FDA Aligns With OKYO’s NEPTUNE Phase 3 Trial of Urcosimod in Neuropathic Corneal Pain

OKYO Pharma has received positive feedback from the U.S. Food and Drug Administration following a Type D meeting, providing regulatory alignment for the clinical development of urcosimod in neuropathic corneal pain (NCP). The agency supported the company’s proposed Phase 3 study design, allowing OKYO to move directly into a global pivotal trial that could potentially support a single-trial registration strategy if the study meets its objectives and receives subsequent FDA agreement.

The company also introduced NEPTUNE (Neuropathic Eye Pain Treatment with Urcosimod & Nerve Evaluation) as the name of its global Phase 3 trial, which will enroll patients across the United States and Europe. The study is expected to recruit approximately 111 participants, randomized in a 2:1 ratio to receive either 0.05% urcosimod or placebo as a single-dose treatment.

The FDA’s feedback provides greater regulatory clarity for OKYO’s development program and reduces uncertainty around the clinical pathway for urcosimod. In parallel, the company plans to seek Breakthrough Therapy Designation (BTD) to potentially accelerate clinical development and regulatory review, reflecting the significant unmet need in NCP.

Urcosimod targets inflammation and pain through a dual mechanism

Urcosimod is an investigational, first-in-class lipid-conjugated chemerin peptide agonist that activates the ChemR23 G protein-coupled receptor. The receptor is expressed on immune cells involved in ocular inflammation as well as neurons and glial cells within the dorsal root ganglion.

Through this dual mechanism, urcosimod has demonstrated both ocular analgesic and anti-inflammatory activity in preclinical and clinical development. The therapy is currently the first investigational drug with an active FDA Investigational New Drug (IND) application specifically for neuropathic corneal pain and has already received Fast Track designation from the agency.

NEPTUNE trial supports pivotal development strategy

The Phase 3 NEPTUNE trial represents the next stage of urcosimod’s clinical development. The global study will evaluate the efficacy and safety of a single 0.05% dose of urcosimod compared with placebo in patients with neuropathic corneal pain, a chronic condition characterized by severe ocular pain caused by dysfunction or injury of the corneal sensory nerves.

Although the company has not disclosed efficacy endpoints, the FDA agreed with the overall study framework during the Type D meeting. Based on those discussions, OKYO believes the trial could serve as the pivotal study supporting a future marketing application, provided the results are positive and the FDA continues to support the proposed regulatory pathway.

FDA feedback strengthens development confidence

Chief Medical Officer Flavio Mantelli, MD, PhD, said the FDA meeting validated the NEPTUNE trial design and reduced regulatory risk while accelerating the company’s clinical development strategy. He noted that the agency’s feedback strengthens confidence in generating clinically meaningful evidence for patients, physicians, and regulators.

Chief Executive Officer Robert J. Dempsey said the combination of a strong financial position and FDA regulatory alignment enables the company to advance directly into the global Phase 3 program. He added that urcosimod has the potential to become the first non-opioid therapy approved specifically for neuropathic corneal pain, a disease that currently has no FDA-approved treatment options.

Addressing a major unmet need in neuropathic corneal pain

Neuropathic corneal pain is a chronic and often debilitating ocular disorder characterized by severe eye pain, light sensitivity, and, in some patients, facial or head pain. Symptoms frequently appear disproportionate to observable clinical findings, making diagnosis and treatment challenging. With no approved therapies available, patients are commonly managed using off-label topical or systemic medications that often provide limited benefit.

If the NEPTUNE trial is successful, urcosimod could become the first approved therapy specifically developed for neuropathic corneal pain while establishing a potentially streamlined regulatory pathway toward registration.

Reference

OKYO Pharma Reports Positive Feedback from FDA Type D Meeting and Accelerates Urcosimod into Global Phase 3 Pivotal Trial for Neuropathic Corneal Pain – OKYO Pharma