Ocular Therapeutix, Inc. announced plans to advance its wet age-related macular degeneration (AMD) candidate AXPAXLI (OTX-TKI) toward regulatory approval in early 2026.
Specifically: The company intends to submit a new drug application (NDA) based on data from its phase 2 SOL-1 clinical trial.
Let’s begin with AXPAXLI.
What it is: Formulated with anti-angigenic properties, this is an investigational, bioresorbable hydrogel intravitreal (IVT) implant
What it does: Continuously delivers axitinib (an FDA-approved small-molecule, multi-target tyrosine kinase inhibitor [TKI]) for proposed retinal indications such as:
- Diabetic macular edema (DME)
- Nonproliferative diabetic retinopathy (NPDR)
- See here for the latest phase 3 clinical trial news
- Wet age-related macular degeneration (AMD)
- Other vascular endothelial growth factor (VEGF)-mediated retinal diseases
Its delivery mode: An IVT-administered 25G needle with a 9-to-12-month target release
How exactly is this release enabled?
That would be via the company’s proprietary and patented ELUTYX technology platform, a bioresorbable polymer matrix hydrogel. Its purpose: To provide a localized sustained-release delivery of drugs (such as AXPAXLI)—effectively maximizing therapeutic efficacy while simultaneously minimizing systemic exposure.
Among its capabilities:
- Profile as an established hydrogel platform
- Continuous and consistent drug delivery over days, weeks, or months
- Biocompatible versatility, and tunable for use in various locations within the eye
Now, more about this wet AMD indication.
AXPAXLI is currently undergoing clinical evaluation in the phase 3 SOL program, consisting of:
- SOL-1 (NCT06223958)
- October 2023: Initiation kicked off, operating under a modified Special Protocol Assessment (SPA) through the FDA
- SOL-R (NCT06495918)
- August 2024: First patients enrolled
- SOL-X (open-label extension with 3-year follow-up)
The intent behind these studies: To enable more infrequent IVT dosing of AXPAXL, ideally every 6 months to (potentially) every 12 months (in other words: 1 to 2 injections per year).
What’s the clinical data showing thus far?
For those numbers, we’ll refer back to the 12-month topline data released from a pivotal phase 1 study NCT04989699), which evaluated AXPAXLI versus aflibercept.
As we reported in 2023, the following was observed among patients treated with a single AXPAXLI implant
- A continuous, sustained best-corrected visual acuity (BCVA; mean change from baseline = -1.0 letters)
- Sustained central subfield thickness (CST; mean change from baseline = +20.2 μm)
- See how this compared to aflibercept-treated patients
- 60% were rescue-free up to 12 months, with an additional four rescue-free at the 12-month mark
- 89% reduction in treatment burden over the 12-month period
- Check out the reported adverse reactions
Just to be clear, there’s no data from the SOL program yet, right?
That’s correct … for now. According to the company: 1-year clinical data from the SOL-1 study (the first of those three in the phase 3 program) is reportedly on track to be released in Q1 2026.
Tell me more about this.
Following recent interactions with the FDA’s Division of Ophthalmology—and pending positive SOL-1 data in Q1—Ocular Therapeutix shared plans to submit its wet AMD-indicated NDA for AXPAXLI in the coming months.
- The timeframe for this: “Shortly after” the release of this (hopefully positive) SOL-1 data.
Explain the significance of SOL-1 for AXPAXLI.
As Pravin U. Dugel, MD, the company’s executive chairman, president, and CEO, noted, the phase 3 trial is currently the only:
- Wet AMD study evaluating superiority compared to a single injection of aflibercept (2 mg)
- Wet AMD registrational trial (that they’re aware of) was conducted 100% in alignment with the FDA’s draft guidance and feedback
See here for more on this supportive reasoning.
And how will the company achieve this accelerated review timeline for the NDA?
By planning to rely on the FDA’s 505(b)(2) pathway.
What this is: A drug approval route through the federal agency that enables companies (such as Ocular Therapeutix) to partly rely on prior clinical findings used to support prior FDA-approved drugs—effectively speeding up the clinical review process.
- In this case: AXPAXLI’s axitinib (its active ingredient) has already received regulatory approval for non-ophthalmic indications.
But aren’t two clinical datasets required for an NDA submission?
Typically … yes; from two “adequate and well-controlled clinical trials to demonstrate the safety and efficacy” of an ophthalmic candidate.
- However, as the company noted, this could be changing: “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.
So SOL-1 could be just the only data needed for AXPAXLI’s application?
Potentially. Ocular Therapeutix added that it’ll be working with the FDA on its submission and the necessary next steps for regulatory approval, particularly following the release of that 1-year SOL-1 data.
As always, stay tuned for updates on this!