Okyo Pharma's Urcosimod Shows Promise in Phase 2 Trial for Neuropathic Corneal Pain

Okyo Pharma has announced positive topline results from its phase 2 trial of urcosimod, a potential first-in-class treatment for neuropathic corneal pain (NCP). The London-based pharmaceutical company reported a significant reduction in pain levels among patients treated with the drug, marking a crucial step towards addressing an unmet medical need.

Trial Results Demonstrate Efficacy

The placebo-controlled, double-masked study, conducted at Tufts Medical Center in Boston, enrolled 18 patients with NCP. Participants received four eye drops daily containing either 0.1% or 0.05% urcosimod for 12 weeks. The 0.05% urcosimod group showed a statistically significant 5.5-point reduction in pain on a 10-point scale, meeting the trial's primary endpoint.

Notably, the 0.1% concentration group demonstrated less efficacy than the 0.05% group, a finding consistent with earlier phase 2 results in dry eye disease. The placebo group also experienced a statistically significant pain reduction of 2.75 points from baseline by week 12.

Dr. Gary Jacob, CEO of Okyo Pharma, expressed optimism about the results, stating, "The results strengthen our conviction that this drug may be particularly effective at showing a significant reduction in pain in patients with a greater degree of neuropathic pain."

Safety Profile and Patient Feedback

No serious adverse events were reported during the trial, suggesting a favorable safety profile for urcosimod. The company also noted positive feedback from patients, with multiple participants requesting continued access to the drug through the FDA's compassionate use program.

Mechanism of Action and Development History

Urcosimod, also known as OK-101, is a protein designed to bind to a G protein-coupled receptor on immune cells in the eye, potentially mitigating inflammation associated with NCP. The compound, Okyo's sole asset, received FDA fast-track designation for NCP in May.

Originally licensed from On Target (OTTx) Therapeutics in March 2017, urcosimod has also been tested in dry eye disease, with a phase 2 trial completed at the end of 2023.

As Okyo Pharma sets its sights on potential FDA approval, these promising results bring hope to patients suffering from the mysterious and debilitating condition of neuropathic corneal pain, for which no approved treatments currently exist.