Scholar Rock presented new efficacy and safety data from its pivotal Phase 3 SAPPHIRE trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The data reinforced apitegromab's potential to provide clinically meaningful improvement in motor function for people with spinal muscular atrophy (SMA) receiving SMN-targeted treatments.
The new analysis showed clinically meaningful and consistent benefit in motor function across pre-specified patient subgroups, including different SMN therapy types, age at initiation of SMN background therapy, and geographic region. Efficacy was also consistent across multiple motor function measures, including HFMSE, RULM, and WHO motor development milestones.
A key highlight was that 30% of apitegromab-treated patients achieved an additional 3-point or greater improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores, compared to only 12.5% in the placebo group. This demonstrates a significant gain in function on top of existing therapies.
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