REGENXBIO Inc. announced new positive interim functional, safety, and biomarker data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 for Duchenne muscular dystrophy on June 5, 2025. The data cut was May 7, 2025.
Functional data from five participants receiving dose level 2 demonstrated consistent benefit at 9 and 12 months, with participants exceeding external natural history controls on the North Star Ambulatory Assessment (NSAA) and timed function tests. At 12 months, RGX-202 recipients improved an average of 4.5 points from baseline on NSAA and 6.8 points compared to natural history.
New biomarker data from an additional 2-year-old patient showed a microdystrophin expression level of 118.6% compared to control. RGX-202 was well tolerated with no serious adverse events or adverse events of special interest. The company expects to submit a Biologics License Application (BLA) using the accelerated approval pathway in mid-2026.
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