Ocular Therapeutix secures FDA agreement for diabetic retinopathy trial

Published 12/08/2025, 12:06
Ocular Therapeutix secures FDA agreement for diabetic retinopathy trial

BEDFORD, Mass. - Ocular Therapeutix, Inc. (NASDAQ:OCUL), currently valued at $2.19 billion and trading near its 52-week high of $12.65, announced Tuesday it has received a Special Protocol Assessment (SPA) agreement from the U.S. Food and Drug Administration for its planned clinical trial of AXPAXLI in non-proliferative diabetic retinopathy (NPDR).

The SPA provides formal FDA alignment with the company’s proposed trial design and primary endpoint for evaluating AXPAXLI, also known as OTX-TKI, a bioresorbable intravitreal hydrogel incorporating axitinib.

Diabetic retinopathy affects nearly 9 million people in the United States, with approximately 6.4 million Americans currently living with NPDR. Despite this prevalence, fewer than 1% of NPDR patients receive treatment.

In the Phase 1 HELIOS trial, patients who received a single AXPAXLI injection showed no disease progression or vision-threatening complications at 48 weeks, while 25% of patients in the control group experienced worsening disease and nearly 40% developed vision-threatening complications.

"A safe, effective, annual intravitreal treatment that aligns with current retina practice dynamics would be a very appealing therapeutic option for people with diabetic retinopathy," said Daniel F. Martin, Vice Chair for Clinical Affairs and Professor of Ophthalmology at Emory University School of Medicine, in the company’s press release.

AXPAXLI is designed to potentially provide annual dosing, which could reduce the treatment burden associated with frequent injections that has limited adoption of current therapies.

The company plans to provide additional details about its strategy for AXPAXLI in NPDR and diabetic macular edema at its upcoming Investor Day scheduled for September 30, 2025, in New York City. The next earnings report is expected on November 10, 2025, with InvestingPro data showing analysts anticipate a sales decline in the current year.

In other recent news, Ocular Therapeutix reported its second-quarter 2025 financial results, revealing a mixed performance. The company posted revenue of $13.5 million, which was slightly below the projected $13.56 million, but it did exceed Raymond James’ estimate of $12.0 million. However, Ocular Therapeutix reported a diluted earnings per share loss of $(0.39), missing the forecasted loss of $(0.35). Expenses for the quarter were $51.1 million, surpassing the anticipated $43.1 million. Despite these results, Raymond James reiterated a Strong Buy rating with a $19.00 price target, while Clear Street maintained a Buy rating at $18.00. TD Cowen also reiterated a Buy rating with a $14.00 price target, as the company anticipates Phase III trial results in early 2026. Revenue from Dextenza saw unit sales up 5% year-over-year, with revenues increasing approximately 25% quarter-over-quarter. However, revenue was down about 18% year-over-year, attributed to challenging reimbursement conditions.

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