Apitegromab meets Phase 3 primary endpoint in SMA; Scholar Rock plans BLA/MAA filing Q1 2025

Scholar Rock Holding Corp

Primary endpoint met: apitegromab improved HFMSE vs placebo (mean diff 1.8 points, p=0.0192) in patients ages 2-12.
30.4% of apitegromab patients had >3 point HFMSE improvement vs 12.5% on placebo.
Benefit observed from first time point at 8 weeks, sustained to 52 weeks.
Safety profile consistent with prior trials; no SAEs related to apitegromab.
Company plans to submit BLA to FDA and MAA to EMA in Q1 2025.

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