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SAREPTA THERAPEUTICS Sarepta Therapeutics Announces Third Quarter 2021 Financial Results and Recent Corporate Developments 11/3/21 • Net product sales for the third quarter of 2021 reached $ 166.9 million , a 37 % increase over the same quarter of prior year • In light of its continued over - performance , Sarepta raises its full - year product revenue guidance by $ 40 million to between $ 605 million to $ 615 million CAMBRIDGE , Mass . , Nov. 03 , 2021 ( GLOBE NEWSWIRE ) -- Sarepta Therapeutics , Inc. ( NASDAQ : SRPT ) , the leader in precision genetic medicine for rare diseases , today reported financial results for the third quarter of 2021 . " We are pleased to report another quarter of strong performance serving the Duchenne community with our three currently approved therapies and on that basis have once again raised our full - year product revenue guidance . In total , we have raised guidance by some $ 70 million this year and are now guiding to $ 605 million to $ 615 million . This represents our 20th straight quarter of strong revenue growth and we anticipate this growth continuing in 2022 , " said Doug Ingram , president and chief executive officer , Sarepta . " We have now initiated Part B of MOMENTUM , our pivotal trial for SRP - 5051 , our next - generation PPMO candidate for exon 51 skip amenable Duchenne patients as well as EMBARK , our pivotal trial for SRP - 9001 , our micro dystrophin gene therapy for Duchenne . Also , this quarter we shared additional compelling data across three studies for SRP - 9001 , providing additional conviction as we execute on EMBARK and prepare to unblind and release Study 102 Part 2 results in the first quarter of next year . As we track out of 2021 and into a milestone - rich 2022 , we are delivering on our approved therapies , seeing successes across our programs , and as of today , with greater than $ 2 billion of cash and cash equivalents on our balance sheet and a first - in - class team of genetic and rare disease professionals , have the resources and talent to deliver on the promise of our multi - platform pipeline . " Third Quarter 2021 and Recent Corporate Developments : • At SRP - 9001 Micro - dystrophin R & D Day Sarepta showed sustained functional improvements in multiple studies of individuals with Duchenne : In October of 2021 , the Company presented new analyses and functional data from its SRP - 9001 development program and details of EMBARK ( Study SRP - 9001-301 ) ; its global pivotal Phase 3 trial of SRP - 9001 for the treatment of Duchenne . 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 . The results presented highlight the breadth , depth and strength of the clinical evidence to date for SRP - 9001 in treating Duchenne . • SRP - 9001-101 ( Study 101 ) : Results from participants treated with SRP - 9001 in Study 101 ( n = 4 , ages 4 to 7 ) found that participants 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 ) . • SRP - 9001-102 ( Study 102 ) : The results from Study 102 Part 1 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 ( p = 0.0129 ) . • ENDEAVOR , SRP - 9001-103 ( Study 103 ) : The first functional results presented from Study 103 , which uses commercially representative SRP - 9001 material , 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 . • Across three clinical studies , the tolerability profile remains consistent in all treated patients . • Initiated EMBARK ( Study SRP - 9001-301 ) , the first global pivotal study of SRP - 9001 for the treatment of Duchenne : The EMBARK study is a multi - center clinical trial initiating in the US , Europe and Asia . It is the first global , randomized , double - blind , placebo - controlled clinical trial of commercially representative SRP - 9001 material and will enroll 120 participants with Duchenne between the ages of 4 to 7. The primary endpoint will assess the change in NSAA total score from baseline to week 52 compared to placebo . Key features include stratification of participants by age and baseline NSAA , with a minimum of 50 percent of patients ages 4 to 5 to be enrolled . Inclusion criteria include a Time to Rise from Floor of less than 5 seconds , a stable daily dose of oral corticosteroids for at least 12 weeks before screening and rAAVrh74 antibody titers of less than 1 : 400 . Participants with mutations between or including exons 1-17 or mutations fully contained within exon 45 ( inclusive ) are not eligible . The Company expects the trial to fully enroll in the first half of 2022 . • Initiated Part B of MOMENTUM ( Study SRP - 5051-201 ) in patients with Duchenne amenable to exon 51 skipping : In the fourth quarter of 2021 , the Company initiated Part B of MOMENTUM , a global trial investigating the use of SRP - 5051 , the Company's next - generation peptide - conjugated phosphorodiamidate morpholino oligomer ( PPMO ) to treat patients with Duchenne who are amenable to exon 51 skipping . The study plans to enroll between 20-40 patients between ages 7 to 21