Okyo Pharma Limited announced plans for the next stage of clinical development of its lead drug candidate, urcosimod, to treat neuropathic corneal pain (NCP). The announcement follows the July release of topline data from Okyo's randomized, double-masked, placebo-controlled phase 2 proof-of-concept trial of urcosimod in 17 NCP patients conducted at Tufts Medical Center in Boston, with Pedram Hamrah, MD, as principal investigator.

After 12 weeks of treatment in that study, 75% of per-protocol patients receiving 0.05% urcosimod showed greater than 80% reduction in neuropathic corneal pain, as measured by visual analog scale, suggesting highly effective treatment in this patient population, Okyo stated in a press release. 

Building on these results, Okyo said it is now focused on advancing to a randomized, placebo-controlled, double-masked, multiple-ascending-dose (MAD) clinical trial designed to:

• Enroll approximately 100 NCP patients across several US clinical sites

• Identify the optimal registration dose for urcosimod for future phase 3 trials

• Develop further understanding of urcosimod’s unique micellar drug characteristics

According to Okyo, negotiations with several leading US clinical sites are underway, and the company anticipates topline data from this trial in 2026.

As part of the registration pathway, Okyo said it continues to progress toward a meeting with the US Food and Drug Administration (FDA) to discuss requirements for an approvable drug to treat NCP and to define the primary endpoint required for potential registration. Urcosimod currently holds Fast Track designation.