RHYTHM PHARMACEUTICALS, INC. 10-Q

Item 1. Financial Statements Rhythm Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2025 2024 ​ ​ ​ ​ ​ ​ ​ Assets ​ ​ ​ ​ ​ ​ Current assets: ​ ​ Cash and cash equivalents ​ $ 106,095 ​ $ 89,137 Short-term investments ​ 208,393 ​ 231,428 Accounts receivable, net ​ ​ 17,825 ​ ​ 18,512 Inventory ​ ​ 19,316 ​ ​ 18,741 Prepaid expenses and other current assets ​ 17,659 ​ 16,382 Total current assets ​ 369,288 ​ 374,200 Property and equipment, net ​ 462 ​ 632 Right-of-use asset ​ ​ 3,367 ​ ​ 3,477 Intangible assets, net ​ ​ 5,960 ​ ​ 6,174 Restricted cash ​ 464 ​ 464 Other long-term assets ​ ​ 7,144 ​ ​ 7,326 Total assets ​ $ 386,685 ​ $ 392,273 Liabilities, Convertible Preferred Stock and Stockholders’ equity ​ ​ Current liabilities: ​ ​ Accounts payable ​ $ 11,912 ​ $ 12,328 Accrued expenses and other current liabilities ​ 59,314 ​ 62,658 Other current liability - LG Chem ​ ​ 38,783 ​ ​ 37,704 Lease liability ​ ​ 224 ​ ​ — Deferred revenue ​ ​ — ​ ​ 1,286 Deferred royalty obligation, current ​ ​ 1,590 ​ ​ 1,541 Total current liabilities ​ 111,823 ​ 115,517 Long-term liabilities: ​ ​ Deferred royalty obligation ​ ​ 107,934 ​ ​ 108,269 Lease liability, non-current ​ 3,839 ​ 3,938 Total liabilities ​ 223,596 ​ 227,724 Commitments and contingencies (Note 13) ​ ​ ​ ​ ​ ​ Series A convertible preferred stock, $ 0.001 par value: 150,000 shares authorized; 150,000 and 150,000 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively. Liquidation preference of $ 150,000 as of March 31, 2025. ​ ​ 144,142 ​ ​ 142,820 Stockholders’ equity: ​ ​ Preferred stock, $ 0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at March 31, 2025 and December 31, 2024 ​ — ​ — Common stock, $ 0.001 par value: 120,000,000 shares authorized; 63,494,892 and 62,390,654 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively ​ 62 ​ 61 Additional paid-in capital ​ ​ 1,223,772 ​ ​ 1,177,045 Accumulated other comprehensive (loss) ​ ​ ( 51 ) ​ ​ ( 39 ) Accumulated deficit ​ ( 1,204,836 ) ​ ( 1,155,338 ) Total stockholders’ equity ​ 18,947 ​ 21,729 Total liabilities, convertible preferred stock and stockholders’ equity ​ $ 386,685 ​ $ 392,273 ​ ​ ​ ​ ​ ​ ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 3 Table of Contents ​ Rhythm Pharmaceuticals, Inc. Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ 2025 2024 Revenues: ​ ​ ​ ​ ​ ​ Product revenue, net ​ $ 37,718 ​ $ 25,967 License revenue ​ ​ ( 5,014 ) ​ ​ — Total revenues ​ ​ 32,704 ​ ​ 25,967 Costs and expenses: ​ ​ ​ ​ ​ ​ Cost of sales ​ ​ 3,648 ​ ​ 2,807 Research and development ​ ​ 36,973 ​ ​ 128,665 Selling, general, and administrative ​ 39,087 ​ 34,382 Total costs and expenses ​ 79,708 ​ 165,854 Loss from operations ​ ( 47,004 ) ​ ( 139,887 ) Other income (expense): ​ ​ Other income (expense), net ​ ( 644 ) ​ 524 Interest expense ​ ​ ( 5,409 ) ​ ​ ( 4,755 ) Interest income ​ 3,639 ​ 3,046 Total other (expense), net ​ ( 2,414 ) ​ ( 1,185 ) Loss before income taxes ​ ​ ( 49,418 ) ​ ​ ( 141,072 ) Provision for income taxes ​ ​ 80 ​ ​ 300 Net loss ​ $ ( 49,498 ) ​ $ ( 141,372 ) Accrued dividends on convertible preferred stock ​ ​ ( 1,322 ) ​ ​ — Net loss attributable to common stockholders ​ $ ( 50,820 ) ​ $ ( 141,372 ) Net loss per share attributable to common stockholders, basic and diluted ​ $ ( 0.81 ) ​ $ ( 2.35 ) ​ ​ ​ ​ ​ ​ ​ Weighted-average common shares outstanding, basic and diluted ​ ​ 63,059,165 ​ ​ 60,143,558 ​ ​ ​ ​ ​ ​ ​ Other comprehensive loss: ​ ​ ​ ​ ​ ​ Net loss attributable to common stockholders ​ $ ( 50,820 ) ​ $ ( 141,372 ) Foreign currency translation adjustment ​ ​ ( 2 ) ​ ​ ( 71 ) Unrealized (loss), net on marketable securities ​ ( 10 ) ​ ( 244 ) Comprehensive loss ​ $ ( 50,832 ) ​ $ ( 141,687 ) The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ ​ 4 Table of Contents Rhythm Pharmaceuticals, Inc. Condensed Consolidated Statements of Convertible Preferred Stock & Stockholders’ Equity (in thousands, except share data) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ Series A Convertible ​ ​ ​ ​ ​ ​ Additional ​ Other ​ ​ ​ ​ Total ​ ​ Preferred Stock ​ Common Stock ​ Paid-In ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ Shares Amount Shares Amount Capital Income (Loss) ​ Deficit Equity Balance at December 31, 2024 ​ 150,000 $ 142,820 62,390,654 ​ $ 61 $ 1,177,045 $ ( 39 ) ​ $ ( 1,155,338 ) $ 21,729 Stock compensation expense ​ — ​ ​ — ​ — ​ — ​ 12,862 ​ — ​ ​ — ​ 12,862 Issuance of common stock in connection with ESPP ​ — ​ ​ — ​ 21,875 ​ ​ — ​ ​ 854 ​ ​ — ​ ​ — ​ ​ 854 Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units ​ — ​ ​ — ​ 494,853 ​ ​ 1 ​ ​ 2,225 ​ ​ — ​ ​ — ​ ​ 2,226 Issuance of common stock upon completion of ATM equity offering, net of $ 739 offering costs ​ — ​ ​ — ​ 587,510 ​ ​ — ​ ​ 32,108 ​ ​ — ​ ​ — ​ ​ 32,108 Accretion of preferred stock dividends ​ — ​ ​ 1,322 ​ — ​ ​ — ​ ​ ( 1,322 ) ​ ​ — ​ ​ — ​ ​ ( 1,322 ) Foreign currency translation adjustment ​ — ​ ​ — ​ — ​ ​ — ​ ​ — ​ ​ ( 2 ) ​ ​ — ​ ​ ( 2 ) Net unrealized loss on marketable securities ​ — ​ ​ — ​ — ​ — ​ — ​ ( 10 ) ​ ​ — ​ ( 10 ) Net loss ​ — ​ ​ — ​ — ​ — ​ — ​ — ​ ​ ( 49,498 ) ​ ( 49,498 ) Balance at March 31, 2025 ​ 150,000 ​ $ 144,142 ​ 63,494,892 ​ $ 62 ​ $ 1,223,772 ​ $ ( 51 ) ​ $ ( 1,204,836 ) ​ $ 18,947 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at December 31, 2023 — — 59,426,559 ​ $ 59 $ 1,064,302 $ 134 ​ $ ( 894,736 ) $ 169,759 Stock-based compensation expense ​ — ​ ​ — ​ — ​ ​ — ​ ​ 7,767 ​ ​ — ​ ​ — ​ ​ 7,767 Issuance of common stock in connection with ESPP ​ — ​ ​ — ​ 28,495 ​ ​ — ​ ​ 673 ​ ​ — ​ ​ — ​ ​ 673 Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units ​ — ​ ​ — ​ 1,077,271 ​ ​ 1 ​ ​ 6,352 ​ ​ — ​ ​ — ​ ​ 6,353 Issuance of common stock as consideration for LGC license ​ — ​ ​ — ​ 432,143 ​ ​ — ​ ​ 18,716 ​ ​ — ​ ​ — ​ ​ 18,716 Foreign currency translation adjustment ​ — ​ ​ — ​ — ​ ​ — ​ ​ — ​ ​ ( 71 ) ​ ​ — ​ ​ ( 71 ) Unrealized loss on marketable securities ​ — ​ ​ — ​ — ​ ​ — ​ ​ — ​ ​ ( 244 ) ​ ​ — ​ ​ ( 244 ) Net loss ​ — ​ ​ — ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 141,372 ) ​ ​ ( 141,372 ) Balance at March 31, 2024 ​ — ​ $ — ​ 60,964,468 ​ $ 60 $ 1,097,810 $ ( 181 ) ​ $ ( 1,036,108 ) $ 61,581 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ 5 Table of Contents ​ Rhythm Pharmaceuticals, Inc. Condensed Consolidated Statements of Cash Flows (in thousands) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ 2025 2024 Operating activities ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 49,498 ) ​ $ ( 141,372 ) Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ Stock-based compensation expense ​ 12,862 ​ 7,767 Depreciation and amortization ​ 383 ​ 405 Non-cash interest expense ​ ​ 5,408 ​ ​ 4,009 Non-cash accretion & amortization of short-term investments ​ ​ ( 1,787 ) ​ ​ ( 1,370 ) Non-cash rent expense ​ 110 ​ 111 Change in fair value of embedded derivative liability ​ ​ 80 ​ ​ ( 490 ) Acquired IPR&D assets classified as investing activities ​ ​ — ​ ​ 92,385 Changes in operating assets and liabilities: ​ ​ ​ ​ Accounts receivable ​ ​ 687 ​ ​ 172 Inventory ​ ​ ( 575 ) ​ ​ 117 Prepaid expenses and other current assets ​ ( 3,204 ) ​ ( 2,421 ) Deferred revenue ​ ​ ( 1,286 ) ​ ​ — Other long-term assets, net ​ 102 ​ 1,195 Accounts payable, accrued expenses and other liabilities ​ ( 3,633 ) ​ ( 1,251 ) Net cash used in operating activities ​ ( 40,351 ) ​ ( 40,743 ) Investing activities ​ ​ Purchases of short-term investments ​ ( 33,521 ) ​ — Maturities of short-term investments ​ 58,333 ​ 70,050 Acquisition of IPR&D assets ​ ​ — ​ ​ ( 40,000 ) Net cash (used in) provided by investing activities ​ 24,812 ​ 30,050 Financing activities ​ ​ Repayment of deferred royalty obligation ​ ​ ( 4,615 ) ​ ​ ( 2,783 ) Proceeds from the exercise of stock options ​ 2,226 ​ 6,353 Proceeds from issuance of common stock from ESPP ​ 854 ​ 673 Proceeds from ATM equity offering ​ ​ 34,034 ​ ​ — Net cash provided by financing activities ​ 32,499 ​ 4,243 Effect of exchange rates on cash ​ ​ ( 2 ) ​ ​ ( 71 ) Net increase (decrease) in cash, cash equivalents and restricted cash ​ 16,958 ​ ( 6,521 ) Cash, cash equivalents and restricted cash at beginning of period ​ 89,601 ​ 60,409 Cash, cash equivalents and restricted cash at end of period ​ $ 106,559 ​ $ 53,888 Supplemental disclosure of non-cash investing and financing activities: ​ ​ ​ ​ ​ ​ Non-current liability issued in exchange for the acquisition of IPR&D ​ $ — ​ $ 33,669 Issuance of common stock in exchange for IPR&D ​ $ — ​ $ 18,716 Accretion of preferred stock dividends ​ $ 1,322 ​ $ — ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 6 Table of Contents Rhythm Pharmaceuticals, Inc. Notes to Unaudited Condensed Consolidated Financial Statements (In thousands, except share and per share information) 1. Nature of Business Rhythm Pharmaceuticals, Inc. (the “Company” or “we”) is a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases. We are focused on advancing our melanocortin-4 (MC4R) receptor agonists, including our lead asset, IMCIVREE ® (setmelanotide), as a precision medicine designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases. While obesity affects hundreds of millions of people worldwide, we are developing therapies for a subset of individuals who have hyperphagia, a pathological insatiable hunger and impaired satiety accompanied by persistent and abnormal food-seeking behaviors, decreased energy expenditure and severe obesity due to diseases such as acquired or congenital hypothalamic obesity, Bardet-Biedl syndrome (BBS) or other diseases caused by impaired MC4R pathway signaling. The MC4R pathway is an endocrine pathway in the brain that is responsible for regulating hunger, caloric intake and energy expenditure, which consequently affect body weight. IMCIVREE, an MC4R agonist for which we hold worldwide rights, is the first-ever therapy developed for patients with certain rare diseases that is approved or authorized in the United States, European Union, and Great Britain, Canada, and other countries and regions. The Company is a Delaware corporation organized in February 2013 under the name Rhythm Metabolic, Inc., and as of October 2015, under the name Rhythm Pharmaceuticals, Inc. The Company has wholly owned subsidiaries in the US, Ireland, the United Kingdom, the Netherlands, France, Germany, Italy, Spain and Canada. The Company is subject to risks and uncertainties common to commercial-stage companies in the biotechnology industry, including but not limited to, risks associated with the commercialization of approved products, completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Commercialization of approved products will require significant resources and in order to market IMCIVREE, the Company must continue to build its sales, marketing, managerial and other non-technical capabilities or make arrangements with third parties to perform these services. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even though the Company has an approved product, and even if the Company’s further product development efforts are successful, it is uncertain when, if ever, the Company will realize sufficient revenue from product sales to fund operations. Liquidity The Company has incurred operating losses and experienced negative cash flows from operations since inception. As of March 31, 2025, the Company had an accumulated deficit of $ 1.2 billion. The Company has funded these losses primarily from the proceeds from the sales of common and preferred stock, product revenue, asset sales, royalty financing, out-license arrangements, as well as capital contributions received from the former parent company, Rhythm Holdings LLC. While the Company is generating product revenue, management expects operating losses to continue for the foreseeable future. The Company has devoted substantially all of its resources to its drug development efforts, comprised of research and development, the acquisition of in process research and development assets, manufacturing, conducting clinical trials for its product candidates, protecting its intellectual property, commercialization activities and general and administrative functions relating to these operations. The future success of the Company is dependent on its ability to develop its product candidates and ultimately upon its ability to attain profitable operations. As of March 31, 2025, the Company had $ 314,488 of cash and cash equivalents and short-term investments on hand. In the future, the Company will be dependent on obtaining funding from third parties, such as proceeds from the 7 Table of Contents issuance of debt, sale of equity, proceeds from out license arrangements, product sales and funded research and development programs to maintain the Company's operations and meet the Company's obligations. There is no guarantee that additional equity or other financing will be available to the Company on acceptable terms, or at all. If the Company fails to obtain additional funding when needed, the Company would be forced to scale back, terminate its operations or seek to merge with or be acquired by another company. Management believes that the Company's existing cash resources will be sufficient to fund the Company’s operations through at least the next twelve months from the filing of this Quarterly Report on Form 10-Q with the SEC . 2. Summary of Significant Accounting Policies Basis of Presentation The Company's unaudited condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States, or GAAP, and the applicable rules and regulations of the Securities and Exchange Commission, or SEC, regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates, or ASU, of the Financial Accounting Standards Board, or FASB. As permitted under these rules, certain footnotes or other financial information that are normally required by GAAP have been condensed or omitted. The accompanying condensed consolidated balance sheet as of March 31, 2025, the condensed consolidated statements of operations and comprehensive loss for the three months ended March 31, 2025 and 2024, the condensed consolidated statements of convertible preferred stock and stockholders’ equity for the three months ended March 31, 2025 and 2024 and the condensed consolidated statements of cash flows for the three months ended March 31, 2025 and 2024 and the related footnote disclosures are unaudited. In management's opinion, the unaudited condensed consolidated financial statements have been prepared on the same basis as the audited consolidated financial statements as of and for the year ended December 31, 2024 and include all adjustments, which are all normal recurring adjustments, necessary for the fair presentation of the interim financial statements. The results for the three months ended March 31, 2025 are not necessarily indicative of the results expected for the full fiscal year, any other interim periods, or any future year or period. The accompanying unaudited condensed consolidated financial statements reflect the application of certain significant accounting policies as described below and elsewhere in these notes to the unaudited condensed consolidated financial statements. As of March 31, 2025, there have been no material changes in the Company's significant accounting policies from those that were disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements if these results differ from historical experience, or other assumptions do not turn out to be substantially accurate, even if such assumptions are reasonable when made. Significant estimates relied upon in preparing these financial statements include estimates related to determining our net product revenue and accruals related to research and development expenses. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ materially from those estimates. Principles of Consolidation The consolidated financial statements include the accounts of Rhythm Pharmaceuticals, Inc. and its wholly-owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation. 8 Table of Contents Segment and Geographic Information Disclosure requirements about segments of an enterprise and related information establishes standards for reporting information regarding operating segments in annual financial statements and requires selected information of those segments to be presented in interim financial reports issued to shareholders. Operating segments are defined as components of an enterprise about which separate discrete financial information is available that is evaluated regularly by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company’s chief operating decision maker is the chief executive officer. The Company and the chief executive officer view the Company’s operations and manage its business as one operating segment. In November 2023, the FASB issued ASU 2023-07 – Segment Reporting (Topic 280) – Improvements to Reportable Segment Disclosures, which improves segment disclosure requirements, primarily through enhanced disclosure requirements for significant segment expenses. The improved disclosure requirements apply to all public entities that are required to report segment information, including those with only one reportable segment. The Company adopted the guidance in the fiscal year beginning January 1, 2024. There was no impact on the Company’s reportable segments identified and additional required disclosures have been included in Note 15. ​ Off-Balance Sheet Risk and Concentrations of Credit Risk Financial instruments, which potentially subject the Company to significant concentration of credit risk, consist primarily of cash and cash equivalents and short-term investments, which are maintained at two federally insured financial institutions. The deposits held at these two institutions are in excess of federally insured limits. The Company has not experienced any losses in such accounts and management believes that the Company is not exposed to significant credit risk due to the financial position of the depository institutions in which those deposits are held. The Company has no off-balance sheet risk, such as foreign exchange contracts, option contracts, or other foreign hedging arrangements. The Company is exposed to risks associated with extending credit to customers related to the sale of products. The Company does not require collateral to secure amounts due from its customers. For the three months ended March 31, 2025 and 2024, approximately 65 % and 74 % of all of the Company’s revenue was generated from a single customer in the United States. As of March 31, 2025 and December 31, 2024, approximately 61 % and 67 %, respectively, of the Company’s accounts receivable was outstanding from a single customer in the United States. The Company relies on third-party manufacturers and suppliers for the manufacture and supply of its product. The inability of the suppliers or manufacturers to fulfill supply requirements of the Company could materially impact future operating results. A change in the relationship with the suppliers or manufacturer, or an adverse change in their business, could materially impact future operating results. The Company relies on separate third parties to perform genetic testing in the United States and Europe, respectively. The inability of the vendors to fulfill testing services for the Company could materially impact future operating results and adversely impact our ability to further develop setmelanotide. A change in the relationship with the genetic testing service providers, or an adverse change in their business, could materially impact future operating results. Cash and Cash Equivalents The Company considers all highly liquid investments with remaining maturity from the date of purchase of three months or less to be cash equivalents. Cash and cash equivalents includes bank demand deposits, U.S. treasury bills and money market funds that invest primarily in U.S. government treasuries. Short-Term Investments Short-term investments consist of investments with maturities greater than 90 days, as of the date of purchase. The Company has classified its investments with maturities beyond one year as short term, based on their highly liquid nature and because such marketable securities represent the investment of cash that is available for current operations. The 9 Table of Contents Company considers its investment portfolio available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices. Unrealized gains and losses are reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity. To the extent the amortized cost basis of the available-for-sale debt securities exceeds the fair value, management assesses the debt securities for credit loss; however, management considers the risk of credit loss to be minimized by the Company's policy of investing in financial instruments issued by highly-rated financial institutions. When assessing the risk of credit loss, management considers factors such as the severity and the reason for the decline in value (i.e., any changes to the rating of the security by a rating agency or other adverse conditions specifically related to the security) and management's intended holding period and time horizon for selling. During the three months ended March 31, 2025 and 2024, the Company did no t recognize any credit losses related to its available-for-sale debt securities. Further, as of March 31, 2025 and December 31, 2024, the Company did no t record an allowance for credit losses related to its available-for-sale debt securities. Accounts Receivable, net Accounts receivable consists of amounts due from customers, net of customer allowances for cash discounts and any estimated expected credit losses. The Company's measurement of expected credit losses is based on relevant information about past events, including historical experience, current conditions, and reasonable and supportable forecasts that affect the collectability of the reported amount. To date, the Company has not experienced any credit losses. The Company's contracts with its customers have customary payment terms that generally require payment within 90 days. The Company analyzes amounts that are past due for collectability, and periodically evaluates the creditworthiness of its customers. As of March 31, 2025 and December 31, 2024, the Company determined an allowance for doubtful accounts was not required based upon our review of contractual payments and our customers’ circumstances. Revenue Recognition The Company recognizes revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers , or ASC 606. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. Product Revenue, net In the United States (the “U.S.”), which accounts for the largest portion of our total revenues, the Company sells its product to one specialty pharmacy. The product is distributed through third-party logistics, or 3PL, distribution agent that does not take title to the product. Once the product is delivered to the Company’s specialty pharmacy provider, our customer in the U.S., the customer (or “wholesaler”) takes title to the product. The wholesaler then distributes the product to patients. In our distribution agreement with the 3PL company, the Company acts as principal because we retain control of the product. Internationally, we make sales primarily to specialty distributors and retail pharmacy chains, as well as hospitals, many of which are government-owned or supported. The Company offers returns of product sold to the customer on a limited basis, however, no material returns have been recognized to date. Revenue from product sales is recognized when the customer obtains control of our product, which occurs at a point in time, upon transfer of title to the customer because at that point in time we have no ongoing obligations to the customer. There are no other performance obligations besides the sale of product. We classify payments to our customers or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses in our consolidated statements of operations and comprehensive loss. Otherwise, payments to a customer or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. Taxes collected from the customer relating to product sales and remitted to governmental authorities are excluded from revenue. Because our payment terms are generally ninety days or less, the Company concluded there is not a significant financing component because the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred since the expected amortization period of the asset that we would have recognized is one year or less. 10 Table of Contents Reserves for Variable Consideration Revenues from product sales are recorded at the net sales price, or the transaction price, which includes estimates of variable consideration for which reserves are established and which result from discounts, rebates, and co-pay assistance that are offered within contracts between us and our customers, health care providers and other indirect customers relating to the sale of IMCIVREE. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. The following are the components of variable consideration related to product revenue: Government rebates: The Company is subject to discount obligations under government programs, including Medicaid programs, Medicare and Tricare in the United States as well as certain government rebates and pricing adjustments in certain international markets that we operate. We estimate these rebates based upon a range of possible outcomes that are probability-weighted for the estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses and other current liabilities on our condensed consolidated balance sheets. On a quarterly basis, we update our estimates and record any adjustments in the period that we identify the adjustments. Trade discounts and allowances: The Company provides customary invoice discounts on IMCIVREE sales to certain of our customers for prompt payment. These are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive and pay for various distribution services from our customers in the distribution channel. For services that are not distinct from the sale of our product, such fees are classified as a reduction of product revenue. Product returns: Our customers have limited return rights related to the product’s damage or defect. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. Based on the distribution model for IMCIVREE, the Company believes there will be minimal returns and these reserves have not been material to date. Other incentives: Other incentives include co-payment assistance the Company provides to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. The estimate is recorded as a reduction of revenue in the same period the related revenue is recognized. Provisions for trade discounts, and allowances are recorded as reductions to accounts receivable, and returns, government rebates, and other incentives are recorded as a component of accrued expenses. License Agreements We generate revenue from license or similar agreements with pharmaceutical companies for the development and commercialization of certain of our products and product candidates. Such agreements may include the transfer of intellectual property rights in the form of licenses, transfer of technological know-how, delivery of drug substances, research and development services, and participation on certain committees with the counterparty. Payments made by the customers may include non-refundable upfront fees, payments upon the exercise of customer options, payments 11 Table of Contents based upon the achievement of defined milestones, and royalties on sales of products and product candidates if they are approved and commercialized. ​ If a license to our intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, we recognize the transaction price allocated to the license as revenue upon transfer of control of the license. We evaluate all other promised goods or services in the agreement to determine if they are distinct. If they are not distinct, they are combined with other promised goods or services to create a bundle of promised goods or services that is distinct. Optional future services where any additional consideration paid to us reflects their standalone selling prices do not provide the customer with a material right and, therefore, are not considered performance obligations. If optional future services are priced in a manner which provides the customer with a significant or incremental discount, they are material rights, and are accounted for as separate performance obligations. ​ We utilize judgment to determine the transaction price. In connection therewith, we evaluate contingent milestones at contract inception to estimate the amount which is not probable of a material reversal to include in the transaction price using the most likely amount method. Milestone payments that are not within our control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received and therefore the variable consideration is constrained. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which we recognize revenue as or when the performance obligations under the contract are satisfied. At the end of each reporting period, we re-evaluate the probability of achieving development milestone payments that may not be subject to a material reversal and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect license and other revenue, as well as earnings, in the period of adjustment. ​ We then determine whether the performance obligations or combined performance obligations are satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. We evaluate the measure of progress, as applicable, for each reporting period and, if necessary, adjust the measure of performance and related revenue recognition. ​ When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract liability is recorded within deferred revenue. Contract liabilities within deferred revenue are recognized as revenue after control of the goods or services is transferred to the customer and all revenue recognition criteria have been met. ​ For arrangements that include sales-based royalties, including sales-based milestone payments, and a license of intellectual property that is deemed to be the predominant item to which the royalties relate, we recognize revenue at the later of when the related sales occur or when the performance obligation to which some or all of the royalties have been allocated has been satisfied (or partially satisfied). See Note 12 – Significant Agreements for discussion related to the Company’s accounting for the RareStone Group, Ltd. agreement. ​ Deferred Royalty Obligation The Company treats the debt obligation to HealthCare Royalty Management, LLC as discussed further in Note 13, “Long-Term Obligations”, as a deferred royalty obligation, amortized using the effective interest rate method over the estimated life of the revenue streams. The Company recognizes interest expense thereon using the effective rate, which is based on our current estimates of future revenues over the life of the arrangement. In connection therewith, the Company periodically assesses its expected revenues using internal projections, imputes interest on the carrying value of the deferred royalty obligation, and records interest expense using the imputed effective interest rate. To the extent the Company’s estimates of future revenues are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, the Company will account for any such changes by adjusting the effective interest rate on a prospective basis, with a corresponding impact to the reclassification of our deferred royalty obligation. The assumptions used in determining the expected repayment term of the deferred royalty obligation and amortization period of the issuance costs requires the Company to make estimates that could impact the classification of such costs, as well as the period over which such costs will be amortized. 12 Table of Contents Inventory Prior to receiving approval from the FDA in November 2020 to sell IMCIVREE in the United States, the Company expensed all costs incurred related to the manufacture of IMCIVREE as research and development expense because of the inherent risks associated with the development of a drug candidate, the uncertainty about the regulatory approval process and the lack of history for the Company of regulatory approval of drug candidates. The Company values inventories at the lower of cost or estimated net realizable value. The Company determines the cost of inventories, which includes amounts related to materials and manufacturing overhead, on a first-in, first-out basis. Raw materials and work in process includes all inventory costs prior to packaging and labelling, including raw materials, active pharmaceutical ingredient, and drug product. Finished goods include packaged and labelled products. Raw materials and work in process that may be used for either research and development or commercial sale are classified as inventory until the material is consumed or otherwise allocated for research and development. If the material is intended to be used for research and development, it is expensed as research and development once that determination is made. Cost of Product Sales Cost of product sales consists of manufacturing costs, transportation and freight, amortization of capitalized intangibles, royalty payments and indirect overhead costs associated with the manufacturing and distribution of IMCIVREE. Cost of product sales may also include periodic costs related to certain manufacturing services and inventory adjustment charges. Finally, cost of sales may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, and manufacturing variances. Intangible Assets, Net Definite-lived intangible assets related to capitalized milestones under license agreements are amortized on a straight-line basis over their remaining useful lives, which are estimated to be the remaining patent life. If our estimate of the product’s useful life is shorter than the remaining patent life, then a shorter period is used. Amortization expense is recorded as a component of cost of sales on the consolidated statements of operations and comprehensive loss. Impairment of Long-Lived Assets The Company evaluates its long-lived assets, which consist primarily of property and equipment and finite lived intangible assets, for impairment whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. The Company measures recoverability of assets to be held and used by comparing the carrying amount of an asset to the future undiscounted net cash flows expected to be generated by the asset. If such assets are considered to be impaired, the Company measures the impairment to be recognized by the amount by which the carrying amount of the asset exceeds the fair value of the asset, less the cost to sell. No events or changes in circumstances existed to require an impairment assessment during the three months ended March 31, 2025 and 2024, respectively. Acquired IPR&D and Milestone Expenses ​ In an asset acquisition, payments incurred prior to regulatory approval to acquire rights to in-process research and development projects are expensed as acquired IPR&D and recorded as a component of research and development expense in the condensed consolidated statements of operations and comprehensive net loss unless the project has an alternative future use. These costs include upfront and development milestone payments related to licensing arrangements, or other asset acquisitions that provide rights to develop, manufacture and/or sell pharmaceutical products. Where contingent development milestone payments are due to third parties, prior to regulatory approval, the payment obligations are expensed when the achievement of the underlying milestone becomes probable. Regulatory and commercial milestone payments made to third parties subsequent to regulatory approval are capitalized as intangible assets and amortized to cost of products sold over the remaining useful life of the related product. 13 Table of Contents Foreign Currency Translation ​ The assets and liabilities of the Company’s subsidiaries with functional currencies other than the U.S. dollar are translated into U.S. dollars at exchange rates in effect at the balance sheet date. Revenue and expense amounts for these subsidiaries are translated using the average exchange rates for the period. Changes resulting from foreign currency translation are included in accumulated other comprehensive income (loss) on the Company’s consolidated statement of stockholders’ equity. Net foreign currency exchange transaction gains (losses), which are included in other (expense) income, net on our consolidated statements of operations, were immaterial for the three months ended March 31, 2025 and 2024, respectively. Fair Value Measurements Fair value is 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. Financial assets and liabilities carried at fair value are classified and disclosed in one of the following three categories: Level 1 — Quoted market prices in active markets for identical assets or liabilities. Level 2 — Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. The Company’s cash equivalents and marketable securities and derivative asset as of March 31, 2025 and December 31, 2024 were carried at fair value, determined according to the fair value hierarchy. See Note 6 for further discussion. The carrying amounts reflected in the condensed consolidated balance sheets for accounts payable and accrued expenses and other current liabilities approximate their fair values due to their short-term maturities as of March 31, 2025 and December 31, 2024, respectively. ​ Net Loss Per Share Basic net loss per share is computed by dividing the net loss attributable to common shareholders by the weighted average number of common shares outstanding during the period, without consideration of potential dilutive securities. Diluted net loss per common share is computed by adjusting the weighted average shares outstanding for the potential dilutive effects of common stock equivalents outstanding during the period calculated in accordance with the more dilutive of the if-converted or the treasury stock method. For purposes of the diluted net loss per share calculation, stock options, performance stock units and restricted stock units are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, basic and diluted net loss per share is the same for all periods presented. 14 Table of Contents The following table includes the potential common shares that were excluded from the computation of diluted net loss per share as their effect would have been anti-dilutive for the periods indicated: ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ March 31, ​ 2025 2024 Stock options ​ 7,157,931 ​ ​ 7,029,546 Restricted stock units 2,704,837 ​ 1,775,146 Performance stock units ​ 249,322 ​ ​ — Common stock reserved for the conversion of Series A convertible preferred stock ​ 3,124,999 ​ ​ — Potential common shares ​ 13,237,089 ​ ​ 8,804,692 ​ Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. See Note 16. Application of New or Revised Accounting Standards From time to time, new accounting pronouncements are issued by the FASB and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its financial position or results of operations upon adoption. In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures , or ASU 2023-09. The new guidance requires that an entity, on an annual basis, disclose additional income tax information, primarily related to the rate reconciliation and income taxes paid. The amendments in the ASU are intended to enhance the transparency and decision usefulness of income tax disclosures. The amendments in this update are effective for us beginning in fiscal year ending December 31, 2025. We are currently evaluating the impact of the new standard on our consolidated financial statements which is expected to result in enhanced disclosures, however, we do not otherwise expect the adoption of the new guidance to have a material impact on our financial condition or results of operations. In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The standard requires disclosure of incremental segment information on an annual and interim basis and allows for multiple measures of a segment’s profit or loss provided that one of those measures is consistent with GAAP. The amendments in this update do not change how a public company identifies its operating segments, aggregates those operating segments, or applies the quantitative thresholds to determine its reportable segments, but rather requires public entities to provide in interim periods all disclosures about a reporting segment’s profit or loss and assets that are currently required annually. ASU 2023-07 becomes effective for the annual period starting on January 1, 2024, and for interim periods starting on January 1, 2025. The company adopted this standard in the year ending December 31, 2024. 3. Asset Acquisitions LG Chem, Ltd. On January 4, 2024, the Company entered into a license agreement and share issuance agreement with LG Chem, Ltd. (“LGC”). Under the terms of the license agreement, the Company obtained worldwide rights to LGC’s proprietary compound bivamelagon and assumed sponsorship of two ongoing LGC Phase 2 studies designed to evaluate safety, tolerability, pharmacokinetics and weight loss efficacy of bivamelagon. ​ 15 Table of Contents The total purchase consideration of $ 92.4 million was composed of $ 40.0 million of cash paid at closing and issued shares of the Company’s common stock with an aggregate value of $ 20.0 million. The shares were issued at a per share price equal to the ten -day volume weighted average closing price for our common stock, calculated as of the trading day immediately prior to January 4, 2024. As of January 4, 2024, the fair value of common stock issued was $ 18.7 million. The total purchase consideration also includes an additional $ 40.0 million license fee payable in 18 months , whose present value at closing was $ 33.7 million, and $ 0.8 million of transaction costs which are recorded as selling, general and administrative expenses. The carrying value of the license fee payable is $ 38.8 million as of March 31, 2025 and is reflected in current liabilities on our condensed consolidated balance sheet. ​ In addition, under the terms of the license agreement, we agreed to pay LGC up to $ 205 million in cash upon achieving various regulatory and sales milestones based on net sales of bivamelagon. In addition, and subject to the completion of Phase 2 development of bivamelagon, the Company has agreed to pay LGC royalties of between low-to-mid single digit percent of net revenues from its MC4R portfolio, including bivamelagon, commencing in 2029 and dependent upon achievement of various regulatory and indication approvals, and subject to customary deductions and anti-stacking. Royalties may further increase to a low double digit percent royalty, though such royalty would only be applicable on net sales of bivamelagon in a region if bivamelagon is covered by a composition of matter or method of use patent controlled by LGC in such region and the Company’s MC4R portfolio is not covered by any composition of matter or method of use patents controlled by the Company in such region. Such increased rate would only apply on net sales of bivamelagon for the limited remainder of the royalty term in the relevant region. ​ The assets acquired were In-Process Research and Development (“IPR&D”) assets. However, since the IPR&D assets were determined to have no alternative future use, the Company recognized the $ 92.4 million of purchase consideration as research and development expense in the three months ended March 31, 2024. The Company determined that the additional contingent consideration did not meet the definition of a derivative as of the acquisition date. Therefore, the Company did not record a contingent consideration liability on the acquisition date. The Company will recognize any future contingent consideration payments related to the LGC transaction in the period in which the achievement of the underlying milestones becomes probable. ​ 4. Inventory Inventory consists of the following: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2025 2024 Raw Materials ​ $ 5,027 ​ $ 6,776 WIP ​ 1,689 ​ 1,250 Finished Goods ​ 12,600 ​ 10,715 Total Inventory ​ $ 19,316 ​ $ 18,741 ​ ​ 16 Table of Contents 5. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consisted of the following: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2025 2024 Research and development costs ​ $ 19,919 ​ $ 17,871 Professional fees ​ 4,735 ​ 4,280 Payroll related ​ 8,586 ​ 18,216 Royalties ​ ​ 1,890 ​ ​ 2,091 Sales allowances ​ ​ 18,383 ​ ​ 15,850 Other ​ 5,801 ​ 4,350 Accrued expenses and other current liabilities ​ $ 59,314 ​ $ 62,658 ​ ​ ​ ​ ​ ​ ​ ​ ​ 6. Fair Value of Financial Assets and Liabilities As of March 31, 2025 and December 31, 2024, the carrying amount of cash and cash equivalents and short-term investments was $ 314,488 and $ 320,565 respectively, which approximates fair value. Cash and cash equivalents and short-term investments includes investments in U.S. treasury securities and money market funds that invest in U.S. government securities that are valued using quoted market prices. Accordingly, money market funds and government funds are categorized as Level 1. The financial assets valued based on Level 2 inputs consist of corporate debt securities and commercial paper, which consist of investments in highly-rated investment-grade corporations. The following tables present information about the Company's financial assets measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair value: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Fair Value Measurements as of ​ ​ March 31, 2025 using: ​ Level 1 Level 2 Level 3 Total Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash equivalents: ​ ​ ​ ​ Money market funds ​ $ 89,850 ​ $ — ​ $ — ​ $ 89,850 Marketable securities: ​ ​ ​ ​ US treasury securities ​ ​ 58,327 ​ ​ — ​ ​ — ​ ​ 58,327 Corporate debt securities and commercial paper ​ ​ — ​ ​ 150,066 ​ ​ — ​ ​ 150,066 Derivative asset ​ ​ — ​ ​ — ​ ​ 190 ​ ​ 190 Total ​ $ 148,177 ​ $ 150,066 ​ $ 190 ​ $ 298,433 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Fair Value Measurements as of ​ ​ December 31, 2024 using: ​ Level 1 Level 2 Level 3 Total Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash equivalents: ​ ​ ​ ​ Commercial Paper ​ $ — ​ $ 2,984 ​ $ — ​ $ 2,984 Money market funds ​ ​ 71,334 ​ ​ — ​ ​ — ​ ​ 71,334 Marketable securities: ​ ​ ​ ​ US treasury securities ​ ​ 65,118 ​ ​ — ​ ​ — ​ ​ 65,118 Corporate debt securities and commercial paper ​ ​ — ​ ​ 166,310 ​ ​ — ​ ​ 166,310 Derivative asset ​ ​ — ​ ​ — ​ ​ 270 ​ ​ 270 Total ​ $ 136,452 ​ $ 169,294 ​ $ 270 ​ $ 306,016 ​ 17 Table of Contents The estimated fair value of the derivative asset relates to our Royalty Interest Financing Agreement (RIFA) with HealthCare Royalty Partners was determined using Level 3 inputs. The fair value measurement of the derivative asset is sensitive to changes in the unobservable inputs used to value the financial instrument. Changes in the inputs could result in changes to the fair value of each financial instrument. The embedded derivative asset associated with our deferred royalty obligation, as discussed further in Note 13, “Long-Term Obligations”, is measured at fair value using an option pricing Monte Carlo simulation model and is included as a component of the deferred royalty obligation on the condensed consolidated balance sheets. The embedded derivative asset is subject to remeasurement at the end of each reporting period, with changes in fair value recognized as a component of other (expense) income, net. The assumptions used in the option pricing Monte Carlo simulation model include: (1) our estimates of the probability and timing of related events; (2) the probability-weighted net sales of IMCIVREE, including worldwide net product sales, upfront payments, milestones and royalties; (3) our risk-adjusted discount rate that includes a company specific risk premium; (4) our cost of debt; (5) volatility; and (6) the probability of a change in control occurring during the term of the instrument. The following tables set forth a summary of the changes in the estimated fair value of our embedded derivative liability (asset) (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended ​ ​ March 31, ​ 2025 ​ 2024 Beginning aggregate estimated fair value of Level 3 liability (asset) ​ $ ( 270 ) ​ $ 1,150 Change in fair value of embedded derivative ​ ​ 80 ​ ​ ( 490 ) Ending aggregate estimated fair value of Level 3 liability (asset) ​ $ ( 190 ) ​ $ 660 ​ Marketable Securities The following tables summarize the Company's marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, 2025 ​ ​ ​ ​ ​ Gross ​ Gross ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair ​ Cost Gains Losses Value Assets ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Corporate debt securities and commercial paper (due within 1 year) ​ $ 150,037 ​ $ 57 ​ $ ( 28 ) ​ $ 150,066 U.S. Treasury Securities ​ ​ 58,268 ​ ​ 72 ​ ​ ( 13 ) ​ ​ 58,327 ​ ​ $ 208,305 ​ $ 129 ​ $ ( 41 ) ​ $ 208,393 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2024 ​ ​ ​ ​ ​ Gross ​ Gross ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair ​ Cost Gains Losses Value Assets ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Corporate debt securities and commercial paper (due within 1 year) ​ $ 166,255 ​ $ 147 ​ $ ( 92 ) ​ $ 166,310 U.S. Treasury Securities ​ ​ 65,074 ​ ​ 79 ​ ​ ( 35 ) ​ ​ 65,118 ​ ​ $ 231,329 ​ $ 226 ​ $ ( 127 ) ​ $ 231,428 ​ ​ ​ ​ ​ ​ 18 Table of Contents 7. Intangible Assets ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of March 31, 2025 ​ As of December 31, 2024 ​ Estimated life (years) Cost Accumulated Amortization Net Cost Accumulated Amortization Net ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Capitalized Milestones ​ 11 ​ $ 9,000 ​ $ ( 3,040 ) ​ $ 5,960 ​ $ 9,000 ​ $ ( 2,826 ) ​ $ 6,174 ​ As of March 31, 2025, the Company’s finite-lived net intangible assets, which totaled $ 5,960 , resulted from the capitalization of certain milestone payments made to Ipsen Pharma, S.A.S., or Ipsen, in accordance with the terms of the Company’s license agreement with Ipsen, in connection with the Company’s first commercial sale of IMCIVREE in the U.S. in March 2021 and in France in March 2022. As of March 31, 2025, amortization expense for the next five years and beyond is summarized as follows: ​ ​ ​ ​ ​ 2025 ​ $ 641 2026 ​ ​ 855 2027 ​ ​ 855 2028 ​ ​ 855 2029 ​ ​ 855 Thereafter ​ 1,899 Total ​ $ 5,960 ​ Amortization expense totaled $ 214 and $ 214 for the three months ended March 31, 2025 and 2024, respectively. Amortization expense is included in cost of sales in the condensed consolidated statements of operations and comprehensive loss. ​ 8. Income Taxes The Company recorded an income tax provision of approximately $ 80 and $ 300 for the three months ended March 31, 2025 and 2024, respectively. The income tax provision is a result of taxable income from the Company’s foreign jurisdictions. The Company expects to maintain a full valuation allowance against its net deferred tax assets for the year ended December 31, 2025. 9. Series A Convertible Preferred Stock On April 1, 2024, the Company entered into an Investment Agreement (the “Investment Agreement”) with certain affiliates of Perceptive Advisors LLC (“Perceptive”) and certain other investors (each, an “Investor” and collectively, the “Investors”), relating to the issuance and sale of 150,000 shares of a new series of the Company’s Series A Convertible Preferred Stock, par value $ 0.001 per share, titled the “Series A Convertible Preferred Stock” (the “Convertible Preferred Stock”), for an aggregate purchase price of $ 147,750 , net of $ 2,250 of issuance costs, or $ 1,000 per share (the “Issuance”). The Issuance closed on April 15, 2024. The Company determined the obligation to issue 150,000 shares of Convertible Preferred Stock to Perceptive and Investors in the future at a set price represented a forward contract which was required to be accounted for at fair value. The fair value of the forward contract was measured as the difference between the fair value of the Convertible Preferred Stock, as determined using a binomial lattice valuation model, and the consideration payable to the Company. The assumptions used in the binomial lattice model include: (1) the Company’s common stock price on the issuance and settlement dates; (2) the Conversion Price as of $ 48.00 as per the Agreement; (3) a 20-year term to maturity; (4) an estimate of the Company’s credit risk-adjusted discount rate; and (5) volatility. The fair value of the forward contract upon issuance was determined to be $ 0 . Upon closing, the value of the forward contract was determined to be $ 8,900 and the fair value of the Convertible Preferred Stock was determined to be $ 141,100 . 19 Table of Contents The Company classifies its Convertible Preferred Stock outside of stockholders’ equity as the redemption of such shares is outside the Company’s control. The Company did not adjust the carrying values of the Convertible Preferred Stock to redemption value as the shares are not probable of becoming redeemable as of March 31, 2025. The Convertible Preferred Stock has the following rights and privileges: Liquidation: The Convertible Preferred Stock will rank senior to the Company’s common stock with respect to the distribution of assets upon the Company’s liquidation, dissolution or winding up. Upon any liquidation, dissolution or winding-up of the Company, whether voluntary or involuntary (“Liquidation”), each holder of Convertible Preferred Stock shall be entitled to receive payment for the greater of (i) 1.75 multiplied by the sum of the Liquidation Preference (i.e., Initial Liquidation Preference of $ 1,000 per share plus Paid-in-Kind (“PIK”) Dividends) plus unpaid Regular Dividends (to the extent such accumulated and unpaid Regular Dividends are not included in such Liquidation Preference) or (ii) the amount such holder would have received if the Convertible Preferred Stock were fully converted to common stock. If the assets available for distribution are not sufficient to pay the holders of the Convertible Preferred Stock pursuant to the preceding sentence, the assets will be distributed ratably to the holders of the Convertible Preferred Stock. Voting: Holders of the Convertible Preferred Stock have the right to vote with the holders of common stock on each matter submitted for a vote on an as-converted basis, subject to the terms of the Convertible Preferred Stock as specified in the Amended and Restated Certificate of Designations. The holders of the Convertible Preferred Stock shall also have certain protective voting rights. Specifically, as long as the Convertible Preferred Stock are outstanding, each of the following events require at least a two thirds affirmative vote of the Convertible Preferred Stock holders: (a) any amendment or modification of the Certificate of Incorporation to authorize or create, or to increase the authorized number of shares of, any class or series of Dividend Parity Stock, Liquidation Parity Stock, Dividend Senior Stock or Liquidation Senior Stock, (b) any amendment, modification, repeal or waiver of any provision of the Certificate of Incorporation or the Amended and Restated Certificate of Designations that adversely affects the rights, preferences, privileges or powers of the Convertible Preferred Stock, (c) increase or decrease the number of authorized shares of Convertible Preferred Stock or issue additional shares of Convertible Preferred Stock, (d) the Company’s consolidation or combination with, or merger with or into, another Person, or any binding or statutory share exchange or involving the Convertible Preferred Stock, in each case unless: (i) the Convertible Preferred Stock either (x) remains outstanding after such consolidation, combination, merger, share exchange or reclassification; or (y) is converted or reclassified into, or is exchanged for, or represents solely the right to receive, preference securities of the continuing, resulting or surviving Person of such consolidation, combination, merger, share exchange or reclassification, or the parent thereof; (ii) the Convertible Preferred Stock that remains outstanding or such preference securities, as applicable, have rights, preferences and voting powers that, taken as a whole, are not materially less favorable to the Holders or the holders thereof, as applicable, than the rights, preferences and voting powers, taken as a whole, of the Convertible Preferred Stock immediately before the consummation of such consolidation, combination, merger, share exchange or reclassification; and (iii) the issuer of the Convertible Preferred Stock that remains outstanding or such preference securities, as applicable, is a corporation duly organized and existing under the laws of the United States of America, any State thereof or the District of Columbia that, if not the Company, will succeed to the Company under the Amended and Restated Certificate of Designations and the Convertible Preferred Stock. Redemption: The Company has the right to redeem all Convertible Preferred Stock after the Redemption Trigger Date, which is the fifth anniversary of the Initial Issue Date of April 15, 2024. The amount payable on the redemption date is equal to the Liquidation Preference (i.e., Initial Liquidation Preference of $ 1,000 per share plus PIK Dividends) plus any unpaid 20 Table of Contents Regular Dividends (to the extent such accumulated and unpaid Regular Dividends are not included in such Liquidation Preference). If a change of control occurs, each holder shall have the right to require the Company to repurchase all, or any whole number of shares that is less than all, of the holder’s Convertible Preferred Stock at an amount equal to 1.75 multiplied by the sum of the Liquidation Preference (i.e., Initial Liquidation Preference of $ 1,000 per share plus PIK Dividends) plus any unpaid Regular Dividends (to the extent such accumulated and unpaid Regular Dividends are not included in such Liquidation Preference). As of March 31, 2025, the Company did not adjust the carrying value of the Convertible Preferred Stock to its redemption value, since a change of control was determined to not be probable. Dividends: After the second anniversary, dividends on the Convertible Preferred Stock accrue quarterly, at a 6 % annual rate, and if not paid out in cash before the quarter end, will become PIK Dividends and added to the liquidation preference, or original issue price plus PIK Dividends. Since dividends do not commence until the second anniversary of the Issuance, the Convertible Preferred Stock is considered increasing rate preferred stock. Accordingly, the Company accretes the dividends, using the effective interest method, from Issuance to the first contractual call date, April 15, 2029. The Company accrued dividends of $ 1,322 for the three months ended March 31, 2025, as a reduction to Additional Paid-In Capital and an increase to the carrying value of Convertible Preferred Stock. The carrying value of Convertible Preferred Stock as of March 31, 2025 is $ 144,142 . Conversions: Holders of Convertible Preferred Stock have the option to convert any number of whole shares at any time. The conversion is based on the sum of the Liquidation Preference plus unpaid Dividends divided by the $ 48.00 Conversion Price. Given the Initial Liquidation Preference of $ 1,000 , each share of Convertible Preferred Stock would be convertible into 20.8333 shares of common stock, prior to any adjustments such as PIK Dividends, unpaid Dividends, stock splits, or voluntary conversion rate increases. Upon conversion, cash will be paid in lieu of any fractional share of common stock. However, based on certain restrictions on the conversion of the Convertible Preferred Stock specified in the Amended and Restated Certificate of Designations, a holder of Convertible Preferred Stock is not entitled to effect a conversion of any portion of its shares of Convertible Preferred Stock, or to vote in its capacity as a holder of shares of Convertible Preferred Stock with respect to matters submitted to holders of the common stock if, after giving effect to such conversion, that holder would beneficially own in excess of 4.99 %, in the case of one holder, or 9.99 %, in the case of the other holder, of the number of shares of common stock outstanding immediately after giving effect to such exercise. ​ On May 7, 2024, the Company filed an Amended and Restated Certificate of Designations in respect of the Convertible Preferred Stock containing certain technical amendments to the terms of the Convertible Preferred Stock. The amendments contained in the Amended and Restated Certificate of Designations (x) limited the voting rights of the Convertible Preferred Stock to 24.9438 shares of the Company’s common stock per $ 1,000 liquidation preference of Convertible Preferred Stock and (y) eliminated a 1 % step up in the interest rate that otherwise would have applied in the unlikely event that the Company was required to obtain and failed to obtain stockholder approval for certain conversion shares underlying the Convertible Preferred Stock. On July 10, 2024, the Company filed with the Securities Exchange Commission (the “SEC”) a prospectus supplement to the prospectus included in the Company’s registration statement on Form S-3ASR filed with the SEC on March 2, 2023, covering the resale from time to time by the Investors of up to an aggregate of 3,124,995 shares of common stock, to satisfy registration rights that the Company granted to such stockholders in connection with the Issuance. ​ 10. Common Stock As of March 31, 2025, an aggregate of 14,096,737 shares of common stock were reserved for issuance under the Company’s stock plans, which includes stock options, restricted stock units, and performance stock units that have been 21 Table of Contents granted covering 10,112,090 shares of common stock, 150,000 shares of Convertible Preferred Stock potentially convertible into 3,125,000 shares of common stock, and 1,256,597 shares of common stock that remain available for future grants under the Company’s 2017 Equity Incentive Plan (the “2017 Plan”), 2017 Employee Stock Purchase Plan and 2022 Employment Inducement Plan (the “Inducement Plan”). On February 29, 2024, the Company and Cowen entered into Amendment No. 1 to Sales Agreement (the “Amendment”) to increase the aggregate offering price of the shares of common stock that may be issued and sold pursuant to the Sales Agreement to $ 200,000,000 (excluding the aggregate offering price of shares of common stock issued and sold pursuant to the Sales Agreement prior to February 29, 2024). In connection with the Amendment, on February 29, 2024, the Company filed with the SEC a prospectus supplement, dated February 29, 2024, which, combined with the Base Prospectus (together, the “New Prospectus”), amended the Prior Prospectus in its entirety. The issuances and sales under the Sales Agreement, as amended by the Amendment, will be made pursuant to the Registration Statement and the New Prospectus. Between December 10, 2024 and December 31, 2024, the Company sold 744,595 shares of common stock in the ATM Program for net proceeds of $ 41.2 million. Between January 1, 2025 and January 21, 2025, the Company sold an additional 587,510 shares of common stock in the ATM Program for net proceeds of approximately $ 32.1 million. On January 4, 2024, the Company issued 432,143 shares of common stock as partial consideration for its acquisition of the worldwide rights to LGC’s proprietary compound bivamelagon. On February 9, 2022, the Company’s board of directors adopted the Inducement Plan, without stockholder approval pursuant to Rule 5635(c)(4) of the Nasdaq Stock Market LLC listing rules or Rule 5635(c)(4). In accordance with Rule 5635(c)(4), awards under the Inducement Plan may only be made to a newly hired employee who has not previously been a member of the Company’s board of directors, or an employee who is being rehired following a bona fide period of non-employment by the Company or a subsidiary, as a material inducement to the employee’s entering into employment with the Company or its subsidiary. An aggregate of 1,000,000 shares of the Company’s common stock have been reserved for issuance under the Inducement Plan. The Company continues to grant awards under the 2017 Plan pursuant to the terms thereof. The exercise price of stock options granted under the Inducement Plan is not less than the fair market value of a share of the Company’s common stock on the grant date. Other terms of awards, including vesting requirements, are determined by the Company’s board of directors and are subject to the provisions of the Inducement Plan. Stock options granted to employees generally vest over a four-year period but may be granted with different vesting terms. Certain options may provide for accelerated vesting in the event of a change in control. Stock options granted under the Inducement Plan expire no more than 10 years from the date of grant. As of March 31, 2025, 556,100 stock option awards and 422,934 restricted stock unit awards have been granted under the Inducement Plan. As of March 31, 2025, 20,966 shares of common stock are available for future grant under the Inducement Plan. 11. Related-Party Transactions Expenses paid directly to related parties for the three months ended March 31, 2025 and 2024, were immaterial. Outstanding payments due to related parties as of March 31, 2025 and December 31, 2024 were also immaterial. ​ 12. Significant Agreements RareStone Group Ltd. ​ On March 14, 2025, we entered into a termination agreement (the “Termination Agreement”) with RareStone Group Ltd. (“RareStone”) and RareStone Medicine (Shenzhen) Co., Ltd. (“RareStone Shenzhen”), pursuant to which the Company, RareStone and RareStone Shenzhen have mutually agreed to terminate (i) the Exclusive License Agreement between the Company and RareStone, dated December 3, 2021 (the “License Agreement”); and (ii) the Share Purchase Agreement between the Company and RareStone, dated December 3, 2021 (the “Share Purchase Agreement”, and with the License Agreement the “RareStone Agreements”). 22 Table of Contents Under the Termination Agreement, the Company agreed to pay $ 6.3 million as a repayment of a portion of the upfront payment made pursuant to Section 7.1 of the License Agreement. In connection with the Termination Agreement, the Company and RareStone also entered into a Share Repurchase Agreement dated March 14, 2025, pursuant to which the Company has agreed to convey all of the shares acquired under the original Share Purchase Agreement back to RareStone, for no additional consideration. The Company had previously written off the value of the shares in the year ended December 31, 2022. Prior to executing the termination agreement, the Company had recorded $ 1.3 million of deferred revenue related to this arrangement for unsatisfied performance obligations. As a result of the Termination Agreement, the Company recognized the $ 6.3 million paid to RareStone as a reduction in previously-recognized license revenue as it represented consideration paid to a customer, and recognized all existing deferred revenue resulting in a net reduction in license revenue of $ 5.0 million. Pursuant to the Termination Agreement, the RareStone Agreements were terminated and all rights and obligations under such agreements ceased. In addition, each party to the Termination Agreement discharged and released the other parties, subsidiaries, divisions, affiliates, predecessors, successors, and each of their past and present officers, directors, employees, attorneys, agents, affiliates, assigns, and representatives of and from any and all claims, demands, actions, or causes of action, known or unknown, contingent or non-contingent, which the parties may or might have against them, by reason of any general, special, or consequential damages, losses, or potential losses, arising out of and/or relating to the RareStone Agreements. 13. Long-Term Obligations On June 16, 2022, we entered into a RIFA with entities managed by HealthCare Royalty Management, LLC, collectively referred to as the Investors. Pursuant to the RIFA and subject to customary closing conditions, the Investors have agreed to pay the Company an aggregate investment amount of up to $ 100,000 , or the Investment Amount. Under the terms of the RIFA, we received $ 37,500 on June 29, 2022 upon FDA approval of IMCIVREE in BBS, referred to as the Initial Investment Amount, and we received an additional $ 37,500 on September 29, 2022 of the Investment Amount upon EMA approval for BBS. On September 12, 2023, we received the remaining $ 24,370 of the Investment Amount, net of debt issuance costs, following the achievement of a specified amount of cumulative net sales of IMCIVREE between July 1, 2022 and September 30, 2023. As consideration for the Investment Amount and pursuant to the RIFA, we agreed to pay the Investors a tiered royalty on our annual net revenues, or Revenue Interest, including worldwide net product sales and upfront payments and milestones. The applicable tiered percentage will initially be 11.5 % on annual net revenues up to $ 125,000 , 7.5 % on annual net revenues of between $ 125,000 and $ 300,000 and 2.5 % on annual net revenues exceeding $ 300,000 . If the Investors have not received cumulative minimum payments equal to 60 % of the amount funded by the Investors to date by March 31, 2027, or 120 % of the amount funded by the Investors to date by March 31, 2029, we must make a cash payment immediately following each applicable date to the Investors sufficient to gross the Investors up to such minimum amounts after giving full consideration of the cumulative amounts paid by us to the Investors through each date, referred to as the Under Performance Payment. As the repayment of the funded amount is contingent upon worldwide net product sales and upfront payments, milestones, and royalties, the repayment term may be shortened or extended depending on actual worldwide net product sales and upfront payments, milestones, and royalties. We made repayments of $ 4,615 in the three months ended March 31, 2025. As of March 31, 2025 we have made cumulative payments of $ 25,045 . The Investors’ rights to receive the Revenue Interests will terminate on the date on which the Investors have received payments equal to a certain percentage of the funded portion of the Investment Amount including the aggregate of all payments made to the Investors as of such date, each percentage tier referred to as the Hard Cap, unless the RIFA is earlier terminated. The total Revenue Interests payable by us to the Investors is capped between 185 % and 250 % of the Investment Amount paid, dependent on the aggregate royalty paid between 2028 and 2032. If a change of control of occurs, the Investors may accelerate payments due under the RIFA up to the Hard Cap plus any other obligations payable under the RIFA. The repayment period commenced on July 8, 2022 for the Initial Investment Amount, and expires on the earlier of (i) the date at which the Investors received cash payments totaling an aggregate of a Hard Cap ranging from 185 % to 250 % of the Initial Investment Amount or (ii) the legal maturity date of July 8, 2034. If the Investors have not received payments equal to 250 % of the Investment Amount by the twelve-year anniversary of the initial closing date, we will be 23 Table of Contents required to pay an amount equal to the Investment Amount plus a specific annual rate of return less payments previously received by Investors. In the event of a change of control, we are obligated to pay Investors an amount equal to the Hard Cap in effect at the time, ranging from 185 % to 250 % plus any Under Performance Payment of the Investment Amount less payments previously received by Investors. In addition, upon the occurrence of an event of default, including, among others, our failure to pay any amounts due to Investors under the deferred royalty obligation, insolvency, our failure to pay indebtedness when due, the revocation of regulatory approval of IMCIVREE in the U.S. or our breach of any covenant contained in the RIFA and our failure to cure the breach within the prescribed time frame, we are obligated to pay Investors an amount equal to the Hard Cap in effect at the time of default ranging from 185 % to 250 % plus any Under Performance Payment of the Investment Amount less payments previously received by Investors. In addition, upon an event of default, Investors may exercise all other rights and remedies available under the RIFA, including foreclosing on the collateral that was pledged to Investors, which consists of all of our present and future assets relating to IMCIVREE. We have evaluated the terms of the RIFA and concluded that the features are similar to those of a debt instrument. Accordingly, we have accounted for the transaction as long-term debt and presented it as a deferred royalty obligation on our condensed consolidated balance sheets. We have further evaluated the terms of the RIFA and determined that the repayment of the Hard Cap in effect at the time which ranges from 185 % to 250 % of the Investment Amount, less any payments made to date, upon a change of control is an embedded derivative that requires bifurcation from the debt instrument and fair value recognition. We determined the fair value of the derivative using an option pricing Monte Carlo simulation model taking into account the probability of change of control occurring and potential repayment amounts and timing of such payments that would result under various scenarios, as further described in Note 2, “Summary of Significant Accounting Policies” to our condensed consolidated financial statements. The aggregate fair value of the embedded derivative asset (liability) was $ 190 and $ 270 as of March 31, 2025 and December 31, 2024, respectively. We will remeasure the embedded derivative to fair value each reporting period until the time the features lapse and/or termination of the deferred royalty obligation. For the three months ended March 31, 2025 and 2024, we recognized other income of ($ 80 ) and $ 490 , respectively, due to the remeasurement of the embedded derivative liability. The carrying value of the deferred royalty obligation as of March 31, 2025 was $ 109,524 based on $ 100,000 of proceeds, net of the fair value of the bifurcated embedded derivative liability upon execution of the RIFA, and debt issuance costs incurred. The carrying value is classified as $ 1,590 within current liabilities and $ 107,934 within long-term liabilities on the consolidated balance sheet as of March 31, 2025. The carrying value of the deferred royalty obligation approximated fair value as of March 31, 2025 and December 31, 2024. The effective interest rate as of March 31, 2025 was 15.99 %. In connection with the deferred royalty obligation, we incurred debt issuance costs totaling $ 3,287 . Debt issuance costs have been netted against the debt and are being amortized over the estimated term of the debt using the effective interest method, adjusted on a prospective basis for changes in the underlying assumptions and inputs. The assumptions used in determining the expected repayment term of the debt and amortization period of the issuance costs requires that we make estimates that could impact the short and long-term classification of these costs, as well as the period over which these costs will be amortized. ​ 14. Commitments and Contingencies Legal Proceedings ​ The Company, from time to time, may be party to various litigation arising in the ordinary course of business. The Company is not presently subject to any pending or threatened litigation that it believes, if determined adversely to the Company, individually, or taken together, would reasonably be expected to have a material adverse effect on its business or financial results. ​ Other ​ The Company is party to various agreements, principally relating to licensed technology, that require future payments relating to milestones whose achievement may become probable in subsequent periods, or royalties on future sales of specified products. Additionally, the Company is party to various contracts with CROs and CMOs that 24 Table of Contents generally provide for termination on notice, with the exact amounts in the event of termination to be based on the timing of the termination and the terms of the agreement. ​ Based on the Company’s current development plans as of March 31, 2025, the Company does not expect to make milestone payments due to third parties during the next 12 months from the filing of this Form 10-Q, in connection with our license agreements. These milestones are generally recognized in the period in which the achievement of the underlying milestones becomes probable. When the achievement of these milestones or sales have not occurred, such contingencies are not recorded in the Company’s consolidated financial statements. ​ 15. Segment and Geographic Information Operating segments are defined as components of an entity about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company currently operates in two business segments, which are U.S. and international segments for the development and commercialization of therapies for patients with rare diseases. A single management team that reports to the Chief Executive Officer comprehensively manages the entire business. The Company meets the aggregation criteria of ASC 280 and therefore has one reportable segment for the year ended March 31, 2025. The table below is a summary of the segment profit or loss, including significant segment expenses (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2025 ​ 2024 Net product revenue - U.S. ​ $ 24,369 ​ $ 19,443 Net product revenue - International ​ ​ 13,349 ​ ​ 6,524 Total net product revenue ​ ​ 37,718 ​ ​ 25,967 License revenue ​ ​ ( 5,014 ) ​ ​ - Total net revenue ​ ​ 32,704 ​ ​ 25,967 ​ ​ ​ ​ ​ ​ ​ Cost of sales ​ ​ 3,648 ​ ​ 2,807 Global headcount expense ​ ​ 34,868 ​ ​ 27,104 Preclinical, clinical and development expense ​ ​ 19,960 ​ ​ 21,954 Commercial & medical affairs ​ ​ 13,249 ​ ​ 12,927 Corporate, general & administrative ​ ​ 7,983 ​ ​ 8,677 Other segment expenses ​ ​ - ​ ​ 92,385 Other (income) expense, net ​ ​ 644 ​ ​ ( 524 ) Interest (income) expense, net ​ ​ 1,770 ​ ​ 1,709 Income taxes ​ ​ 80 ​ ​ 300 Net loss ​ $ ( 49,498 ) ​ $ ( 141,372 ) ​ Other segment expenses is composed of acquired in-process research and development costs associated with the acquisition of LGC’s proprietary compound bivamelagon in the three months ended March 31, 2024. ​ ​ ​ ​ ​ 25 Table of Contents Geographic Data The Company allocates, for the purpose of geographic data reporting, its revenue based upon the location of its customers. Total product revenue, net, by geographic area was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ 2025 ​ 2024 US ​ $ 24,369 ​ $ 19,443 International ​ ​ 13,349 ​ ​ 6,524 Total product revenue, net ​ $ 37,718 ​ $ 25,967 ​ ​ As of March 31, 2025 and December 31, 2024, long-lived assets at locations outside the United States were not material. ​ 16. Subsequent Events ​ The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. No events or transactions occurred subsequent to the balance sheet date that require disclosure. ​ ​