Avidity Biosciences has announced positive, healthy volunteer data from the Phase 1/2 EXPLORE44™ study evaluating AOC 1044 for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44).
This is the first data readout from the EXPLORE44 trial and showed AOC 1044 to be well tolerated and delivered unprecedented concentrations of phosphorodiamidate morpholino oligomers (PMOs – synthetic DNA analogs that inhibit gene expression in a sequence-dependent manner) in skeletal muscle. 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 and increased exon skipping in all participants.
- 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.
- AOC 1044 was well tolerated in healthy volunteers. All treatment-emergent adverse events in participants dosed with AOC 1044 were mild to moderate. There were no symptomatic hemoglobin changes, no hypomagnesemia and no renal events.
A first look at data from participants with Duchenne is planned in the second half of 2024, and Defeat Duchenne Canada will be eager to share with the wider community.