Avidity Biosciences announces ‘positive’ data from MARINA-OLE trial

Avidity Biosciences announced new positive long-term AOC 1001 data from the MARINA open-label extension trial showing reversal of disease progression in people living with myotonic dystrophy type 1 across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time, and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024. “The long-term data from the MARINA-OLE study demonstrating that del-desiran improved measures of disease progression in DM1 patients compared to natural history data is remarkable,” said John W. Day, MD, PhD, Professor of Neurology and Pediatrics, and Director, Division of Neuromuscular Medicine, Stanford University School of Medicine, an investigator of the MARINA and MARINA-OLE trials. “The favorable long-term safety data and consistent, durable improvement in myotonia, muscle strength and patient-reported outcomes measures show the potential of del-desiran to make a meaningful difference in the lives of DM1 patients. I am very encouraged by the prospect of del-desiran as a potential treatment for DM1.”