Avidity Biosciences, Inc. announced new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE?) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) 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 (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) 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. Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran.

Del-desiran (AOC 1001) is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.