FDA Grants Fast Track Designation for Antibody Oligonucleotide Conjugate to Treat Duchenne Muscular Dystrophy

AOC 1044 (Avidity Biosciences, Inc, San Diego, CA), a drug in the new class of RNA therapeutics called antibody oligonucleotide conjugates (AOCs), was granted Fast Track designation by the Food and Drug Administration (FDA) to treat Duchenne muscular dystrophy (DMD) in those with mutations amenable to exon 44 skipping (DMD44). “Fast Track” represents a designation associated with expedited development and review of drugs designed to treat serious medical conditions and fill an unmet medical need. At present, there are no approved therapies for DMD44.

Steve Hughes, MD, Chief Medical Officer at Avidity Biosciences, Inc, issued a statement about the announcement: "We will continue to work closely with the FDA as we advance AOC 1044 and look forward to the anticipated data readout from the healthy volunteer portion of our Phase 1/2 EXPLORE44 clinical trial later this year." EXPLORE44 (NCT05670730) is a phase 1/2 clinical trial currently recruiting which is designed to study thesafety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD44.

AOCs represent a combination of monoclonal antibodies and oligonucleotides that increasingly have been used in a variety of applications such as imaging, detection, and therapies. DMD is a rare genetic, x-linked recessive disease that affects 1 in 3500 to 5000 individuals, mostly boys, worldwide.