Fresh off of submitting an FDA request for Fast Track designation earlier this month, OKYO Pharma Limited announced that positive data on its neuropathic corneal pain (NCP) candidate indicates its long-term stability.
Catch me up to speed on this asset.
As OKYO’s lead investigational therapeutic, urcosimod (formerly known as OK-101) is a non-steroidal anti-inflammatory and non-opioid analgesic.
A more technical description: Urcosimod is a lipid-conjugated chemerin peptide antagonist of the ChemR23 G-protein-coupled receptor (GPCR).
- Take note: Among its other purposes, the ChemR23 G-protein plays a critical role in managing the body’s inflammation and immune responses and is encoded by the chemerin chemokine-like receptor 1 (CMKLR1) gene.
And how does it work?
Urcosimod utilizes OKYO’s proprietary membrane-anchored-peptide (MAP) platform, which enables it to produce a novel long-acting drug candidate.
Its application: Through topical administration, the asset is formulated to prevent washout via a lipid “anchor” built directly into the drug molecule.
- The intent: To increase the amount of time it can reside within the ocular environment.
Is it under evaluation for more than NCP?
Indeed (check out the pipeline details here):
- Allergic conjunctivitis
- Dry eye disease (DED; its original indication)
- Uveitis
Got it. So where is it at in the regulatory process?
Aside from the recent Fast Track designation submission—see here for the significance of this—the FDA already accepted:
- Urcosimod’s NCP-indicated investigational new drug (IND) application in February 2024
- Urcosimod’s IND app for its DED indication in December 2022
Now to this positive data.
In a nutshell: OKYO stated that daily administration of urcosimod via single-use ampoules “has been shown to be stable for over 2.5 years” and minimizes the risk for contamination.
The basis for this conclusion: “Recently completed GMP (Good Manufacturing Practice) stability assay testing of single-use ampoules containing urcosimod in ophthalmic solution, stored under refrigerated conditions,” according to CSO Raj Patil, PhD.
- And the supporting data: Two varying doses of urcosimod demonstrated high percentages of stability:
- 0.05% urcosimod (94.8%)
- 0.1% urcosimod (97.1%)
And why is it so important for urcosimod to have long-term stability?
Because it’s an FDA-mandated requirement as part of the federal chemistry, manufacturing, and controls (CMC) part of a drug’s new drug application (NDA) submission package for marketing approval.
As CEO Gary S. Jacob, PhD, noted, a drug (such as urcosmiod) must be stable to degradation for a considerable amount of time.
Got it. Has any other stability testing been conducted on it?
That’s actually ongoing …
In fact, Dr. Patil shared that the company is currently conducting long-term stability testing on urcosimod at room temperature (as opposed to refrigerated, as was done for this most recent testing).
- So far: The 3-month results at room temperature are at or above 100%.
“We have been pleased not only with the drop comfort score observed with this drug but also with the efficacy results from an earlier trial of urcosimod to treat DED,” Dr. Patil added.
Speaking of earlier trials, what has previous data shown so far?
First: Mouse model-based clinical evaluations have demonstrated urcosimod’s ability to produce anti-inflammatory and pain-reducing activities in NCP and DED, respectively.
Next: A DED-based, first-in-human (FIH) phase 2 study on the therapeutic further supported its potential—with a few of the outcomes finding:
- Urcosimod demonstrated favorable safety and tolerability
- Statistically significant improvements noted in dry eye symptoms
See here for more (and here for other favorable safety data).
And how about for NCP?
As we previously reported, a randomized, real-world, 12-week phase 2 trial is currently ongoing among 48 patients diagnosed with NCP (see all the study details here).
The trial was initiated in October 2024 and is expected to report topline by Q3 2025.