Ocular Therapeutix Secures FDA Special Protocol Assessment for NPDR Trial of Axpaxli

Ocular Therapeutix has received written agreement from the US Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the registrational trial design of Axpaxli (OTX-TKI) in the treatment of nonproliferative diabetic retinopathy (NPDR).

About Axpaxli (OTX-TKI)

Axpaxli is an investigational, bioresorbable intravitreal hydrogel incorporating axitinib, a small molecule, multi-target tyrosine kinase inhibitor with anti-angiogenic properties. The therapy is under development for wet age-related macular degeneration (wet AMD), diabetic retinopathy, diabetic macular edema (DME), and other retinal diseases.

“Securing a Special Protocol Assessment agreement from the FDA for our planned NPDR trial represents a major milestone as we look to broaden the impact of Axpaxli beyond wet AMD and into diabetic eye disease,” said Pravin U. Dugel, MD, Executive Chairman, President, and CEO of Ocular Therapeutix. “This formal agreement ensures direct FDA alignment with our proposed approach for NPDR and provides us with a clear regulatory path forward. We are confident Axpaxli will be successful in the trial design and primary endpoint agreed upon with the FDA.”

Promising Early-Stage Data from HELIOS Trial

According to Daniel F. Martin, MD, Vice Chair for Clinical Affairs and Professor of Ophthalmology at the Emory University School of Medicine, results from the Phase 1 HELIOS trial showed encouraging signals:

       • With a single Axpaxli injection, no NPDR patients experienced disease progression or vision-threatening complications at 48 weeks.

       • In the sham control arm, 25% of patients had worsening disease, and nearly 40% developed a vision-threatening complication.

       • All patients treated with Axpaxli who entered the study with non-center involved DME experienced improvement.

“Though these data are early, they point to the possibility of not only improving outcomes but also reshaping how physicians approach proactive care in NPDR with and without DME,” Dr. Martin noted.

Next Steps and Strategic Plans

Ocular Therapeutix will present additional details on its NPDR and DME strategy at its Investor Day in New York City on September 30, 2025. Registration for the event is available through the company’s website.