Video

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

Author(s):

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.

Related Videos
Jörn Schattenberg, MD | Credit: Novo Nordisk
Diabetes Dialogue: Diabetes Tech Updates from November 2024 | Image Credit: HCPLive
Jörn Schattenberg, MD | Credit: Novo Nordisk
The APAC Recap: Cardiomyopathy at CAPP Live 2024 with Greg Duck, PA-C | Image Credit: APAC
Orrin Troum, MD: Accurately Imaging Gout With DECT Scanning
The APAC Recap: Peripheral Artery Disease at CAPP Live 2024 with Bob Ross, PA-C | Image Credit: APAC
How to Manage Aspirin-Exacerbated Respiratory Disease
John Stone, MD, MPH: Continuing Progress With IgG4-Related Disease Research
© 2024 MJH Life Sciences

All rights reserved.