OKYO Pharma Limited announced a clinical update and its interim results for the 6 months ended Sept. 30, 2024. The company said in a press release that it anticipates completing enrollment of the 48-patient trial for OK-101 to treat neuropathic corneal pain (NCP), by the end of 2Q 2025 and releasing top-line data on the Phase 2a trial in 4Q 2025.
The Phase 2a study is designed as a double-masked, randomized, 12-week placebo-controlled trial in NCP patients. A total of 48 patients are planned to be enrolled in the trial, with NCP disease confirmed via confocal microscopy, patient symptoms and medical histories.
OK-101, a novel, non-opioid therapeutic candidate, is designed to target and alleviate the debilitating pain associated with corneal nerve damage. NCP, often resulting from conditions such as dry eye disease (DED), surgery, or infections, can severely impact a patient’s quality of life.
In February 2024, OKYO Pharma announced that it was the first company to receive an investigational new drug application clearance by the US Food and Drug Administration (FDA) to clinically evaluate a drug specifically to treat NCP with the clearance of the IND for OK-101.
The OK-101 trial is designed as a single-center trial and is being led by Pedram Hamrah, MD, of Tufts Medical Center, as principal investigator, the company said. In addition to his work on OK-101, of which he is a co-inventor and member of OKYO’s Scientific Advisory Board, Dr. Hamrah is also conducting studies on developing new corneal nerve function tests and is a consultant to the company covering non-clinical studies of OK-101 to evaluate its mechanism of action.
In January of 2024, OKYO reported positive top line data from the phase 2b trial of OK-101 to treat DED patients. This OK-101 first-in-human phase 2b trial established a clear clinical path for potential further clinical development via a phase 3 study design using FDA-recognized endpoints, the company said. OK-101 demonstrated statistically significant benefit in a “sign” endpoint, namely “total conjunctival staining” as measured by the Ora Calibra Staining Scale as early as Day 29 (p = 0.034).
OK-101 also improved at least two “symptom” endpoints of DED, including “burning/stinging” as measured by the Ora Calibra 4-symptom questionnaire, and as measured by a visual analog scale as early as Day 15 (p = 0.04 and p = 0.03, respectively), as well as “blurred vision”, with statistically significant improvement in blurred vision occurring by Day 29 (p = 0.01).
Of the 240 patients treated in the trial, treatment emergent adverse events (TEAEs) were observed to be similar to the placebo-treated group. No severe drug related ocular TEAEs were seen. Possible drug-related TEAEs were observed in one patient in the OK-101 0.05% treatment group and 3 patients in the placebo-treated group, again highlighting the favorable safety profile of OK-101.
OKYO said it is planning to engage with the FDA on the next clinical plans for OK-101 to treat DED.