REGENXBIO Announces Completion of Enrollment in Pivotal Trials of Subretinal Surabgene Lomparvovec for Wet AMD

Core Insights - REGENXBIO Inc. has completed enrollment in pivotal studies ATMOSPHERE and ASCENT for surabgene lomparvovec (sura-vec) targeting wet age-related macular degeneration (wet AMD) [1][2][8] - The studies aim to evaluate the efficacy and safety of sura-vec as a potential first gene therapy for wet AMD, with topline data expected in Q4 2026 [4][8] Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline including treatments for rare and retinal diseases [7] - The company has pioneered AAV gene therapy since its founding in 2009 and has treated thousands of patients with its AAV platform [7] Clinical Trials - The ATMOSPHERE trial compares sura-vec to ranibizumab, while the ASCENT trial compares it to aflibercept, with over 1,200 participants enrolled across more than 200 sites [2][8] - Primary endpoints include non-inferiority based on changes in Best Corrected Visual Acuity (BCVA) at 54 weeks and one year, with secondary endpoints assessing safety, central retinal thickness, and need for additional anti-VEGF injections [2][3] Treatment Efficacy - Long-term follow-up studies indicate that sura-vec is well tolerated, showing stable or improved vision up to four years and a reduction in the need for anti-VEGF injections [3] - Sura-vec is designed to inhibit the VEGF pathway, which is responsible for the formation of leaky blood vessels in the retina, a key factor in wet AMD [5][6] Market Context - Wet AMD is a significant cause of vision loss, affecting up to 2 million people in the U.S., Europe, and Japan, with current treatments requiring frequent injections that can be burdensome for patients [6] - The introduction of sura-vec could provide a one-time treatment option, potentially transforming the management of wet AMD [2][8]