OKYO Pharma Ltd (NASDAQ:OKYO) on Wednesday reported promising top-line results from a Phase 2 trial of its investigational drug urcosimod for neuropathic corneal pain (NCP), with 75% of patients showing greater than 80% pain reduction after 12 weeks of treatment.
Patients treated with the 0.05% formulation of urcosimod saw a statistically significant reduction in pain scores compared to baseline, as measured by the Visual Analogue Scale (VAS), with an average improvement of 5.5 points versus 2.75 in the placebo group.
The placebo-controlled trial, conducted at Tufts Medical Center in Boston, is the first clinical study targeting NCP, a chronic ocular condition with no approved therapies.
“The drug showed a dramatic reduction in VAS pain scores in patients who have particularly high VAS pain scores, indicating a more severe form of the disease,” noted Dr Pedram Hamrah, vice chair of research at Tufts and the trial’s principal investigator.
Efficacy was observed as early as Week 4 and sustained through the 12-week study. In the intent-to-treat population, 67% of patients in the urcosimod group experienced at least a 50% improvement in pain, compared to 33% in the placebo group. The effect size, measured using the Cohen-d metric, was greater than 1.2—indicating a strong treatment effect.
No serious adverse events were reported among the 18 patients enrolled.
“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,” said OKYO Pharma CEO Gary Jacob.
“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 said it plans to meet with the US Food and Drug Administration to discuss next steps for urcosimod, which has received Fast Track designation. The company also intends to explore expanded access for patients who participated in the trial and others who may qualify under FDA compassionate use guidelines.
Founder and chairman Gabriele Cerrone said the company is proud to be pioneering the first topical drug for NCP. “Our goal now is to bring this drug to the market as quickly as possible to help patients suffering from this terrible disease.”
Additional data from the study will be released following further analysis.