Fresh off a successful end-of-phase-2 meeting with the FDA, Cloudbreak Therapeutics LLC reportedly announced phase 3 plans to advance its investigational topical ophthalmic solution for vascularized pinguecula.
The eventual intent: A new drug application (NDA) submission.
Let’s begin with Cloudbreak.
Cloudbreak is a subsidiary of Cloudbreak Pharma Inc., a clinical-stage global biotechnology company headquartered in Irvine, California.
Its focus: Developing pipeline candidates targeting anterior- and posterior-segment ocular diseases, including juvenile (pediatric) myopia, meibomian gland dysfunction (MGD), presbyopia, and (our topic of conversation) vascularized pinguecula.
- See here for a full look of assets and their respective ophthalmic disease indications.
Zero in on pinguecula.
What it is: A round, yellow(ish), and elevated tissue that manifests on the conjunctiva (adjacent to the cornea).
- How it develops: Its prevalence is known to increase with both increasing age and ultraviolet (UV) exposure
- The common symptoms: Ocular discomfort, pain, foreign body sensation, tearing, itching, and redness.
And the issue: Though methods such as surgery and prescription (steroid) eye drops are used as common treatment methods to relieve symptoms, there's currently no FDA-approved treatment.
Which brings us to this asset.
Precisely. Enter in: CBT-004,
What it is: A multi-kinase inhibitor targeting vascular endothelial growth factor receptors (VEGFs) and platelet-derived growth factor receptors (GFRs)—formulated as a topical ophthalmic eye drop
- Its purpose: To reduce abnormal conjunctival vascularity and related inflammation.
And its clinical performance thus far?
While the formulation’s preclinical data reportedly showed good effectiveness and safety in non-clinical animal disease models and animal toxicological tests, its human-based performance comes from a recent U.S.-based phase 2 clinical trial.
What we know about its design: The study evaluated three patient groups (a high-dose CBT-004 group, a low-dose CBT-004 group, and a placebo group).
Now to those findings.
In April 2025, Cloudbreak reported the following preliminary data analysis from the phase 2 study:
- CBT-004 demonstrated an excellent ocular safety profile
- The study met its primary efficacy endpoint (conjunctival hyperemia)
- Both CBT-004 treatment groups showed "statistically significant reductions in conjunctival hyperemia scores versus a placebo group
- A dose-response was observed, with the high-dose CBT-004 group performing better than the low-dose group
- Multiple secondary efficacy endpoints also showed “statistically significant differences” between both CBT-004 groups and the placebo group
- “Meaningful clinical effects” were observed
Sounds pretty promising … so what did this recent FDA meeting determine?
Essentially, the federal agency meeting confirmed CBT-004’s drug safety and efficacy, enabling its progression to phase 3 clinical evaluation.
As reports noted: “This development marks a significant step towards the commercialization of CBT-004” and could (potentially, if successful) advance Cloudbreak’s position in the ophthalmic pharma market.
Nice! Any timeframe on the phase 3 trial yet?
Details have yet to be released on those plans … so stay tuned.
And in the meantime, here’s another therapeutic development Cloudbreak is involved in:
- The company reported in June 2025 patient enrollment completion for an international, multi-center phase 3 clinical trial evaluating yet another therapeutic candidate.
- The asset: CBT-001 (formulated as an eye drop)
- Its proposed indication: Pterygium (in patients aged 12+)
See here for more details.