REGENXBIO (NASDAQ: RGNX) has announced new positive interim data from the Phase 1/2 AFFINITY DUCHENNE trial of RGX-202—a potential best-in-class therapy for Duchenne muscular dystrophy (DMD).
Trial investigator Carolina Tesi-Rocha, MD, clinical professor, neurology, Stanford School of Medicine, Stanford Children’s Health, will present the AFFINITY DUCHENNE trial data, including new functional, safety, biomarker, and cardiac MRI measures, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
In a statement, Steve Pakola, MD, CMO of REGENXBIO, commented, “Today’s new Phase 1/2 interim data demonstrate continued positive impact on function, stable cardiac health, and a favorable safety profile, highlighting the potential of RGX-202 to be a differentiated gene therapy option for Duchenne. Our proactive, comprehensive approach to safety combined with our novel microdystrophin construct are supported by the sustained safety and durable functional outcomes through two years post-treatment. As we approach our topline pivotal data readout in early Q2, we are very encouraged by this thorough new dataset and the opportunity to advance RGX-202 as a potential meaningful treatment option for patients.”
