RegenXBio announces positive topline results from pivotal Phase 3 microdystrophin gene therapy trial

RegenXBio shared positive topline results from their pivotal Phase 3 AFFINITY DUCHENNE study of RGX-202 in 31 individuals with Duchenne aged 1 year and older.  The study met the primary endpoint with statistical significance, with 93% of participants achieving microdystrophin expression above 10%.  There was also a statistically significant correlation between microdystrophin expression and functional improvement on NSAA (Northstar Ambulatory Assessment).  In 9 participants who reached one year post-dosing, functional improvement (changes from baseline) were demonstrated on the NSAA and timed function tests. 

RegenXBio plans to pursue accelerated approval for RGX-202, with a potential commercial launch in 2027.  

You can learn more about this exciting data during a webinar that CureDuchenne is hosting with RegenXBio on Tuesday, May 19, 2026.  Register for the webinar: https://cureduchenne.org/event/regenxbio-webinar/

Link to press release:  https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-positive-topline-results-pivotal-phase-iii