RGX-202 - (AFFINITY DUCHENNE)

June 24, 2026: REGENXBIO Inc. (NASDAQ: RGNX) announced it has completed dosing in the confirmatory study for RGX-202, a gene therapy candidate for Duchenne muscular dystrophy, completing the registrational development program ahead of schedule. The company said it remains on track to initiate a Biologics License Application (BLA) under the FDA accelerated approval pathway in Q3 2026, with a potential FDA approval in 2H 2027.

REGENXBIO said the planned BLA will include safety data from the AFFINITY DUCHENNE pivotal plus confirmatory studies (n=63) and efficacy data from the pivotal portion (n=30), and is expected to include 12-month functional data for at least half of pivotal participants. In topline pivotal results, RGX-202 met its primary endpoint: 10% microdystrophin expression in >93% of patients at Week 12. Twelve-month functional data available so far (n=9) showed a large magnitude of effect across multiple timed function tests and the North Star Ambulatory Assessment (NSAA). RGX-202 was described as well tolerated with a favorable safety profile, and microdystrophin expression at Week 12 was reported to correlate with interim functional improvement at one year.