Partial Hold on Avidity Bio Therapy

FDA has issued Avidity Biosciences a partial clinical hold on new participant enrollment in its Phase 1/2 MARINA clinical trial of AOC 100, an antibody oligonucleotide conjugate, in adults with myotonic dystrophy Type 1 (DM1). Close to 40 participants are currently enrolled in the MARINA and MARINA open label extension (MARINA-OLE) trials, the company says, adding that all participants may continue in their current dosing cohort. A preliminary assessment of the MARINA study is expected in the fourth quarter, Avidity says.

DM1 is described as an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options. The partial hold was triggered by a serious adverse event reported in a single participant in the 4mg/kg cohort of the MARINA study. “Avidity is working closely with the FDA and the trial investigator to assess the cause of this event,” the company says.