Business Wire

Scholar Rock Announces Design of Phase 3 SAPPHIRE Clinical Trial Evaluating Apitegromab in Non-Ambulatory Patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA)

– Phase 3 trial is a randomized, double-blind, placebo-controlled trial of apitegromab as add-on to background SMN therapy in non-ambulatory Type 2/3 SMA – Trial design is informed by the positive ...
Nasdaq

Scholar Rock to Present Additional Clinical Data from the Phase 3 SAPPHIRE Trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Oral presentation to include additional efficacy data from the Phase 3 SAPPHIRE trial of apitegromab in patients with spinal muscular atrophy (SMA) Poster presentations include preclinical data on a ...
Business Wire

Scholar Rock to Present Phase 3 SAPPHIRE Trial Design at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022)

“Enrollment in the ongoing pivotal Phase 3 SAPPHIRE trial, designed to further evaluate the potential of apitegromab in people with non-ambulatory Types 2 and 3 SMA, is progressing, and we are ...
Nasdaq

Scholar Rock Presents New Phase 3 SAPPHIRE Data at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

Primary endpoint showed clinically meaningful improvement in patients with SMA receiving apitegromab as measured by gold standard Hammersmith Functional Motor Scale Expanded (HFMSE) versus placebo ...