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SAREPTA THERAPEUTICS 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 1/7/21 -- Study met the primary biological endpoint of micro - dystrophin protein expression at 12 weeks post - treatment , as measured by western blot , in SRP - 9001 - treated participants versus placebo -- -- SRP - 9001 - treated participants showed an increase in NSAA total score compared to placebo at 48 weeks ; however , the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post - treatment -- -- 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 -- -- No new safety signals identified for SRP - 9001 , reinforcing the favorable safety profile observed to date -- -- Sarepta to host conference call at 4:30 p.m. Eastern time -- CAMBRIDGE , Mass . , Jan. 07 , 2021 ( GLOBE NEWSWIRE ) -- Sarepta Therapeutics , Inc. ( NASDAQ : SRPT ) , the leader in precision genetic medicine for rare diseases , 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 ( rAAVrh74.MHCK7.micro - dystrophin ) 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 ( P < 0.0001 ) . Participants who received SRP - 9001 ( n = 20 ) 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 ( P = 0.37 ) . 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 ( P = 0.009 ) , 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 ( n = 21 , P = 0.1411 ) . 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 . "