Ocular Therapeutix™ Announces First Patient Randomized in HELIOS-3 Phase 3 Registrational Program for AXPAXLI™ in NPDR

First patient randomized into HELIOS-3, which along with HELIOS-2 form the basis of AXPAXLI’s registrational program in non-proliferative diabetic retinopathy (NPDR)

HELIOS-2 and HELIOS-3 are global, complementary superiority trials using a novel ordinal diabetic retinopathy severity scale (DRSS) primary endpoint

Targeting a broad diabetic retinopathy (DR) label by including patients with non-center-involved diabetic macular edema (non-CI-DME)

Excerpt from the Press Release:

BEDFORD, Mass., Nov. 24, 2025 (GLOBE NEWSWIRE) — Ocular Therapeutix, Inc. (NASDAQ: OCUL, “Ocular”), an integrated biopharmaceutical company committed to redefining the retina experience, today announced that the first patient has been randomized in the HELIOS-3 Phase 3 registrational program for AXPAXLI (also known as OTX-TKI) for the treatment of non-proliferative diabetic retinopathy (NPDR).

“Initiating the HELIOS registrational program marks a pivotal step toward redefining treatment for diabetic retinal disease. While there are more than 6 million NPDR patients in the U.S., fewer than 1% receive therapy today, due mostly to the burden of frequent injections in this working age population. AXPAXLI’s potential to deliver efficacy with attractive durability could help hundreds of thousands, if not millions more patients preserve vision,” said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix. “HELIOS-2 and HELIOS-3 are complementary superiority studies targeting a comprehensive and broad DR label. The HELIOS study designs reflect our intentional and strategic clinical philosophy: combining scientific rigor, outstanding clinical execution, and a focus on de-risking, with regulatory alignment to optimize the potential for success and a label that readily translates into broad adoptability. By aiming for a superiority-based DR label that spans the full continuum of disease – including NPDR and DME – we believe AXPAXLI could deliver a market opportunity that is not merely incremental, but transformative for patients, physicians, and payors around the world.”

The HELIOS program is comprised of two complementary superiority studies, HELIOS-2 and HELIOS-3, designed to evaluate whether early AXPAXLI treatment, as infrequent as every 12 months, can meaningfully alter the course of non-proliferative diabetic retinopathy (NPDR).

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