by: Parent Project Muscular Dystrophy
Astellas has shared results of the Phase 1b study of ASP0367 (MA-0211), an oral investigational drug. The study aimed to evaluate safety, tolerability, and preliminary efficacy in individuals living with Duchenne aged 8 to 16 years old.
The study was stopped early, in November 2022, because not enough patients were available to take part in the study. While the study’s small sample size limits the data interpretation, the results indicate that ASP0367 is safe and well-tolerated. Additionally, for the key efficacy endpoints, the ASP0367 treatment group showed a clinically relevant improvement in parameters compared with those observed in the placebo treatment group.
To learn more about the results of the study please see the Plain Language Summary linked in the community letter.
Astellas has indicated that they look forward to continuing to work with patients and families to develop new therapies for patients.
