Ocular Therapeutix, Inc. announced positive topline findings from the SOL-1 phase 3 superiority study evaluating its investigational candidate AXPAXLI (OTX-TKI) for the treatment of wet age-related macular degeneration (AMD).
The update follows the company’s December 2025 announcement of plans to submit a new drug application (NDA) with an accelerated review period (but more on that later).
First, a look at this candidate.
AXPAXLI is an investigational, bioresorbable hydrogel intravitreal (IVT) implant formulated with anti-angiogenic properties.
What it does: Provides a continuous delivery of an FDA-approved small-molecule, multi-target tyrosine kinase inhibitor (TKI) known as axitinib.
- Its delivery mode: An IVT-administered 25G needle with a 9-to-12-month target release
Its proposed retinal indications (other than wet AMD):
- Diabetic macular edema (DME)
- Nonproliferative diabetic retinopathy (NPDR)
- Other vascular endothelial growth factor (VEGF)-mediated retinal diseases
Explain how the implant releases axitinib.
For those details, click here to learn about the company’s proprietary and patented ELUTYX technology platform.
And its clinical performance thus far?
AXPAXLI is currently undergoing evaluation in the phase 3 SOL program, consisting of the SOL-1 study (our topic of discussion) and two other studies (see here and check out a status update on them here).
As such: The wet AMD data available up until this point was 12-month findings from a pivotal phase 1 study evaluating a single injection of AXPAXLI versus aflibercept.
Now let’s talk SOL-1.
The purpose behind this study (and the others in the SOL program): 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).
And for the sake of brevity: SOL-1 is evaluating a single injection of AXPAXLI (versus a single aflibercept 2 mg injection) among randomized newly-diagnosed wet AMD patients.
- Click here for a rundown on the superiority study’s setup, participants (a population specifically selected to lose vision), and outcome measures.
- See here for the randomization and re-dosing processes, in which patients were randomized after showing a peak response to two aflibercept injections.
Also, keep in mind: SOL-1 is operating under a Special Protocol Assessment (SPA).
And the primary objective?
To refresh: The primary outcome is the number of patients who maintain visual acuity (VA), defined as a loss of <15 Early Treatment Diabetic Retinopathy (ETDRS) letters of best-corrected visual acuity (BCVA) from baseline, measured at 36 weeks.
- Notably: This endpoint was designed to support a “potential superiority label in wet AMD,” as indicated in that aforementioned SPA agreement.
So did the study meet its goal?
Indeed—at Week 36 with a “high statistical significance.”
Specifically: 74.1% of patients in the AXPAXLI versus 55.8% of patients in the aflibercept arm lost <15 ETDRS letters from baseline to Week 36.
- Risk difference: 17.15%
- P-value = 0.0006 (per the pre-specified statistical model)
- Observed difference: 18.3%
How about secondary objectives?
This involved the number of patients who maintained VA at Week 52 (using the same analysis as the primary objective).
The outcome: AXPAZLI also met this “durability assessment” with “high statistical significance.”
- The numbers: 65.9% of patients in the AXPAXLI arm (compared to 44.2% in the aflibercept arm) achieved this.
- Risk difference: 21.1%
- P-value of < 0.0001
- Observed difference: 21.7%
- Risk difference: 21.1%
Any other outcomes worth noting?
Two other pre-specified exploratory endpoints:
- Patients who did not require rescue injections (as specified by the protocol rescue criteria)
- Rescue-free rates for Weeks 24, 36, and 52
- AXPAXLI arm: 80.6%, 74.7%, and 68.8%
- Aflibercept arm: 72.1%, 56.4%, and 47.7%
- The observed differences for these weeks (in favor of the AXPAXLI arm): 8.5%, 18.3%, and 21.1%
- Rescue-free rates for Weeks 24, 36, and 52
- Patients who maintained central subfield thickness (CST) within 30 μm from baseline
Now to the safety data.
As of Feb. 5 (Week 52), AXPAXLI was reported to be “generally well-tolerated” with no treatment-related ocular or systemic serious adverse events (SAEs) observed, Ocular Therapeutix reported.
These included no events of the following in the AXPAXLI arm: Endophthalmitis, occlusive or non-occlusive retinal vasculitis, retinal detachment, or implant migration to the anterior chamber.
And what did the company have to say about these findings?
Executive Chairman, President, and CEO Pravin U. Dugel, MD, noted the “exceptionally difficult bar” SOL-1 achieved by demonstrating superiority in wet AMD versus a single dose of aflibercept—and that two-thirds of AXPAXLI-treated patients were rescue-free through Week 52.
- He added: “The fact that AXPAXLI delivered these consistent, durable, and superior outcomes in this trial provides further evidence of axitinib’s potency and pan-VEGF suppression paired with the robustness and safety of our proprietary hydrogel platform.”
Nice! And what’s next for this study?
Considering it’s still ongoing, the company shared that participants will remain masked and have already received their second injection (AXPAXLI or aflibercept) at their Week 52 visit.
Moving forward: They’ll receive yet another injection at the Week 76 mark and will continue to be followed (on a masked-basis) for safety through the end of Week 104.
… and how about that aforementioned NDA?
As Ocular Therapeutix previously indicated, it intends to submit an NDA for AXPAXLI’s wet AMD indication based on this SOL-1 data (subject to planned discussion with the FDA, of course).
The timeframe for that: Within the next few months (or possibly even sooner).
- As for the potential: If approved, AXPAXLI could become the first TKI commercialized for wet AMD—and the “only therapy with a superiority label and best-in-disease durability."
In the meantime, the company plans to share more detailed clinical data on the SOL-1 study at the 2026 Macula Society Annual Meeting next week.