Edgewise Therapeutics, Inc. 10-Q

Item 1. Financial Statements EDGEWISE THERAPEUTICS, INC. Condensed Balance Sheets (In thousands, except share and per share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of ​ As of ​ ​ June 30, ​ December 31, ​ ​ ​ ​ 2026 ​ ​ ​ 2025 Assets ​ (unaudited) ​ ​ Current assets ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 72,319 ​ ​ ​ $ 61,148 Marketable securities, available for sale ​ 388,390 ​ 468,961 Prepaid expenses and other assets ​ 6,445 ​ 13,276 Current assets held for sale ​ ​ 4,364 ​ ​ — Total current assets ​ 471,518 ​ 543,385 ​ ​ ​ ​ ​ ​ ​ Property and equipment, net ​ 6,892 ​ 7,831 Operating lease right-of-use asset ​ ​ 1,278 ​ ​ 1,387 ​ ​ ​ ​ ​ ​ ​ Total assets ​ $ 479,688 ​ $ 552,603 ​ ​ ​ ​ ​ ​ ​ Liabilities and stockholders' equity ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Current liabilities ​ ​ ​ ​ Accounts payable ​ $ 4,666 ​ $ 6,006 Accrued compensation ​ 8,576 ​ 12,430 Accrued other expenses ​ 5,189 ​ 7,920 Operating lease liability, current portion ​ ​ 1,021 ​ ​ 1,014 Current liabilities held for sale ​ ​ 9,590 ​ ​ — Total current liabilities ​ 29,042 ​ 27,370 Operating lease liability, net of current portion ​ ​ 2,569 ​ ​ 2,976 ​ ​ ​ ​ ​ ​ ​ Total liabilities ​ 31,611 ​ 30,346 ​ ​ ​ ​ ​ ​ ​ 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 June 30, 2026 and December 31, 2025 ​ ​ — ​ ​ — Common stock, $ .0001 par value per share; 1,000,000,000 shares authorized as of June 30, 2026 and December 31, 2025; 107,776,380 shares and 106,249,579 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively ​ 10 ​ 10 Additional paid-in capital ​ 1,101,278 ​ 1,067,941 Accumulated other comprehensive income (loss) ​ ​ ( 500 ) ​ ​ 677 Accumulated deficit ​ ( 652,711 ) ​ ( 546,371 ) Total stockholders' equity ​ 448,077 ​ 522,257 ​ ​ ​ ​ ​ ​ ​ Total liabilities and stockholders' equity ​ $ 479,688 ​ $ 552,603 ​ The accompanying notes are an integral part of these condensed financial statements. 6 Table of Contents EDGEWISE THERAPEUTICS, INC. Condensed Statements of Operations and Comprehensive Loss (In thousands, except share and per share data) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six months ended June 30, ​ ​ ​ ​ 2026 ​ ​ ​ 2025 ​ ​ ​ 2026 ​ ​ ​ 2025 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating expenses ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Research and development ​ $ 47,548 ​ $ 33,558 ​ $ 90,199 ​ ​ ​ $ 70,315 General and administrative ​ 14,395 ​ 9,052 ​ 25,859 ​ 18,254 Total operating expenses ​ 61,943 ​ 42,610 ​ 116,058 ​ 88,569 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Loss from operations ​ ( 61,943 ) ​ ( 42,610 ) ​ ( 116,058 ) ​ ( 88,569 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Other income ​ ​ ​ ​ ​ ​ ​ ​ Interest income ​ 4,616 ​ 6,495 ​ ​ 9,718 ​ 11,656 Total other income ​ 4,616 ​ 6,495 ​ 9,718 ​ 11,656 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ ​ ( 57,327 ) ​ ​ ( 36,115 ) ​ ​ ( 106,340 ) ​ ​ ( 76,913 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Other comprehensive income (loss): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Unrealized loss on available-for-sale securities, net ​ ​ ( 248 ) ​ ​ ( 197 ) ​ ​ ( 1,177 ) ​ ​ ( 216 ) Total comprehensive loss ​ $ ( 57,575 ) ​ $ ( 36,312 ) ​ $ ( 107,517 ) ​ $ ( 77,129 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss per share, basic and diluted ​ $ ( 0.53 ) ​ $ ( 0.34 ) ​ $ ( 0.99 ) ​ $ ( 0.77 ) Weighted-average shares outstanding, basic and diluted ​ 107,669,774 ​ 104,940,493 ​ 107,394,769 ​ 100,062,373 ​ The accompanying notes are an integral part of these condensed financial statements. ​ ​ 7 Table of Contents ​ EDGEWISE THERAPEUTICS, INC. Condensed Statements of Stockholders’ Equity (In thousands, except share data) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ Common Stock ​ Additional ​ Other Comprehensive ​ Accumulated ​ ​ ​ ​ ​ Shares ​ Amount ​ Paid-In Capital ​ Income (Loss) ​ Deficit ​ Total Balance as of December 31, 2024 94,838,466 ​ $ 9 ​ $ 837,363 ​ $ 420 ​ $ ( 378,576 ) ​ $ 459,216 Exercise of stock options 367,217 ​ — ​ 2,261 ​ ​ — ​ — ​ 2,261 Stock-based compensation — ​ — ​ 9,059 ​ ​ — ​ — ​ 9,059 Other comprehensive loss ​ — ​ — ​ — ​ ​ ( 19 ) ​ — ​ ( 19 ) Net loss ​ — ​ — ​ — ​ ​ — ​ ( 40,798 ) ​ ( 40,798 ) Balance as of March 31, 2025 95,205,683 ​ $ 9 ​ $ 848,683 ​ $ 401 ​ $ ( 419,374 ) ​ $ 429,719 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 ​ 151,535 ​ ​ — ​ ​ 233 ​ ​ — ​ ​ — ​ ​ 233 Purchase of common stock under Employee Stock Purchase Plan ​ 48,659 ​ ​ — ​ ​ 597 ​ ​ — ​ ​ — ​ ​ 597 Stock-based compensation ​ — ​ ​ — ​ ​ 7,733 ​ ​ — ​ ​ — ​ ​ 7,733 Other comprehensive loss ​ — ​ ​ — ​ ​ — ​ ​ ( 197 ) ​ ​ — ​ ​ ( 197 ) Net loss ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 36,115 ) ​ ​ ( 36,115 ) Balance as of June 30, 2025 ​ 105,341,296 ​ $ 10 ​ $ 1,044,346 ​ $ 204 ​ $ ( 455,489 ) ​ $ 589,071 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ Common Stock ​ Additional ​ Other Comprehensive ​ Accumulated ​ ​ ​ ​ ​ Shares ​ Amount ​ Paid-In Capital ​ Income (Loss) ​ Deficit ​ Total Balance as of December 31, 2025 ​ 106,249,579 ​ $ 10 ​ $ 1,067,941 ​ $ 677 ​ $ ( 546,371 ) ​ $ 522,257 Exercise of stock options and vesting of restricted stock units 1,231,943 ​ — ​ 12,173 ​ ​ — ​ — ​ 12,173 Stock-based compensation — ​ — ​ 8,715 ​ ​ — ​ — ​ 8,715 Other comprehensive loss — ​ — ​ — ​ ​ ( 929 ) ​ — ​ ( 929 ) Net loss — ​ — ​ — ​ ​ — ​ ( 49,013 ) ​ ( 49,013 ) Balance as of March 31, 2026 107,481,522 ​ $ 10 ​ $ 1,088,829 ​ $ ( 252 ) ​ $ ( 595,384 ) ​ $ 493,203 Exercise of stock options and vesting of restricted stock units ​ 234,659 ​ ​ — ​ ​ 2,294 ​ ​ — ​ ​ — ​ 2,294 Purchase of common stock under Employee Stock Purchase Plan ​ 60,199 ​ ​ — ​ ​ 793 ​ ​ — ​ ​ — ​ 793 Stock-based compensation ​ — ​ ​ — ​ ​ 9,362 ​ ​ — ​ ​ — ​ 9,362 Other comprehensive loss ​ — ​ ​ — ​ ​ — ​ ​ ( 248 ) ​ ​ — ​ ​ ( 248 ) Net loss ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 57,327 ) ​ ( 57,327 ) Balance as of June 30, 2026 ​ 107,776,380 ​ $ 10 ​ $ 1,101,278 ​ $ ( 500 ) ​ $ ( 652,711 ) ​ $ 448,077 The accompanying notes are an integral part of these condensed financial statements. ​ 8 Table of Contents EDGEWISE THERAPEUTICS, INC. Condensed Statements of Cash Flows (In thousands) (Unaudited) ​ ​ ​ ​ ​ ​ ​ ​ ​ Six months ended June 30, ​ ​ ​ 2026 ​ ​ ​ 2025 Cash flows from operating activities ​ ​ ​ ​ ​ Net loss ​ $ ( 106,340 ) ​ ​ ​ $ ( 76,913 ) Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ Depreciation ​ ​ 1,078 ​ 1,036 Stock-based compensation ​ 18,077 ​ 16,792 Accretion of discount on marketable securities, net ​ ​ ( 1,822 ) ​ ​ ( 4,126 ) Amortization of right-of-use asset ​ ​ 109 ​ ​ 85 Changes in assets and liabilities: ​ ​ ​ ​ Prepaid expenses and other assets ​ 2,466 ​ ( 3,950 ) Accounts payable ​ 2,227 ​ 2,707 Accrued compensation ​ ( 3,854 ) ​ ( 4,420 ) Accrued other expenses and other liabilities ​ 3,446 ​ ( 1,293 ) Lease liability ​ ( 399 ) ​ ​ ( 366 ) Net cash used in operating activities ​ ( 85,012 ) ​ ​ ( 70,448 ) ​ ​ ​ ​ ​ ​ ​ Cash flows from investing activities ​ ​ ​ ​ ​ Purchases of marketable securities ​ ( 163,237 ) ​ ( 385,942 ) Sales of marketable securities ​ ​ 26,766 ​ ​ 38,650 Maturities of marketable securities ​ ​ 217,686 ​ ​ 229,118 Purchases of property and equipment ​ ( 292 ) ​ ( 94 ) Net cash provided by (used in) investing activities ​ 80,923 ​ ( 118,268 ) ​ ​ ​ ​ ​ ​ ​ Cash flows from financing activities ​ ​ ​ ​ Proceeds from issuance of common stock, net of offering costs ​ ​ — ​ ​ 187,362 Exercise of stock options ​ 14,467 ​ 2,494 Payment of deferred offering costs ​ ​ — ​ ​ — Proceeds from Employee Stock Purchase Plan ​ ​ 793 ​ ​ 598 Net cash provided by financing activities ​ 15,260 ​ 190,454 ​ ​ ​ ​ ​ ​ ​ Net change in cash and cash equivalents ​ 11,171 ​ 1,738 Cash and cash equivalents at beginning of period ​ 61,148 ​ 41,666 Cash and cash equivalents at end of period ​ $ 72,319 ​ $ 43,404 Supplemental disclosures of non-cash investing and financing activities: ​ ​ ​ ​ Property and equipment purchases included in accounts payable ​ $ 47 ​ $ 78 ​ The accompanying notes are an integral part of these condensed financial statements. 9 Table of Contents EDGEWISE THERAPEUTICS, INC. Notes to Condensed Financial Statements (Unaudited) NOTE 1 DESCRIPTION OF BUSINESS Organization and Description of Business Edgewise Therapeutics, Inc. (the Company) is a late-stage clinical biopharmaceutical company focused on advancing therapies for people living with for serious cardiovascular diseases. The Company’s pipeline includes EDG-7500, a novel, oral, selective, cardiac sarcomere modulator, in development for the treatment of hypertrophic cardiomyopathy (HCM), including both obstructive HCM (oHCM) and nonobstructive HCM (nHCM). The Company is also advancing EDG-15400, an oral next-generation cardiac sarcomere modulator in clinical development for the treatment of heart failure with preserved ejection fraction (HFpEF). In addition, the Company is leveraging its precision medicine platform to advance additional research programs focused on genetically defined cardiovascular and cardiometabolic diseases. On May 31, 2026, the Company entered into an Asset Purchase Agreement (Sevasemten Purchase Agreement) with Servier Pharmaceuticals LLC and Les Laboratoires Servier (Servier) for the sale of the sevasemten muscular dystrophy program, including certain related assets, intellectual property, know-how, select employees, contracts, regulatory filings and clinical data, and assumed certain related liabilities (Sevasemten Sale). As a result, the related disposal group was classified as held for sale as of June 30, 2026, and the Sevasemten Sale closed on July 10, 2026. Following completion of the Sevasemten Sale, the Company is focused on advancing its cardiovascular pipeline and related research and development activities. 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 $ 652.7 million and cash, cash equivalents and marketable securities of $ 460.7 million as of June 30, 2026. 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 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. ​ As a subsequent event, on July 10, 2026, the Company completed the Sevasemten Sale receiving $ 1,550 million in upfront cash proceeds and is eligible to receive up to $ 1,100 million in potential future milestone payments, including (a) a potential milestone payment upon achieving U.S. marketing approval for sevasemten for 10 Table of Contents Becker muscular dystrophy in the amount of (i) $ 200 million, in cash, payable in the event of an approved labelling including specified adult and adolescent populations or (ii) $ 100 million in cash, payable in the event of an approved labelling including only specified adult populations (if (i) has not previously been achieved); (b) a potential milestone payment of $ 600 million in cash, payable upon the achievement of U.S. marketing approval for sevasemten for Duchenne muscular dystrophy; and (c) a potential milestone payment of $ 300 million in cash, payable upon the achievement of annual U.S. net sales of sevasemten products exceeding $ 550 million. Together, the upfront cash proceeds and potential future milestone payments constitute aggregate potential consideration of up to $ 2,650 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 $ 460.7 million of cash, cash equivalents and marketable securities on hand as of June 30, 2026 and the $ 1,550 million in proceeds from the Sevasemten Sale received on July 10, 2026 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 condensed financial statements and related notes to the condensed financial statements should be read in conjunction with the audited financial statements and the related notes thereto for the year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 26, 2026. There have been no material changes to the summary of significant accounting policies as disclosed in that filing. These interim financial statements have been prepared in accordance with U.S. generally accepted accounting principles for interim financial information and include all adjustments consisting of normal recurring adjustments that management believes are necessary for a fair presentation of the periods presented and are not necessarily indicative of results expected for the full fiscal year or for any subsequent interim period. 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 11 Table of Contents securities. The Company has not experienced any realized losses on its deposits of cash, cash equivalents, and marketable securities since inception. ​ 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 June 30, 2026 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ ​ ​ ​ Hierarchy ​ ​ ​ Cost Basis ​ ​ ​ Gains ​ ​ ​ Losses ​ ​ ​ Value Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds Level 1 ​ $ 72,107 ​ $ — ​ $ — ​ $ 72,107 Marketable securities, available for sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ Asset-backed securities Level 2 ​ 34,115 ​ ​ 4 ​ ​ ( 23 ) ​ 34,096 Corporate debt securities Level 2 ​ 144,071 ​ ​ 6 ​ ​ ( 212 ) ​ 143,865 Commercial paper Level 2 ​ 28,908 ​ ​ 2 ​ ​ ( 36 ) ​ 28,874 U.S. government treasury and agency securities ​ Level 2 ​ 181,797 ​ ​ 9 ​ ​ ( 250 ) ​ 181,555 Total ​ ​ $ 460,998 ​ $ 21 ​ $ ( 521 ) ​ $ 460,497 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 ​ 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 June 30, 2026, the remaining contractual maturities of $ 388.9 million of marketable securities were less than one year . 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 June 30, 2026 were primarily due to changes in interest rates, including market credit spreads, and not due to increased credit risks associated with specific securities. ​ 12 Table of Contents Accounting Standards Not Yet Adopted In November 2024, the FASB issued Accounting Standards Update (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 Improvement s. This ASU is expected to improve the navigability of the required interim disclosures, clarify when that guidance is applicable, and provide additional guidance on what disclosures should be included in interim reporting periods. It also adds a principle requiring 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. ​ 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 June 30, 2026, 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 June 30, 2026, there were 7,832,215 shares of common stock available for future issuance under the 2021 Plan. 13 Table of Contents ​ ​ ​ 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 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 June 30, 2026, there were 352,000 shares available for future issuance under the Inducement Plan. 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 and expire 15 years after the date of grant. This grant is separate from the Company’s equity incentive plans discussed above. As of June 30, 2026, 1,147,365 options were both outstanding and exercisable. 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 June 30, 2026, there were 3,848,431 shares of common stock available for future issuance under 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. ​ ​ ​ 14 Table of Contents Total stock-based compensation expense related to all equity plans was allocated as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six months ended June 30, ​ ​ ​ ​ 2026 ​ ​ ​ 2025 ​ ​ ​ 2026 ​ ​ ​ 2025 Research and development ​ $ 5,418 $ 4,489 $ 10,515 $ 10,356 General and administrative ​ 3,944 ​ 3,244 ​ 7,562 ​ 6,436 Total stock-based compensation expense ​ $ 9,362 ​ $ 7,733 ​ $ 18,077 ​ $ 16,792 ​ ​ NOTE 5 COMMITMENTS AND CONTINGENCIES 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 three and six months ended June 30, 2026 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 June 30, 2026. ​ NOTE 6 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): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six Months Ended June 30, ​ ​ ​ ​ 2026 ​ ​ 2025 ​ ​ ​ 2026 ​ ​ ​ 2025 Numerator ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 57,327 ) ​ $ ( 36,115 ) ​ $ ( 106,340 ) $ ( 76,913 ) Denominator ​ ​ ​ ​ ​ ​ ​ ​ Weighted-average shares outstanding used in computing net loss per share, basic and diluted ​ ​ 107,669,774 ​ ​ 104,940,493 ​ ​ 107,394,769 ​ ​ 100,062,373 Net loss per share, basic and diluted ​ $ ( 0.53 ) ​ $ ( 0.34 ) ​ $ ( 0.99 ) ​ $ ( 0.77 ) ​ 15 Table of Contents 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: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six Months Ended June 30, ​ ​ ​ ​ 2026 ​ ​ 2025 ​ ​ 2026 ​ ​ ​ 2025 Options to purchase common stock ​ ​ 18,439,730 ​ ​ 16,825,819 ​ ​ 18,554,483 ​ ​ 16,809,703 Unvested restricted stock units ​ ​ 1,205,783 ​ ​ 633,509 ​ ​ 1,217,161 ​ ​ 651,722 Total ​ ​ 19,645,513 ​ ​ 17,459,328 ​ ​ 19,771,644 ​ ​ 17,461,425 ​ NOTE 7 PROPERTY AND EQUIPMENT Property and equipment consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of June 30, ​ As of December 31, ​ ​ ​ ​ 2026 ​ ​ ​ 2025 Leasehold improvements ​ $ 9,815 ​ $ 9,646 Laboratory equipment ​ ​ 4,117 ​ ​ 4,070 Computers and software ​ ​ 296 ​ ​ 296 Furniture and fixtures ​ ​ 511 ​ ​ 511 Construction in process ​ ​ — ​ ​ 77 Property and equipment, at cost ​ ​ 14,739 ​ ​ 14,600 Less: accumulated depreciation ​ ​ ( 7,847 ) ​ ​ ( 6,769 ) Property and equipment, net ​ $ 6,892 ​ $ 7,831 ​ ​ Depreciation expense was $ 0.5 million and $ 1.1 million for the three and six months ended June 30, 2026 and $ 0.5 million and $ 1.0 million for the three and six months ended June 30, 2025. NOTE 8 ACCRUED OTHER EXPENSES Accrued other expenses consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of June 30, ​ As of December 31, ​ ​ ​ ​ 2026 ​ ​ ​ 2025 Accrued research and development costs ​ $ 4,228 ​ $ 7,161 Accrued other ​ ​ 961 ​ ​ 759 Total accrued other expenses ​ $ 5,189 ​ $ 7,920 ​ ​ ​ ​ ​ ​ ​ 16 Table of Contents ​ NOTE 9 SEGMENT REPORTING There have been no changes in the Company’s assessment of the basis of segmentation or in the basis of measurement of segment profit or loss since the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 26, 2026. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six Months Ended June 30, ​ ​ ​ ​ 2026 ​ ​ 2025 ​ ​ ​ ​ 2026 ​ ​ ​ 2025 Operating expenses: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contracted research expense ​ $ 27,838 ​ $ 19,017 ​ $ 51,780 ​ $ 40,316 Personnel expense ​ ​ 15,817 ​ ​ 10,894 ​ ​ 30,541 ​ ​ 21,518 Stock-based compensation expense ​ ​ 9,362 ​ ​ 7,733 ​ ​ 18,077 ​ ​ 16,792 Other segment expense (a) ​ ​ 8,385 ​ ​ 4,443 ​ ​ 14,582 ​ ​ 8,907 Depreciation ​ ​ 541 ​ ​ 523 ​ ​ 1,078 ​ ​ 1,036 Segment net loss ​ ​ ( 61,943 ) ​ ​ ( 42,610 ) ​ ​ ( 116,058 ) ​ ​ ( 88,569 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Reconciliation of net loss ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Interest income ​ ​ 4,616 ​ ​ 6,495 ​ ​ 9,718 ​ ​ 11,656 Net Loss ​ $ ( 57,327 ) ​ $ ( 36,115 ) ​ $ ( 106,340 ) ​ $ ( 76,913 ) 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 . ​ NOTE 10 ASSETS AND LIABILITIES HELD FOR SALE On May 31, 2026, the Company entered into the Sevasemten Purchase Agreement with Servier, to sell the assets and transfer the liabilities comprising its sevasemten program (the Disposal Group) for (i) closing consideration of $ 1,550.0 million in cash and (ii) up to $ 1,100.0 million of additional contingent consideration payable upon the achievement of specified clinical, regulatory and commercial milestones for the sevasemten program. The transaction was completed on July 10, 2026. In connection with the closing of the Sevasemten Sale, the Company incurred fees payable to its financial advisor and legal counsel, of approximately $ 47.2 million in the aggregate. These fees were contingent upon successful closing of the Sevasemten Sale and were not payable, and no liability existed, as of June 30, 2026. Upon closing, these amounts became fixed and determinable obligations of the Company and will be recognized as a reduction of the gain on sale in connection with the disposal. The assets and liabilities of the Disposal Group met the criteria for classification as held for sale under ASC 360-10-45-9 effective May 31, 2026, and have been presented as Current assets held for sale and Current liabilities held for sale in the accompanying unaudited condensed balance sheet as of June 30, 2026. The comparative condensed balance sheet as of December 31, 2025 has not been reclassified. The Disposal Group has been measured at the lower of its carrying amount or fair value less costs to sell. Because the estimated fair value less costs to sell of the Disposal Group exceeds its carrying amount, no impairment loss was recognized upon its classification as held for sale for the three months ended June 30, 2026. The Company expects to recognize a gain within Other income upon completion of the sale for the three months ended September 30, 2026. The Disposal Group does not include property and equipment or other long-lived assets subject to depreciation or amortization. The Disposal Group is reported within the Company's single operating and reportable segment. For the three and six months ended June 30, 2026, the Disposal Group's pretax loss, recorded within operating expenses, was $ 21.5 million and $ 39.9 million, respectively. ​ ​ ​ 17 Table of Contents ​ ​ ​ ​ ​ ​ As of June 30, ​ ​ ​ ​ 2026 Assets held for sale: ​ ​ ​ Prepaid expenses and other assets ​ $ 4,364 Total assets held for sale ​ $ 4,364 ​ ​ ​ ​ Liabilities held for sale: ​ ​ ​ Accounts payable ​ $ 3,413 Accrued other expenses ​ ​ 6,177 Total liabilities held for sale ​ $ 9,590 ​ The Company did not present the disposal group as discontinued operations because the disposition did not meet the applicable discontinued-operations criteria. The sevasemten intellectual property, know-how, regulatory filings, clinical data and related contracts were internally generated and, as a result, related costs were expensed as incurred and no intangible asset was included in the disposal group. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​