Positive data in study of drug for Duchenne muscular dystrophy with exon 44 skipping

Avidity Biosciences Inc. has announced positive data in healthy volunteers from its ongoing phase 1/2 clinical trial of a proprietary monoclonal antibody to treat Duchenne muscular dystrophy mutations amenable to exon 44 skipping.

According to a company release, AOC 1044 delivered high concentrations of phosphorodiamidate morpholino oligomers (PMO) in skeletal muscle with up to 50 times greater concentrations of PMO after a single dose compared with peptide conjugated PMOs in healthy volunteers.

Additionally, data showed AOC 1044 was well-tolerated, demonstrated “statistically significant” exon 44 skipping compared with placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg and increased exon skipping in all participants, Avidity said in the release.

AOC 1044 was granted orphan drug designation in August by the FDA and European Medicines Agency, as well as fast track designation by the FDA.

“We are excited with the early data set of AOC 1044 demonstrating unprecedented delivery of therapeutic oligonucleotide in skeletal muscle and consistent exon skipping in healthy volunteers,” Avidity President and CEO Sarah Boyce said in the release. “Data from our clinical programs continue to reinforce the broad and disruptive potential of our [antibody oligonucleotide conjugate] platform for the treatment of high burden muscle diseases.”

Avidity plans to release a "first look" at the data on AOC 1044 during the second half of 2024, according to the release.

Editor’s note: This story was updated Dec. 26, 2023, to indicate that a first look at the data will be released in 2024. Healio regrets the error.