Avidity Biosciences' Muscle Disease Candidate Shows Improvement in Multiple Additional Functional Endpoints, Safety

Avidity Biosciences Inc RNA announced new AOC 1001 data demonstrating improvement in multiple additional functional endpoints and favorable long-term safety and tolerability for myotonic dystrophy type 1 (DM1). 

AOC 1001, Avidity's lead clinical program utilizing its AOC platform, is designed to address the root cause of DM1. 

The AOC 1001 data from the Phase 1/2 MARINA trial and MARINA open-label extension (MARINA-OLE) study will be highlighted at the 28th Annual Congress of the World Muscle Society.

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 demonstrates favorable safety and tolerability, with most adverse events (AEs) mild to moderate.

Data from 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 plans to share AOC 1001 data from the MARINA-OLE study in the first half of 2024 and is finalizing the Phase 3 study design and global regulatory path for AOC 1001.

Price Action: RNA shares are down 1.13% at $6.12 on the last check Monday.