OKYO Pharma Announces OK-101 Successfully Achieved Statistical Significance for Both Sign and Symptom Endpoints in its First-in-Human Phase 2 Trial of OK-101 in Patients with Dry Eye Disease

2024-01-08

临床2期临床结果临床3期

LONDON, UK and NEW YORK, NY, USA I January 08, 2024 I OKYO Pharma Limited (NASDAQ: OKYO), a clinical-stage biopharmaceutical company developing innovative therapies for the treatment of inflammatory dry eye disease (DED), a multi-billion-dollar market, and for neuropathic corneal pain, a severe ocular condition without an FDA approved therapy, today reports positive safety and efficacy results in its Phase 2, randomized, double-masked, placebo-controlled trial evaluating the safety and efficacy of OK-101 ophthalmic solution in subjects with DED. This first-in-human trial of OK-101 established a clear and informed path for further development in Phase 3 registration trials.

The double-masked, randomized, placebo-controlled Phase 2 trial was conducted at six sites in the U.S. and enrolled 240 subjects with DED dosed twice-daily (BID). Patients were randomly divided into 3 cohorts, with one of the cohorts dosed with 0.05% OK-101 (n=80), a second with 0.1% OK-101 (n=80), and the third cohort with vehicle (n=80). The duration of a patient’s treatment was 14 weeks, including a 2-week run-in period on placebo, to exclude placebo responders from the study, followed by 12 weeks in the randomized portion of the study.

Highlights of OK-101 Phase 2 Trial

“It is remarkable that in this first in human study of OK-101 ophthalmic solution, an analysis of all randomized subjects demonstrated a persistent, statistically significant improvement in multiple dry eye-related symptoms as early as day 15, along with a sign, total conjunctival lissamine green staining, by day 29. Ameliorating this unique constellation of signs and symptoms may reflect the differentiated mechanism of action of OK-101. Also of note, drop comfort was excellent and the safety profile favorable. This is evidenced by fewer subjects discontinuing study medication in the OK-101 arm (2.5%) than the placebo arm (3.8%) due to treatment-emergent adverse events. This highly favorable tolerability profile is very significant because many patients commonly discontinue currently available dry eye medications due to unwanted side effects, such as blurred vision, conjunctival redness or altered taste sensation (dyseguesia),” said Jay Pepose, M.D., Ph.D., Founder and Medical Director of Pepose Vision Institute and Professor of Clinical Ophthalmology at Washington University School of Medicine in St. Louis.

“We were pleased to identify both a statistically significant “sign” endpoint – “total conjunctival staining”, as well as two statistically significant “symptom” endpoints – “stinging/burning” and “blurred vision” in our first trial in man. These results are extremely encouraging and will steer the selection of the future primary endpoints of subsequent Phase 3 trials of OK-101 to treat DED,” said Gary S. Jacob, Ph.D., CEO of OKYO Pharma. “Moreover, we were pleased to see these benefits showing up as early as 15 days after dosing, along with showing meaningful durability until the end of the trial. While we didn’t meet the original primary endpoints, the totality of the data support advancing forward with a new study design that leverages our learnings of the novel mechanism of action of OK-101. We plan to advance OK-101 into Phase 3 clinical trials, with the goal of developing a highly differentiated dry eye product to help patients underserved by current treatments. These results also further support the potential of OK-101 for the treatment for corneal neuropathic pain, which is our parallel development focus for OK-101 in 2024.”

“To our knowledge, there are no FDA approved DED drugs that have been shown in clinical studies to improve conjunctival staining, a primary “sign’” endpoint for DED drug approval,” said Dr. Raj Patil CSO of OKYO Pharma. “OK-101 is a drug with multiple novel and differentiated mechanisms of action that include anti-inflammatory activity and restoring the mucin secreting goblet cells in conjunctiva, as observed in animal models of DED. In addition, we see improvement in blurred vision, a primary “symptom” endpoint for DED drug approval, in OK-101 treated DED patients that is plausibly tied to enhancing the tear film stability and lubrication of the ocular surface by normalizing the mucin component in the tear film secreted by goblet cells. We will work with our scientific advisers and seek additional FDA guidance on next steps to designing a Phase 3 trial to confirm OK-101’s novel mechanism of action.”

“I am extremely proud of our team who executed seamlessly to complete the OK-101 trial by the end of the year and want to thank our dedicated site investigators and key opinion leaders,” said Gabriele Cerrone, Non-Executive Chairman of OKYO Pharma. "We successfully completed a Phase 2 trial of a novel compound that was previously never tested in humans that detected unequivocal efficacy signals that warrants further investigation in a larger trial.”

OKYO management plans to host a conference call to provide further data on the trial results once the Company has finished a more comprehensive analysis of the data from the trial including additional signs and symptoms of dry eye disease as well as efficacy within the more severe patient population at baseline. The conference call is planned for Q1, 2024.

About Dry Eye Disease

Dry eye disease is a common condition that occurs when an individual’s tears are unable to adequately lubricate the eyes. This condition affects approximately 49 million people in the U.S. alone and has been a difficult one to positively diagnose and to treat due to the multifactorial nature of the condition. A number of contributing factors can lead to this condition, including age, sex, certain medical conditions, reduced tear production and tear film dysfunction. Tear film instability typically leads to inflammation and damage to the ocular surface.

About OK-101

OK-101 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. OK-101 was developed using a membrane-anchored-peptide technology to produce a novel long-acting drug candidate for treating dry eye disease. OK-101 has been shown to produce anti-inflammatory and pain-reducing activities in mouse models of dry eye disease and corneal neuropathic pain (NCP), respectively, and is designed to combat washout through the inclusion of the lipid anchor built into the drug molecule to enhance the residence time of OK-101 within the ocular environment. OK-101 showed clear statistical significance in multiple endpoints in a recently completed Phase 2, multi-center, double-blind, placebo-controlled trial to treat dry eye disease.

About OKYO

OKYO Pharma Limited (NASDAQ: OKYO) is a clinical stage biopharmaceutical company developing innovative therapies for the treatment of DED and NCP, with ordinary shares listed for trading on the NASDAQ Capital Market. OKYO is focused on the discovery and development of novel molecules to treat inflammatory DED and ocular pain. In addition to the recently completed Phase 2 DED trial, OKYO also has plans underway for the opening of a Phase 2 trial for OK-101 to treat NCP in patients with this debilitating condition. For further information, please visit www.okyopharma.com.

SOURCE: OKYO Pharma