REGENXBIO Initiates Phase IIb/III Clinical Trial for Diabetic Retinopathy Treatment.

REGENXBIO Inc. announced the initiation of a pivotal Phase IIb/III clinical trial for surabgene lomparvovec (sura-vec) in diabetic retinopathy using suprachoroidal delivery. The decision comes after positive two-year data from the Phase II ALTITUDE trial, which demonstrated durable safety and efficacy through a single, in-office injection of sura-vec in non-proliferative diabetic retinopathy patients. The company will receive $100 million upon the first subject dosed in the Phase IIb/III trial and an additional $100 million upon the first subject dosed in a second Phase III trial.

July 2, 2025 - REGENXBIO Inc. (Nasdaq: RGNX) has announced the initiation of a pivotal Phase IIb/III clinical trial for its investigational drug surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy (DR) using suprachoroidal delivery. This move follows positive two-year data from the Phase II ALTITUDE® trial, which demonstrated durable safety and efficacy through a single, in-office injection of sura-vec in non-proliferative diabetic retinopathy (NPDR) patients [1].

The new trial is supported by the Phase II dose level 3 data and will have a primary endpoint of 2-step DRSS improvement at 1 year. REGENXBIO will receive $100 million upon the first subject dosed in the Phase IIb/III trial and an additional $100 million upon the first subject dosed in a second Phase III clinical trial. AbbVie, in collaboration with REGENXBIO, will pay for all costs for Phase IIb of the Phase IIb/III trial [1].

"Advancing our DR program to late-stage development brings sura-vec closer to being a potentially transformative new treatment for the millions of people living with DR," said Steve Pakola, M.D., Chief Medical Officer, REGENXBIO. "We remain committed to advancing this program to maximize its value and impact for patients worldwide" [1].

Diabetic retinopathy (DR) is a progressive disease that affects nearly 10 million people in the United States alone. It is the leading cause of blindness among working-age adults and can lead to vision-threatening complications, including diabetic macular edema (DME) and neovascularization [2].

Sura-vec is a potential one-time treatment for wet AMD, DR, and other chronic retinal conditions. It consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF) [1].

REGENXBIO, a biotechnology company on a mission to improve lives through the curative potential of gene therapy, has pioneered the field of AAV gene therapy. The company is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including surabgene lomparvovec for the treatment of wet AMD and diabetic retinopathy [1].

References:
[1] https://www.marketscreener.com/news/regenxbio-announces-pivotal-program-for-surabgene-lomparvovec-in-diabetic-retinopathy-ce7c5edfda88ff26
[2] Cheung N, Mitchell P, Wong TY. Diabetic retinopathy. Lancet. 2010;376(9735):124–36.