January 18, 2023
1 minute read
FDA grants Avidity Biosciences Inc. Fast Track designation for its novel RNA therapeutic AOC 1020, an antibody-oligonucleotide conjugate being studied for the treatment of facioscapulohumeral muscular dystrophy .
According to a release from Avidity, AOC 1020 combines a proprietary monoclonal antibody with a muscle-targeted small interfering RNA designed to reduce the expression of double homeobox 4 (DUX4) mRNA. In patients with facioscapulohumeral muscular dystrophy (FSHD), aberrant expression of the DUX4 protein can alter gene expression in muscle cells, leading to a steady loss of muscle function.

The FDA has granted priority review designation to a California-based bioscience company for a new therapy intended for the treatment of facioscapulohumeral muscular dystrophy. Source: Adobe Stock
AOC 1020 is currently being evaluated in the Phase 1/2 FORTITUDE clinical trial. This is a randomized, placebo-controlled, double-blind trial enrolling approximately 70 adults diagnosed with FSHD. This study examines the safety, tolerability, and pharmacokinetics of IV administration of the drug. There are currently no FDA-approved treatments for people with this disease.
“FDA Fast Track designation for AOC 1020 underscores the importance of finding effective treatments to help people living with FSHD. Steve Hughes MD, Avidity’s chief medical officer said in a release. “AOC 1020 is designed to directly target the disease-causing gene DUX4 to address the underlying causes of FSHD.”
Avidity also said in the release that it plans to share early evaluation results of AOC 1020 with approximately half of the FORTITUDE study participants in the first half of 2024.