OK-101 for Neuropathic Corneal Pain

Overseen By Nancy Gee, MPH

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Okyo Pharma Ltd

Must not be taking: Topical medications

Disqualifiers: Ocular infection, Intraocular inflammation, Corneal scar, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called OK-101, an experimental eye drop, to determine its effectiveness in reducing neuropathic corneal pain. This type of eye pain can feel like burning, stinging, or discomfort. Participants will receive eye drops containing either the new treatment or a placebo, applied four times a day for 12 weeks, to compare the treatment's effectiveness and safety. The trial seeks individuals who have experienced these eye pain symptoms for at least three months and can adhere to the study's routine. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

You will need to stop using any topical eye medications 8 days before the trial starts. However, you can continue taking your oral medications for neuropathic corneal pain as long as there are no changes during the study.

Is there any evidence suggesting that OK-101 is likely to be safe for humans?

Research shows that OK-101 is intended as a safe treatment for nerve-related eye pain. In earlier studies, patients generally tolerated OK-101 well, with no major safety issues. Reports from these studies indicate that the number of unwanted side effects was similar to those in the placebo group, suggesting that OK-101 is relatively safe. Since OK-101 is in a Phase 2 trial, it has already passed initial safety checks, demonstrating a good safety record in earlier stages. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for neuropathic corneal pain?

Researchers are excited about OK-101 for neuropathic corneal pain because it offers a potentially new approach to treatment. Unlike current options like topical analgesics or oral pain medications, which often have systemic side effects, OK-101 is applied directly to the eye, minimizing these risks. The active compound in OK-101 is designed to target pain pathways specifically in the cornea, which could provide more effective relief with fewer side effects. Additionally, the treatment is delivered in a precise, controlled manner through eye drops, which may enhance patient convenience and adherence compared to other methods requiring more frequent or invasive administration.

What evidence suggests that OK-101 could be an effective treatment for neuropathic corneal pain?

This trial will compare different dosages of OK-101 for neuropathic corneal pain. Research has shown that OK-101, also known as urcosimod, may help treat nerve-related eye pain. In one study, patients using a 0.05% dose reported over 80% less pain after 12 weeks. Another study found that people taking OK-101 experienced an average pain reduction of 5.5 on a 10-point scale. These results suggest that OK-101 could effectively reduce eye pain for many patients. While research continues, these findings are promising for those considering this treatment. ^{1 3 6 7 8}

Who Is on the Research Team?

Pedram Hamrah, MD

Principal Investigator

Tufts Medical Center

Are You a Good Fit for This Trial?

This trial is for individuals experiencing neuropathic corneal pain, which is a type of nerve pain in the eye. Participants should be able to apply eye drops four times a day. The specific inclusion and exclusion criteria are not provided, but typically these would detail necessary conditions or characteristics participants must have or lack.

Inclusion Criteria

Satisfying all Informed Consent requirements

Ability and willingness to comply with study procedures

I have had symptoms like burning or pain in my eye for at least 3 months.

Exclusion Criteria

Evidence of any corneal scar/corneal edema

I have signs of inflammation inside my eye.

Participation in another clinical trial concurrently

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive OK-101 or placebo instilled in affected eye(s) four times daily for 12 weeks

12 weeks

Regular visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• OK-101

Trial Overview The study tests two concentrations of OK-101 (0.05% and 0.1%) against a placebo to see if they can reduce eye pain when used as eye drops four times daily. Effectiveness will be measured using a visual analogue scale (VAS), which helps patients rate their pain level.

How Is the Trial Designed?

Group I: Arm B Experimental Treatment 1 Intervention

OK-101, 0.1% - one drop of study drug (0.1%) will be instilled in affected eye(s), four times daily (every 4 hours up to 16 hours, e.g., 9:00 am, 01:00 pm; 05:00 pm, 09:00pm) for 12 weeks.

Group II: Arm A Experimental Treatment 1 Intervention

OK-101, 0.05% - one drop of study drug (0.05%) will be instilled in affected eye(s), four times daily (every 4 hours up to 16 hours, e.g., 9:00 am, 01:00 pm; 05:00 pm, 09:00pm) for 12 weeks

Group III: Arm C Placebo Group 1 Intervention

Placebo - one drop of placebo will be instilled in affected eye(s), four times daily (every 4 hours up to 16 hours, e.g., 9:00 am, 01:00 pm; 05:00 pm, 09:00pm) for 12 weeks.

Who Is Running the Clinical Trial?

Okyo Pharma Ltd

Lead Sponsor

Trials

2

Recruited

290+

Tufts Medical Center

Collaborator

Trials

264

Recruited

264,000+

Published Research Related to This Trial

Neuropathic corneal pain (NCP) is a challenging condition to manage due to its ill-defined nature, and this review provides guidelines for diagnosis and treatment based on a systematic literature search from 1966 to 2017, including evidence from randomized controlled trials.

First-line treatments for NCP include topical neuroregenerative and anti-inflammatory agents, as well as systemic medications like tricyclic antidepressants and anticonvulsants, while complementary therapies such as acupuncture and omega-3 supplementation may offer additional benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Neuropathic Corneal Pain: Approaches for Management. Dieckmann, G., Goyal, S., Hamrah, P.[2022]

Corneal neuropathic pain can occur in patients with dry eye disease even when there are no visible signs, making diagnosis and treatment challenging.

Understanding corneal neuropathic pain as a distinct condition is crucial for developing better treatment options for patients suffering from this debilitating issue.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Corneal pain without stain: is it real? Rosenthal, P., Baran, I., Jacobs, DS.[2022]

In a rat model of dry eye disease (DED), mirogabalin significantly reduced hyperalgesia and chronic ocular pain, indicating its potential effectiveness for treating similar symptoms in humans.

Mirogabalin administration led to a notable decrease in pain-related behaviors and reduced markers of pain in the brain, suggesting it could be a promising treatment option for patients suffering from DED-related ocular pain.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of Mirogabalin on Hyperalgesia and Chronic Ocular Pain in Tear-Deficient Dry-Eye Rats. Kikuchi, K., Tagawa, Y., Murata, M., et al.[2023]

In a case series of three patients with severe neuropathic dry eye-like pain, two patients experienced significant pain relief within 48 hours after treatment for hidden Meibomian gland dysfunction, highlighting the importance of identifying and treating underlying ocular surface issues.

The findings suggest that addressing peripheral causes of pain, such as obstructive Meibomian gland dysfunction, can effectively reverse symptoms of neuropathic pain, potentially reducing the need for systemic treatments that carry risks of serious side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Successful reversal of neuropathic eye pain by treatment of occult ocular surface disease: Case series and implications. Maskin, SL.[2022]

Topical corticosteroids and blood-derived tear preparations have been found to be effective in treating corneal neuropathic pain, which often does not respond to standard dry eye treatments.

Emerging therapies like topical lacosamide and low-dose naltrexone show promise as additional options for managing this type of pain, highlighting the need for diverse treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topical Therapeutic Options in Corneal Neuropathic Pain. Nortey, J., Smith, D., Seitzman, GD., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06637527

The Study Assessing the Safety and Efficacy of OK-101 ... The primary objective of this study, in subjects with Neuropathic Corneal Pain, is to evaluate the efficacy and safety of OK-101 0.05% and 0.1% as compared to ...

2. okyopharma.com okyopharma.com/okyo-pharma-unveils-strong-phase-2-clinical-trial-results-for-urcosimod-to-treat-neuropathic-corneal-pain/

OKYO Pharma Unveils Strong Phase 2 Clinical Trial ... After 12 weeks of treatment, 75% of per-protocol patients receiving 0.05% urcosimod showed greater than 80% reduction in neuropathic corneal ...

3. ophthalmologytimes.com ophthalmologytimes.com/view/okyo-pharma-announces-positive-results-from-phase-2-trial-of-urcosimod-for-neuropathic-corneal-pain

OKYO Pharma announces positive results from phase 2 ... OKYO Pharma has announced positive top-line data from the recently closed phase 2 trial of urcosimod (formerly called OK-101) to treat neuropathic corneal pain ...

4. clinicaltrialsarena.com clinicaltrialsarena.com/news/okyo-trial-ncp-therapy/

OKYO reports top-line data from trial of NCP therapy OKYO Pharma has reported encouraging top-line outcomes from the randomised Phase II trial of urcosimod (previously referred to as OK-101), ...

5. fiercebiotech.com fiercebiotech.com/biotech/okyo-sets-sights-first-approval-neuropathic-eye-pain-after-phase-2-win

Okyo's eye drops reduce neuropathic eye pain in phase 2 ... Okyo Pharma's urcosimod reduced pain levels by an average of 5.5 on a 10-point scale, hitting the main goal of a phase 2 trial and teeing up a ...

6. ophthalmologytimes.com ophthalmologytimes.com/view/first-patient-dosed-in-clinical-trial-to-treat-neuropathic-corneal-pain

First patient dosed in clinical trial to treat neuropathic ... The Phase 2 clinical trial of OK-101 is a double-masked, randomized, 12-week placebo-controlled study in patients with neuropathic corneal pain ...

7. okyopharma.com okyopharma.com/wp-content/uploads/2025/06/OKYO-Corp-Presentation-JUNE-2025-FINAL-V2.pdf

Clinical Development of Urcosimod to Treat Neuropathic ... To evaluate the efficacy of urcosimod compared to placebo instilled QID in subjects with neuropathic corneal pain, assessed by visual analogue scale (VAS).

8. firstwordpharma.com firstwordpharma.com/story/5931340

OKYO Pharma to Complete Enrollment of Neuropathic ... OK-101, a novel, non-opioid therapeutic candidate, is designed to target and alleviate the debilitating pain associated with corneal nerve ...