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SAREPTA THERAPEUTICS Sarepta Therapeutics ' SRP - 9001 Shows Sustained Functional Improvements in Multiple Studies of Patients with Duchenne 10/11/21 • Results presented at ' Micro - dystrophin Day ' highlight breadth , depth and strength of the clinical evidence to date for SRP - 9001 in treating Duchenne muscular dystrophy ; tolerability profile remains consistent across treated patients • Results from Study SRP - 9001-101 found that SRP - 9001 - treated participants ( n = 4 , ages 4 to 7 years ) improved 8.6 points on the North Star Ambulatory Assessment ( NSAA ) compared to a matched natural history cohort three years after treatment • Results from Study SRP - 9001-102 found that SRP - 9001 - treated participants ( n = 12 , ages 6 to 7 ) had a positive 2.9 - point difference on NSAA compared to a matched natural history cohort one year after treatment • Functional results from Study SRP - 9001-103 Cohort 1 ( n = 11 , ages 4-7 ) found participants improved 3.0 points on NSAA six months after treatment • EMBARK , the first global Phase 3 pivotal double - blind gene therapy trial in Duchenne to enroll 120 patients in the US , Europe and Asia , conducted in partnership with Roche CAMBRIDGE , Mass . , Oct. 11 , 2021 ( GLOBE NEWSWIRE ) -- Sarepta Therapeutics , Inc. ( NASDAQ : SRPT ) , the leader in precision genetic medicine for rare diseases , today shared new analyses and functional data from its SRP - 9001 ( rAAVrh74.MHCK7.micro - dystrophin ) development program and details of Study SRP - 9001-301 , known as EMBARK , its global pivotal Phase 3 trial of SRP - 9001 for the treatment of Duchenne muscular dystrophy . SRP - 9001 , being developed in partnership with Roche , 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 . In new analyses presented at " SRP - 9001 Micro - dystrophin Day , " results from participants treated with SRP - 9001 in Study SRP - 9001-101 ( n = 4 , ages 4 to 7 ) found that participants in Study 101 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 ( p < 0.0001 ) . In Study SRP - 9001-102 , 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 ( p = 0.0129 ) . In addition , the first functional results were presented from Study SRP - 9001-103 ( ENDEAVOR ) , which uses commercially representative SRP - 9001 material . Results from 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 . " With 77 patients treated to date , the multi - study development program for SRP - 9001 represents the most comprehensive and long - term dataset for a Duchenne muscular dystrophy gene therapy in existence . The totality of evidence shows that SRP - 9001 is a significantly differentiated gene therapy product candidate with one - time dosing and a stable tolerability profile , results in robust expression and evidence of sustained functional benefits across our various studies , " said Doug Ingram , president and chief executive officer , Sarepta . " We commence our EMBARK pivotal trial - currently the only truly global Phase 3 trial with a Duchenne gene therapy - with great conviction in the transformative potential of SRP - 9001 . But while our expression , tolerability , functional evidence and CMC achievements may place SRP - 9001 alone among potential therapies , we never forget that we remain in a daily race against a life - ending degenerative disease . To that end , Sarepta , along with our partner Roche , will continue working with tenacity and urgency to bring this potentially transformative treatment to individuals with Duchenne around the world . " " When compared to a matched natural history cohort , individuals with Duchenne who received SRP - 9001 are performing better on the NSAA or showing stabilization of NSAA scores where we would expect to see a decline . The functional results from Study 103 , as early as 6 months following treatment , provide additional confidence in our ability to confirm a treatment effect with SRP - 9001 as we advance our pivotal Phase 3 trial , " said Louise Rodino - Klapac , Ph.D. , executive vice president and chief scientific officer , Sarepta . " Given our experience with the AAVrh74 vector in more than 80 individuals with Duchenne and limb - girdle muscular dystrophy , we are very encouraged by the sustained and meaningful clinical results and consistency of the safety profile of SRP - 9001 . " The safety and tolerability profile of SRP - 9001 is similar to past reports . Across all three studies , treatment - related adverse events ( TRAEs ) generally occurred within 90 days of treatment and subsequently resolved . No clinically relevant complement activation was observed in any of the studies . The most common treatment - related adverse event was vomiting , generally within the first week post - infusion . Increases in liver enzymes were transient and responsive to steroids . In Study 9001-103 , safety data were consistent with data from previous studies of SRP - 9001 ( 101 and 102 ) . There was one immune - mediated myositis serious adverse event in Cohort 2 specific to the participant's mutation ; participant received treatment including plasmapheresis and has since returned to pre - event function . About the SRP - 9001 Clinical Development Program The SRP - 9001 clinical development program currently consists of four studies :