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ROCKVILLE, Md.,Oct. 6, 2025/PRNewswire/ -- REGENXBIO Inc. (Nasdaq:RGNX) today announced the completion of enrollment in the ATMOSPHERE®and ASCENT®pivotal studies evaluating surabgene lomparvovec (sura-vec, ABBV-RGX-314) in wet age-related macular degeneration (wet AMD) using subretinal delivery.
"Completing enrollment in this large, global pivotal program is an exciting milestone in our efforts to deliver sura-vec to patients as the potential first gene therapy for wet AMD," said Steve Pakola, M.D., Chief Medical Officer, REGENXBIO. "The millions of patients worldwide with wet AMD are in need of a treatment option that can preserve vision, prevent disease progression, and reduce the significant burden of frequent, life-long eye injections required with today's standard of care. We are highly encouraged by the safety and long-term durability seen in multiple earlier-stage trials. We look forward to sharing the topline data next year and advancing global registration of this potentially transformative treatment."
ATMOSPHERE and ASCENT are multi-center, randomized, active-controlled trials designed to support the global regulatory submissions of sura-vec. ATMOSPHERE, conducted in the U.S., is evaluating sura-vec versus ranibizumab; ASCENT, conducted in the U.S. and 13 other countries, is evaluating sura-vec versus aflibercept. The primary endpoint is non-inferiority based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks and one year, respectively. Secondary endpoints include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections in the treatment arms. Together, these pivotal studies have enrolled over 1,200 participants across more than 200 sites.
In along-term follow up study, sura-vec was well tolerated and demonstrated durable treatment effect with stable or improved vision up to four years. An additional Phase II pharmacodynamic study evaluated sura-vec at the same dose levels as the pivotal trials. At one year, sura-vec was well tolerated in 60 treated participants with no drug-related serious adverse events, stable to improved vision and anatomy, and meaningful reduction in anti-VEGF injections in participants with high treatment burden.
Topline data is expected in the fourth quarter of 2026.
About Surabgene Lomparvovec (sura-vec, ABBV-RGX-314)Sura-vecis being investigated as a potential one-time treatment for wetAMD, diabetic retinopathy and other chronic retinal conditions.Sura-vecconsists of theNAV®AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF).Sura-vecis believed to inhibit theVEGFpathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
About Wet AMDWet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long frequent, repeated intraocular injections to maintain efficacy. Due to the burden of treatment, it is difficult for patients to adhere to frequent injections, which has been shown to lead to a decline in vision over time.
ABOUT REGENXBIO Inc.REGENXBIOis a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009,REGENXBIOhas pioneered the field ofAAVgene therapy.REGENXBIOis advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment ofDuchenne;clemidsogene lanparvovec(RGX-121) for the treatment ofMPSII and RGX-111 for the treatment ofMPSI, both in partnership withNippon Shinyaku; andsurabgene lomparvovec(ABBV-RGX-314) for the treatment of wetAMDand diabetic retinopathy, in collaboration withAbbVie. Thousands of patients have been treated withREGENXBIO's AAVplatform, including those receivingNovartis'ZOLGENSMA®.REGENXBIO'sinvestigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visitwww.regenxbio.com.
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