Scholar Rock’s SMA treatment trials show promising results

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

Scholar Rock Holding Corporation has announced encouraging results from its Phase 3 SAPHIRE trial, showing significant improvements in motor function in patients with spinal muscular atrophy (SMA) treated with apitegromab, a myostatin-inhibiting antibody. The study met its primary endpoint: patients showed early and sustained improvements in their motor skills. Based on these positive results, Scholar Rock is preparing to submit license applications in both the US and EU in early 2025. The safety profile of the treatment was positive and consistent with previous Phase 2 results. More detailed results will be presented at an upcoming medical conference.

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