Scholar Rock Holding SRRK inventory is buying and selling increased on Monday after the corporate launched topline outcomes from the Section 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy.
The examine achieved its main endpoint, demonstrating a statistically vital and clinically significant enchancment for apitegromab versus placebo in motor operate in SMA sufferers on continual dosing of standard-of-care therapies.
Customary therapies embody Biogen Inc‘s BIIB Spinraza (nusinersen) and Roche Holdings AG‘s RHHBY Evrysdi (risdiplam).
In the primary efficacy inhabitants (ages 2-12), the imply distinction in change from baseline in Hammersmith Purposeful Motor Scale Expanded (HFMSE) was 1.8 factors (p=0.0192) for all sufferers receiving apitegromab 10 mg/kg and 20 mg/kg (n=106) in comparison with placebo (n=50).
Sufferers receiving 20 mg/kg of apitegromab (n=53) confirmed a 1.4-point imply distinction in comparison with placebo (p=0.1149).
The prespecified evaluation of the ten mg/kg dose confirmed that sufferers receiving 10 mg/kg of apitegromab (n=53) improved by 2.2 factors (nominal p=0.0121) in comparison with placebo.
Motor operate outcomes have been significant and constant throughout the primary efficacy inhabitants, and the exploratory inhabitants ages 13-21 favored apitegromab (n=22) in comparison with placebo (n=10).
Thirty p.c of sufferers receiving apitegromab had a >3-point enchancment in HFMSE in comparison with 12.5% of sufferers on placebo.
Sufferers receiving apitegromab demonstrated early motor operate enchancment in comparison with placebo from the primary measured time level at 8 weeks, profit expanded at 52 weeks as measured by HFMSE.
Therapy with apitegromab was well-tolerated throughout all age teams.
The corporate plans to submit an FDA and European advertising functions within the first quarter of 2025.
Value Motion: SRRK inventory is up 320.2% to $31.43 ultimately verify Monday.
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