Ocular Therapeutix Reports Superiority Data for AXPAXLI in Wet AMD Phase 3 Trial

Ocular Therapeutix, Inc. announced positive topline findings from the SOL-1 Phase 3 superiority study evaluating its investigational candidate AXPAXLI for the treatment of wet age-related macular degeneration. The landmark trial met its primary endpoint and demonstrated statistically significant improvements in vision outcomes compared to aflibercept.

At Week 36, 74.1% of patients in the AXPAXLI arm versus 55.8% of patients in the aflibercept arm lost fewer than 15 Early Treatment Diabetic Retinopathy letters from baseline, with a risk difference of 17.15% and a p-value of 0.0006 per the pre-specified statistical model. The observed difference was 18.3%.

AXPAXLI also met its secondary objective, a durability assessment at Week 52, with high statistical significance. At Week 52, 65.9% of patients in the AXPAXLI arm compared to 44.2% in the aflibercept arm maintained visual acuity, with a risk difference of 21.1%, an observed difference of 21.7%, and a p-value of less than 0.0001.

Pre-specified exploratory endpoints showed that patients who did not require rescue injections had rescue-free rates at Weeks 24, 36, and 52 of 80.6%, 74.7%, and 68.8% in the AXPAXLI arm versus 72.1%, 56.4%, and 47.7% in the aflibercept arm. The observed differences for these weeks in favor of the AXPAXLI arm were 8.5%, 18.3%, and 21.1%.

As of February 5, 2026 (Week 52), AXPAXLI was reported to be generally well-tolerated with no treatment-related ocular or systemic serious adverse events observed. There were no events of endophthalmitis, occlusive or non-occlusive retinal vasculitis, retinal detachment, or implant migration to the anterior chamber in the AXPAXLI arm.

AXPAXLI is an investigational, bioresorbable hydrogel intravitreal implant formulated with anti-angiogenic properties that provides continuous delivery of axitinib, an FDA-approved small-molecule, multi-target tyrosine kinase inhibitor. The implant is administered via an intravitreal 25G needle with a 9-to-12-month target release. The therapy leverages the company's proprietary ELUTYX bioresorbable hydrogel technology.

SOL-1 is operating under a Special Protocol Assessment. The study evaluated a single injection of AXPAXLI versus a single aflibercept 2 mg injection among randomized newly-diagnosed wet AMD patients. Patients were randomized after showing a peak response to two aflibercept injections.

Ocular Therapeutix will present detailed results from the SOL-1 trial at the 49th Macula Society Annual Meeting on February 27, 2026. The company will also host an investor webcast on March 2, 2026, to review the data and discuss the presentations.

Ocular intends to submit a New Drug Application based on SOL-1 data, subject to planned formal discussions with the U.S. FDA. If approved, AXPAXLI could be the first tyrosine kinase inhibitor to be commercialized in wet AMD, and potentially the only therapy with a superiority label and best-in-disease durability.

AXPAXLI is being developed for the treatment of wet age-related macular degeneration, a leading cause of vision loss among older adults, and is also under investigation for diabetic retinal diseases, including non-proliferative diabetic retinopathy. Proposed retinal indications other than wet AMD include diabetic macular edema, nonproliferative diabetic retinopathy, and other vascular endothelial growth factor-mediated retinal diseases.

Upcoming milestones include the SOL-R Trial, a Phase 3 non-inferiority study in wet AMD with topline data expected in Q1 2027, and the SOL-X Extension, an open-label safety follow-up study planned for Q2 2026, allowing patients from SOL-1 and SOL-R to continue for long-term monitoring. The HELIOS-3 Trial is an ongoing Phase 3 superiority study assessing AXPAXLI in patients with moderately severe to severe non-proliferative diabetic retinopathy without center-involved diabetic macular edema.

The company ended the year 2025 with total cash and cash equivalents of $737.1 million.