OKYO Pharma Reports Successful FDA Meeting to Support Phase 2b/3 Development of Urcosimod for Neuropathic Corneal Pain

OKYO Pharma announced that it has held a successful Type C meeting with the FDA regarding the planned phase 2b/3 clinical trial of urcosimod for the treatment of NCP.

The meeting resulted in meaningful regulatory alignment across clinical, statistical, and manufacturing aspects of the program, significantly de-risking the path toward a potential pivotal trial and future registration. Among the key outcomes, the FDA confirmed that the proposed primary endpoint—reduction in pain as measured by the Visual Analogue Scale (VAS) at Week 12—is clinically meaningful. According to OKYO, the agency explicitly acknowledged that a ≥2-point improvement on the VAS scale represents a meaningful treatment effect for patients with neuropathic corneal pain.

The FDA also provided statistical guidance intended to enhance the robustness of the study. The agency noted that if the statistical analysis plan (SAP) is finalized prior to unmasking and the trial produces strong results, the data could potentially provide substantial evidence of effectiveness at a future end-of-phase 2b/3 meeting. In addition, the FDA endorsed the proposed study design, sample size, and powering assumptions, and agreed that the Ocular Pain Assessment Survey (OPAS) is appropriate as supportive quality-of-life evidence.

Further alignment was achieved on Chemistry, Manufacturing and Controls (CMC) strategy and key clinical elements, with no material issues raised by the agency. This alignment further supports the feasibility of advancing urcosimod into a pivotal trial and, if results are robust, toward potential registration.

“We were very pleased to achieve meaningful progress with the FDA toward alignment on urcosimod’s clinical development program,” said Raj Patil, PhD, Chief Scientific Officer of OKYO Pharma. “This alignment represents an important step forward and underscores the potential to address a significant unmet medical need in patients with neuropathic corneal pain.”

"This positive outcome reflects years of disciplined scientific and clinical execution. Moreover, the FDA’s alignment on our Phase 2b/3 strategy meaningfully de-risks the program," said Robert Dempsey, Chief Executive Officer of OKYO Pharma. "This meeting represents 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.”

As previously disclosed, urcosimod was granted the first Investigational New Drug (IND) approval to treat patients with NCP and has received Fast Track designation from the FDA. OKYO Pharma expects to initiate a 120-patient phase 2b/3 multiple-dose clinical trial of urcosimod for NCP in the first half of this year.