Solid Biosciences Stock Gains After Duchenne Dystrophy Gene Therapy Data

  • Solid Biosciences Inc SLDB reports 1.5-year functional data and patient-reported outcome measures in the ongoing IGNITE DMD Phase 1/2 trial of SGT-001. 
  • SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy for Duchenne muscular dystrophy (DMD).
  • The data will be presented today at the World Muscle Society 2021 Virtual Congress.
  • The data demonstrated durable expression and function of microdystrophin protein after 12 to 24 months post-dosing.
  • Related Content: Solid Bio's DMD Gene Therapy Shows Promising Early Data, No New Safety Concerns, But Fails To Cheer Investors.
  • NSAA scores (measurement of functional motor abilities) showed minimal change compared with baseline and suggested benefit after treatment compared to trajectories typically observed in natural history data. 
  • Natural history analyses suggest that patients would usually exhibit a 4.5-point decline in NSAA over 1.5 years. 
  • In contrast, patients exhibited a mean decrease of 1.7 points from baseline and a mean difference of +2.8 points compared with natural history data.
  • 6-Minute Walk Test (6MWT) distances were maintained 1.5 years post-dosing, while natural history analyses suggested a 63.5-meter decline. 
  • The mean increase in the 6MWT was 15.3 meters compared with baseline, and the mean difference compared with natural history data was +78.8 meters.
  • The percent predicted FVC (lung function tests) continued to show stability or improvement, while natural history analyses suggest a decline of 7.5%. 
  • Patient-reported outcome measures showed meaningful, sustained improvements at 1.5 years compared with baseline.
  • No new drug-related safety findings were reported.
  • Price Action: SLDB stock is up 1% at $3.03 during the market session on the last check Thursday.
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