Marinus Pharmaceuticals (MRNS) 10-Q 8 May 24 2024 Q1 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	3 Months Ended
	Mar. 31, 2024	May 03, 2024
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2024
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 Rd
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 Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Non-accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		54,933,774
Entity Central Index Key	0001267813
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q1
Amendment Flag	false

CONSOLIDATED BALANCE SHEETS

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 104,253	$ 120,572
Short-term investments	9,000	29,716
Accounts receivable, net	3,513	3,799
Inventory	5,783	2,413
Prepaid expenses and other current assets	8,727	8,746
Total current assets	131,276	165,246
Property and equipment, net	3,735	3,843
Other assets	2,339	1,819
Total assets	137,350	170,908
Current liabilities:
Accounts payable	6,125	4,003
Current portion of notes payable	15,401	11,551
Current portion of revenue interest financing payable	2,511	2,211
Accrued expenses	18,721	22,859
Total current liabilities	42,758	40,624
Notes payable, net of deferred financing costs	58,072	61,423
Revenue interest financing payable, net of deferred financing costs	34,642	33,766
Contract liabilities, net	17,730	17,545
Other long-term liabilities	582	785
Total liabilities	153,784	154,143
Stockholders' equity:
Common stock, $0.001 par value; 150,000,000 shares authorized, 54,938,349 issued and 54,931,042 outstanding at March 31, 2024 and 54,585,428 issued and 54,578,121 outstanding at December 31, 2023	55	55
Additional paid-in capital	594,106	588,656
Treasury stock at cost, 7,307 shares at March 31, 2024 and December 31, 2023
Accumulated other comprehensive loss		(20)
Accumulated deficit	(610,595)	(571,926)
Total stockholders' (deficit) equity	(16,434)	16,765
Total liabilities and stockholders' (deficit) equity	$ 137,350	$ 170,908

CONSOLIDATED BALANCE SHEETS (Pa

CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Mar. 31, 2024	Dec. 31, 2023
CONSOLIDATED BALANCE SHEETS
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	54,938,349	54,585,428
Common stock, shares outstanding	54,931,042	54,578,121
Treasury stock, shares	7,307	7,307

CONSOLIDATED STATEMENTS OF OPER

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Revenue:
Federal contract revenue	$ 152	$ 7,048
Total revenue	7,679	10,380
Expenses:
Research and development	24,118	27,933
Selling, general and administrative	18,626	15,204
Total expenses	43,500	43,343
Loss from operations	(35,821)	(32,963)
Interest income	1,462	2,343
Interest expense	(4,346)	(4,147)
Other income, net	36	37
Net loss applicable to common shareholders	$ (38,669)	$ (34,730)
Per share information:
Net loss per share of common stock-basic	$ (0.68)	$ (0.67)
Net loss per share of common stock-diluted	$ (0.68)	$ (0.67)
Basic weighted average shares outstanding	56,851,811	51,769,685
Diluted weighted average shares outstanding	56,851,811	51,769,685
Other comprehensive income:
Unrealized gain on available-for-sale securities	$ 20	$ 74
Total comprehensive loss	(38,649)	(34,656)
Product revenue, net
Revenue:
Revenue	7,509	3,332
Expenses:
Cost of product revenue	756	$ 206
Collaboration revenue
Revenue:
Revenue	$ 18

CONSOLIDATED STATEMENTS OF CASH

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Cash flows from operating activities
Net loss	$ (38,669)	$ (34,730)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	131	157
Amortization of debt issuance costs	609	480
Accretion of revenue interest financing debt, net of cash paid	1,065	1,440
Amortization of discount on short-term investments	(149)	(228)
Stock-based compensation expense	5,193	3,741
Amortization of net contract asset/liability	(337)	(413)
Noncash lease expense	47	53
Noncash lease liability	224	96
Write off of fixed assets		62
Changes in operating assets and liabilities:
Net contract asset/liability	522	544
Prepaid expenses and other current assets, non-current assets, inventory and accounts receivable	(3,833)	(8,963)
Accounts payable and accrued expenses	(2,264)	(3,717)
Net cash used in operating activities	(37,461)	(41,478)
Cash flows from investing activities
Maturities of short-term investments	20,885
Purchases of short-term investments		(51,995)
Net cash provided by (used in) investing activities	20,885	(51,995)
Cash flows from financing activities
Proceeds from exercise of stock options	257
Other cash flows from financing activities		(174)
Net cash provided by (used in) financing activities	257	(174)
Net decrease in cash and cash equivalents	(16,319)	(93,647)
Cash and cash equivalents-beginning of period	120,572	240,551
Cash and cash equivalents-end of period	104,253	146,904
Supplemental disclosure of cash flow information
Unrealized gain on short-term investments	20	74
Cash paid for interest during the period	$ 2,671	$ 2,156

CONSOLIDATED STATEMENTS OF (DEF

CONSOLIDATED STATEMENTS OF (DEFICIT) 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, 2022	$ 4,043	$ 50	$ 542,428			$ (430,521)	$ 116,000
Balance Beginning (in shares) at Dec. 31, 2022	4,300	49,642,767		7,307
Increase (Decrease) in Stockholders' Equity (Deficit)
Stock-based compensation expense			3,741				3,741
Net issuance of common stock in connection with the vesting of restricted stock (in shares)		22,350
Unrealized gain on short-term investments					$ 74		74
Net Income (Loss)						(34,730)	(34,730)
Balance Ending at Mar. 31, 2023	$ 4,043	$ 50	546,169		74	(465,251)	85,085
Balance Ending (in shares) at Mar. 31, 2023	4,300	49,665,117		7,307
Balance Beginning at Dec. 31, 2023		$ 55	588,656		(20)	(571,926)	16,765
Balance Beginning (in shares) at Dec. 31, 2023		54,578,121		7,307
Increase (Decrease) in Stockholders' Equity (Deficit)
Stock-based compensation expense			5,193				5,193
Exercise of stock options			257				257
Exercise of stock options (in shares)		57,665
Net issuance of common stock in connection with the vesting of restricted stock (in shares)		295,256
Unrealized gain on short-term investments					$ 20		20
Net Income (Loss)						(38,669)	(38,669)
Balance Ending at Mar. 31, 2024		$ 55	$ 594,106			$ (610,595)	$ (16,434)
Balance Ending (in shares) at Mar. 31, 2024		54,931,042		7,307

Description of the Business and

Description of the Business and Liquidity	3 Months Ended
	Mar. 31, 2024
Description of the Business and Liquidity
Description of the Business and Liquidity	​ 1. Description of the Business and Liquidity 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 (SE). On March 18, 2022, the U.S. Food and Drug Administration (FDA) approved our new drug application (NDA) for the use of ZTALMY® (ganaxolone) 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). 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. Patients with SE who do not respond to first-line benzodiazepine treatment are classified as having Established Status Epilepticus (ESE) and those who then progress to and subsequently fail at least one second-line antiepileptic drug are classified as having RSE. ​ 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 ​ 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 $38.7 million for the three months ended March 31, 2024. 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 March 31, 2024 was $610.6 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 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 $113.3 million as of March 31, 2024. We believe that such amount is not sufficient to fund our operations for the one-year period after the date these 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. Cost reduction activities are being implemented, with expected impact beginning in the second quarter of 2024. Management’s plans that are intended to further mitigate this risk include securing additional funding in the future from one or more equity or debt financings, government funding, collaborations, licensing transactions, other commercial or strategic transactions or other sources. 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	3 Months Ended
	Mar. 31, 2024
Summary of Significant Accounting Policies
Summary of Significant Accounting Policies	2. Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited interim consolidated financial statements include the accounts of Marinus Pharmaceuticals, Inc. (a Delaware corporation) as well as the accounts of Marinus Pharmaceuticals Emerald Limited (an Ireland company incorporated in February 2021), a wholly owned subsidiary requiring consolidation. Marinus Pharmaceuticals Emerald Limited serves as a corporate presence in the European Union for regulatory purposes. The unaudited interim consolidated financial statements included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (SEC). Accordingly, they do not include all information and disclosures necessary for a presentation of our financial position, results of operations and cash flows in conformity with generally accepted accounting principles in the U.S. (GAAP) for annual financial statements. In the opinion of management, these unaudited interim consolidated financial statements reflect all adjustments, consisting primarily of normal recurring accruals, necessary for a fair presentation of our financial position and results of operations and cash flows for the periods presented. The results of operations for interim periods are not necessarily indicative of the results for the full year. These unaudited interim consolidated financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2023 and accompanying notes thereto included in our Annual Report on Form 10-K filed with the SEC on March 5, 2024. Use of Estimates The preparation of financial statements in conformity with 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 income and expenses during the reporting period. Actual results could differ from such estimates. 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 in the third quarter of 2022. 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. 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 interim consolidated balance sheets. This revenue is not within the scope of ASC 606 – Revenue from contracts with customers. 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 income (loss) within stockholders’ equity. All of our investments were short-term in nature as of March 31, 2024. Accounts Receivable, net Net trade receivables related to ZTALMY sales, which are recorded in Accounts receivable, net on the consolidated balance sheets, were approximately $2.8 million and $2.6 million as of March 31, 2024 and December 31, 2023, respectively. As of both March 31, 2024 and December 31, 2023, we had no allowance for doubtful accounts. 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 12). Inventory Debt Issuance Costs Debt issuance costs incurred in connection with Note payable (Note 10) and Revenue interest financing payable (Note 11) are amortized to Interest expense over the term of the respective financing arrangement using the effective-interest method. Debt issuance costs, net of related amortization, are deducted from the carrying value of the related debt. Contract Liabilities, net When consideration is received, or such consideration is unconditionally due, from a customer prior to completing our performance obligation to the customer under the terms of a contract, a Contract liability is recorded. Contract liabilities expected to be recognized as revenue or a reduction of expense within the 12 months following the balance sheet date are classified as Current liabilities. Contract liabilities not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as Long-term liabilities. In accordance with ASC 210-20, our Contract liabilities were partially offset by our Contract assets at March 31, 2024, as further discussed in Note 12. Liability Related to Revenue Interest Financing and Non-Cash Interest Expense In October 2022, we recognized a liability related to the Revenue Interest Financing Agreement with Sagard Healthcare Royalty Partners, LP (Sagard) under ASC 470-10 Deb Interest - Imputation of Interest Collaboration and Licensing Revenue We may enter into collaboration and licensing arrangements for research and development, manufacturing, and commercialization activities with counterparties for the development and commercialization of our product candidates. These arrangements may contain multiple components, such as (i) licenses, (ii) research and development activities, and (iii) the manufacturing of certain material. Payments pursuant to these arrangements may include non-refundable and refundable payments, payments upon the achievement of significant regulatory, development and commercial milestones, sales of product at certain agreed-upon amounts, and royalties on product sales. The amount of variable consideration is constrained until it is probable that the revenue is not at a significant risk of reversal in a future period. In determining the appropriate amount of revenue to be recognized as we fulfill our obligations under a collaboration agreement, we perform the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services 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. 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.

Cash, Cash Equivalents and Shor

Cash, Cash Equivalents and Short-Term Investments	3 Months Ended
	Mar. 31, 2024
Cash, Cash Equivalents and Short-Term Investments
Cash, Cash Equivalents and Short-Term Investments	3. Cash, Cash Equivalents and Short-Term Investments million in money market funds. As of December 31, 2023, our Cash and cash equivalents included million, respectively, of accrued interest receivable related to our Short-term investments. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized Cost Unrealized Gains Unrealized Losses Fair Value ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury securities ​ $ 9,000 ​ $ 155 ​ $ (155) ​ $ 9,000 Total ​ $ 9,000 ​ $ 155 ​ $ (155) ​ $ 9,000 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 ​

Fair Value Measurements

Fair Value Measurements	3 Months Ended
	Mar. 31, 2024
Fair Value Measurements
Fair Value Measurements	4. 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 (the 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 March 31, 2024 and December 31, 2023, all of our financial assets and liabilities were classified as Level 1 or Level 2 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 March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash ​ $ 1,420 ​ $ — ​ $ — ​ $ 1,420 ​ Money market funds (cash equivalents) ​ ​ 102,833 ​ ​ — ​ ​ — ​ ​ 102,833 ​ U.S. Treasury securities ​ ​ — ​ ​ 9,000 ​ ​ — ​ ​ 9,000 ​ Total assets ​ $ 104,253 ​ $ 9,000 ​ $ — ​ $ 113,253 ​ 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 ​ Agency securities ​ ​ — ​ ​ 2,881 ​ ​ — ​ ​ 2,881 ​ Total assets ​ $ 120,572 ​ $ 29,716 ​ $ — ​ $ 150,288 ​ ​

Inventory

Inventory	3 Months Ended
	Mar. 31, 2024
Inventory
Inventory	5. Inventory ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ ​ ​ 2024 ​ 2023 ​ Raw materials ​ $ 3,600 ​ $ 436 Work in process ​ ​ 1,591 ​ ​ 1,075 ​ Finished goods ​ ​ 592 ​ ​ 902 ​ Total Inventories ​ $ 5,783 ​ $ 2,413 ​ ​

Accrued Expenses

Accrued Expenses	3 Months Ended
	Mar. 31, 2024
Accrued Expenses
Accrued Expenses	6. Accrued Expenses Accrued expenses consisted of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ ​ ​ 2024 ​ 2023 ​ Payroll and related costs ​ $ 2,830 ​ $ 7,746 Clinical trials and drug development ​ ​ 5,694 ​ ​ 4,701 ​ Accrued license agreement payment ​ ​ 2,000 ​ ​ 4,000 ​ Professional fees ​ ​ 1,669 ​ ​ 1,236 ​ Selling and commercial liabilities ​ ​ 4,502 ​ ​ 3,901 ​ Short-term lease liabilities ​ ​ 1,306 ​ ​ 774 ​ Other ​ ​ 720 ​ ​ 501 ​ Total accrued expenses ​ $ 18,721 ​ $ 22,859 ​ ​

Loss Per Share of Common Stock

Loss Per Share of Common Stock	3 Months Ended
	Mar. 31, 2024
Loss Per Share of Common Stock
Loss Per Share of Common Stock	7. Loss Per Share of Common Stock Basic loss per share of common stock is computed by dividing Net loss attributable to common stockholders by the Weighted average number of shares of common stock outstanding during each period. Diluted loss per share of common stock 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, 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 8. ​ The pre-funded warrants to purchase common stock 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 8. ​ The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares of common stock outstanding, as they would be anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ ​ ​ ​ March 31, ​ ​ ​ ​ 2024 ​ 2023 ​ ​ Convertible preferred stock ​ — ​ 860,000 ​ Restricted stock awards and restricted stock units ​ 2,293,878 ​ 1,542,115 ​ Stock options ​ 8,671,041 ​ 7,143,397 ​ ​ ​ 10,964,919 ​ 9,545,512 ​ ​

Stockholders' Equity

Stockholders' Equity	3 Months Ended
	Mar. 31, 2024
Stockholders' Equity
Stockholders' Equity	8. Stockholders’ Equity In 2005, we adopted the 2005 Stock Option and Incentive Plan (2005 Plan) that authorizes us to grant stock options, restricted stock and other equity-based awards. As of March 31, 2024, no 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 stock options, restricted stock, and other equity-based awards, subject to adjustment in accordance with the 2014 Plan. As of March 31, 2024, 6,389,758 options to purchase shares of common stock were outstanding pursuant to grants in connection with the 2014 Plan, and 260,569 shares of common stock were available for future issuance. The amount, terms of grants, and exercisability provisions are determined and set by our board of directors. 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 3,090,220. Stock Options There were 8,671,041 stock options outstanding as of March 31, 2024 at a weighted average exercise price of $9.69 per share, including 2,281,283 stock options outstanding outside of the 2014 Plan, granted as inducements to new employees. Restricted Stock and Restricted Stock Units All issued and outstanding restricted shares of common stock are time-based, and become vested within two years of 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 March 31, 2024, we did not have any restricted shares of common stock outstanding. During the three months ended March 31, 2024, we granted 1,350,244 restricted stock units, which generally vest within three years of the grant date, pursuant to the 2014 Plan. As of March 31, 2024, we had 2,293,878 restricted stock units outstanding. Total compensation cost recognized for all stock options, restricted stock awards and restricted stock units in the statements of operations is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ ​ ​ March 31, ​ ​ ​ 2024 ​ 2023 ​ Research and development $ 1,832 $ 1,342 Selling, general and administrative ​ 3,361 ​ 2,399 ​ Total ​ $ 5,193 ​ $ 3,741 ​ ​ ​ Preferred Stock As of March 31, 2024 all shares of our Series A Convertible Preferred Stock (Preferred Stock) had been converted and none remained outstanding. In the three months ended March 31, 2023, 4,300 shares of our Preferred stock remained outstanding, convertible into 860,000 shares of our common stock. In the three months ended March 31, 2023, there were no conversions of shares of our Preferred Stock into shares of our common stock. 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 with its affiliates, would beneficially own more than 9.99% of the number of shares of common stock outstanding immediately after giving effect to such exercise. A holder of Pre-funded Warrants may increase or decrease this percentage not in excess of 19.99% by providing at least 61 days’ prior notice to us. 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 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. We did not sell any shares of our common stock during the three months ended March 31, 2024 and March 31, 2023 under the EDA.

Leases

Leases	3 Months Ended
	Mar. 31, 2024
Leases
Leases	9. Leases We have entered into one operating lease for real estate and several operating leases for clinical site equipment. Our real estate operating lease has a term of 78 months, and includes renewal terms which can extend the lease term by 60 months, which we include in the lease term when it is reasonably certain that we will exercise the option. As of March 31, 2024, our operating lease had a remaining lease term of 18 months. Our operating leases for clinical site equipment each have a term of 18 months and include renewal terms that can extend the lease terms monthly at the end of each applicable term. As of March 31, 2024, our operating leases for clinical site equipment had an average remaining term of 14 months. The right-of-use (ROU) assets are included in Other assets on our interim consolidated balance sheets as of March 31, 2024 and December 31, 2023, and represent our right to use the underlying assets for the applicable lease terms. Our obligations to make lease payments are included in both Accrued expenses and Other long-term liabilities on our interim consolidated balance sheets as of March 31, 2024 and December 31, 2023. 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 March 31, 2024 and December 31, 2023, ROU assets were $1.5 million and $1.0 million, respectively, and operating lease liabilities were $1.9 million and $1.4 million, respectively. We have entered into 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 the three months ended March 31, 2024 and 2023, we recognized $0.3 million and $0.1 million, respectively, in total lease costs, of which less than $0.1 million related to short-term operating leases for each period. Because the rate implicit in each 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 ROU assets and lease liabilities was 11.0%, derived from a corporate yield curve based on a synthetic credit rating model using a market signal analysis. 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 March 31, 2024 and December 31, 2023, we have not recognized any impairment losses for our ROU assets. Maturities of operating lease liabilities as of March 31, 2024 were as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Remainder of 2024 ​ $ 1,073 ​ 2025 ​ 955 ​ ​ ​ ​ 2,028 ​ Less: imputed interest ​ ​ (140) ​ Total lease liabilities ​ $ 1,888 ​ ​ ​ ​ ​ ​ Current operating lease liabilities ​ $ 1,306 ​ Non-current operating lease liabilities ​ ​ 582 ​ Total lease liabilities ​ $ 1,888 ​ ​

Notes Payable

Notes Payable	3 Months Ended
	Mar. 31, 2024
Notes Payable
Notes Payable	​ 10. 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 increased 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, 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 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 March 31, 2024, 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-liabilities on our consolidated balance sheets and are being recognized as Interest expenses 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-liabilities on the consolidated balance sheet and are being recognized as Interest expenses over the term of the loan using the effective-interest method. For the three months ended March 31, 2024, we recognized interest expense of $2.7 million, of which $2.2 million was interest on the Term Loans and $0.5 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 March 31, 2024 (in thousands): ​ ​ ​ ​ ​ Gross proceeds ​ $ 75,000 Contractual exit fee ​ 2,003 Unamortized debt discount and issuance costs ​ (3,530) Total note payable ​ $ 73,473 ​ ​ ​ ​ Current portion of note payable ​ ​ 15,401 Non-current portion of note payable ​ ​ 58,072 Total note payable ​ $ 73,473 ​ The aggregate maturities of Notes payable as of March 31, 2024 are as follows (in thousands): ​ ​ ​ ​ Remainder of 2024 ​ $ 11,250 2025 ​ ​ 15,000 2026 ​ ​ 48,750 Total ​ $ 75,000 ​

Revenue Interest Financing Agre

Revenue Interest Financing Agreement	3 Months Ended
	Mar. 31, 2024
Revenue Interest Financing Payable
Revenue Interest Financing Payable	11. Revenue Interest Financing Agreement ​ On October 28, 2022 (Closing Date), we entered into a revenue interest financing agreement (Revenue Interest Financing Agreement) with S agard 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 10. ​ Issuance costs pursuant to the Revenue Interest Financing Agreement consisted primarily of advisory and legal fees million. These issuance costs were recorded as a direct deduction to the carrying amount of the liability and will be amortized under the effective interest method over the estimated period the liability will be repaid. For the three months ended March 31, 2024, w ​ The following table summarizes the activity of the Revenue Interest Financing Agreement for the three months ended March 31, 2023 and March 31, 2024 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the three months ended March 31, 2023 ​ ​ ​ ​ Revenue Interest Financing Balance at December 31, 2022 ​ $ 30,877 ​ Non-cash interest expense in the three months ended March 31, 2023 ​ ​ 1,440 ​ Amortization of debt discount in the three months ended March 31, 2023 ​ ​ 71 ​ Payments made in the three months ended March 31, 2023 ​ ​ (174) ​ Revenue Interest Financing Balance at March 31, 2023 ​ $ 32,214 ​ ​ ​ ​ ​ ​ Current portion of revenue interest financing liability ​ ​ 1,321 ​ Long-term portion of revenue interest financing liability ​ ​ 30,893 ​ Revenue Interest Financing Balance at March 31, 2023 ​ $ 32,214 ​ ​ ​ ​ ​ ​ For the three months ended March 31, 2024 ​ ​ ​ ​ Revenue Interest Financing Balance at December 31, 2023 ​ $ 35,977 ​ Non-cash interest expense in the three months ended March 31, 2024 ​ ​ 1,556 ​ Amortization of debt discount in the three months ended March 31, 2024 ​ ​ 111 ​ Payments made in the three months ended March 31, 2024 ​ ​ (491) ​ Revenue Interest Financing Balance at March 31, 2024 ​ $ 37,153 ​ ​ ​ ​ ​ ​ Current portion of revenue interest financing liability ​ $ 2,511 ​ Long-term portion of revenue interest financing liability ​ ​ 34,642 ​ Revenue Interest Financing Balance at March 31, 2024 ​ $ 37,153 ​

Collaboration Revenue

Collaboration Revenue	3 Months Ended
	Mar. 31, 2024
Collaboration Revenue
Collaboration Revenue	12. 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 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 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, 2023, there was a total contract liability of $13.7 million and a total contract asset of $2.9 million. In accordance with ASC 210-20, the contract liability was offset by the contract asset related to the reimbursement of research and development costs, which resulted in a net contract liability of $10.8 million as of December 31, 2023. ​ Transaction Price and Net Contract Liability as of December 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 ​ During the three months ended March 31, 2024, we amortized $0.3 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 $13.4 million as of March 31, 2024. In accordance with ASC 210-20, the contract liability of $13.4 million is offset by the contract asset of $2.4 million related to the reimbursement of research and development costs, resulting in a net contract liability of $11.0 million as of March 31, 2024. Transaction Price and Net Contract Liability as of March 31, 2024: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cumulative Collaboration ​ ​ ​ Transaction ​ Revenue Recognized ​ Contract ​ Price as of March, 2024 Liability License $ 21,660 ​ $ 21,660 ​ $ - Development and Regulatory Services ​ 6,717 ​ ​ 2,848 ​ ​ 3,869 Supply of Licensed Product ​ 9,503 ​ ​ - ​ ​ 9,503 ​ $ 37,880 ​ $ 24,508 ​ $ 13,372 ​ ​ ​ ​ ​ ​ ​ ​ ​ Less Total Contract Asset ​ ​ ​ ​ ​ ​ ​ 2,372 Net Contract Liability ​ ​ ​ ​ ​ ​ $ 11,000 ​ We incurred $2.0 million of incremental costs in connection with 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 other current assets and other assets, which are being amortized as Development and Regulatory Services and Supply of Licensed 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 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. 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. ​ There was no activity during each of the three months ended March 31, 2024 and 2023. The cumulative collaboration revenue recognized as of March 31, 2024 and December 31, 2023 is $3.0 million, which was the $3.0 million transaction price associated with the License as revenue at contract inception. No license revenue was recorded during each of the three months ended March 31, 2024 and 2023. There was a total contract liability of $7.0 million as of both March 31, 2024 and December 31, 2023. In accordance with ASC 210-20, the contract liability of $7.0 million is offset by a contract asset of $0.7 million, resulting in a net contract liability of $6.3 million as of both March 31, 2024 and December 31, 2023. Transaction Price and Net Contract Liability as of March 31, 2024 and December 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cumulative Collaboration ​ ​ ​ Transaction ​ Revenue Recognized ​ Contract ​ Price as of March 31, 2024 and December 31, 2023 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, 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 distribution 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

Subsequent Events

Subsequent Events	3 Months Ended
	Mar. 31, 2024
Subsequent Events
Subsequent Events	13. Subsequent Events modification. We have decided to complete enrollment in the RAISE trial at 100 patients with top-line results expected in the summer of 2024. Cost reduction activities are being implemented with expected impact beginning in the second quarter of 2024. On April 30, 2024, we implemented a reduction-in-force (RIF) which impacted approximately 20% of our workforce.

Summary of Significant Accoun_2

Summary of Significant Accounting Policies (Policies)	3 Months Ended
	Mar. 31, 2024
Summary of Significant Accounting Policies
Basis of Presentation	Basis of Presentation The accompanying unaudited interim consolidated financial statements include the accounts of Marinus Pharmaceuticals, Inc. (a Delaware corporation) as well as the accounts of Marinus Pharmaceuticals Emerald Limited (an Ireland company incorporated in February 2021), a wholly owned subsidiary requiring consolidation. Marinus Pharmaceuticals Emerald Limited serves as a corporate presence in the European Union for regulatory purposes. The unaudited interim consolidated financial statements included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (SEC). Accordingly, they do not include all information and disclosures necessary for a presentation of our financial position, results of operations and cash flows in conformity with generally accepted accounting principles in the U.S. (GAAP) for annual financial statements. In the opinion of management, these unaudited interim consolidated financial statements reflect all adjustments, consisting primarily of normal recurring accruals, necessary for a fair presentation of our financial position and results of operations and cash flows for the periods presented. The results of operations for interim periods are not necessarily indicative of the results for the full year. These unaudited interim consolidated financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2023 and accompanying notes thereto included in our Annual Report on Form 10-K filed with the SEC on March 5, 2024.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with 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 income and expenses during the reporting period. Actual results could differ from such estimates.
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 in the third quarter of 2022. 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. 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 interim consolidated balance sheets. This revenue is not within the scope of ASC 606 – Revenue from contracts with customers.
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 income (loss) within stockholders’ equity. All of our investments were short-term in nature as of March 31, 2024.
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 $2.8 million and $2.6 million as of March 31, 2024 and December 31, 2023, respectively. As of both March 31, 2024 and December 31, 2023, we had no allowance for doubtful accounts. 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 12).
Inventory	Inventory
Debt Issuance Costs	Debt Issuance Costs Debt issuance costs incurred in connection with Note payable (Note 10) and Revenue interest financing payable (Note 11) are amortized to Interest expense over the term of the respective financing arrangement using the effective-interest method. Debt issuance costs, net of related amortization, are deducted from the carrying value of the related debt.
Contract Liabilities, net	Contract Liabilities, net When consideration is received, or such consideration is unconditionally due, from a customer prior to completing our performance obligation to the customer under the terms of a contract, a Contract liability is recorded. Contract liabilities expected to be recognized as revenue or a reduction of expense within the 12 months following the balance sheet date are classified as Current liabilities. Contract liabilities not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as Long-term liabilities. In accordance with ASC 210-20, our Contract liabilities were partially offset by our Contract assets at March 31, 2024, as further discussed in Note 12.
Liability Related to Revenue Interest Financing and Non-Cash Interest Expense	Liability Related to Revenue Interest Financing and Non-Cash Interest Expense In October 2022, we recognized a liability related to the Revenue Interest Financing Agreement with Sagard Healthcare Royalty Partners, LP (Sagard) under ASC 470-10 Deb Interest - Imputation of Interest
Collaboration and Licensing Revenue	Collaboration and Licensing Revenue We may enter into collaboration and licensing arrangements for research and development, manufacturing, and commercialization activities with counterparties for the development and commercialization of our product candidates. These arrangements may contain multiple components, such as (i) licenses, (ii) research and development activities, and (iii) the manufacturing of certain material. Payments pursuant to these arrangements may include non-refundable and refundable payments, payments upon the achievement of significant regulatory, development and commercial milestones, sales of product at certain agreed-upon amounts, and royalties on product sales. The amount of variable consideration is constrained until it is probable that the revenue is not at a significant risk of reversal in a future period. In determining the appropriate amount of revenue to be recognized as we fulfill our obligations under a collaboration agreement, we perform the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services 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. 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.

Cash, Cash Equivalents and Sh_2

Cash, Cash Equivalents and Short-Term Investments (Tables)	3 Months Ended
	Mar. 31, 2024
Cash, Cash Equivalents and Short-Term Investments
Schedule of short-term investments	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized Cost Unrealized Gains Unrealized Losses Fair Value ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury securities ​ $ 9,000 ​ $ 155 ​ $ (155) ​ $ 9,000 Total ​ $ 9,000 ​ $ 155 ​ $ (155) ​ $ 9,000 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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

Fair Value Measurements (Tables

Fair Value Measurements (Tables)	3 Months Ended
	Mar. 31, 2024
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 March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash ​ $ 1,420 ​ $ — ​ $ — ​ $ 1,420 ​ Money market funds (cash equivalents) ​ ​ 102,833 ​ ​ — ​ ​ — ​ ​ 102,833 ​ U.S. Treasury securities ​ ​ — ​ ​ 9,000 ​ ​ — ​ ​ 9,000 ​ Total assets ​ $ 104,253 ​ $ 9,000 ​ $ — ​ $ 113,253 ​ 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 ​ Agency securities ​ ​ — ​ ​ 2,881 ​ ​ — ​ ​ 2,881 ​ Total assets ​ $ 120,572 ​ $ 29,716 ​ $ — ​ $ 150,288 ​ ​
Schedule of short-term investments	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized Cost Unrealized Gains Unrealized Losses Fair Value ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury securities ​ $ 9,000 ​ $ 155 ​ $ (155) ​ $ 9,000 Total ​ $ 9,000 ​ $ 155 ​ $ (155) ​ $ 9,000 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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

Inventory (Tables)

Inventory (Tables)	3 Months Ended
	Mar. 31, 2024
Inventory
Schedule of inventory	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ ​ ​ 2024 ​ 2023 ​ Raw materials ​ $ 3,600 ​ $ 436 Work in process ​ ​ 1,591 ​ ​ 1,075 ​ Finished goods ​ ​ 592 ​ ​ 902 ​ Total Inventories ​ $ 5,783 ​ $ 2,413 ​ ​

Accrued Expenses (Tables)

Accrued Expenses (Tables)	3 Months Ended
	Mar. 31, 2024
Accrued Expenses
Schedule of accrued expenses	Accrued expenses consisted of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ ​ ​ 2024 ​ 2023 ​ Payroll and related costs ​ $ 2,830 ​ $ 7,746 Clinical trials and drug development ​ ​ 5,694 ​ ​ 4,701 ​ Accrued license agreement payment ​ ​ 2,000 ​ ​ 4,000 ​ Professional fees ​ ​ 1,669 ​ ​ 1,236 ​ Selling and commercial liabilities ​ ​ 4,502 ​ ​ 3,901 ​ Short-term lease liabilities ​ ​ 1,306 ​ ​ 774 ​ Other ​ ​ 720 ​ ​ 501 ​ Total accrued expenses ​ $ 18,721 ​ $ 22,859 ​ ​

Loss Per Share of Common Stock

Loss Per Share of Common Stock (Tables)	3 Months Ended
	Mar. 31, 2024
Loss Per Share of Common Stock
Schedule of the computation of basic and diluted loss per share	​
Schedule of antidilutive securities excluded from the computation of diluted weighted average shares outstanding	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ ​ ​ ​ March 31, ​ ​ ​ ​ 2024 ​ 2023 ​ ​ Convertible preferred stock ​ — ​ 860,000 ​ Restricted stock awards and restricted stock units ​ 2,293,878 ​ 1,542,115 ​ Stock options ​ 8,671,041 ​ 7,143,397 ​ ​ ​ 10,964,919 ​ 9,545,512 ​ ​

Stockholders' Equity (Tables)

Stockholders' Equity (Tables)	3 Months Ended
	Mar. 31, 2024
Stockholders' Equity
Schedule of total compensation cost recognized in the statement of operations	Total compensation cost recognized for all stock options, restricted stock awards and restricted stock units in the statements of operations is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ ​ ​ March 31, ​ ​ ​ 2024 ​ 2023 ​ Research and development $ 1,832 $ 1,342 Selling, general and administrative ​ 3,361 ​ 2,399 ​ Total ​ $ 5,193 ​ $ 3,741 ​

Leases (Tables)

Leases (Tables)	3 Months Ended
	Mar. 31, 2024
Leases
Schedule of maturities of operating lease liabilities	Maturities of operating lease liabilities as of March 31, 2024 were as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Remainder of 2024 ​ $ 1,073 ​ 2025 ​ 955 ​ ​ ​ ​ 2,028 ​ Less: imputed interest ​ ​ (140) ​ Total lease liabilities ​ $ 1,888 ​ ​ ​ ​ ​ ​ Current operating lease liabilities ​ $ 1,306 ​ Non-current operating lease liabilities ​ ​ 582 ​ Total lease liabilities ​ $ 1,888 ​ ​

Notes Payable (Tables)

Notes Payable (Tables)	3 Months Ended
	Mar. 31, 2024
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 March 31, 2024 (in thousands): ​ ​ ​ ​ ​ Gross proceeds ​ $ 75,000 Contractual exit fee ​ 2,003 Unamortized debt discount and issuance costs ​ (3,530) Total note payable ​ $ 73,473 ​ ​ ​ ​ Current portion of note payable ​ ​ 15,401 Non-current portion of note payable ​ ​ 58,072 Total note payable ​ $ 73,473
Schedule of maturities of notes payable over the next five years	The aggregate maturities of Notes payable as of March 31, 2024 are as follows (in thousands): ​ ​ ​ ​ Remainder of 2024 ​ $ 11,250 2025 ​ ​ 15,000 2026 ​ ​ 48,750 Total ​ $ 75,000 ​

Revenue Interest Financing Ag_2

Revenue Interest Financing Agreement (Tables)	3 Months Ended
	Mar. 31, 2024
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 three months ended March 31, 2023 and March 31, 2024 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the three months ended March 31, 2023 ​ ​ ​ ​ Revenue Interest Financing Balance at December 31, 2022 ​ $ 30,877 ​ Non-cash interest expense in the three months ended March 31, 2023 ​ ​ 1,440 ​ Amortization of debt discount in the three months ended March 31, 2023 ​ ​ 71 ​ Payments made in the three months ended March 31, 2023 ​ ​ (174) ​ Revenue Interest Financing Balance at March 31, 2023 ​ $ 32,214 ​ ​ ​ ​ ​ ​ Current portion of revenue interest financing liability ​ ​ 1,321 ​ Long-term portion of revenue interest financing liability ​ ​ 30,893 ​ Revenue Interest Financing Balance at March 31, 2023 ​ $ 32,214 ​ ​ ​ ​ ​ ​ For the three months ended March 31, 2024 ​ ​ ​ ​ Revenue Interest Financing Balance at December 31, 2023 ​ $ 35,977 ​ Non-cash interest expense in the three months ended March 31, 2024 ​ ​ 1,556 ​ Amortization of debt discount in the three months ended March 31, 2024 ​ ​ 111 ​ Payments made in the three months ended March 31, 2024 ​ ​ (491) ​ Revenue Interest Financing Balance at March 31, 2024 ​ $ 37,153 ​ ​ ​ ​ ​ ​ Current portion of revenue interest financing liability ​ $ 2,511 ​ Long-term portion of revenue interest financing liability ​ ​ 34,642 ​ Revenue Interest Financing Balance at March 31, 2024 ​ $ 37,153 ​

Collaboration Revenue (Tables)

Collaboration Revenue (Tables)	3 Months Ended
	Mar. 31, 2024
Collaboration Revenue
Schedule of allocation of the transaction price to the performance obligations	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 March, 2024 Liability License $ 21,660 ​ $ 21,660 ​ $ - Development and Regulatory Services ​ 6,717 ​ ​ 2,848 ​ ​ 3,869 Supply of Licensed Product ​ 9,503 ​ ​ - ​ ​ 9,503 ​ $ 37,880 ​ $ 24,508 ​ $ 13,372 ​ ​ ​ ​ ​ ​ ​ ​ ​ Less Total Contract Asset ​ ​ ​ ​ ​ ​ ​ 2,372 Net Contract Liability ​ ​ ​ ​ ​ ​ $ 11,000 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cumulative Collaboration ​ ​ ​ Transaction ​ Revenue Recognized ​ Contract ​ Price as of March 31, 2024 and December 31, 2023 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

Description of the Business a_2

Description of the Business and Liquidity (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Liquidity
Net loss	$ 38,669	$ 34,730
Accumulated deficit	610,595		$ 571,926
Cash, cash equivalents and investment balances	$ 113,300

Summary of Significant Accoun_3

Summary of Significant Accounting Policies - Other (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($) customer	Dec. 31, 2023 USD ($)
Product Revenue, net
Number of customers | customer	3
Trade Receivables, net
Accounts receivable, net	$ 3,513	$ 3,799
Allowance for doubtful accounts.	$ 0	0
Payment terms	30 days
ZTALMY
Trade Receivables, net
Accounts receivable, net	$ 2,800	$ 2,600
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

Cash, Cash Equivalents and Sh_3

Cash, Cash Equivalents and Short-Term Investments - Cash and Cash Equivalents (Details) - USD ($) $ in Millions	Mar. 31, 2024	Dec. 31, 2023
Cash, Cash Equivalents and Short-Term Investments
Cash accounts in banking institutions	$ 1.4	$ 1.3
Money market funds	102.9	119.3
Accrued interest receivable	$ 0.1	$ 0.2

Cash, Cash Equivalents and Sh_4

Cash, Cash Equivalents and Short-Term Investments - Short-Term Investments (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Short-term Investments
Amortized Cost	$ 9,000	$ 29,736
Unrealized Gains	155	169
Unrealized Losses	(155)	(189)
Fair Value	9,000	29,716
U.S. Treasury securities
Short-term Investments
Amortized Cost	9,000	26,852
Unrealized Gains	155	138
Unrealized Losses	(155)	(155)
Fair Value	$ 9,000	26,835
U.S. Government Agency securities
Short-term Investments
Amortized Cost		2,884
Unrealized Gains		31
Unrealized Losses		(34)
Fair Value		$ 2,881

Fair Value Measurements - Fair

Fair Value Measurements - Fair Value Recurring Basis (Details) - Recurring basis - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Assets
Total assets	$ 113,253	$ 150,288
U.S. Treasury securities
Assets
Investments	9,000	26,835
U.S. Government Agency securities
Assets
Investments		2,881
Cash.
Assets
Cash and cash equivalents, fair value	1,420	1,255
Money market funds
Assets
Cash and cash equivalents, fair value	102,833	119,317
Level 1
Assets
Total assets	104,253	120,572
Level 1 | Cash.
Assets
Cash and cash equivalents, fair value	1,420	1,255
Level 1 | Money market funds
Assets
Cash and cash equivalents, fair value	102,833	119,317
Level 2
Assets
Total assets	9,000	29,716
Level 2 | U.S. Treasury securities
Assets
Investments	$ 9,000	26,835
Level 2 | U.S. Government Agency securities
Assets
Investments		$ 2,881

Inventory (Details)

Inventory (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Inventory
Raw materials	$ 3,600	$ 436
Work in process	1,591	1,075
Finished goods	592	902
Total Inventories	$ 5,783	$ 2,413

Accrued Expenses (Details)

Accrued Expenses (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Accrued Expenses
Payroll and related costs	$ 2,830	$ 7,746
Clinical trials and drug development	5,694	4,701
Accrued license agreement payment	2,000	4,000
Professional fees	1,669	1,236
Selling and commercial liabilities	4,502	3,901
Short-term lease liabilities	1,306	774
Other	720	501
Total accrued expenses	$ 18,721	$ 22,859

Loss Per Share of Common Stoc_2

Loss Per Share of Common Stock - Basic and diluted net loss per share as the exercise price (Details) - $ / shares	Mar. 31, 2024	Dec. 31, 2023
Loss Per Share of Common Stock
Warrant exercise price (in dollars per share)	$ 0.001	$ 0.001

Loss Per Share of Common Stoc_3

Loss Per Share of Common Stock - Anti-dilutive securities (Details) - shares	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Antidilutive securities
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares)	10,964,919	9,545,512
Convertible preferred stock
Antidilutive securities
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares)		860,000
RSAs and RSUs
Antidilutive securities
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares)	2,293,878	1,542,115
Stock options
Antidilutive securities
Antidilutive securities excluded from the computation of diluted weighted average shares outstanding (in shares)	8,671,041	7,143,397

Stockholders' Equity - Other (D

Stockholders' Equity - Other (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	3 Months Ended
	Nov. 30, 2022	Mar. 31, 2024	Mar. 31, 2023	Jan. 01, 2024
Stockholders' Equity
Total compensation cost		$ 5,193	$ 3,741
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%
Public Offering.
Stockholders' Equity
Stock Issued During Period, Shares, New Issues	12,421,053
Number of pre-funded warrants (in shares)	2,105,264
Proceeds from equity offerings, net of offering costs	$ 64,500
Research and development.
Stockholders' Equity
Total compensation cost		1,832	1,342
Selling, general and administrative
Stockholders' Equity
Total compensation cost		$ 3,361	$ 2,399
Series A Convertible Preferred Stock
Shares
Conversion of Series A Convertible Preferred Stock (in shares)			0
Number of common shares issuable upon conversion of stock			860,000
Preferred stock, shares outstanding		0	4,300
Employee Stock Option
Stock Options
Outstanding (in shares)		8,671,041
Outstanding, weighted-average exercise price (in dollars per share)		$ 9.69
Granted (in shares)		1,617,094
Granted (in dollars per share)		$ 9.76
Restricted common stock
Stockholders' Equity
Vesting period		2 years
2005 Plan
Stockholders' Equity
Common stock reserved for issuance (in shares)		0
2005 Plan | Employee Stock Option
Stock Options
Outstanding (in shares)		0
2014 Plan
Stockholders' Equity
Common stock reserved for issuance (in shares)		260,569		3,090,220
2014 Plan | Employee Stock Option
Stock Options
Outstanding (in shares)		6,389,758
Granted (in shares)		1,540,519
2014 Plan | Restricted Stock Units
Stockholders' Equity
Vesting period		3 years
Shares
Issued (in shares)		1,350,244
Awards outstanding (in shares)		2,293,878
Equity Incentive Plan, Employee Inducements | Employee Stock Option
Stock Options
Outstanding (in shares)		2,281,283
Granted (in shares)		76,575

Stockholders' Equity - Equity D

Stockholders' Equity - Equity Distribution Agreement (Details) - USD ($) shares in Millions	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
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%
Issuance of shares under equity distribution agreement (in shares)	0	0

Leases - Other (Details)

Leases - Other (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Leases
Operating lease agreement term	78 months
Operating lease renewal term	60 months
Weighted average remaining lease term	18 months
Right-of-use assets	$ 1,500	$ 1,000
Operating Lease, Right-of-Use Asset, Statement of Financial Position [Extensible List]	Other Assets, Noncurrent	Other Assets, Noncurrent
Operating lease liabilities	$ 1,888	$ 1,400

Leases - Costs (Details)

Leases - Costs (Details) - USD ($) $ in Millions	3 Months Ended		12 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Leases
Total lease costs	$ 0.3	$ 0.1
Weighted-average incremental borrowing rate used to determine right-of-use assets and lease liabilities	11%
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%
Maximum
Leases
Short-term operating lease costs	$ 0.1	$ 0.1

Leases - Maturities (Details)

Leases - Maturities (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Maturities of operating lease liabilities:
Remainder of 2024	$ 1,073
2025	955
Total lease payments	2,028
Less: imputed interest	(140)
Total lease liabilities	1,888	$ 1,400
Current operating lease liabilities	$ 1,306
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List]	Accrued Liabilities, Current	Accrued Liabilities, Current
Non-current operating lease liabilities	$ 582
Operating Lease, Liability, Noncurrent, Statement of Financial Position [Extensible List]	Other Accrued Liabilities, Noncurrent	Other Accrued Liabilities, Noncurrent

Notes Payable - Narratives (Det

Notes Payable - Narratives (Details) $ in Thousands		1 Months Ended			3 Months Ended
	May 11, 2021 USD ($) tranche	Mar. 31, 2022 USD ($)	Sep. 30, 2021 USD ($)	May 31, 2021 USD ($)	Mar. 31, 2024 USD ($)	Mar. 31, 2023 USD ($)	Aug. 31, 2023 USD ($)	Oct. 31, 2022	Oct. 27, 2022 USD ($)	Sep. 30, 2022
Notes Payable
Contractual exit fee					$ 2,003
Interest expense					4,346	$ 4,147
Amortization of debt issuance costs					609	$ 480
Total debt commitments					75,000		$ 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)	0.75%
Commitment fee accrual period	120 days
Debt issuance costs									$ 500
Debt issuance costs		1,800	1,200	4,400
Contractual exit fee			600	300
Interest expense					2,700
Interest on term loan					2,200
Amortization of debt issuance costs					$ 500
Exit fee, percentage	2.67%							2.67%	2%	2%
Oaktree Capital Management LP Credit Agreement | Maximum
Notes Payable
Contractual exit fee		600
Oaktree Capital Management LP Credit Agreement | Scenario Of Prepayment On Or Before May 11, 2023
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	Mar. 31, 2024	Dec. 31, 2023	Aug. 31, 2023
Notes Payable
Gross proceeds	$ 75,000		$ 75,000
Contractual exit fee	2,003
Unamortized debt discount and issuance costs	(3,530)
Total note payable	73,473
Current portion of note payable	15,401	$ 11,551
Non-current portion of note payable	$ 58,072	$ 61,423

Notes Payable - Debt maturities

Notes Payable - Debt maturities (Details) $ in Thousands	Mar. 31, 2024 USD ($)
Notes Payable
Remainder of 2024	$ 11,250
2025	15,000
2026	48,750
Total	$ 75,000

Revenue Interest Financing Ag_3

Revenue Interest Financing Agreement - Terms (Details) - USD ($) $ in Thousands		3 Months Ended
	Oct. 28, 2022	Mar. 31, 2024	Dec. 31, 2023
Revenue Interest Financing Payable
Professional fees		$ 1,669	$ 1,236
Total issuance costs		$ 2,600
Estimated effective annual interest rate, percentage		18%
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
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 Ag_4

Revenue Interest Financing Agreement - Summary of Activity (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Revenue Interest Financing Payable
Revenue Interest Financing Starting Balance	$ 35,977	$ 30,877
Non-cash interest expense	1,556	1,440
Amortization of debt discount	111	71
Payments	(491)	(174)
Revenue Interest Financing Ending Balance	37,153	32,214
Current portion of revenue interest financing liability	2,511	1,321	$ 2,211
Long-term portion of revenue interest financing liability	34,642	30,893	33,766
Revenue Interest Financing Balance	$ 37,153	$ 32,214	$ 35,977

Collaboration Revenue - Narrati

Collaboration Revenue - Narrative (Details) $ in Thousands, € in Millions			1 Months Ended		3 Months Ended		12 Months Ended
	Jul. 30, 2021 USD ($)	Jul. 30, 2021 EUR (€) item	May 31, 2023 USD ($)	Dec. 31, 2022 USD ($) item product	Mar. 31, 2024 USD ($)	Mar. 31, 2023 USD ($)	Dec. 31, 2022 USD ($) item	Dec. 31, 2023 USD ($)
License and Collaboration Revenue
Contract liability					$ 400			$ 0
Net Contract Liability					17,730			17,545
Collaboration revenue
License and Collaboration Revenue
Collaboration revenue					18
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
Collaboration revenue							$ 12,700
Transaction Price					37,880			37,880
Contract liability					13,372			13,709
Contract asset					2,372			2,912
Net Contract Liability					11,000			10,797
Offset by contract asset					2,372			2,912
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
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					300
Transaction Price					6,717			6,717
Contract liability					3,869			4,206
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			2
Number of commitments represent distinct performance | item				2			2
Transaction Price					10,000			10,000
Contract liability					7,002			7,002
Contract asset					700			700
Net Contract Liability					6,302			6,302
Offset by contract asset					700			700
Incremental costs incurred in obtaining the agreement					1,000
Capitalized contract costs					700
Cost of product 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	$ 0
Transaction Price					2,998			2,998
Collaboration Agreement with Tenacia | Collaboration revenue
License and Collaboration Revenue
Transaction Price					3,000			3,000
Collaboration Agreement with Tenacia | Supply of License Product
License and Collaboration Revenue
Transaction Price					7,002			7,002
Contract liability					7,002			$ 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

Collaboration Revenue - Allocat

Collaboration Revenue - Allocation of the transaction price to the performance obligations (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
License and Collaboration Revenue
Contract Liability	$ 400	$ 0
Net Contract Liability	17,730	17,545
Collaboration Agreement with Orion
License and Collaboration Revenue
Transaction Price	37,880	37,880
Cumulative Collaboration Revenue Recognized	24,508	24,171
Contract Liability	13,372	13,709
Less Total Contract Asset	2,372	2,912
Net Contract Liability	11,000	10,797
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	2,848	2,511
Contract Liability	3,869	4,206
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	10,000
Cumulative Collaboration Revenue Recognized	2,998	2,998
Contract Liability	7,002	7,002
Less Total Contract Asset	700	700
Net Contract Liability	6,302	6,302
Collaboration Agreement with Tenacia | License Revenue
License and Collaboration Revenue
Transaction Price	2,998	2,998
Cumulative Collaboration Revenue Recognized	2,998	2,998
Collaboration Agreement with Tenacia | Supply of License Product
License and Collaboration Revenue
Transaction Price	7,002	7,002
Contract Liability	$ 7,002	$ 7,002

Subsequent Events (Details)

Subsequent Events (Details) - Subsequent Event - item	Apr. 30, 2024	Apr. 15, 2024
Subsequent Events
Number of patients enrolled in Raise Trial		100
Reduction-in-Force
Subsequent Events
Percentage of workforce impacted by RIF	20%

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (38,669)	$ (34,730)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
	Mar. 31, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	Trading Plans ​ During the three months ended March 31, 2024, none of our directors or officers (as defined in Rule 16a-1(f) of the Exchange Act) adopted a “Rule 10b5-1 trading arrangement” (as those terms are defined under Item 408 of Regulation S-K), except as follows: ​ ​ ​ ​ ​ ​ ​ Name Title Reporting Action Plan Start Date Plan End Date Shares of Common Stock to be Sold Intended to Satisfy Rule 10b5-1(c)? Elan Ezickson Director Plan Adoption January 18, 2025 January 31, 2025 1,600 Yes Marvin H. Johnson, Jr. Director Plan Adoption June 10, 2024 June 10, 2025 6,999 Yes Sarah Noonberg, M.D., Ph.D. Director Plan Adoption January 18, 2025 January 31, 2025 To be determined as sale of common stock to cover tax obligation related to expected January 2025 RSU vesting Yes Christina Shafer Chief Commercial Officer Plan Adoption June 10, 2024 June 10, 2025 236,932 Yes
Elan Ezickson
Trading Arrangements, by Individual
Name	Elan Ezickson
Title	Director
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	January 18, 2025
Aggregate Available	1,600
Expiration Date	January 31, 2025
Marvin H. Johnson, Jr.
Trading Arrangements, by Individual
Name	Marvin H. Johnson, Jr.
Title	Director
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	June 10, 2024
Aggregate Available	6,999
Expiration Date	June 10, 2025
Sarah Noonberg, M.D., Ph.D.
Trading Arrangements, by Individual
Name	Sarah Noonberg, M.D., Ph.D.
Title	Director
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	January 18, 2025
Expiration Date	January 31, 2025
Christina Shafer
Trading Arrangements, by Individual
Name	Christina Shafer
Title	Chief Commercial Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	June 10, 2024
Aggregate Available	236,932
Expiration Date	June 10, 2025