Gene Therapy RGX-314 Benefits nAMD Treatment Burden in Phase 2 AAVIATE

May 5, 2022
Kevin Kunzmann

Study author Arshad Khanani, MD, MA, discusses how the novel-targeting gene therapy candidate may uniquely benefit patients with treated retina disease.

Investigative suprachoroidal gene therapy RGX-314 provided tolerability, safe outcomes and meaningful benefit in best-corrected visual acuity (BCVA) and central retinal thickness (CRT) at 6 months, according to new phase 2 findings.

In new data from the Regenxbio AAVIATE clinical trial, presented at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Meeting this week, a team investigators reported the one-time gene therapy injection additionally reduced burden of long-term intravitreal anti-VEGF injection treatments among treated patients with neovascular age-related macular degeneration (nAMD).

The phase 2 findings, which featured 40 patients with nAMD previously treated with anti-VEGF randomized to either the suprachoroidal-delivered RGX-314 or ranibizumab, contribute toward evidence and rhetoric supporting the promising gene therapy as a long-term agent in patients with nAMD or diabetic retinopathy (DR).

In an interview with HCPLive regarding the findings, study author Arshad Khanani, MD, MA, director of clinical research at Sierra Eye Associates, discussed the significance of the novel-targeting gene therapy for this patient population.

"Remember, this is the first trial looking at a suprachoroidal gene therapy, and we’re learning a lot about efficacy as well as safety,” Khanani said. “It was tolerated very, very well, and in terms of efficacy, we saw greater than 70% reduction of treatment burden in these patients treated with RGX-314.”

Khanani said the team is currently enrolling patients to be administered an even greater dose in the ongoing phase 2 trial, and will also conduct analysis of patients with baseline positive neutralizing antibodies to compare efficacy and safety outcomes.

In the meantime, the future of gene therapy management of nAMD and retina disease looks slightly more promising. Though RGX-314 would not be a curative agent, Khanani pointed to its significant benefit in reducing treatment burden for patients previously reliant on regular anti-VEGF injections—as well as its availability as a one-time, in-clinic treatment.

“It’s really exciting to see what the next dose level shows in terms of efficacy and safety,” Khanani said. “In the first 2 doses at 6 months’ data, we had stabilization of anatomy and visual acuity. So it’s really exciting times for patients.”

The study, "Suprachoroidal Delivery of RGX-314 Gene Therapy for Neovascular AMD: The Phase II AAVIATE™ Study," was presented at ARVO 2022.


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