Scholar Rock Presents New Phase 3 SMA Data

 Scholar Rock (NASDAQ: SRRK) announced new Phase 3 SAPPHIRE trial data for apitegromab, their investigational muscle-targeted therapy for Spinal Muscular Atrophy (SMA). The trial met its primary endpoint with statistically significant improvement in motor function, showing a mean difference of 1.8 points (p=0.0192) in Hammersmith Functional Motor Scale Expanded (HFMSE) versus placebo.

Key secondary endpoints revealed that 30.4% of patients receiving apitegromab achieved ≥3-point HFMSE improvement compared to 12.5% on placebo (p=0.0156). The treatment demonstrated consistent benefits across all subgroups and showed improvements in Revised Upper Limb Module (RULM) and WHO motor development milestones.

The drug was well-tolerated with no serious adverse events related to apitegromab, positioning it as a potential complementary therapy to existing SMN-targeted treatments.

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