Scholar Rock's Apitegromab Achieves Phase 3 Success, Boosting Stock and Future Prospects

Scholar Rock's apitegromab achieved significant success in its Phase 3 SAPPHIRE trial for spinal muscular atrophy (SMA), resulting in a substantial improvement in motor function among patients aged 2-12[1]. The trial met its primary endpoint, with a 1.8-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE) compared to placebo, marked by a statistically significant p-value of 0.0192[1][2]. Scholar Rock is now preparing for regulatory submissions in the U.S. and Europe, aiming to launch apitegromab by early 2025. This breakthrough could potentially transform the therapeutic landscape for SMA, with analysts noting the drug's capacity to boost Scholar Rock's regulatory standing and market prospects[3].