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REGENXBIO Reports First Quarter 2025 Financial Results and Recent Operational Highlights
RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026Pivotal trial more than half enrolled, with completion expected in 2025 Additional
About this update from Regenxbio Inc.
[{"type":"text","content":"RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026Pivotal trial more than half enrolled, with completion expected in 2025 Additional Phase I/II functional data expected 1H 2025 Initiating commercial supply manufacturing in Q3 2025FDA acceptance of Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121, partnered with Nippon Shinyaku) expected in May 2025 Pivotal data evaluating the safety and efficacy of the subretinal delivery of surabgene lomparvovec (ABBV-RGX-314, partnered with AbbVie) in patients with wet age-related macular degeneration are expected in 2026 and planning of diabetic retinopathy pivotal study continuesConference call today at 4:30 p.m. ETROCKVILLE, Md., May 12, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the first quarter ended March 31, 2025.\n\"We have made tremendous progress towards delivering multiple commercial gene therapies, starting this year,\" said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. \"Each of our late-stage assets is demonstrating differentiation against standard of care or available treatments, giving us a pipeline of potential first- or best-in-class gene therapies for rare and retinal diseases. With advanced clinical programs, commercial-ready manufacturing in-house at our Rockville, MD headquarters, and a strong balance sheet, REGENXBIO is well-equipped to deliver potentially transformative gene therapies to patients in need.\"\nPROGRAM HIGHLIGHTS AND MILESTONES\nNeuromuscular Disease: RGX-202 is designed to use the NAV® AAV8 vector to deliver a differentiated, novel microdystrophin gene for improved muscle function and outcomes for patients living with Duchenne. It is the only investigational or approved microdystrophin gene therapy construct to include the C-Terminal (CT) domain found in naturally occurring dystrophin.\nThe pivotal Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202 is ongoing in ambulatory patients and is expected to enroll approximately 30 patients aged 1+ in the U.S. and Canada by 2025, with more than half already enrolled to support the pivotal dataset.REGENXBIO expects to share topline data in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.C...