-= Sarepta Therapeutics (SRPT) : Disappointing gene-therapy study results

 

Sarepta Therapeutics announces top-line results for Part 1 of Study 102; primary functional endpoint did not achieve statistical significance

Sarepta Therapeutics today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy, and tolerability of a single dose of SRP-9001 in 41 patients with Duchenne muscular dystrophy.

• SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

At 12 weeks post-treatment compared to baseline, the study met its primary biological endpoint of micro-dystrophin protein expression.

• Participants who received SRP-9001 had mean micro-dystrophin expression of 28.1%, as measured by western blot.
• Accompanying secondary biological endpoints including vector genome copies per nucleus, percent positive fibers, intensity, and reduction in creatine kinase (exploratory) were also met.

In the primary functional endpoint, SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the difference was not statistically significant.

• At every time point measured, the cohort of SRP-9001 treated participants outperformed the placebo group, and, at 48 weeks, participants in the treatment group demonstrated a statistically significant increase of 1.7 points in NSAA total score compared to baseline, while participants in the placebo group saw an increase of 0.9 points on the NSAA total score compared to baseline, which was not statistically significant.

In the pre-specified analysis by age-group, by which the randomization was stratified, participants aged 4-5 years at time of treatment with SRP-9001 demonstrated a statistically significant improvement in NSAA total score versus the age-matched placebo cohort, achieving a 4.3-point improvement on NSAA at 48 weeks post-treatment from baseline.

The results from Study 102 reinforce the "favorable" safety and tolerability profile of SRP-9001, with no new safety signals identified.