Urcosimod Shows Positive Phase 2 Results for NCP

OKYO Pharma has announced promising top-line results from its recently concluded Phase 2 clinical trial evaluating urcosimod (formerly OK-101) for the treatment of neuropathic corneal pain (NCP). The data marks a significant advancement for a condition that currently lacks effective treatment options.

Urcosimod: A Targeted Approach to NCP

Urcosimod is described by OKYO Pharma as a lipid-conjugated chemerin peptide agonist that targets the ChemR23 G-protein coupled receptor. This receptor is found on immune cells involved in ocular inflammation and on neurons and glial cells within the dorsal root ganglion, making it a compelling therapeutic candidate for managing both inflammatory and neuropathic pain mechanisms.

Trial Overview and Design

The Phase 2 study was a randomized, double-masked, placebo-controlled, proof-of-concept trial conducted at Tufts Medical Center in Boston, Massachusetts. The trial was led by Dr. Pedram Hamrah, a leading expert in neuropathic corneal pain. Although the original plan was to enroll 48 patients, the trial was closed early in April 2025 after 17 patients completed the study. This early termination was driven by the company’s strong interest in unmasking the clinical data for an early read-out on potential drug efficacy.

Primary Endpoint and Pain Measurement

The trial’s primary endpoint was the change in mean pain scores from baseline (visit 1, day 0) to the end of treatment (visit 4, day 84), measured using a Visual Analogue Scale (VAS) ranging from 0 to 10.

Efficacy Results: Per-Protocol Population

In the per-protocol population, patients receiving 0.05% urcosimod reported a mean pain score of 5.5 compared to 2.75 in the placebo group after the 12-week treatment period. Notably, 75% of patients in the urcosimod group experienced greater than 80% improvement in pain severity according to VAS scores. A marked reduction in pain was observed as early as week 4. The company emphasized that the placebo group showed only half the mean improvement seen with urcosimod.

Results in the Intent-to-Treat Population

Among the intent-to-treat population, 67% of patients treated with 0.05% urcosimod achieved over 50% improvement in VAS scores. In comparison, only 33% of those in the placebo group reached the same threshold, highlighting urcosimod’s potential clinical benefit.

Expert Insights

Commenting on the results, Dr. Pedram Hamrah stated, “I have been dedicated to the pursuit of a drug to treat NCP for a considerable period of time, and these top-line results, along with the findings of our previous in-house animal model study of urcosimod to treat NCP, are very exciting and extremely encouraging. I find it particularly encouraging that the drug showed a dramatic reduction in VAS pain scores in patients who have particularly high VAS pain scores, indicating a more severe form of the disease.”

Future Development Plans and Drug Stability

OKYO Pharma has indicated that it will accelerate the clinical development of urcosimod with further trials expected in the near future. Additionally, the company recently reported positive findings regarding the drug’s long-term stability. Urcosimod has demonstrated over 2.5 years of stability in single-use ampoules used for its administration, supporting its viability as a long-term therapeutic option.