- \n
- FDA accepted apitegromab Biologics License Application (BLA) for treatment of children and adults with spinal muscular atrophy (SMA) with September 30, 2026 Prescription Drug User Fee Act (PDUFA) action date<\/i><\/li>\n
- Accepted apitegromab BLA includes two fill-finish facilities, Catalent Indiana LLC (part of Novo Nordisk), and a second U.S.-based facility<\/i><\/li>\n
- FDA has completed reinspection of Catalent Indiana; classification of facility expected within 90 days following reinspection, in accordance with FDA guidelines<\/i><\/li>\n
- Second fill-finish facility on track to have commercial apitegromab supply in early Q3 2026<\/i><\/li>\n
- Scholar Rock is prepared for U.S. apitegromab launch immediately upon FDA approval, which may be granted at any time through September 30, 2026<\/i><\/li>\n
- Cash, cash equivalents, and marketable securities of $480 million as of March 31, 2026; includes an additional $100 million in debt and $98 million in net cash proceeds from the Company\u2019s at-the-market (ATM) program<\/i><\/li>\n
- Management to host a 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 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 first quarter ended March 31, 2026, and provided an update on recent company developments.<\/p>\n\n\u201cWith the FDA\u2019s acceptance of our apitegromab BLA, we have achieved another critical milestone as we work with urgency to deliver on our mission to bring the world\u2019s first muscle-targeted treatment to the SMA community,\u201d said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. \u201cWe are grateful for the FDA\u2019s continued high level of engagement, and we are pleased that important progress continues to be made at both of our fill-finish facilities. Our U.S. commercial team stands ready to launch apitegromab on or at any time prior to the September 30th<\/sup> PDUFA date.\u201d<\/p>\n
\nMr. Hallal continued, \u201cOur balance sheet is strong, our clinical-stage pipeline continues to advance, and we are poised, now more than ever, to usher in the next phase of innovation for patients with SMA.\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 a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE).<\/p>\n
SMA Program<\/b><\/p>\n
- \n
- Apitegromab BLA accepted by FDA with September 30, 2026 PDUFA action date. <\/b>The accepted BLA includes two fill-finish facilities, Catalent Indiana and a second U.S.-based fill-finish facility.\n<\/li>\n
- FDA completed reinspection of Catalent Indiana. <\/b>Following the FDA\u2019s acceptance of the apitegromab BLA, the Agency completed reinspection of Catalent Indiana. In accordance with FDA guidelines, classification of the facility is anticipated within 90 days following reinspection.\n<\/li>\n
- Significant progress continues at second fill-finish facility. <\/b>Apitegromab commercial supply from this facility is expected to be available early in the third quarter of 2026.\n<\/li>\n
- Preparations ongoing for U.S. commercial launch. <\/b>The Commercial team continues to expand its reach and engagement with key stakeholders, including a significant presence at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which was held March 8 \u2013 11, 2026 in Orlando, FL and at the upcoming 2026 Annual SMA Conference, which is being held June 25 \u2013 28, 2026 in Orlando, FL. The U.S. commercial team is prepared to launch apitegromab immediately upon FDA approval.\n<\/li>\n
- European Medicines Agency (EMA)<\/b>regulatory review ongoing. <\/b>A Committee for Medicinal Products for Human Use (CHMP) opinion for the apitegromab Marketing Authorisation Application (MAA) is anticipated near mid-2026. The Scholar Rock team in Europe continues to engage with key stakeholders on SMA disease awareness and education initiatives, including at the 5th<\/sup> International Scientific Congress on SMA, which was held March 11 \u2013 14, 2026 in Budapest, Hungary. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany.\n<\/li>\n
- Enrollment progressing in Phase 2 OPAL clinical trial<\/b>. Patients continue to be enrolled and dosed in the Phase 2 OPAL study (NCT07047144). The trial is evaluating apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy.\n<\/li>\n
- Subcutaneous apitegromab development continues to progress. <\/b>Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed. Further development activities are ongoing, including anticipated FDA and EMA regulatory engagements following apitegromab approvals.\n<\/li>\n<\/ul>\n
FSHD Program<\/b><\/p>\n
- \n
- Phase 2 FORGE trial on track for initiation in mid-2026. <\/b>Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. Initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, is expected in mid-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
- Phase 1 healthy volunteer study ongoing. <\/b>A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026.\n<\/li>\n<\/ul>\n
First Quarter 2026 Financial Results<\/b><\/p>\n
\nScholar Rock reported a net loss of $105.5 million, including stock-based compensation of $18.2 million, for the quarter ended March 31, 2026, compared to a net loss of $74.7 million, including stock-based compensation of $13.4 million, for the quarter ended March 31, 2025. Net loss per common share was $0.83 for the quarter ended March 31, 2026, compared to $0.67 per common share for the quarter ended March 31, 2025.<\/p>\n
- \n
- \nThe Company did not record any revenue for the quarters ended March 31, 2026 and 2025.<\/p>\n<\/li>\n
- \nResearch and development expense was $51.8 million, including $6.5 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $48.7 million, including $4.0 million in stock-based compensation, for the quarter ended March 31, 2025.<\/p>\n<\/li>\n
- \nGeneral and administrative expense was $50.2 million, including $11.7 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $28.4 million, including $9.4 million in stock-based compensation, for the quarter ended March 31, 2025.<\/p>\n<\/li>\n
- \nAs of March 31, 2026, Scholar Rock had cash, cash equivalents, and marketable securities of $479.9 million. This reflects a drawdown of $100.0 million from the Company\u2019s debt facility and net cash proceeds of $98.0 million from the Company\u2019s at-the-market (ATM) program.<\/p>\n<\/li>\n<\/ul>\n
Conference Call Information<\/b><\/p>\n
\nScholar Rock will host a conference call and webcast today, Thursday, May 7, at 8:00 a.m. ET to review its first quarter 2026 financial results and discuss recent business updates. To access the live audio webcast, please go to \u201cEvents and Presentations\u201d in the Investors section of the Scholar Rock website at http:\/\/investors.scholarrock.com<\/a>.<\/p>\n
\n
\n \u00a0<\/td>\n<\/tr>\n \n Scholar Rock Holding Corporation<\/b><\/td>\n<\/tr>\n \n Condensed Consolidated Statements of Operations<\/b><\/td>\n<\/tr>\n \n (unaudited)<\/td>\n<\/tr>\n \n (in thousands, except share and per share data)<\/td>\n<\/tr>\n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n Three Months Ended March 31,<\/b><\/td>\n<\/tr>\n \n \n \n \n\u00a0<\/p>\n<\/td>\n
\n 2026<\/b><\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n 2025<\/b><\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Operating expenses<\/td>\n \n \n <\/tr>\n \n Research and development<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n51,814<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n48,678<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n General and administrative<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n50,202<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n28,412<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n Total operating expenses<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n102,016<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n77,090<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n Loss from operations<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(102,016<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(77,090<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n<\/tr>\n
\n Other income (expense), net<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n(3,494<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n2,367<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n Net loss<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n(105,510<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(74,723<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Net loss per share, basic and diluted<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n(0.83<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n(0.67<\/p>\n<\/td>\n
\n \n)<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Weighted average common shares outstanding, basic and diluted<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n127,277,144<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n111,838,272<\/p>\n<\/td>\n
\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n<\/table>\n \n
\n <\/tr>\n \n \n Scholar Rock Holding Corporation<\/b><\/p>\n<\/td>\n<\/tr>\n
\n \n Condensed Consolidated Balance Sheets<\/b><\/p>\n<\/td>\n<\/tr>\n
\n \n \n(unaudited)<\/p>\n<\/td>\n<\/tr>\n
\n \n \n(in thousands)<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n March 31, 2026<\/b><\/td>\n \n December 31, 2025<\/b><\/td>\n<\/tr>\n \n Assets<\/b><\/td>\n \n \n <\/tr>\n \n Cash, cash equivalents and marketable securities<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n479,944<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n367,563<\/p>\n<\/td>\n<\/tr>\n
\n Other current assets<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n35,340<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n17,584<\/p>\n<\/td>\n<\/tr>\n
\n Total current assets<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n515,284<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n385,147<\/p>\n<\/td>\n<\/tr>\n
\n Other assets<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n19,970<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n19,125<\/p>\n<\/td>\n<\/tr>\n
\n Total assets<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n535,254<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n404,272<\/p>\n<\/td>\n<\/tr>\n
\n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Liabilities and Stockholders’ Equity<\/b><\/td>\n \n \n <\/tr>\n \n Current liabilities<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n59,643<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n55,419<\/p>\n<\/td>\n<\/tr>\n
\n Long-term liabilities<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n199,598<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n103,365<\/p>\n<\/td>\n<\/tr>\n
\n Total liabilities<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n259,241<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n158,784<\/p>\n<\/td>\n<\/tr>\n
\n Total stockholders’ equity<\/td>\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n276,013<\/p>\n<\/td>\n
\n \n \n\u00a0<\/p>\n<\/td>\n
\n \n245,488<\/p>\n<\/td>\n<\/tr>\n
\n Total liabilities and stockholders’ equity<\/td>\n \n \n$<\/p>\n<\/td>\n
\n \n535,254<\/p>\n<\/td>\n
\n \n \n$<\/p>\n<\/td>\n
\n \n404,272<\/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\/20260507365694\/en\/<\/a><\/span><\/p>\n
Investor Contact
\n<\/b>
Laura Ekas, Ph.D.
\n
ir@scholarrock.com
\n<\/a>
917-439-0374<\/p>\nMedia Contact
\n
<\/b>Molly MacLeod, Ph.D.
\n
media@scholarrock.com
\n<\/a>
802-579-5995<\/p>\nKEYWORDS:<\/b> Massachusetts United States North America<\/p>\n
INDUSTRY KEYWORDS:<\/b> Medical Supplies FDA Health Genetics Clinical Trials Biotechnology<\/p>\n
- Phase 1 healthy volunteer study ongoing. <\/b>A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026.\n<\/li>\n<\/ul>\n
- FDA completed reinspection of Catalent Indiana. <\/b>Following the FDA\u2019s acceptance of the apitegromab BLA, the Agency completed reinspection of Catalent Indiana. In accordance with FDA guidelines, classification of the facility is anticipated within 90 days following reinspection.\n<\/li>\n
- Accepted apitegromab BLA includes two fill-finish facilities, Catalent Indiana LLC (part of Novo Nordisk), and a second U.S.-based facility<\/i><\/li>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20260507365694\/en\/2723099\/3\/ScholarRock_Logo_Registered_Stacked_Color.jpg\"){kind=logo}
<\/td>\n<\/tr>\n