Avidity announces positive topline data from DMD clinical trial skipping exon 44

CureDuchenne provided early funding to Avidity Biosciences, and are pleased to share they have released positive topline data from the EXPLORE44 trial in individuals amenable to skipping exon 44.  Data was consistent across two cohorts, dosed either with 5 mg/kg every 6 weeks or 10 mg/kg every 8 weeks.  Delpacibart zotadirsen (abbreviated as del-zota) demonstrated statistically significant increases of approximately 25% of normal dystrophin production, restoring total dystrophin up to 58% of normal.  In addition, creatine kinase levels were reduced by more than 80%, to near normal levels.  On the strength of this data, Avidity is planning a year end 2025 BLA submission to the FDA for accelerated approval of del-zota dosed at 5 mg/kg every 6 weeks. 

https://aviditybiosciences.investorroom.com/2025-03-17-Avidity-Biosciences-Announces-Positive-Topline-Del-zota-Data-Demonstrating-Consistent,-Statistically-Significant-Improvements-in-Dystrophin,-Exon-Skipping-and-Creatine-Kinase-in-People-Living-with-Duchenne-Muscular-Dystrophy-Amenable-to-Exon-44