Scholar Rock Stock Jumps On Additional Data From Pivotal Trial Of Muscular Dystrophy Treatment

Scholar Rock Holding Corp. SRRK said on Sunday the company will present new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.

The Phase 3 SAPPHIRE trial evaluated the efficacy and safety of apitegromab, an investigational muscle-targeted therapy being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving SMN-targeted treatments.

Also Read: Scholar Rock Sets Sights on $2 Billion Apitegromab Revenue Amid Spinal Muscular Atrophy Market Growth

The topline data released in October 2024 showed the study achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in motor function for apitegromab versus placebo in SMA patients on chronic dosing of standard-of-care therapies.

New analysis performed in the pooled population (ages 2-21) showed clinically meaningful and consistent improvement in HFMSE across pre-specified subgroups (type of SMN-targeted therapy, age at SMN-targeted therapy initiation) and geographic region.

For secondary endpoints measured on patients ages 2-12 receiving apitegromab (10 mg/kg and 20 mg/kg) or placebo, the following improvements were observed:

• A greater proportion of patients treated with apitegromab had improvements of ≥3 points in their HFMSE scores compared to placebo with odds ratio of 3.0 (nominal p-value = 0.0256).
• Additionally, 30.4% of patients receiving apitegromab had ≥ 3-point improvement in HFMSE versus 12.5% of patients on placebo (nominal p-value= 0.0156).
• Consistent improvement in motor function with a greater proportion of participants on apitegromab achieving HFMSE improvements versus placebo across all five-point thresholds at 52 weeks.

Treatment with apitegromab was well-tolerated across all age groups, consistent with the established safety profile and with no clinically relevant differences by dose.

SRRK Price Action: Scholar Rock Holding stock is up 0.87% at $34.63 at publication Monday.

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