AOC 1001

Avidity Biosciences, Inc. (RNA) specializes in RNA therapeutics, focusing on rare diseases with its AOC products. Currently, AOC 1001 for myotonic dystrophy type 1 is in phase 1/2 trials. The company, based in San Diego, has a market cap of $3.6B and faces financial challenges with a negative EPS of -2.88.

Increased R&D in myotonic dystrophy drives market growth, attracting funding and partnerships. DelveInsight's 'Myotonic Dystrophy Pipeline Insight 2024' reports on 20+ companies developing 22+ therapies, including AOC 1001, Tideglusib, and PGN EDODM1. Key events include FDA designations for ATX-01 and DYNE-101, and Fast Track designation for PGN-EDODM1.

HNSA-5487 showed robust IgG reduction in NICE-1 trial, potentially advancing treatment in chronic autoimmune diseases. FDA removed partial hold on AOC 1001 for myotonic dystrophy type 1. EMBARK study found no cardiac impact from delandistrogene moxeparvovec in Duchenne muscular dystrophy.

FDA lifts partial hold on Avidity's AOC 1001 for myotonic dystrophy type 1 (DM1), allowing continuation of MARINA trial. AOC 1001, a monoclonal antibody-siRNA conjugate targeting DMPK, showed positive long-term data in MARINA-OLE, with consistent improvements in DM1 measures. The agent received breakthrough therapy designation for DM1 treatment.

Avidity Biosciences' stock rose 12% after FDA lifted partial clinical hold on its muscular dystrophy drug, AOC 1001. Goldman Sachs analyst Corinne Johnson reiterated her buy recommendation.