REGENXBIO Reports Positive Interim Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202 for Duchenne Muscular Dystrophy
Interim results from seven participants show improved functional performance, stable cardiac function, and strong microdystrophin expression1.
Among four patients at pivotal dose, all surpassed expected disease trajectory on North Star Ambulatory Assessment (NSAA), with average improvements of 6.6 points at 12 months and 7.4 points at 18 months2.
RGX-202 demonstrates favorable safety:
no serious adverse events, no liver injury, reductions in muscle damage biomarkers at one year1.
Phase III topline data expected in Q2 2026; BLA submission planned mid-2026 via accelerated approval12.
Trial (NCT05693142) is ongoing, enrolling boys aged 1+ with DMD34.
Sources:
1. https://cureduchenne.org/general/regenxbio-reports-new-positive-interim-data-from-phase-i-ii-affinity-duchenne-gene-therapy-trial/
2. https://www.neurologylive.com/view/patients-treated-regenxbio-dmd-gene-therapy-rgx-202-exceed-expected-disease-trajectory-nsaa