Ocular Therapeutix receives FDA agreement under special protocol assessment for registrational trial of AXPAXLI

Ocular Therapeutix announced it has received written agreement regarding a registrational trial design from the US Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for its planned clinical trial of AXPAXLI (also known as OTX-TKI) for the treatment of non-proliferative diabetic retinopathy (NPDR).

The company describes AXPAXLI as an investigational, bioresorbable, intravitreal hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties. It is currently being evaluated for the treatment of wet AMD, diabetic retinopathy, diabetic macular edema, and other retinal diseases.¹

Recently, the company closed enrollment in its SOL-R registrational trial of AXPAXLI in wet age-related macular degeneration (wet AMD). For the trial, the company noted¹ that it believes it has enrolled enough patients to ensure the target randomization of at least 555 patients in SOL-R. Patients are enrolled across approximately 100 sites in the US, Argentina, India, and Australia.²

Pravin U. Dugel, MD, executive chairman, president and CEO of Ocular Therapeutix commented on the written agreement from the FDA in a press release from the company.

“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 Dugel.

“We believe AXPAXLI, with its potential for annual dosing, could offer a transformative approach to improving outcomes while preventing or substantially reducing the rate of vision-threatening complications in these patients,” he continued.

Daniel F. Martin, MD, vice chair for clinical affairs and professor of ophthalmology at the Emory University School of Medicine commented on the efficacy of AXPAXLI in the press release saying, “Diabetic retinopathy remains the leading cause of vision loss among working-age adults. 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.”

The company noted that it plans to review its strategy and the next steps for AXPAXLI in NPDR and DME at its Investor Day in New York City on September 30, 2025.

References:

1. Ocular Therapeutix™ Receives FDA Agreement Under Special Protocol Assessment (SPA) for Registrational Trial of AXPAXLI™ in NPDR. Published August 12, 2025. Accessed August 13, 2025. https://www.biospace.com/press-releases/ocular-therapeutix-receives-fda-agreement-under-special-protocol-assessment-spa-for-registrational-trial-of-axpaxli-in-npdr

2. Harp MD. Ocular Therapeutix closes enrollment in phase 3 SOL-R clinical trial for wet AMD. Published June 2, 2025. Accessed August 13, 2025. https://www.ophthalmologytimes.com/view/ocular-therapeutix-closes-enrollment-in-phase-3-sol-r-clinical-trial-for-wet-amd