OKYO Pharma Limited has received positive feedback from the FDA supporting the continued advancement of urcosimod—its lead investigational candidate for neuropathic corneal pain (NCP)—toward phase 3 clinical evaluation.
Lots to unpack here … starting with urcosimod.
What it is: A non-steroidal, anti-inflammatory, and non-opioid analgesic under clinical development for NCP (plus three other potential indications).
Some facts:
- It’s also a lipid-conjugated chemerin peptide antagonist that targets ChemR23 G-protein-coupled receptor (GPCR).
- Click here for details on how that protein targets inflammation/immune response.
- It utilizes OKYO’s proprietary membrane-anchored-peptide (MAP) platform technology.
- Its mode of delivery is via topical application.
Also worth noting: The therapeutic was granted FDA Fast Track designation for its NCP indication last year (and its investigational new drug [IND] was previously approved in 2024).
And what was the last update on its clinical progress?
That would be the latest favorable phase 2 data, which we reported on in December 2025. This dataset was a follow-up to prior topline results released in July 2025.
For a refresh: See this coverage on the 12-week, randomized, double-masked, placebo-controlled trial’s setup of three cohorts: two urcosimod doses (0.05% and 0.1%) and a placebo evaluated over a 12-month period.
- The primary endpoint: Pain relief measured via the Visual Analog Scale (VAS) of 1-10.
Give me the highlights from both data readouts.
Check out this recap of that topline data—and see here for the full rundown.
As for that December 2025 reporting: This involved corneal image analyses.
The general findings: Patients in the 0.05% urcosimod group demonstrated:
- A positive reduction in NCP
- Favorable changes in corneal nerve structure (which was not observed in the placebo group)
- A favorable impact on corneal nerve health (that data here)
So based on that, what did OKYO have to say?
We’ll do you one better—and share feedback from that phase 2 study’s principal investigator: Pedram Hamrah, MD, FARVO.
Per Dr. Hamrah, 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.
Nice! Now let’s talk about this new FDA feedback.
Important to note: The feedback followed a Type D meeting between the federal agency and OKYO.
- For context: This type of meeting is an expedited interaction with the FDA that’s strictly limited to one or two focused topics and up to three to five questions, enabling a drug developer to receive rapid guidance without the extensive review times associated with Type C meetings (which are more comprehensive in nature and conduct).
- This rundown offers an explanation on various FDA meeting types.
And what, exactly, was discussed?
As we mentioned earlier: The company’s regulatory and clinical pathway for urcosimod—specifically the FDA’s support for advancing the therapeutic into a global phase 3 pivotal trial for NCP.
- That trial will be dubbed Neuropathic Eye Pain Treatment with Urcosimod & Nerve Evaluation (NEPTUNE).
OKYO also shared that the feedback in question offered increased clarity around the company’s updated regulatory strategy as well as reduced uncertainty and further support for its plan to advance urcosimod via a “potentially streamlined clinical development pathway.”
Speaking of this trial … what do we know?
For starters: NEPTUNE is expected to be conducted across the United States and Europe.
Its design will include:
- Enrolling an estimated 111 patients (diagnosed with NCP, of course)
- Randomizing patients 2:1 to receive either urcosimod 0.05% or a placebo
And based on its discussion with the FDA, the company plans to position the planned NEPTUNE trial “as a pivotal study that could support a potential single-trial registration strategy, subject to successful study results and continued FDA review.”
Anything else?
One more piece to this: OKYO also intends to seek FDA Breakthrough Therapy Designation (BTD) for urcosimod.
To refresh: BTD is reserved for investigational drugs designed to treat a serious or life-threatening disease—and is intended to expedite its clinical development as well as future new drug application (NDA) review process.
- Check out the criteria a drug must meet to qualify.
Lastly, what’s the big-picture significance of this?
While there’s no timeframe yet as to when the NEPTUNE trial may get underway, the potential significance of urcosimod for NCP—which currently has no FDA-approved therapy—is already clear.