Cloudbreak Pharma's Eye Drop Shows Promise in Phase 2 Trial for Vascularized Pinguecula

Cloudbreak Pharmaceuticals has announced positive results from a phase 2 clinical trial of its investigational eye drop, CBT-004, for the treatment of vascularized pinguecula. The promising outcomes position the company to advance the candidate towards phase 3 studies and engage in discussions with the FDA regarding the path to approval.

Trial Results Demonstrate Efficacy and Safety

The phase 2 trial, which enrolled 88 adults with vascularized pinguecula and associated ocular redness, met its primary endpoint. Patients received one of two doses of CBT-004 or a placebo for 28 days. Key findings include:

• Significant improvements in conjunctival hyperemia (redness) were observed with both CBT-004 doses compared to the control group.
• The higher dose of CBT-004 showed significant improvements from Day 7 onward, as assessed by an independent reading center using digital imaging.
• Statistically significant improvements were reported in patient-reported symptoms, including burning, stinging, itching, foreign body sensation, eye discomfort, and pain.
• No treatment-related adverse events were reported, with most adverse events being mild to moderate in nature.
• No clinically meaningful changes in visual acuity or intraocular pressure were observed.

Mechanism of Action and Potential Market Impact

CBT-004 targets vascular endothelial and platelet-derived growth factor receptors (VEGFRs/PDGFRs), aiming to reduce abnormal vascularity associated with late-stage pinguecula. This condition is characterized by the formation of yellowish, elevated tissue on the eye, which can become inflamed and cause discomfort.

If approved, CBT-004 would be the first drug specifically indicated for vascularized pinguecula. Current treatments are limited to lubricating eye drops and off-label use of nonsteroidal anti-inflammatory drugs or steroid eye drops. Cloudbreak believes there is a significant unmet need for safer and more effective treatments in this area.

Next Steps and Regulatory Strategy

Cloudbreak Pharma is planning to provide updates on the design and timing of its phase 3 trial in the coming months. The company intends to pursue approval in the United States through the 505(b)(2) regulatory pathway, leveraging existing data on Pfizer's oral VEGFR inhibitor, Inlyta, to support their filing.

This development comes as Cloudbreak recently listed its shares in Hong Kong, with the majority of its IPO proceeds earmarked for its core products, CBT-001 and CBT-009, targeting pterygium and juvenile myopia, respectively. CBT-004 is part of a broader portfolio of assets under development at Cloudbreak, showcasing the company's commitment to addressing various ophthalmic conditions.