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Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
Topline data from Phase 3 trial expected in Q4 2024 Company to present encore 36-month efficacy, safety, and patient-reported outcomes data from the Phase 2
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n\nTopline data from Phase 3 trial expected in Q4 2024\n\n\n\nCompany to present encore 36-month efficacy, safety, and patient-reported outcomes data from the Phase 2 TOPAZ trial extension at the 2023 World Muscle Society (WMS) Annual Congress\n\n\n\nApitegromab is the only muscle-targeted therapy candidate with clinical data showing proof of concept in spinal muscular atrophy\n\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, announced today that it has completed enrollment of the SAPPHIRE Phase 3 trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam). The company also announced that it plans to present encore data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes in SMA after 36 months of treatment with apitegromab. Efficacy and safety data at 36 months, and an analysis of patient-reported outcome measures such as fatigue, mobility, and activities of daily living, will be presented during two poster presentations at the 28th Annual Congress of the World Muscle Society from October 3 – 7 in Charleston, South Carolina, USA.\n\n\n“Completing SAPPHIRE enrollment is a critical step on the path to bringing apitegromab, a potentially transformative therapy, to individuals living with SMA. SAPPHIRE was designed to build on the positive TOPAZ results, and we look forward to reporting the topline results from this registrational study in Q4 next year,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “The 36-month data from TOPAZ, which reinforced long-term sustained and clinically meaningful improvement in motor function on top of SMN therapy, gives us strong conviction that apitegromab could provide substantial benefit beyond SMN therapy alone.”\n\n\nDetails of the poster presentations are as follows:\n\n\nTitle: Effect of apitegromab on PEDI-CAT and PROMIS-fatigue questionnaire at 36 months in patients with spinal muscular atrophy\nPresentation type: Poster presentation\nPresenter: Thomas O. Crawford, M.D., Professor of Neurology and Pediatrics, Johns Hopkins Univ...