OKYO Pharma Announces Registration Pathway with 100 Patient Multi-Center Clinical Trial of Urcosimod in Neuropathic Corneal Pain
September 22, 2025 – Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical – OKYO Pharma, clinical trials, neuropathic corneal pain, ophthalmology
22 September 2025 — London, UK and New York, US — OKYO Pharma Limited, an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod to treat neuropathic corneal pain (NCP), an ocular condition associated with chronic and often severe nerve-related pain but without an FDA-approved therapy, today announced plans for the next stage of clinical development for its lead drug candidate, urcosimod, to treat neuropathic corneal pain (NCP).
The announcement follows the release in July of topline data from OKYO’s randomized, double-masked, placebo-controlled Phase 2 proof-of-concept trial of urcosimod in 17 NCP patients conducted at Tufts Medical Center in Boston, MA, with Pedram Hamrah, M.D., a world-leading expert in NCP, as Principle Investigator.
After 12 weeks of treatment, 75% of per-protocol patients receiving 0.05% urcosimod showed greater than 80% reduction in neuropathic corneal pain in that study, as measured by visual analog scale, suggesting highly effective treatment in this patient population.
Building on these promising results, OKYO is now focused on advancing to a multiple-ascending-dose (MAD) clinical trial designed to:
- Enroll approximately 100 NCP patients across several U.S. clinical sites in this randomized, placebo-controlled, double-masked trial
- Identify the optimal registration dose for urcosimod for future Phase 3 trials
- Develop further understanding of urcosimod’s unique micellar drug characteristics.
Negotiations with several leading U.S. clinical sites are underway, and OKYO anticipates topline data from this trial in 2026.
As part of the registration pathway, OKYO continues to progress towards a meeting with the U.S. Food and Drug Administration (FDA) to discuss requirements for an approvable drug to treat NCP and to define the primary endpoint required for potential registration. Urcosimod currently holds Fast-Track designation, which is expected to enable quick turn-around on having the meeting with the FDA.
“The results from our first-in-patient Phase 2 trial were exceptionally encouraging, with three-quarters of the per-protocol 0.05% dose patients experiencing major pain relief in the study. These results suggest an important step forward for NCP patients, most of whom are searching for an effective treatment for their NCP condition,” commented Gary S. Jacob, Ph.D., CEO of OKYO Pharma. “Our next MAD clinical trial is designed to give us the data needed to define an optimal registration pathway for urcosimod, and we are moving rapidly to initiate this study. With Fast-Track designation in place and a constructive engagement anticipated with FDA, we are well-positioned to bring this novel therapy closer to patients in need.”
OKYO remains committed to addressing this major unmet medical need and will provide updates as its development program progresses.
About OKYO
OKYO Pharma Limited is a clinical stage biopharmaceutical company developing innovative therapies for the treatment of neuropathic corneal pain and dry eye disease, with ordinary shares listed for trading on the NASDAQ Capital Market. OKYO is focused on the discovery and development of novel molecules to treat neuropathic corneal pain and dry eye disease. A Phase 2 trial of urcosimod to treat neuropathic corneal pain patients was just completed by OKYO. For further information, please visit www.okyopharma.com.
