OKYO Pharma reports favorable phase 2 data for NCP asset

OKYO Pharma, Inc. has released new corneal image analyses from its phase 2 clinical trial evaluating urcosimod, its investigational candidate under evaluation for the treatment of neuropathic corneal pain (CNP).

Before we get to this data, let’s get a recap of urcosimod.

Previously referred to as OK-101, urcosimod is a non-steroidal anti-inflammatory and non-opioid analgesic under clinical development as OKYO’s lead asset for NCP—as well as three other potential indications.

What to know about it:

This is a lipid-conjugated chemerin peptide antagonist that targets ChemR23 G-protein-coupled receptor (GPCR).
- See how that protein targets inflammation/immune response.
Urcosimod utilizes OKYO’s proprietary membrane-anchored-peptide (MAP) platform technology (details here and here).
Its mode of delivery: Topical application

Anything else?

Earlier this year, the FDA granted Fast Track designation for urcosimod’s NCP indication.

How about its clinical performance?

For those details, we’ll refer to this randomized, double-masked, placebo-controlled phase 2 trial (our topic of conversation)—more specifically: its topline data reported in July 2025.

Check out this coverage on the 12-week study’s setup of three cohorts: two urcosimod doses and a placebo evaluated over a 12-month period.

The primary endpoint: Pain relief measured via the Visual Analog Scale (VAS) of 1-10.

So what did topline data find?

Among the highlights following 12 weeks of treatment:

75% per-protocol patients receiving 0.05% urcosimod demonstrated >80% reduction in NCP
This reduction was seen among the 0.05% urcosimod group as early as Week 4 (and sustained throughout the trial)
- Mean change of 5.25 (versus 3.0 for placebo group)
67% of intent-to-treat population patients in 0.05% urcosimod group demonstrated >50% pain improvement (versus 33% in placebo group)
- Mean reduction in pain severity: 4.2 (0.05% urcosimod group) versus 2.5 (placebo group

See here for the full rundown.

Now to the latest data rollout.

Analyzed using in vivo confocal microscopy, this newest data was compiled from nerve growth analyses (an exploratory endpoint) on the study’s 18 participants.

And in general: OKYO reported that patients in the 0.05% urcosimod group demonstrated “not only a positive reduction” in NCP, but also “favorable changes in corneal nerve structure,” which weren’t observed in the placebo group.

And what did nerve growth analyses show?

A “favorable impact on corneal nerve health.”

Case in point: 0.05% urcosimod-treated patients showed median increases in:

Total nerve fiber count: +2.0, n/0.16 mm², interquartile range (IQR) 0.54 to 3.63
Total nerve fiber length: +2.6 mm/mm², IQR 1.55 to 5.67; p = 0.057

Conversely, placebo-treated showed median decreases in:

Total nerve fiber count: –1.92, n/0.16 mm², IQR –2.79 to –0.04
Total nerve fiber length: –1.63 mm/mm², IQR –3.76 to 0.63

So … what does all of this mean?

OKYO noted that 0.05% urcosmid's demonstration of “consistent and meaningful directional improvements” across its key anatomical endpoints is indicative of its key potential for NCP.

Specifically: According to Pedram Hamrah, MD, FARVO, the study’s principal investigator, the data “suggests that urcosimod may not only reduce pain but also support the restoration of corneal nerve architecture”— a critical step toward establishing additional mechanistic insights.

And don’t forget: With no FDA-approved drug to treat NCP, urcosimod has the potential to become the first-ever treatment.

Duly noted. And what’s next for this candidate?

As OKYO shared in September 2025, the company intends to focus on advancing urcosimod to a U.S.-based multiple-ascending dose (MAD) clinical trial.

What we know about the study (so far):

The design: Randomized, placebo-controlled, double-masked study
The participants: An estimated 100 NCP patients
The purpose: To identify the optimal registration dose of urcosimod for future phase 3 trial as well as understand urcosimod’s “unique micellar drug characteristics”

Lastly: What’s the timeframe for that MAD trial?

Topline data is expected to be available in 2026 (no specific quarter has been identified as of yet).