Business
REGENXBIO Reports First Quarter 2026 Financial Results and Operational Highlights
Company announced positive topline results from pivotal Phase III AFFINITY DUCHENNE® study of RGX-202Primary endpoint achieved with high statistical
About this update from Regenxbio Inc.
Company announced positive topline results from pivotal Phase III AFFINITY DUCHENNE® study of RGX-202 Primary endpoint achieved with high statistical significance Statistically significant correlation between RGX-202 microdystrophin expression and functional improvement (NSAA, n=9), supporting validity of surrogate endpoint Surabgene lomparvovec (sura-vec, ABBV-RGX-314) on track toward key catalysts Sites activated in pivotal Phase IIb/III study for diabetic retinopathy; first patient dosed expected Q2 2026 Subretinal wet AMD topline pivotal data expected in Q4 2026 Clinical hold lifted for RGX-121 Webcast today at 8:00 a.m. ET to discuss RGX-202 topline pivotal data and Q1 2026 earnings ROCKVILLE, Md. , May 14, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the first quarter ended March 31, 2026 . " REGENXBIO enters a transformative year with positive momentum, reaching significant late-stage milestones to support our potential first- and best-in-class gene therapies," said Curran Simpson , President and Chief Executive Officer, REGENXBIO . "On our mission to bring meaningful new therapies to rare disease communities facing limited options, we remain focused on advancing RGX-202 for Duchenne toward potential BLA submission and are excited to share topline pivotal data in our webcast today; we also continue engaging with the FDA regarding a potential path forward for RGX-121 for Hunter syndrome. In chronic retinal diseases, we are executing against our multi-indication strategy and preparing for the Phase 3 wet AMD readout with partner AbbVie. Additionally, we continue harnessing internal manufacturing capabilities to support anticipated commercial needs. We remain well-positioned to capitalize on several near-term, high-value opportunities and bring these highly differentiated treatments to patients." PROGRAM HIGHLIGHTS AND MILESTONES Neuromuscular Disease: RGX-202 is a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. RGX-202 is designed for improved muscle function, durability and positive safety outcomes and is being evaluated in the Phase I/II/III AFFINITY DUCHENNE® trial in ambulatory patients aged 1+. REGENXBIO today announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE trial of RGX-202, including primary endpoint (n=30 at Week 12), interim safety (n=31), and interim functional data (n=9 at 12 months):[1] Achieved primary endpoint with high statistical significance; 93% of patients achieved RGX-202 microdystrophin expression above 10% (p