OKYO Pharma Ltd (NASDAQ:OKYO) has announced positive safety and efficacy results in its Phase 2 trial of OK-101 ophthalmic solution for the treatment of inflammatory dry eye disease (DED). The trial, conducted at six US sites with 240 subjects, demonstrated promising outcomes and established a clear path for Phase 3 registration trials.
The randomized, double-masked, placebo-controlled Phase 2 trial spanned 14 weeks, including a two-week run-in period on placebo, followed by 12 weeks in the randomized portion. Patients were divided into three cohorts, with varying doses of OK-101. The trial's endpoints adhered to Food and Drug Administration (FDA) guidelines for Dry Eye: Developing Drugs for Treatment.
OK-101 exhibited significant superiority over placebo in multiple aspects. The conjunctival staining endpoint showed statistical significance as early as Day 29 (p = 0.034). Symptom improvement, including burning and burning/stinging, was noted by Day 15 (p = 0.04 and p = 0.03, respectively). Blurred vision also demonstrated a statistically significant improvement on Day 29 (p = 0.01).
The safety profile of OK-101 was favorable, with treatment-emergent adverse events (TEAEs) comparable to the placebo group. Notably, fewer subjects in the OK-101 arm discontinued the study medication (2.5%) compared to the placebo arm (3.8%).
“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," commented Dr Jay Pepose, founder and medical director of Pepose Vision Institute and professor of Clinical Ophthalmology at Washington University School of Medicine in St Louis.
"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."
OKYO CEO Gary Jacob highlighted the encouraging results, steering the future design of Phase 3 trials for OK-101.
“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," Jacob said.
"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."
Although not meeting the original primary endpoints, OKYO Pharma said it plans to advance OK-101 into Phase 3 trials, aiming to develop a highly differentiated dry eye product. The potential for treating corneal neuropathic pain is also under consideration.
OKYO chief scientific officer Dr Raj Patil highlighted the novelty of OK-101, citing its multiple mechanisms of action, including anti-inflammatory activity and restoration of mucin-secreting goblet cells in the conjunctiva.
“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,” Patil added.
“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."
Patil said further discussions with scientific advisers and seeking FDA guidance on Phase 3 trial design are planned.
"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 non-executive chairman Gabriele Cerrone concluded.
The company said a comprehensive data analysis, including additional signs and symptoms, will be presented in a first-quarter 2024 conference call once it 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.
OKYO Pharma is focusing on the discovery and development of novel molecules to treat inflammatory DED and ocular pain.
Its therapeutic candidate 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.