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

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

Axpaxli (OTX-TKI) is an investigational, bioresorbable intravitreal hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, under development for wet AMD, diabetic retinopathy, 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. 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,” said Pravin U. Dugel, MD, Executive Chairman, President and CEO of Ocular Therapeutix.

According to Daniel F. Martin, MD, Vice Chair for Clinical Affairs and Professor of Ophthalmology at the Emory University School of Medicine, in the phase 1 HELIOS trial, with a single Axpaxli injection, there were no NPDR patients who had disease progression or vision threatening complications at 48 weeks, while 25% of patients in the sham control arm experienced worsening disease and nearly 40% developed a vision threatening complication. In addition, there was improvement in all patients treated with Axpaxli who came into the study with non-center involved DME. 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.”

Ocular Therapeutix will share additional details on its NPDR and diabetic macular edema (DME) strategy at its Investor Day in New York City on September 30, 2025. Registration is available via the company’s website.