Ocular Therapeutix says the FDA has agreed that one finished Phase 3 trial, plus a safety readout from a second, is enough to support its application for AXPAXLI in wet age-related macular degeneration.
The company laid out the plan at an investor day on June 17. It stems from a Type C meeting with the agency in May 2026. The new drug application is due in the fourth quarter of 2026, built on week 52 efficacy and safety data from SOL-1 and interim safety data from SOL-R.
SOL-R’s own efficacy results are no longer part of the package. An interim safety analysis in the fourth quarter is meant to push the safety database past 300 patients across the two trials, a number the company says matches what the FDA requires.
Ocular will file under the 505(b)(2) pathway, which it says could cut up to 60 days from the review. A pre-NDA meeting with the agency was planned for the third quarter to settle the format and content of the filing. If the drug is approved, the company said, patients could have it in 2027.
SOL-R gets a harder job
With the filing no longer leaning on SOL-R, the company is rebuilding the trial. It will stay masked through week 96, and topline results now slip to the first quarter of 2028. A new key secondary endpoint will test whether AXPAXLI beats 8 mg aflibercept on mean change in best corrected visual acuity at that point. Ocular also hopes to show that its drug prevents fibrosis and atrophy better than 2 mg aflibercept.
Chief executive Pravin Dugel described SOL-1 as the first successful wet AMD trial in which a novel agent showed superiority over an approved anti-VEGF drug. “The data for AXPAXLI from SOL-1 showed unmatched durability and a level of sustained disease control that is simply unprecedented in this space,” he said. He added that the company is now “in the extraordinary position” of using SOL-R to pursue its long-term aim of becoming the best-in-disease agent for wet AMD.
The company says the superiority endpoint could sharpen the drug’s label and help it win broad global adoption, if approved.
Diabetic retinopathy trimmed to one trial
Ocular is also narrowing its diabetic retinopathy work. HELIOS-3, which compares AXPAXLI dosed every 12 months against sham in non-proliferative diabetic retinopathy, becomes the single registrational trial. Dugel pointed to up to 12 months of durability seen in SOL-1 and HELIOS-1, and said market research shows retina specialists strongly favor a once-yearly regimen for that disease.
AXPAXLI, also called OTX-TKI, is a bioresorbable hydrogel implant that carries axitinib, a tyrosine kinase inhibitor with anti-angiogenic properties. It goes into the eye by intravitreal injection.
The company is based in Bedford, Massachusetts, and trades on Nasdaq as OCUL. The investor event began at 2:00 PM Eastern in New York, with virtual access.
Sources
- FDA Alignment Boosts Wet AMD Treatment Path
- Ocular Therapeutix Eyes Q4 FDA Filing for AXPAXLI After SOL-1 Win
- Ocular Therapeutix™ Investor Day to Highlight U.S. FDA Alignment on AXPAXLI™ NDA with Submission Planned for Fourth Quarter 2026
