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REGENXBIO Inc. announced it will initiate a pivotal phase IIb/III clinical trial for investigational surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy (DR) using suprachoroidal delivery and a corresponding amendment to its eyecare collaboration with AbbVie.
This clinical advancement follows new, positive two-year data from the phase II ALTITUDE trial and long-term follow-up study, which enables the initiation of a global clinical program for DR.
"Advancing our DR programme 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."
"DR is a progressive disease, with most patients eventually developing vision threatening events (VTEs) and is the leading cause of blindness among working age adults," said Primal Kaur, M.D., senior vice president, global development of immunology, neuroscience, eye care and specialty, AbbVie. "We are excited to advance this clinical development program with the goal of helping to address these unmet needs and bring an additional option to patients living with DR."
In the phase II ALTITUDE trial, sura-vec was well tolerated in subjects with non-proliferative diabetic retinopathy (NPDR) at dose levels 1, 2, and 3. As of June 9, 2025, there were no drug-related serious adverse events. No intraocular inflammation was observed through two years at dose level 3 (1.0x1012 GC/eye) (n = 15) with short-course topical prophylactic steroids.
Updated phase II ALTITUDE results will be presented at a future medical meeting.
A pivotal two-part placebo-controlled phase IIb/III trial will be initiated. Supported by the phase II dose level 3 data, the primary endpoint will be > 2-step DRSS improvement at 1 year. Site selection is in progress.
AbbVie and REGENXBIO executed an amendment to the collaboration and license agreement established between the two companies on September 10, 2021. The amendment includes an updated milestone structure for the DR programme, under the terms of which AbbVie will pay REGENXBIO $100 million upon first subject dosed in the phase IIb/III trial and an additional $100 million upon first subject dosed in a second phase III clinical trial. REGENXBIO will pay for all costs for phase IIb of the phase IIb/III trial.
The amendment also reflects AbbVie's continued investment across the broader sura-vec program. AbbVie will independently advance and pay all costs for a new phase III ACHIEVE trial in wet AMD. This randomized controlled trial will assess the potential reduction in injection burden and preservation of long-term vision of sura-vec compared to standard of care.
Sura-vec is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy and other chronic retinal conditions. Sura-vec consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). Sura-vec is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide. DR affects nearly 10 million people in the United States alone. The spectrum of DR severity ranges from non-proliferative diabetic retinopathy (NPDR) to proliferative diabetic retinopathy (PDR). As DR progresses, a large proportion of patients develop vision threatening complications, including diabetic macular edema (DME) and neovascularization that can lead to blindness. Current treatment options for patients with NPDR typically include "watchful waiting" or anti-VEGF treatment. For patients with PDR, current treatment options include anti-VEGF treatment or retinal laser; surgical treatment may be required for advanced PDR.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy.
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