Scholar Rock Inventory Jumps On Extra Information From Pivotal Trial Of Muscular Dystrophy Remedy – Scholar Rock Holding (NASDAQ:SRRK)

Scholar Rock Holding Corp. SRRK stated on Sunday the corporate will current new efficacy and security information from the Part 3 pivotal SAPPHIRE trial on the 2025 Muscular Dystrophy Affiliation (MDA) Scientific & Scientific Convention.

The Part 3 SAPPHIRE trial evaluated the efficacy and security of apitegromab, an investigational muscle-targeted remedy being developed to supply clinically significant enchancment in motor operate for individuals dwelling with spinal muscular atrophy (SMA) who’re receiving SMN-targeted remedies.

Additionally Learn: Scholar Rock Units Sights on $2 Billion Apitegromab Income Amid Spinal Muscular Atrophy Market Progress

The topline information launched in October 2024 confirmed the examine achieved its major endpoint, demonstrating a statistically vital and clinically significant enchancment in motor operate for apitegromab versus placebo in SMA sufferers on continual dosing of standard-of-care therapies.

New evaluation carried out within the pooled inhabitants (ages 2-21) confirmed clinically significant and constant enchancment in HFMSE throughout pre-specified subgroups (sort of SMN-targeted remedy, age at SMN-targeted remedy initiation) and geographic area.

For secondary endpoints measured on sufferers ages 2-12 receiving apitegromab (10 mg/kg and 20 mg/kg) or placebo, the next enhancements have been noticed:

• A larger proportion of sufferers handled with apitegromab had enhancements of ≥3 factors of their HFMSE scores in comparison with placebo with odds ratio of three.0 (nominal p-value = 0.0256).
• Moreover, 30.4% of sufferers receiving apitegromab had ≥ 3-point enchancment in HFMSE versus 12.5% of sufferers on placebo (nominal p-value= 0.0156).
• Constant enchancment in motor operate with a larger proportion of individuals on apitegromab attaining HFMSE enhancements versus placebo throughout all five-point thresholds at 52 weeks.

Remedy with apitegromab was well-tolerated throughout all age teams, according to the established security profile and with no clinically related variations by dose.

SRRK Value Motion: Scholar Rock Holding inventory is up 0.87% at $34.63 at publication Monday.

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