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Scholar Rock to Present Comprehensive Update at 2025 Annual Cure SMA Research and Clinical Care Meeting, Including Positive Results from Pivotal Phase 3 SAPPHIRE Trial
Oral presentation on the positive Phase 3 SAPPHIRE trial of apitegromab for people living with spinal muscular atrophy (SMA), which demonstrated
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n\nOral presentation on the positive Phase 3 SAPPHIRE trial of apitegromab for people living with spinal muscular atrophy (SMA), which demonstrated statistically significant improvement and clinically meaningful benefit as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE)\n\n\n\nApitegromab Biologics License Application (BLA) accepted under priority review for patients with SMA, and U.S. Food and Drug Administration (FDA) Prescription Drug User Fee Act (PDUFA) date set for September 22\n\n\n\nEuropean Medicines Agency (EMA) validated Marketing Authorisation Application (MAA) for apitegromab for patients with SMA\n\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, announced today that it will present data from its Phase 3 SAPPHIRE clinical trial (NCT05156320) in an oral presentation at Cure SMA’s Annual SMA Research & Clinical Care Meeting being held June 25-27, 2025, in Anaheim, California.\n\n\nSAPPHIRE evaluated the safety and efficacy of apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments. Scholar Rock previously shared positive topline data from the trial in October 2024 and announced in March 2025 that the U.S. FDA accepted its BLA for apitegromab. The application has a priority review designation, and the FDA has assigned a PDUFA target action date of September 22, 2025.\n\n\nDetails of the oral presentation are as follows:\n\n\nTitle: Efficacy and Safety of Apitegromab in Individuals with Type 2 and Type 3 Spinal Muscular Atrophy Evaluated in the Phase 3 SAPPHIRE Trial\nPresentation type: Oral presentation\nPresenter: Basil T. Darras, M.D., Associate Neurologist-in-Chief, Boston Children’s Hospital and Professor of Neurology, Harvard Medical School\nLocation: Sequoia Ballroom, Disney’s Grand Californian Hotel\nDate and time: Friday, June 27, 11:20 a.m. PDT\n\n\nScholar Rock will also present a poster on a systematic literature review of characterization of the longer-term effectiveness of SMN-targeted therapies for SMA. The det...