REGENXBIO reports three-year retinopathy improvement in 6 of 10
The small ALTITUDE follow-up lacks a control comparison; the dose is being tested in the sham-controlled NAAVIGATE trial.
Adrian Kessler
An improvement of more than two steps in diabetic retinopathy severity was recorded for six of 10 higher-dose participants at three years, as REGENXBIO Inc. NASDAQRGNX reported new ALTITUDE follow-up data on Thursday. The result adds follow-up evidence for the dose in the Phase IIb/III NAAVIGATE trial, which measures improvement against sham at its 1-year primary endpoint.
The Sept. 24 release covers participants with non-proliferative diabetic retinopathy who received one suprachoroidal injection of surabgene lomparvovec, known as sura-vec. Among the 10 Dose Level 3 participants with three-year visits, six reached the severity-scale threshold without further treatment for diabetic retinopathy or a vision-threatening event.
REGENXBIO is a Maryland-based small-cap gene therapy developer working on retinal and rare inherited diseases.
Its NASDAQRGNX shares were virtually unchanged at US$7.25 at 2:38 p.m. EDT on Sept. 25.
The Three-Year Group Had No Reported Control Comparison
The company's data cutoff was Aug. 17. It also said three of four participants who had improved by one step at one year without supplemental anti-VEGF injections reached the greater than two-step threshold by year three without additional diabetic-retinopathy treatment.
No new sura-vec-related safety signal or intraocular inflammation was reported among 17 participants followed for three years after short-course preventive steroid drops. The Sept. 24 update did not provide a control-group result, p-value or confidence interval for the 10-person efficacy subset, so the percentage alone cannot establish an advantage over another treatment or observation.
"Building on the positive data seen at two and two and a half years after a single treatment, the three-year data continues to show meaningful improvements in DRSS alongside a reduction in disease progression," REGENXBIO chief medical officer Steve Pakola said in the Sept. 24 release.
The gene therapy is designed to make an antibody fragment that blocks vascular endothelial growth factor, a driver of abnormal, leaky blood vessels in the retina. The company is developing it with AbbVie Inc. NYSEABBV, whose own trial page lists an RGX-314 Phase II study in diabetic retinopathy.
NAAVIGATE Will Test the Dose Against Sham Treatment
In its Aug. 2025 pivotal-program announcement, REGENXBIO set a primary endpoint of greater than two-step improvement on the Diabetic Retinopathy Severity Scale at one year. Its Aug. 6, 2026 quarterly release described NAAVIGATE as randomized, masked and sham-controlled, and said the first participant had been dosed in June. Neither the pivotal-program announcement nor the Sept. 24 update describes FDA feedback on that design.
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REGENXBIO calculated pro forma cash, equivalents and marketable securities of about US$313 million as of June 30, including July funding. That funding included a US$100 million AbbVie milestone and about US$108 million in net share-offering proceeds. In its Aug. 6 update, the company projected a cash runway into the fourth quarter of 2027.
The commercial benchmark includes Regeneron Pharmaceuticals, Inc. NASDAQREGN, whose Eylea HD received FDA approval for diabetic retinopathy in August 2023. Regeneron's release says patients with that indication receive injections every eight to 12 weeks after three initial monthly doses. A single-dose benefit would address that repeat-treatment schedule if confirmed in a controlled trial.
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The iShares Biotechnology ETF NASDAQIBB traded at US$209.50 at 2:39 p.m. EDT on Sept. 25.
REGENXBIO expects topline results in the fourth quarter of 2026 from ATMOSPHERE and ASCENT, pivotal trials of sura-vec in wet age-related macular degeneration using a different delivery route.
