OKYO Pharma Ltd. announced on Monday that the U.S. Food and Drug Administration has approved a single-patient expanded access Investigational New Drug application for urcosimod (0.05%). The expanded access authorization permits the investigational topical agent to be administered to a patient with severe neuropathic corneal pain at the University of South Florida.
The treating physician, Dr. Pedram Hamrah, described neuropathic corneal pain as involving both immune-mediated inflammation and dysfunctional nerve signaling. OKYO’s compound is proposed to have a dual mechanism of action that could potentially address both pathways, a rationale cited in the expanded access request.
This regulatory action underscores the degree of unmet medical need in the condition. Neuropathic corneal pain is a debilitating disorder for which there are currently no FDA-approved therapies and for which patients frequently have limited treatment options. The condition is characterized by severe eye pain resulting from damaged corneal sensory nerves.
OKYO has outlined a clinical development pathway that includes initiating a 120-patient Phase 2b/3 multiple-dose trial of urcosimod for neuropathic corneal pain in 2026. The planned study size and combined Phase 2b/3 designation indicate the company is advancing toward a potentially pivotal program, contingent on future trial conduct and outcomes.
On the corporate front, OKYO has implemented leadership changes. Robert J. Dempsey has been named chief executive officer. Gary S. Jacob, the outgoing CEO, will transition to the role of chief development officer and will remain a member of the company’s board of directors.
Market reaction and analyst perspectives were reflected in coverage following the announcement. H.C. Wainwright reiterated its Buy rating on OKYO and maintained a $7.00 price target. That stance is consistent with the broader range of analyst price targets on file for the company, which span from $5 to $13. At current market levels the stock was trading at $2.22, corresponding to a market capitalization of $83.5 million, and shares have appreciated roughly 111% over the past year.
Separately, InvestingPro analysis referenced in market commentary characterizes the stock as appearing overvalued at its then-current levels; supplemental financial details and analysis are cited as available to subscribers of that service.
The FDA authorization for single-patient expanded access permits Dr. Hamrah at the University of South Florida to administer the investigational urcosimod formulation to a patient who has limited treatment alternatives. The compassionate use approval covers only that specific, individual use under the expanded access pathway.
These developments - the compassionate use authorization, the planned 2026 Phase 2b/3 trial and the executive leadership change - represent steps in OKYO’s ongoing clinical and corporate progression as the company continues to focus resources on advancing urcosimod for neuropathic corneal pain.
Summary
The FDA has granted single-patient expanded access for OKYO Pharma’s urcosimod (0.05%) to treat severe neuropathic corneal pain at the University of South Florida. OKYO plans a 120-patient Phase 2b/3 multiple-dose trial in 2026 and has appointed Robert J. Dempsey as CEO while Gary S. Jacob moves to chief development officer. H.C. Wainwright reiterated a Buy rating with a $7.00 target; analyst price targets range from $5 to $13. The stock was trading at $2.22 with a market cap of $83.5 million and has risen 111% in the past year.