OKYO Pharma Announces Plans to Accelerate the Clinical Development of Urcosimod to Treat Neuropathic Corneal Pain
Rhea-AI Summary
OKYO Pharma announces plans to accelerate the development of urcosimod for treating Neuropathic Corneal Pain (NCP). The company is closing its Phase 2 trial early after 17 patients completed the study at Tufts Medical Center in Boston.
The trial, initiated in October 2024, was designed as a double-masked, randomized, 12-week placebo-controlled study. The decision to end early stems from OKYO's desire to analyze the current data and expand to a multicenter trial. Several patients who completed the trial have requested continued compassionate use of urcosimod.
The company expects data analysis to take approximately 6 weeks and plans to meet with the FDA to discuss accelerated development plans. Urcosimod has already shown favorable safety and placebo-like tolerability in a previous 240-patient Phase 2 trial. NCP currently has no FDA-approved therapy, making this development significant for patients who have had limited success with other treatments.
Positive
- Early trial closure enables faster FDA meeting and potential accelerated development pathway
- 17 patients completed Phase 2 trial with data analysis expected within 6 weeks
- Previous Phase 2 trial with 240 patients showed favorable safety profile
- Strong patient interest for future registrational trial indicates market demand
- Patients requesting compassionate use suggests potential treatment effectiveness
Negative
- Early trial closure before full patient enrollment could indicate data limitations
- Single trial site limitation may affect data robustness
- No current FDA-approved therapy in market suggests challenging regulatory pathway
- Additional multicenter trials will require significant time and resources
Insights
OKYO's early Phase 2 trial closure with just 17 patients creates uncertainty despite positioning as strategic acceleration.
OKYO Pharma's decision to prematurely conclude its Phase 2 trial for urcosimod in Neuropathic Corneal Pain warrants careful analysis. The company has only 17 patients who completed the study, significantly fewer than typical Phase 2 trials. This early termination is framed as a strategic acceleration to unblind data and plan for a multi-center trial, rather than completing enrollment as originally designed.
The company's stated rationale—accelerating development by analyzing current data to request an FDA meeting—represents an unconventional development approach. While potentially saving time, this strategy introduces statistical limitations due to the small sample size. The anticipated 6-week timeline for data analysis appears feasible for this limited dataset.
Two aspects merit attention: First, the mention that patients have requested compassionate use continuation could suggest patient-perceived benefit, though this cannot be confirmed without unblinded efficacy data. Second, urcosimod has previously demonstrated favorable safety in a 240-patient trial, mitigating some risk concerns.
This development represents a pivotal moment for OKYO's urcosimod program, with the upcoming data analysis determining whether the company proceeds toward registrational studies or requires additional proof-of-concept work.
OKYO's strategic pivot on urcosimod trial reflects urgency in addressing NCP's unmet need despite limited patient data.
OKYO Pharma's early conclusion of its urcosimod Phase 2 trial addresses a significant ophthalmological challenge. Neuropathic Corneal Pain (NCP) represents an important unmet medical need characterized by chronic, severe ocular pain with no FDA-approved therapies currently available.
The trial specifically targeted a difficult-to-treat population—patients with long-term chronic NCP who previously failed multiple therapies. This patient selection strategy appropriately focuses on those with the greatest need but also represents a high efficacy hurdle for any investigational treatment.
The transition from a single-site study at Tufts Medical Center to a planned multi-center approach is clinically sound, potentially improving geographical access and patient diversity. However, the limited cohort of 17 completed patients raises questions about statistical power for detecting meaningful efficacy signals.
Patient requests for compassionate use continuation suggest potential clinical benefit worthy of further investigation, though efficacy outcomes remain masked pending analysis. The reference to favorable safety from a previous larger trial provides reassurance regarding tolerability profile.
This strategic pivot reflects the challenging balance between thorough investigation and accelerating potential treatments for conditions with significant patient burden and limited therapeutic options.
- Urcosimod phase 2 trial treating Neuropathic Corneal Pain (“NCP”) patients was initiated in October 2024 and designed as a double-masked, randomized, 12-week placebo-controlled trial.
- OKYO Pharma plans to analyze the efficacy data from the 17 patients who have now completed the Phase 2 trial.
- OKYO Pharma plans for a meeting with FDA following evaluation of clinical data.
LONDON and NEW YORK, April 30, 2025 (GLOBE NEWSWIRE) -- OKYO Pharma Limited (NASDAQ: OKYO), an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod (formerly called OK-101) to treat NCP, an ocular condition associated with chronic and often severe pain but without an FDA-approved therapy, announces plans to accelerate the clinical development of urcosimod to treat NCP through the analysis of its data following the early closure of its Phase 2 trial.
The Phase 2 trial of urcosimod to treat NCP was being conducted at a single trial site at Tufts Medical Center in Boston, MA. The decision to close the trial now, with 17 patients having presently completed the study, is due to OKYO’s strong desire to access the currently masked data and use it to plan its expanded development program. The patients participating in the trial have all been diagnosed with long term chronic neuropathic corneal pain and had previously been treated with multiple therapies with very limited or no response. Significant interest has been seen in the trial from long-term sufferers of NCP, and OKYO believes it should expand the development program and move forward with a multicenter trial. A considerable number of potential patients have expressed an interest in participating in a future registrational trial.
A number of patients who have now completed the trial have requested continued compassionate use of urcosimod which the Company is seeking to arrange with Tufts Medical Center, subject to the necessary FDA consents.
“By ending this trial now, we believe we can save time in advancing the program to a meeting with FDA to discuss further development of urcosimod,” commented Gary S. Jacob, Ph.D., Chief Executive Officer of OKYO Pharma. “We anticipate that the analysis of the data from these patients with the closing of this trial should take no longer than 6 weeks. Urcosimod has already demonstrated favorable safety and placebo-like tolerability in our previous 240 patient Phase 2 trial. The decision to close the trial now significantly cuts the time to our requesting an end-of-phase 2 meeting with FDA to explore accelerated plans for the drug’s further clinical development. I want to also thank our patients and study site team for their participation and commitment to this important trial of urcosimod to treat NCP.”
About NCP
Neuropathic corneal pain 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. Neuropathic corneal pain, which can exhibit as a severe, chronic, debilitating condition in patients suffering from a host of ophthalmic conditions, is presently treated by various topical and systemic treatments in an off-label fashion. Notably, there is no FDA approved drug to treat this debilitating condition.
About the Urcosimod Phase 2 Trial in NCP Patients
The Phase 2 study was designed as a double-masked, randomized, 12-week placebo-controlled trial in NCP patients. A total of 48 patients were planned to enroll for the study, with NCP disease confirmed via confocal microscopy.
About Urcosimod (Formerly called OK-101)
Urcosimod is 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. Urcosimod was developed using a membrane-anchored-peptide technology to produce a novel long-acting drug candidate for treating dry eye disease. Urcosimod has been shown to produce anti-inflammatory and pain-reducing activities in mouse models of dry eye disease and corneal neuropathic pain, respectively, and is designed to combat washout through the inclusion of the lipid anchor built into the drug molecule to potentially enhance the residence time of urcosimod within the ocular environment. Urcosimod showed clear statistical significance in multiple endpoints in a recently completed Phase 2, multi-center, double-masked, placebo-controlled trial to treat dry eye disease, and was also being evaluated in a randomized, placebo-controlled, double-masked Phase 2 trial to treat neuropathic corneal pain patients.
About OKYO
OKYO Pharma Limited (NASDAQ: OKYO) is a clinical stage biopharmaceutical company developing innovative therapies for the treatment of neuropathic corneal pain and dry eye disease, with ordinary shares listed for trading on the NASDAQ Capital Market. OKYO is focused on the discovery and development of novel molecules to treat neuropathic corneal pain and dry eye disease. In addition to the completed Phase 2 trial of urcosimod to treat dry eye disease patients, OKYO is also currently evaluating urcosimod to treat neuropathic corneal pain patients.
For further information, please visit www.okyopharma.com.
Enquiries:
| OKYO Pharma Limited | Gary S. Jacob, Chief Executive Officer | 917-497-7560 |
| Business Development & Investor Relations | Paul Spencer | +44 (0)20 7495 2379 |
FAQ
What are the results of OKYO's Phase 2 trial for urcosimod in treating neuropathic corneal pain?
Why did OKYO (NASDAQ: OKYO) end its Phase 2 urcosimod trial early in April 2025?
How many patients completed OKYO's Phase 2 trial for neuropathic corneal pain treatment?
What is the next step for OKYO's urcosimod development after the Phase 2 trial?
Has urcosimod shown any safety concerns in OKYO's clinical trials?
