Defeat Duchenne Canada is thrilled that REGENXBIO will be moving to a pivotal Phase 3 trial of their investigational gene therapy RGX-202 for the treatment of Duchenne muscular dystrophy (DMD). On July 31, 2024, REGENXBIO announced positive interim results from their Phase 1/2 AFFINITY trial of RGX-202 in people ages 1 to 11 years of age with DMD. On August 1st’s earning’s call, the company shared that they recently held a successful End-of-Phase II meeting with the U.S. FDA and is moving forward with plans to initiate their pivotal Phase 3 trial in Q4 2024.

High level results of the Phase 1/2 AFFINITY study include that all 7 children with DMD who received treatment with RGX-202 experienced increases in microdystrophin. Participants in the study who received dose level 2 experienced an increase of microdystrophin expression of 77.2% in the 4 to 7 year old children and 46.5% in the 8 to 11 year old group. As of the July 8 data cut off, treatment was well tolerated with no serious side effects. REGENXBIO plans to complete enrollment in dose level 2 as early as end of September and has initiated enrollment in a cohort for children 1-3 years of age with DMD.

We look forward to additional data from both cohorts before the end of 2024. The full press release of their Phase 2 data is available here.

Want to know more about Duchenne Research?

Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: