The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is underway in Dallas, Texas, bringing together leading experts, researchers, and advocates in the neuromuscular disease community. Today, several significant announcements were made by companies developing therapies for Duchenne. These updates include:

Avidity Biosciences Announces Positive Topline Del-zota Data in Phase 1/2 EXPLORE44® Trial

Avidity Biosciences, Inc., has announced positive del-zota (delpacibart zotadirsen, formerly AOC 1044) topline data from the Phase 1/2 EXPLORE44® trial in people living with Duchenne amenable to exon 44 skipping. According to Avidity, the data demonstrates consistent, statistically significant improvements in dystrophin production and creatine kinase levels, as well as favorable safety and tolerability across the dose cohorts. Avidity reports that, based on the company’s interactions with FDA on accelerated approval, it believes dystrophin data from EXPLORE44 combined with the safety data from the fully enrolled EXPLORE44-OLE trial will support Avidity’s planned BLA submission at the end of 2025. Read Avidity’s press release here and Letter to the Community here.

Please join PPMD and Avidity Biosciences for a community webinar on Friday, March 28, 2025 at 2:00 PM ET. Avidity will share topline data from their EXPLORE44® clinical trial in greater detail and answer questions from the community. Please submit questions in advance here. Register for the webinar here.

Capricor Therapeutics: Long-Term Efficacy of Deramiocel

Capricor Therapeutics has announced positive long-term data from its ongoing HOPE-2 open-label extension (OLE) clinical trial, highlighting the potential of deramiocel, the company’s novel cell therapy for the treatment of individuals diagnosed with Duchenne cardiomyopathy, to slow disease progression and preserve upper limb function in Duchenne patients. Over a three-year period, patients treated with deramiocel experienced a 52% reduction in disease progression, as measured by a slower decline in Performance of Upper Limb function, compared to an external comparator group. The therapy was well-tolerated, with no new safety signals identified, reinforcing its favorable long-term benefit-risk profile. Read Capricor’s press release here.

Capricor recently joined PPMD for a community webinar to provide a current update on the company’s Biologics License Application (BLA) seeking full approval of deramiocel. Watch the webinar recording here.

Dyne Therapeutics: Sustained Functional Improvement with DYNE-251

Dyne Therapeutics announced new long-term clinical data from its Phase 1/2 DELIVER trial of DYNE-251, demonstrating sustained functional improvement in individuals with DMD amenable to exon 51 skipping. Individuals treated with the registrational dose of 20 mg/kg every four weeks showed meaningful improvements from baseline in multiple functional endpoints through 12 months. DYNE-251 also exhibited a favorable safety profile, with no new treatment-related serious adverse events observed. The company plans to submit a Biologics License Application (BLA) for U.S. accelerated approval in early 2026. Read Dyne’s press release here.

ITF Therapeutics: Long-Term Benefits of DUVYZAT™ (Givinostat)

ITF Therapeutics presented seven abstracts highlighting the long-term efficacy and safety of DUVYZAT™ (givinostat) for the treatment of Duchenne. Notably, data from the Phase 3 EPIDYS study and ongoing open-label extension studies demonstrated that patients treated with givinostat experienced a median age of loss of ambulation at 18.1 years, compared to 15.2 years in a matched natural history control group. Additionally, givinostat was associated with a delayed decline in respiratory function, and its long-term administration was well-tolerated, with no new safety signals observed. Read ITF’s press release here.