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Highlights:
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Scholar Rock announced positive topline results from the Phase 3 SAPPHIRE clinical trial for apitegromab, showing a statistically significant improvement in motor function in patients with Spinal Muscular Atrophy (SMA).
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The trial met its primary endpoint, with 30.4% of patients on apitegromab demonstrating a greater than 3-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to 12.5% in the placebo group.
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Scholar Rock plans to submit regulatory applications for apitegromab in the U.S. and EU in the first quarter of 2025.
Scholar Rock {NASDAQ:SRRK} has announced encouraging topline results from its Phase 3 SAPPHIRE clinical trial, which evaluated apitegromab, an investigational muscle-targeted therapy for patients with Spinal Muscular Atrophy (SMA). The trial successfully met its primary endpoint, indicating a statistically significant and clinically meaningful improvement in motor function, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE).
The trial's findings revealed that 30.4% of patients receiving apitegromab experienced a greater than 3-point improvement in their HFMSE scores, in contrast to only 12.5% of patients on placebo. Furthermore, early improvements in motor function were noted as early as eight weeks into the treatment, with benefits continuing to expand through the 52-week mark. The results demonstrated a clinically meaningful advantage across all age groups studied, ranging from 2 to 21 years.
In addition to its efficacy, apitegromab showcased a favorable safety profile consistent with the long-term data previously observed in the Phase 2 TOPAZ trial. Given these promising results, Scholar Rock plans to advance the drug's development by submitting regulatory applications in both the U.S. and the European Union during the first quarter of 2025. This potential regulatory pathway underscores the company's commitment to providing innovative treatments for individuals affected by SMA.
