Cloudbreak Pharma Inc. announced positive topline results from its Phase 2 clinical trial evaluating CBT-004 ophthalmic solution in patients with vascularized pinguecula and associated conjunctival hyperemia. The preservative-free eye drop formulation achieved its primary endpoint, demonstrating statistically significant improvements in conjunctival hyperemia compared to vehicle at Day 28, as assessed by an independent reading center using digital imaging.
Phase 2 Trial Results Exceed Expectations
The multicenter, randomized, double-masked, vehicle-controlled Phase 2 study enrolled 88 adult patients with vascularized pinguecula and associated conjunctival hyperemia. Both investigated concentrations of CBT-004 met the primary endpoint, with significant improvements observed as early as Day 7 with the highest investigated concentration, and benefits persisting through the 28-day treatment period.
Beyond the primary endpoint, CBT-004 demonstrated statistically significant improvements in five common patient-reported symptoms including burning/stinging, itching, foreign body sensation, eye discomfort, and pain compared to vehicle. The treatment exhibited an excellent safety profile with no treatment-related adverse events observed, and most adverse events were mild to moderate. No clinically meaningful changes in visual acuity or intraocular pressure were reported.
Addressing Significant Unmet Medical Need
Vascularized pinguecula affects millions of Americans and represents a substantial unmet medical need in ophthalmology. This common, benign conjunctival growth becomes problematic when it develops abnormal blood vessels and inflammation, leading to persistent redness, irritation, pain, and foreign body sensation. Current management options are limited, with many patients relying on off-label corticosteroids or surgical excision, both of which carry significant limitations and potential complications.
"There is a significant unmet need for patients suffering from symptomatic pinguecula, as current therapies are largely off-label and may carry safety concerns with long-term use," said Dr. John Hovanesian, Clinical Professor of Ophthalmology and recognized key opinion leader in anterior segment disease. "The results from this trial are exciting, as they demonstrate that a targeted, non-steroidal therapy can meaningfully improve both the clinical signs and symptoms that impact patients' quality of life."
Novel Mechanism of Action
CBT-004 is a novel, preservative-free topical ophthalmic solution containing a potent and selective inhibitor of vascular endothelial growth factor (VEGF) receptors and platelet-derived growth factor (PDGF) receptors. The formulation is specifically designed to reduce abnormal blood vessel growth and inflammation associated with vascularized pinguecula while minimizing potential ocular surface toxicity through its preservative-free composition.
Dr. Sherif El-Harazi, Medical Director at Global Research Management and principal investigator in the study, commented: "I was impressed by the consistency and magnitude of improvement in both objective redness and patient-reported discomfort with CBT-004. The safety profile was excellent, and I believe this therapy could represent a meaningful advance for our patients with vascularized pinguecula."
Market Opportunity and Next Steps
The vascularized pinguecula market represents a significant commercial opportunity with limited therapeutic options, affecting over 1.1 billion patients globally according to market projections. The prevalence of pinguecula increases with age and UV exposure, affecting a substantial portion of the aging population. With no FDA-approved treatments specifically indicated for this condition, CBT-004 has the potential to become a category-defining therapy in this underserved market projected to reach $1.5 billion globally by 2033.
Based on these positive Phase 2 results, Cloudbreak Pharma plans to advance CBT-004 into Phase 3 development and initiate discussions with the U.S. Food and Drug Administration (FDA) to establish the regulatory pathway toward potential approval. The company anticipates providing updates on Phase 3 study design and timing in the coming months.
