The MARINA trial is a double-blind, placebo-controlled Phase I/II trial which evaluated AOC 1001 for six months in Steinert’s disease (or DM1). It was followed by an open-label extension, the results of which were announced at the Muscular Dystrophy Association congress in March 2024. After one year of treatment, they confirm the preliminary results previously communicated:
- a significant improvement in hand myotonia, muscular strength and the Activ-DM1 scale, reflecting personal dynamism in daily life,
- and good tolerability of treatment.
A Phase III HARBOR trial is scheduled to start in 2024.
Initial Results of the Phase 2 Open-Label Extension Study of AOC 1001 in Adults With Myotonic Dystrophy Type 1: MARINA-OLE – Johnson N, Day J, Hamel J et al. MDA Clinical & Scientific Conference Mars 2024
Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial – Avidity Biosciences, 4 March 2024 press release
