Gene therapy RGX-314 shows promise in the treatment of nAMD

Clinical trials have demonstrated that RGX-314, administered through a single subretinal delivery, provides sustained anti-VEGF therapy, potentially reducing the need for frequent anti-VEGF injections in the treatment of age-related macular degeneration (nAMD), according to data presented at the EURETINA Congress 2023.

Patients in the study experienced stable to improved visual acuity, and a significant reduction in injection frequency.

A Phase 1/2a trial in previously treated patients with nAMD demonstrated sustained anti-VEGF levels up to 2 years post RGX-314 administration. In the trial, 5 escalating dose levels of RGX-314 were evaluated in 42 previously treated patients who had responded to ranibizumab before receiving RGX-314.

As of August 29th, 2022, 37 patients enrolled in the long-term follow-up (LTFU) study reported continued positive tolerance of RGX-314, with no new drug-related ocular adverse events in Cohorts 1-4. While 1 patient in Cohort 5 experienced a vision decrease during the LTFU study, it was attributed to specific circumstances in the Phase 1/2a trial.

Patients in Cohort 3 demonstrated an improvement of 12 letters in visual acuity at 4 years, while those in Cohort 4 maintained their visual acuity with only a 5-letter reduction at 3 years. Additionally, both cohorts saw a significant reduction in annualized injection rates, marking a 67.0% and 58.4% decrease in injections, respectively, compared to the year prior to RGX-314 administration.

Reference
Vajzovic L. Long-Term Follow-Up Study Results from the Phase I/IIa RGX-314 Subretinal Delivery Gene Therapy Program in Neovascular Age-Related Macular Degeneration (nAMD). Presented at: EURETINA Congress 2023.