OKYO Advances Urcosimod into Phase 2b/3 Trial After FDA Alignment Meeting

OKYO Pharma Limited shares climbed in pre-market trading following a significant development with the U.S. Food and Drug Administration. The company secured FDA backing for its clinical development strategy for Urcosimod, a novel treatment being developed for neuropathic corneal pain (NCP), a severe ocular condition currently lacking any approved therapies.

FDA Type C Meeting Validates Phase 2b/3 Clinical Strategy

During a recent Type C consultation, OKYO achieved critical alignment with the FDA on its proposed trial design. The regulatory agency confirmed that the Visual Analogue Scale (VAS) pain score reduction at Week 12 represents a clinically meaningful primary endpoint. The FDA also determined that a two-point or greater improvement on the VAS scale constitutes a meaningful therapeutic response.

The regulatory review validated the Phase 2b/3 study design specifications, including the planned sample size and statistical powering assumptions. Additionally, the FDA supported the use of the Ocular Pain Assessment Survey (OPAS) as a secondary efficacy measure. The agency raised no material concerns regarding the company’s Chemistry, Manufacturing and Controls (CMC) strategy—a positive signal for the development timeline.

Notably, the FDA indicated that robust Phase 2b/3 results, combined with a finalized statistical analysis plan disclosed before trial unmasking, could potentially support approval-level evidence of effectiveness at a subsequent End-of-Phase meeting.

Understanding Urcosimod’s Mechanism and Development

Urcosimod, previously designated OK-101, is a lipid-conjugated chemerin peptide agonist designed to modulate the ChemR23 receptor. This mechanism targets both inflammatory and neuronal pathways implicated in corneal pain generation. The company recently completed a Phase 2 trial demonstrating statistically significant reductions in patient pain scores, providing proof-of-concept for the therapeutic approach.

The upcoming Phase 2b/3 trial represents a critical inflection point, combining efficacy confirmation with Phase 3 pivotal elements into a single streamlined protocol. This integrated design aims to expedite the development pathway while maintaining robust clinical evidence standards.

Neuropathic Corneal Pain: An Unmet Clinical Need

Neuropathic corneal pain represents a chronic, disabling ocular condition stemming from corneal nerve dysfunction. Affected patients experience characteristic symptoms including burning sensations, stabbing pain, or electric shock-like discomfort. Current treatment options rely on off-label medications with limited effectiveness, leaving significant unmet medical needs.

With no FDA-approved therapies specifically indicated for NCP, Urcosimod could potentially establish a first-in-class treatment category. OKYO plans to launch the 120-patient Phase 2b/3 multiple-dose trial in the first half of 2026, positioning the program for near-term data readouts.

Market Perspective and Stock Movement

OKYO has historically traded within a range of $0.90 to $3.34 over the preceding twelve-month period. The regulatory breakthrough reflected positively in trading activity, with shares gaining in early trading sessions following the FDA announcement. The company’s validated development pathway strengthens its position in pursuing approval for this underserved patient population.