Regenxbio Announces Lancet Publication of Phase 1/2a Study Evaluating ABBV-RGX-314 as a One-Time Gene Therapy for Wet AMD
Regenxbio announced the publication of results from the phase 1/2a trial evaluating the safety and tolerability of a single dose of subretinal ABBV-RGX-314 for the treatment of wet age-related macular degeneration (AMD). Two-year data were published in The Lancet in a paper titled "Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study." These positive study results informed the ongoing pivotal trials of ABBV-RGX-314, a potential one-time gene therapy, for the treatment of wet AMD.
"We have started 2024 with strong, positive new data from the ABBV-RGX-314 program, and we believe that there is multi-billion-dollar potential for ABBV-RGX-314 to become a first-in-class gene therapy for wet AMD and the standard of care to treat and prevent progression of diabetic retinopathy," Kenneth T. Mills, President and Chief Executive Officer of Regenxbio, said in a company news release. "To have these phase 1/2a data published in The Lancet highlights the groundbreaking work of our scientists and investigators, and further validates the clinically transformative nature of ABBV-RGX-314 as a potential one-time gene therapy for wet AMD that may help patients maintain or improve their vision."
The findings demonstrated that a single administration of ABBV-RGX-314 was generally well tolerated. Stable or improved visual acuity and retinal thickness was observed with few or no supplemental anti-VEGF injections in most patients at 2 years. Patients who received therapeutic doses demonstrated sustained levels of ABBV-RGX-314 protein and stable or improved vision and retinal anatomy with few, to no, supplemental anti-VEGF injections in most participants up to 2 years. Regenxbio also reported additional positive interim data from a long-term follow-up study of ABBV-RGX-314 supporting that treatment continues to be well-tolerated and demonstrates long-term, durable treatment effect up to four years.
ABBV-RGX-314 is currently being evaluated in patients with wet AMD in two pivotal trials called ATMOSPHERE and ASCENT. Enrollment is on track and the trials are expected to support global regulatory submissions with the FDA and the European Medicines Agency in late 2025 through the first half of 2026.
ABBV-RGX-314, being developed in collaboration with AbbVie, is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. ABBV-RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). ABBV-RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
Regenxbio is advancing research in two separate routes of administration of ABBV-RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. Regenxbio has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical to deliver gene therapy treatments to the suprachoroidal space of the eye.