FDA Approves Compassionate Use of Urcosimod (0.05%) for the Treatment of Neuropathic Corneal Pain

OKYO Pharma (Nasdaq: OKYO) announced the FDA authorized a single-patient expanded access Investigational New Drug (IND 176297) application for urcosimod 0.05% to treat a patient with severe neuropathic corneal pain (NCP).

Rhea-AI Summary

OKYO Pharma (Nasdaq: OKYO) announced the FDA authorized a single-patient expanded access Investigational New Drug (IND 176297) application for urcosimod 0.05% to treat a patient with severe neuropathic corneal pain (NCP).

The company said it will provide urcosimod under the physician-sponsored compassionate use IND and noted urcosimod previously received FDA fast track designation. OKYO expects to initiate a 120-patient Phase 2b/3 multiple-dose study of urcosimod for NCP in 2026.

Positive

• FDA authorized single-patient expanded access IND 176297
• Urcosimod holds FDA fast track designation
• Planned 120-patient Phase 2b/3 study expected in 2026

Negative

• Expanded access covers a single patient, not a pivotal trial
• No FDA-approved treatments exist for neuropathic corneal pain
• Urcosimod remains clinical-stage pending Phase 2b/3 results

Argus Jan 23 session

-6.33% close to close Open Argus

Details

News Market Reaction – OKYO

On Jan 23, the day this news came out, OKYO closed 6.33% below the previous close.

Data tracked by StockTitan Argus for the Jan 23 session.

Market Context

On Jan 23, the day this news came out, the stock closed 6.3% below the previous close. A negative re...

Analysis

On Jan 23, the day this news came out, the stock closed 6.3% below the previous close. A negative reaction despite FDA authorization for compassionate use and a planned 120-patient Phase 2b/3 trial would contrast with prior responses where clinical and strategic news often aligned with gains. Historical divergence mainly followed insider purchase disclosures rather than development updates. Any weakness could have reflected profit-taking or skepticism about single-patient expanded access, making subsequent clinical execution an important focus for reassessing sentiment.

Key Figures

Urcosimod concentration: 0.05% Compassionate-use patient: 1 patient Expanded access IND: IND 176297 +1 more

Urcosimod concentration

0.05%

Ophthalmic formulation used for neuropathic corneal pain

Compassionate-use patient

1 patient

Single-patient expanded access IND for severe neuropathic corneal pain

Expanded access IND

IND 176297

FDA-authorized single-patient expanded access application

Planned Phase 2b/3 size

120 patients

Planned multiple-dose Phase 2b/3 study for neuropathic corneal pain

Historical Context

5 past events · Latest: Jan 05

5 events

1. Jan 05

   Leadership change

   24h Move

   +7.6%

   New CEO with ophthalmology experience appointed to advance urcosimod.

2. Dec 19

   Corporate milestone

   24h Move

   +8.6%

   Nasdaq opening bell event highlighting pipeline and positive Phase 2 results.

3. Dec 15

   Insider share purchase

   24h Move

   -8.3%

   Chairman increased holdings via purchase of 24,551 ordinary shares.

4. Dec 11

   Clinical data update

   24h Move

   +6.9%

   Phase 2 trial data showed favorable corneal nerve outcomes in NCP.

5. Dec 03

   Insider share purchase

   24h Move

   -5.8%

   Chairman acquired 27,051 shares, lifting total holdings above 10.49M.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

neuropathic corneal pain, expanded access, compassionate use, investigational new drug (ind), +4 more

8 terms

neuropathic corneal pain medical

"developing investigational therapies for the treatment of neuropathic corneal pain (NCP)"

Neuropathic corneal pain is chronic, often severe eye pain caused by damage or malfunction in the tiny nerves of the cornea, where the eye surface can feel burning, stinging, or like something is in it despite little visible damage. It matters to investors because it represents a persistent, hard-to-treat medical need that drives demand for new drugs, diagnostics, devices and regulatory approvals, similar to how fixing a frayed electrical wire stops false alarm signals.

expanded access regulatory

"authorized a single-patient expanded access (compassionate use) Investigational New Drug (IND)"

Access that lets patients use an experimental drug or medical device outside of a formal clinical trial when no approved options are available. Think of it like allowing someone to test-drive a prototype car because they have no other transportation; for investors, expanded access can affect short-term demand, provide additional safety or usage information, and carry reputational or regulatory risks that may influence a company’s prospects and potential future sales.

compassionate use regulatory

"authorized a single-patient expanded access (compassionate use) Investigational New Drug"

Compassionate use is a regulated program that lets patients access an experimental drug or medical device outside a formal clinical trial when no approved treatment is available. For investors, it matters because such access can provide early real-world safety and demand signals, affect regulatory relationships and public perception, and slightly alter near-term revenue prospects or liability exposure — similar to a company offering a prototype to select customers before full approval.

investigational new drug (ind) regulatory

"expanded access (compassionate use) Investigational New Drug (IND) application submitted"

An investigational new drug (IND) is a drug or biologic that is being tested but has not yet been approved for general use; it is the application and formal status that allows a company to begin human clinical trials under regulator oversight. Investors care because an IND marks the transition from lab work to human testing — like getting a permit to run real-world experiments — which creates important milestones, costs, timelines and regulatory risk that drive a development-stage company's value.

immune-mediated inflammation medical

"a complex condition involving both immune-mediated inflammation and dysfunctional nerve signaling"

An immune-mediated inflammation is swelling and tissue reaction caused by the body's own defense system responding to a perceived threat; imagine a home security system that sometimes overreacts and damages the house while trying to stop an intruder. It matters to investors because such reactions drive drug safety profiles, trial outcomes, regulatory reviews and potential liabilities, all of which can speed up, delay or change the commercial value of therapies and medical products.

nerve signaling medical

"condition involving both immune-mediated inflammation and dysfunctional nerve signaling"

Nerve signaling is the process by which nerve cells send electrical pulses and chemical messages that let the body sense things, move, and regulate internal functions like heartbeat or digestion. For investors, products or drugs that change nerve signaling can relieve symptoms or restore function in many conditions, so progress in this area affects clinical trial success, regulatory approval prospects and potential market size — think of it like repairing a home’s wiring to restore normal operation.

fast track designation regulatory

"urcosimod was granted the first IND to treat patients with NCP and was awarded fast track designation"

Fast track designation is a status the U.S. Food and Drug Administration grants to drugs intended to treat serious conditions and address an unmet medical need. It gives the developer more frequent communication with the FDA and can allow parts of the application to be reviewed on a rolling basis, and it may pave the way to priority review or accelerated approval. It can shorten development timelines, though it does not guarantee approval.

phase 2b/3 medical

"expects to initiate a 120-patient Phase 2b/3 multiple-dose study of urcosimod"

A phase 2b/3 trial is a combined late-stage clinical study that first refines the best dose and measures how well a treatment works (phase 2b) then expands to a larger, definitive test of safety and effectiveness needed for regulatory approval (phase 3). For investors, results from a phase 2b/3 act like a dress rehearsal that turns into opening night: positive, well-controlled outcomes substantially raise the chance of approval and future sales, while failures can sharply reduce a drug’s value.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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LONDON and NEW YORK, Jan. 23, 2026 (GLOBE NEWSWIRE) -- OKYO Pharma Limited (Nasdaq: OKYO), a clinical-stage biopharmaceutical company developing investigational therapies for the treatment of neuropathic corneal pain (NCP) and for inflammatory eye diseases, today announced that the U.S. Food and Drug Administration (FDA) has authorized a single-patient expanded access (compassionate use) Investigational New Drug (IND) application submitted by Pedram Hamrah, MD, at the University of South Florida, for the use of urcosimod (0.05%) in a patient with neuropathic corneal pain.

Under the FDA-authorized expanded access (IND 176297), urcosimod is being provided by OKYO Pharma for use in a patient with severe neuropathic corneal pain who has limited therapeutic options and no FDA-approved treatments available.

“We are grateful that this FDA authorization allows us to explore the potential of urcosimod in a patient with severe neuropathic corneal pain through a physician-sponsored expanded access IND,” said Pedram Hamrah, MD, Vice Chair of Academic Medicine, Department of Ophthalmology, University of South Florida. “Neuropathic corneal pain is a complex condition involving both immune-mediated inflammation and dysfunctional nerve signaling. Urcosimod’s proposed dual mechanism of action, potentially targeting both pathways provides a strong scientific rationale for investigation in this setting.”

“Supporting physician-sponsored expanded access to urcosimod under compassionate use in the U.S. highlights the pressing global need for innovative therapies to specifically address neuropathic corneal pain,” said Robert J. Dempsey, Chief Executive Officer, OKYO Pharma. “This also reflects the potential new hope for patients battling this debilitating painful condition with no FDA approved treatment available today.”

As previously communicated, urcosimod was granted the first IND to treat patients with NCP and was awarded fast track designation by the Food and Drug Administration (FDA). The company expects to initiate a 120-patient Phase 2b/3 multiple-dose study of urcosimod to treat NCP this year.

About Neuropathic Corneal Pain (NCP)

Neuropathic corneal pain (NCP) is a chronic, often debilitating condition characterized by severe pain and sensitivity of the eyes, and in some cases the face or head. It is thought to result from damage or dysfunction of corneal sensory nerves, often in combination with inflammatory processes, and may occur in patients with a range of underlying ophthalmic conditions. There are currently no FDA-approved therapies specifically for NCP, resulting in patients being treated with limited or no success using various topical and systemic medications in an off-label manner.

About Urcosimod (formerly called OK-101)

Urcosimod is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor which is typically found on immune cells of the eye responsible for the inflammatory response, as well as on neurons and glial cells in the dorsal root ganglion. Urcosimod has been shown to produce anti-inflammatory and pain-reducing activities in a mouse model of dry eye disease and in a neuropathic corneal pain mouse model, respectively. OKYO recently announced positive data on NCP pain reduction in a randomized, placebo-controlled, double-masked Phase 2 trial involving 18 neuropathic corneal pain patients. Urcosimod showed clear statistical significance in multiple endpoints in an earlier 240-patient Phase 2, multi-center, double-masked, placebo-controlled trial to treat dry eye disease.

About OKYO Pharma

OKYO Pharma Limited (Nasdaq: OKYO) is a clinical-stage biopharmaceutical company developing innovative therapies for the treatment of neuropathic corneal pain (NCP) and inflammatory eye diseases, with ordinary shares listed for trading on the Nasdaq Capital Market. OKYO is focused on the discovery and development of novel molecules to treat neuropathic corneal pain and other ocular diseases. OKYO recently completed a successful phase 2 trial of its flagship drug urcosimod in patients with NCP and plans to initiate a 120-patient Phase 2b/3 multiple-dose study of urcosimod to treat NCP this year.

For further information, please visit www.okyopharma.com.

For further inquiries:

OKYO Pharma Ltd
Paul Spencer, Business Development, and Investor Relations
+44 (0) 207 495 2379
Email: info@okyopharma.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did OKYO (OKYO) announce about FDA expanded access on January 23, 2026?

FDA authorized a single-patient expanded access IND (IND 176297) for urcosimod 0.05% to treat a patient with severe NCP.

What is the IND number for the compassionate use authorization for urcosimod?

The FDA-authorized compassionate use IND number is 176297.

Does urcosimod have any special FDA designations for neuropathic corneal pain?

Yes, urcosimod was previously awarded FDA fast track designation for NCP.

When will OKYO start the Phase 2b/3 study of urcosimod (OKYO)?

The company expects to initiate a 120-patient Phase 2b/3 multiple-dose study in 2026.

What does the single-patient compassionate use authorization mean for urcosimod evidence?

It permits treatment of one patient under an expanded access IND but is not a substitute for clinical trial efficacy data.

How might the FDA authorization affect OKYO shareholders (OKYO)?

The authorization may signal regulatory progress and clinical interest but does not guarantee approval or commercial outcome.