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Stock and Warrants - Schedule of Authorized Shares of Common Stock Reserved for Future Issuance (Details) - shares	Sep. 30, 2025	Dec. 31, 2024
Total common shares reserved for future issuance (in shares)	19,143,514	19,804,372
Common stock warrants
Total common shares reserved for future issuance (in shares)	4,436,637	5,483,537
Awards under equity incentive plans
Total common shares reserved for future issuance (in shares)	7,228,555	7,789,658
Possible future issuances under equity incentive plans
Total common shares reserved for future issuance (in shares)	6,422,637	5,383,165
Possible future issuances under employee stock purchase plans
Total common shares reserved for future issuance (in shares)	1,055,685	1,148,012

Stock-Based Compensation

9 Months Ended

Sep. 30, 2025

Share-Based Payment Arrangement, Disclosure [Abstract]

Stock-Based Compensation

In November 2014, the Board of Directors of the Company (the “Board”), and in April 2015, the Company’s stockholders, approved the Company’s 2014 Equity Incentive Plan (the “2014 Plan”), which became effective in April 2015. The 2014 Plan provides for the grant of stock options, other forms of equity compensation, and performance cash awards. In June 2021, the Company's stockholders approved an Amended and Restated 2014 Equity Incentive Plan (the “A&R 2014 Plan”), following its adoption by the Board in April 2021, which, among other things, added 4,900,000 shares to the maximum number of shares of common stock to be issued under the plan and extended the annual automatic increases (discussed further below) until January 1, 2031 and eliminated individual grant limits that applied under the 2014 Plan to awards that were intended to comply with the exemption for “performance-based compensation” under Code Section 162(m). The maximum number of shares of common stock that may be issued under the A&R 2014 Plan was 12,079,711 as of September 30, 2025. The number of shares of common stock reserved for issuance under the A&R 2014 Plan automatically increases on January 1 of each year, beginning on January 1, 2016, and ending on and including January 1, 2031, by 4% of the total number of shares of the Company’s capital stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares determined by the Board. Pursuant to the terms of the A&R 2014 Plan, on January 1, 2025, the common stock reserved for issuance under the A&R 2014 Plan automatically increased by 2,146,828 shares.

During the three and nine months ended September 30, 2025, 86,628 and 1,162,133 stock options were exercised, respectively. During the three and nine months ended September 30, 2024, 27,750 and 761,500 stock options were exercised, respectively.

In June 2021, the Company's stockholders approved an Employee Stock Purchase Plan (the “ESPP”), following its adoption by the Board in April 2021. The maximum number of shares of common stock that may be issued under the ESPP is 1,500,000. The first offering period under the ESPP began on October 1, 2021, and the first purchase date occurred on May 31, 2022. As of September 30, 2025, 444,315 shares have been issued under the ESPP.

In January 2023, the Board approved the 2023 Employment Inducement Award Plan (as amended, the “2023 Plan”). The maximum number of shares of common stock that may be issued under the 2023 Plan was 4,500,000 as of September 30, 2025.

In February 2025, the Board approved the Tenth Amended and Restated Non-Employee Director Compensation Policy (the “Non-Employee Director Compensation Policy”). The equity compensation granted pursuant to the Non-Employee Director Compensation Policy is granted under the A&R 2014 Plan.

Stock-based compensation expense recorded under the A&R 2014 Plan, ESPP and 2023 Plan is included in the following line items in the accompanying unaudited condensed consolidated statements of operations (in thousands):


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024
Research and development	$	250	$	1,441	$	721	$	3,322
Selling, general and administrative	2,521		4,696		7,629		7,566
Total stock-based compensation expense	$	2,771	$	6,137	$	8,350	$	10,888

There was no stock-based compensation expense related to performance-based awards recognized during the three and nine months ended September 30, 2025. During the three and nine months ended September 30, 2024, the Company recognized approximately $2.5 million in stock-based compensation expense related to performance-based awards.

UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Current assets:
Cash and cash equivalents	$ 54,439	$ 33,785
Securities at fair value, current	145,070	35,711
Accounts and other receivables	16,841	10,509
Prepaid expenses and other current assets	7,398	4,052
Inventories, current	1,367	1,970
Total current assets	225,115	86,027
Securities at fair value, noncurrent	30,865	6,010
Inventories, noncurrent	720	10,999
Property and equipment, net	488	356
Operating lease right-of-use assets	1,337	657
Goodwill	4,701	4,701
Intangible assets, net	6,737	68,993
Other long-term assets	153	384
Total assets	270,116	178,127
Current liabilities:
Accounts payable and accrued expenses	11,072	25,456
Current portion of operating lease liabilities	441	420
Current portion of discount and rebate liabilities	5,609	4,989
Other current liabilities	8,990	3,200
Total current liabilities	26,112	34,065
Long-term debt	61,310	59,504
Warrant liability	13,767	17,804
Income tax payable	17,464	14,431
Operating lease liabilities, less current portion	966	372
Discount and rebate liabilities, less current portion	13,337	7,655
Other long-term liabilities	3,995	4,630
Total liabilities	136,951	138,461
Commitments and contingencies (Note M)
Stockholders’ equity:
Undesignated preferred stock	0	0
Common stock	6	5
Additional paid-in capital	580,783	555,302
Treasury stock, at cost	(10,983)	(10,983)
Accumulated deficit	(434,225)	(505,289)
Accumulated other comprehensive (loss) income	(2,416)	631
Total stockholders' equity	133,165	39,666
Total liabilities and stockholders' equity	$ 270,116	$ 178,127

Fair Value of Financial Instruments - Fair Value Measurement Inputs and Valuation Techniques (Details)	9 Months Ended	12 Months Ended
	Sep. 30, 2025 $ / shares	Dec. 31, 2024 $ / shares
Weighted average fair value (in dollars per share)	$ 3.10	$ 3.25
Risk-free interest rate \| Minimum
Warrants and rights outstanding, measurement input	0.0355	0.0408
Risk-free interest rate \| Maximum
Warrants and rights outstanding, measurement input	0.0392	0.0423
Volatility \| Minimum
Warrants and rights outstanding, measurement input	0.6061	0.6214
Volatility \| Maximum
Warrants and rights outstanding, measurement input	0.6388	0.6868
Dividend yield
Warrants and rights outstanding, measurement input	0	0
Expected term (years) \| Minimum
Warrants and rights outstanding, measurement input	0.27	1.02
Expected term (years) \| Maximum
Warrants and rights outstanding, measurement input	3.15	3.89

UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Sep. 30, 2025	Dec. 31, 2024
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred stock, shares authorized (in shares)	10,000,000	10,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Common stock, shares authorized (in shares)	250,000,000	250,000,000
Common stock, shares issued (in shares)	57,793,414	55,246,401
Common stock, shares outstanding (in shares)	56,217,722	53,670,709

Goodwill & Intangible Assets - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended		9 Months Ended
	Oct. 31, 2024	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024	Dec. 31, 2024
Goodwill		$ 4,701		$ 4,701		$ 4,701
Non-amortizable intangible assets		2,000		2,000		2,000
Impairment of intangible assets		0	$ 0	58,710	$ 0
Amortization expense		$ 315	1,545	$ 3,546	4,619
Patents
Useful life		13 years		13 years
Amortization expense		$ 0	1,500	$ 2,600	4,600
Patents \| XOMA License Agreement
Useful life		5 years		5 years
Amortization expense		$ 300	$ 0	$ 900	$ 0
Patents \| XOMA License Agreement \| Capitalization, Royalty Expense
Payments to acquire intangible assets	$ 6,000
OLPRUVA
Impairment of intangible assets		$ 0		$ 58,700
Carrying value of intangible asset						$ 61,300

Revenue, net (Tables)

9 Months Ended

Sep. 30, 2025

Revenue from Contract with Customer [Abstract]

Schedule of Accounts and Other Receivables

Accounts and other receivables consist of the following (in thousands):


	September 30, 2025		December 31, 2024
Commercial accounts receivable	$	8,770	$	4,010
Receivables related to product reimbursements	6,056		5,380
Royalties accounts receivable	1,200		786
Other receivables	815		333
Total receivables	$	16,841	$	10,509

Goodwill & Intangible Assets - Schedule of Estimated Amortization Expense For Intangible Assets (Details) $ in Thousands	Sep. 30, 2025 USD ($)
Goodwill and Intangible Assets Disclosure [Abstract]
2025 (excluding the nine months ended September 30, 2025)	$ 316
2026	1,263
2027	1,263
2028	1,263
2029	$ 632

Summary of Significant Accounting Policies (Details) - USD ($)				3 Months Ended		9 Months Ended		12 Months Ended
	Apr. 01, 2025	Feb. 27, 2025	Aug. 30, 2023	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024	Dec. 31, 2024
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Fair value				$ 175,900,000		$ 175,900,000		$ 41,700,000
Unrealized gain on investments						119,000		$ 18,000
Debt securities mature within one year				145,100,000		145,100,000
Debt securities mature within year one through five				30,900,000		30,900,000
Estimated proceeds from sale of priority review voucher		$ 150,000,000.0
Proceeds from sale of PRV	$ 148,300,000					150,000,000	$ 0
Inventory write-down				$ 0	$ 2,000,000.0	$ 11,681,000	$ 5,244,000
Aquestive Therapeutics \| License \| AZSTARYS License Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Royalty percentage						10.00%
Acer and Relief Therapeutics SA \| License \| Relief License Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Royalty percentage			10.00%
Contractual obligation			$ 45,000,000.0
Acer and Relief Therapeutics SA \| License \| Relief License Agreement \| Maximum
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Contractual obligation			$ 56,500,000

Leases- Schedule of Component of Lease Expenses (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Leases [Abstract]
Amortization of right-of-use assets	$ 1	$ 12	$ 4	$ 36
Total finance lease cost	1	12	4	36
Operating lease cost	205	119	612	330
Short-term lease cost	32	59	127	177
Variable lease cost	10	26	36	39
Less: sublease income	(27)	(50)	(105)	(128)
Total lease costs	$ 221	$ 166	$ 674	$ 454

Revenue, net - Narrative (Details) - USD ($)	3 Months Ended		9 Months Ended		12 Months Ended
Revenue, net - Narrative (Details) - USD ($)	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024	Dec. 31, 2024	Sep. 03, 2019
Disaggregation of Revenue [Line Items]
Revenue, net	$ 26,063,000	$ 3,695,000	$ 72,345,000	$ 11,569,000
Revenue percentage of discounted allowances for products	4.00%	0.00%	1.00%	0.00%
Contract with customer, liability	$ 0		$ 0		$ 0
Reserve or allowance for doubtful accounts	0		0		0
Arimoclomol Purchase Agreement
Disaggregation of Revenue [Line Items]
Recognized revenue	2,400,000	$ 2,600,000	7,300,000	$ 8,000,000.0
Clawback liability	1,500,000	1,400,000	4,500,000	4,400,000
Estimated reserve liability			18,900,000		12,600,000
OLPRUVA
Disaggregation of Revenue [Line Items]
Revenue, net	100,000	0	500,000	0
MIPLYFFA
Disaggregation of Revenue [Line Items]
Revenue, net	22,400,000		61,000,000.0
License
Disaggregation of Revenue [Line Items]
Revenue, net			0
License \| AZSTARYS License Agreement
Disaggregation of Revenue [Line Items]
Recognized revenue		$ 1,100,000		$ 3,600,000
Contract with customer, liability, revenue recognized, including opening balance	1,200,000		3,300,000
Contract with customer, liability	0		0		$ 0
License \| Commave Therapeutics SA \| AZSTARYS License Agreement
Disaggregation of Revenue [Line Items]
Milestone payments						$ 590,000,000.0
License \| Relief Therapeutics
Disaggregation of Revenue [Line Items]
Recognized revenue	$ 0		$ 0
Royalty percentage			10.00%

Summary of Significant Accounting Policies	9 Months Ended
Summary of Significant Accounting Policies	Sep. 30, 2025
Accounting Policies [Abstract]
Summary of Significant Accounting Policies	Summary of Significant Accounting Policies Use of Estimates The preparation of these unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires the Company to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. On an ongoing basis, the Company evaluates its estimates and assumptions, including those related to revenue recognition, the useful lives of property and equipment, the recoverability of long-lived assets, the incremental borrowing rate for leases, and assumptions used for purposes of determining stock-based compensation, income taxes, the fair value of the warrant liability and discount and rebate liabilities, among others. The Company bases its estimates on historical experience and on various other assumptions that it believes to be reasonable, the results of which form the basis for making judgments about the carrying value of assets and liabilities. Investments The Company maintains investment securities that are classified as available-for-sale securities for which the Company has elected the fair value option under Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 825, Financial Instruments. As such, these securities are carried at fair value with unrealized gains and losses included in fair value adjustment related to investments on the unaudited condensed consolidated statements of operations. The securities primarily consist of U.S. Treasury securities and corporate bonds and are included in securities at fair value in the unaudited condensed consolidated balance sheets. As of September 30, 2025, and December 31, 2024, the Company held securities with an aggregate fair value of $175.9 million and $41.7 million, respectively, that contained an aggregate unrealized gain of $119,000 and $18,000, respectively. For securities held at September 30, 2025, approximately $145.1 million mature within one year and approximately $30.9 million mature in one to three years. Applying fair value accounting to these debt securities more accurately represents the Company's investment strategy due to the fact that excess cash is currently being invested for the purpose of funding future operations. Interest income is recognized as earned using an effective yield method giving effect to the amortization of premium and accretion of discount and is based on the economic life of the securities. Interest income is included in interest and other income, net, in the unaudited condensed consolidated statements of operations. Variable Interest Entities The primary beneficiary of a variable interest entity (“VIE”) is required to consolidate the assets and liabilities of the VIE. When the Company obtains a variable interest in another entity, it assesses at the inception of the relationship and upon occurrence of certain significant events whether the entity is a VIE and, if so, whether the Company is the primary beneficiary of the VIE based on its power to direct the activities of the VIE that most significantly impact the VIE's economic performance and the Company's obligation to absorb losses or the rights to receive benefits from the VIE that could potentially be significant to the VIE. To assess whether the Company has the power to direct the activities of the VIE that most significantly impact the VIE's economic performance, the Company considers all the facts and circumstances, including the Company's role in establishing the VIE and the Company's ongoing rights and responsibilities. The assessment includes identifying the activities that most significantly impact the VIE's economic performance and identifying which party, if any, has the power to direct those activities. In general, the parties that make the most significant decisions affecting the VIE (management and members of the Board of Directors) are deemed to have the power to direct the activities of a VIE. To assess whether the Company has the obligation to absorb losses of the VIE or the rights to receive benefits from the VIE that could potentially be significant to the VIE, the Company considers all of its economic interests that are deemed to be variable interests in the VIE. This assessment requires judgment in determining whether these interests, in the aggregate, are considered potentially significant to the VIE. On August 30, 2023, the Company entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Acer Therapeutics Inc. (“Acer”). On November 17, 2023 (the “Closing Date”), Zevra completed the acquisition of Acer (the “Merger”). Pursuant to the Merger Agreement, on the Closing Date, Acer continued as the surviving entity and as a wholly-owned subsidiary of Zevra. As of September 30, 2025, and December 31, 2024, the Company identified Acer to be the Company's sole interest in a VIE. As Zevra is the final decision maker for all of Acer's research, development, and commercialization of drug candidates that it is producing, the Company directs the activities of Acer that most significantly impact its performance. Therefore, the Company is the primary beneficiary of this VIE for accounting purposes and consolidates the assets and liabilities of the VIE. Gain on Sale of PRV The Company received a transferable rare priority review voucher (“PRV”) in conjunction with the FDA approval of MIPLYFFA. On February 27, 2025, the Company and its subsidiary, Zevra Denmark A/S, entered into an asset purchase agreement with a buyer, pursuant to which the Company agreed to sell the PRV to the buyer for aggregate proceeds of $150.0 million, payable in cash, upon the closing of the sale. On April 1, 2025, the asset sale was consummated and title of the PRV transferred to the buyer, resulting in net proceeds of $148.3 million to the Company. The PRV did not have a carrying value at the time of sale. In accordance with ASC 610-20, Gains and Losses from the Derecognition of Nonfinancial Assets, the net proceeds from the sale were recorded as a gain on sale of PRV in the Company's unaudited condensed consolidated statements of operations for the nine months ended September 30, 2025. Revenue Recognition The Company recognizes revenue in accordance with the provisions of ASC 606, Revenue from Contracts with Customers (“ASC 606”) and, as a result, follows the five-step model when recognizing revenue: 1) identifying a contract; 2) identifying the performance obligations; 3) determining the transaction price; 4) allocating the price to performance obligations; and 5) recognizing revenue when the performance obligations have been fulfilled. Product Revenues, Net Net revenues from product sales are recognized at the transaction price when the customer obtains control of the Company's product, which occurs at a point in time, typically upon receipt of the product by the customer. The Company's current single customer is a specialty pharmacy provider. In accordance with ASC 606, the Company recognizes revenue when fulfilling its performance obligation by transferring control of promised goods or services to its customer, in an amount that reflects the consideration that the Company expects to receive in exchange for those goods or services. In determining when the customer obtains control of the product, the Company considers certain indicators, including whether the Company has a present right to payment from the customer, whether title and/or significant risks and rewards of ownership have transferred to the customer and whether customer acceptance has been received. The Company's net revenues represent total revenues adjusted for discounts and allowances, including estimated cash discounts, chargebacks, rebates, returns, copay assistance, data fees and wholesaler fees for services. These adjustments represent variable consideration under ASC 606 and are recorded as a reduction of revenue. These adjustments are established by management as its best estimate based on available information and will be adjusted to reflect known changes in the factors that impact such allowances. Adjustments for variable consideration are determined based on the contractual terms with customers, historical trends, communications with customers and the levels of inventory remaining in the distribution channel, as well as expectations about the market for the product and anticipated introduction of competitive products. Arimoclomol French AC Net revenue includes revenue from the sale of arimoclomol for the treatment of NPC under the remunerated expanded access program in France, Accès Compassionnel (“French AC”). An expanded access program is a program giving specific patients access to a drug that is not yet approved for commercial sale. Only drugs targeting serious or rare indications and for which there is currently no appropriate treatment are considered for expanded access programs. Further, to be considered for the expanded access program, the drug must have proven efficacy and safety and must either be undergoing price negotiations or seeking marketing approval. In accordance with ASC 606, the Company recognizes revenue when fulfilling its performance obligation under the French AC by transferring control of promised goods or services to its customer, in an amount that reflects the consideration that the Company expects to receive in exchange for those goods or services. In determining when the customer obtains control of the product, the Company considers certain indicators, including whether the Company has a present right to payment from the customer, whether title and/or significant risks and rewards of ownership have transferred to the customer and whether customer acceptance has been received. Revenue is recognized net of sales deductions, including discounts, rebates, applicable distributor fees, and revenue-based taxes. The French Health Authorities and the manufacturer have agreed to a price for sales under the French AC, but the final transaction price depends on the terms and conditions in the contracts with the French Health Authorities, following market approval. Any excess in the price charged by the manufacturer compared to the price agreed with the health authorities once the drug product is approved in France must be repaid. The repayment is considered in the clawback liability (rebate). An estimate of net revenue and clawback liability are recognized using the ‘expected value’ method. Accounting for net revenue and clawback liability requires determination of the most appropriate method for the expected final transaction price. This estimate also requires assumptions with respect to inputs into the method, including current pricing of comparable marketed products within the rare disease area in France. Management has considered the expected final sales price as well as the price of similar drug products. The Company is operating within a rare disease therapeutic area where there is unmet treatment need and hence a limited number of comparable commercialized drug products. The limited available relevant market information for directly comparable commercialized drugs within rare disease increases the uncertainty in management's estimate. Licensing Agreements The terms of the Company’s licensing agreements typically include one or more of the following: (i) upfront fees; (ii) milestone payments related to the achievement of development, regulatory, or commercial goals; and (iii) royalties on net sales of licensed products. Each of these payments may result in licensing revenues. As part of the accounting for these agreements, the Company 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. Generally, the estimation of the stand-alone selling price may include such estimates as independent evidence of market price, forecasted revenues or costs, development timelines, discount rates, and probability of regulatory success. The Company evaluates each performance obligation to determine if it can be satisfied at a point in time or over time, and it measures the services delivered to the licensee, which are periodically reviewed based on the progress of the related program. The effect of any change made to an estimated input component and, therefore, revenue or expense recognized, would be recorded as a change in estimate. In addition, variable consideration (e.g., milestone payments) must be evaluated to determine if it is constrained and, therefore, excluded from the transaction price. Upfront Fees: If a license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from the transaction price allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time. Milestone Payments: At the inception of each arrangement that includes milestone payments (variable consideration), the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the Company’s or the licensee’s control, such as non-operational developmental and regulatory approvals, are generally not considered probable of being achieved until those approvals are received. At the end of each reporting period, the Company re-evaluates the probability of achievement of milestones that are within its or the licensee’s control, such as operational developmental milestones and any related constraint, and, if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenues and earnings in the period of adjustment. Revisions to the Company’s estimate of the transaction price may also result in negative licensing revenues and earnings in the period of adjustment. Inventories The value of inventories is recorded at net realizable value. The Company determines the cost of its inventories, which include amounts related to materials and manufacturing overhead, on a first-in, first-out basis. Inventories that are not expected to be sold within 12 months are classified as inventories, noncurrent. The Company may scale-up and make commercial quantities of its product candidates prior to the date it anticipates that such product will receive final regulatory approval. The scale-up and commercial production of pre-launch inventory involves the risk that such products may not be approved for marketing on a timely basis, or ever. This risk notwithstanding, the Company may scale-up and build pre-launch inventory of products that have not received final regulatory approval when the Company believes such action is appropriate in relation to the commercial value of the product launch opportunity. We capitalize inventory costs associated with our products prior to regulatory approval when, based on management's judgment, future commercialization is considered probable and the future economic benefit is expected to be realized; otherwise, such costs are expensed as research and development. The determination to capitalize inventory costs is based on various factors, including status and expectations of the regulatory approval process, any known safety or efficacy concerns, potential labeling restrictions, and any other impediments to obtaining regulatory approval. The Company had no pre-approval inventory on its unaudited condensed consolidated balance sheets as of September 30, 2025, or December 31, 2024. Inventory used in clinical trials is also expensed as research and development expense when selected for such use. Inventory that can be used in the production of either clinical or commercial products is expensed as research and development costs when identified for use in a clinical manufacturing campaign. The cost of finished goods inventory that is shipped to a customer to support the Company’s patient assistance programs is expensed when those shipments take place. As of September 30, 2025, and December 31, 2024, the Company did not have pre-launch inventory that qualified for capitalization. The Company performs an assessment of the recoverability of capitalized inventory during each reporting period and writes down any excess and obsolete inventory to its net realizable value in the period in which the impairment is first identified. Such impairment charges, should they occur, are recorded as a component of cost of product revenue in the unaudited condensed consolidated statements of operations. The determination of whether inventory costs will be realizable requires the use of estimates by management. If actual market conditions are less favorable than projected by management, additional write downs of inventory may be required. Additionally, the Company’s products are subject to strict quality control and monitoring, which is performed throughout the manufacturing process. In the event that certain batches or units of product do not meet quality specifications, the Company will record a charge to cost of product revenue, to write down any unsaleable inventory to its estimated net realizable value. For the three and nine months ended September 30, 2025, the Company recognized charges of approximately $0 and $11.7 million, respectively, related to write-downs for unsaleable inventory. For the three and nine months ended September 30, 2024, the Company recognized charges of approximately $2.0 million and $5.2 million, respectively, related to write-downs for unsaleable inventory. Cost of Product Revenue The components of cost of product revenue are royalties and expenses directly attributable to revenue. To date, the Company has generated revenue from product sales of MIPLYFFA and OLPRUVA, reimbursements received under the French AC, royalties or net sales milestone payments generated under the Collaboration and License Agreement with Commave Therapeutics SA (the “AZSTARYS® License Agreement”), and consulting agreements. Prior to our acquisition of the assets of Orphazyme A/S (“Orphazyme”) in May 2022, Orphazyme had entered into an asset purchase agreement with LadRx Corporation, which was assigned to XOMA (US) LLC, a wholly-owned subsidiary of XOMA Corporation (“XOMA”), in June 2023 (“XOMA License Agreement”). Under the XOMA License Agreement, XOMA is entitled to a mid-single digit percentage royalty with respect to net sales of MIPLYFFA as well as milestone payments based on future potential sales and regulatory milestones. As a condition to entering into the Merger Agreement, Acer and Relief Therapeutics SA (“Relief”) entered into an exclusive license agreement on August 30, 2023 (the “Relief License Agreement”). Pursuant to the Relief License Agreement, Zevra was obligated to pay royalties of 10% of U.S. net sales of OLPRUVA up to a maximum of $45.0 million, plus specified regulatory milestones, for total payments to Relief of up to $56.5 million. On April 10, 2025, the rights to this royalty were sold to Soleus Capital Management L.P. In connection with the AZSTARYS License Agreement, the Company pays Aquestive Therapeutics, Inc. (“Aquestive”) a royalty equal to 10% of all regulatory milestone and royalty payments. Income Taxes The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the financial reporting and tax basis of assets and liabilities, as well as for operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using the tax rates that are expected to apply to taxable income for the years in which those tax assets and liabilities are expected to be realized or settled. Valuation allowances are recorded to reduce deferred tax assets to the amount the Company believes is more likely than not to be realized. Uncertain tax positions are recognized only when the Company believes it is more likely than not that the tax position will be upheld on examination by the taxing authorities based on the merits of the position. The Company recognizes interest and penalties, if any, related to unrecognized income tax uncertainties in income tax expense. The Company files income tax returns in the United States for federal and various state jurisdictions. With few exceptions, the Company is no longer subject to U.S. federal and state and local income tax examinations for years prior to 2015, although carryforward attributes that were generated prior to 2017 may still be adjusted upon examination by the Internal Revenue Service if used in a future period. No income tax returns are currently under examination by taxing authorities. On December 22, 2017, the U.S. government enacted H.R. 1, “An Act to provide for reconciliation pursuant to titles II and V of the concurrent resolution on the budget for fiscal year 2018” (“Tax Act”). Effective January 1, 2018, the Tax Act provides for a new global intangible low-taxed income (“GILTI”) provision. Under the GILTI provision, certain foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets are included in U.S. taxable income. The Company has not recorded any deferred taxes for future GILTI inclusions as any future inclusions are expected to be treated as a period expense and offset by net operating loss carryforwards in the U.S. On July 4, 2025, the One Big Beautiful Bill Act (the “OBBB”), which includes a broad range of tax reform provisions, was signed into law in the United States. The Company continues to assess the impact of the OBBB but currently does not expect the OBBB to have a material impact on the Company's estimated annual effective tax rate in 2025. Segment and Geographic Information Operating segments are defined as components of an enterprise (business activity from which it earns revenue and incurs expenses) for which discrete financial information is available and regularly reviewed by the chief operating decision maker (“CODM”) in deciding how to allocate resources and in assessing performance. The Company’s CODM is its Chief Executive Officer. The Company views its operations and manages its business as a single operating and reporting segment. Foreign Currency Assets and liabilities are translated into the reporting currency using the exchange rates in effect on the balance sheet dates. Equity accounts are translated at historical rates, except for the change in retained earnings during the year, which is the result of the income statement translation process. Revenue and expense accounts are translated using the weighted average exchange rate during the period. The cumulative translation adjustments associated with the net assets of foreign subsidiaries are recorded in accumulated other comprehensive income (loss) in the accompanying unaudited condensed consolidated statements of changes in stockholders’ equity. Debt Issuance Costs Debt issuance costs incurred in connection with financing arrangements are recorded as a reduction of the related debt on the unaudited condensed consolidated balance sheets and amortized over the life of the respective financing arrangement using the effective interest method. Warrants The Company accounts for warrants as either equity-classified or liability-classified instruments based on an assessment of the warrant’s specific terms and applicable authoritative guidance in FASB ASC Topic 480, Distinguishing Liabilities from Equity (“ASC 480”) and FASB ASC Topic 815, Derivatives and Hedging (“ASC 815”). The assessment considers whether the warrants are freestanding financial instruments pursuant to ASC 480, meet the definition of a liability pursuant to ASC 480, and whether the warrants meet all of the requirements for equity classification under ASC 815, including whether the warrants are indexed to the Company's own stock and whether the warrant holders could potentially require “net cash settlement” in a circumstance outside of the issuing company’s control, among other conditions for equity classification. This assessment, which requires the use of professional judgment, is conducted at the time of warrant issuance and as of each subsequent quarterly period end date while the warrants are outstanding. For warrants that meet all criteria for equity classification, the warrants are required to be recorded as a component of additional paid-in capital on the unaudited condensed consolidated statements of changes in stockholders’ equity at the time of issuance. For warrants that do not meet all the criteria for equity classification, the warrants are required to be recorded at their initial fair value on the date of issuance, and on each balance sheet date thereafter. Changes in the estimated fair value of the warrants are recognized as a non-cash gain or loss in other expense, net, on the unaudited condensed consolidated statements of operations. The fair value of the warrants was estimated using the Black-Scholes-Merton (“BSM”) option pricing model. New Accounting Pronouncements Not Yet Adopted Income Taxes (Topic 740): Improvements to Income Tax Disclosures In December 2023, the FASB issued Accounting Standards Update (“ASU”) No. 2023-09, Income Taxes (“Topic 740”): Improvements to Income Tax Disclosures, which expands disclosures in an entity’s income tax rate reconciliation table and disclosures regarding cash taxes paid both in the U.S. and in foreign jurisdictions. The update will be effective for annual periods beginning after December 15, 2024. The Company is currently evaluating the impact that this guidance will have on the presentation of its financial statements and accompanying notes.

Leases (Tables)

9 Months Ended

Sep. 30, 2025

Leases [Abstract]

Schedule of Component of Lease Expenses

The components of lease expense were as follows (in thousands):


	Three months ended September 30,				Nine months ended September 30,
Lease Cost	2025		2024		2025		2024
Finance lease cost:
Amortization of right-of-use assets	$	1	$	12	$	4	$	36
Total finance lease cost	1		12		4		36
Operating lease cost	205		119		612		330
Short-term lease cost	32		59		127		177
Variable lease cost	10		26		36		39
Less: sublease income	(27)		(50)		(105)		(128)
Total lease costs	$	221	$	166	$	674	$	454

Schedule of Supplemental Cash Flow Information Related To Leases

Supplemental cash flow information related to leases was as follows (in thousands):


	Nine months ended September 30,
	2025		2024
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	688	$	405
Operating cash flows from short-term leases	127		177
Operating cash flows from variable lease costs	36		39

Right-of-use assets obtained in exchange for lease liabilities:
Operating leases	$	1,115	$	419

Schedule Of Supplemental Balance Sheet Information Related To Leases

Supplemental balance sheet information related to leases was as follows (in thousands, except weighted average remaining lease term and weighted average discount rate):


	September 30, 2025			December 31, 2024
Finance Leases
Property and equipment, at cost	$	1,031		$	1,031
Less: accumulated depreciation and amortization	(1,026)			(1,023)
Property and equipment, net	$	5		$	8

Operating Leases
Operating lease right-of-use assets	$	1,337		$	657


Current portion of operating lease liabilities	$	441		$	420
Operating lease liabilities, less current portion	966			372
Total operating lease liabilities	$	1,407		$	792

Weighted Average Remaining Lease Term
Operating leases (in years)	3			3

Weighted Average Discount Rate
Operating leases	12.9		%	9.9		%

Schedule of Maturities of Operating Lease Liabilities

Maturities of lease liabilities were as follows (in thousands):


Year Ended December 31,	Operating Leases
2025 (excluding the nine months ended September 30, 2025)	$	172
2026	552
2027	542
2028	393
2029	39

Total lease payments	1,698
Less: future interest expense	(291)
Lease liabilities	$	1,407

Leases

9 Months Ended

Sep. 30, 2025

Leases [Abstract]

Leases

K. Leases

The Company has operating and finance leases for office space, laboratory facilities and various laboratory equipment, furniture and office equipment and leasehold improvements. The Company determines if an arrangement is a lease at contract inception. Lease assets and lease liabilities are recognized based on the present value of lease payments over the lease term at the commencement date. The Company does not separate lease and non-lease components. Leases with a term of 12 months or less at commencement are not recorded on the unaudited condensed consolidated balance sheets. Lease expense for these arrangements is recognized on a straight-line basis over the lease term. The Company’s leases have remaining lease terms of approximately 1 year and up to approximately 3 years, and some which include options to terminate the leases within 1 year.

The components of lease expense were as follows (in thousands):


	Three months ended September 30,				Nine months ended September 30,
Lease Cost	2025		2024		2025		2024
Finance lease cost:
Amortization of right-of-use assets	$	1	$	12	$	4	$	36
Total finance lease cost	1		12		4		36
Operating lease cost	205		119		612		330
Short-term lease cost	32		59		127		177
Variable lease cost	10		26		36		39
Less: sublease income	(27)		(50)		(105)		(128)
Total lease costs	$	221	$	166	$	674	$	454

Supplemental cash flow information related to leases was as follows (in thousands):


	Nine months ended September 30,
	2025		2024
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	688	$	405
Operating cash flows from short-term leases	127		177
Operating cash flows from variable lease costs	36		39

Right-of-use assets obtained in exchange for lease liabilities:
Operating leases	$	1,115	$	419

Supplemental balance sheet information related to leases was as follows (in thousands, except weighted average remaining lease term and weighted average discount rate):


	September 30, 2025			December 31, 2024
Finance Leases
Property and equipment, at cost	$	1,031		$	1,031
Less: accumulated depreciation and amortization	(1,026)			(1,023)
Property and equipment, net	$	5		$	8

Operating Leases
Operating lease right-of-use assets	$	1,337		$	657


Current portion of operating lease liabilities	$	441		$	420
Operating lease liabilities, less current portion	966			372
Total operating lease liabilities	$	1,407		$	792

Weighted Average Remaining Lease Term
Operating leases (in years)	3			3

Weighted Average Discount Rate
Operating leases	12.9		%	9.9		%

Maturities of lease liabilities were as follows (in thousands):


Year Ended December 31,	Operating Leases
2025 (excluding the nine months ended September 30, 2025)	$	172
2026	552
2027	542
2028	393
2029	39

Total lease payments	1,698
Less: future interest expense	(291)
Lease liabilities	$	1,407

Leases

K. Leases

The components of lease expense were as follows (in thousands):


	Three months ended September 30,				Nine months ended September 30,
Lease Cost	2025		2024		2025		2024
Finance lease cost:
Amortization of right-of-use assets	$	1	$	12	$	4	$	36
Total finance lease cost	1		12		4		36
Operating lease cost	205		119		612		330
Short-term lease cost	32		59		127		177
Variable lease cost	10		26		36		39
Less: sublease income	(27)		(50)		(105)		(128)
Total lease costs	$	221	$	166	$	674	$	454

Supplemental cash flow information related to leases was as follows (in thousands):


	Nine months ended September 30,
	2025		2024
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	688	$	405
Operating cash flows from short-term leases	127		177
Operating cash flows from variable lease costs	36		39

Right-of-use assets obtained in exchange for lease liabilities:
Operating leases	$	1,115	$	419

Supplemental balance sheet information related to leases was as follows (in thousands, except weighted average remaining lease term and weighted average discount rate):


	September 30, 2025			December 31, 2024
Finance Leases
Property and equipment, at cost	$	1,031		$	1,031
Less: accumulated depreciation and amortization	(1,026)			(1,023)
Property and equipment, net	$	5		$	8

Operating Leases
Operating lease right-of-use assets	$	1,337		$	657


Current portion of operating lease liabilities	$	441		$	420
Operating lease liabilities, less current portion	966			372
Total operating lease liabilities	$	1,407		$	792

Weighted Average Remaining Lease Term
Operating leases (in years)	3			3

Weighted Average Discount Rate
Operating leases	12.9		%	9.9		%

Maturities of lease liabilities were as follows (in thousands):


Year Ended December 31,	Operating Leases
2025 (excluding the nine months ended September 30, 2025)	$	172
2026	552
2027	542
2028	393
2029	39

Total lease payments	1,698
Less: future interest expense	(291)
Lease liabilities	$	1,407

Fair Value of Financial Instruments

9 Months Ended

Sep. 30, 2025

Fair Value Disclosures [Abstract]

Fair Value of Financial Instruments

The accounting standard for fair value measurements provides a framework for measuring fair value and requires disclosures regarding fair value measurements. Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date, based on the Company’s principal or, in absence of a principal, most advantageous market for the specific asset or liability.

The Company uses a three-tier fair value hierarchy to classify and disclose all assets and liabilities measured at fair value on a recurring basis, as well as assets and liabilities measured at fair value on a non-recurring basis, in periods subsequent to their initial measurement. The hierarchy requires the Company to use observable inputs, when available, and to minimize the use of unobservable inputs when determining fair value. The three tiers are defined as follows:

•Level 1: Observable inputs that reflect quoted market prices (unadjusted) for identical assets or liabilities in active markets;

•Level 2: Observable inputs other than quoted prices in active markets that are observable either directly or indirectly in the marketplace for identical or similar assets and liabilities; and

•Level 3: Unobservable inputs that are supported by little or no market data, which require the Company to develop its own assumptions.

The carrying amounts of certain financial instruments, including cash and cash equivalents, accounts and other receivables, and accounts payable and accrued expenses approximate their respective fair values due to the short-term nature of such instruments.

Assets and Liabilities Measured at Fair Value on a Recurring Basis

The Company evaluates its financial assets and liabilities subject to fair value measurements on a recurring basis to determine the appropriate level in which to classify them for each reporting period. This determination requires significant judgments to be made. The following table summarizes the conclusions reached regarding fair value measurements as of September 30, 2025, and December 31, 2024 (in thousands):


	Balance as of September 30, 2025		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
CVR liability	$	1,750	$	—	$	—	$	1,750
Warrant liabilities	13,767		—		—		13,767
Total liabilities	$	15,517	$	—	$	—	$	15,517

Securities:
U.S. Treasury securities	$	107,083	$	107,083	$	—	$	—
Corporate bonds	68,852		—		68,852		—
Total assets	$	175,935	$	107,083	$	68,852	$	—


	Balance as of December 31, 2024		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
CVR liability	$	3,500	$	—	$	—	$	3,500
Warrant liabilities	17,804		—		—		17,804
Total liabilities	$	21,304	$	—	$	—	$	21,304

Securities:
U.S. Treasury securities	$	35,711	$	35,711	$	—	$	—
Corporate bonds	6,010		—		6,010		—
Total assets	$	41,721	$	35,711	$	6,010	$	—

Warrants

The common stock warrant liabilities were recorded at fair value using the BSM option pricing model. The following assumptions were used in determining the fair value of the warrant liabilities valued using the BSM option pricing model as of September 30, 2025, and December 31, 2024:


	September 30, 2025			December 31, 2024
Risk-free interest rate	3.55% - 3.92%			4.08% - 4.23%
Volatility	60.61% - 63.88%			62.14% - 68.68%
Dividend yield	—		%	—		%
Expected term (years)	0.27 - 3.15			1.02 - 3.89
Weighted average fair value	$	3.10		$	3.25

The following table is a reconciliation for the common stock warrant liabilities measured at fair value using Level 3 unobservable inputs (in thousands):


Balance as of December 31, 2024	$	17,804
Change in fair value measurement of warrant liabilities	3,129
Warrants exercised	(7,166)
Balance as of September 30, 2025	$	13,767

For the nine months ended September 30, 2025, the changes in fair value of the warrant liabilities primarily resulted from changes in the discount rate.

Contingent Consideration

Contingent consideration liabilities relate to the Company's liabilities arising in connection with the CVRs issued as a result of the Merger. The contingent consideration is classified as Level 3 in the fair value hierarchy. The fair value is measured based on a Monte Carlo simulation or a scenario-based method, depending on the earn-out achievement objectives, utilizing projections about future performance. Significant inputs include volatility and projected financial information, including projections representative of a market participant's view of the expected cash payments associated with the agreed upon regulatory milestones based on probabilities of technical success, timing of the potential milestone events for the compounds, and estimated discount rates.

The following table provides a reconciliation of the beginning and ending balances related to the contingent consideration liabilities for the CVRs (dollars in thousands):


Balance as of December 31, 2024	$	3,500
Change in fair value measurement of contingent consideration liabilities	(1,750)
Balance as of September 30, 2025	$	1,750

For the nine months ended September 30, 2025, the changes in fair value of contingent consideration primarily resulted from changes in market data and revenue projections.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 107
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-107

Reference 3: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 100
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(3)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2E
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2E

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (h)
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-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 940
-SubTopic 820
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478119/940-820-50-1

+ Details

Net Income (Loss) Per Share - Schedule of Earnings Per Share, Basic and Diluted (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2025	Jun. 30, 2025	Mar. 31, 2025	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2025	Sep. 30, 2024
Basic net (loss) income per share of common stock:
Net (loss) income	$ (544)	$ 74,707	$ (3,099)	$ (33,225)	$ (19,925)	$ (16,622)	$ 71,064	$ (69,772)
Earnings allocated to participating securities	0						(5,309)
Net (loss) income attributable to shares of common stock	(544)						65,755
Less: Dividends declared or accumulated	0						0
Undistributed net (loss) income attributable to shares of common stock, basic	$ (544)						$ 65,755
Weighted-average number of shares of common stock outstanding - basic (in shares)	55,951,572			47,808,817			54,949,483	43,843,851
Basic net income per share of common stock (in dollars per share)	$ (0.01)			$ (0.69)			$ 1.20	$ (1.59)
Diluted net (loss) income per share of common stock:
Net (loss) income attributable to shares of common stock, diluted	$ (544)						$ 65,755
Less: Fair value adjustment income related to warrant liability	0						0
Net (loss) income attributable to shares of common stock, diluted	$ (544)						$ 65,755
Weighted-average number of shares of common stock outstanding - basic (in shares)	55,951,572			47,808,817			54,949,483	43,843,851
Dilutive effect of outstanding stock options (as converted to common stock) (in shares)	0						1,834,000
Weighted-average shares of common stock outstanding, diluted (in shares)	55,951,572			47,808,817			56,782,763	43,843,851
Diluted net income per share of common stock (in dollars per share)	$ (0.01)			$ (0.69)			$ 1.16	$ (1.59)

Fair Value of Financial Instruments - Fair Value, Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - Fair Value, Recurring - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	$ 15,517	$ 21,304
Total assets	175,935	41,721
U.S. Treasury securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	107,083	35,711
Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	68,852	6,010
Quoted Prices in Active Markets for Identical Assets (Level 1)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	0	0
Total assets	107,083	35,711
Quoted Prices in Active Markets for Identical Assets (Level 1) \| U.S. Treasury securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	107,083	35,711
Quoted Prices in Active Markets for Identical Assets (Level 1) \| Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	0	0
Significant Other Observable Inputs (Level 2)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	0	0
Total assets	68,852	6,010
Significant Other Observable Inputs (Level 2) \| U.S. Treasury securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	0	0
Significant Other Observable Inputs (Level 2) \| Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	68,852	6,010
Significant Unobservable Inputs (Level 3)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	15,517	21,304
Total assets	0	0
Significant Unobservable Inputs (Level 3) \| U.S. Treasury securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	0	0
Significant Unobservable Inputs (Level 3) \| Corporate bonds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total assets	0	0
CVR liability \| Derivative Financial Instruments, Liabilities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	1,750	3,500
CVR liability \| Derivative Financial Instruments, Liabilities \| Quoted Prices in Active Markets for Identical Assets (Level 1)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	0	0
CVR liability \| Derivative Financial Instruments, Liabilities \| Significant Other Observable Inputs (Level 2)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	0	0
CVR liability \| Derivative Financial Instruments, Liabilities \| Significant Unobservable Inputs (Level 3)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	1,750	3,500
Warrant liabilities \| Derivative Financial Instruments, Liabilities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	13,767	17,804
Warrant liabilities \| Derivative Financial Instruments, Liabilities \| Quoted Prices in Active Markets for Identical Assets (Level 1)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	0	0
Warrant liabilities \| Derivative Financial Instruments, Liabilities \| Significant Other Observable Inputs (Level 2)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	0	0
Warrant liabilities \| Derivative Financial Instruments, Liabilities \| Significant Unobservable Inputs (Level 3)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total liabilities	$ 13,767	$ 17,804

Segment Information - Segment Reporting Information, by Segment (Details) - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2025	Jun. 30, 2025	Mar. 31, 2025	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2025	Sep. 30, 2024
Segment Reporting, Revenue Reconciling Item [Line Items]
Total revenues	$ 26,063			$ 3,695			$ 72,345	$ 11,569
Research and development identified to programs	3,432			10,945			10,123	33,743
Selling, general and administrative identified to programs	16,935			16,208			57,262	38,743
Impairment of intangible assets	0			0			58,710	0
Income tax (benefit) expense	433			0			(2,948)	0
Depreciation and amortization expense							3,692	4,741
Interest expense	2,051			2,312			6,029	5,157
Net (loss) income	(544)	$ 74,707	$ (3,099)	(33,225)	$ (19,925)	$ (16,622)	71,064	(69,772)
Reportable Segment
Segment Reporting, Revenue Reconciling Item [Line Items]
Total revenues	26,063			3,695			72,345	11,569
Impairment of intangible assets	0			0			58,710	0
Income tax (benefit) expense	(433)			0			2,948	0
Interest income	(2,333)			(899)			(5,308)	(2,366)
Depreciation and amortization expense	392			1,617			3,692	4,741
Interest expense	2,051			2,312			6,029	5,157
Other expense (income), net	6,563			6,737			(132,175)	1,323
Net (loss) income	(544)			(33,225)			71,064	(69,772)
Direct Operating Costs \| Reportable Segment
Segment Reporting, Revenue Reconciling Item [Line Items]
Research and development identified to programs	1,780			4,916			6,480	18,369
Selling, general and administrative identified to programs	5,144			4,864			19,144	12,560
Indirect Operating Costs \| Reportable Segment
Segment Reporting, Revenue Reconciling Item [Line Items]
Research and development identified to programs	1,652			6,029			3,643	15,374
Selling, general and administrative identified to programs	$ 11,791			$ 11,344			$ 38,118	$ 26,183

Revenue, net - Schedule of Accounts and Other Receivables (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	$ 16,841	$ 10,509
Commercial accounts receivable
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	8,770	4,010
Receivables related to product reimbursements
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	6,056	5,380
Royalties accounts receivable
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	1,200	786
Other receivables
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	$ 815	$ 333

Segment Information - Narrative (Details) $ in Millions	9 Months Ended
Segment Information - Narrative (Details) $ in Millions	Sep. 30, 2025 USD ($) segment	Dec. 31, 2024 USD ($)
Segment Reporting, Revenue Reconciling Item [Line Items]
Number of operating segments \| segment	1
UNITED STATES
Segment Reporting, Revenue Reconciling Item [Line Items]
Segment long-lived assets	$ 2.2	$ 13.4
Europe
Segment Reporting, Revenue Reconciling Item [Line Items]
Segment long-lived assets	$ 0.4	$ 0.5

Fair Value of Financial Instruments (Tables)

9 Months Ended

Sep. 30, 2025

Fair Value Disclosures [Abstract]

Schedule of Fair Value, Assets and Liabilities Measured at Fair Value on a Recurring Basis

The following table summarizes the conclusions reached regarding fair value measurements as of September 30, 2025, and December 31, 2024 (in thousands):


	Balance as of September 30, 2025		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
CVR liability	$	1,750	$	—	$	—	$	1,750
Warrant liabilities	13,767		—		—		13,767
Total liabilities	$	15,517	$	—	$	—	$	15,517

Securities:
U.S. Treasury securities	$	107,083	$	107,083	$	—	$	—
Corporate bonds	68,852		—		68,852		—
Total assets	$	175,935	$	107,083	$	68,852	$	—


	Balance as of December 31, 2024		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)
CVR liability	$	3,500	$	—	$	—	$	3,500
Warrant liabilities	17,804		—		—		17,804
Total liabilities	$	21,304	$	—	$	—	$	21,304

Securities:
U.S. Treasury securities	$	35,711	$	35,711	$	—	$	—
Corporate bonds	6,010		—		6,010		—
Total assets	$	41,721	$	35,711	$	6,010	$	—

Schedule of Fair Value Measurement Inputs and Valuation Techniques

The following assumptions were used in determining the fair value of the warrant liabilities valued using the BSM option pricing model as of September 30, 2025, and December 31, 2024:


	September 30, 2025			December 31, 2024
Risk-free interest rate	3.55% - 3.92%			4.08% - 4.23%
Volatility	60.61% - 63.88%			62.14% - 68.68%
Dividend yield	—		%	—		%
Expected term (years)	0.27 - 3.15			1.02 - 3.89
Weighted average fair value	$	3.10		$	3.25

Schedule Of Common Stock Warrant Liabilities Measured At Fair Value

The following table is a reconciliation for the common stock warrant liabilities measured at fair value using Level 3 unobservable inputs (in thousands):


Balance as of December 31, 2024	$	17,804
Change in fair value measurement of warrant liabilities	3,129
Warrants exercised	(7,166)
Balance as of September 30, 2025	$	13,767

The following table provides a reconciliation of the beginning and ending balances related to the contingent consideration liabilities for the CVRs (dollars in thousands):


Balance as of December 31, 2024	$	3,500
Change in fair value measurement of contingent consideration liabilities	(1,750)
Balance as of September 30, 2025	$	1,750

Fair Value of Financial Instruments - Common Stock Warrant Liabilities Measured At Fair Value (Details) $ in Thousands	9 Months Ended
	Sep. 30, 2025 USD ($)
Warrant liabilities
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Beginning balance	$ 17,804
Change in fair value measurement of warrant liabilities	3,129
Warrants exercised	(7,166)
Ending balance	13,767
Contingent consideration liabilities
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Beginning balance	3,500
Change in fair value measurement of warrant liabilities	(1,750)
Ending balance	$ 1,750

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE (LOSS) INCOME - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Statement of Comprehensive Income [Abstract]
Net (loss) income	$ (544)	$ (33,225)	$ 71,064	$ (69,772)
Other comprehensive income (loss):
Foreign currency translation adjustment	12	(536)	(3,047)	(72)
Other comprehensive income (loss):	12	(536)	(3,047)	(72)
Comprehensive (loss) income	$ (532)	$ (33,761)	$ 68,017	$ (69,844)

- Definition

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income and other comprehensive income, attributable to parent entity. Excludes changes in equity resulting from investments by owners and distributions to owners.

+ References + Details

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

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+ Details

- Definition

Amount after tax, before reclassification adjustments of gain (loss) on foreign currency translation adjustments, foreign currency transactions designated and effective as economic hedges of a net investment in a foreign entity and intra-entity foreign currency transactions that are of a long-term-investment nature.

+ References + Details

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024
Cash flows from operating activities:
Net (loss) income	$ 71,064	$ (69,772)
Adjustments to reconcile net income (loss) to net cash used in operating activities:
Stock-based compensation expense	8,350	10,888
Impairment of intangible assets	58,710	0
Inventory obsolescence charge	11,681	5,244
Income tax expense	2,948	0
Depreciation and amortization expense	3,692	4,741
Non-cash interest expense	2,037	1,489
Fair value adjustment related to warrant and CVR liability	1,379	(4,660)
Accretion on investments	(2,271)	0
Fair value adjustment related to investments	(119)	(64)
Consulting fees paid in common stock	75	399
Loss on foreign currency exchange rates	126	147
Gain on sale of PRV	(148,325)	0
Change in assets and liabilities:
Accounts and other receivables	(5,610)	9,587
Prepaid expenses and other current assets	(3,342)	(452)
Inventories	(780)	(4,159)
Operating lease right-of-use assets	476	389
Accounts payable and accrued expenses	(15,999)	(10,590)
Discount and rebate liabilities	4,517	4,824
Operating lease liabilities	(546)	(395)
Other liabilities	4,823	(1,031)
Net cash used in operating activities	(7,114)	(53,415)
Cash flows from investing activities:
Purchases of property and equipment	(866)	0
Disposals of property and equipment	589	0
Purchases of investments	(222,823)	(41,488)
Maturities of investments	91,000	24,798
Proceeds from sale of PRV	150,000	0
Net cash provided by (used in) investing activities	17,900	(16,690)
Cash flows from financing activities:
Proceeds from issuance of debt, net of lender fees	0	58,990
Payment of third-party debt issuance costs	0	(2,091)
Repayment of debt	0	(42,700)
Proceeds from insurance financing arrangements	0	1,082
Proceeds from Employee Stock Purchase Plan	413	643
Proceeds from issuance of stock	0	64,516
Proceeds from issuance of common stock for options exercised or RSUs vested	3,520	1,303
Proceeds from issuance of common stock for warrants exercised	5,957	0
Payments of principal on insurance financing arrangements	(372)	(431)
Net cash provided by financing activities	9,518	81,312
Effect of exchange rate changes on cash and cash equivalents	350	(217)
Net increase in cash and cash equivalents	20,654	10,990
Cash and cash equivalents, beginning of period	33,785	43,049
Cash and cash equivalents, end of period	54,439	54,039
Supplemental cash flow information:
Cash paid for interest	3,992	3,668
Right-of-use assets obtained in exchange for lease liabilities	$ 1,115	$ 419

Stock and Warrants (Tables)

9 Months Ended

Sep. 30, 2025

Equity [Abstract]

Schedule of Authorized Shares of Common Stock Reserved for Future Issuance

As of September 30, 2025, and December 31, 2024, the Company had reserved authorized shares of common stock for future issuance as follows:


	September 30, 2025	December 31, 2024
Outstanding awards under equity incentive plans	7,228,555	7,789,658
Outstanding common stock warrants	4,436,637	5,483,537
Possible future issuances under equity incentive plans	6,422,637	5,383,165
Possible future issuances under employee stock purchase plans	1,055,685	1,148,012
Total common shares reserved for future issuance	19,143,514	19,804,372

Schedule of Common Stock Activity

The following table summarizes common stock activity for the three and nine months ended September 30, 2025:


						Shares of Common Stock
Balance as of January 1, 2025						53,670,709
Common stock issued as compensation to third parties						9,306
Common stock issued as a result of stock options exercised or RSUs vested						999,338
Common stock issued as a result of stock warrants exercised						10
Balance as of March 31, 2025						54,679,363
Common stock issued as a result of stock options exercised or RSUs vested						312,514
Common stock issued as a result of the Employee Stock Purchase Plan						92,327
Balance as of June 30, 2025						55,084,204
Common stock issued as a result of stock options exercised or RSUs vested						86,628
Common stock issued as a result of stock warrants exercised						1,046,890
Balance as of September 30, 2025						56,217,722

Goodwill & Intangible Assets (Tables)

9 Months Ended

Sep. 30, 2025

Goodwill and Intangible Assets Disclosure [Abstract]

Schedule of Estimated Amortization Expense For Intangible Assets

For intangible assets subject to amortization, estimated amortization expense for the five fiscal years subsequent to September 30, 2025, is expected to be as follows:


2025 (excluding the nine months ended September 30, 2025)	$	316
2026	1,263
2027	1,263
2028	1,263
2029	632

Commitments and Contingencies	9 Months Ended
Commitments and Contingencies	Sep. 30, 2025
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	Commitments and Contingencies From time to time, the Company is involved in various legal proceedings arising in the normal course of business. For some matters, a liability is not probable, or the amount cannot be reasonably estimated and, therefore, an accrual has not been made. However, for such matters when it is probable that the Company has incurred a liability and can reasonably estimate the amount, the Company accrues and discloses such estimates. Litigation Related to the AZSTARYS License Agreement In September 2024, the Company became engaged in a legal dispute regarding the AZSTARYS License Agreement. The litigation is currently in the discovery phase. The Company cannot predict with certainty the timing or ultimate outcome of this litigation or its potential impact on the Company's business, financial condition, or results of operations. At this time, the Company has not recorded any accrual for contingent liability associated with this matter. The AZSTARYS License Agreement remains in effect during this litigation, and both parties continue to perform their respective obligations thereunder. However, there can be no assurance that this dispute will not have an adverse impact on the Company's relationship with Commave or on the Company's business. The Company will continue to monitor developments in this matter and will assess the potential impact on the Company's financial statements in future periods. The Company expects to incur significant legal expenses in connection with this litigation, which may materially affect its results of operations in future periods. As of September 30, 2025, and December 31, 2024, no accruals were made related to commitments and contingencies.

Leases - Schedule Of Supplemental Balance Sheet Information Related To Leases (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Finance Leases
Property and equipment, at cost	$ 1,031	$ 1,031
Less: accumulated depreciation and amortization	(1,026)	(1,023)
Property and equipment, net	$ 5	$ 8
Finance Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration]	Property and equipment, net	Property and equipment, net
Operating Leases
Operating lease right-of-use assets	$ 1,337	$ 657
Total operating lease right-of-use assets	1,337	657
Current portion of operating lease liabilities	441	420
Operating lease liabilities, less current portion	966	372
Total operating lease liabilities	$ 1,407	$ 792
Weighted Average Remaining Lease Term
Operating leases (in years)	3 years	3 years
Weighted Average Discount Rate
Operating leases	12.90%	9.90%

Stock and Warrants

9 Months Ended

Sep. 30, 2025

Equity [Abstract]

Stock and Warrants

Authorized, Issued, and Outstanding Common Shares

As of September 30, 2025, and December 31, 2024, the Company had authorized shares of common stock of 250,000,000 shares. Of the authorized shares, 57,793,414 and 55,246,401 shares of common stock were issued as of September 30, 2025, and December 31, 2024, respectively, and 56,217,722 and 53,670,709 shares of common stock were outstanding as of September 30, 2025, and December 31, 2024, respectively.

As of September 30, 2025, and December 31, 2024, the Company had reserved authorized shares of common stock for future issuance as follows:


	September 30, 2025	December 31, 2024
Outstanding awards under equity incentive plans	7,228,555	7,789,658
Outstanding common stock warrants	4,436,637	5,483,537
Possible future issuances under equity incentive plans	6,422,637	5,383,165
Possible future issuances under employee stock purchase plans	1,055,685	1,148,012
Total common shares reserved for future issuance	19,143,514	19,804,372

Common Stock Activity

The following table summarizes common stock activity for the three and nine months ended September 30, 2025:


						Shares of Common Stock
Balance as of January 1, 2025						53,670,709
Common stock issued as compensation to third parties						9,306
Common stock issued as a result of stock options exercised or RSUs vested						999,338
Common stock issued as a result of stock warrants exercised						10
Balance as of March 31, 2025						54,679,363
Common stock issued as a result of stock options exercised or RSUs vested						312,514
Common stock issued as a result of the Employee Stock Purchase Plan						92,327
Balance as of June 30, 2025						55,084,204
Common stock issued as a result of stock options exercised or RSUs vested						86,628
Common stock issued as a result of stock warrants exercised						1,046,890
Balance as of September 30, 2025						56,217,722

Authorized, Issued, and Outstanding Preferred Stock

As of September 30, 2025, and December 31, 2024, the Company had 10,000,000 shares of authorized preferred stock, none of which were designated, issued, or outstanding.

Warrants to Purchase Common Stock

The Company has issued warrants to purchase common stock to various third parties, of which 4,436,637 remain outstanding as of September 30, 2025, and are immediately exercisable. These warrants qualify as participating securities under ASC Topic 260, Earnings per Share, and are treated as such in the net income (loss) per share calculation. The Company may be required to redeem these warrants for a cash amount equal to the BSM value of the portion of the warrants to be redeemed.

While the warrants are outstanding (but unexercised), the warrant holders will participate in any dividend or other distribution of the Company’s assets to its common stockholders by way of return of capital or otherwise. As of September 30, 2025, 1,046,900 of the warrants had been exercised. No warrants had been exercised as of December 31, 2024. The warrants have been evaluated to determine the appropriate accounting and classification pursuant to ASC 480 and ASC 815. Generally, freestanding warrants should be classified as (i) liabilities if the warrant terms allow settlement of the warrant exercise in cash and (ii) equity if the warrant terms only allow settlement in shares of common stock.

The Company determined that its outstanding warrants should be recorded as a liability and stated at fair value at each reporting period. Changes to the fair value of the warrant liability are recorded through the unaudited condensed consolidated statements of operations as a fair value adjustment related to warrant and CVR liability. As of September 30, 2025, and December 31, 2024, the fair value of the liability associated with these warrants was approximately $13.8 million and $17.8 million, respectively. The fair value adjustment related to these warrants for the three and nine months ended September 30, 2025 was $5.1 million and $3.1 million of loss, respectively. The fair value adjustment related to these warrants for the three and nine months ended September 30, 2024 was $6.0 million of loss and $2.2 million of income, respectively.

- Definition

The entire disclosure for equity.

+ References

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+ Details

Segment Information (Tables)

9 Months Ended

Sep. 30, 2025

Segment Reporting [Abstract]

Schedule of Segment Reporting Information, by Segment

The following table presents the operating results of the Company's segment for the three and nine months ended September 30, 2025, and 2024:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024
Total revenues	$	26,063	$	3,695	$	72,345	$	11,569
Less significant segment expenses:
Research and development directly identified to programs	1,780		4,916		6,480		18,369
Research and development not directly identified to programs	1,652		6,029		3,643		15,374
Selling, general and administrative directly identified to programs	5,144		4,864		19,144		12,560
Selling, general and administrative not directly identified to programs	11,791		11,344		38,118		26,183
Other segment items:
Impairment of intangible assets	—		—		58,710		—
Income tax (benefit) expense	(433)		—		2,948		—
Interest income	(2,333)		(899)		(5,308)		(2,366)
Depreciation and amortization expense	392		1,617		3,692		4,741
Interest expense	2,051		2,312		6,029		5,157
Other expense (income), net (a)	6,563		6,737		(132,175)		1,323
Segment net (loss) income	$	(544)	$	(33,225)	$	71,064	$	(69,772)

(a) Other expense, net, included in segment net (loss) income includes the gain on the sale of the PRV, foreign currency exchange gains and losses, cost of product revenue (excluding intangible asset amortization), fair value adjustment related to warrant and contingent value right (“CVR”) liabilities, fair value adjustment related to investments, and other overhead expenses.

Debt Obligations - Schedule of Future Minimum Principal Payments (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Debt Disclosure [Abstract]
2025 (excluding the nine months ended September 30, 2025)	$ 0
2026	0
2027	0
2028	0
2029	63,090
Total minimum payments	63,090	$ 61,552
Less: unamortized debt discount, debt issuance costs and paid in kind interest	(1,780)
Long-term debt	$ 61,310	$ 59,504

- Definition

Amount of unamortized debt discount (premium) and debt issuance costs.

+ References + Details

- Definition

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

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+ Details

Inventories (Tables)

9 Months Ended

Sep. 30, 2025

Inventory Disclosure [Abstract]

Schedule of Inventory, Noncurrent

The components of inventory are summarized as follows (in thousands):


	September 30, 2025		December 31, 2024
Raw materials	$	—	$	7,928
Work in progress	1,224		3,260
Finished goods	863		1,781
Total inventories	$	2,087	$	12,969

Stock and Warrants - Schedule of Common Stock Activity (Details) - shares	3 Months Ended			9 Months Ended
	Sep. 30, 2025	Jun. 30, 2025	Mar. 31, 2025	Sep. 30, 2025
Movement In Common Stock Activity [Roll Forward]
Beginning balance (in shares)	55,084,204	54,679,363	53,670,709	53,670,709
Common stock issued as compensation to third parties (in shares)			9,306
Common stock issued as a result of stock options exercised or RSUs vested (in shares)	86,628	312,514	999,338
Common stock issued as a result of stock warrants exercised (in shares)	1,046,890		10
Common stock issued as a result of the Employee Stock Purchase Plan (in shares)		92,327		444,315
Ending balance (in shares)	56,217,722	55,084,204	54,679,363	56,217,722

Stock-Based Compensation (Tables)

9 Months Ended

Sep. 30, 2025

Share-Based Payment Arrangement, Disclosure [Abstract]

Share-Based Payment Arrangement, Expensed and Capitalized


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024
Research and development	$	250	$	1,441	$	721	$	3,322
Selling, general and administrative	2,521		4,696		7,629		7,566
Total stock-based compensation expense	$	2,771	$	6,137	$	8,350	$	10,888

Subsequent Events	9 Months Ended
Subsequent Events	Sep. 30, 2025
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events The Company evaluated events and transactions occurring subsequent to September 30, 2025, through November 5, 2025, the date the accompanying unaudited condensed consolidated financial statements were issued. During this period, there were no subsequent events that required recognition in the accompanying unaudited condensed consolidated financial statements, nor were there any additional non-recognized subsequent events that required disclosure.

Net Income (Loss) Per Share - Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Total securities excluded from the calculation of weighted average number of shares of common stock outstanding (in shares)	11,485,859	14,471,534	1,771,663	14,471,534
Awards under equity incentive plans
Total securities excluded from the calculation of weighted average number of shares of common stock outstanding (in shares)	7,049,222	8,987,997	1,771,663	8,987,997
Common stock warrants
Total securities excluded from the calculation of weighted average number of shares of common stock outstanding (in shares)	4,436,637	5,483,537	0	5,483,537

Segment Information

9 Months Ended

Sep. 30, 2025

Segment Reporting [Abstract]

Segment Information

Zevra manages its business activities on a consolidated basis and operates as a single operating segment dedicated to the research and development, manufacturing, commercialization and sale of innovative medicines and therapies. The Company primarily derives its revenue from reimbursements received from product sales of MIPLYFFA and OLPRUVA, reimbursements received under the French AC, and royalties or net sales milestone payments generated under the AZSTARYS License Agreement. The accounting policies of the segment are the same as those described in Note B.

Zevra's CODM is the Company's Chief Executive Officer, Neil F. McFarlane. The CODM uses net income (loss), as reported in the Company's unaudited condensed consolidated statements of operations, in evaluating performance of its segment and determining how to allocate resources of the Company as a whole, including investing in its research and development, commercialization efforts, and acquisition strategy. The CODM does not review assets in evaluating the results of the segment, and, therefore, such information is not presented.

The following table presents the operating results of the Company's segment for the three and nine months ended September 30, 2025, and 2024:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024
Total revenues	$	26,063	$	3,695	$	72,345	$	11,569
Less significant segment expenses:
Research and development directly identified to programs	1,780		4,916		6,480		18,369
Research and development not directly identified to programs	1,652		6,029		3,643		15,374
Selling, general and administrative directly identified to programs	5,144		4,864		19,144		12,560
Selling, general and administrative not directly identified to programs	11,791		11,344		38,118		26,183
Other segment items:
Impairment of intangible assets	—		—		58,710		—
Income tax (benefit) expense	(433)		—		2,948		—
Interest income	(2,333)		(899)		(5,308)		(2,366)
Depreciation and amortization expense	392		1,617		3,692		4,741
Interest expense	2,051		2,312		6,029		5,157
Other expense (income), net (a)	6,563		6,737		(132,175)		1,323
Segment net (loss) income	$	(544)	$	(33,225)	$	71,064	$	(69,772)

The Company holds long-lived assets in the United States of $2.2 million and $13.4 million as of September 30, 2025, and December 31, 2024, respectively. The Company holds long-lived assets in Europe of $0.4 million and $0.5 million as of September 30, 2025, and December 31, 2024, respectively.

- Definition

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

+ References

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+ Details

Leases - Schedule of Supplemental Cash Flow Information Related To Leases (Details) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$ 688	$ 405
Operating cash flows from short-term leases	127	177
Operating cash flows from variable lease costs	36	39
Right-of-use assets obtained in exchange for lease liabilities:
Operating leases	$ 1,115	$ 419

Summary of Significant Accounting Policies (Policies)	9 Months Ended
Summary of Significant Accounting Policies (Policies)	Sep. 30, 2025
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The Company prepared the unaudited condensed consolidated financial statements in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and the rules and regulations of the Securities and Exchange Commission (“SEC”) and, in the Company’s opinion, reflect all adjustments, including normal recurring items that are necessary. All significant intercompany accounts and transactions have been eliminated in consolidation.
Reclassifications	Reclassifications Certain reclassifications were made to historical period's unaudited condensed consolidated financial statements to conform to the classifications used in the current year. These reclassifications had no impact on the consolidated net (loss) income, changes in stockholder's equity, or cash flows previously reported.
Use of Estimates	Use of Estimates The preparation of these unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires the Company to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. On an ongoing basis, the Company evaluates its estimates and assumptions, including those related to revenue recognition, the useful lives of property and equipment, the recoverability of long-lived assets, the incremental borrowing rate for leases, and assumptions used for purposes of determining stock-based compensation, income taxes, the fair value of the warrant liability and discount and rebate liabilities, among others. The Company bases its estimates on historical experience and on various other assumptions that it believes to be reasonable, the results of which form the basis for making judgments about the carrying value of assets and liabilities.
Investments	Investments The Company maintains investment securities that are classified as available-for-sale securities for which the Company has elected the fair value option under Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 825, Financial Instruments. As such, these securities are carried at fair value with unrealized gains and losses included in fair value adjustment related to investments on the unaudited condensed consolidated statements of operations. The securities primarily consist of U.S. Treasury securities and corporate bonds and are included in securities at fair value in the unaudited condensed consolidated balance sheets. As of September 30, 2025, and December 31, 2024, the Company held securities with an aggregate fair value of $175.9 million and $41.7 million, respectively, that contained an aggregate unrealized gain of $119,000 and $18,000, respectively. For securities held at September 30, 2025, approximately $145.1 million mature within one year and approximately $30.9 million mature in one to three years. Applying fair value accounting to these debt securities more accurately represents the Company's investment strategy due to the fact that excess cash is currently being invested for the purpose of funding future operations. Interest income is recognized as earned using an effective yield method giving effect to the amortization of premium and accretion of discount and is based on the economic life of the securities. Interest income is included in interest and other income, net, in the unaudited condensed consolidated statements of operations.
Variable Interest Entities	Variable Interest Entities The primary beneficiary of a variable interest entity (“VIE”) is required to consolidate the assets and liabilities of the VIE. When the Company obtains a variable interest in another entity, it assesses at the inception of the relationship and upon occurrence of certain significant events whether the entity is a VIE and, if so, whether the Company is the primary beneficiary of the VIE based on its power to direct the activities of the VIE that most significantly impact the VIE's economic performance and the Company's obligation to absorb losses or the rights to receive benefits from the VIE that could potentially be significant to the VIE. To assess whether the Company has the power to direct the activities of the VIE that most significantly impact the VIE's economic performance, the Company considers all the facts and circumstances, including the Company's role in establishing the VIE and the Company's ongoing rights and responsibilities. The assessment includes identifying the activities that most significantly impact the VIE's economic performance and identifying which party, if any, has the power to direct those activities. In general, the parties that make the most significant decisions affecting the VIE (management and members of the Board of Directors) are deemed to have the power to direct the activities of a VIE. To assess whether the Company has the obligation to absorb losses of the VIE or the rights to receive benefits from the VIE that could potentially be significant to the VIE, the Company considers all of its economic interests that are deemed to be variable interests in the VIE. This assessment requires judgment in determining whether these interests, in the aggregate, are considered potentially significant to the VIE. On August 30, 2023, the Company entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Acer Therapeutics Inc. (“Acer”). On November 17, 2023 (the “Closing Date”), Zevra completed the acquisition of Acer (the “Merger”). Pursuant to the Merger Agreement, on the Closing Date, Acer continued as the surviving entity and as a wholly-owned subsidiary of Zevra. As of September 30, 2025, and December 31, 2024, the Company identified Acer to be the Company's sole interest in a VIE. As Zevra is the final decision maker for all of Acer's research, development, and commercialization of drug candidates that it is producing, the Company directs the activities of Acer that most significantly impact its performance. Therefore, the Company is the primary beneficiary of this VIE for accounting purposes and consolidates the assets and liabilities of the VIE.
Revenue Recognition	Revenue Recognition The Company recognizes revenue in accordance with the provisions of ASC 606, Revenue from Contracts with Customers (“ASC 606”) and, as a result, follows the five-step model when recognizing revenue: 1) identifying a contract; 2) identifying the performance obligations; 3) determining the transaction price; 4) allocating the price to performance obligations; and 5) recognizing revenue when the performance obligations have been fulfilled. Product Revenues, Net Net revenues from product sales are recognized at the transaction price when the customer obtains control of the Company's product, which occurs at a point in time, typically upon receipt of the product by the customer. The Company's current single customer is a specialty pharmacy provider. In accordance with ASC 606, the Company recognizes revenue when fulfilling its performance obligation by transferring control of promised goods or services to its customer, in an amount that reflects the consideration that the Company expects to receive in exchange for those goods or services. In determining when the customer obtains control of the product, the Company considers certain indicators, including whether the Company has a present right to payment from the customer, whether title and/or significant risks and rewards of ownership have transferred to the customer and whether customer acceptance has been received. The Company's net revenues represent total revenues adjusted for discounts and allowances, including estimated cash discounts, chargebacks, rebates, returns, copay assistance, data fees and wholesaler fees for services. These adjustments represent variable consideration under ASC 606 and are recorded as a reduction of revenue. These adjustments are established by management as its best estimate based on available information and will be adjusted to reflect known changes in the factors that impact such allowances. Adjustments for variable consideration are determined based on the contractual terms with customers, historical trends, communications with customers and the levels of inventory remaining in the distribution channel, as well as expectations about the market for the product and anticipated introduction of competitive products. Arimoclomol French AC Net revenue includes revenue from the sale of arimoclomol for the treatment of NPC under the remunerated expanded access program in France, Accès Compassionnel (“French AC”). An expanded access program is a program giving specific patients access to a drug that is not yet approved for commercial sale. Only drugs targeting serious or rare indications and for which there is currently no appropriate treatment are considered for expanded access programs. Further, to be considered for the expanded access program, the drug must have proven efficacy and safety and must either be undergoing price negotiations or seeking marketing approval. In accordance with ASC 606, the Company recognizes revenue when fulfilling its performance obligation under the French AC by transferring control of promised goods or services to its customer, in an amount that reflects the consideration that the Company expects to receive in exchange for those goods or services. In determining when the customer obtains control of the product, the Company considers certain indicators, including whether the Company has a present right to payment from the customer, whether title and/or significant risks and rewards of ownership have transferred to the customer and whether customer acceptance has been received. Revenue is recognized net of sales deductions, including discounts, rebates, applicable distributor fees, and revenue-based taxes. The French Health Authorities and the manufacturer have agreed to a price for sales under the French AC, but the final transaction price depends on the terms and conditions in the contracts with the French Health Authorities, following market approval. Any excess in the price charged by the manufacturer compared to the price agreed with the health authorities once the drug product is approved in France must be repaid. The repayment is considered in the clawback liability (rebate). An estimate of net revenue and clawback liability are recognized using the ‘expected value’ method. Accounting for net revenue and clawback liability requires determination of the most appropriate method for the expected final transaction price. This estimate also requires assumptions with respect to inputs into the method, including current pricing of comparable marketed products within the rare disease area in France. Management has considered the expected final sales price as well as the price of similar drug products. The Company is operating within a rare disease therapeutic area where there is unmet treatment need and hence a limited number of comparable commercialized drug products. The limited available relevant market information for directly comparable commercialized drugs within rare disease increases the uncertainty in management's estimate. Licensing Agreements The terms of the Company’s licensing agreements typically include one or more of the following: (i) upfront fees; (ii) milestone payments related to the achievement of development, regulatory, or commercial goals; and (iii) royalties on net sales of licensed products. Each of these payments may result in licensing revenues. As part of the accounting for these agreements, the Company 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. Generally, the estimation of the stand-alone selling price may include such estimates as independent evidence of market price, forecasted revenues or costs, development timelines, discount rates, and probability of regulatory success. The Company evaluates each performance obligation to determine if it can be satisfied at a point in time or over time, and it measures the services delivered to the licensee, which are periodically reviewed based on the progress of the related program. The effect of any change made to an estimated input component and, therefore, revenue or expense recognized, would be recorded as a change in estimate. In addition, variable consideration (e.g., milestone payments) must be evaluated to determine if it is constrained and, therefore, excluded from the transaction price. Upfront Fees: If a license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from the transaction price allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time. Milestone Payments: At the inception of each arrangement that includes milestone payments (variable consideration), the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the Company’s or the licensee’s control, such as non-operational developmental and regulatory approvals, are generally not considered probable of being achieved until those approvals are received. At the end of each reporting period, the Company re-evaluates the probability of achievement of milestones that are within its or the licensee’s control, such as operational developmental milestones and any related constraint, and, if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenues and earnings in the period of adjustment. Revisions to the Company’s estimate of the transaction price may also result in negative licensing revenues and earnings in the period of adjustment.
Inventories	Inventories The value of inventories is recorded at net realizable value. The Company determines the cost of its inventories, which include amounts related to materials and manufacturing overhead, on a first-in, first-out basis. Inventories that are not expected to be sold within 12 months are classified as inventories, noncurrent. The Company may scale-up and make commercial quantities of its product candidates prior to the date it anticipates that such product will receive final regulatory approval. The scale-up and commercial production of pre-launch inventory involves the risk that such products may not be approved for marketing on a timely basis, or ever. This risk notwithstanding, the Company may scale-up and build pre-launch inventory of products that have not received final regulatory approval when the Company believes such action is appropriate in relation to the commercial value of the product launch opportunity. We capitalize inventory costs associated with our products prior to regulatory approval when, based on management's judgment, future commercialization is considered probable and the future economic benefit is expected to be realized; otherwise, such costs are expensed as research and development. The determination to capitalize inventory costs is based on various factors, including status and expectations of the regulatory approval process, any known safety or efficacy concerns, potential labeling restrictions, and any other impediments to obtaining regulatory approval. The Company had no pre-approval inventory on its unaudited condensed consolidated balance sheets as of September 30, 2025, or December 31, 2024. Inventory used in clinical trials is also expensed as research and development expense when selected for such use. Inventory that can be used in the production of either clinical or commercial products is expensed as research and development costs when identified for use in a clinical manufacturing campaign. The cost of finished goods inventory that is shipped to a customer to support the Company’s patient assistance programs is expensed when those shipments take place. As of September 30, 2025, and December 31, 2024, the Company did not have pre-launch inventory that qualified for capitalization. The Company performs an assessment of the recoverability of capitalized inventory during each reporting period and writes down any excess and obsolete inventory to its net realizable value in the period in which the impairment is first identified. Such impairment charges, should they occur, are recorded as a component of cost of product revenue in the unaudited condensed consolidated statements of operations. The determination of whether inventory costs will be realizable requires the use of estimates by management. If actual market conditions are less favorable than projected by management, additional write downs of inventory may be required. Additionally, the Company’s products are subject to strict quality control and monitoring, which is performed throughout the manufacturing process. In the event that certain batches or units of product do not meet quality specifications, the Company will record a charge to cost of product revenue, to write down any unsaleable inventory to its estimated net realizable value.
Cost of Product Revenue	Cost of Product Revenue The components of cost of product revenue are royalties and expenses directly attributable to revenue. To date, the Company has generated revenue from product sales of MIPLYFFA and OLPRUVA, reimbursements received under the French AC, royalties or net sales milestone payments generated under the Collaboration and License Agreement with Commave Therapeutics SA (the “AZSTARYS® License Agreement”), and consulting agreements. Prior to our acquisition of the assets of Orphazyme A/S (“Orphazyme”) in May 2022, Orphazyme had entered into an asset purchase agreement with LadRx Corporation, which was assigned to XOMA (US) LLC, a wholly-owned subsidiary of XOMA Corporation (“XOMA”), in June 2023 (“XOMA License Agreement”). Under the XOMA License Agreement, XOMA is entitled to a mid-single digit percentage royalty with respect to net sales of MIPLYFFA as well as milestone payments based on future potential sales and regulatory milestones. As a condition to entering into the Merger Agreement, Acer and Relief Therapeutics SA (“Relief”) entered into an exclusive license agreement on August 30, 2023 (the “Relief License Agreement”). Pursuant to the Relief License Agreement, Zevra was obligated to pay royalties of 10% of U.S. net sales of OLPRUVA up to a maximum of $45.0 million, plus specified regulatory milestones, for total payments to Relief of up to $56.5 million. On April 10, 2025, the rights to this royalty were sold to Soleus Capital Management L.P. In connection with the AZSTARYS License Agreement, the Company pays Aquestive Therapeutics, Inc. (“Aquestive”) a royalty equal to 10% of all regulatory milestone and royalty payments.
Income Taxes	Income Taxes The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the financial reporting and tax basis of assets and liabilities, as well as for operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using the tax rates that are expected to apply to taxable income for the years in which those tax assets and liabilities are expected to be realized or settled. Valuation allowances are recorded to reduce deferred tax assets to the amount the Company believes is more likely than not to be realized. Uncertain tax positions are recognized only when the Company believes it is more likely than not that the tax position will be upheld on examination by the taxing authorities based on the merits of the position. The Company recognizes interest and penalties, if any, related to unrecognized income tax uncertainties in income tax expense. The Company files income tax returns in the United States for federal and various state jurisdictions. With few exceptions, the Company is no longer subject to U.S. federal and state and local income tax examinations for years prior to 2015, although carryforward attributes that were generated prior to 2017 may still be adjusted upon examination by the Internal Revenue Service if used in a future period. No income tax returns are currently under examination by taxing authorities. On December 22, 2017, the U.S. government enacted H.R. 1, “An Act to provide for reconciliation pursuant to titles II and V of the concurrent resolution on the budget for fiscal year 2018” (“Tax Act”). Effective January 1, 2018, the Tax Act provides for a new global intangible low-taxed income (“GILTI”) provision. Under the GILTI provision, certain foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets are included in U.S. taxable income. The Company has not recorded any deferred taxes for future GILTI inclusions as any future inclusions are expected to be treated as a period expense and offset by net operating loss carryforwards in the U.S. On July 4, 2025, the One Big Beautiful Bill Act (the “OBBB”), which includes a broad range of tax reform provisions, was signed into law in the United States. The Company continues to assess the impact of the OBBB but currently does not expect the OBBB to have a material impact on the Company's estimated annual effective tax rate in 2025.
Segment and Geographic Information	Segment and Geographic Information Operating segments are defined as components of an enterprise (business activity from which it earns revenue and incurs expenses) for which discrete financial information is available and regularly reviewed by the chief operating decision maker (“CODM”) in deciding how to allocate resources and in assessing performance. The Company’s CODM is its Chief Executive Officer. The Company views its operations and manages its business as a single operating and reporting segment.
Foreign Currency	Foreign Currency Assets and liabilities are translated into the reporting currency using the exchange rates in effect on the balance sheet dates. Equity accounts are translated at historical rates, except for the change in retained earnings during the year, which is the result of the income statement translation process. Revenue and expense accounts are translated using the weighted average exchange rate during the period. The cumulative translation adjustments associated with the net assets of foreign subsidiaries are recorded in accumulated other comprehensive income (loss) in the accompanying unaudited condensed consolidated statements of changes in stockholders’ equity.
Debt Issuance Costs	Debt Issuance Costs Debt issuance costs incurred in connection with financing arrangements are recorded as a reduction of the related debt on the unaudited condensed consolidated balance sheets and amortized over the life of the respective financing arrangement using the effective interest method.
Warrants	WarrantsThe Company accounts for warrants as either equity-classified or liability-classified instruments based on an assessment of the warrant’s specific terms and applicable authoritative guidance in FASB ASC Topic 480, Distinguishing Liabilities from Equity (“ASC 480”) and FASB ASC Topic 815, Derivatives and Hedging (“ASC 815”). The assessment considers whether the warrants are freestanding financial instruments pursuant to ASC 480, meet the definition of a liability pursuant to ASC 480, and whether the warrants meet all of the requirements for equity classification under ASC 815, including whether the warrants are indexed to the Company's own stock and whether the warrant holders could potentially require “net cash settlement” in a circumstance outside of the issuing company’s control, among other conditions for equity classification. This assessment, which requires the use of professional judgment, is conducted at the time of warrant issuance and as of each subsequent quarterly period end date while the warrants are outstanding.For warrants that meet all criteria for equity classification, the warrants are required to be recorded as a component of additional paid-in capital on the unaudited condensed consolidated statements of changes in stockholders’ equity at the time of issuance. For warrants that do not meet all the criteria for equity classification, the warrants are required to be recorded at their initial fair value on the date of issuance, and on each balance sheet date thereafter. Changes in the estimated fair value of the warrants are recognized as a non-cash gain or loss in other expense, net, on the unaudited condensed consolidated statements of operations. The fair value of the warrants was estimated using the Black-Scholes-Merton (“BSM”) option pricing model.
New Accounting Pronouncements Not Yet Adopted	New Accounting Pronouncements Not Yet Adopted Income Taxes (Topic 740): Improvements to Income Tax Disclosures In December 2023, the FASB issued Accounting Standards Update (“ASU”) No. 2023-09, Income Taxes (“Topic 740”): Improvements to Income Tax Disclosures, which expands disclosures in an entity’s income tax rate reconciliation table and disclosures regarding cash taxes paid both in the U.S. and in foreign jurisdictions. The update will be effective for annual periods beginning after December 15, 2024. The Company is currently evaluating the impact that this guidance will have on the presentation of its financial statements and accompanying notes.

Debt Obligations - Schedule of Long-Term Debt (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Debt Disclosure [Abstract]
Notes payable	$ 63,090	$ 61,552
Unamortized original issue discount	(801)	(921)
Less: debt issuance costs	(979)	(1,127)
Long-term debt	$ 61,310	$ 59,504

- Definition

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

+ References

+ Details

Debt Obligations

9 Months Ended

Sep. 30, 2025

Debt Disclosure [Abstract]

Debt Obligations

Term Loans

On April 5, 2024 (the “Term Loans Closing Date”), the Company entered into a credit agreement (the “Credit Agreement”) with HCR Stafford Fund II, L.P., HCR Potomac Fund II, L.P., and Perceptive Credit Holdings IV, LP (collectively, the “Lenders”), and Alter Domus (US) LLC, as administrative agent (the “Administrative Agent”).

Under the terms of the Credit Agreement, the Lenders provided a senior secured loan facility to the Company in the aggregate principal amount of $100.0 million, which is divided into three tranches as follows: (i) $60.0 million, which was funded in full on the Term Loans Closing Date; (ii) $20.0 million, which was available to the Company in up to two drawings, each in an amount not to exceed $10.0 million, at the Company's option until October 5, 2025; and; (iii) $20.0 million, which was available to the Company upon approval by the FDA of the NDA for MIPLYFFA for the treatment of NPC, at the Company’s option until December 31, 2024 (collectively, the “Term Loans”). The Company did not draw down the amounts described in (ii) and (iii) above prior to their applicable expiration dates.

The principal amount of the Term Loans outstanding (the “Outstanding Principal Amount”) will bear interest at a rate equal to 3-Month Term Secured Overnight Financing Rate (“SOFR”) plus 7.00% per annum. If the net product sales for the calendar year ending December 31, 2025 exceed $100.0 million, the Outstanding Principal Amount will bear interest at 3-Month Term SOFR plus 6.00% per annum. If the net product sales for the calendar year ending December 31, 2025 do not exceed $100.0 million, then for any subsequent period of four consecutive fiscal quarters ending on or after March 31, 2026, in which net product sales exceed $125.0 million, the Outstanding Principal Amount will bear interest at 3-Month Term SOFR plus 6.50% per annum. In all cases, the 3-Month Term SOFR rate will be subject to a floor of 4.00% per annum. Interest will be payable quarterly in arrears on the last day of each calendar quarter. The Company has the option to pay up to 25% of the interest in-kind beginning on the Term Loans Closing Date, through and including June 30, 2026. The Company has recognized approximately $2.7 million and $1.4 million of interest-in-kind as of September 30, 2025, and December 31, 2024, which is included in long-term debt in the unaudited condensed consolidated balance sheets. The Term Loans will mature on the fifth anniversary of the Term Loans Closing Date. In connection with the Credit Agreement, the Company incurred approximately $2.2 million of costs, which primarily consisted of underwriting, legal and other professional fees, and are included as a reduction to the carrying amount of the related debt liability and are deferred and amortized over the remaining life of the financing using the effective interest method.

The Credit Agreement contains customary affirmative and negative covenants by the Company, which, among other things, will require the Company to provide certain financial reports to the Lenders within 60 days after the end of each of the first three fiscal quarters of each fiscal year and 105 days after the end of each fiscal year, meet certain minimum net product sales amounts, and limit the ability of the Company to incur or guarantee additional indebtedness, engage in certain transactions, and effect a consolidation or merger without consent. In addition, as long as the line of credit remains active, the Company must maintain a minimum cash balance of $20.0 million to ensure the Company can meet its immediate capital needs. The obligations of the Company under the Credit Agreement may be accelerated upon customary events of default, including non-payment of principal, interest, fees and other amounts, covenant defaults, insolvency, material judgments, or inaccuracy of representations and warranties. The Term Loans are secured by a first priority perfected lien on, and security interest in, substantially all current and future assets of the Company. The proceeds of the Term Loans were used to refinance certain existing indebtedness of the Company and its subsidiaries. The Company will use the remaining proceeds to pay fees and expenses related to the debt financing and commercialization of MIPLYFFA and OLPRUVA, and to further the development of its other product candidates.

Long-term debt consisted of the following (in thousands):


	September 30, 2025		December 31, 2024
Notes payable	$	63,090	$	61,552
Unamortized original issue discount	(801)		(921)
Less: debt issuance costs	(979)		(1,127)
	$	61,310	$	59,504

Future minimum principal payments under the Term Loans as of September 30, 2025, were as follows (in thousands):


Year Ended December 31,
2025 (excluding the nine months ended September 30, 2025)	$	—
2026	—
2027	—
2028	—
2029	63,090
Total minimum payments	63,090
Less: unamortized debt discount, debt issuance costs and paid in kind interest	(1,780)
Long-term debt	$	61,310

- Definition

The entire disclosure for information about short-term and long-term debt arrangements, which includes amounts of borrowings under each line of credit, note payable, commercial paper issue, bonds indenture, debenture issue, own-share lending arrangements and any other contractual agreement to repay funds, and about the underlying arrangements, rationale for a classification as long-term, including repayment terms, interest rates, collateral provided, restrictions on use of assets and activities, whether or not in compliance with debt covenants, and other matters important to users of the financial statements, such as the effects of refinancing and noncompliance with debt covenants.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Debt Obligations - Narrative (Details) $ in Thousands		9 Months Ended
Debt Obligations - Narrative (Details) $ in Thousands	Apr. 05, 2024 USD ($) tranche drawing	Sep. 30, 2025 USD ($)	Dec. 31, 2024 USD ($)
Debt Instrument [Line Items]
Debt issuance costs net		$ 979	$ 1,127
Debt instrument, number of days to provide report to lenders, quarterly duration		60 days
Debt instrument, number of days to provide report to lenders, annual duration		105 days
Term Loans Under Credit Agreement
Debt Instrument [Line Items]
Debt instrument face amount	$ 100,000
Debt instrument, number of tranches \| tranche	3
Proceeds from issuance of debt	$ 60,000
Debt instrument, available amount until 18 months following closing date	$ 20,000
Debt instrument, number of drawings \| drawing	2
Debt instrument, available amount until 18 months following closing date, maximum each drawing	$ 10,000
Debt instrument, available amount until December 31, 2024	$ 20,000
Basis spread on variable rate	7.00%
Debt instrument, net product sales performance benchmark	$ 100,000
Debt instrument, basis spread on variable rate when performance benchmark meet	6.00%
Debt instrument, net product sales performance benchmark in next year	$ 125,000
Debt instrument, basis spread on variable rate when performance benchmark meet in next year	6.50%
Debt instrument, floor rate	4.00%
Debt instrument, maximum interest payment, percentage	25.00%
Interest payable		$ 2,700	$ 1,400
Debt issuance costs net		2,200
Debt instrument minimum cash balance		$ 20,000

Stock-Based Compensation - Stock-based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	$ 2,771	$ 6,137	$ 8,350	$ 10,888
Research and development
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	250	1,441	721	3,322
Selling, general and administrative
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	$ 2,521	$ 4,696	$ 7,629	$ 7,566

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Inventories (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Inventory Disclosure [Abstract]
Raw materials	$ 0	$ 7,928
Work in progress	1,224	3,260
Finished goods	863	1,781
Total inventories	$ 2,087	$ 12,969

Stock and Warrants - Narrative (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended		12 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024	Dec. 31, 2024	Jun. 30, 2025	Mar. 31, 2025
Class of Warrant or Right [Line Items]
Common stock, shares authorized (in shares)	250,000,000		250,000,000		250,000,000
Common stock, shares issued (in shares)	57,793,414		57,793,414		55,246,401
Common stock, shares outstanding (in shares)	56,217,722		56,217,722		53,670,709	55,084,204	54,679,363
Preferred stock, shares authorized (in shares)	10,000,000		10,000,000		10,000,000
Preferred stock, shares issued (in shares)	0		0		0
Preferred stock, shares outstanding (in shares)	0		0		0
Class of warrant or right, outstanding (in shares)	4,436,637		4,436,637
Fair value adjustment related to warrant and CVR liability	$ 5,506	$ 4,746	$ 1,379	$ (4,660)
Fair Value, Recurring
Class of Warrant or Right [Line Items]
Total liabilities	15,517		15,517		$ 21,304
Warrant liabilities \| Derivative Financial Instruments, Liabilities \| Fair Value, Recurring
Class of Warrant or Right [Line Items]
Total liabilities	13,767		13,767		$ 17,804
Fair value adjustment related to warrant and CVR liability	$ (5,100)	$ (6,000)	$ (3,100)	$ 2,200
Acer Warrant
Class of Warrant or Right [Line Items]
Class of warrants or right, exercised (in shares)			1,046,900		0

Leases - Narrative (Details)	9 Months Ended
Leases - Narrative (Details)	Sep. 30, 2025
Lessee, Lease, Description [Line Items]
Options to terminate leases (in years)	1 year
Minimum
Lessee, Lease, Description [Line Items]
Remaining lease term (in years)	1 year
Maximum
Lessee, Lease, Description [Line Items]
Remaining lease term (in years)	3 years

Stock-Based Compensation - Narrative (Details) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended			9 Months Ended
	Jan. 01, 2025	Jun. 30, 2021	Sep. 30, 2025	Jun. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Employee stock purchase plan, maximum number of issuable shares (in shares)		1,500,000
Stock issued during period, shares, employee stock purchase plans (in shares)				92,327		444,315
Stock based compensation expense			$ 2,771		$ 6,137	$ 8,350	$ 10,888
Performance Shares
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Stock based compensation expense			$ 0		$ 2,500	$ 0	$ 2,500
2014 Equity Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Share-based payment award, number of additional shares authorized (in shares)		4,900,000				12,079,711
Number of shares reserved for issuance, incremental percentage of capital stock outstanding		4.00%
Increase in common stock reserved for issuance (in shares)	2,146,828
Common stock issued as a result of stock options exercised or released (in shares)			86,628		27,750	1,162,133	761,500
The 2023 Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Share-based payment award, number of additional shares authorized (in shares)						4,500,000

Goodwill & Intangible Assets

9 Months Ended

Sep. 30, 2025

Goodwill and Intangible Assets Disclosure [Abstract]

Goodwill & Intangible Assets

L. Goodwill & Intangible Assets

The Company's goodwill balance was $4.7 million as of September 30, 2025, and December 31, 2024.

As of September 30, 2025, and December 31, 2024, non-amortizable intangible assets include $2.0 million related to in-process research and development associated with the Merger.

The definite-lived intangible assets that are subject to amortization have been reviewed for impairment whenever events or changes in circumstances indicate that their carrying amounts may not be recoverable. In the second quarter of 2025, the Company assessed the results of its refined commercial efforts related to OLPRUVA. This was determined to be a triggering event that could result in a decrease in future expected cash flows, and thus indicated the carrying amount of the OLPRUVA asset group may not be fully recoverable. The Company performed an undiscounted cash flow analysis over the OLPRUVA asset group and determined that the carrying value of the asset group is not recoverable. Future cash flows specific to OLPRUVA, which most significantly includes an estimate of forecasted revenues, are based on reasonable and supportable assumptions regarding the cash flows expected to result from the use of the asset and its eventual disposition. The Company then estimated the fair value of the asset group to measure the impairment loss for the period. The fair value measurement was based on Level 3 inputs including projected sales driven by market share and product sales price estimates, associated expenses, growth rates, and the discount rate used to measure the fair value of the net cash flows associated with this asset group. The Company recorded an intangible asset impairment charge of $0 and $58.7 million, respectively, in the unaudited condensed consolidated statement of operations for the three and nine months ended September 30, 2025. As of December 31, 2024, the Company had a definite-lived intangible asset, net, related to the acquisition of OLPRUVA of $61.3 million.

Prior to the impairment discussed above, the OLPRUVA definite-lived intangible asset was being amortized on a straight-line basis over the OLPRUVA patent life of 13 years. Amortization expense is recorded as intangible asset amortization in the unaudited condensed consolidated statements of operations and was $0 and $2.6 million for the three and nine months ended September 30, 2025, respectively. There was $1.5 million and $4.6 million of amortization expense related to this intangible asset for the three and nine months ended September 30, 2024, respectively.

In connection with the XOMA License Agreement, a regulatory milestone payment of $6.0 million was due to XOMA upon approval of MIPLYFFA in the U.S., which the Company paid in October 2024. This definite-lived intangible asset is amortized on a straight-line basis over the MIPLYFFA patent life of approximately five years and is reviewed periodically for impairment. Amortization expense is recorded as intangible asset amortization in the unaudited condensed consolidated statements of operations and was $0.3 million and $0.9 million for the three and nine months ended September 30, 2025, respectively. Amortization expense related to this intangible asset was de minimis for the three and nine months ended September 30, 2024.

For intangible assets subject to amortization, estimated amortization expense for the five fiscal years subsequent to September 30, 2025, is expected to be as follows:


2025 (excluding the nine months ended September 30, 2025)	$	316
2026	1,263
2027	1,263
2028	1,263
2029	632

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Parenthetical) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Income Statement [Abstract]
Intangible asset amortization	$ 315	$ 1,545	$ 3,546	$ 4,619

- Definition

The aggregate expense charged against earnings to allocate the cost of intangible assets (nonphysical assets not used in production) in a systematic and rational manner to the periods expected to benefit from such assets.

+ References

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Reference 6: http://www.xbrl.org/2003/role/exampleRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Net Income (Loss) Per Share (Tables)

9 Months Ended

Sep. 30, 2025

Earnings Per Share [Abstract]

Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share

The following securities, presented on a common stock equivalent basis, have been excluded from the calculation of weighted-average number of shares of common stock outstanding because their effect is anti-dilutive:


	Three months ended September 30,		Nine months ended September 30,
	2025	2024	2025	2024
Awards under equity incentive plans	7,049,222	8,987,997	1,771,663	8,987,997
Common stock warrants	4,436,637	5,483,537	—	5,483,537
Total securities excluded from the calculation of weighted average number of shares of common stock outstanding	11,485,859	14,471,534	1,771,663	14,471,534

Schedule of Earnings Per Share, Basic and Diluted

A reconciliation from net income to basic net income attributable to common stockholders per share of common stock and diluted net income attributable to common stockholders per share of common stock for the three and nine months ended September 30, 2025, is as follows (in thousands):


	Three Months Ended September 30, 2025		Nine Months Ended September 30, 2025
Basic net (loss) income per share of common stock:
Net (loss) income	$	(544)	$	71,064
Earnings allocated to participating securities	—		(5,309)
Net (loss) income attributable to shares of common stock	(544)		65,755
Less: Dividends declared or accumulated	—		—
Undistributed net (loss) income attributable to shares of common stock, basic	(544)		65,755
Weighted-average shares of common stock outstanding	55,952		54,949
Basic net (loss) income per share of common stock	$	(0.01)	$	1.20

Diluted net (loss) income per share of common stock:
Net (loss) income attributable to shares of common stock	$	(544)	$	65,755
Less: Fair value adjustment income related to warrant liability	—		—
Net (loss) income attributable to shares of common stock, diluted	(544)		65,755
Weighted-average number of shares of common stock outstanding	55,952		54,949
Dilutive effect of outstanding stock options (as converted to common stock)	—		1,834
Weighted-average shares of common stock outstanding, diluted	55,952		56,783
Diluted net (loss) income per share of common stock	$	(0.01)	$	1.16

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY - USD ($) $ in Thousands	Total	Public Offering	Common Stock	Common Stock Public Offering	Additional Paid-in Capital	Additional Paid-in Capital Public Offering	Treasury Stock, at cost	Accumulated Deficit	Other Comprehensive Income (Loss)
Beginning balance at Dec. 31, 2023	$ 61,864		$ 4		$ 472,664		$ (10,983)	$ (399,778)	$ (43)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	(16,622)							(16,622)
Stock-based compensation expense	2,190				2,190
Issuance of common stock in exchange for consulting services	56				56
Issuance of common stock for options exercised or RSUs vested	1,146				1,146
Other comprehensive (loss) income	184								184
Ending balance at Mar. 31, 2024	48,818		4		476,056		(10,983)	(416,400)	141
Beginning balance at Dec. 31, 2023	61,864		4		472,664		(10,983)	(399,778)	(43)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	(69,772)
Other comprehensive (loss) income	(72)
Ending balance at Sep. 30, 2024	69,770		5		550,413		(10,983)	(469,550)	(115)
Beginning balance at Mar. 31, 2024	48,818		4		476,056		(10,983)	(416,400)	141
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	(19,925)							(19,925)
Stock-based compensation expense	2,688				2,688
Issuance of common stock in exchange for consulting services	193				193
Issuance of common stock as part of the Employee Stock Purchase Plan	424				424
Other comprehensive (loss) income	280								280
Ending balance at Jun. 30, 2024	32,478		4		479,361		(10,983)	(436,325)	421
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	(33,225)							(33,225)
Stock-based compensation expense	6,229				6,229
Issuance of common stock in exchange for consulting services	150				150
Issuance of common stock for options exercised or RSUs vested	157				157
Issuance of common stock in public offering		$ 64,517		$ 1		$ 64,516
Other comprehensive (loss) income	(536)								(536)
Ending balance at Sep. 30, 2024	69,770		5		550,413		(10,983)	(469,550)	(115)
Beginning balance at Dec. 31, 2024	39,666		5		555,302		(10,983)	(505,289)	631
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	(3,099)							(3,099)
Stock-based compensation expense	3,115				3,115
Issuance of common stock in exchange for consulting services	75				75
Issuance of common stock for options exercised or RSUs vested	1,979				1,979
Other comprehensive (loss) income	(713)								(713)
Ending balance at Mar. 31, 2025	41,023		5		560,471		(10,983)	(508,388)	(82)
Beginning balance at Dec. 31, 2024	39,666		5		555,302		(10,983)	(505,289)	631
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	71,064
Other comprehensive (loss) income	(3,047)
Ending balance at Sep. 30, 2025	133,165		6		580,783		(10,983)	(434,225)	(2,416)
Beginning balance at Mar. 31, 2025	41,023		5		560,471		(10,983)	(508,388)	(82)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	74,707							74,707
Stock-based compensation expense	2,464				2,464
Issuance of common stock for options exercised or RSUs vested	969		1		968
Issuance of common stock as part of the Employee Stock Purchase Plan	413				413
Other comprehensive (loss) income	(2,346)								(2,346)
Ending balance at Jun. 30, 2025	117,230		6		564,316		(10,983)	(433,681)	(2,428)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net (loss) income	(544)							(544)
Stock-based compensation expense	2,771				2,771
Issuance of common stock for options exercised or RSUs vested	573				573
Issuance of common stock for stock warrants exercised	13,123				13,123
Other comprehensive (loss) income	12								12
Ending balance at Sep. 30, 2025	$ 133,165		$ 6		$ 580,783		$ (10,983)	$ (434,225)	$ (2,416)

Net Income (Loss) Per Share

9 Months Ended

Sep. 30, 2025

Earnings Per Share [Abstract]

Net Income (Loss) Per Share

The two-class method requires earnings for the period to be allocated between common stock and participating securities based upon their respective rights to receive distributed and undistributed earnings. Under the two-class method, for periods with net income attributable to common stockholders, basic net income attributable to common stockholders per share of common stock is computed by dividing the net income attributable to common stockholders by the weighted average number of shares of common stock outstanding during the period. Undistributed net income attributable to common stockholders is computed by subtracting from net income the portion of current period earnings that participating securities would have been entitled to receive pursuant to their dividend rights had all of the period’s earnings been distributed. No such adjustment to earnings is made during periods with a net loss as the holders of the participating securities have no obligation to fund losses. Diluted net income attributable to common stockholders per share of common stock is computed under the two-class method by using the weighted average number of shares of common stock outstanding plus the potential dilutive effects of stock options and warrants. In addition to analyzing under the two-class method, the Company analyzes the potential dilutive effect of stock options and warrants under the treasury-stock method when calculating diluted income (loss) attributable to common stockholders per share of common stock, in which it is assumed that the stock options and warrants convert into common stock at the beginning of the period or date of issuance, if the stock option or warrant was issued during the period. The Company reports the more dilutive of the approaches (two-class or treasury-stock/if-converted) as its diluted net income (loss) attributable to common stockholders per share of common stock during the period.

Diluted net loss per share of common stock is the same as basic net loss per share of common stock for the three and nine months ended September 30, 2024, because the effects of potentially dilutive items were anti-dilutive for the respective periods. The following securities, presented on a common stock equivalent basis, have been excluded from the calculation of weighted-average number of shares of common stock outstanding because their effect is anti-dilutive:


	Three months ended September 30,		Nine months ended September 30,
	2025	2024	2025	2024
Awards under equity incentive plans	7,049,222	8,987,997	1,771,663	8,987,997
Common stock warrants	4,436,637	5,483,537	—	5,483,537
Total securities excluded from the calculation of weighted average number of shares of common stock outstanding	11,485,859	14,471,534	1,771,663	14,471,534


	Three Months Ended September 30, 2025		Nine Months Ended September 30, 2025
Basic net (loss) income per share of common stock:
Net (loss) income	$	(544)	$	71,064
Earnings allocated to participating securities	—		(5,309)
Net (loss) income attributable to shares of common stock	(544)		65,755
Less: Dividends declared or accumulated	—		—
Undistributed net (loss) income attributable to shares of common stock, basic	(544)		65,755
Weighted-average shares of common stock outstanding	55,952		54,949
Basic net (loss) income per share of common stock	$	(0.01)	$	1.20

Diluted net (loss) income per share of common stock:
Net (loss) income attributable to shares of common stock	$	(544)	$	65,755
Less: Fair value adjustment income related to warrant liability	—		—
Net (loss) income attributable to shares of common stock, diluted	(544)		65,755
Weighted-average number of shares of common stock outstanding	55,952		54,949
Dilutive effect of outstanding stock options (as converted to common stock)	—		1,834
Weighted-average shares of common stock outstanding, diluted	55,952		56,783
Diluted net (loss) income per share of common stock	$	(0.01)	$	1.16

Leases - Schedule of Maturities of Operating Lease Liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Operating Leases
2025 (excluding the nine months ended September 30, 2025)	$ 172
2026	552
2027	542
2028	393
2029	39
Total lease payments	1,698
Less: future interest expense	(291)
Lease liabilities	$ 1,407	$ 792

Description of Business, Basis of Presentation, and Significant Transactions	9 Months Ended
	Sep. 30, 2025
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Description of Business, Basis of Presentation, and Significant Transactions	Description of Business, Basis of Presentation, and Significant Transactions Organization Zevra Therapeutics, Inc. (the “Company” or “Zevra”) is a purpose-driven, commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases. The Company has a diverse portfolio of products and product candidates, which includes pre-clinical, clinical, and commercial stage assets. On September 20, 2024, the U.S. Food and Drug Administration (“FDA”) approved the New Drug Application (“NDA”) for MIPLYFFA® (arimoclomol), an orally-delivered treatment for Niemann-Pick disease type C (“NPC”), which is an ultra-rare and progressive neurodegenerative disease. MIPLYFFA, the first FDA-approved treatment for NPC, is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients two years of age and older. MIPLYFFA has also been granted orphan medicinal product designation for the treatment of NPC by the European Commission. The Company's other commercial stage asset, OLPRUVA® (sodium phenylbutyrate) for oral suspension, is approved by the FDA for the treatment of certain urea cycle disorders (“UCDs”). Additionally, the Company has a pipeline of investigational product candidates, including celiprolol for the treatment of Vascular Ehlers-Danlos syndrome (“VEDS”) in patients with a confirmed type III collagen mutation and KP1077, the Company's clinical development product candidate being developed to treat idiopathic hypersomnia (“IH”), a rare neurological sleep disorder, and narcolepsy. The sole active pharmaceutical ingredient of KP1077 is serdexmethylphenidate (“SDX”), the Company's proprietary prodrug of d-methylphenidate (“d-MPH”). The FDA has granted KP1077 orphan drug designation for the treatment of IH. Basis of Presentation The Company prepared the unaudited condensed consolidated financial statements in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and the rules and regulations of the Securities and Exchange Commission (“SEC”) and, in the Company’s opinion, reflect all adjustments, including normal recurring items that are necessary. All significant intercompany accounts and transactions have been eliminated in consolidation. Registration Statements on Form S-3 On February 5, 2024, Zevra filed a registration statement on Form S-3 (File No. 333-276856) registering an aggregate of 2,269,721 shares of Zevra’s common stock. On April 5, 2024, the Company filed an amendment to such registration statement, which was declared effective on April 8, 2024. On June 4, 2024, the Company filed a registration statement on Form S-3 (File No. 333-279941) (the “June 2024 Registration Statement”) under which the Company may sell securities, including as may be issuable upon conversion, redemption, repurchase, exchange or exercise of securities, in one or more offerings up to a total aggregate offering price of $350.0 million, $75.0 million of which was allocated to the sale of the shares of common stock issuable under the 2024 ATM Agreement (as described further below). The registration statement was declared effective on June 13, 2024. August 2024 Offering On August 8, 2024, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with Cantor Fitzgerald & Co. and William Blair & Company, L.L.C., as representatives of the several underwriters named therein (collectively, the “Underwriters”), in connection with the offering, issuance and sale by the Company of 9,230,770 shares of the Company’s common stock at a public offering price of $6.50 per share, pursuant to the June 2024 Registration Statement and a related prospectus supplement dated August 8, 2024, filed with the SEC (the “August 2024 Offering”). Under the terms of the Underwriting Agreement, the Company also granted the Underwriters an option exercisable for 30 days to purchase up to an additional 1,384,615 shares of its Common Stock at the public offering price, less underwriting discounts and commissions, which the Underwriters exercised in full on August 9, 2024. The August 2024 Offering closed on August 12, 2024. Total shares issued were 10,615,385. Net proceeds from the offering were approximately $64.5 million, after deducting underwriting discounts and commissions and estimated offering expenses payable by the Company. The Company intends to use the net proceeds of the offering to support the commercialization of its approved products and the continued development of its product candidates, and for other general corporate purposes. Entry into 2024 ATM Agreement On July 12, 2024, the Company entered into an equity distribution agreement (the “2024 ATM Agreement”) with Citizens JMP Securities LLC (“Citizens JMP”) under which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock having an aggregate offering price of up to $75.0 million through Citizens JMP as its sales agent. The issuance and sale, if any, of common stock by the Company under the 2024 ATM Agreement will be made pursuant to the June 2024 Registration Statement, the accompanying prospectus, and the related prospectus supplement dated July 12, 2024. Citizens JMP may sell the common stock by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415 of the Securities Act. Citizens JMP will use commercially reasonable efforts to sell the common stock from time to time, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company will pay Citizens JMP a commission equal to 3.0% in the aggregate of the gross sales proceeds of any common stock sold through Citizens JMP under the 2024 ATM Agreement. As of September 30, 2025, no shares have been issued or sold under the 2024 ATM Agreement. Reclassifications Certain reclassifications were made to historical period's unaudited condensed consolidated financial statements to conform to the classifications used in the current year. These reclassifications had no impact on the consolidated net (loss) income, changes in stockholder's equity, or cash flows previously reported.

Description of Business, Basis of Presentation, and Significant Transactions (Details) - USD ($) $ / shares in Units, $ in Millions			9 Months Ended
	Aug. 12, 2024	Aug. 08, 2024	Sep. 30, 2025	Jul. 12, 2024	Jun. 04, 2024	Feb. 05, 2024
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Common stock registered, shares (in shares)						2,269,721
Aggregate offering price					$ 350.0
ATM Agreement Sales
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Aggregate offering price					$ 75.0
ATM Agreement Sales \| JMP
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Stock issued (in shares)			0
Aggregate offering price				$ 75.0
Percentage of aggregate gross sales proceeds				3.00%
Underwriting Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Stock issued (in shares)		9,230,770
Shares issued, price per share (in dollars per share)		$ 6.50
Over-Allotment Option
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Option to purchase additional shares, exercise period		30 days
Over-Allotment Option \| Maximum
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Stock issuable during the period (in shares)		1,384,615
Underwriting Agreement and Over-allotment Option
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Stock issued (in shares)	10,615,385
Proceeds from issuance of common stock, net	$ 64.5

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Income Statement [Abstract]
Revenue, net	$ 26,063	$ 3,695	$ 72,345	$ 11,569
Cost of product revenue	1,238	2,303	14,962	6,051
Intangible asset amortization	315	1,545	3,546	4,619
Impairment of intangible assets	0	0	58,710	0
Operating expenses:
Research and development	3,432	10,945	10,123	33,743
Selling, general and administrative	16,935	16,208	57,262	38,743
Total operating expenses	20,367	27,153	67,385	72,486
Income (loss) from operations	4,143	(27,306)	(72,258)	(71,587)
Other (expense) income:
Gain on sale of PRV	0	0	148,325	0
Interest expense	(2,051)	(2,312)	(6,029)	(5,157)
Fair value adjustment related to warrant and CVR liability	(5,506)	(4,746)	(1,379)	4,660
Fair value adjustment related to investments	124	90	119	64
Interest and other income, net	2,313	1,049	5,234	2,248
Total other (expense) income	(5,120)	(5,919)	146,270	1,815
(Loss) income before income taxes	(977)	(33,225)	74,012	(69,772)
Income tax benefit (expense)	433	0	(2,948)	0
Net (loss) income	$ (544)	$ (33,225)	$ 71,064	$ (69,772)
Net (loss) income per share of common stock:
Basic (in dollars per share)	$ (0.01)	$ (0.69)	$ 1.20	$ (1.59)
Diluted (in dollars per share)	$ (0.01)	$ (0.69)	$ 1.16	$ (1.59)
Weighted-average shares of common stock outstanding:
Basic (in shares)	55,951,572	47,808,817	54,949,483	43,843,851
Diluted (in shares)	55,951,572	47,808,817	56,782,763	43,843,851

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Sep. 30, 2025
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

Cover - shares	9 Months Ended
Cover - shares	Sep. 30, 2025	Nov. 03, 2025
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2025
Document Transition Report	false
Entity File Number	001-36913
Entity Registrant Name	Zevra Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	20-5894398
Entity Address, Address Line One	1180 Celebration Boulevard, Suite 103
Entity Address, City or Town	Celebration
Entity Address, State or Province	FL
Entity Address, Postal Zip Code	34747
City Area Code	321
Local Phone Number	939-3416
Title of 12(b) Security	Common Stock, $0.0001 par value per share
Trading Symbol	ZVRA
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		56,297,535
Entity Central Index Key	0001434647
Document Fiscal Year Focus	2025
Document Fiscal Period Focus	Q3
Amendment Flag	false
Current Fiscal Year End Date	--12-31

Inventories

9 Months Ended

Sep. 30, 2025

Inventory Disclosure [Abstract]

Inventories

The components of inventory are summarized as follows (in thousands):


	September 30, 2025		December 31, 2024
Raw materials	$	—	$	7,928
Work in progress	1,224		3,260
Finished goods	863		1,781
Total inventories	$	2,087	$	12,969

Debt Obligations (Tables)

9 Months Ended

Sep. 30, 2025

Debt Disclosure [Abstract]

Schedule of Long-Term Debt

Long-term debt consisted of the following (in thousands):


	September 30, 2025		December 31, 2024
Notes payable	$	63,090	$	61,552
Unamortized original issue discount	(801)		(921)
Less: debt issuance costs	(979)		(1,127)
	$	61,310	$	59,504

Schedule of Future Minimum Principal Payments

Future minimum principal payments under the Term Loans as of September 30, 2025, were as follows (in thousands):


Year Ended December 31,
2025 (excluding the nine months ended September 30, 2025)	$	—
2026	—
2027	—
2028	—
2029	63,090
Total minimum payments	63,090
Less: unamortized debt discount, debt issuance costs and paid in kind interest	(1,780)
Long-term debt	$	61,310

- Definition

Tabular disclosure of long-debt instruments or arrangements, including identification, terms, features, collateral requirements and other information necessary to a fair presentation. These are debt arrangements that originally required repayment more than twelve months after issuance or greater than the normal operating cycle of the entity, if longer.

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+ Details

Revenue, net

9 Months Ended

Sep. 30, 2025

Revenue from Contract with Customer [Abstract]

Revenue, net

For the three and nine months ended September 30, 2025, the Company recorded $26.1 million and $72.3 million, respectively, of revenue. Included in revenue for the nine months ended September 30, 2025 is a de minimis amount related to the licensing of certain IP. For the three and nine months ended September 30, 2024, the Company recorded $3.7 million and $11.6 million, respectively, of revenue.

Product Revenues, Net

On December 27, 2022, the FDA approved OLPRUVA (sodium phenylbutyrate), a prescription medicine used along with certain therapy, including changes in diet, for the chronic management of adults and children with certain UCDs. For the three and nine months ended September 30, 2025, sales of OLPRUVA were $0.1 million and $0.5 million, respectively. For the three and nine months ended September 30, 2024, sales of OLPRUVA were de minimis.

On September 20, 2024, the FDA approved MIPLYFFA (arimoclomol), an orally-delivered treatment for NPC, which is an ultra-rare and progressive neurodegenerative disease, for treatment in combination with miglustat. For the three and nine months ended September 30, 2025, sales of MIPLYFFA were $22.4 million and $61.0 million, respectively.

The Company currently utilizes a single specialty pharmacy provider as its sole distributor for both MIPLYFFA and OLPRUVA. The Company also enters into arrangements with health care providers and payors that provide for government mandated and/or privately negotiated rebates with respect to the purchase of its products. To commercialize MIPLYFFA and OLPRUVA in the U.S., the Company has built marketing, sales, medical affairs, distribution, managerial and other non-technical capabilities or has made arrangements with third parties to perform these services. All revenues derived from sales of MIPLYFFA and OLPRUVA are in the United States.

Adjustments to product revenue recognized as a result of changes in estimates for discounts and allowances for products shipped in previous periods were 4% and 1% of revenue, net, on the unaudited condensed consolidated statements of operations during the three and nine months ended September 30, 2025, respectively. No adjustments to product revenue were recognized as a result of changes in estimates during the three and nine months ended September 30, 2024.

Arimoclomol French AC

For the three and nine months ended September 30, 2025, the Company recognized revenue related to the French AC of $2.4 million and $7.3 million, respectively, net of a clawback liability of $1.5 million and $4.5 million, respectively, and other gross to net adjustments. For the three and nine months ended September 30, 2024, the Company recognized revenue related to the French AC of

$2.6 million and $8.0 million, respectively, net of a clawback liability of $1.4 million and $4.4 million, respectively, and other gross to net adjustments.

The total estimated reserve liability as of September 30, 2025, and December 31, 2024, was $18.9 million and $12.6 million, respectively. As of September 30, 2025, and December 31, 2024, this estimated reserve liability is recorded as discount and rebate liabilities in the unaudited condensed consolidated balance sheets and is separated into current and long-term based upon the timing of the expected payment to the French regulators.

AZSTARYS License Agreement

The Company entered into a Collaboration and License Agreement (the “AZSTARYS License Agreement”) with Commave Therapeutics SA (formerly known as Boston Pharmaceuticals Holdings SA) (“Commave”), an affiliate of Gurnet Point Capital, L.P., dated September 3, 2019. Under the AZSTARYS License Agreement, as amended, the Company granted to Commave an exclusive, worldwide license to develop, manufacture and commercialize the Company’s product candidates containing SDX and d-MPH, including AZSTARYS, or any other product candidates containing SDX and developed to treat ADHD or any other central nervous system disorder. Corium Inc. was tasked by Commave to lead all commercialization activities for AZSTARYS under the AZSTARYS License Agreement. Pursuant to the AZSTARYS License Agreement, Commave agreed to pay milestone payments up to an aggregate of $590.0 million upon the occurrence of specified regulatory milestones related to AZSTARYS, additional fixed payments upon the achievement of specified U.S. sales milestones, and quarterly, tiered royalty payments based on a range of percentages of net sales (as defined in the AZSTARYS License Agreement). Commave is obligated to make such royalty payments on a product-by-product basis until expiration of the royalty term for the applicable product.

The Company concluded that these regulatory milestones, sales milestones and royalty payments each contain a significant uncertainty associated with a future event. As such, these milestone and royalty payments are constrained at contract inception and are not included in the transaction price, as the Company could not conclude that it is probable a significant reversal in the amount of cumulative revenue recognized will not occur surrounding these milestone payments. At the end of each reporting period, the Company updates its assessment of whether the milestone and royalty payments are constrained by considering both the likelihood and magnitude of the potential revenue reversal. For the three and nine months ended September 30, 2025, the Company recognized revenue under the AZSTARYS License Agreement of $1.2 million and $3.3 million, respectively. For the three and nine months ended September 30, 2024, the Company recognized revenue under the AZSTARYS License Agreement of $1.1 million and $3.6 million, respectively. There was no deferred revenue related to this agreement as of September 30, 2025, or December 31, 2024. All revenues recognized under this agreement were derived in the United States.

The AZSTARYS License Agreement is within the scope of ASC 606, as the transaction represents a contract with a customer where the participants function in a customer/vendor relationship and are not exposed equally to the risks and rewards of the activities contemplated under the AZSTARYS License Agreement.

Relief Exclusive License Agreement

Pursuant to the Relief License Agreement, Relief will hold exclusive development and commercialization rights for OLPRUVA in the EU, Liechtenstein, San Marino, Vatican City, Norway, Iceland, Principality of Monaco, Andorra, Gibraltar, Switzerland, United Kingdom, Albania, Bosnia, Kosovo, Montenegro, Serbia and North Macedonia (“Geographical Europe”). The Company has the right to receive a royalty of up to 10% of the net sales of OLPRUVA in Geographical Europe. For the three and nine months ended September 30, 2025, and 2024, the Company did not recognize any revenue under the Relief License Agreement. There was no deferred revenue related to this agreement as of September 30, 2025, and December 31, 2024.

Accounts and Other Receivables

Accounts and other receivables consist of receivables from product sales of MIPLYFFA and OLPRUVA, reimbursements received under the French AC, royalties generated under the AZSTARYS License Agreement, and other receivables due to the Company. Receivables under the AZSTARYS License Agreement are recorded for amounts due to the Company related to reimbursable third-party costs as well as milestones and royalties on product sales. Receivables are recorded for commercial sales of MIPLYFFA and OLPRUVA to a single specialty pharmacy and product sales under the French AC. The Company provides reserves against receivables for estimated losses that may result from a customer's inability to pay. Receivables are evaluated to determine if any reserve or allowance should be recorded based on consideration of the current economic environment, expectations of future economic conditions, specific circumstances and the Company’s own historical collection experience. Amounts determined to be uncollectible are charged or written-off against the reserve.

Accounts and other receivables consist of the following (in thousands):


	September 30, 2025		December 31, 2024
Commercial accounts receivable	$	8,770	$	4,010
Receivables related to product reimbursements	6,056		5,380
Royalties accounts receivable	1,200		786
Other receivables	815		333
Total receivables	$	16,841	$	10,509

As of September 30, 2025, and December 31, 2024, no reserve or allowance for doubtful accounts had been established.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

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+ Details