OKYO Pharma Announces Plans to Accelerate the Clinical Development of Urcosimod to Treat Neuropathic Corneal Pain
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.
OKYO Pharma annuncia l'intenzione di accelerare lo sviluppo di urcosimod per il trattamento del Dolore Corneale Neuropatico (NCP). L'azienda ha deciso di concludere anticipatamente la sperimentazione di Fase 2 dopo che 17 pazienti hanno completato lo studio presso il Tufts Medical Center di Boston.
Lo studio, iniziato nell'ottobre 2024, era progettato come uno studio randomizzato, in doppio cieco, controllato con placebo della durata di 12 settimane. La decisione di terminare in anticipo nasce dal desiderio di OKYO di analizzare i dati attuali ed espandere la sperimentazione a uno studio multicentrico. Diversi pazienti che hanno completato lo studio hanno richiesto l'uso compassionevole continuativo di urcosimod.
L'azienda prevede che l'analisi dei dati richiederà circa 6 settimane e ha in programma un incontro con la FDA per discutere i piani di sviluppo accelerato. Urcosimod ha già dimostrato un profilo di sicurezza favorevole e una tollerabilità simile al placebo in una precedente sperimentazione di Fase 2 su 240 pazienti. Attualmente, per il NCP non esiste una terapia approvata dalla FDA, rendendo questo sviluppo particolarmente importante per i pazienti che hanno avuto scarso successo con altri trattamenti.
OKYO Pharma anuncia planes para acelerar el desarrollo de urcosimod para el tratamiento del Dolor Corneal Neuropático (NCP). La compañía está cerrando anticipadamente su ensayo de Fase 2 tras la finalización del estudio por parte de 17 pacientes en el Tufts Medical Center de Boston.
El ensayo, iniciado en octubre de 2024, fue diseñado como un estudio aleatorizado, doble ciego y controlado con placebo de 12 semanas. La decisión de finalizar antes se debe al deseo de OKYO de analizar los datos actuales y ampliar el estudio a un ensayo multicéntrico. Varios pacientes que completaron el estudio han solicitado continuar con el uso compasivo de urcosimod.
La compañía espera que el análisis de datos tome aproximadamente 6 semanas y planea reunirse con la FDA para discutir planes de desarrollo acelerado. Urcosimod ya ha demostrado un perfil de seguridad favorable y una tolerabilidad similar al placebo en un ensayo previo de Fase 2 con 240 pacientes. Actualmente, no existe una terapia aprobada por la FDA para el NCP, lo que hace que este desarrollo sea significativo para los pacientes que han tenido poco éxito con otros tratamientos.
OKYO Pharma가 신경병성 각막통증(NCP) 치료를 위한 우르코시모드 개발 가속 계획을 발표했습니다. 보스턴의 터프츠 메디컬 센터에서 17명의 환자가 연구를 완료한 후 2상 임상을 조기 종료하기로 했습니다.
2024년 10월에 시작된 이 임상은 12주간 위약 대조, 이중 눈가림, 무작위 배정 방식으로 설계되었습니다. 조기 종료 결정은 현재 데이터를 분석하고 다기관 임상으로 확장하려는 OKYO의 의지에서 비롯되었습니다. 임상을 완료한 여러 환자들이 우르코시모드의 지속적인 동정적 사용을 요청했습니다.
회사는 데이터 분석에 약 6주가 소요될 것으로 예상하며, FDA와 만나 개발 가속화 계획을 논의할 예정입니다. 우르코시모드는 이전 240명 대상 2상 임상에서 안전성 및 위약과 유사한 내약성을 이미 입증했습니다. 현재 NCP에 대해 FDA 승인 치료제가 없어, 이 개발은 기존 치료에 제한적 효과를 본 환자들에게 중요한 의미를 갖습니다.
OKYO Pharma annonce son intention d'accélérer le développement de urcosimod pour le traitement de la Douleur Cornéenne Neuropathique (NCP). La société met fin prématurément à son essai de phase 2 après que 17 patients ont terminé l'étude au Tufts Medical Center de Boston.
L'essai, lancé en octobre 2024, était conçu comme une étude randomisée en double aveugle, contrôlée par placebo, d'une durée de 12 semaines. La décision d'interrompre prématurément découle du souhait d'OKYO d'analyser les données actuelles et d'élargir l'étude à un essai multicentrique. Plusieurs patients ayant terminé l'essai ont demandé la poursuite de l'utilisation compassionnelle d'urcosimod.
La société prévoit que l'analyse des données prendra environ 6 semaines et envisage une rencontre avec la FDA pour discuter des plans de développement accéléré. Urcosimod a déjà démontré une bonne sécurité et une tolérance similaire au placebo lors d'un précédent essai de phase 2 portant sur 240 patients. À ce jour, aucune thérapie n'est approuvée par la FDA pour le NCP, ce qui rend ce développement particulièrement important pour les patients ayant eu peu de succès avec d'autres traitements.
OKYO Pharma kündigt Pläne zur Beschleunigung der Entwicklung von urcosimod zur Behandlung von Neuropathischem Hornhautschmerz (NCP) an. Das Unternehmen beendet die Phase-2-Studie vorzeitig, nachdem 17 Patienten die Studie im Tufts Medical Center in Boston abgeschlossen haben.
Die im Oktober 2024 gestartete Studie war als randomisierte, doppelblinde, 12-wöchige Placebo-kontrollierte Studie konzipiert. Die Entscheidung zur vorzeitigen Beendigung resultiert aus dem Wunsch von OKYO, die aktuellen Daten zu analysieren und die Studie auf eine multizentrische Studie auszuweiten. Mehrere Patienten, die die Studie abgeschlossen haben, haben um eine fortgesetzte einfühlsame Anwendung von urcosimod gebeten.
Das Unternehmen erwartet, dass die Datenanalyse etwa 6 Wochen dauern wird, und plant ein Treffen mit der FDA, um die Pläne zur beschleunigten Entwicklung zu besprechen. Urcosimod hat bereits in einer vorherigen Phase-2-Studie mit 240 Patienten eine günstige Sicherheit und eine Placebo-ähnliche Verträglichkeit gezeigt. Für NCP gibt es derzeit keine von der FDA zugelassene Therapie, was diese Entwicklung für Patienten, die mit anderen Behandlungen nur begrenzte Erfolge hatten, besonders bedeutsam macht.
- 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
- 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
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