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 company's lead asset demonstrated significant pain reduction in patients with this mysterious eye condition, paving the way for further development and potential FDA approval.

Trial Results and Clinical Significance

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. Using Cohen's d to compare treatment and placebo effects yielded a d-value of 1.2, indicating a strong treatment effect.

Dr. Gary Jacob, CEO of Okyo Pharma, expressed optimism about the results: "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."

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 reducing inflammation associated with NCP. Okyo Pharma licensed the compound from On Target (OTTx) Therapeutics in March 2017.

The molecule has shown promise beyond NCP, with a completed phase 2 trial in dry eye disease at the end of 2023. In May, urcosimod received FDA fast-track designation for NCP, highlighting the unmet medical need in this area.

Future Outlook and Patient Impact

With no FDA-approved treatments currently available for NCP, Okyo Pharma's urcosimod represents a potential breakthrough for patients suffering from this chronic condition. The company plans to conduct additional trials of urcosimod for NCP, and multiple patients from the phase 2 study have requested continued access to the molecule through the FDA's compassionate use program.

As Okyo Pharma prepares for discussions with the FDA regarding next steps, the pharmaceutical industry watches closely. If successful, urcosimod could become the first approved treatment for NCP, offering hope to patients living with the life-altering effects of this relentless neuropathic pain.