Sarepta Therapeutics Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy - Seite 2

Study randomization was stratified by age group and, in the pre-specified analysis of participants aged 4-5 (n=16) at the time of treatment, the treatment group demonstrated a statistically significant 4.3-point improvement on NSAA total score at 48 weeks post treatment compared to a 1.9-point improvement in the age-matched placebo group (P=0.0172). The functional status at baseline for participants in the 4-5 age group was balanced across the placebo and treatment cohorts. A statistically significant imbalance (P=0.0046) in baseline NSAA total score was present in the cohort of 6-7-year-old participants (n=25), resulting in milder participants in the placebo arm (n=13) than in the treated arm (n=12). The significantly different baseline characteristics between treatment and control groups in the 6-7 age group may have contributed to the inability to observe a treatment effect in the 6-7 age group at the week 48 timepoint in Part 1.

The results from Study 102 reinforce the favorable safety and tolerability profile of SRP-9001 with no new safety signals identified. In line with previously reported clinical data, no clinical complement activation was observed. 85% of participants in the treatment group experienced at least one treatment-related adverse event compared to 43% in the placebo group. Among participants with treatment-related adverse events, 82% were mild or moderate in severity, and 4 participants experienced serious treatment-related adverse events including 3 participants in the treatment group (2 cases of rhabdomyolysis, 2 transaminase elevations) and 1 participant in the placebo (rhabdomyolysis).

Study 102 is ongoing and remains blinded to participants, investigators, site staff and sponsor staff with direct site interaction. All 41 participants have completed their Part 1, 48-week assessment and have entered the Part 2 crossover phase. Participants continue to be monitored for safety and will undergo another biopsy at week 12 in Part 2 to assess expression and biological markers, in addition to longer-term assessments of functional outcomes.

“Study 102 reinforces our confidence in the potentially transformative benefits of SRP-9001, including among other things, the fact that in the Study’s pre-specified analysis, the participants in the 4-5 age group robustly achieved a statistically significant and clinically meaningful improvement in NSAA over placebo, as predicted by our prior Study 101. For the entire population, while we saw separation at every time point between the active and placebo cohorts, Study 102 did not achieve statistical significance on the primary functional endpoint. In this regard, we are very disappointed that the randomization process resulted in a significant imbalance in baseline NSAA scores between the active and placebo cohorts of the participants ages 6-7, making the 6-7 age groups non-comparable and likely substantially contributing to the inability to achieve statistical significance,” said Doug Ingram, president and chief executive officer, Sarepta. “Study 102 remains blinded and we will analyze the functional results for all patients, including cross-over participants, once they have achieved the 48-week timepoint in Part 2. We have already enrolled and dosed 11 participants in Study 103, using our commercial process material, and we will have biomarker and safety results from that cohort in the second quarter. And very importantly, Study 102 has provided us with a wealth of information and insight which we will use to refine and complete the protocol for our upcoming trial using commercial process material. We intend to continue to move forward with diligence and urgency to generate the evidence necessary to bring SRP-9001 to waiting Duchenne patients around the world.”