OKYO Pharma announced the acceptance of OK-101 as an investigational new drug following clinical studies investigating the candidate in patients with neuropathic corneal pain, the first to do so.
OKYO Pharma issued a press release announcing investigational new drug (IND) approval by the FDA for OK-101 in neuropathic corneal pain (NCP). The lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor was developed using a membrane-anchored peptide technology to produce a novel long-acting drug candidate, which was originally intended to treat dry eye disease (DED). OK-101 combats washout via the lipid anchor. In mouse models, OK-101 reduced inflammation and pain caused by DED and NCP, respectively, causing OKYO to expand the investigation and propose an open-label design for the clinical trial in the initial IND submission.
Based on FDA feedback, the Phase 2 study is now a double-masked, randomized, 12-week placebo-controlled trial comparing OK-101 to placebo in NCP patients. NCP must be confirmed via confocal microscopy, and the primary end point is measured with VAS pain relief scores. OKYO intends to being the trial in Q2 2024.
The trial, led by Pedram Hamrah, MD, of Tufts Medical Center as Principal Investigator, garnered significant results and met multiple end points for DED. 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,” Hamrah said in a press release. “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.”
NCP, an orphan disease that causes pain and sensitivity in the eyes, face, or head, is a large unmet medical need, and the clinical trials of OK-101 are the first to investigate a therapy for it. While the cause is unknown, NCP is thought to result from a combination of inflammation and corneal nerve damage. There are no approved therapies to relieve patients with NCP, and providers are forced to use off-label systemic and topical therapies to help patients find relief.
In January 2024, OKYO released the results of the Phase 2 first-in-human, randomized, double-masked, placebo-controlled clinical trial of OK-101 for DED. The trial was conducted at 6 sites across the US and enrolled 240 subjects with DED dosed twice-daily. 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). Duration of treatment was 14 weeks with a 2-week run-in period on placebo. Significance was achieved in multiple end points for DED, including tolerability.
“We are pleased to gain IND clearance for OK-101 to treat NCP as a second important disease target for the company,” Gary S. Jacob, PhD, CEO of OKYO, said in a press release. “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.”