- A single ABBV-RGX-314 gene therapy treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing wet AMD due to the treatment burden of chronic anti-VEGF injections
- Patients who received therapeutic doses resulted in stable or improved vision and retinal anatomy up to 2 years
- Additional long-term follow-up data has demonstrated durable treatment effect, with stable or improved vision, up to 4 years
- Enrollment is on track in pivotal trials of ABBV-RGX-314 in wet AMD that are expected to support global regulatory submissions in late 2025 through the first half of 2026
REGENXBIO Inc. 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 (wet 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," said Kenneth T. Mills, president and CEO of REGENXBIO. "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 highlighted in The Lancet 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 two 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 two years. REGENXBIO has 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.
"The publication of the ABBV-RGX-314 phase 1/2a trial results in The Lancet reinforces the encouraging long-term clinical data observed using subretinal delivery and underscores the potential of ABBV-RGX-314 gene therapy to offer a new approach to the clinical management of wet AMD," said Jeffrey S. Heier, MD, director of the Vitreoretinal Service and director of Retina Research at Ophthalmic Consultants of Boston and primary investigator for the trial. "Wet AMD is a chronic, life-long disease and real-world evidence shows patients are losing significant vision over time, and the burden of frequent anti-VEGF injections needed to manage their wet AMD is a major reason why. A single treatment of ABBV-RGX-314 that can potentially provide long-lasting treatment outcomes and a strong safety profile would offer a novel approach to treating this serious and blinding disease."
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 these trials are expected to support global regulatory submissions with the U.S. Food and Drug Administration and the European Medicines Agency in late 2025 through the first half of 2026.
About ABBV-RGX-314
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, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.