
Contract Liability | $ 7,002 |
Commitments and Contingencies -
Commitments and Contingencies - Employee Benefit Plan and License Agreements (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Mar. 29, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | |
Employee Benefit Plan | |||
Percentage of contribution | 3% | ||
Amount of contribution | $ 1,100 | $ 1,000 | |
Vesting period | 4 years | ||
License agreement | |||
Issuance of common stock (in shares) | 3,700,000 | ||
Product revenue, net | |||
License agreement | |||
Revenue | $ 19,561 | 2,872 | |
Exclusive patent license agreement with Ovid Therapeutics Inc. | |||
License agreement | |||
Amount agreed to be paid | $ 1,500 | ||
Number of shares agreed to be issued (in shares) | 123,255 | ||
Option to obtain shares of common stock exercisable, following the filing of annual report, maximum period (in days) | 5 days | ||
Issuance of common stock (in shares) | 123,255 | ||
IP license fee expenses | $ 1,200 | ||
License Agreement | Purdue | |||
License agreement | |||
Expiration period of obligation to pay royalties | 10 years |
(Loss) Gain from Sale of Prio_2
(Loss) Gain from Sale of Priority Review Voucher (PRV) (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Jul. 13, 2022 | Feb. 29, 2024 | Dec. 31, 2023 | Dec. 31, 2022 | Aug. 31, 2022 | |
(Loss) Gain from Sale of Priority Review Voucher (PRV) | |||||
Accrued liability related to PRV | $ 4,000 | ||||
Net loss from sale of PRV | (4,000) | $ 107,375 | |||
PRV Asset Purchase Agreement | |||||
(Loss) Gain from Sale of Priority Review Voucher (PRV) | |||||
Amount of consideration to be paid by the acquirer to the Company upon closing of the asset purchase transaction | $ 110,000 | ||||
PRV Asset Purchase Agreement | Purdue | |||||
(Loss) Gain from Sale of Priority Review Voucher (PRV) | |||||
Amount claimed by counterparty from sale of PRV | $ 5,500 | ||||
Amount of payment to be made by Company towards amount claimed by counterparty from the sale of PRV | $ 4,000 | ||||
Accrued liability related to PRV | 4,000 | ||||
Net loss from sale of PRV | $ 4,000 |
Income Taxes - Jurisdiction (De
Income Taxes - Jurisdiction (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Current | ||
U.S. State | $ (1,538) | $ 2,445 |
Foreign | 944 | |
Total current | (1,538) | 3,389 |
Provision for income taxes | $ (1,538) | $ 3,389 |
Income Taxes - Other (Details)
Income Taxes - Other (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Loss before income taxes | ||
U.S. operations | $ 142,943 | $ 16,427 |
Loss before income taxes | 142,943 | 16,427 |
U.S. State | (1,538) | 2,445 |
Foreign | 944 | |
Gross deferred tax assets: | ||
Net operating loss carryforwards | 72,792 | 58,056 |
Accrued expenses | 1,754 | 124 |
Amortization of intangible | 468 | 454 |
Stock-based compensation | 8,741 | 8,091 |
Research and development and other credits and other carryforwards | 39,588 | 30,627 |
Lease liability | 345 | 440 |
Capitalized research and development expenses | 37,643 | 22,382 |
Royalty/ Deferred Revenue | 4,298 | 3,781 |
Capital Loss | 74 | 94 |
Interest carryforward | 2,152 | |
Other | 152 | 64 |
Total gross deferred tax assets | 168,007 | 124,113 |
Gross deferred tax liabilities: | ||
ROU Asset | (237) | (291) |
Depreciation | (458) | (224) |
Unrealized income | (209) | (192) |
Total gross deferred tax liabilities | (904) | (707) |
Net deferred tax assets | 167,103 | 123,406 |
Less: valuation allowance | (167,103) | (123,406) |
Increase (decrease) in valuation allowance | 43,700 | 7,700 |
Unrecognized tax benefits | 0 | 0 |
Liability for uncertain tax positions | 0 | 0 |
Interest and penalties accrued related to unrecognized tax benefits | 0 | 0 |
Income tax benefit | $ 1,538 | $ (3,389) |
Reconciliation of income tax expense (benefit) at the statutory federal income tax rate and income taxes | ||
Federal income tax expense at statutory rate (as a percent) | 21% | 21% |
Permanent items (as a percent) | (0.40%) | (2.60%) |
State income tax, net of federal benefit (as a percent) | 4.10% | (9.00%) |
R&D tax credits (as a percent) | 6.30% | 88.10% |
Change in state apportionment (as a percent) | 2.50% | (59.50%) |
Foreign withholding tax (as a percent) | (5.70%) | |
Change in valuation allowance (as a percent) | (30.60%) | (47.00%) |
Other (as a percent) | (1.80%) | (5.90%) |
Effective income tax rate (as a percent) | 1.10% | (20.60%) |
Federal | ||
Loss before income taxes | ||
Net operating loss carry forwards available to offset future federal and state taxable income | $ 292,200 | $ 233,500 |
Net operating loss carry forwards subject to expiration | 81,100 | |
State | ||
Loss before income taxes | ||
Net operating loss carry forwards available to offset future federal and state taxable income | 240,100 | 204,600 |
Tax credit carryovers | 400 | |
Collaboration Agreement with Tenacia | ||
Loss before income taxes | ||
Foreign | 900 | |
Research and development | ||
Loss before income taxes | ||
U.S. State | $ 2,400 | |
Research and development | Federal | ||
Loss before income taxes | ||
Tax credit carryovers | $ 39,200 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Pay vs Performance Disclosure | ||
Net Income (Loss) | $ (141,405) | $ (19,816) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Dec. 31, 2023 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |