- \n
- Completed constructive and collaborative in-person Type A meeting with U.S. Food and Drug Administration (FDA) on November 12th<\/sup> for apitegromab biologics license application (BLA) for the treatment of children and adults with spinal muscular atrophy (SMA)<\/i><\/li>\n
- Catalent Indiana, LLC (part of <\/i>Novo <\/i>Nordisk) participated in the Type A meeting in person and presented the progress made in implementing the remediation plan; confirmed to FDA that the site is on-track to be reinspection ready by the end of this year<\/i><\/li>\n
- Resubmission <\/i>of BLA and U.S. launch following approval of apitegromab for children and adults with SMA anticipated in 2026<\/i><\/li>\n
- Timelines accelerated with additional apitegromab U.S. fill-finish facility; tech transfer underway with commercial capacity reserved beginning in Q1 2026<\/i><\/li>\n
- Dosing underway in Phase 2 OPAL study evaluating apitegromab in infants and toddlers <2 years of age with SMA<\/i><\/li>\n
- FDA cleared SRK-439 Investigational New Drug (IND) application; dosing in healthy volunteers to commence in Q4 2025<\/i><\/li>\n
- Cash, cash equivalents and marketable securities of $369.6 million as of September 30, 2025; expected to fund operations into 2027<\/i><\/li>\n
- Company to host conference call today at 8:00 a.m. ET<\/i><\/li>\n<\/ul>\n
CAMBRIDGE, Mass.–(BUSINESS WIRE<\/a>)–
\nScholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today reported financial results for the third quarter ended September 30, 2025, and provided an update on recent company developments.<\/p>\n\n\u201cWe are unwavering in our commitment to bring apitegromab, the world\u2019s first and only muscle-targeted treatment to improve motor function, to children and adults living with SMA,\u201d said David L. Hallal, Chairman and Chief Executive of Scholar Rock. \u201cWe are grateful to the FDA, Cure SMA, and our colleagues at Novo Nordisk for the positive engagement at our in-person Type A meeting earlier this week. We are encouraged by the discussion and by our shared understanding of the urgency to bring this important treatment to the SMA community as rapidly as possible.\u201d<\/p>\n
\nMr. Hallal continued, \u201cIn parallel, we are equally focused on establishing Scholar Rock as the global leader in myostatin biology and muscle-targeted therapeutics, and we will continue to advance key strategic programs with tight financial discipline.\u201d<\/p>\n
Business Highlights and Upcoming Milestones<\/b><\/p>\n
Apitegromab<\/span><\/b><\/p>\n
\nApitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in SMA to demonstrate statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE).<\/p>\n
SMA Program<\/b><\/p>\n
- \n
- Completed <\/b>Type A meeting with FDA. <\/b>On November 12, 2025, Scholar Rock had a Type A meeting with the U.S. Food and Drug Administration (FDA). The meeting was held in person and was constructive. Catalent Indiana, LLC (part of Novo Nordisk) joined Scholar Rock at the meeting and detailed the progress it has made in implementing the remediation plan, and affirmed that it expects the facility to be ready for reinspection by the end of this year. Resubmission of the BLA and U.S. launch following approval of apitegromab for children and adults with SMA is anticipated in 2026.\n<\/li>\n
- Accelerated <\/b>timelines with second fill-finish facility.<\/b> Scholar Rock has accelerated timelines with an additional fill-finish facility for apitegromab. The facility is a commercially approved facility that is based in the U.S. and has a proven track record of successful site inspections. Tech transfer is underway, and commercial capacity has been reserved beginning in the first quarter of 2026.\n<\/li>\n
- European Medicines Agency (EMA)<\/b>regulatory review ongoing. <\/b>Scholar Rock continues to expect a decision on its apitegromab Marketing Authorisation Application (MAA) by mid-2026.\n<\/li>\n
- Disease awareness and education activities remain underway. <\/b>U.S. customer-facing teams are in the field and continue to engage with key stakeholders on SMA disease awareness initiatives as well as on the unmet need and the importance of targeting muscle. In Europe, disease awareness and market access initiatives continue in key markets.\n<\/li>\n
- Initiated <\/b>dosing in Phase 2 OPAL clinical trial.\u00a0<\/b>This trial is designed to evaluate apitegromab in infants and toddlers with SMA under two years of age who have been treated with an SMN1-targeted gene therapy or who are receiving treatment with an approved SMN2-targeted therapy.\n<\/li>\n<\/ul>\n
Additional Indications<\/b><\/p>\n
- \n
- Clinical development activities in second indication to initiate by YE 2025.<\/b> Scholar Rock expects to initiate clinical development activities in a second neuromuscular disorder by year-end 2025. The Company plans to provide additional information on the disease and the clinical development strategy in early 2026.\n<\/li>\n<\/ul>\n
SRK-439<\/span><\/b><\/p>\n
\nSRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass.<\/p>\n
- \n
- FDA cleared IND in Q4 2025.<\/b> Scholar Rock expects to initiate dosing of SRK-439 in healthy volunteers in the fourth quarter of 2025.\n<\/li>\n<\/ul>\n
Third Quarter 2025 Financial Results<\/b><\/p>\n
\nScholar Rock reported a net loss of $102.2 million, including stock-based compensation of $18.3 million, for the quarter ended September 30, 2025, compared to a net loss of $64.5 million, including stock-based compensation of $9.7 million, for the quarter ended September 30, 2024. Net loss per common share was $0.90 for the quarter ended September 30, 2025 compared to $0.66 per common share for the quarter ended September 30, 2024.<\/p>\n
- \n
- \nThe Company did not record any revenue for the quarter ended September 30, 2025 or for the quarter ended September 30, 2024.<\/p>\n<\/li>\n
- \nResearch and development expense was $50.5 million, including $5.5 million in stock-based compensation, for the quarter ended September 30, 2025, compared to $48.7 million, including $4.4 million in stock-based compensation, for the quarter ended September 30, 2024. The increase of $1.8 million was primarily driven by investment in commercial manufacturing and launch readiness for apitegromab as well as employee related costs.<\/p>\n<\/li>\n
- \nGeneral and administrative expense was $53.1 million, including $12.8 million in stock-based compensation, for the quarter ended September 30, 2025, compared to $16.1 million, including $5.2 million in stock-based compensation, for the quarter ended September 30, 2024. The increase of $37.0 million was primarily driven by investments in infrastructure to support launch readiness for apitegromab, including an increase in employee related costs of $13.2 million, an increase in non-cash stock-based compensation expense of $7.6 million related to increased headcount, and an increase in professional services fees of $14.3 million. Additionally, there were one-time severance-related costs of $1.1 million.<\/p>\n<\/li>\n
- \nAs of September 30, 2025, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $369.6 million. This reflects net proceeds of $91.7 million from the sale of 2.77 million common shares and a drawdown of $50.0 million from the Company\u2019s debt facility. This cash, in addition to approximately $60 million anticipated to be available to Scholar Rock from common warrants that expire on December 31, 2025, is expected to be sufficient to fund operations into 2027.<\/p>\n<\/li>\n<\/ul>\n
Conference Call Information<\/b><\/p>\n
\nScholar Rock will host a conference call and webcast today, Friday, November 14, at 8:00 a.m. ET to review its third quarter 2025 financial results and discuss recent business updates. To access the live audio webcast, please go to \u201cEvents and Presentations\u201d in the Investors section of the Scholar Rock website at http:\/\/investors.scholarrock.com<\/a>.<\/p>\n
\n
\n Scholar Rock Holding Corporation<\/b><\/td>\n<\/tr>\n \n Condensed Consolidated Statements of Operations<\/b><\/td>\n<\/tr>\n \n (unaudited)<\/td>\n<\/tr>\n \n (in thousands, except share and per share data)<\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n Three Months Ended September 30,<\/b><\/td>\n \n Nine Months Ended September 30,<\/b><\/td>\n<\/tr>\n \n \n \n \n \n\u00a0<\/p>\n<\/td>\n
\n 2025<\/b><\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n 2024<\/b><\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n 2025<\/b><\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n 2024<\/b><\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Operating expenses<\/td>\n \n \n \n \n \n \n <\/tr>\n \n \n Research and development<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n50,486<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n48,719<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n161,565<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n134,185<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n \n General and administrative<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n53,064<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n16,061<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n131,184<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n48,512<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n Total operating expenses<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n103,550<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n64,780<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n292,749<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n182,697<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n Loss from operations<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(103,550<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(64,780<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(292,749<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(182,697<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n<\/tr>\n
\n Other income (expense), net<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n1,330<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n301<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n5,775<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n2,857<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n Net loss<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n(102,220<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(64,479<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(286,974<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(179,840<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Net loss per share, basic and diluted<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n(0.90<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(0.66<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(2.54<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(1.86<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Weighted average common shares outstanding, basic and diluted<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n113,729,850<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n97,050,637<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n112,763,974<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n96,587,149<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n
\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n Scholar Rock Holding Corporation<\/b><\/p>\n<\/td>\n<\/tr>\n
\n \n Condensed Consolidated Balance Sheets<\/b><\/td>\n<\/tr>\n \n \n (unaudited)<\/td>\n<\/tr>\n \n \n (in thousands)<\/td>\n<\/tr>\n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n September 30, 2025<\/b><\/td>\n \n December 31, 2024<\/b><\/td>\n<\/tr>\n \n Assets<\/b><\/td>\n \n \n <\/tr>\n \n \n Cash, cash equivalents and marketable securities<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n369,630<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n437,278<\/p>\n<\/td>\n<\/tr>\n
\n \n Other current assets<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n21,081<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n13,887<\/p>\n<\/td>\n<\/tr>\n
\n \n Total current assets<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n390,711<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n451,165<\/p>\n<\/td>\n<\/tr>\n
\n \n Other assets<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n21,007<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n23,757<\/p>\n<\/td>\n<\/tr>\n
\n Total assets<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n411,718<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n474,922<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Liabilities and Stockholders’ Equity<\/b><\/td>\n \n \n <\/tr>\n \n \n Current liabilities<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n61,972<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n46,936<\/p>\n<\/td>\n<\/tr>\n
\n \n Long-term liabilities<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n104,751<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n59,352<\/p>\n<\/td>\n<\/tr>\n
\n \n Total liabilities<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n166,723<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n106,288<\/p>\n<\/td>\n<\/tr>\n
\n \n Total stockholders’ equity<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n244,995<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n368,634<\/p>\n<\/td>\n<\/tr>\n
\n Total liabilities and stockholders’ equity<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n411,718<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n474,922<\/p>\n<\/td>\n<\/tr>\n<\/table>\n
\n\u00a0<\/p>\n
<\/p>\n
View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20251114762340\/en\/<\/a><\/span><\/p>\n
Investor Contact
\n<\/b>
Laura Ekas, Ph.D.
\n
Scholar Rock
\n
ir@scholarrock.com
\n<\/a>
917-439-0374<\/p>\nMedia Contact
\n<\/b>
Molly MacLeod
\n
media@scholarrock.com
\n<\/a>
802-579-5995<\/p>\nKEYWORDS:<\/b> Massachusetts United States North America<\/p>\n
INDUSTRY KEYWORDS:<\/b> Biotechnology FDA Health Genetics Pharmaceutical<\/p>\n
- FDA cleared IND in Q4 2025.<\/b> Scholar Rock expects to initiate dosing of SRK-439 in healthy volunteers in the fourth quarter of 2025.\n<\/li>\n<\/ul>\n
- Accelerated <\/b>timelines with second fill-finish facility.<\/b> Scholar Rock has accelerated timelines with an additional fill-finish facility for apitegromab. The facility is a commercially approved facility that is based in the U.S. and has a proven track record of successful site inspections. Tech transfer is underway, and commercial capacity has been reserved beginning in the first quarter of 2026.\n<\/li>\n
- Catalent Indiana, LLC (part of <\/i>Novo <\/i>Nordisk) participated in the Type A meeting in person and presented the progress made in implementing the remediation plan; confirmed to FDA that the site is on-track to be reinspection ready by the end of this year<\/i><\/li>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20251114762340\/en\/2643887\/3\/Scholar_Rock_Logo.jpg\"){kind=logo}
<\/td>\n<\/tr>\n