REGENXBIO Reports First Quarter 2026 Financial Results and Operational Highlights

• 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