Key Takeaways

Okyo Pharma announced new positive findings from an exploratory analysis of patient-reported outcomes in its recently completed phase 2a trial of urcosimod (0.05%).

The new data, highlighting improvements in quality of life (QoL), will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2026 annual meeting. The presentation will detail results from the first-in-human study of urcosimod, including reductions in pain and improvements in patient-reported well-being.

Neuropathic corneal pain is a chronic and often debilitating condition with no FDA-approved treatments.

The randomized, double-masked, placebo-controlled phase 2a study evaluated 12 patients, with six receiving urcosimod and six receiving placebo over a 12-week period. Patients treated with urcosimod demonstrated greater improvements across several QoL measures compared with placebo, based on a 0–10 scale.

Key findings included a mean reduction of −4.5 in scores related to enjoyment of life and relationships among urcosimod-treated patients, compared to no change in the placebo group. Mood scores improved by −1.5 in the treatment group versus −0.5 for placebo, while time spent thinking about eye pain decreased by −3.0 compared to −1.5 with placebo.

These QoL improvements build on previously reported primary efficacy results showing meaningful reductions in pain as measured by the Visual Analogue Scale (VAS). Researchers also observed signals suggesting potential restoration of corneal nerve structure, pointing to a possible disease-modifying effect.

“We are pleased to see these patient-reported quality-of-life benefits emerging alongside the primary pain reduction signals from urcosimod,” said Flavio Mantelli, MD, PhD, Chief Medical Officer at Okyo Pharma. “Like pain, QoL is a clinically meaningful endpoint, and this exploratory analysis reinforces the holistic therapeutic profile of urcosimod.”

Raj Patil, PhD, Chief Scientific Officer at Okyo Pharma, added that the consistent improvements across emotional and functional measures further support the therapy’s potential impact. 

Okyo Pharma plans to advance urcosimod into larger confirmatory studies, including a multicenter phase 2b/3 trial, to validate these findings and further evaluate its therapeutic potential.