▎Edited by the WuXi AppTec content team

REGENXBIO Inc. recently announced positive interim results from the Phase 2 clinical trial of the investigational gene therapy ABBV-RGX-314, developed in collaboration with AbbVie Inc., for the treatment of wet age-related macular degeneration (AMD). Patients who received the treatment demonstrated good safety and tolerability.Patients receiving the highest dose of ABBV-RGX-314 showed an up to 80% reduction in the need for subsequent anti-vascular endothelial growth factor (VEGF) treatment.ABBV-RGX-314 is a single, in-clinic treatment that provides sustained anti-VEGF therapy to stabilize or improve vision in patients with wet AMD.

This trial is a Phase 2, multi-center, open-label, randomized, active-controlled, dose-escalation study. It focuses on evaluating the efficacy, safety, and tolerability of ABBV-RGX-314 administered via the suprachoroidal route in patients with wet AMD. The primary endpoint is the mean change in visual acuity at Week 40, measured by Best Corrected Visual Acuity (BCVA), in patients treated with ABBV-RGX-314 compared to those receiving monthly ranibizumab injections. Secondary endpoints include the mean change in central retinal thickness (CRT) and the number of anti-VEGF drug injections required after treatment with ABBV-RGX-314.

As of November 6, 2023, 106 patients responded well to the suprachoroidal delivery of ABBV-RGX-314 across three dose levels, with no drug-related serious adverse events (SAEs) reported. During the 6-month study period, all treatment-related adverse events (TEAEs) associated with the eye were mild or moderate, including increased intraocular pressure, scleritis, and conjunctival hyperemia. The incidence of mild intraocular inflammation was similar at the first and second dose levels, while mild to moderate intraocular inflammation was observed in Groups 4 and 5 at the third dose level. All cases of intraocular inflammation resolved after treatment with topical corticosteroids. Notably, no cases of intraocular inflammation were observed in Group 6 (n=21) at the third dose level, where patients received short-term prophylactic topical corticosteroid treatment following ABBV-RGX-314 administration.

▲ABBV-RGX-314 treatment significantly reduces the need for patients to receive anti-VEGF therapy (Image source: REGENXBIO Inc. official website)

Patients treated with ABBV-RGX-314 continue to show stable BCVA and CRT after six months.In addition, a significant reduction in the need for anti-VEGF treatments was observed following treatment with ABBV-RGX-314, wherePatients receiving the third dose level treatment experienced the highest reduction in annualized injection rates, reaching 80%, with 50% of patients not requiring further injections.

ABBV-RGX-314 uses an adeno-associated virus 8 (AAV8) vector to carry a transgene expressing an anti-VEGF antibody fragment. The protein expressed in vivo blocks VEGF signaling, inhibiting angiogenesis and retinal fluid accumulation.Beyond the treatment of wet AMD, the two companies are also exploring the potential of ABBV-RGX-314 in treating diabetic retinopathy and other chronic retinal diseases.

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