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SAREPTA THERAPEUTICS Sarepta Therapeutics Announces Second Quarter 2021 Financial Results and Recent Corporate Developments 8/4/21 • Sarepta announces successful completion of end - of - phase - 2 meeting for SRP - 9001 micro - dystrophin with FDA's Office of Tissues and Advanced Therapies ( OTAT ) and plans to initiate pivotal trial , SRP - 9001-301 ( Study 301 or EMBARK ) , in September of 2021 • Net product sales for the second quarter of 2021 reached $ 141.8 million , a 27 % increase over the same quarter of prior year • In light of its over - performance , Sarepta raises its full - year product revenue guidance by nearly $ 30 million to between $ 565 million to $ 575 million CAMBRIDGE , Mass . , Aug. 04 , 2021 ( GLOBE NEWSWIRE ) -- Sarepta Therapeutics , Inc. ( NASDAQ : SRPT ) , the leader in precision genetic medicine for rare diseases , today reported financial results for the second quarter of 2021 . " I am pleased to report that the Sarepta team continues to execute , serving our patient community and advancing our portfolio . In the second quarter , our net product revenue stood at $ 141.8 million , a 27 % increase over the same quarter last year , " stated Doug Ingram , Sarepta's president and chief executive officer . " We also advanced our broad genetic medicine portfolio . Significantly and representing years of work and enormous investment into process and analytical development for SRP - 9001 , our gene therapy for Duchenne muscular dystrophy , we announced very positive results from our first cohort of Study SRP - 9001-103 ( ENDEAVOR ) to test the performance of our commercially representative material . From there , we scheduled and have now completed a very productive and informative end - of - phase - 2 meeting with the FDA and on that basis we anticipate initiating Study SRP - 9001-301 ( EMBARK ) , our pivotal trial for SRP - 9001 in September of this year in the United States and globally . This is an extraordinarily important moment for our program and for the many families living with Duchenne . We intend to move as fast as science will permit to confirm the benefits of SRP - 9001 and , if successful , to rapidly advance this therapy to waiting Duchenne patients around the world . " Second Quarter 2021 and Recent Corporate Developments : • Sarepta announced that it concluded its end - of - phase - 2 meeting with OTAT : In July of 2021 , Sarepta completed a successful end - of - phase - 2 meeting with the United States Food and Drug Administration's Office of Tissues and Advanced Therapies ( OTAT ) regarding the Company's proposed pivotal trial , Study SRP - 9001-301 ( EMBARK ) , and the commercially representative material to be used in that trial . Based on the meeting with OTAT , Sarepta anticipates initiating Study 301 , as submitted to the Division , in September of this year . • Sarepta executes licensing agreement for gene therapy program from Nationwide Children's Hospital to treat limb - girdle muscular dystrophy type 2A : Today Sarepta announced that upon completion of a number of preclinical and safety studies , the Company executed its licensing agreement to an investigational gene therapy candidate , calpain 3 ( CAPN - 3 ) , to treat limb - girdle muscular dystrophy type 2A ( LGMD2A ) . The candidate was developed by the Abigail Wexner Research Institute at Nationwide Children's Hospital . Also known as calpainopathy , LGMD2A is caused by mutations in the CAPN - 3 gene and is the most common type of LGMD , accounting for almost a third of cases . Like SRP - 9001 , Sarepta's lead investigational gene transfer therapy for Duchenne , and the Company's five other LGMD programs , the LGMD2A program uses the AAVrh74 vector , designed to systematically and robustly deliver treatment to skeletal muscle making it an ideal candidate to treat muscle disease . • Sarepta announced positive 12 - week expression and safety results for SRP - 9001-103 , the first results from a clinical trial using SRP - 9001 commercially representative material : In May 2021 , the Company announced results from the first 11 participants enrolled in Study SRP - 9001-103 , an open - label study known as ENDEAVOR being conducted in partnership with Roche . SRP - 9001 is an investigational gene transfer therapy being developed for the treatment of Duchenne muscular dystrophy . In the open - label study , 20 participants between the ages of four and seven were treated with a single infusion of SRP - 9001 at a dose of 1.33x1014 vg / kg . Results include : • All patients demonstrated robust transduction , with mean micro - dystrophin expression of 55.4 % of normal , as measured by western blot . • Muscle dystrophin levels demonstrated a mean of 70.5 % ( baseline 12.8 % ) muscle fibers expressing micro - dystrophin at 12