Okyo Pharma’s first investigational new drug (IND) application for OK-101 has been cleared by the U.S. Food and Drug Administration (FDA), marking a pivotal moment in the development of potential therapies for neuropathic corneal pain.
Initially proposed as an open-label clinical trial, the study's design has been refined following constructive feedback from the FDA. The phase 2 trial is now set to be double-masked, randomized, and placebo-controlled over a 12-week period. It will involve comparing OK-101 with a placebo in patients suffering from NCP.
A total of 54 patients will participate, with NCP diagnosis confirmed through confocal microscopy. The primary endpoint of the trial will be assessed using visual analog scale (VAS) pain relief scores. These protocol adjustments are aimed at ensuring a statistically sound evaluation of OK-101's efficacy in alleviating NCP symptoms, as per Okyo's statement. The trial is slated to commence in the second quarter of 2024.
Led by Dr. Pedram Hamrah, MD, of Tufts Medical Center, the OK-101 trial will be conducted as a single-center study. Dr. Hamrah, a distinguished ophthalmologist and clinician-scientist, serves as 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. Renowned for his expertise in NCP, Dr. Hamrah is not only the Principal Investigator for the OK-101 trial but also a co-inventor of the patented OK-101 formulation. Additionally, he is a valued member of Okyo's Scientific Advisory Board, contributing his insights to the company's research endeavors in ocular health.
“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.”