Regenxbio announced that the first patient has been dosed in the phase 2b/3 NAAVIGATE trial evaluating the investigational gene therapy surabgene lomparvovec (sura-vec; ABBV-RGX-314) for nonproliferative diabetic retinopathy (NPDR).
Sura-vec is an investigational adeno-associated virus serotype 8 (AAV8)-based gene therapy designed to provide sustained intraocular expression of an anti-VEGF antibody fragment following a single administration. Regenxbio and AbbVie are jointly developing sura-vec for diabetic retinopathy (DR), neovascular age-related macular degeneration (nAMD), and other chronic retinal diseases under a collaboration and license agreement established in 2021. The companies amended the agreement in 2025 to support the pivotal DR program. Dosing the first patient in NAAVIGATE triggered a $100 million milestone payment from AbbVie to Regenxbio under the amended agreement.
NAAVIGATE is a multicenter, randomized, masked, sham-controlled study evaluating suprachoroidal delivery of sura-vec in patients with NPDR without center-involved diabetic macular edema. Participants receive a single administration of sura-vec at a dose of 1.0×10^12 genome copies (GC)/eye, followed by a short course of topical corticosteroids. The primary endpoint is the proportion of patients achieving greater than a 2-step improvement on the Diabetic Retinopathy Severity Scale at 1 year. The phase 2b portion is expected to enroll approximately 135 patients in the United States.
The pivotal study follows 2-year results from the phase 2 ALTITUDE trial, in which dose level 3 demonstrated durable efficacy with no intraocular inflammation among 15 treated patients, according to the company.
Regenxbio also plans to present 2.5-year follow-up data from ALTITUDE and 5-year data from its phase 1/2a subretinal nAMD study at the American Society of Retina Specialists annual meeting in July. Topline results from the pivotal ATMOSPHERE and ASCENT trials evaluating subretinal sura-vec for nAMD are expected in the fourth quarter of 2026. OM