Ocular Therapeutix accelerates NDA submission plans for AXPAXLI for wet AMD

Ocular Therapeutix announced it plans to accelerate its intended New Drug Application (NDA) for AXPAXLI (OTX-TKI) for the treatment of wet age-related macular degeneration (wet AMD) to the US Food and Drug Administration (FDA).

The company plans to submit the NDA following positive 1-year data from the ongoing SOL-1 phase 3 clinical trial, which the company noted is on track for the first quarter of 2026.¹

SOL-1 (NCT06223958) is a multi-center, double-masked, randomized (1:1), parallel group study in more than 100 clinical trial sites located in the US and Argentina. As reported earlier,² the trial completed randomization of 344 evaluable treatment-naïve subjects with a diagnosis of wet AMD in the study eye in December 2024. The primary endpoint is the proportion of patients who maintain visual acuity, defined as a loss of <15 early treatment diabetic retinopathy study (ETDRS) letters of best corrected visual acuity (BCVA), at week 36.

The trial had an 8-week loading segment prior to randomization, during which patients who have 20/80 vision or better and a central subfield thickness (CSFT) of ≤500 μm received 2 doses of aflibercept 2 mg at week -8 and week -4. Patients who achieved BCVA of 20/20 on day 1 or gained at least 10 ETDRS letters along with a CSFT of ≤350 μm were then randomized to receive one dose of AXPAXLI or aflibercept 2 mg.

Patients are then re-dosed at week 52 and week 76 with their respective initial treatment and will be given monthly assessments until the end of year 2.

Pravin U Dugel, MD, executive chairman, president, and CEO of Ocular Therapeutix, commented on the trial in a press release from the company.

“At Ocular Therapeutix, we have always been courageous, opportunistic, and bold in our efforts to redefine retina. Based on recent developments, we now intend to submit our NDA for AXPAXLI in wet AMD shortly after SOL-1 year one data, assuming positive results.”

“SOL-1 is the only ongoing Phase 3 retina trial currently being conducted under a [SPA] agreement. It is also the only current wet AMD trial exploring superiority compared to a single injection of aflibercept (2 mg) and the only wet AMD registrational trial that we are aware of that is being run completely in alignment with the FDA’s draft guidance and feedback. This triad of factors helps us make a compelling case for a truly differentiated NDA for AXPAXLI in the treatment of wet AMD,” said Dugel.

According to the company, historically the FDA has required 2 “adequate and well-controlled clinical trials” to demonstrate the safety and efficacy of ophthalmic product candidates, most notably for larger indications of use such as wet AMD. The company stated that “recent statements from FDA leadership indicate that the agency is potentially moving to requiring only a single registrational trial for approval, as long as the trial is adequately powered and controlled.”

Due to this, combined with its SPA agreement for SOL-1, the company plans to work with the FDA to submit its NDA for AXPAXLI in wet AMD following year 1 results from SOL-1.

“Recent FDA leadership comments on the potential for a single registrational trial for new product candidates stressed the importance that these studies be well powered and well controlled. Being a superiority trial, SOL-1 is substantially powered compared to non-inferiority trials. Moreover, SOL-1 is well controlled, as the active and control arms have the same dosing cadence and do not use sham injections for masking, as per the FDA’s draft guidance and feedback. Since axitinib, the active component of AXPAXLI, is already approved in non-ophthalmic indications, we intend to leverage the 505(b)(2) pathway, which we believe has the potential to further accelerate our review timeline,” concluded Dugel.

AXPAXLI is an investigational, bioresorbable, intravitreal hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties.

References:

1. Ocular Therapeutix™ Announces Plans to Accelerate NDA Submission Timeline for AXPAXLI™ in Wet AMD. Published December 8, 2025. Accessed December 8, 2025. https://investors.ocutx.com/news-releases/news-release-details/ocular-therapeutixtm-announces-plans-accelerate-nda-submission

2. Crago SM. Over 300 patients randomized in Ocular Therapeutix's SOL-1 phase 3 trial for OTX-TKI. Published December 4, 2024. Accessed December 8, 2025. https://www.ophthalmologytimes.com/view/over-300-patients-randomized-in-ocular-therapeutix-sol-1-phase-3-trial-for-otx-tki