OKYO Pharma Announces Registration Pathway with 100 Patient Multi-Center Clinical Trial of Urcosimod in Neuropathic Corneal Pain
OKYO Pharma (NASDAQ: OKYO) has announced plans for an expanded clinical trial of urcosimod, its lead drug candidate for treating neuropathic corneal pain (NCP). Following successful Phase 2 results where 75% of patients showed over 80% pain reduction, the company will conduct a 100-patient multi-center clinical trial across multiple U.S. sites.
The new multiple-ascending-dose (MAD) trial aims to identify optimal dosing for Phase 3 trials and further understand urcosimod's micellar characteristics. The company expects topline data in 2026. With Fast-Track designation from the FDA, OKYO is preparing for regulatory discussions to define primary endpoints for potential registration.
OKYO Pharma (NASDAQ: OKYO) ha annunciato piani per un ampliamento dello studio clinico di urcosimod, candidato principale per trattare il dolore neuropatico corneale (NCP). In seguito ai positivi risultati della fase 2, dove il 75% dei pazienti ha mostrato una riduzione del dolore superiore all’80%, l’azienda procederà con un trial clinico multicentro con 100 pazienti in diverse sedi negli Stati Uniti.
Il nuovo trial a dosi multiple ascendenti (MAD) mira a identificare il dosaggio ottimale per gli studi di fase 3 e a comprendere ulteriormente le caratteristiche micellari di urcosimod. L’azienda prevede dati principali nel 2026. Con la designazione Fast-Track da parte della FDA, OKYO si prepara a discussioni regolatorie per definire gli endpoint primari per una potenziale registrazione.
OKYO Pharma (NASDAQ: OKYO) ha anunciado planes para ampliar el ensayo clínico de urcosimod, su candidato principal para tratar el dolor neuropático de la córnea (NCP). Tras resultados exitosos de la fase 2 donde el 75% de los pacientes mostró una reducción del dolor de más del 80%, la empresa realizará un ensayo clínico multicentro de 100 pacientes en múltiples sitios de Estados Unidos.
El nuevo ensayo de dosis múltiples ascendente (MAD) tiene como objetivo identificar la dosis óptima para los ensayos de fase 3 y comprender mejor las características micelerales de urcosimod. La compañía espera datos preliminares en 2026. Con la designación de Fast-Track de la FDA, OKYO se está preparando para discusiones regulatorias para definir los endpoints primarios para una posible registro.
OKYO Pharma(NASDAQ: OKYO)가 신경학적 각막 통증(NCP) 치료를 위한 주력 후보물질인 우르코시모드의 확대 임상 계획을 발표했습니다. 2상에서 환자의 75%가 통증을 80% 이상 감소시켰다는 성과에 이어, 회사는 미국 전역의 다기관에서 100명 규모의 다기관 임상시험을 수행할 예정입니다.
새로운 다용량 증량(MAD) 시험은 3상 시험의 최적 용량을 식별하고 우르코시모드의 미셀 특징을 더 이해하는 것을 목표로 합니다. 회사는 2026년 상반기 데이터를 기대합니다. FDA의 고속승인(Fast-Track) 지정을 바탕으로 OKYO는 잠재적 등록을 위한 주요 평가변수(primary endpoints)를 정의하기 위한 규제 논의를 준비하고 있습니다.
OKYO Pharma (NASDAQ: OKYO) a annoncé des plans d’extension du essai clinique d’urcosimod, son candidat phare pour le traitement de la douleur neuropathique cornéenne (NCP). Suite à des résultats positifs de la phase 2 où 75 % des patients ont montré une réduction de la douleur de plus de 80 %, la société mènera un essai clinique multicentrique de 100 patients dans plusieurs sites des États-Unis.
Le nouvel essai à doses montantes multiples (MAD) vise à identifier la posologie optimale pour les essais de Phase 3 et à mieux comprendre les caractéristiques micellaires d’urcosimod. L’entreprise prévoit données préliminaires en 2026. Avec la désignation Fast-Track par la FDA, OKYO se prépare à des discussions réglementaires pour définir les endpoints primaires en vue d’un enregistrement potentiel.
OKYO Pharma (NASDAQ: OKYO) hat Pläne angekündigt, die klinischen Studien von Urcosimod, dem führenden Kandidaten zur Behandlung von peripheren Hornhautschmerzen (NCP), auszuweiten. Nach erfolgreichen Phase-2-Ergebnissen, bei denen 75% der Patienten eine Schmerzlinderung von über 80% zeigten, wird das Unternehmen eine 100-Patienten-Studie multicenter an mehreren Standorten in den USA durchführen.
Die neue Mehrfachdosierungs-aufsteigende (MAD) Studie zielt darauf ab, die ideale Dosierung für Phase-3-Studien zu identifizieren und die mikellaren Eigenschaften von Urcosimod weiter zu verstehen. Das Unternehmen erwartet Topline-Daten im Jahr 2026. Mit der FDA-Fast-Track-Zuordnung bereitet sich OKYO auf regulatorische Gespräche vor, um primäre Endpunkte für eine potenzielle Zulassung festzulegen.
OKYO Pharma (ناسداك: OKYO) أعلنت عن خطط لتوسيع التجربة السريرية لـ urcosimod، المرشح الرائد لديها لعلاج ألم القرنية العصبي (NCP). عقب نتائج المرحلة 2 الناجحة حيث أظهر 75% من المرضى انخفاضاً في الألم يتجاوز 80%، ستجري الشركة تجربة سريرية متعددة المراكز تشمل 100 مريض عبر مواقع متعددة في الولايات المتحدة.
والتجربة الجديدة بجرعات مضافة متزايدة (MAD) تهدف إلى تحديد الجرعة المثلى للاختبارات في المرحلة 3 وفهم ملامح العديدية (ميسلر) لـ urcosimod بشكل أعمق. تتوقع الشركة بيانات أولية في 2026. مع تصنيف FDA Fast-Track، تستعد OKYO لمناقشات تنظيمية لتحديد نقاط النهاية الأساسية لإمكانية التسجيل.
OKYO Pharma（纳斯达克股票代码：OKYO）宣布扩大其领先药物候选药 urcosimod 的临床试验计划，以治疗神经性角膜疼痛（NCP）。在第二阶段取得成功的结果后，有75%的患者痛感降低超过80%，公司将进行一项覆盖美国多家中心的 100名患者的多中心临床试验。
新的多剂量递增（MAD）试验旨在确定 Phase 3 试验的最佳给药剂量，并进一步了解 urcosimod 的微胶束特性。公司预计 2026 年公布初步数据。在 FDA 的快速通道（Fast-Track） designation 下，OKYO 正在为定义潜在注册的主要终点进行监管讨论做准备。
- 75% of patients showed over 80% pain reduction in Phase 2 trial
- Fast-Track designation from FDA enables expedited regulatory process
- Expansion to 100-patient multi-center trial demonstrates progression
- Treatment addresses unmet medical need with no current FDA-approved therapy
- Final trial results not expected until 2026
- Optimal registration dose still needs to be determined
- FDA meeting and primary endpoint definition still pending
Insights
OKYO's urcosimod shows promising Phase 2 results for neuropathic corneal pain; advancing to larger trial with FDA fast-track designation.
OKYO Pharma has revealed a significant advancement in their clinical development program for urcosimod, their lead drug candidate targeting neuropathic corneal pain (NCP) - a condition characterized by severe nerve-related ocular pain with no FDA-approved treatments currently available.
The company's recently completed Phase 2 proof-of-concept trial produced remarkably positive results, with
OKYO is now progressing to a more substantial multiple-ascending-dose (MAD) trial that will enroll approximately 100 NCP patients across multiple U.S. clinical sites. This randomized, placebo-controlled study will help identify the optimal registration dose for future Phase 3 trials and further characterize the drug's micellar properties. The company expects topline data from this expanded trial in 2026.
Notably, urcosimod has already secured FDA Fast-Track designation, which should accelerate the regulatory process. OKYO is preparing for FDA discussions to define primary endpoints and registration requirements, a crucial step toward potential approval.
The robust pain reduction seen in early trials, combined with the Fast-Track status and clear development pathway, positions OKYO to potentially bring the first approved therapy to NCP patients - representing a significant opportunity in an untapped therapeutic space with considerable patient need.
LONDON and NEW YORK, Sept. 22, 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, today announced plans for the next stage of clinical development for its lead drug candidate, urcosimod, to treat neuropathic corneal pain (NCP).
The announcement follows the release in July of topline data from OKYO’s randomized, double-masked, placebo-controlled Phase 2 proof-of-concept trial of urcosimod in 17 NCP patients conducted at Tufts Medical Center in Boston, MA, with Pedram Hamrah, M.D., a world-leading expert in NCP, as Principle Investigator.
After 12 weeks of treatment,
Building on these promising results, OKYO is now focused on advancing to a multiple-ascending-dose (MAD) clinical trial designed to:
- Enroll approximately 100 NCP patients across several U.S. clinical sites in this randomized, placebo-controlled, double-masked trial.
- Identify the optimal registration dose for urcosimod for future Phase 3 trials.
- Develop further understanding of urcosimod’s unique micellar drug characteristics.
Negotiations with several leading U.S. clinical sites are underway, and OKYO anticipates topline data from this trial in 2026.
As part of the registration pathway, OKYO continues to progress towards a meeting with the U.S. Food and Drug Administration (FDA) to discuss requirements for an approvable drug to treat NCP and to define the primary endpoint required for potential registration. Urcosimod currently holds Fast-Track designation, which is expected to enable quick turn-around on having the meeting with the FDA.
“The results from our first-in-patient Phase 2 trial were exceptionally encouraging, with three-quarters of the per-protocol
OKYO remains committed to addressing this major unmet medical need and will provide updates as its development program progresses.
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.
Inquiries:
| Business Development & Investor Relations | Paul Spencer | +44 (0)20 7495 2379 |
FAQ
What were the Phase 2 trial results for OKYO's urcosimod in treating neuropathic corneal pain?
How many patients will be enrolled in OKYO's new clinical trial for urcosimod?
When will OKYO Pharma release results from its new urcosimod clinical trial?
What is the regulatory status of OKYO's urcosimod for neuropathic corneal pain?
What is the current treatment landscape for neuropathic corneal pain (NCP)?
