Upcoming 2024 DMD Treatment Trial Results

The recent approval of ITF Therapeutics' Duvyzat in March represents a significant milestone in the treatment of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. This marks the third FDA-approved treatment for DMD in less than a year, following the approval of Sarepta’s Elevidys in June 2023 as the first gene therapy for the condition and Santhera Pharmaceuticals’ corticosteroid Agamree in October.

The pipeline for DMD treatments is robust, with numerous investigational therapies in various stages of clinical trials, and several companies anticipate sharing trial results in 2024. Michael Kelly, chief scientific advisor at CureDuchenne, emphasized the importance of ongoing trials and investments in research, stating that this year could provide valuable insights into the long-term efficacy and safety of current treatments and those in development. He highlighted the potential of gene therapy programs from Pfizer, REGENXBIO, and Solid Biosciences, as well as the next generation of exon-skipping therapies from Avidity Biosciences, Dyne Therapeutics, PepGen, and Entrada Therapeutics.

Jeff Chamberlain, president of the American Society for Gene and Cell Therapy, acknowledged the unmet needs in DMD treatment but expressed optimism about the direction of research. BioSpace has identified five investigational therapies with expected results in the coming year:

Pfizer's Gene Therapy: Pfizer is expecting data from a Phase III trial of fordadistrogene movaparvovec in the latter half of 2024. This gene therapy targets ambulatory DMD patients and aims to transform the patient’s phenotype from Duchenne to the milder Becker muscular dystrophy, despite not providing a full-length dystrophin gene.

Dyne Therapeutics' Exon-Skipping Biologic: Dyne Therapeutics anticipates results from the higher dose cohort of the Phase I/II DELIVER trial of DYNE-251. This therapy is designed to promote exon skipping to produce a truncated but functional dystrophin protein, showing promising early results.

Edgewise Therapeutics' Small Molecule: Edgewise Therapeutics is set to report data from the Phase II LYNX trial of EDG-5506, a candidate designed to reduce mechanical stress on muscles lacking functional dystrophin.

Capricor Therapeutics' Cell Therapy: Capricor Therapeutics is conducting a pivotal Phase III study on CAP-1002, a cell therapy composed of cardiosphere-derived cells that secrete exosomes to modulate inflammation and stimulate tissue regeneration.

Avidity Biosciences' Exon Skipping Biologic: Avidity Biosciences is developing AOC 1044 for patients with exon 44 mutations. The therapy has shown positive results in healthy volunteers and is expected to report data from DMD patients later in 2024.

These advancements in DMD treatment reflect a concerted effort by the scientific and medical communities to address the complex needs of patients with this debilitating genetic disorder. The future holds promise for more effective and targeted therapies that could significantly improve the quality of life for those affected by DMD.