Scholar Rock(SRRK) - 2025 Q2 - Quarterly Report

PART I Item 1. Financial Statements (Unaudited) Presents unaudited condensed consolidated financial statements and notes on financial position and performance Condensed Consolidated Balance Sheets Presents the company's financial position at specific dates, detailing assets, liabilities, and equity | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | | :----- | :--------------------------- | :------------------------------- | :-------------------- | | Cash and cash equivalents | $152,925 | $177,878 | $(24,953) | | Marketable securities | $142,088 | $259,400 | $(117,312) | | Total current assets | $319,126 | $451,165 | $(132,039) | | Total assets | $340,045 | $474,922 | $(134,877) | | Total liabilities | $106,752 | $106,288 | $464 | | Total stockholders' equity | $233,293 | $368,634 | $(135,341) | Condensed Consolidated Statements of Operations and Comprehensive Loss Details the company's revenues, expenses, and net loss over specified three and six-month periods | Metric (in thousands) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | Change ($) | Change (%) | | :-------------------- | :--------------------------- | :--------------------------- | :--------- | :--------- | | Research and development | $62,401 | $42,373 | $20,028 | 47.3% | | General and administrative | $49,708 | $17,125 | $32,583 | 190.3% | | Total operating expenses | $112,109 | $59,498 | $52,611 | 88.4% | | Net loss | $(110,031) | $(58,508) | $(51,523) | 88.1% | | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change ($) | Change (%) | | :-------------------- | :--------------------------- | :--------------------------- | :--------- | :--------- | | Research and development | $111,079 | $85,466 | $25,613 | 30.0% | | General and administrative | $78,120 | $32,451 | $45,669 | 140.7% | | Total operating expenses | $189,199 | $117,917 | $71,282 | 60.5% | | Net loss | $(184,754) | $(115,361) | $(69,393) | 60.2% | Condensed Consolidated Statements of Stockholders' Equity Tracks changes in the company's equity over the six months, including net loss and equity compensation | Metric (in thousands) | Balance at Dec 31, 2024 | Balance at June 30, 2025 | Change | | :-------------------- | :---------------------- | :----------------------- | :----- | | Total Stockholders' Equity | $368,634 | $233,293 | $(135,341) | | Net loss (6 months) | N/A | $(184,754) | N/A | | Equity-based compensation expense (6 months) | N/A | $37,846 | N/A | | Exercise of stock options (6 months) | N/A | $8,126 | N/A | Condensed Consolidated Statements of Cash Flows Summarizes cash inflows and outflows from operating, investing, and financing activities over six months | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change | | :-------------------- | :--------------------------- | :--------------------------- | :----- | | Net cash used in operating activities | $(155,616) | $(98,981) | $(56,635) | | Net cash provided by investing activities | $120,020 | $84,006 | $36,014 | | Net cash provided by financing activities | $10,643 | $6,492 | $4,151 | | Net decrease in cash, cash equivalents and restricted cash | $(24,953) | $(8,483) | $(16,470) | Notes to Condensed Consolidated Financial Statements Provides detailed explanations and additional information supporting the consolidated financial statements 1. Nature of the Business Describes Scholar Rock's focus as a late-stage biopharmaceutical company and its key product candidates - Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for spinal muscular atrophy (SMA) and other neuromuscular diseases, utilizing a proprietary platform targeting the TGFβ superfamily[22][79] - Apitegromab achieved its primary endpoint in the pivotal Phase 3 SAPPHIRE trial for non-ambulatory SMA, is under FDA priority review with a PDUFA target action date of September 22, 2025, and received EMA MAA validation. A U.S. commercial launch is expected in Q4 2025, with Europe to follow in 2026, if approved[23][82] - The company is expanding its anti-myostatin program with SRK-439, a novel, fully human anti-myostatin monoclonal antibody, with an investigational new drug (IND) application planned for the second half of 2025[24][85] - SRK-181, a highly selective inhibitor of latent TGFβ1 activation for checkpoint inhibitor-resistant cancers, completed its Phase 1 DRAGON trial in June 2025, showing encouraging responses[25][88] 2. Summary of Significant Accounting Policies Outlines the company's accounting policies and the impact of recent and future accounting pronouncements - The company's significant accounting policies remain materially unchanged from those disclosed in its Annual Report on Form 10-K for the year ended December 31, 2024[31] - ASU 2023-07 (Segment Reporting) was adopted and did not have a material impact on the condensed consolidated financial position and results of operations[35] - The company is evaluating the impact of ASU 2023-09 (Income Taxes) and ASU 2024-03 (Expense Disaggregation) on future disclosures, with effective dates in 2025 and 2026/2027, respectively[36][37] 3. Fair Value of Financial Assets and Liabilities Details fair value measurements of financial assets, primarily marketable securities and cash equivalents | Asset Category (in thousands) | June 30, 2025 (Total Fair Value) | December 31, 2024 (Total Fair Value) | | :---------------------------- | :------------------------------- | :----------------------------------- | | Money market funds | $132,321 | $97,290 | | U.S. treasury obligations (cash & cash equivalents) | $12,963 | $63,171 | | Marketable securities (U.S. treasury & government agency) | $142,088 | $259,400 | | Total assets at fair value | $287,372 | $419,861 | - All financial assets measured at fair value on a recurring basis (money market funds, U.S. treasury obligations, and government agency securities) are categorized as Level 1, indicating valuation using quoted market prices[38] 4. Marketable Securities