Avidity Biosciences (NASDAQ:RNA) reported positive topline data from the Phase 1/2 MARINA clinical trial of AOC 1001 for the treatment of myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
“The AOC 1001 topline data demonstrated directional improvement across a variety of functional assessments in patients with DM1, including myotonia and muscle strength in a six-month period,” Nicholas Johnson, M.D. and lead investigator in the MARINA trial, said in a statement.
“This result is more than we could have anticipated in such a short time,” he added. “These AOC 1001 data are remarkable and could make a real impact for people living with DM1.”
Sarah Boyce, president and CEO of Avidity, said data from the study delivered a “robust data package to support advancement into a pivotal study. We are focused on designing the quickest path to bring AOC 1001 to patients and look forward to discussions with the FDA.”
Avidity continues to work with the FDA regarding the partial clinical hold on new participant enrollment in the AOC 1001 program. The company continues to dose participants in the MARINA open-label extension study (MARINA-OLE) to evaluate the long-term safety and tolerability of AOC 1001 in participants with DM1 who were previously enrolled in the Phase 1/2 MARINA trial.
Avidity remains on track to share a first look at the data from the MARINA-OLE study at the end of 2023.
