Regenxbio reports positive results of phase 1/2a RGX-314 trial
Regenxbio will initiate a pivotal program for subretinal delivery of RGX-314 in patients with wet age-related macular degeneration later this year, following the release of positive 1-year data from a phase 1/2a trial.
The company plans to advance to AAVIATE, a phase 2 trial of the drug, with patient enrollment beginning in the third quarter, according to a press release.
The phase 1/2a trial evaluated 42 patients with severe wet AMD who required frequent anti-VEGF injections. In five cohorts, the doses ranged from 3 × 109 GC per eye to 2.5 × 1011 GC per eye.
Generally, all cohorts tolerated RGX-314 well, with 18 serious adverse events reported in 11 patients, 17 of which were not related to the drug. The one event that may have been drug related involved a significant decrease in vision in a patient experiencing retinal pigmentary changes involving the macula.
Nonserious adverse events included postoperative conjunctival hemorrhage in 69% of patients, postoperative inflammation in 36%, eye irritation in 17%, eye pain in 17% and postoperative visual acuity reduction in 17%.
Patients in cohort 4 and 5 had stable visual acuity with an average change in best corrected visual acuity of +4 letters and –2 letters, respectively, from baseline. Retinal thickness decreased by an average of –61 µm and –79 µm, respectively. Both cohorts had a significant reduction in anti-VEGF treatment burden compared with the previous 12 months. Cohort 4 had a 61% reduction in anti-VEGF injections, while cohort 5 had an 85% reduction.
Regenxbio plans to announce interim data from AAVIATE by the end of the year. AAVIATE is a multicenter, open-label, randomized, active-controlled, dose-escalation study measuring efficacy, safety and tolerability of the suprachoroidal delivery of RGX-314.