Avidity Biosciences announces AOC 1001 data from MARINA trial, MARINA-OLE study

Avidity Biosciences announced new AOC 1001 data demonstrating improvement in multiple additional functional endpoints and favorable long-term safety and tolerability in people living with myotonic dystrophy type 1. AOC 1001, Avidity’s lead clinical program utilizing its AOC platform, is designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. The AOC 1001 data from the Phase 1/2 MARINA trial and MARINA open-label extension study will be highlighted in an oral presentation at the 28th Annual Congress of the World Muscle Society in Charleston, South Carolina and can be found on Avidity’s website on the Publications page. The new AOC 1001 data demonstrate improvement in additional functional measures including hand grip, muscle strength and patient reported outcomes, augmenting previously reported positive data showing improvements in myotonia, muscle strength and mobility. With new long-term safety data from over 200 infusions totaling 46.2 patient-years of exposure, AOC 1001 continues to demonstrate favorable safety and tolerability with most adverse events mild to moderate. In May 2023, the U.S. Food and Drug Administration eased the partial clinical hold on AOC 1001, allowing Avidity to double the number of participants in the MARINA-OLE study receiving 4 mg/kg of AOC 1001 from 12 to 24 participants. Data from the 12 participants dose-escalated from 2 mg/kg to 4 mg/kg of AOC 1001 as part of the easement of the partial clinical hold showed no neurological events and no MRI changes following dosing. The company continues to work as quickly as possible to resolve the partial clinical hold. New AOC 1001 data demonstrate improvement in additional functional measures augmenting previously reported positive data that demonstrated improvements in functional assessments of myotonia, strength and mobility.