FDA eases partial clinical hold on Avidity trial of AOC 1001

The US Food and Drug Administration (FDA) has eased the partial clinical hold on AOC 1001, allowing Avidity Biosciences (Nasdaq: RNA), the drug’s developer, to double the number of participants in the MARINA Open-Label Extension (MARINA-OLE) study receiving 4 mg/kg of AOC 1001.

Initial market reaction was positive, send Avidity’s shares up 14% pre-market, but they were down 1.6% at $10.83 in mid-morning trading.

The FDA is also allowing new participant enrollment for AOC 1001 at 2mg/kg. Data from the MARINA-OLE study will be used to finalize the AOC 1001 pivotal dose and Phase III study design for adults with myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options.