Scholar Rock Holding Corp (NASDAQ: SRRK) announced new data from the Phase 2 TOPAZ trial extension period evaluating apitegromab in non-ambulatory patients with Types 2 and 3 Spinal Muscular Atrophy (SMA) receiving an SMN therapy.
The trial assessed patient outcomes after 24 months of treatment.
All 35 non-ambulatory patients (Cohorts 2 and 3) and 12 of 23 ambulatory patients (Cohort 1) were receiving Biogen Inc's (NASDAQ: BIIB) Spinraza (nusinersen) maintenance therapy.
The mean change from baseline in HFMSE was 4 points in pooled non-ambulatory patients at 24 months. At 12 months, the change stood at 3.6 points.
The HFMSE is a validated measure for assessing gross motor function in SMA.
The mean change from the baseline in RULM was 1.3 points at 12 months and 1.9 points at 24 months.
RULM is validated to evaluate upper limb motor performance.
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