Scholar Rock’s SMA Treatment Trials Show Promising Results

Scholar Rock Holding ( (SRRK) ) has provided an announcement.

Scholar Rock Holding Corporation has announced promising results from its Phase 3 SAPPHIRE trial, showing significant improvements in motor function in patients with Spinal Muscular Atrophy (SMA) treated with apitegromab, a myostatin-inhibiting antibody. The trial met its primary endpoint, with patients exhibiting early and sustained enhancements in motor abilities. Following these positive outcomes, Scholar Rock is gearing up to submit licensing applications in both the U.S. and the EU in early 2025. The treatment’s safety profile was favorable, aligning with previous Phase 2 findings, and further detailed results will be shared at an upcoming medical conference.

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