REGENXBIO Inc. announced positive interim data from the Phase II AAVIATE® trial of ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) using suprachoroidal delivery. Wet AMD is a chronic, life-long disease with available anti-VEGF treatment options that may reduce the risk of blindness, but require frequent injections. Investigational ABBV-RGX the314 using suprachoroidal delivered is designed to be a one-time, in office treatment that has the potential to sustain constant anti-VEGF therapy and stabilize or improve vision long-term for wet AMD patients.

The new data presented at the Hawaiian Eye and Retina meeting in Maui, HI by John Pitcher, M.D., Eye Associates of New Mexico, includes 6-month results from two additional dose level 3 cohorts (Cohorts 5 and 6). ABBV-RGX -314, being developed in collaboration with AbbVie, is also being investigated as a potential one-time gene therapy for the treatment of diabetic retinopathy and other chronic retinal conditions. AAVIATE is a Phase II, multi-center, open-label, randomized, active-controlled, dose-escalation trial that is evaluating the efficacy, safety and tolerability of suprachoroidal delivery of ABBV-RGX-314 in patients with wet AMD.

The primary endpoint of the trial is mean change in vision in patients dosed with ABBV-RGX-314, as measured by best corrected visual acuity (BCVA) at Week 40 from baseline, compared to patients receiving monthly injections of ranibizumab. Other endpoints include mean change in central retinal thickness (CRT) and number of anti-vascular endothelial growth factor (anti-VEGF) intravitreal injections received following administration of ABBV-RGX-314. As of November 6, 2023, ABBV-RGX-314 suprachoroidal delivery was well tolerated across 106 patients from three dose levels.

No drug-related SAEs were reported. All treatment emergent adverse events (TEAEs) through 6 months in the study eye were mild or moderate and included conjunctival hemorrhage, increased intraocular pressure, episcleritis, and conjunctival hyperemia. Mild intraocular inflammation was reported at similar incidence rate in the first and second dose levels, with mild to moderate intraocular inflammation seen at the third dose level in Cohort 4 and 5. All intraocular inflammation resolved with topical corticosteroids.

Notably, there were zero cases of intraocular inflammation in dose level 3, Cohort 6 (n=21), in which patients received a short-course of prophylactic topical steroids following administration of ABBV-RGX-314. Patients treated with ABBV-RGX-314 continue to demonstrate stable BCVA and CRT at six months. In addition, a meaningful reduction in anti-VEGF treatment burden was observed following administration of ABBV-RGX-314, The highest reduction was seen in dose level 3, demonstrating an 80% reduction in annualized injection rate with 50% of patients remaining injection-free.