Scholar Rock’s Apitegromab Shows Significant Improvement in Spinal Muscular Atrophy Treatment

Clinical Success:
Scholar Rock's experimental add-on treatment, apitegromab, has shown significant and clinically meaningful improvement in motor function for patients with spinal muscular atrophy (SMA) in a Phase 3 clinical trial.

Primary Endpoint:
The study met its primary endpoint, demonstrating a statistically significant improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) at week 52 compared to placebo.

Motor Function Outcomes:
30.4% of patients receiving apitegromab had a >3 point improvement in HFMSE versus 12.5% of patients on placebo, indicating early and sustained motor function improvement.

Safety Profile:
The treatment showed a favorable safety profile consistent with the long-term safety profile observed in the Phase 2 TOPAZ trial.

Regulatory Plans:
Scholar Rock plans to submit a U.S. Biologics License Application and a European Union marketing authorization application in Q1 2025.

Impact:
The positive results mark a critical milestone for the SMA community, offering a potentially transformative treatment that could drive clinically meaningful improvements in motor function across a broad SMA population.