The biotech company Scholar Rock reported Monday that its experimental medicine improved motor function in patients with a muscle-wasting disease called spinal muscular atrophy, paving the way for the likely approval of the drug.
In a year-long Phase 3 trial, the company tested the drug, called apitegromab, on top of existing treatments for the disease. Among the main population studied, children ages 2 to 12, treated patients experienced a 1.8-point greater improvement versus the placebo group on a test known as the Hammersmith Functional Motor Scale.
Additionally, 30% of patients on apitegromab experienced a greater than 3-point improvement on the motor scale, compared with 12.5% of patients on placebo. Treated patients experienced greater improvement in motor function starting at eight weeks, the first time point that was measured, and the benefit continued to expand over the course of the year.
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