Edgewise Therapeutics, Inc. 10-K

Item 8. Financial Statements and Supplementary Data INDEX TO FINANCIAL STATEMENTS EDGEWISE THERAPEUTICS, INC. ​ ​ Page Report of Independent Registered Public Accounting Firm (KPMG LLP, Denver, CO, Auditor Firm ID: 185 ) 137 BALANCE SHEETS 139 STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS 140 STATEMENTS OF AND STOCKHOLDERS’ EQUITY 141 STATEMENTS OF CASH FLOWS 142 NOTES TO FINANCIAL STATEMENTS 143 136 Table of Contents Report of Independent Registered Public Accounting Firm To the Stockholders and Board of Directors Edgewise Therapeutics, Inc.: Opinions on the Financial Statements and Internal Control Over Financial Reporting We have audited the accompanying balance sheets of Edgewise Therapeutics, Inc. (the Company) as of December 31, 2025 and 2024, the related statements of operations and comprehensive loss, stockholders’ equity, and cash flows for each of the years in the three-year period ended December 31, 2025, and the related notes (collectively, the financial statements). We also have audited the Company’s internal control over financial reporting as of December 31, 2025, based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission. In our opinion, the financial statements referred to above present fairly, in all material respects, the financial position of the Company as of December 31, 2025 and 2024, and the results of its operations and its cash flows for each of the years in the three-year period ended December 31, 2025, in conformity with U.S. generally accepted accounting principles. Also in our opinion, the Company maintained, in all material respects, effective internal control over financial reporting as of December 31, 2025 based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission. Basis for Opinions The Company’s management is responsible for these financial statements, for maintaining effective internal control over financial reporting, and for its assessment of the effectiveness of internal control over financial reporting, included in the accompanying Management's Report on Internal Control Over Financial Reporting. Our responsibility is to express an opinion on the Company’s financial statements and an opinion on the Company’s internal control over financial reporting based on our audits. We are a public accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB. We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audits to obtain reasonable assurance about whether the financial statements are free of material misstatement, whether due to error or fraud, and whether effective internal control over financial reporting was maintained in all material respects. Our audits of the financial statements included performing procedures to assess the risks of material misstatement of the financial statements, whether due to error or fraud, and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the financial statements. Our audit of internal control over financial reporting included obtaining an understanding of internal control over financial reporting, assessing the risk that a material weakness exists, and testing and evaluating the design and operating effectiveness of internal control based on the assessed risk. Our audits also included performing such other procedures as we considered necessary in the circumstances. We believe that our audits provide a reasonable basis for our opinions. Definition and Limitations of Internal Control Over Financial Reporting A company’s internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles. A company’s internal control over financial reporting includes those policies and procedures that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company; (2) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company; and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of the company’s assets that could have a material effect on the 137 Table of Contents financial statements. Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate. Critical Audit Matter The critical audit matter communicated below is a matter arising from the current period audit of the financial statements that was communicated or required to be communicated to the audit committee and that: (1) relates to accounts or disclosures that are material to the financial statements and (2) involved our especially challenging, subjective, or complex judgments. The communication of a critical audit matter does not alter in any way our opinion on the financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates. Evaluation of prepaid and accrued research and development expenses As discussed in Notes 2 and 10 to the financial statements, the Company records expenses related to external research and development services based on its estimates of the services received and efforts expended pursuant to quotes and contracts with contract research organizations (CROs) and contract development and manufacturing organizations (CDMOs). At the end of each reporting period, the Company reviews open contracts and purchase orders, communications with their personnel, and communications from external service providers to estimate the level of service performed and associated cost incurred. Depending on the timing of payments to the external service providers and the progress that the Company estimates has been made as a result of the service provided, the Company may record prepaid or accrued expense related to these costs. Accrued research and development costs as of December 31, 2025 were $7.2 million. Prepaid expenses and other current assets as of December 31, 2025 were $13.3 million, which includes prepaid research and development expenses. We identified the evaluation of prepaid and accrued research and development expenses for CROs and CDMOs as a critical audit matter. Specifically, evaluating the estimates of costs incurred by third parties required subjective auditor judgment due to the nature of available evidence. The following are the primary procedures we performed to address this critical audit matter. We evaluated the design and tested the operating effectiveness of certain internal controls related to prepaid and accrued research and development expenses for CROs and CDMOs. This included controls related to the evaluation of the estimation of costs incurred by third parties. For a selection of prepaid and accrued research and development expenses provided by CROs and CDMOs, we evaluated management’s estimate of the prepaid or accrued expense based on confirmation of project status obtained from the relevant third-party, inquiry of project managers, and third-party invoices paid. /s/ KPMG LLP We have served as the Company’s auditor since 2020. Denver, Colorado February 26, 2026 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 138 Table of Contents ​ ​ EDGEWISE THERAPEUTICS, INC. BALANCE SHEETS (In thousands, except share and per share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ As of ​ As of ​ ​ December 31, ​ December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 Assets ​ ​ ​ ​ Current assets ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 61,148 ​ ​ ​ $ 41,666 Marketable securities, available for sale ​ 468,961 ​ 428,504 Prepaid expenses and other assets ​ 13,276 ​ 5,313 Total current assets ​ 543,385 ​ 475,483 ​ ​ ​ ​ ​ ​ ​ Property and equipment, net ​ 7,831 ​ 9,503 Operating lease right-of-use asset ​ ​ 1,387 ​ ​ 1,569 Other non-current assets ​ — ​ 262 ​ ​ ​ ​ ​ ​ ​ Total assets ​ $ 552,603 ​ $ 486,817 ​ ​ ​ ​ ​ ​ ​ Liabilities and stockholders' equity ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Current liabilities ​ ​ ​ ​ Accounts payable ​ $ 6,006 ​ $ 5,579 Accrued compensation ​ 12,430 ​ 10,056 Accrued other expenses ​ 7,920 ​ 7,229 Operating lease liability, current portion ​ ​ 1,014 ​ ​ 996 Total current liabilities ​ 27,370 ​ 23,860 Operating lease liability, net of current portion ​ ​ 2,976 ​ ​ 3,741 ​ ​ ​ ​ ​ ​ ​ Total liabilities ​ 30,346 ​ 27,601 ​ ​ ​ ​ ​ ​ ​ Commitments and contingencies (see note 5) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Stockholders' equity: ​ ​ ​ ​ Preferred stock, $ .0001 par value per share; 200,000,000 shares authorized and no shares issued or outstanding as of December 31, 2025 and December 31, 2024 ​ ​ — ​ ​ — Common stock, $ .0001 par value per share; 1,000,000,000 shares authorized as of December 31, 2025 and December 31, 2024; 106,249,579 shares and 94,838,466 shares issued and outstanding as of December 31, 2025 and December 31, 2024, respectively ​ 10 ​ 9 Additional paid-in capital ​ 1,067,941 ​ 837,363 Accumulated other comprehensive income ​ ​ 677 ​ ​ 420 Accumulated deficit ​ ( 546,371 ) ​ ( 378,576 ) Total stockholders' equity ​ 522,257 ​ 459,216 ​ ​ ​ ​ ​ ​ ​ Total liabilities and stockholders' equity ​ $ 552,603 ​ $ 486,817 ​ The accompanying notes are an integral part of these financial statements. ​ ​ 139 Table of Contents ​ EDGEWISE THERAPEUTICS, INC. STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands, except share and per share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating expenses ​ ​ ​ ​ ​ ​ ​ Research and development ​ $ 151,389 ​ ​ ​ $ 126,966 ​ ​ ​ $ 90,905 General and administrative ​ 40,017 ​ 31,866 ​ 23,452 Total operating expenses ​ 191,406 ​ 158,832 ​ 114,357 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Loss from operations ​ ( 191,406 ) ​ ( 158,832 ) ​ ( 114,357 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Other income ​ ​ ​ ​ ​ ​ Interest income ​ ​ 23,611 ​ 25,019 ​ 14,194 Total other income ​ 23,611 ​ 25,019 ​ 14,194 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ ​ ( 167,795 ) ​ ​ ( 133,813 ) ​ ​ ( 100,163 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Other comprehensive income (loss): ​ ​ ​ ​ ​ ​ ​ ​ ​ Unrealized gain on available-for-sale securities ​ ​ 257 ​ ​ 321 ​ ​ 1,454 Total comprehensive loss ​ $ ( 167,538 ) ​ $ ( 133,492 ) ​ $ ( 98,709 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss per share, basic and diluted ​ $ ( 1.63 ) ​ $ ( 1.45 ) ​ $ ( 1.57 ) Weighted-average shares outstanding, basic and diluted ​ 102,930,744 ​ 92,414,626 ​ 63,723,600 ​ The accompanying notes are an integral part of these financial statements. ​ ​ 140 Table of Contents ​ EDGEWISE THERAPEUTICS, INC. STATEMENTS OF STOCKHOLDERS’ EQUITY (In thousands, except share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Other ​ ​ ​ ​ ​ ​ ​ ​ Common Stock ​ Additional ​ Comprehensive ​ Accumulated ​ ​ ​ ​ ​ ​ ​ Shares ​ ​ ​ Amount ​ ​ ​ Paid-In Capital ​ ​ ​ Income (Loss) ​ ​ ​ Deficit ​ ​ ​ Total Balance as of December 31, 2022 ​ 63,257,376 ​ $ 6 ​ $ 492,665 ​ $ ( 1,355 ) ​ $ ( 144,600 ) ​ $ 346,716 Issuance of common stock, net of offering costs 6,928,044 ​ ​ 1 ​ ​ 52,549 ​ ​ — ​ — ​ 52,550 Exercise of stock options and vesting of restricted stock units 172,663 ​ ​ — ​ ​ 117 ​ ​ — ​ — ​ 117 Purchase of common stock under employee stock purchase plan ​ 95,259 ​ ​ — ​ ​ 596 ​ ​ — ​ — ​ 596 Stock-based compensation ​ — ​ ​ — ​ ​ 17,560 ​ ​ — ​ — ​ 17,560 Other comprehensive income ​ — ​ ​ — ​ ​ — ​ ​ 1,454 ​ — ​ 1,454 Net loss — ​ ​ — ​ ​ — ​ ​ — ​ ( 100,163 ) ​ ( 100,163 ) Balance as of December 31, 2023 ​ 70,453,342 ​ $ 7 ​ $ 563,487 ​ $ 99 ​ $ ( 244,763 ) ​ $ 318,830 Issuance of common stock, net of offering costs ​ 22,450,206 ​ ​ 2 ​ ​ 238,797 ​ ​ — ​ ​ — ​ ​ 238,799 Exercise of stock options and vesting of restricted stock units ​ 1,787,832 ​ ​ — ​ ​ 9,480 ​ ​ — ​ ​ — ​ ​ 9,480 Purchase of common stock under employee stock purchase plan ​ 147,086 ​ ​ — ​ ​ 888 ​ ​ — ​ ​ — ​ ​ 888 Stock-based compensation ​ — ​ ​ — ​ ​ 24,711 ​ ​ — ​ ​ — ​ ​ 24,711 Other comprehensive income ​ — ​ ​ — ​ ​ — ​ ​ 321 ​ ​ — ​ ​ 321 Net loss ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 133,813 ) ​ ​ ( 133,813 ) Balance as of December 31, 2024 ​ 94,838,466 ​ $ 9 ​ $ 837,363 ​ $ 420 ​ $ ( 378,576 ) ​ $ 459,216 Issuance of common stock, net of offering costs ​ 9,935,419 ​ ​ 1 ​ ​ 187,100 ​ ​ — ​ ​ — ​ ​ 187,101 Exercise of stock options and vesting of restricted stock units ​ 1,400,533 ​ ​ — ​ ​ 7,803 ​ ​ — ​ ​ — ​ ​ 7,803 Purchase of common stock under employee stock purchase plan ​ 75,161 ​ ​ — ​ ​ 923 ​ ​ — ​ ​ — ​ ​ 923 Stock-based compensation ​ — ​ ​ — ​ ​ 34,752 ​ ​ — ​ ​ — ​ ​ 34,752 Other comprehensive income ​ — ​ ​ — ​ ​ — ​ ​ 257 ​ ​ — ​ ​ 257 Net loss ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 167,795 ) ​ ​ ( 167,795 ) Balance as of December 31, 2025 ​ 106,249,579 ​ $ 10 ​ $ 1,067,941 ​ $ 677 ​ $ ( 546,371 ) ​ $ 522,257 ​ ​ The accompanying notes are an integral part of these financial statements. ​ ​ ​ 141 Table of Contents EDGEWISE THERAPEUTICS, INC. STATEMENTS OF CASH FLOWS (In thousands) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Cash flows from operating activities ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 167,795 ) ​ ​ ​ $ ( 133,813 ) ​ ​ ​ $ ( 100,163 ) Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ ​ ​ Depreciation ​ ​ 2,084 ​ 2,069 ​ 1,551 Stock-based compensation ​ 34,752 ​ 24,712 ​ 17,560 Accretion of discount on marketable securities, net ​ ​ ( 7,665 ) ​ ​ ( 12,545 ) ​ ​ ( 9,517 ) Amortization of right-of-use asset ​ ​ 182 ​ ​ 221 ​ ​ 181 Changes in assets and liabilities: ​ ​ ​ ​ ​ ​ Prepaid expenses and other assets ​ ( 7,963 ) ​ 3,292 ​ ( 3,553 ) Accounts payable ​ 272 ​ 1,737 ​ ( 92 ) Accrued compensation ​ 2,374 ​ 4,361 ​ 1,663 Accrued other expenses and other liabilities ​ 691 ​ 1,158 ​ 526 Lease liability ​ ( 748 ) ​ ​ ( 220 ) ​ ​ ( 104 ) Net cash used in operating activities ​ ( 143,816 ) ​ ​ ( 109,028 ) ​ ​ ( 91,948 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash flows from investing activities ​ ​ ​ ​ ​ ​ ​ ​ Purchases of marketable securities ​ ( 527,226 ) ​ ( 477,055 ) ​ ( 255,866 ) Sales of marketable securities ​ ​ 67,384 ​ ​ 15,794 ​ ​ 20,118 Maturities of marketable securities ​ ​ 427,308 ​ ​ 277,917 ​ ​ 344,379 Purchases of property and equipment ​ ( 256 ) ​ ( 1,312 ) ​ ( 5,745 ) Net cash (used in) provided by investing activities ​ ( 32,790 ) ​ ( 184,656 ) ​ 102,886 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash flows from financing activities ​ ​ ​ ​ ​ ​ Proceeds from issuance of common stock, net of offering costs ​ ​ 187,362 ​ ​ 239,147 ​ ​ 52,618 Exercise of stock options ​ 7,803 ​ 9,480 ​ 117 Payment of deferred offering costs ​ ​ — ​ ​ ( 262 ) ​ ​ ( 165 ) Proceeds from Employee Stock Purchase Plan ​ ​ 923 ​ ​ 888 ​ ​ 596 Net cash provided by financing activities ​ 196,088 ​ 249,253 ​ 53,166 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net change in cash and cash equivalents ​ 19,482 ​ ( 44,431 ) ​ 64,104 Cash and cash equivalents at beginning of period ​ 41,666 ​ 86,097 ​ 21,993 Cash and cash equivalents at end of period ​ $ 61,148 ​ $ 41,666 ​ $ 86,097 Supplemental disclosures of non-cash investing and financing activities: ​ ​ ​ ​ ​ ​ Right-of-use asset obtained in exchange for new operating lease liability, net of tenant improvement receivable ​ $ — ​ $ — ​ $ 1,110 Property and equipment purchases included in accounts payable and accrued other expenses ​ $ 199 ​ $ 44 ​ $ 227 ​ The accompanying notes are an integral part of these financial statements. 142 Table of Contents EDGEWISE THERAPEUTICS, INC. NOTES TO FINANCIAL STATEMENTS ​ NOTE 1 DESCRIPTION OF BUSINESS Organization and Description of Business Edgewise Therapeutics, Inc. (the Company) was incorporated as a Delaware corporation in May 2017, and is headquartered in Boulder, Colorado. The Company is a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe muscle diseases for which there is significant unmet medical need. The Company’s lead product candidates are sevasemten and EDG-7500: sevasemten is an allosteric, selective, fast myofiber (type II) myosin small molecule inhibitor designed to address contraction-induced muscle injury currently being studied in multiple Phase 2 trials in Becker muscular dystrophy (Becker) and Duchenne muscular dystrophy (Duchenne), which are being held in the U.S., Israel, and certain countries in Europe and Australasia, and EDG-7500 is a novel, oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with hypertrophic cardiomyopathy (HCM) and other diseases of diastolic dysfunction currently being studied in a multipart Phase 2 trial for the potential treatment of obstructive and nonobstructive HCM. The Company is also developing EDG-15400, currently in a Phase 1 trial of healthy adults with the future disease target of heart failure with preserved ejection fraction (HFpEF), and using its proprietary drug discovery platform to develop a pipeline of precision medicine product candidates that target key muscle proteins and modulators to address a broad array of serious muscle disorders. Risks and Uncertainties The board of directors of the Company discusses with management macroeconomic and geopolitical developments, including inflation, instability in the banking and financial services sector, tightening of the credit markets, the impact of changes in the U.S. government administration and policy positions, international conflicts, public health pandemics, cybersecurity, sanctions, and changes in tariffs so that the Company can be prepared to react to new developments as they arise. The board of directors and the management of the Company are carefully monitoring these developments and the resulting economic impact on its financial condition and results of operations. Liquidity and Capital Resources The Company has an accumulated deficit of $ 546.4 million and, cash, cash equivalents and marketable securities of $ 530.1 million as of December 31, 2025. The Company’s ability to fund ongoing operations is highly dependent upon raising additional capital through the issuance of equity securities and issuing debt or other financing vehicles. On June 16, 2023, the Company entered into a Sales Agreement (Sales Agreement) with BofA Securities, Inc. (BofA Securities) under which the Company could offer and sell shares of common stock, having aggregate sales proceeds of up to $ 125.0 million from time to time, through an “at the market offering” program (ATM Program) under which BofA Securities acted as sales agent. Effective January 19, 2024, the Company suspended and terminated the prospectus related to the Company’s common stock issuable pursuant to the terms of the Sales Agreement (the ATM Prospectus). As of the date of the suspension of the ATM Prospectus, the Company had sold 7,560,068 shares of our common stock at a weighted average price of $ 7.93 per share. The gross proceeds were $ 59.9 million, and the net proceeds were $ 59.4 million after deducting underwriting discounts and commissions of $ 0.2 million and offering expenses of $ 0.3 million. On January 23, 2024, the Company closed an underwritten registered direct offering of 21,818,182 shares of common stock at a public offering price of $ 11.00 per share (January 2024 Offering). The aggregate gross proceeds from the January 2024 Offering were $ 240.0 million, and the net proceeds were $ 231.9 million after deducting underwriting discounts and commissions of $ 7.5 million and offering expenses of $ 0.6 million. 143 Table of Contents On May 10, 2024, the Company filed an automatic shelf registration statement on Form S-3ASR that allows the Company to undertake various equity and debt offerings. Additionally, on May 10, 2024, the Company filed a prospectus supplement to the shelf registration statement and entered into a sales agreement with Leerink Partners LLC (Leerink Sales Agreement) under which the Company may offer and sell shares of common stock, having aggregate sales proceeds of up to $ 175.0 million from time to time, through an “at the market offering” program (Leerink ATM) under which Leerink Partners LLC will act as sales agent. The Company has not yet offered or sold any shares of common stock related to the Leerink ATM. On April 3, 2025, the Company closed an underwritten registered direct offering of 9,935,419 shares of common stock at a public offering price of $ 20.13 per share (April 2025 Offering). The aggregate gross proceeds from the April 2025 Offering were $ 200.0 million, and the net proceeds were $ 187.1 million, after deducting underwriting discounts and commissions of $ 12.0 million and offering expenses of $ 0.9 million. The Company’s ability to secure capital is dependent upon success in developing its technology and product candidates. The Company cannot provide assurance that additional capital will be available on acceptable terms, if at all. The issuance of additional equity or debt securities will likely result in substantial dilution to the Company’s stockholders. Should additional capital not be available to the Company in the near term, or not be available on acceptable terms, the Company may be unable to realize value from the Company’s assets or discharge liabilities in the normal course of business, which may, among other alternatives, cause the Company to delay, substantially reduce, or discontinue operational activities to conserve cash balances, which could have a material adverse effect on the Company’s ability to achieve its intended business objectives. The accompanying financial statements have been prepared assuming that the Company will continue as a going concern, which contemplates the realization of assets and the settlement of liabilities and commitments in the normal course of business. The financial statements do not reflect any adjustments relating to the recoverability and reclassification of assets and liabilities that might be necessary if the Company is unable to continue as a going concern. The Company believes that the $ 530.1 million of cash, cash equivalents and marketable securities on hand as of December 31, 2025 will be sufficient to fund its operations in the normal course of business and meet its liquidity needs through at least the next 12 months from the issuance of these financial statements. NOTE 2 SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Basis of Presentation The accompanying financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (U.S. GAAP). Segment Information The Company is organized as a single operating and reportable segment, focused on the discovery, development, and manufacture of drug products for the treatment of severe muscle diseases; segment information is presented in Note 11. All equipment and other fixed assets are physically located in the United States. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Actual results could differ from those estimates. Cash Equivalents The Company considers all liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents as of December 31, 2025 and 2024 primarily consist of money market funds and cash. Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentration of credit risk consist primarily of cash, cash equivalents and marketable securities. Periodically, the Company may maintain deposits in 144 Table of Contents financial institutions in excess of government insured limits. The Company believes that it is not exposed to significant credit risk as its deposits are held at financial institutions that management believes to be of high credit quality. The Company has not experienced any losses on deposits since inception. The Company regularly invests excess cash with major financial institutions in money market funds, corporate debt securities, and commercial paper, all of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from our holdings of these financial instruments is mitigated based on the fact that many of these securities are of high credit rating. Deferred Offering Costs The Company capitalizes certain legal, professional, accounting and other third-party fees that are directly associated with in-process equity issuances as deferred offering costs until such equity issuances are consummated. After consummation of the equity issuance, these costs are recorded as a reduction in the capitalized amount associated with the equity issuance. Should the equity issuance be abandoned, the deferred offering costs are expensed immediately as a charge to operating expenses in the statement of operations. Deferred offering costs were $ 0 as of December 31, 2025 and $ 0.3 million as of December 31, 2024. Such costs are classified in other non-current assets in the accompanying balance sheets. Property and Equipment Property and equipment is stated at cost less accumulated depreciation and amortization. Depreciation of property and equipment is computed using the straight-line method over the estimated useful life of the related asset, which is generally three to seven years , and in the case of leasehold improvements, the shorter of the estimated useful lives of the assets or the term of the lease. Leases The Company accounts for its leases under Accounting Standards Codification (ASC) Topic 842, Leases (ASC 842). At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than 12 months are recognized on the balance sheet as Right-of-Use (ROU) assets and current and non-current lease liabilities, as applicable. The Company has elected not to recognize on the balance sheet leases with terms of 12 months or less. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its material leases on a quarterly basis. Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. For all asset classes of its leases, the Company has elected to account for the lease and non-lease components together for existing classes of underlying asset. Costs determined to be variable and not based on an index or rate are not included in the measurement of the lease liability. Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or circumstances indicate that the carrying value of such assets may not be fully recoverable. Impairment is evaluated based on the sum of undiscounted estimated future cash flows expected to result from use of the related asset compared to its carrying value. If impairment is recognized, the carrying value of the impaired asset is reduced to its fair value. There were no impairment charges or long-lived assets disposed of during the years ended December 31, 2025 and 2024. 145 Table of Contents Income Taxes Deferred income taxes are provided on temporary differences between financial statement and income tax reporting. Temporary differences are differences between the amounts of assets and liabilities reported for financial statement purposes and their tax bases. Deferred tax assets are recognized for temporary differences that will be deductible in future years’ tax returns and for operating loss and tax credit carryforwards. Deferred tax assets are reduced by a valuation allowance if such deferred tax assets are deemed more likely than not that some or all of the deferred tax assets will not be realized. Historically, the Company has not recognized these potential benefits in its financial statements and has fully reserved for such net deferred tax assets, as it believes it is more likely than not that the full benefit of these net deferred tax assets will not be realized. Deferred tax liabilities are recognized for temporary differences that will be taxable in future years. The Company evaluated its tax positions and determined it has no uncertain tax positions as of December 31, 2025. Fair Value of Financial Instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. The Financial Accounting Standards Board (FASB) ASC Topic 820, Fair Value Measurements and Disclosures (ASC 820), establishes a hierarchy of inputs used when available. Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are those that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the best information available in the circumstances. The fair value hierarchy applies only to the valuation inputs used in determining the reported fair value of financial instruments and is not a measure of the investment credit quality. The three levels of the fair value hierarchy are described below: Level 1—quoted prices in active markets for identical assets and liabilities. Level 2—other significant observable inputs (including quoted prices for similar assets and liabilities, interest rates, credit risk, etc.). Level 3—significant unobservable inputs (including the Company’s own assumptions in determining the fair value of assets and liabilities). Marketable Securities, Available For Sale All marketable securities have been classified as “available-for-sale” and are carried at fair value, based upon quoted market prices. The Company considers its available-for-sale portfolio as available for use in current operations. Accordingly, the Company classifies its investments as short-term marketable securities, even though the stated maturity date may be one year or more beyond the current balance sheet date. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive income (loss) and reported as a separate component of stockholders’ equity until realized. Interest income, realized gains and losses, and declines in value judged to be other than temporary, if any, on available-for-sale securities are included in other income. The cost of securities sold is based on the specific-identification method. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. In accordance with the Company’s investment policy, management invests in money market funds, corporate debt securities, commercial paper, asset-backed securities and government securities. The Company has not experienced any realized losses on its deposits of cash, cash equivalents, and marketable securities since inception. 146 Table of Contents The following tables summarize the Company’s financial assets measured at fair value on a recurring basis by level within the fair value hierarchy (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2025 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ ​ ​ ​ Hierarchy ​ ​ ​ Cost Basis ​ ​ ​ Gains ​ ​ ​ Losses ​ ​ ​ Value Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds Level 1 ​ $ 60,965 ​ $ — ​ $ — ​ $ 60,965 Marketable securities, available for sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ Asset-backed securities Level 2 ​ 55,426 ​ ​ 78 ​ ​ — ​ 55,504 Corporate debt securities Level 2 ​ 193,479 ​ ​ 270 ​ ​ ( 5 ) ​ 193,744 Commercial paper Level 2 ​ 10,424 ​ ​ 7 ​ ​ — ​ 10,431 U.S. government treasury and agency securities ​ Level 2 ​ 208,955 ​ ​ 327 ​ ​ — ​ 209,282 Total ​ ​ $ 529,249 ​ $ 682 ​ $ ( 5 ) ​ $ 529,926 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2024 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ ​ Hierarchy ​ ​ ​ Cost Basis ​ ​ ​ Gains ​ ​ ​ Losses ​ ​ ​ Value Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds Level 1 ​ $ 41,474 ​ $ — ​ $ — ​ $ 41,474 Marketable securities, available for sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ Asset-backed securities Level 2 ​ 50,262 ​ 69 ​ — ​ 50,331 Corporate debt securities Level 2 ​ 193,769 ​ 179 ​ ( 81 ) ​ 193,867 Commercial paper ​ Level 2 ​ ​ 10,432 ​ ​ 3 ​ ​ — ​ ​ 10,435 U.S. government treasury and agency securities ​ Level 2 ​ ​ 173,621 ​ ​ 279 ​ ​ ( 29 ) ​ ​ 173,871 Total ​ ​ $ 469,558 ​ $ 530 ​ $ ( 110 ) ​ $ 469,978 ​ The Company’s money market funds are classified as Level 1 because they are valued using quoted market prices. Investments in asset-backed securities, corporate debt securities, commercial paper and U.S. government treasury and agency securities, and supranational and sovereign government securities have been classified as Level 2 as they are valued using quoted prices in less active markets or other directly or indirectly observable inputs. Fair values of asset-backed securities, corporate debt securities, commercial paper, and U.S. government treasury and agency securities were derived based on input of market prices from multiple sources at each reporting period. With regard to commercial paper, all of the securities had high credit ratings and one year or less to maturity; therefore, fair value was derived from accretion of purchase price to face value over the term of maturity or quoted market prices for similar instruments if available. There were no transfers of financial assets between Level 1, Level 2, or Level 3, during the periods presented. As of December 31, 2025, remaining contractual maturities of $ 435.1 million of marketable securities were less than one year and $ 33.2 million of marketable securities were between 1 and 2 years . The Company periodically reviews its portfolio of debt securities to determine if any investment is impaired due to credit loss or other potential valuation concerns. For debt securities where the fair value of the investment is less than the amortized cost basis, the Company has assessed at the individual security level for various quantitative factors including, but not limited to, the nature of the investments, changes in credit ratings, interest rate fluctuations, industry analyst reports, and the severity of impairment. Unrealized losses on marketable securities at December 31, 2025 were primarily due to changes in interest rates, including market credit spreads, and not due to increased credit risks associated with specific securities. Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in equity during a period from transactions and other events and/or circumstances from non-owner sources. The Company’s only element of other comprehensive income (loss) was net unrealized gain (loss) on marketable securities. 147 Table of Contents Stock-Based Compensation In accordance with ASC Topic 718, Compensation—Stock Compensation , the Company recognizes compensation expense for all stock-based awards issued to employees based on the estimated grant-date fair value, which is recognized as expense on a straight-line basis over the requisite service period. The Company has elected to recognize forfeitures as they occur. For restricted stock unit awards, the fair value is based on the closing price of the Company's common stock on the date of grant. The fair value of stock options is determined using the Black-Scholes option-pricing model. The determination of fair value for stock-based awards on the date of grant using an option-pricing model requires management to make certain assumptions including expected volatility, expected term, risk-free interest rate and expected dividends in addition to the Company’s common stock valuation (see Note 4). Research and Development Expenses and Accrued Research and Development Expenses Expenditures made for research and development are charged to expense as incurred. External costs consist primarily of payments to contract research organizations (CROs), contract development and manufacturing organizations (CDMOs), sample acquisition costs and laboratory supplies purchased in connection with the Company’s discovery and preclinical activities, and process development and clinical development activities. Internal costs consist primarily of employee-related costs, facilities, depreciation and costs related to compliance with regulatory requirements. Non-refundable advance payments for goods and services that will be used in future research and development activities are capitalized and recorded as an expense in the period that the Company receives the goods or when services are performed. The Company records expenses related to external research and development services based on its estimates of the services received and efforts expended pursuant to quotes and contracts with multiple CROs and CDMOs that supply, conduct and manage preclinical studies and clinical trials on its behalf. The financial terms of these contracts vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided under such contracts. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual or the amount of prepaid expenses accordingly. Recently Adopted Accounting Pronouncements In December 2023, the FASB issued Accounting Standards Update (ASU) 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures . This ASU enhanced the transparency and decision usefulness of income tax disclosures by requiring public business entities on an annual basis to disclose specific categories in the rate reconciliation, additional information for reconciling items that meet a quantitative threshold, and certain information about income taxes paid. ASU 2023-09 was adopted for the Company’s Annual Report for the fiscal year ended December 31, 2025 on a retrospective basis which resulted in enhanced income tax disclosures (see Note 6). Accounting Standards Not Yet Adopted In November 2024, the FASB issued ASU 2024-03, Income Statement–Reporting Comprehensive Income–Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses . This ASU is expected to improve the disclosures about a public business entity’s expenses and address requests from investors for more detailed information about the types of expenses (including purchases of inventory, employee compensation, depreciation, amortization, and depletion) in commonly presented expense captions (such as cost of sales, SG&A, and research and development). This ASU is effective beginning with the Company’s 2027 fiscal year annual reporting period and interim periods thereafter, with early adoption permitted. The Company is currently evaluating the impact that the adoption of this standard will have on its financial statements. In December 2025, the FASB issued ASU 2025-11, Interim Reporting (Topic 270): Narrow Scope Improvements . This ASU is expected to improve the navigability of the required interim disclosures and clarify when that guidance is applicable, and provide additional guidance on what disclosures should be provided in interim reporting periods, as well as add a principle that requires entities to disclose events since the end of the last annual reporting period that have a material impact on the entity. This ASU is effective for interim reporting periods within annual reporting periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the impact that the adoption of this standard will have on its interim financial statements. 148 Table of Contents NOTE 3 PREFERRED STOCK AND COMMON STOCK The Company is authorized to issue two classes of stock designated as common stock and preferred stock. As of December 31, 2025 the total number of shares authorized was 1,200,000,000 . The total number of shares of common stock authorized was 1,000,000,000 . The total number of shares of preferred stock authorized was 200,000,000 . All shares of the Company’s capital stock have a par value of $ 0.0001 per share. Common stockholders are entitled to dividends if and when declared by the board of directors of the Company and after any convertible preferred share dividends are fully paid. The holder of each share of common stock is entitled to one vote. NOTE 4 STOCK-BASED COMPENSATION AWARDS Equity Incentive Plans In March 2021, the Company’s board of directors adopted, and its stockholders approved, the Company’s 2021 Equity Incentive Plan (2021 Plan), which became effective in March 2021 in connection with the IPO. Upon adoption of the 2021 Plan, the Company restricted the grant of future equity awards under its 2017 Equity Incentive Plan, as amended and restated (2017 Plan). The 2021 Plan provides for the grant of incentive stock options, within the meaning of Section 422 of the Internal Revenue Code, to the Company’s employees and any of its parent and subsidiary corporations’ employees, and for the grant of nonstatutory stock options, restricted stock, restricted stock units (RSUs), stock appreciation rights, performance units, and performance shares to its employees, directors, and consultants and its subsidiary corporations’ employees and consultants. The vesting of stock options is stated in each individual grant agreement, which is generally four years . Options granted expire 10 years after the date of grant. An RSU represents the right to receive one share of common stock upon vesting of the RSU. The fair value of each RSU is based on the closing price of the Company’s common stock on the date of grant and generally vest over 2 to 4 years . A total of 5,040,000 shares of the Company’s common stock were initially reserved for issuance pursuant to the 2021 Plan. The 2021 Plan share reserve increases by the number of shares under the 2017 Plan that are repurchased, forfeited, expired or cancelled after the effective date of the 2021 Plan up to the limit under the 2021 Plan. The number of shares available for issuance under the 2021 Plan increases annually on the first day of each fiscal year beginning with the Company’s 2022 fiscal year, equal to the least of (1) 5,040,000 shares, (2) five percent ( 5 %) of the outstanding shares of its common stock as of the last day of the immediately preceding fiscal year; or (3) such other amount as the Company’s board of directors may determine. As of December 31, 2025, there were 2,599,220 shares available for future issuance under the 2021 Plan. Inducement Equity Incentive Plan Effective August 10, 2024, the Company’s board of directors adopted the Company’s 2024 Inducement Equity Incentive Plan (Inducement Plan) and, subject to the adjustment provisions of the Inducement Plan, reserved 2,000,000 shares of the Company’s common stock for issuance pursuant to equity awards granted under the Inducement Plan. The Inducement Plan was adopted without stockholder approval pursuant to the applicable The Nasdaq Stock Market LLC’s (Nasdaq) Listing Rules. The Inducement Plan provides for the grant of equity-based awards, including nonstatutory stock options, stock appreciation rights, restricted stock, restricted stock units, and performance awards, and its terms are substantially similar to the 2021 Plan, including with respect to treatment of equity awards in the event of a “merger” or “change in control” as defined under the Inducement Plan, but with such other terms and conditions intended to comply with the Nasdaq inducement award exception or to comply with the Nasdaq acquisition and merger exception. In accordance with the Nasdaq Listing Rules, awards under the Inducement Plan may only be made to individuals not previously employees or non-employee directors of the Company (or following such individuals’ bona fide period of non-employment with the Company), as an inducement material to the individuals’ entry into employment with the Company, or, to the extent permitted by the Nasdaq Listing Rules, in connection with a merger or acquisition. The 149 Table of Contents vesting of stock options is stated in each individual grant agreement, which is generally four years , and expires 10 years after the date of grant. The fair value of each RSU is based on the closing price of the Company’s common stock on the date of grant and vests over 4 years . As of December 31, 2025, there were 1,009,750 shares available for future issuance under the Inducement Plan. Stock Options Stock option activity for the year ended December 31, 2025 is as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Average ​ ​ ​ ​ Weighted ​ ​ ​ ​ Remaining ​ ​ ​ ​ Average ​ Total Intrinsic ​ Contractual Life ​ ​ ​ ​ Options ​ ​ ​ Exercise Price ​ ​ ​ Value (000's) ​ ​ ​ (Years) Outstanding as of December 31, 2024 15,416,761 $ 9.40 $ 267,397 ​ 7.5 Granted 4,267,256 ​ $ 15.78 ​ ​ ​ ​ ​ Exercised ( 993,495 ) ​ $ 8.45 ​ ​ ​ ​ ​ Cancelled ​ ( 529,284 ) ​ $ 12.79 ​ ​ ​ ​ ​ Outstanding as of December 31, 2025 ​ 18,161,238 ​ $ 10.85 ​ $ 255,726 ​ 7.2 Options exercisable as of December 31, 2025 ​ 10,687,753 ​ $ 8.46 ​ $ 175,917 ​ 6.0 ​ The vesting of stock options is stated in each individual grant agreement, which is generally four years . As of December 31, 2025, there was unrecognized stock-based compensation cost of $ 64.9 million, which is expected to be recognized over a weighted-average term of 2.8 years. The aggregate intrinsic value of options exercised during the years ended December 31, 2025, 2024 and 2023 was $ 12.9 million, $ 30.3 million, and $ 1.0 million, respectively. For options granted during the years ended December 31, 2025, 2024 and 2023, the weighted-average grant date fair value was $ 10.92 , $ 12.73 , and $ 5.25 per share, respectively. The Company did not have any material awards modified during the years ended December 31, 2025, 2024 and 2023. In previous years, there were additional options issued outside of this plan as discussed under Founder Stock Options. Fair Value Assumptions The fair value of option grants is estimated on the date of grant using the Black-Scholes option-pricing model, which requires the use of the following assumptions: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Expected term (Years) ​ 0.36 - 6.85 ​ 5.10 - 6.85 ​ 5.44 - 6.85 Expected volatility ​ 69.23 % - 81.21 % ​ 78.67 % - 83.21 % ​ 80.68 % - 85.68 % Risk-free interest rate ​ 3.51 % - 4.41 % ​ 3.59 % - 4.66 % ​ 3.47 % - 4.80 % Expected dividend rate ​ - ​ - ​ - Fair value common stock ​ $ 13.11 - $ 28.02 ​ $ 16.33 - $ 33.57 ​ $ 5.73 - $ 10.29 ​ The expected term is based on the “simplified method” described in the U.S. Securities and Exchange Commission’s Staff Accounting Bulletin Topic 14 which is determined as the midpoint between the vesting date and the contractual end of the option grant. Stock price volatility was estimated based on the estimated stock price volatility of a peer group of publicly traded companies over a similar term. The risk-free interest rate for periods within the contractual life of the option is based on the U.S. Treasury yield in effect at the time of grant. The dividend yield was zero as the Company has never declared or paid dividends and has no plans to do so in the foreseeable future. Founder Stock Options On September 19, 2017, the Company granted one of its founders the option to purchase 1,795,880 shares of the Company’s common stock at an exercise price of $ 0.18 per share which vested monthly over a four-year period that expires 15 years after the date of grant. This grant is separate from the Company’s equity incentive plans discussed above. 150 Table of Contents As of December 31, 2025, 1,147,365 options were both outstanding and exercisable and there was no unrecognized stock-based compensation expense. During the years ended December 31, 2025 and 2024, 209,415 options with intrinsic value of $ 3.8 million and 175,000 options with intrinsic value of $ 4.6 million were exercised, respectively. There were no options exercised during the year ended December 31, 2023. As of December 31, 2025, the intrinsic value of options outstanding and exercisable was $ 28.3 million with a weighted-average remaining contractual life of 6.5 years. Restricted Stock Units A RSU represents the right to receive one share of common stock upon vesting of the RSU. The fair value of each RSU is based on the closing price of the Company’s common stock on the date of grant and generally vest over 3 or 4 years . RSU activity for the year ended December 31, 2025 is as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted Average ​ ​ ​ ​ Grant Date ​ ​ ​ ​ RSUs ​ ​ ​ Fair Value Issued and unvested as of December 31, 2024 639,516 $ 15.86 Granted 908,723 ​ $ 14.48 Vested ( 213,411 ) ​ $ 13.68 Cancelled ​ ( 59,876 ) ​ $ 15.87 Issued and unvested as of December 31, 2025 ​ 1,274,952 ​ $ 15.24 ​ As of December 31, 2025, there was unrecognized stock-based compensation cost of $ 15.5 million, which is expected to be recognized over a weighted-average term of 3.2 years. The total fair value of RSUs vested during the years ended December 31, 2025, 2024 and 2023 was $ 3.1 million, $ 0.7 million, and $ 0.7 million, respectively. 2021 Employee Stock Purchase Plan The 2021 Employee Stock Purchase Plan (2021 ESPP) enables eligible employees of the Company to purchase shares of common stock at a discount. A total of 504,000 shares of the Company’s common stock were initially reserved for issuance pursuant to the 2021 ESPP. The number of shares available for issuance under the 2021 ESPP increases annually on the first day of each fiscal year beginning with the Company’s 2022 fiscal year, equal to the least of (1) 1,008,000 shares, (2) one percent ( 1 %) of the outstanding shares of common stock as of the last day of the immediately preceding fiscal year; or (3) such other amount as the Company’s board of directors may determine. As of December 31, 2025, the Company has reserved for issuance 2,900,630 shares of common stock pursuant to the 2021 ESPP. The 2021 ESPP provides for two offering periods of approximately twelve months’ duration, with purchase periods commencing on the first trading day on or after May 15 and November 15 and terminating on the last trading day on or before November 15 of the same year and May 15 of the following year, respectively. Contributions under the 2021 ESPP are limited to 15 % of an employee’s eligible compensation, IRS limitations, and a maximum of 6,000 shares of common stock during each offering period. 2021 ESPP participants will purchase shares of common stock at a price per share equal to 85 % of the lesser of (1) the fair market value per share of the common stock on the first trading day of the offering period or (2) the fair market value of the common stock on the purchase date. Additionally, during the years ended December 31, 2025, 2024 and 2023, a total of 75,161 , 147,086 , and 95,259 shares of common stock were issued under the ESPP at a weighted-average per share price of $ 12.28 , $ 6.03 , and $ 6.26 , respectively. 151 Table of Contents Total stock-based compensation expense related to all equity plans, including Founder Stock Options was allocated as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Research and development $ 19,647 $ 13,956 $ 10,029 General and administrative ​ 15,105 ​ 10,755 ​ 7,531 Total stock-based compensation expense ​ $ 34,752 ​ $ 24,711 ​ $ 17,560 ​ ​ ​ NOTE 5 COMMITMENTS AND CONTINGENCIES Lease Agreements In January 2022, the Company entered into a lease agreement for approximately 18,614 square feet of office and laboratory space in Boulder, Colorado (the New Boulder Lease) with aggregate base rent payments of approximately $ 3.3 million over the initial 8.2 -year term of the lease. Further, the Company provided a standby letter of credit (LOC) of $ 0.8 million during the term of the lease as collateral for the Company’s obligations under the lease. The New Boulder Lease includes two tenant improvement allowances, which includes one for $ 1.0 million in construction costs to be fully reimbursed by the lessor (the First Allowance) and one for $ 2.0 million in construction costs to be repaid to the lessor as additional rent payments over the initial term of the lease (the Second Allowance). Both the First Allowance and Second Allowance have been received in full. The receipt of $ 2.0 million under the Second Allowance resulted in an increase to operating lease liabilities and an increase to aggregate base rent payments totaling $ 2.5 million. In February 2023, the New Boulder Lease was modified to occupy an additional 9,624 square feet of office space (the Expansion Space) with aggregate payments of approximately $ 1.5 million over the initial 7.3 year term of the lease. The Expansion space includes an improvement allowance in the amount of $ 0.5 million to be fully reimbursed by the lessor. The allowance associated with the expansion space has been received in full. Under the New Boulder Lease and the Expansion Space (collectively, the Lease), the Company has the option to extend the Lease for two additional terms of five years each. The Company is obligated to pay the lessor an amount not to exceed 5 % of the net rents from the property for operating costs. Such amounts are not included in the measurement of the lease liabilities and are recognized as variable lease expense when they are incurred. Variable lease expense was $ 0.4 million, $ 0.5 million, and $ 0.3 million for the years ended December 31, 2025, 2024 and 2023, respectively. The Lease is classified as an operating lease. The Company recorded lease liabilities and ROU lease assets for the Lease based on the present value of lease payments over the expected lease term, discounted using the Company’s incremental borrowing rate. The option to extend the Lease was not recognized as part of the Company’s lease liabilities and ROU lease assets, as such extensions are not reasonably certain to occur. As of December 31, 2025, the weighted-average remaining lease term and the weighted-average discount rate for the Lease was 4.3 years and 6.5 %, respectively. Rent expense under the Lease was $ 0.5 million for each of the years ended December 31, 2025, 2024 and 2023. Future minimum lease payments under the Lease as of December 31, 2025 are as follows (in thousands): ​ ​ ​ ​ ​ Year Ending December 31, ​ ​ ​ 2026 ​ ​ ​ $ 1,048 2027 ​ ​ 1,066 2028 ​ 1,084 2029 ​ ​ 1,103 2030 ​ ​ 278 Thereafter ​ ​ — Total undiscounted future minimum lease payments ​ ​ 4,579 Less: discount ​ ​ ( 589 ) Total lease liability ​ $ 3,990 ​ 152 Table of Contents Litigation Liabilities for loss contingencies arising from claims, assessments, litigation, fines, penalties and other sources are recorded when it is probable that a liability has been incurred and the amount can be reasonably estimated. From time to time, the Company may become involved in legal proceedings arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the year ended December 31, 2025 and no material legal proceedings are currently pending or threatened. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners and other parties with respect to certain matters including, but not limited to, losses arising from breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company is not aware of any claims under indemnification arrangements, and it has not accrued any liabilities related to such obligations in its financial statements as of December 31, 2025. NOTE 6 INCOME TAXES Since inception, the Company has incurred net taxable losses, and accordingly, no current provision for income taxes has been recorded. Net loss before income tax expense or benefit of $ 167.8 million, $ 133.8 million, and $ 100.2 million for the years ended December 31, 2025, 2024 and 2023, respectively, was incurred in the United States. The Company has not recorded any United States federal or foreign income tax expense or benefit. ​ The effective income tax rate of the provision for income taxes differs from the federal statutory rate as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Income tax benefit at federal statutory rate ​ ​ ( 35,238 ) 21.0 % ​ ​ ( 28,101 ) 21.0 % ​ ​ ( 21,034 ) 21.0 % State and local income taxes, net of federal income tax effect ​ ​ 4 ( 0.0 ) % ​ ​ 3 ( 0.0 ) % ​ ​ 4 ( 0.0 ) % Federal tax credits ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Orphan drug ​ ​ ( 15,704 ) 9.4 % ​ ​ ( 670 ) 0.5 % ​ ​ ( 691 ) 0.7 % Research and development ​ ​ ( 2,510 ) 1.5 % ​ ​ ( 6,499 ) 4.9 % ​ ​ ( 5,346 ) 5.3 % Change in valuation allowance ​ ​ 49,707 ( 29.6 ) % ​ ​ 34,832 ( 26.0 ) % ​ ​ 25,071 ( 25.0 ) % Non-deductible or non-taxable items ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Section 162(m) limitations ​ ​ 4,450 ( 2.7 ) % ​ ​ 4,629 ( 3.5 ) % ​ ​ 1,432 ( 1.4 ) % Share based compensation ​ ​ ( 528 ) 0.3 % ​ ​ ( 4,596 ) 3.4 % ​ ​ 551 ( 0.6 ) % Other ​ ​ ( 177 ) 0.1 % ​ ​ 405 ( 0.3 ) % ​ ​ 17 ( 0.0 ) % Effective income tax rate ​ ​ 4.0 ( 0.0 ) % ​ ​ ​ 3.0 ( 0.0 ) % ​ ​ ​ 4.0 ( 0.0 ) % ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 153 Table of Contents The tax effect of temporary differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases that give rise to deferred tax assets and liabilities is as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 Deferred tax assets: ​ ​ ​ ​ ​ ​ Federal net operating loss carryforward ​ $ 67,128 ​ $ 30,714 Capitalized research expenses ​ ​ 40,957 ​ ​ 49,054 Federal and state R&D credits ​ ​ 21,616 ​ ​ 16,854 Orphan Drug Credit ​ ​ 16,311 ​ ​ 606 State net operating loss carryforward ​ ​ 14,671 ​ ​ 6,956 Stock-based compensation ​ ​ 6,442 ​ ​ 4,929 Accrued expenses ​ ​ 1,808 ​ ​ 1,661 Lease liability ​ ​ 1,034 ​ ​ 1,236 Property and equipment ​ ​ 261 ​ ​ 107 Total deferred tax assets before valuation allowance ​ ​ 170,228 ​ ​ 112,117 Valuation allowance ​ ​ ( 169,741 ) ​ ​ ( 111,692 ) Total deferred tax assets ​ ​ 487 ​ ​ 425 Deferred tax liabilities: ​ ​ ​ ​ ​ ​ Other ​ ​ ( 128 ) ​ ​ ( 15 ) ROU asset ​ ​ ( 359 ) ​ ​ ( 410 ) Total deferred tax liabilities ​ ​ ( 487 ) ​ ​ ( 425 ) Net deferred tax asset (liability) ​ $ — ​ $ — ​ For the period ended December 31, 2025, the Company has federal and state post-apportioned net operating loss (NOL) carryforwards of $ 319.7 million and $ 333.3 million, respectively. Of the federal amount, $ 1.2 million have a limited carryforward period and will begin to expire in 2037; the remaining $ 318.5 million will have an indefinite carryforward period. Of the state post-apportioned amount, $ 300.6 million have a limited carryforward period and will begin to expire in 2037; the remaining $ 32.7 million will have an indefinite carryforward period. The Company also has federal and state tax credit carryforwards of $ 34.8 million and $ 3.1 million, respectively. The full federal amount of $ 34.8 million has a limited carryforward period and will begin to expire in 2039. Of the state tax credit carryforwards, $ 2.3 million will begin to expire in 2037; the remaining $ 0.8 million have an indefinite carryforward period. In accordance with Section 382 and Section 383, utilization of the NOL and tax credit carryforwards may be subject to limitations based on prior or future ownership changes. After weighing all available positive and negative evidence, the Company determined a full valuation allowance was necessary, consistent with prior year. The Company is subject to income tax in multiple jurisdictions, including federal and several states. The Company has federal and state income tax returns that are open to examination from 2022 and 2021 forward, respectively. In addition, the utilization of NOLs and tax credit carryforwards, from periods prior to those previously mentioned may also be audited by the taxing authorities once utilized. As a result, the Company continuously monitors its current and prior filing positions in order to determine if any unrecognized tax positions need to be recorded. The analysis involves considerable judgement and is based on the best information available. For the period ended December 31, 2025, the Company is not aware of any positions which require an uncertain tax position liability. NOTE 7 EMPLOYEE BENEFIT PLANS In 2017, the Company established a qualified 401(k) plan which covers all employees who meet eligibility requirements. The Company’s contribution to the plan, as determined by the Company’s Board of Directors, was discretionary until September 2021 when the Company initiated a match with a maximum amount of 4 % of the participant’s compensation. During the years ended December 31, 2025, 2024 and 2023, the Company made matching contributions of $ 1.2 million, $ 1.0 million, $ 0.6 million, respectively. 154 Table of Contents NOTE 8 NET LOSS PER SHARE Basic net loss per common share is calculated by dividing the net loss by the weighted-average number of common shares outstanding during the period, without consideration of potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss by the weighted-average number of common shares and potentially dilutive securities outstanding for the period. For purposes of the diluted net loss per share calculation, common stock options and unvested restricted stock units are considered to be potentially dilutive securities. The Company’s participating securities do not have a contractual obligation to share in the Company’s losses. As such, the net loss was attributed entirely to common stockholders. As the Company has reported a net loss for all periods presented, diluted net loss per common share is the same as basic net loss per common share for those periods. The following table sets forth the computation of the basic and diluted net loss per share (in thousands, except share and per share data): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Numerator ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 167,795 ) $ ( 133,813 ) $ ( 100,163 ) Denominator ​ ​ ​ ​ ​ ​ Weighted-average shares outstanding used in computing net loss per share, basic and diluted ​ ​ 102,930,744 ​ ​ 92,414,626 ​ ​ 63,723,600 Net loss per share, basic and diluted ​ $ ( 1.63 ) ​ $ ( 1.45 ) ​ $ ( 1.57 ) ​ The following weighted average outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Options to purchase common stock ​ ​ 17,823,597 ​ ​ 16,340,740 ​ ​ 13,764,188 Unvested restricted stock units ​ ​ 877,323 ​ ​ 328,984 ​ ​ 207,628 Total ​ ​ 18,700,920 ​ ​ 16,669,724 ​ ​ 13,971,816 ​ ​ NOTE 9 PROPERTY AND EQUIPMENT Property and equipment consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ As of December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 Leasehold improvements ​ $ 9,646 ​ $ 9,646 Laboratory equipment ​ ​ 4,070 ​ ​ 3,735 Computers and software ​ ​ 296 ​ ​ 296 Furniture and fixtures ​ ​ 511 ​ ​ 511 Construction in process ​ ​ 77 ​ ​ — Property and equipment, at cost ​ ​ 14,600 ​ ​ 14,188 Less: accumulated depreciation ​ ​ ( 6,769 ) ​ ​ ( 4,685 ) Property and equipment, net ​ $ 7,831 ​ $ 9,503 ​ Depreciation expense was $ 2.1 million, $ 2.1 million, and $ 1.6 million for years ended December 31, 2025, 2024 and 2023, respectively. ​ 155 Table of Contents NOTE 10 ACCRUED OTHER EXPENSES Accrued other expenses consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ As of December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 Accrued research and development costs ​ $ 7,161 ​ $ 6,488 Accrued other ​ ​ 759 ​ ​ 741 Total accrued other expenses ​ $ 7,920 ​ $ 7,229 ​ ​ NOTE 11 SEGMENT REPORTING The Company has one reportable segment focused on the discovery, development, and manufacture of drug products for the treatment of various muscular disorders. Our determination that we operate as a single segment is consistent with the financial information regularly reviewed by the chief operating decision maker (CODM) for purposes of evaluating performance, allocating resources, and planning and forecasting for future periods. Our business activities are managed on a consolidated basis based on net loss that is also reported on the Statement of Operations and Comprehensive Loss as Net Loss. The Company’s CODM is the President and Chief Executive Officer (CEO). The measure of segment assets is reported on the balance sheet as total assets. ​ Substantially all of the Company’s assets are used to support the research, manufacture, and development of drug products for the treatment of muscular disorders; clinical and research data are key drivers in deciding how to allocate resources. The CEO uses net loss and significant segment expenses to monitor budget versus actual results and make decisions on whether to invest in internal or external resources to support the Company’s research and development programs, as well as determine if additional funding is needed for the Company’s research efforts. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 Operating expenses: ​ ​ ​ ​ ​ ​ ​ Contracted research expense ​ $ 87,987 ​ $ 80,214 ​ $ 57,497 Personnel expense ​ ​ 47,606 ​ ​ 35,797 ​ ​ 24,604 Stock-based compensation expense ​ ​ 34,752 ​ ​ 24,712 ​ ​ 17,556 Other segment expense (a) ​ ​ 18,977 ​ ​ 16,040 ​ ​ 13,149 Depreciation ​ ​ 2,084 ​ ​ 2,069 ​ ​ 1,551 Segment net loss ​ ​ ( 191,406 ) ​ ​ ( 158,832 ) ​ ​ ( 114,357 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Reconciliation of net loss ​ ​ ​ ​ ​ ​ ​ ​ ​ Interest income ​ ​ 23,611 ​ ​ 25,019 ​ ​ 14,194 Net Loss ​ $ ( 167,795 ) ​ $ ( 133,813 ) ​ $ ( 100,163 ) a Other segment expense included in Segment net loss includes contracted administrative expenses, intellectual property fees, software costs, occupancy & equipment costs, and other overhead expenses. ​ ​ ​ Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure None