Patients with a mysterious eye pain disease may be one step closer to seeing the first approved treatment for their condition. Okyo Pharma’s urcosimod reduced pain levels by an average of 5.5 on a 10-point scale, hitting the main goal of a phase 2 trial and teeing up a meeting with the FDA about next steps.
London-based Okyo released top-line data July 16 from 18 patients with neuropathic corneal pain (NCP) enrolled in the ongoing midstage trial, all of whom received four eye drops a day of solution containing either 0.1% or 0.05% urcosimod for 12 weeks. The placebo-controlled, double-masked study is being conducted at Tufts Medical Center in Boston.
NCP leads to chronic pain and sensitivity in the eyes, face and head, and its cause is currently unknown. There are no FDA-approved treatments for the disease, and Okyo said in the release that it is the first company to conduct a clinical study for NCP.
The statistically significant 5.5-point reduction in pain came from the 0.05% urcosimod group, which Okyo’s initial data drop focuses on. The 0.1% group showed less efficacy than the 0.05% group, according to the release, which Okyo said is similar to findings from an earlier phase 2 study of the drug candidate in dry eye disease.
The placebo group’s pain levels dropped by 2.75 points from baseline by Week 12, which was also statistically significant. Using a statistical measure called "Cohen’s d" to compare the treatment and placebo effects revealed a d-value of 1.2, which means the treatment is considered to have a strong effect.
No serious adverse events were reported during the trial, according to Okyo.
“The results strengthen our conviction that this drug may be particularly effective at showing a significant reduction in pain in patients with a greater degree of neuropathic pain,” Okyo CEO Gary Jacob, Ph.D., said in the release. “Throughout the trial we were also pleased by the feedback from patients and are excited about the opportunity to provide relief to NCP patients currently living day-to-day with the life-altering effects of this relentless neuropathic pain."
Okyo is planning to run more trials of urcosimod for NCP in the future and said that multiple patients from the phase 2 study asked the FDA for continued access to the molecule through the compassionate use program.
Urcosimod, also called OK-101, is a protein designed to bind to a G protein-coupled receptor on immune cells in the eye and tame inflammation thought to be at the heart of NCP. The compound is Okyo’s sole asset and snagged an FDA fast track designation in May for NCP.
Okyo originally licensed OK-101 from On Target Therapeutics in March 2017. Okyo has also tested urcosimod in dry eye disease, with a phase 2 trial completed at the end of 2023.