FDA grants single-patient expanded access for urcosimod in neuropathic corneal pain

The US Food and Drug Administration has authorized a single-patient expanded access (compassionate use) Investigational New Drug (IND) application for the use of urcosimod (0.05%) in a patient with neuropathic corneal pain (NCP). The application was submitted by Pedram Hamrah, MD, vice chair of academic medicine, Department of Ophthalmology, University of South Florida.

Hamrah commented on the FDA decision in a press release from OKYO, saying, “We are grateful that this FDA authorization allows us to explore the potential of urcosimod in a patient with severe [NCP] through a physician-sponsored expanded access IND. [NCP] is a complex condition involving both immune-mediated inflammation and dysfunctional nerve signaling. Urcosimod’s proposed dual mechanism of action, potentially targeting both pathways provides a strong scientific rationale for investigation in this setting.”

NCP is a condition that causes severe pain and sensitivity of the eyes, face, or head. The exact cause of neuropathic corneal pain is unknown but is thought to result from nerve damage to the cornea combined with inflammation. There are currently no FDA-approved therapies specifically for NCP.

OKYO describes urcosimod (formerly OK-101) as a lipid-conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor, which is typically found on immune cells of the eye responsible for the inflammatory response, as well as on neurons and glial cells in the dorsal root ganglion.

The company released further data from its placebo-controlled, randomized, double-masked, phase 2 clinical trial of urcosimod in NCP at the end of 2025.² According to the company, results show patients treated with urcosimod 0.05% demonstrated a positive reduction in NCP, but also favorable changes in corneal nerve structure which were not observed in the placebo group.

Patients treated with urcosimod 0.05% showed median increases in total nerve fiber count (+2.0, n/0.16 mm², Inter Quartile Range [IQR] 0.54 to 3.63) and total nerve fiber length (+2.6 mm/mm², IQR 1.55 to 5.67; p = 0.057 vs placebo). While the placebo group exhibited median decreases in total nerve fiber count (–1.92, n/0.16 mm², IQR –2.79 to –0.04) and total nerve fiber length (–1.63 mm/mm², IQR –3.76 to 0.63).

Raj Patil, PhD, chief scientific officer of OKYO Pharma, commented on those findings, saying, “While exploratory, these findings reinforce our long-standing belief that targeting the chemerin receptor pathway may open a new therapeutic avenue for patients with neuropathic corneal pain. We are encouraged by the biological activity observed and believe they provide an important foundation for the continued development of urcosimod.”

In July 2025, OKYO also released positive top-line data from the recently closed phase 2 trial of urcosimod showing that after 12 weeks of treatment, 75% of per-protocol patients receiving 0.05% urcosimod showed greater than 80% reduction in NCP. Additionally, the company announced it had received $1.9 million in non-dilutive funding to support its ongoing research and development programs.^3,4

The company states it is focusing on advancing to a multiple-ascending-dose (MAD) clinical trial following the results of the phase 2 trial, which aims to enroll 100 NCP patients across several US sites and will be a randomized, placebo-controlled, double-masked trial.⁵

Gary S. Jacob, PhD, CEO of OKYO Pharma, commented, saying, “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. With Fast-Track designation in place and a constructive engagement anticipated with the FDA, we are well-positioned to bring this novel therapy closer to patients in need.”

The company anticipates topline data to be available in 2026.⁵

References:

1. FDA Approves Compassionate Use of Urcosimod (0.05%) for the Treatment of Neuropathic Corneal Pain Published January 23, 2026. Accessed January 23, 2026. https://www.globenewswire.com/news-release/2026/01/23/3224608/0/en/FDA-Approves-Compassionate-Use-of-Urcosimod-0-05-for-the-Treatment-of-Neuropathic-Corneal-Pain.html

2. OKYO Pharma Announces New Data Showing Favorable Corneal Nerve Outcomes in Phase 2 Study for Neuropathic Corneal Pain. Published December 11, 2025. Accessed January 23, 2026. https://okyopharma.com/okyo-pharma-announces-new-data-showing-favorable-corneal-nerve-outcomes-in-phase-2-study-for-neuropathic-corneal-pain/

3. Harp MD. OKYO Pharma announces positive results from phase 2 trial of urcosimod for neuropathic corneal pain. Published July 18, 2025. Accessed January 23, 2026 https://www.ophthalmologytimes.com/view/okyo-pharma-announces-positive-results-from-phase-2-trial-of-urcosimod-for-neuropathic-corneal-pain

4. Harp MD. OKYO Pharma receives $1.9 million in non-dilutive funding. Published July 21, 2025. Accessed January 23, 2026. https://www.ophthalmologytimes.com/view/okyo-pharma-receives-1-9-million-in-non-dilutive-funding

5. Harp MD. OKYO Pharma details next clinical stage of urcosimod development. Published September 22, 2025. Accessed January 23, 2026. https://www.ophthalmologytimes.com/view/okyo-pharma-details-next-clinical-stage-of-urcosimod-development