Phase 2 data and FDA-aligned Phase 3 design in hand for neuropathic corneal pain; approval rests on NEPTUNE trial replicating the Phase 2 effect in a single pivotal study

OKYO Pharma is a clinical-stage company developing urcosimod, a targeted drug for neuropathic corneal pain, which is not yet approved.

Urcosimod, a small molecule targeting neuropathic corneal pain, a debilitating orphan-like condition with no approved treatment, produced statistically significant pain reduction in a Phase 2 trial and earned FDA endorsement of a single-trial registration pathway. The thesis resolves on whether NEPTUNE, enrolling ~111 patients globally, replicates the Phase 2 pain signal with sufficient magnitude to support approval on single-arm-adjacent, placebo-controlled but small-trial data. The primary risk is that a 111-patient single pivotal study leaves limited margin for effect-size attenuation, and any FDA concern about endpoint generalizability could require additional evidence.