CBT-004

Cloudbreak Pharma on Sunday reported positive top-line data from a mid-stage study of CBT-004, an investigational ophthalmic solution to treat vascularised pinguecula, supporting its advancement to late-stage development.The study enrolled 88 adults with vascularised pinguecula and associated conjunctival hyperaemia, who were randomised to receive one of two concentrations of CBT-004 or a vehicle for 28 days. Cloudbreak noted the trial’s primary endpoint was met, with both tested concentrations of CBT-004 achieving statistically significant improvements in conjunctival hyperaemia versus vehicle at day 28, assessed via digital imaging. Furthermore, improvements with the highest CBT-004 dose emerged by day 7, sustaining through day 28.Additionally, CBT-004 also delivered statistically significant relief across five patient-reported symptoms — burning/stinging, itching, foreign body sensation, eye discomfort and pain — compared with vehicle. Principal investigator Sherif El-Harazi dubbed the “consistency and magnitude of improvement in both objective redness and patient-reported discomfort” impressive. Meanwhile, eye drop’s safety profile was favourable, with no treatment-related adverse events and absence of any meaningful changes in visual acuity or intraocular pressure.According to Cloudbreak, CBT-004 is designed to block VEGF and PDGF receptors, targeting abnormal vessel growth and inflammation while minimising ocular surface toxicity. The company plans to initiate Phase III testing and begin discussions with the FDA on a regulatory pathway.

IRVINE, Calif.--(BUSINESS WIRE)--Cloudbreak Pharma Inc. a clinical-stage ophthalmology company developing innovative therapies for ocular surface diseases, today announced positive topline results from its Phase 2 clinical trial evaluating CBT-004 ophthalmic solution in patients with vascularized pinguecula and associated conjunctival hyperemia. "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 recognize Key Phase 2 Results Primary Endpoint Achieved: Both investigated concentrations of CBT-004 demonstrated statistically significant improvements in conjunctival hyperemia compared to vehicle at Day 28, as assessed by an independent reading center using digital imaging. Rapid Onset and Sustained Efficacy: Significant improvements were observed as early as Day 7 with the highest investigated concentration CBT-004 , with benefits persisting through the 28-day treatment period. Significant Symptom Relief: Both CBT-004 concentrations showed statistically significant improvements in five common patient-reported symptoms including burning/stinging, itching, foreign body sensation, eye discomfort, and pain compared to vehicle. Excellent Safety Profile: No treatment-related adverse events were observed. Most adverse events were mild to moderate. No clinically meaningful changes in visual acuity or intraocular pressure were reported. Addressing a Significant Unmet Medical Need Vascularized pinguecula affects millions of Americans and represents a substantial unmet medical need in ophthalmology. This common, benign conjunctival growth can become problematic when it develops abnormal blood vessels and inflammation, which can lead 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. Study Design and Results The multicenter, randomized, double-masked, vehicle-controlled Phase 2 study enrolled 88 adult patients with vascularized pinguecula and associated conjunctival hyperemia. Participants were randomized to receive one of two concentrations of CBT-004, or vehicle. The primary endpoint was the change from baseline in conjunctival hyperemia at Day 28, as measured by an independent reading center using standardized digital imaging protocols. Expert Commentary "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." "As a principal investigator in this study, I was impressed by the consistency and magnitude of improvement in both objective redness and patient-reported discomfort with CBT-004," commented Dr. Sherif El-Harazi, Medical Director at Global Research Management. "The safety profile was excellent, and I believe this therapy could represent a meaningful advance for our patients with vascularized pinguecula." About CBT-004 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. Next Steps and Regulatory Strategy Based on these positive Phase 2 results, Cloudbreak Pharma Inc. 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. Market Opportunity The vascularized pinguecula market represents a significant commercial opportunity with limited therapeutic options. 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. About Cloudbreak Pharma Inc. Cloudbreak Pharma Inc. is a clinical-stage ophthalmology company dedicated to developing innovative therapies for ocular surface diseases with high unmet medical need. The company's pipeline focuses on novel treatments targeting inflammation, vascularization, and other pathological processes affecting the ocular surface. Cloudbreak is committed to improving outcomes for patients with challenging eye conditions through scientifically-driven therapeutic development. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the potential therapeutic benefits of CBT-004, the company's clinical development plans, regulatory strategy, and market opportunity. These forward-looking statements are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Factors that could cause actual results to differ include, but are not limited to, the uncertainty of clinical trial results, regulatory approval processes, competitive developments, and other risks detailed in the company's filings. The company undertakes no obligation to update these forward-looking statements except as required by law. Please note that the information contained in this press release may not be complete. For further details about Cloudbreak Pharma Inc., and our drug candidates, please refer to our company’s website (at www.cloudbreakpharma.com).