Financial Performance and Liquidity
Operating Expenses
Pipeline and Operational Developments
The company advanced its Duchenne muscular dystrophy program, reporting that patients treated with the pivotal dose of RGX-202 demonstrated an average improvement of 7.4 points on the North Star Ambulatory Assessment compared to the expected disease trajectory at 18 months. Topline pivotal data for RGX-202 is anticipated in early Q2 2026. In retinal diseases, topline pivotal data for subretinal delivery of sura-vec is expected in Q4 2026. Concurrently, the neurodegenerative pipeline faced regulatory setbacks, with the FDA placing clinical holds on both RGX-111 and RGX-121 following a report of an intraventricular CNS tumor in an RGX-111 trial participant. Additionally, the FDA issued a complete response letter for the RGX-121 Biologics License Application in February 2026.