Clinical Trial Overview and Scientific Foundations

Ocular Therapeutix, Inc. highlighted compelling clinical data for its investigational axitinib intravitreal hydrogel (OTX-TKI, also known as AXPAXLI™) at Clinical Trials at the Summit 2025 in Las Vegas. The presentation encompassed results from the Phase 1/2 HELIOS trial in diabetic retinopathy (DR), and the registrational Phase 3 SOL-R trial in wet age-related macular degeneration (wet AMD).

AXPAXLI employs Ocular’s proprietary ELUTYX™ bioresorbable hydrogel technology, designed to provide sustained drug delivery of axitinib over 6 to 12 months, significantly extending treatment intervals beyond current anti-VEGF therapies requiring monthly or bimonthly injections.

Professor Justis Ehlers, MD, an investigator at the Cleveland Clinic, emphasized the importance: “HELIOS data reveal durable volumetric retinal fluid reductions and meaningful disease stabilization in nonproliferative diabetic retinopathy without center-involved diabetic macular edema. OTX-TKI holds potential to transform long-term disease management for this patient population.”

Key Efficacy Outcomes from HELIOS and SOL-R

In HELIOS, OTX-TKI demonstrated sustained reduction in retinal-RPE volume, central subfield thickness, and intraretinal fluid without rescue anti-VEGF interventions over 48 weeks, compared to sham controls. Visual function stability and absence of progression to proliferative diabetic retinopathy or diabetic macular edema further substantiate clinical benefit.

The SOL-R trial, conducted across approximately 100 locations globally, is a multicenter, double-masked, randomized three-arm study comparing OTX-TKI dosed every six months to aflibercept dosed every 8 weeks (2 mg) and aflibercept high-dose every six months (8 mg). Unique trial design addressed masking challenges by aligning dosing intervals. Full enrollment was completed in mid-2025.

Pravin Dugel, MD, Executive Chairman & CEO at Ocular Therapeutix, stated: “Completing enrollment so rapidly in the largest retinal TKI study underscores our commitment to innovation. Together with SOL-1, SOL-R provides a complementary regulatory foundation to support a label for OTX-TKI with longer dosing intervals and potential superiority claims.”

Safety Profile and Manufacturing

Across both trials, OTX-TKI’s safety profile was consistent with known risks of intravitreal injections but with no unexpected adverse events or serious ocular inflammation. The bioresorbable hydrogel platform offers a reproducible manufacturing process that supports scale-up for commercial demand while maintaining product integrity.

Forward-Looking Statements and Market Potential

Ocular Therapeutix plans submissions to regulatory agencies following availability of 56-week data from SOL-R, with a view to redefine treatment paradigms in retinal vascular diseases through flexible, durable therapies. Funding rounds securing $85 million this quarter bolster operational readiness for commercial launch and pipeline expansion.