OKYO Pharma Ltd (NASDAQ:OKYO) plans to run a 100-patient, multi-centre study of its eye-drop candidate Urcosimod in neuropathic corneal pain, laying out a path it hopes can lead to US approval in a condition with no Food & Drug Administration-approved therapies.
The company said the next study will be a multiple-ascending-dose (MAD) trial, researcher-speak for testing several dose levels, run as a randomized, placebo-controlled, double-masked design across several U.S. sites.
The goals: pick an “optimal registration dose” for Phase 3 and learn more about the drug’s micellar (droplet-based) formulation.
OKYO said it expects topline results in 2026 and is moving toward an FDA meeting to confirm the primary endpoint for potential approval. Urcosimod holds Fast-Track designation, a status that can speed meetings and review timelines.
The plan follows July topline data from a small, single-centre Phase 2 proof-of-concept study at Tufts Medical Center in Boston.
After 12 weeks of treatment, “75% of per-protocol patients receiving 0.05% Urcosimod showed greater than 80% reduction in neuropathic corneal pain… as measured by visual analog scale,” OKYO said.
The Phase 2 trial was closed early and unmasked after 17 patients completed the study; participants had long-standing NCP and had tried multiple treatments with limited benefit.
Chief executive Gary Jacob said: “The results from our first-in-patient Phase 2 trial were exceptionally encouraging, with three-quarters of the per-protocol 0.05% dose patients experiencing major pain relief in the study.… 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.”
Neuropathic corneal pain is thought to stem from corneal nerve damage and inflammation and can trigger severe, chronic eye and facial pain.
With no approved drugs, physicians often rely on off-label therapies. Fast-Track status, granted to drugs targeting serious conditions with unmet need, can allow rolling FDA submissions and more frequent interactions with the agency, though it does not guarantee approval.
Urcosimod, formerly OK-101, is a lipid-conjugated peptide designed to activate the ChemR23 receptor found on immune and nerve cells in the eye. The lipid “anchor” is intended to help the drop stay on the eye’s surface longer, potentially improving efficacy.
Beyond NCP, Urcosimod previously hit multiple endpoints in a 240-patient Phase 2 study in dry eye disease.
OKYO said it will provide further updates as the development program advances.