Sarepta's Duchenne Gene Therapy Shows Sustained Functional Improvements
- Sarepta Therapeutics Inc (NASDAQ: SRPT) shared new analyses and functional data from its SRP-9001 development program and the details of Study SRP-9001-301 (EMBARK) Phase 3 trial of SRP-9001 for Duchenne muscular dystrophy.
- SRP-9001, being developed in partnership with Roche Holdings AG (OTC: RHHBY), is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue.
- In new analyses of study 101 (n=4, ages 4 to 7), SRP-9001 treated participants improved 8.6 points on the North Star Ambulatory Assessment (NSAA) compared to a matched natural history cohort three years following a single administration of SRP-9001.
- In Study 102, SRP-9001 treated participants ages 6 to 7 (n=12) had a positive 2.9-point difference on NSAA change from baseline compared to a matched natural history control.
- The first 11 participants in Cohort 1, ages 4 to 7, demonstrated a 3.0-point improvement from baseline on NSAA six months after treatment.
- Related: Sarepta's Stock Trading Higher As Duchenne Muscular Dystrophy Gene Therapy Shows Promise In Open-Label Study.
- Price Action: SRPT shares are down 6.62% at $92.84 during the market session on the last check Monday.
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