Key Takeaways

  • Ocular Therapeutix has aligned with the FDA on a planned fourth-quarter 2026 NDA submission for Axpaxli in wet AMD based on SOL-1 efficacy and safety data plus interim SOL-R safety results
  • The company is modifying the SOL-R trial to focus on long-term differentiation from aflibercept, including visual acuity superiority and retinal outcome assessments through Week 96
  • Ocular is also streamlining its diabetic retinopathy program, advancing a single global phase 3 study, HELIOS-3, evaluating once-yearly Axpaxli in non-proliferative diabetic retinopathy.

Ocular Therapeutix announced that it has reached alignment with the FDA on plans to submit a new drug application (NDA) for Axpaxli (also known as OTX-TKI) for the treatment of wet age-related macular degeneration (AMD) in the fourth quarter of 2026. The planned submission is expected to be based on efficacy and safety findings from the phase 3 SOL-1 trial together with interim safety data from the ongoing SOL-R study.

The company disclosed the regulatory update ahead of its Investor Day presentation, describing the FDA interaction as a key milestone in advancing Axpaxli toward potential commercialization. According to Ocular, the agency agreed that SOL-1 could serve as the pivotal efficacy study supporting the application, with additional confirmatory evidence and safety data included as part of the submission package.

“Our consistently strong execution has brought us to a pivotal milestone today,” said Pravin U. Dugel, MD, executive chairman, president, and CEO of Ocular Therapeutix, in a company statement. “We are thrilled to announce that we have FDA alignment on our plans to submit the Axpaxli NDA for wet AMD in the fourth quarter of 2026, based on SOL-1 efficacy and safety data, combined with interim safety data from SOL-R.”

The company reported that SOL-1 met its primary endpoint, demonstrating superiority on maintenance of vision at Week 36 with a P{-value of 0.0006. Ocular noted that the treatment effect strengthened over time and was supported by anatomical outcomes and statistically significant sensitivity analyses. The study was conducted under a Special Protocol Assessment agreement with the FDA.

Ocular plans to submit the NDA through the FDA’s 505(b)(2) pathway, which may allow for a more streamlined review process for modified versions or formulations of previously approved therapies. The company said the submission will include a broad package of confirmatory evidence, including mechanistic, pharmacodynamic, animal model, natural history, class-consistency, and real-world data.

To satisfy safety exposure requirements, Ocular intends to conduct an interim analysis of SOL-R in the fourth quarter of 2026. The combined safety database from SOL-1 and SOL-R is expected to exceed 300 patients with at least 1 year of treatment exposure, a benchmark the company cited as consistent with FDA expectations for neovascular AMD development programs.

SOL-R Modified to Support Long-Term Differentiation

With SOL-R efficacy data no longer expected to be part of the initial NDA review package, Ocular is revising the trial to focus on longer-term differentiation from aflibercept. The company said it plans to maintain masking through Week 96 and add new secondary endpoints, including an assessment of superiority in best-corrected visual acuity compared with aflibercept 8 mg. Investigators will also evaluate fibrosis and atrophy outcomes relative to aflibercept 2 mg. Topline results from SOL-R are now anticipated in the first quarter of 2028.

“SOL-1 trial results potentially meet this high bar of a single trial approval with the combined data from the planned SOL-R interim analysis providing the required greater than 300 patients for the safety dataset,” said Arshad M. Khanani, MD, MA, FASRS, director of clinical research at Sierra Eye Associates and steering committee chair for the SOL program.

Diabetic Retinopathy Program Streamlined

In a separate development, Ocular announced plans to streamline its diabetic retinopathy development strategy by prioritizing a single global phase 3 registrational study, HELIOS-3. The trial will evaluate once-yearly Axpaxli against sham treatment in patients with non-proliferative diabetic retinopathy. The primary endpoint will assess a two-step or greater improvement in diabetic retinopathy severity score at Week 56.

The company said the revised strategy is intended to support global regulatory objectives while capitalizing on physician interest in a once-yearly treatment paradigm for diabetic retinal disease.

If approved, Ocular expects Axpaxli could reach the market in 2027 and potentially offer a longer-duration treatment option for patients with wet AMD.