Business
Scholar Rock Reports First Quarter 2025 Financial Results and Highlights Business Progress
Apitegromab BLA accepted under priority review for patients with Spinal Muscular Atrophy (SMA) and FDA’s PDUFA date set for September 22, 2025; commercial
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n\nApitegromab BLA accepted under priority review for patients with Spinal Muscular Atrophy (SMA) and FDA’s PDUFA date set for September 22, 2025; commercial readiness progresses\n\n\n\nEuropean Medicines Agency validated Marketing Authorisation Application (MAA) for apitegromab for patients with SMA\n\n\n\nTopline results from Phase 2 EMBRAZE proof-of-concept trial in adult patients with obesity on track for June 2025\n\n\n\nCash, cash equivalents and marketable securities of $364.4 million as of March 31, 2025; expected to support commercial and development programs into 2027\n\n\n\nManagement to host update call today at 8:15 a.m. ET\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (SMA) and other severe and debilitating neuromuscular diseases, today reported financial results and updates for the first quarter ended March 31, 2025.\n\n“Scholar Rock is at an inflection point as we evolve to a commercial-stage biopharmaceutical company, and our team remains focused on key priorities in preparation for the potential U.S. launch of apitegromab in Q3 2025,” said David L. Hallal, Chief Executive Officer of Scholar Rock. “With the FDA granting our BLA priority review and review of the MAA underway in Europe, I am delighted we have bolstered our leadership with the appointments of Akshay Vaishnaw, Keith Woods and Vikas Sinha as we scale our operations to serve patients with SMA in the U.S., Europe and in many additional countries around the world.”\n\nCompany Highlights and Upcoming Milestones\n\nSpinal Muscular Atrophy (SMA) Program\n\nApitegromab is an investigational monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Apitegromab is the only muscle-targeted therapeutic candidate for patients with SMA to demonstrate statistically significant improvement and clinically meaningful benefit in a pivotal Phase 3 (SAPPHIRE) clinical trial.\n\n\nGranted priority review for BLA. In March 2025, the FDA accepted the Company’s BLA for apitegromab for people with SMA and granted the application Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. In anti...