Sarepta Therapeutics Announces Top-Line Results for Part 1 of Study SRP-9001-102 (Study 102)
January 07, 2021 at 04:05 pm EST
Share
Sarepta Therapeutics, Inc. 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 (PAbout SRP-9001-102 Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 102 uses clinical process SRP-9001 and has two primary endpoints: micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks compared to placebo. Secondary endpoints include certain timed functional tests; micro-dystrophin expression measured by immunofluorescence (IF) fiber intensity; and micro-dystrophin expression measured by IF percent dystrophin positive fibers. In Part 1, results from the treatment and placebo groups are compared through 48 weeks following treatment. In Part 2, the study remains blinded while all participants in the placebo group cross over to active treatment and all participants are followed for another 48 weeks while safety and efficacy continue to be evaluated. SRP-9001 (rAAVrh74.MHCK7.micro-dys trophin): SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital.
Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company. The Company is focused on helping patients through the discovery and development of ribonucleic acid (RNA)-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. It has developed multiple approved products for the treatment of Duchenne muscular dystrophy (Duchenne) and is developing potential therapeutic candidates for a range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. It has developed and commercialized four approved products for the treatment of Duchenne: EXONDYS 51 (eteplirsen), Injection (EXONDYS 51), VYONDYS 53 (golodirsen) Injection (VYONDYS 53), AMONDYS 45 (casimersen) Injection (AMONDYS 45), and ELEVIDYS. Its pipeline includes approximately 40 programs at various stages of discovery, pre-clinical and clinical development.