OKYO Pharma Ltd (NASDAQ:OKYO) announced that it has received positive feedback from the US Food and Drug Administration (FDA) following a Type C meeting on the design of its planned Phase 2b/3 clinical trial of its investigational therapeutic urcosimod for the treatment of neuropathic corneal pain (NCP).
The company said the FDA agreed that the proposed primary endpoint, reduction in pain measured by the Visual Analogue Scale at Week 12, is clinically meaningful, including acknowledgment that a two-point or greater improvement on the scale represents a meaningful treatment effect.
According to OKYO Pharma, the agency also provided statistical guidance intended to strengthen the trial’s design, noting that if the statistical analysis plan is finalized before unmasking and the results are strong, the data could support a finding of substantial evidence of effectiveness at a future End-of-Phase 2b/3 meeting.
The FDA also endorsed the proposed study design, sample size, and powering assumptions, and agreed that the Ocular Pain Assessment Survey could serve as appropriate supportive quality-of-life evidence.
In addition, the agency aligned with the company on its chemistry, manufacturing and controls strategy and key clinical elements, with no material issues raised, which OKYO Pharma said reduces risk as it moves toward a pivotal trial and potential registration, subject to robust study results.
OKYO Pharma said it expects to initiate a 120-patient Phase 2b/3 multiple-dose study of urcosimod in the first half of this year.
“We were very pleased to achieve meaningful progress with the FDA toward alignment on urcosimod’s clinical development program,” OKYO Pharma chief scientific officer Dr Raj Patil said in a statement.
“This alignment represents an important step forward and underscores the potential to address a significant unmet medical need in patients with neuropathic corneal pain.”
OKYO Pharma CEO Robert Dempsey added that the FDA’s alignment with the company’s Phase 2b/3 strategy “meaningfully de-risks the program.”
“The results of this meeting also represent a meaningful value-inflection point for urcosimod and, most importantly, a step forward for patients suffering from neuropathic corneal pain, where the unmet need remains profound,” Dempsey said.
Neuropathic corneal pain is a chronic condition associated with severe eye pain and sensitivity, and there are currently no FDA-approved therapies specifically indicated for the disease. Urcosimod previously received the first investigational new drug authorization to treat NCP and was granted fast track designation by the FDA.
Shares of OKYO were up around 7.8% on Wednesday morning.