Scholar Rock ( SRRK ) Holding Corp. 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.

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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 ( SRRK ) stock is up 0.87% at $34.63 at publication Monday.

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