RGX-314 Gene Therapy Efficacious in Wet AMD: Robert L. Avery, MD

This content originally appeared on our sister site, HCPLive.

HCPLive spoke with Robert L. Avery, MD, CEO, California Retina Consultants, to learn more about the data he presented at the American Academy of Ophthalmology 2021 Meeting on the REGENXBIO study of RGX-314. The gene therapy is being investigated as a potential treatment for wet age-related macular degeneration (AMD) and diabetic retinopathy.

Avery presented positive data from the Phase 1/2a study of a single intravitreal injection of RGX-314. The study enrolled 42 patients, with 5 ascending dose escalation cohorts. In the first 2 cohorts, no benefit was seen with a lower dose.

However, Avery noted that in cohort 3, 4, and 5, they saw a definite effect on patients who had been anti-VEGF dependent, or frequent fliers, as Avery called them. They had on average 9 or more injections per year, potentially for numerous years.

“They were heavily treated patients, it was a dramatic reduction in their need for therapy, while maintaining good vision, stable vision and good thickness results,” he said.

The study did show 20 severe adverse events in 13 patients that were felt to not have been related to the drug treatment, aside from a single event that was pigmentary changes in the macula of one patient.

Avery went on to note that 69% of the patients develop pigmentary changes in the retina, mostly in the far interior periphery. However, they were considered mild, except if there was an effect on visual acuity. He explained that these patients were not treatment naive and had a history of atrophy before enrollment.

“Other than that the drug was really well tolerated, Avery said. “There were no signs of significant inflammation as opposed to many other gene therapies. There was no prophylactic steroid treatment given for this trial, just the typical drops we would do after vitrectomy and tapered off, but really no significant inflammation, which is, I think, a very important point with this study.”

Further studies include ATMOSPHERE and a second unnamed study which begin enrolling in the 4th quarter of 2021, with about 300 patients. They are randomized to 2 doses of RGX-314, or ranibizumab.

“Two Year Results from the Subretinal RGX-314 Gene Therapy Phase 1/2a Study for the Treatment of nAMD, and an Update on Suprachoroidal Trials,” was presented at AAO 2021.