A recently completed study indicates that a subretinal gene therapy in development for neovascular (“wet”) age-related macular degeneration (nAMD) is both safe and effective when dosed bilaterally. Arshad M. Khanani, MD, MA, FASRS, presented the data on ABBV-RGX-314 (Regenxbio/AbbVie) during the late-breaking developments session during Saturday’s Retina Subspecialty program at the American Academy of Ophthalmology (AAO) meeting in Chicago.
ABBV-RGX-314 is an adeno-associated virus serotype 8 (AAV8) gene therapy for nAMD, designed to express monoclonal antibody fragment that neutralize vascular endothelial growth factor (VEGF) activity. Based on a follow-up study that reported data up to 4 years, a single treatment with ABBV-RGX-314 has the potential for long-term durable therapeutic anti-VEGF expression. ABBV-RGX-314 is currently being studied in a pair of phase 3 pivotal trials, ATMOSPHERE and ASCENT.
The bilateral dosing study represented the first time that a gene therapy for neovascular AMD has been dosed in the fellow eye, said Dr. Khanani. He noted that nAMD eventually affects both eyes in most patients and treating both eyes in patients with bilateral nAMD should decrease treatment burden and potentially produce better vision outcomes in the real world.
Dr. Khanani reported that 78% of patients were injection free at the 9-month mark, and that there was a 97% reduction in overall treatment burden. “Most of these patients were getting injections every 1 to 2 months with an average of 8 actual injections before they were enrolled in the study,” Dr. Khanani noted. Visual acuity and retinal thickness were stable or improved after a single treatment with ABBV-RGX-314, he said. The data did not show any new safety signals or any cases of intraocular inflammation, he commented.
“This is a milestone for the field of gene therapy for nAMD," said Dr. Khanani. "The results from this study are promising and show that fellow eye dosing with ABBV-RGX-314 is efficacious and safe and has the potential to make a meaningful difference for patients with bilateral wet AMD.” RP
