OKYO Pharma Receives FDA Approval of IND for OK-101 in Neuropathic Corneal Pain

Rhea-AI Summary

OKYO Pharma Receives FDA Clearance for OK-101 to Treat Neuropathic Corneal Pain, Launches Phase 2 Clinical Trial

Positive

• OKYO Pharma obtains FDA clearance for OK-101 to address neuropathic corneal pain (NCP)
• The Phase 2 clinical trial for OK-101 to treat NCP is set to begin in Q2 2024
• NCP is classified as an Orphan disease with no FDA-approved therapy
• The trial will include 54 patients with NCP confirmed via confocal microscopy
• Primary endpoint to be measured using VAS pain relief scores

Negative

• None.

Medical Research Analyst

The IND clearance for OK-101 by the FDA marks a significant milestone in the treatment of neuropathic corneal pain (NCP), which is a condition with limited therapeutic options. The clinical trial design—randomized, placebo-controlled, double-masked—is robust, enhancing the credibility of the outcomes. A placebo-controlled trial is essential in establishing the efficacy of a new drug, as it helps to eliminate the placebo effect and provides a clear comparison between the active drug and an inactive substance.

The use of confocal microscopy for confirming NCP disease is noteworthy as it represents a precise diagnostic approach, ensuring the selection of appropriate trial participants. The primary endpoint utilizing Visual Analogue Scale (VAS) pain relief scores is a widely accepted method for assessing patients' pain, which can provide reliable and quantifiable data on the drug's effectiveness in alleviating symptoms.

From a research perspective, the transition from an open-label to a double-masked study design, upon FDA feedback, indicates a strategic move to strengthen the trial's methodological rigor. This can increase the potential for OK-101 to meet the FDA's efficacy and safety standards, which is crucial for eventual market approval.

Financial Analyst

The IND clearance for a novel drug like OK-101 has the potential to positively impact OKYO Pharma's financial outlook. As NCP is an orphan disease, OK-101 could benefit from various incentives, including market exclusivity, tax credits and waiver of certain FDA fees, which can enhance the drug's commercial viability. Moreover, the successful completion of the Phase 2 trial could lead to a significant appreciation of the company's stock value, as investors often respond positively to advancements in a company's drug pipeline.

It's also important to note that the multi-billion-dollar market for inflammatory dry eye disease (DED) treatments suggests a substantial commercial opportunity for OK-101, should it prove effective in treating NCP as well. The drug's dual applicability could position OKYO Pharma favorably within the ocular therapy space, potentially leading to diversified revenue streams and an increased market share.

Market Research Analyst

The clinical advancement of OK-101 for treating NCP addresses a major unmet medical need, which can be a key driver for market adoption upon successful trial outcomes. The rarity of NCP and the lack of FDA-approved therapies place OK-101 in a unique position to become the first-mover in this niche market. A successful trial could not only benefit patients but also create a new therapeutic segment within the ocular pain market.

Furthermore, the involvement of Dr. Pedram Hamrah, a leading expert in NCP, lends additional credibility to the clinical study and could facilitate the adoption of OK-101 by healthcare professionals. The expertise and reputation of the principal investigator often play a role in the perception of a drug's potential, influencing both medical community acceptance and investor confidence.

• OK-101 is the first IND clearance granted by FDA for a drug to begin clinical studies specifically to treat patients suffering with neuropathic corneal pain (NCP), a major unmet medical need
• The initial trial of OK-101 to treat NCP is designed as a randomized, placebo-controlled, double-masked Phase 2 clinical trial and is planned to begin in 2Q 2024
• NCP is an Orphan disease as listed in the National Organization for Rare Disorders

LONDON and NEW YORK, Feb. 09, 2024 (GLOBE NEWSWIRE) -- OKYO Pharma Limited (NASDAQ: OKYO), a clinical-stage biopharmaceutical company developing innovative ocular therapies for the treatment of inflammatory dry eye disease (DED), a multi-billion-dollar market, and for neuropathic corneal pain (NCP), an ocular condition associated with pain but without an FDA approved therapy, announced today that the U.S. Food and Drug Administration (FDA) has cleared OK-101 as its first Investigational New Drug (IND) application for the treatment of NCP.

Notably, the initial IND submission to FDA proposed an open-label design for the clinical trial. Based on positive feedback from FDA, the Phase 2 study is now designed as a double-masked, randomized, 12-week placebo-controlled trial comparing OK-101 to placebo in NCP patients. A total of 54 patients are planned for the study, with NCP disease confirmed via confocal microscopy. The primary endpoint will be measured utilizing VAS pain relief scores. These protocol changes will enable a statistically valid demonstration of a true drug effect of OK-101 on NCP symptoms. OKYO Pharma is scheduling this trial to begin in Q2 2024.

The OK-101 trial, designed as a single-center trial, will be led by Pedram Hamrah, MD, of Tufts Medical Center, as Principal Investigator. Dr. Hamrah is Professor and Vice Chair of Research and Academic Programs, Co-Director of the Cornea Service and Director of the Center for Translational Ocular Immunology at Tufts Medical Center. An ophthalmologist and a clinician-scientist, Dr. Hamrah is a leading expert in NCP and co-inventor on the OK-101 patent. He is also a member of OKYO’s Scientific Advisory Board.

“I am very pleased that we have gained FDA IND clearance for the first drug to be tested for NCP, a debilitating disease,” said Dr. Hamrah. “Receiving the IND clearance in an important and novel indication was not a trivial hurdle to overcome. Now that the path has been opened for drugs to be tested in NCP, I am looking forward to working with the OKYO team to launch this important trial.”

“We are pleased to gain IND clearance for OK-101 to treat NCP as a second important disease target for the Company,” said Dr. Gary S. Jacob, CEO of OKYO. “OK-101 recently demonstrated favorable tolerability in a Phase 2 trial of dry eye patients along with statistically significant improvements in dry eye symptoms such as stinging/burning and blurred vision, which are also hallmarks of NCP. OK-101 was also shown in a cutting-edge mouse model of NCP to significantly reduce ocular neuropathic pain. We are looking forward to advancing OK-101 to potentially treat NCP, a chronically painful ocular disease with no FDA-approved therapy and a major unmet medical need for patients suffering from this condition.”

About NCP
Neuropathic corneal pain (NCP) is a condition that causes pain and sensitivity of the eyes, face, or head. The exact cause of NCP is unknown but thought to result from nerve damage to the cornea combined with inflammation. NCP, which can exhibit as a severe, chronic, or 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. There are no approved commercial treatments currently available for this condition.

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 recently showed clear statistical significance in multiple endpoints in a recently completed Phase 2, multi-center, double-blind, placebo-controlled trial of OK-101 to treat DED.

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.

Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements, including with respect to the anticipated timing of completion of enrolment of the Company’s Phase 2 trial of topical ocular OK-101 to treat DED and the release of top-line data therefrom. 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. 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 forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

Enquiries:

OKYO Pharma Limited	Gary S. Jacob, Chief Executive Officer	917-497-7560
Business Development & Investor Relations	Paul Spencer	+44 (0)20 7495 2379

What is the significance of OK-101 being granted FDA clearance?

OKYO Pharma has received FDA clearance for OK-101 to treat neuropathic corneal pain, marking a significant milestone in addressing this unmet medical need.

When is the Phase 2 clinical trial for OK-101 scheduled to start?

The Phase 2 clinical trial for OK-101 to treat neuropathic corneal pain is planned to commence in the second quarter of 2024.

How many patients are expected to participate in the trial for OK-101?

A total of 54 patients with neuropathic corneal pain confirmed via confocal microscopy are planned to be included in the study.

What will be the primary endpoint measured in the Phase 2 trial for OK-101?

The primary endpoint in the Phase 2 trial for OK-101 will be measured using VAS pain relief scores to assess the drug's effect on neuropathic corneal pain.

Who will lead the single-center trial for OK-101?

Dr. Pedram Hamrah of Tufts Medical Center will lead the single-center trial for OK-101 as the Principal Investigator.