Data presented at a pair of recent meetings show that RGX-314 (Regenxbio), an adeno-associated viral (AAV) vector encoding an antibody fragment that inhibits VEGF, has promise in the treatment of wet AMD and diabetic retinopathy (DR). In a pair of phase 2 trials, the drug, which is delivered suprachoroidally, met a primary endpoint showing no serious drug-related adverse events while also meeting important benchmarks specific to each disease.

At the Retina Society meeting in Chicago, Nikolas London, MD, FACS, chief of ophthalmology at Scripps Memorial Hospital and a partner in and director of research at Retina Consultants of San Diego, reported that a cohort of 20 wet AMD patients receiving either RGX-314 at a dose level of 2.5x10¹¹ genomic copies per eye (GC/eye) or ranibizumab (Lucentis; Genentech) 0.5 mg monthly. At 6 months, patients in this trial, called AAVIATE, who received a single injection of RGX-314 showed stable visual acuity and retinal thickness, as well as a 75.9% reduction in the number of anti-VEGF injections following RGX-314.

The following week, at the American Society of Retina Specialists (ASRS) Annual Meeting in San Antonio, Texas, Dennis Marcus, MD, FASRS, president of Southeast Retina Center in Atlanta, presented data from the ALTITUDE study of RGX-314 in DR. Twenty patients in this cohort were randomized to receive either the same dose of RGX-314 as in the AAVIATE cohort or an observational control. At 3 months, of 15 patients receiving the drug, a third showed a ≥2-step improvement from baseline on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale, with 1 achieving a 4-step improvement. The response rate was better in patients with nonproliferative DR.

“We look forward to further enhancing our understanding of the potential of RGX-314 when delivered to the suprachoroidal space,” said Steve Pakola, MD, chief medical officer of Regenxbio, in a news release.