OKYO Pharma details next clinical stage of urcosimod development

OKYO Pharma has announced its plans for the next stage of clinical development of urcosimod (formerly OK-101), its lead drug candidate to treat neuropathic corneal pain (NCP).

NCP is a condition that causes severe pain and sensitivity of the eyes, face, or head. The exact cause of neuropathic corneal pain is unknown but is thought to result from nerve damage to the cornea combined with inflammation.¹

The company describes urcosimod as a lipid-conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor, which is typically found on immune cells of the eye responsible for the inflammatory response, as well as on neurons and glial cells in the dorsal root ganglion.

In July, OKYO released positive top-line data from the recently closed phase 2 trial of urcosimod and announced it had received $1.9 million in non-dilutive funding to support its ongoing research and development programs.^2,3

In the trial, 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.

The company states it is now focusing on advancing to a multiple-ascending-dose (MAD) clinical trial following the results of the phase 2 trial.

The MAD trial aims to enroll 100 NCP patients across several US sites and will be a randomized, placebo-controlled, double-masked trial.

Additionally, the company noted it plans to identify the optimal registration dose for urcosimod for future phase 3 trials and develop a further understanding of urcosimod’s unique micellar drug characteristics.

Gary S. Jacob, PhD, CEO of OKYO Pharma, commented on the news in a press release from the company, saying, “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 the FDA, we are well-positioned to bring this novel therapy closer to patients in need.”

The company anticipates topline data to be available in 2026 and is in negotiations with several leading US clinical sites for the trial.

References:

1. OKYO Pharma Announces Registration Pathway with 100 Patient Multi-Center Clinical Trial of Urcosimod in Neuropathic Corneal Pain. Published September 22, 2025. Accessed September 22, 2025. https://okyopharma.com/okyo-pharma-announces-registration-pathway-with-100-patient-multi-center-clinical-trial-of-urcosimod-in-neuropathic-corneal-pain

2. Harp MD. OKYO Pharma announces positive results from phase 2 trial of urcosimod for neuropathic corneal pain. Published July 18, 2025. Accessed September 22, 2025. https://www.ophthalmologytimes.com/view/okyo-pharma-announces-positive-results-from-phase-2-trial-of-urcosimod-for-neuropathic-corneal-pain

3. Harp MD. OKYO Pharma receives $1.9 million in non-dilutive funding. Published July 21, 2025. Accessed September 22, 2025. https://www.ophthalmologytimes.com/view/okyo-pharma-receives-1-9-million-in-non-dilutive-funding