5 DMD Data Releases to Watch in 2024

In March, the FDA approved Duvyzat, ITF Therapeutics' treatment for Duchenne muscular dystrophy (DMD), a genetic disorder leading to muscle weakness and degeneration. This marked the third new treatment for DMD in just ten months. The FDA had earlier approved Sarepta’s Elevidys, the first gene therapy for DMD, in June 2023, followed by Santhera Pharmaceuticals’ corticosteroid Agamree in October.

The DMD treatment landscape is rapidly evolving, with numerous novel therapies advancing through clinical trials. Several companies anticipate significant data releases in 2024, underscoring the progress made in recent years. Michael Kelly, Chief Scientific Advisor at CureDuchenne, expressed optimism about the forthcoming data, emphasizing its potential to enhance understanding of the long-term efficacy and safety of both ongoing trials and approved medications. He highlighted Pfizer, REGENXBIO, and Solid Biosciences' gene therapy initiatives and the next-generation exon-skipping programs from Avidity Biosciences, Dyne Therapeutics, PepGen, and Entrada Therapeutics as particularly noteworthy.

Jeff Chamberlain, President of the American Society for Gene and Cell Therapy, acknowledged the substantial unmet needs within the DMD community but noted the significant strides being made in the right direction. BioSpace explored five promising investigational therapies expected to yield results this year.

Pfizer is set to release data from a Phase III trial of fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in the latter half of 2024. This investigational therapy utilizes an adeno-associated virus to deliver a shortened dystrophin gene to DMD patients who can walk. Kelly noted that while this therapy aims to transform the severe DMD phenotype to the milder Becker muscular dystrophy, it isn't a cure. Pfizer's journey with fordadistrogene movaparvovec has faced challenges, including a temporary halt in 2021 due to a patient’s death. However, the Phase III trial has since resumed.

Dyne Therapeutics anticipates data from the higher dose cohort of their Phase I/II DELIVER trial for DYNE-251 in the latter half of the year. DYNE-251 is an exon-skipping biologic aimed at promoting the skipping of exon 51 to produce functional dystrophin protein in patients with specific mutations. Preliminary results have shown promising levels of dystrophin expression and a favorable safety profile, encouraging further optimization of the dosage for future trials.

Edgewise Therapeutics is expected to report three-month controlled dose-ranging data from the Phase II LYNX trial of EDG-5506 this quarter. EDG-5506 aims to mitigate mechanical stress on muscles caused by the lack of dystrophin, enabling healthier muscle contractions. The FDA has granted Fast Track designation to this candidate. Edgewise is also conducting the Phase II FOX trial in DMD patients who have undergone gene therapy.

Capricor Therapeutics is carefully monitoring its cell therapy CAP-1002, currently in a pivotal Phase III study, with topline data expected by year’s end. CAP-1002 consists of cardiosphere-derived cells that secrete exosomes to reduce inflammation and promote tissue regeneration. Capricor is also exploring CAP-1002's potential synergy with other emerging therapies.

Avidity Biosciences is developing AOC 1044 for patients with exon 44 mutations, an area with no current disease-modifying treatments. Utilizing its proprietary AOC platform, AOC 1044 aims to deliver morpholino oligomers to skip exon 44 and enable dystrophin production. Early data from the Phase I/II EXPLORE44 trial showed significant exon 44 skipping and good tolerance. Avidity plans to present data from DMD patients in the second half of 2024.