REGENXBIO Says 93% of Duchenne Patients Hit RGX-202 Phase III Endpoint, Eyes 2027 Launch
Updated
Updated · PR Newswire · May 14
REGENXBIO Says 93% of Duchenne Patients Hit RGX-202 Phase III Endpoint, Eyes 2027 Launch
7 articles · Updated · PR Newswire · May 14
93% of evaluable patients in AFFINITY DUCHENNE cleared the trial’s primary bar of more than 10% microdystrophin expression at Week 12, with p<0.0001 in 30 participants.
Nine boys with one-year follow-up showed statistically significant improvement on NSAA and timed function tests, and microdystrophin levels correlated with functional gains, supporting the biomarker as a surrogate endpoint.
RGX-202 was described as well tolerated in 31 patients: two serious adverse events—subacute myocarditis and asymptomatic liver injury—resolved within weeks, while common drug-related side effects were mild or moderate.
More than 20 additional patients have entered the confirmatory study, and REGENXBIO expects to finish dosing all 60 patients across pivotal and confirmatory trials by mid-year.
FDA has indicated microdystrophin could support accelerated approval if tied to clinical outcomes; REGENXBIO plans further talks with the agency and is preparing for a potential 2027 commercial launch.
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