Sarepta Therapeutics

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

WaVe Life Sciences Ltd. (NASDAQ:WVE) focuses on RNA editing technology for genetic diseases, with a promising DMD program showing 53% exon skipping. The company faces competition from firms like Sarepta Therapeutics and KRRO, but recent analyst price targets suggest growing confidence. WaVe's future hinges on clinical trial success and strategic partnerships, particularly in RNA editing, which is seen as a transformative technology in genetic medicine.

Roche and Dyno Therapeutics expand their collaboration to develop next-gen AAV vectors for neurological diseases using Dyno’s AI-driven LEAP technology, with potential payments exceeding $1 billion.

Elevidys, a $3.2 million gene therapy for Duchenne muscular dystrophy, failed to meet its primary endpoint in a Phase 3 trial, showing no significant motor function improvement over placebo.

Delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy missed primary endpoint in EMBARK trial but showed differences in secondary outcomes, including clinically relevant improvements in time to rise and 10-meter walk/run. FDA granted full approval based on totality of evidence, despite primary endpoint failure.

CGTs disrupt treatment for genetic diseases, with 69 marketed worldwide and 7 novel FDA approvals in 2023. Oncology leads CGTs, projected to reach $37bn by 2030. Outsourcing vs. in-house manufacturing is crucial, with Lonza Group as the leading CMO for marketed CGTs.

Pfizer withdrew Oxbryta from global markets due to increased risk of death, joining other drugs like Aduhelm, Exkivity, Ukoniq, and Zydelig that were approved under the FDA's accelerated pathway but later withdrawn. The pathway, implemented in 1992, has helped bring nearly 300 drugs to market but faces scrutiny over delayed confirmatory trials and surrogate endpoints. Sarepta's Elevidys is an exception, securing full approval despite missing primary efficacy endpoints.

Delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy (DMD) was safe over 5 years, with 75 adverse events, mostly mild or moderate, and no serious AEs. Treated patients showed significant improvements in time to rise (TTR) and 10-meter walk/run time compared to an external control cohort, maintaining ambulation while the control group lost it. The therapy's North Star Ambulatory Assessment (NSAA) total score also improved significantly over 5 years, diverging from natural history predictions.

Rare diseases affect 7% of the world’s population, mostly genetic. Precision medicine offers tailored treatments, overcoming traditional pharmaceutical research limitations. Success stories include therapies for spinal muscle atrophy, Batten’s disease, Duchenne muscular dystrophy, and cystic fibrosis. Challenges remain in cost, ethical considerations, and data sharing, but advancements in genomic sequencing, AI, and digital technology hold promise.