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Sarepta Planning BLA Submission of DMD Gene Therapy

The announcement follows positive data updates from the ENDEAVOR and SRP-9001-101 studies.

Sarepta Therapeutics has announced their intent to submit a biologic license application (BLA) for delandistrogene moxeparvovec (SRP-9001), their investigative gene therapy for the treatment of Duchenne muscular dystrophy (DMD).1

“We are delighted to confirm that based on the feedback we received following a thorough and in-depth review, we intend to submit a BLA for our SRP-9001 gene therapy to treat Duchenne muscular dystrophy this fall. We look forward to a collaborative review commencing this year and running through the first half of 2023,” Doug Ingram, president and chief executive officer, Sarepta Therapeutics, said in a statement.1 “Duchenne robs children daily and hourly of their muscle, stealing them bit by bit from their families and loved ones. Guided by rigorous science and productive regulatory discussions, our goal is to move with the urgency desperately needed by the patient community, and our upcoming BLA filing for SRP-9001 serves that goal.”

SRP-9001 is being developed in partnership with Roche as of a December 2019 partnership. The gene transfer therapy is designed to deliver the microdystrophin gene to muscle tissue to restore production of the microdystrophin protein. The FDA has granted fast track, rare pediatric disease, and orphan drug designations (ODD) to the therapy. It has also been granted ODD in the European Union, Switzerland, and Japan.

SRP-9001 recently demonstrated promising data from 20 participants in the phase 1 SRP-9001-103 study (ENDEAVOR, NCT04626674) and 4 participants in the phase 1/2 Study SRP-9001-101 (NCT03375164).2

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“We are absolutely delighted by these most recent results. We now have positive results across multiple studies and multiple time points, including 1-, 2- and 4-years after treatment, and are very pleased with the consistent safety profile across more than 80 treated patients,” Ingram said in a previous statement.2 “We are particularly excited about the results of cohort 1 of Study 103, as these results come from our commercially representative process at our intended commercial dose. We robustly powered our 120-patient phase 3 study, known as EMBARK, and the results from Study 103 provide even greater conviction on the powering and probability of success of EMBARK. Duchenne is a relentlessly degenerative disease and every day of delay is a day of muscle and function loss that cannot be recovered. Sarepta and our partner Roche are dedicated and determined to bring this potentially transformative gene therapy to patients around the globe as rapidly as possible.”

Participants in ENDEAVOR showed an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control group (P <.0001).

Participants from Study SRP-9001-101 (NCT03375164) demonstrated a 7-point improvement above pre-treatment baselines on the NSAA, a 9.9-point unadjusted means and 9.4-point least squared means versus a propensity-weighted external control (P = .0125) at 4 years. An integrated analysis of 52 patients across studies SRP-9001-101, SRP-9001-102 (NCT03769116), and SRP-9001-103 also showed that at 1 year, patients treated with SRP-9001 at the target dose improved 3.1 points (unadjusted means) and 2.4 points (least squared means) on NSAA versus propensity-weighted external control (P <.0001).

“The data from our 103 study is particularly important because it’s from our commercially-representative material,” Louise Rodino-Klapac, PhD, executive vice president and chief scientific officer, Sarepta, said on a conference call regarding the data on July 6, 2022.3 “It can be extremely challenging to achieve the same level of safety and efficacy when scaling up from small-scale to commercial-scale, so we are thrilled to see that these results are not only consistent, but may be improved over previously reported results.”

1. Sarepta Therapeutics announces intent to submit an accelerated approval biologics license application for its gene therapy SRP-9001 to treat Duchenne muscular dystrophy. News release. Sarepta Therapeutics.
2. Sarepta Therapeutics’ investigational gene therapy SRP-9001 for Duchenne muscular dystrophy demonstrates significant functional improvements across multiple studies. News release. Sarepta Therapeutics, Inc. July 6, 2022. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-investigational-gene-therapy-srp-9001
3. SRP-9001: New clinical data and integrated analysis. Webcast. Sarepta Therapeutics. July 6, 2022. https://investorrelations.sarepta.com/events/event-details/sarepta-therapeutics-share-new-clinical-data-and-integrated-analysis-srp-9001