Ocular Therapeutix has received a Special Protocol Assessment (SPA) from the U.S. Food and Drug Administration (FDA) for its planned registrational trial of AXPAXLI™ (OTX-TKI) in non-proliferative diabetic retinopathy (NPDR). The SPA provides formal FDA agreement on trial design, endpoints, and analysis, giving Ocular a clear regulatory pathway for advancing AXPAXLI beyond wet age-related macular degeneration into diabetic eye disease.
The company plans to outline its development strategy for NPDR and diabetic macular edema (DME) during its Investor Day on September 30, 2025, in New York City.
“[This] represents a major milestone as we look to broaden the impact of AXPAXLI beyond wet AMD and into diabetic eye disease,” notes Pravin U. Dugel, Executive Chairman, President, and CEO of the company. “With diabetic retinopathy affecting nearly 9 million people in the US alone, we believe AXPAXLI… could offer a transformative approach to improving outcomes while preventing or substantially reducing the rate of vision-threatening complications in these patients.”
Clinical experts welcomed the news. In the Phase 1 HELIOS trial, NPDR patients who received a single AXPAXLI injection showed no signs of disease progression or vision-threatening complications at 48 weeks, compared to nearly 40% who developed complications in the sham control arm.
“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,” noted Daniel F. Martin, Vice Chair for Clinical Affairs and Professor of Ophthalmology at the Emory University School of Medicine.
David M. Brown, Retina Consultants of America, added that a durable treatment like AXPAXLI could “shift the paradigm from reactive treatment to proactive disease management” in a largely untreated high-risk population.
The company plans to outline its development strategy for NPDR and diabetic macular edema (DME) during its Investor Day on September 30, 2025, in New York City.
“[This] represents a major milestone as we look to broaden the impact of AXPAXLI beyond wet AMD and into diabetic eye disease,” notes Pravin U. Dugel, Executive Chairman, President, and CEO of the company. “With diabetic retinopathy affecting nearly 9 million people in the US alone, we believe AXPAXLI… could offer a transformative approach to improving outcomes while preventing or substantially reducing the rate of vision-threatening complications in these patients.”
Clinical experts welcomed the news. In the Phase 1 HELIOS trial, NPDR patients who received a single AXPAXLI injection showed no signs of disease progression or vision-threatening complications at 48 weeks, compared to nearly 40% who developed complications in the sham control arm.
“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,” noted Daniel F. Martin, Vice Chair for Clinical Affairs and Professor of Ophthalmology at the Emory University School of Medicine.
David M. Brown, Retina Consultants of America, added that a durable treatment like AXPAXLI could “shift the paradigm from reactive treatment to proactive disease management” in a largely untreated high-risk population.
