- 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.
- Related: Scholar Rock Shares Jumps On Updating Strategic Priorities.
- 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.
- Price Action: SRRK is trading higher by 26.75% at $6.16 during the market session on the last check Friday.
