Avidity Biosciences announces data from Phase 1/2 EXPLORE44 clinical trial

Avidity Biosciences announced positive AOC 1044 data in healthy volunteers from the Phase 1/2 EXPLORE44 clinical trial for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping. AOC 1044 delivered unprecedented concentrations of phosphorodiamidate morpholino oligomers in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 and increased exon skipping in all participants. Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024. AOC 1044 is designed to deliver PMO to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production. AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency, and Fast Track Designation by the FDA. AOC 1044 is the first of multiple AOCs the company is developing for DMD. AOC 1044 delivered unprecedented, dose-dependent increases in PMO concentrations in skeletal muscle following a single dose of 5 mg/kg or 10 mg/kg, providing up to 50-times greater concentrations of PMO in skeletal muscle when compared to a single dose of peptide conjugated PMOs in healthy volunteers. AOC 1044 produced statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 at Day 29. AOC 1044 increased exon skipping in all participants.