Regenxbio’s RGX-202 shows efficacy in Phase 1/2 Duchenne trial

Regenxbio (RGNX) announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, Regenxbio’s potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy. The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202. The data further supports the planned Biologics License Application submission under accelerated approval in mid-2026. Regenxbio announced positive interim functional results from the first five participants, aged approximately 6 to 12 years at dosing, receiving RGX-202 at dose level 2. RGX-202 continues to demonstrate evidence of positively impacting disease trajectory. At 9 months, RGX-202 participants demonstrated improvement in function and exceeded external controls on all measures. Four out of the five participants reached 12-months post dosing. Results at 12 months are similar to those seen at 9 months. RGX-202 participants demonstrated improved performance on timed function tests and NSAA, exceeding external natural history controls at 12 months. All participants within this cohort demonstrated improvement on all timed function tests compared to baseline. RGX-202 was appropriately localized to the sarcolemma, demonstrating that the differentiated construct is appropriately targeting the muscle. RGX-202 was well tolerated with no serious adverse events and no AEs of special interest. Regenxbio is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202. The pivotal trial is expected to support a Biologics License Application submission using the accelerated approval pathway in mid-2026. Regenxbio expects to share top-line data in the first half of 2026.