REGENXBIO Inc. announced plans to initiate a pivotal phase IIb/III clinical trial for its investigational therapy, surabgene lomparvovec (sura-vec, ABBV-RGX-314), in diabetic retinopathy (DR) via suprachoroidal delivery.
This move is paired with an amendment to its eye-care collaboration with AbbVie.
Key Drivers for the Trial Launch
- Positive two-year phase II ALTITUDE trial data.
- Long-term follow-up results enabling a global clinical program for DR.
Executive Comments
Steve Pakola, M.D., Chief Medical Officer, REGENXBIO:
“Advancing our DR programme to late-stage development brings sura-vec closer to becoming a transformative treatment for millions living with DR.”
Primal Kaur, M.D., SVP, Global Development, AbbVie:
“DR is progressive, leading to vision-threatening events and is the leading cause of blindness among working-age adults. We aim to address unmet needs and offer new patient options.”
Phase II ALTITUDE Trial Insights
- Patient Group: Non-proliferative diabetic retinopathy (NPDR).
- Dose Levels Tested: 1, 2, and 3.
- Safety Profile:
- Well tolerated across all dose levels.
- No drug-related serious adverse events.
- No intraocular inflammation through two years at dose level 3 (1.0×10¹² GC/eye) with short-course topical prophylactic steroids. - Sample Size: n = 15 for dose level 3.
- Updated results to be presented at a future medical meeting.
Upcoming Phase IIb/III Trial
- Design: Two-part, placebo-controlled.
- Primary Endpoint: > 2-step DRSS improvement at 1 year.
- Support Data: Phase II dose level 3 results.
- Status: Site selection in progress.
Collaboration and Licensing Amendment
- Original agreement signed: September 10, 2021.
- Updated Milestone Structure:
- $100M from AbbVie upon first subject dosed in phase IIb/III.
- Additional $100M upon first subject dosed in a second phase III trial. - Funding:
- REGENXBIO to fund phase IIb.
- AbbVie to fund the phase III ACHIEVE trial in wet AMD.
ACHIEVE Trial in Wet AMD
- Objective: Assess reduction in injection burden and preservation of long-term vision.
- Design: Randomized controlled trial.
- Comparison: Sura-vec vs standard of care.
About Sura-Vec
- Mechanism: NAV AAV8 vector encoding an antibody fragment to inhibit vascular endothelial growth factor (VEGF).
- Potential: One-time treatment for wet AMD, DR, and other chronic retinal conditions.
- Action: Blocks VEGF pathway to prevent growth of leaky blood vessels and retinal fluid accumulation.
Diabetic Retinopathy – Background
- Prevalence: Affects nearly 10 million people in the U.S.
- Age Group: Leading cause of vision loss in adults aged 24–75.
- Progression:
- Ranges from NPDR to proliferative diabetic retinopathy (PDR).
- Can lead to diabetic macular edema (DME) and blindness. - Current Treatments:
- NPDR: Watchful waiting or anti-VEGF treatment.
- PDR: Anti-VEGF, retinal laser, or surgery for advanced cases.
About REGENXBIO
Founded in 2009, REGENXBIO is a biotechnology leader in AAV gene therapy, dedicated to harnessing the curative potential of genetic medicine.
