Biotech firms, including Regenxbio, Novartis and Dyne, are converging on regulatory milestones for therapies targeting Duchenne muscular dystrophy and myotonic dystrophy type 1, signaling a flurry of potential filings and confirmatory trials.
Regenxbio reported mixed pivotal data for its gene therapy RGX-202: the Phase 3 portion met its primary endpoint with 93% of patients achieving at least 10% microdystrophin expression at week 12, but two serious adverse events—one liver injury and one myocarditis—were recorded; the company previously guided a mid-2026 submission and the product is expected to be approved and launch in 2027, according to the company.
Dyne said it had a positive pre-BLA engagement for exon-51 skipper DYNE-251, is targeting a second-quarter submission with a potential Q1 2027 launch, and has initiated a 72-week Phase 3 confirmatory trial; the company also reported progress on DYNE-101 for DM1, with target enrollment reached in a global confirmatory study.
Novartis’s $12 billion acquisition of Avidity brought exon-skipping and DM1 assets, including del-zota and del-desiran, the latter with a Phase 3 study of 159 patients expected to complete in September.
Solid Biosciences has dosed the first patient in a Phase 3 study of its gene therapy SGT-003 and highlights a favorable safety profile to date, a contrast to recent safety and confirmatory trial setbacks that have affected parts of the field.
