Scholar Rock Holding Corporation announced positive topline results from its Phase 3 SAPPHIRE clinical trial for apitegromab, an investigational muscle-targeted therapy for spinal muscular atrophy (SMA). The trial successfully met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in motor function.
Patients treated with apitegromab, who were already receiving SMN-targeted treatments, showed a 1.8-point improvement on the Hammersmith Functional Motor Scale-Expanded (HFMSE) compared to placebo at 52 weeks. This outcome highlights apitegromab's potential to address the unmet need for therapies that directly target muscle health in SMA.
The company plans to leverage these positive results to seek U.S. and European Union regulatory approvals for apitegromab in the first quarter of the upcoming year. This marks a significant step towards potentially bringing a new treatment option to SMA patients.
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