Published in Pipeline

FDA approves Okyo Pharma's IND for neuropathic corneal pain candidate

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5 min read

Just 4 months after filing, OKYO Pharma Limited announced the FDA has cleared OK-101, its first investigational new drug (IND) application, to treat neuropathic corneal pain (NCP).

Quick rundown on this company, please.

OKYO Pharma is a London, United Kingdom-based, pre-clinical biopharmaceutical company focused on developing novel molecules to treat inflammatory dry eye disease (DED) and chronic ocular pain.

Now this candidate.

OK-101 is a lipid-conjugated chemerin peptide antagonist of the ChemR23 G-protein coupled receptor.

Note: this protein can be found on immune cells of the eye, is responsible for the inflammatory response, and is encoded in humans by the chemerin chemokine-like receptor 1 (CMKLR1) gene.

One more note: OK-101 was originally developed as a drug candidate for treating DED. Further, it’s also in preclinical development for allergic conjunctivitis and uveitis as a potential treatment for these diseases.

Explain how it operates.

Developed using a membrane-anchored-peptide (MAP) technology, OK-101 has been shown to produce anti-inflammatory and pain-reducing activities in mouse models of DED and NCP.

It is designed to mitigate washout via a lipid “anchor” that is contained in the candidate drug molecule to enhance the residence time of OK-101 within the ocular surface environment.

Gotcha. So what does this IND mean?

OKYO’s IND submission included an open-label-designed clinical trial, which the FDA gave positive feedback on.

This approval is enabling the company to design a double-masked, randomized, 12-week, placebo-controlled phase 2 trial evaluating OK-101 compared to a placebo for NCP—its first for this indication (more on that in a moment).

Is there any clinical data on OK-101 yet?

There is!  But the data on it thus far is strictly for its potential indication for DED.

In January 2024, OKYO released data from its first-in-human (FIH) phase 2 multi-center, placebo-controlled trial (NCT05759208) for DED, reporting that OK-101 successfully achieved statistical significance for both sign and symptom endpoints.

Key highlights included:

  • Statistically significant symptom improvement noted in efficacy endpoints as early as the 15-day first visit following dosing
    • Included burning, as measured by the Ora Calibra 4-symptom questionnaire as well as burning/stinging measured by a visual analogue scale (VAS)
  • Demonstrated superiority for total conjunctival staining  as early as Day 29
    • Measured by the Ora Calibra Staining Scale
  • Statistical significant improvement in blurred vision at Day 29
  • OK-101 illustrated placebo-like tolerability, low adverse event profile, and no severe drug-related ocular adverse events

Note: These endpoints were FDA-recognized.

Of course, this followed the company’s previous reports of favorable safety data from the trial in October 2023. See here for those details.

Gotcha. Now talk about this upcoming NCP trial.

Based on the FDA’s acceptance, the single-center phase 2 study is anticipated to enroll 52 patients—with an NCP diagnosis confirmed via confocal microscopy—and include a primary endpoint measured via VAS pain relief scores.

Who’s conducting it?

Per the company, the study is being conducted at Tufts Medical Center in Boston, Massachusetts, by principal investigator Pedram Hamrah, MD, co-inventor of the OK-101 patent.

Dr. Hamrah is a professor and vice chair of Research and Academic Programs as well as co-director and director of the Center for Translational Ocular Immunology at Tufts Medical Center, and a member of OKYO Pharma’s Scientific Advisory Board.

What else can you tell me?

Per OKYO, the trial has an expected 6 to 9-month study period.

And the company previously reported that it’s expected to clock in at just under $1 million, including funding for drug manufacturing and formulation for investigational use.

And the significance of this?

According to OKYO CEO Gary S. Jacob, PhD, NCP currently has no FDA-approved therapy and is “a major unmet medical need for patients suffering from his condition.”