A phase 2 trial of Cloudbreak Pharma’s eye disease prospect CBT-004 has hit its primary endpoint, teeing the company up to advance the candidate and talk to the FDA about the route to approval.
CBT-004 targets vascular endothelial and platelet-derived growth factor receptors (VEGFRs/PDGFRs). Cloudbreak has identified the mechanism as a way to reduce abnormal vascularity associated with late-stage pinguecula, a condition characterized by the formation of yellowish, elevated tissue on the eye. If the tissue becomes inflamed, patients can experience eye pain, itching and redness.
Seeking to show whether CBT-004 can stop or reverse the progression of the lesions, Cloudbreak enrolled 88 adults with vascularized pinguecula and associated redness of the eye. Patients took one of two doses of CBT-004, delivered as an eye drop, or placebo for 28 days.
Cloudbreak reported significant improvements in conjunctival hyperemia, the medical term for redness of the conjunctiva, on both CBT-004 doses compared to the control. An independent reading center assessed conjunctival hyperemia using digital imaging. Researchers saw significant improvements on the high dose from Day 7 onward.
The biotech also reported statistically significant improvements in patient-reported symptoms including burning, stinging, itching, foreign body sensation, eye discomfort and pain compared to the control. No patients had treatment-related adverse events. Most adverse events were mild to moderate, Cloudbreak said, and no clinically meaningful changes in visual acuity or intraocular pressure were reported.
Cloudbreak plans to provide updates on the design and timing of its phase 3 trial in the coming months. The company is aiming to bring CBT-004 to the U.S. market via the 505(b)(2) regulatory pathway, using data on Pfizer’s oral VEGFR inhibitor Inlyta to support the filing.
If approved, CBT-004 will be the first drug for vascularized pinguecula. Existing treatments include lubricating eye drops and off-label use of nonsteroidal anti-inflammatory drugs or steroid eye drops. Cloudbreak believes there is a need for safer, more effective treatments.
The company recently listed (PDF) its shares in Hong Kong. Cloudbreak designated CBT-001 and CBT-009, drugs to treat the eye conditions pterygium and juvenile myopia, respectively, as its core products. Most of the IPO haul is earmarked for the core products. CBT-004 is part of a clutch of other assets in development at Cloudbreak.