OKYO Pharma Unveils Strong Phase 2 Clinical Trial Results for Urcosimod to Treat Neuropathic Corneal Pain
OKYO Pharma (NASDAQ: OKYO) has announced positive Phase 2 clinical trial results for urcosimod in treating neuropathic corneal pain (NCP). The trial demonstrated that 75% of per-protocol patients receiving 0.05% urcosimod achieved over 80% reduction in pain after 12 weeks of treatment.
Key findings include a statistically significant reduction in mean pain scores (p-value = 0.025) for the 0.05% urcosimod group, with efficacy observed as early as Week 4. The drug showed particularly strong results in patients with moderate to severe NCP pain scores. Importantly, no serious adverse events were reported among the 18 trial participants.
The company plans to meet with the FDA to discuss next steps for urcosimod, which has already received Fast Track designation. OKYO is also planning to arrange an Expanded Access program for the drug, pending FDA approval.
OKYO Pharma (NASDAQ: OKYO) ha annunciato risultati positivi dalla sperimentazione clinica di Fase 2 sull'urcosimod nel trattamento del dolore corneale neuropatico (NCP). Lo studio ha dimostrato che il 75% dei pazienti per protocollo trattati con urcosimod allo 0,05% ha ottenuto una riduzione del dolore superiore all'80% dopo 12 settimane di trattamento.
I risultati principali includono una riduzione statisticamente significativa dei punteggi medi di dolore (valore p = 0,025) nel gruppo trattato con urcosimod allo 0,05%, con efficacia osservata già dalla quarta settimana. Il farmaco ha mostrato risultati particolarmente efficaci nei pazienti con punteggi di dolore da moderato a grave. È importante sottolineare che non sono stati segnalati eventi avversi gravi tra i 18 partecipanti allo studio.
L'azienda prevede di incontrare la FDA per discutere i prossimi passi per l'urcosimod, che ha già ottenuto la designazione Fast Track. OKYO sta inoltre pianificando di organizzare un programma di Accesso Espanso per il farmaco, in attesa dell'approvazione della FDA.
OKYO Pharma (NASDAQ: OKYO) ha anunciado resultados positivos en el ensayo clínico de Fase 2 para urcosimod en el tratamiento del dolor corneal neuropático (NCP). El estudio demostró que el 75% de los pacientes por protocolo que recibieron urcosimod al 0,05% lograron una reducción del dolor superior al 80% después de 12 semanas de tratamiento.
Los hallazgos clave incluyen una reducción estadísticamente significativa en las puntuaciones medias de dolor (valor p = 0,025) para el grupo de urcosimod al 0,05%, con eficacia observada desde la semana 4. El medicamento mostró resultados especialmente sólidos en pacientes con puntuaciones de dolor NCP de moderadas a graves. Es importante destacar que no se reportaron eventos adversos graves entre los 18 participantes del ensayo.
La compañía planea reunirse con la FDA para discutir los próximos pasos para urcosimod, que ya ha recibido la designación Fast Track. OKYO también está planificando organizar un programa de Acceso Expandido para el medicamento, pendiente de la aprobación de la FDA.
OKYO Pharma (NASDAQ: OKYO)는 신경병성 각막 통증(NCP) 치료를 위한 우르코시모드의 2상 임상시험에서 긍정적인 결과를 발표했습니다. 임상시험에서 프로토콜 준수 환자의 75%가 0.05% 우르코시모드를 투여받아 12주 치료 후 통증이 80% 이상 감소한 것으로 나타났습니다.
주요 결과로는 0.05% 우르코시모드 그룹에서 평균 통증 점수가 통계적으로 유의미하게 감소(p값 = 0.025)했으며, 4주차부터 효과가 관찰되었습니다. 특히 중등도에서 중증 NCP 통증 점수를 가진 환자들에게서 우수한 효과가 나타났습니다. 중요하게도, 18명의 시험 참가자 중 심각한 부작용은 보고되지 않았습니다.
회사는 이미 Fast Track 지정을 받은 우르코시모드의 다음 단계 논의를 위해 FDA와의 미팅을 계획하고 있습니다. 또한 FDA 승인 대기 중인 우르코시모드의 확대 접근 프로그램도 준비 중입니다.
OKYO Pharma (NASDAQ : OKYO) a annoncé des résultats positifs de son essai clinique de phase 2 concernant l'urcosimod dans le traitement de la douleur cornéenne neuropathique (NCP). L'étude a montré que 75 % des patients selon le protocole ayant reçu de l'urcosimod à 0,05 % ont obtenu une réduction de la douleur de plus de 80 % après 12 semaines de traitement.
Les résultats clés incluent une réduction statistiquement significative des scores moyens de douleur (valeur p = 0,025) dans le groupe urcosimod 0,05 %, avec une efficacité observée dès la semaine 4. Le médicament a montré des résultats particulièrement forts chez les patients présentant des scores de douleur NCP modérés à sévères. Il est important de noter qu’aucun effet indésirable grave n’a été rapporté parmi les 18 participants à l’essai.
La société prévoit de rencontrer la FDA pour discuter des prochaines étapes concernant l’urcosimod, qui a déjà obtenu la désignation Fast Track. OKYO prévoit également de mettre en place un programme d'accès élargi pour le médicament, sous réserve de l'approbation de la FDA.
OKYO Pharma (NASDAQ: OKYO) hat positive Ergebnisse der Phase-2-Studie zu Urcosimod bei der Behandlung von neuropathischen Hornhautschmerzen (NCP) bekannt gegeben. Die Studie zeigte, dass 75 % der per Protokoll behandelten Patienten mit 0,05 % Urcosimod nach 12 Wochen eine Schmerzlinderung von über 80 % erreichten.
Wichtige Erkenntnisse umfassen eine statistisch signifikante Reduktion der durchschnittlichen Schmerzwerte (p-Wert = 0,025) in der 0,05 %-Urcosimod-Gruppe, wobei die Wirksamkeit bereits ab Woche 4 beobachtet wurde. Das Medikament zeigte besonders starke Ergebnisse bei Patienten mit mäßigen bis starken NCP-Schmerzpunkten. Wichtig ist, dass keine schwerwiegenden Nebenwirkungen unter den 18 Studienteilnehmern gemeldet wurden.
Das Unternehmen plant, sich mit der FDA zu treffen, um die nächsten Schritte für Urcosimod zu besprechen, das bereits die Fast Track-Zulassung erhalten hat. OKYO plant außerdem, ein Expanded Access-Programm für das Medikament einzurichten, vorbehaltlich der FDA-Zulassung.
- 75% of patients receiving 0.05% urcosimod achieved >80% pain reduction
- Statistically significant reduction in pain scores (p-value = 0.025)
- Strong treatment effect demonstrated (Cohen-d value >1.2)
- No serious adverse events reported
- Fast Track designation already received from FDA
- Drug showed efficacy as early as Week 4 of treatment
- Higher concentration (0.1%) showed less efficacy than 0.05% dose
- Placebo group also showed statistically significant improvement (p-value = 0.035)
Insights
OKYO's urcosimod showed impressive 80% pain reduction in 75% of NCP patients with strong safety profile, addressing critical unmet need.
The Phase 2 results for urcosimod represent a significant breakthrough in neuropathic corneal pain (NCP) treatment. The data demonstrates 75% of per-protocol patients receiving 0.05% urcosimod achieved >80% reduction in pain after 12 weeks, measured by Visual Analogue Scale. This is particularly remarkable considering these patients had moderate to severe NCP despite prior maximum medical therapy - indicating efficacy in difficult-to-treat cases.
The trial revealed a statistically significant reduction in mean pain scores (p-value = 0.025) in the per-protocol group, with a substantial 5.5-point reduction compared to 2.75 in placebo. The Cohen-d value >1.2 confirms a strong treatment effect. What's clinically important is the rapid onset of action, with marked pain reduction by Week 4, suggesting patients could experience relief relatively quickly.
From a safety perspective, no serious adverse events were reported among the 18 participants, which is crucial for a chronic condition requiring long-term management. The FDA Fast Track designation already granted for urcosimod underscores the recognition of NCP as a serious condition with significant unmet need.
As the first company conducting a clinical study specifically for NCP, OKYO is addressing a major gap in ophthalmology. NCP causes severe nerve-related pain with currently no FDA-approved therapies. The trial's positive outcomes could position urcosimod as a first-in-class treatment, potentially transforming care for these patients who currently have limited relief options.
OKYO's positive Phase 2 data supports potential first-to-market advantage in untapped NCP market with accelerated regulatory pathway opportunities.
OKYO Pharma's Phase 2 results represent a significant milestone in their clinical development program. The strong efficacy signal in this trial provides critical validation for urcosimod as the company positions itself in the untapped neuropathic corneal pain market. The 75% response rate with >80% pain reduction in per-protocol patients at the 0.05% concentration demonstrates a compelling therapeutic profile.
From a regulatory perspective, OKYO holds several advantages. The drug has already secured Fast Track designation, which can expedite development and review timelines. Additionally, as the first company conducting clinical trials specifically for NCP, OKYO is establishing itself as a pioneer in this therapeutic area with potential first-mover advantages.
The company's strategic focus on the 0.05% concentration rather than 0.1% shows data-driven decision-making, as they've consistently observed superior efficacy at this lower concentration across multiple conditions. Their plans to pursue expanded access (compassionate use) while continuing development demonstrates both commercial awareness and patient-centric approach.
The market opportunity is significant - NCP represents an unmet medical need with no FDA-approved therapies. Patient demand is evidenced by the "significant interest" mentioned and requests for expanded access. With a differentiated product, demonstrated efficacy, clean safety profile, and regulatory advantages, OKYO is well-positioned to potentially advance urcosimod toward becoming the first approved therapy for this debilitating condition.
- After 12 weeks of treatment,
75% of per-protocol patients receiving0.05% urcosimod showed greater than80% reduction in neuropathic corneal pain (NCP), as measured by Visual Analogue Scale (VAS), demonstrating highly effective treatment. - Urcosimod (
0.05% ) demonstrated a marked reduction in pain scores as early as Week 4, with sustained efficacy maintained throughout the trial. - A statistically significant reduction in mean pain scores was observed from Visit 1 to the end of treatment Visit 4 (p-value = 0.025) in the per-protocol
0.05% urcosimod group, indicating the drug’s effectiveness over the study period. - Notably, all these responders entered the study with moderate to severe NCP pain scores despite prior use of maximum medical therapy.
- No serious adverse events were reported among the 18 patients throughout the trial.
- Following completion of full data analysis, OKYO plans a meeting with FDA to discuss next steps for urcosimod which has already received Fast Track designation for treating NCP.
LONDON and NEW YORK, July 16, 2025 (GLOBE NEWSWIRE) -- OKYO Pharma Limited (NASDAQ: OKYO), an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod to treat neuropathic corneal pain (NCP), an ocular condition associated with chronic and often severe nerve-related pain but without an FDA-approved therapy, is pleased to announce positive top-line data from the recently closed 18-patient Phase 2 trial of urcosimod (formerly called OK-101) to treat NCP. OKYO is the first company to conduct a clinical study to treat NCP disease, a major unmet medical need.
This randomized, double-masked, placebo-controlled, Phase 2 Proof-of-Concept trial of urcosimod to treat NCP was conducted at a single trial site at Tufts Medical Center in Boston, MA, with Pedram Hamrah, M.D., a leading expert in NCP, as Principal Investigator.
Top Line Data
Note: Primary Endpoint of Phase 2 trial was change in mean pain scores from baseline (Visit 1, Day 0) to end of treatment (Visit 4, Day 84), as measured by a VAS scale of 0-10.
For the per-protocol population, change in mean pain score was 5.5 in the
In the intent-to-treat population,
The drug-effect size of
“I have been dedicated to the pursuit of a drug to treat NCP for a considerable period of time, and these top-line results, along with the findings of our previous in-house animal model study of urcosimod to treat NCP, are very exciting and extremely encouraging,” said Dr. Hamrah, Vice Chair of Research and Academic Programs for the Department of Ophthalmology at Tufts Medical Center and Principal Investigator of the phase 2 trial of urcosimod. “I find it particularly encouraging that the drug showed a dramatic reduction in VAS pain scores in patients who have particularly high VAS pain scores, indicating a more severe form of the disease.”
“We are thrilled with these initial top-line results in patients treated with urcosimod from this first in-human phase 2 trial,” said Gary S. Jacob, Ph.D., Chief Executive Officer of OKYO Pharma. “In particular, the results strengthen our conviction that this drug may be particularly effective at showing a significant reduction in pain in patients with a greater degree of neuropathic pain. Throughout the trial we were also pleased by the feedback from patients and are excited about the opportunity to provide relief to NCP patients currently living day-to-day with the life-altering effects of this relentless neuropathic pain. I want to thank Dr. Pedram Hamrah, one of the world’s foremost experts on NCP, and our patients and study site team for their participation and commitment to this important trial of urcosimod to treat NCP.”
“I am very proud that OKYO Pharma is pioneering the development of the first topical drug with the potential to treat Neuropathic Corneal Pain, a debilitating disease, which currently has no FDA approved therapy,’’ said Mr. Gabriele Cerrone, Founder and Chairman of OKYO Pharma. “Our goal now is to bring this drug to the market as quickly as possible to help patients suffering from this terrible disease.”
Significant interest in urcosimod has been brought to OKYO’s attention by sufferers of NCP during this phase 2 trial, and OKYO will be pushing to accelerate clinical development of urcosimod with further trials in the near future. In addition, several patients from the just completed trial have also requested availability of the drug through FDA’s “Expanded Access” program (also referred to as “Compassionate Use”) which the Company is planning to arrange, contingent on necessary FDA approvals, for those present patients and future patients who have specifically completed clinical trials on urcosimod.
In line with earlier findings from a previously conducted Phase 2 trial to evaluate urcosimod to treat dry eye disease, results from the
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 randomized, double-masked, placebo-controlled, Phase 2 trial of urcosimod to treat NCP was conducted at a single trial site at Tufts Medical Center in Boston, MA, and was planned to enroll 48 patients. In April 2025, OKYO Pharma announced the decision to close the trial early, with 17 patients having completed the study, based on OKYO’s strong desire to unmask the clinical data for an early read-out on a drug effect. Patients participating in the trial had all been suffering from long-term chronic NCP and had previously been treated with multiple therapies with very limited or no response. The Phase 2 study was designed as a double-masked, randomized, 12-week placebo-controlled trial in NCP patients.
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 ocular diseases. 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 efficacy of urcosimod within the ocular environment. Urcosimod showed clear statistical significance in multiple endpoints in a 240-patient completed Phase 2, multi-center, double-masked, placebo-controlled trial to treat dry eye disease, and just completed a randomized, placebo-controlled, double-masked Phase 2 trial of urcosimod to treat neuropathic corneal pain.
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. A Phase 2 trial of urcosimod to treat neuropathic corneal pain patients was just completed by OKYO.
For further information, please visit www.okyopharma.com.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Company’s current expectations, estimates, and projections about its industry, its beliefs, and assumptions. Words such as ‘anticipates,’ ‘expects,’ ‘intends,’ ‘plans,’ ‘believes,’ ‘seeks,’ ‘estimates,’ and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company’s control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. These and additional risks and uncertainties are described more fully in the company’s filings with the SEC, including those factors identified as “Risk Factors” in our most recent Annual Report on Form 20-F, for the fiscal year ended March 31, 2024. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these
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