Avidity Biosciences, Inc. has announced topline results from its Phase 1/2 EXPLORE44 trial of delpacibart zotadirsen (del-zota) for treating Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). The data demonstrated successful delivery of phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle, increased exon 44 skipping, improved dystrophin production, and reduced creatine kinase levels.

The trial results also indicate favorable safety and tolerability, supporting further development. Avidity has selected a 5 mg/kg dosage every six weeks for ongoing and future studies, with plans to submit a Biologics License Application (BLA) by the end of 2025. The company continues to pursue an accelerated approval path in the United States.