Regenxbio's RGX-202 Duchenne Gene Therapy Shows Positive Interim Data, Pivotal Results Expected Q2 2026 for FDA Accelerated Approval
Phase I/II AFFINITY DUCHENNE trial demonstrates robust microdystrophin expression (up to 77.2% of control in older patients), improved NSAA scores, stable cardiac function, and favorable safety with no serious adverse events.
FDA aligned on expanded Phase 1/2 study as pivotal trial (~30 patients aged 1+), using microdystrophin levels at 3 months as primary endpoint for accelerated approval.
Pivotal topline data expected early Q2 2026; pre-BLA meeting mid-2026, with confirmatory portion ongoing.
Early data shows higher microdystrophin than Sarepta's Elevidys, dose response, and functional improvements vs. external controls.
RGX-202 includes C-Terminal domain for potentially better muscle protection; commercial manufacturing underway.