REGENXBIO announced earlier this year the start of ASCENT TM, the second of two Phase III pivotal trials evaluating the viability of subretinal delivery of RGX-314 in people with wet age-related macular degeneration (AMD). ASCENT is the first trial that REGENXBIO is initiating with the collaboration with AbbVie. RGX-314 is being studied based on its potential to be a single-dose gene therapy for the treatment of wet AMD. ASCENT is a randomized, multi-center and active-controlled trial that evaluates the safety and effectiveness of delivering RGX-314 subretinally.
In this trial, two dose arms are being studied and compared to intravitreal injections of aflibercept, which take place bi-monthly. The trial aims to establish results that are not inferior to aflibercept. The results are based on the change from baseline in the Best Correct Visual Acuity (BCVA) at the one-year mark. ASCENT plans to enroll about 465 patients, and REGENXBIO aims to submit the Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) in 2024.
More about RGX-314
RGX-314 is a REGENXBIO Inc. product that is a candidate for treating wet AMD. The novel aspect of this drug is that it is a one-time subretinal treatment for wet AMD, and it includes the NAV AAV8 vector, which contains a gene that encodes for a monoclonal antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). Wet AMD causes vision loss due to structural changes that occur via excess formation of new leaky blood vessels and retinal fluid accumulation. VEGF is responsible for this angiogenesis, and as this process continues, vision loss can occur due to scar formation as a result of inflammation and hemorrhaging. This demonstrates the importance of VEGF in the progression of wet AMD, which has been the target of standard care for this condition. To inhibit the advancement of vision loss, current anti-VEGF therapies require numerous intraocular injections, administered every four to eight weeks to maintain their efficacy. These various injections can negatively impact patients’ comfort and compliance, which can surmount vision deterioration. Therefore, having a novel treatment that could potentially be administered only once and have long-term effects could be life-changing.
