On October 31, 2023, Roche provided a community letter to share timely updates about Roche Duchenne muscular dystrophy (DMD) research efforts. The topline results of Part 1 (1-year data) of the EMBARK Phase 3 study have been announced.
● The study did not meet its primary endpoint of showing a statistically significant difference in a change on the North Star Ambulatory Assessment (NSAA) total score after treatment with delandistrogene moxeparvovec (ElevidysTM) gene therapy, compared with placebo. However, participants did show an improvement in motor function abilities, compared with placebo-treated participants.
● Clinically meaningful and statistically significant improvements were observed in both key pre-specified secondary functional endpoints (time to rise from the floor and 10-meter walk test).
● The safety of delandistrogene moxeparvovec was consistent with other studies, and no new safety signals were observed.
To read the community letter in full, please see it below.
