Upcoming 2024 DMD Treatment Data

In recent years, the medical field has witnessed significant advancements in the treatment of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness. The approval of ITF Therapeutics’ Duvyzat in March marked a milestone, being the third new therapy approved by the FDA within a span of 10 months. This followed the historic green light given to Sarepta’s Elevidys in June 2023, the first gene therapy for DMD, and Santhera Pharmaceuticals’ Agamree, a novel corticosteroid, in October.

The pipeline for DMD treatments is robust, with numerous investigational therapies in various stages of clinical trials. Companies anticipate releasing trial data in 2024, which could potentially lead to new treatment options. Michael Kelly, the chief scientific advisor at CureDuchenne, expressed optimism about the long-term efficacy and safety data expected from ongoing trials and approved drugs.

Investigational therapies are diverse, ranging from gene therapy to exon-skipping biologics and small molecules. Pfizer’s fordadistrogene movaparvovec, a gene therapy, is expected to report data from a Phase III trial in 2024. This therapy aims to introduce a mini-dystrophin gene to DMD patients, transforming their condition to the less severe Becker muscular dystrophy, although it is not a complete cure.

Dyne Therapeutics is also looking forward to results from its Phase I/II DELIVER trial of DYNE-251, an exon-skipping biologic designed to create a functional dystrophin protein for patients with specific mutations. Early data indicate promising levels of dystrophin expression and a favorable safety profile.

Edgewise Therapeutics is working on EDG-5506, a small molecule intended to reduce the mechanical stress caused by the lack of functional dystrophin. The company plans to report data from its Phase II LYNX trial, with the FDA granting Fast Track designation for this candidate in DMD.

Capricor Therapeutics is developing CAP-1002, a cell therapy composed of cardiosphere-derived cells that could potentially work in synergy with emerging disease-modifying therapies. The company is conducting a pivotal Phase III study with expectations of topline data by year-end.

Lastly, Avidity Biosciences is advancing AOC 1044, an exon-skipping biologic for patients with exon 44 mutations. The therapy has shown positive results in Phase I/II trials, with significant exon skipping and good tolerability. Avidity plans to share initial data from DMD patients in the latter half of 2024.

These developments reflect a concerted effort to address the unmet needs of DMD patients, with the hope that a combination of therapies targeting dystrophin restoration, alongside other approaches, will pave the way for more effective treatments.