Avidity Biosciences announced in a press release the first results of the MARINA phase 1-2 trial of AOC 1001 in 44 people with Steinert’s disease (DM1) in the US.
- The product caused only moderate side effects in most participants. However, one person who received the highest dose of the drug candidate experienced serious health problems, which has led to the suspension of inclusions for the time being. The trial continues for those participants already included.
- AOC 1001 reduced DMPK by an average of 45% in the muscle of all those who received it.
- A decrease in DM1-specific splicing abnormalities was also demonstrated.
- An improvement in hand myotonia was observed in some participants, indicating a possible early clinical effect of the treatment, to be confirmed in the further course of the trial.
Avidity Announces Positive AOC 1001 Phase 1/2 MARINA™ Data Demonstrating First-Ever Successful Targeted Delivery of RNA to Muscle – Revolutionary Advancement for the Field of RNA Therapeutics Avidity Biosciences press release, December 14, 2022.
