Cloudbreak Pharma reports positive phase 2 results for CBT-004 in vascularized pinguecula

(Image Credit: AdobeStock/Zarina Lukash)

Cloudbreak Pharma announced positive results from its phase 2 trial evaluating CBT-004 ophthalmic solution in patients with vascularized pinguecula and associated conjunctival hyperemia.

The phase 2 trial was a multicenter, randomized, double-masked, vehicle-controlled study that enrolled 88 patients with vascularized pinguecula and associated conjunctival hyperemia. Patients were randomized to receive 1 of 2 concentrations of CBT-004 or vehicle.¹

The company describes CBT-004 as 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.¹

The primary endpoint in the trial was the change from baseline in conjunctival hyperemia at day 28, as measured by an independent reading center using standardized digital imaging protocols.

According to the company, the trial met its primary endpoint, with both investigated concentrations of CBT-004 demonstrating statistically significant improvements in conjunctival hyperemia compared to the vehicle at day 28.

Additionally, both CBT-004 concentrations showed statistically significant improvements in 5 common patient-reported symptoms, including burning/stinging, itching, foreign body sensation, eye discomfort, and pain, compared to the vehicle. Significant improvements were seen as early as day 7 with the highest investigated concentration of CBT-004, with benefits persisting through the 28-day treatment period.

John Hovanesian, MD, clinical professor of ophthalmology, commented on the trial in a press release from the company, saying, "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. The results from this trial are exciting, as they demonstrate that a targeted, nonsteroidal therapy can meaningfully improve both the clinical signs and symptoms that impact patients' quality of life."

Pinguecula is a benign, common degeneration of the conjunctiva that appears as a grey-white-yellow mass on the bulbar conjunctiva. It presents in people exposed to wind, dust, and ultraviolet light and working outdoors for a long duration and does not affect visual acuity. Though it is asymptomatic, it causes cosmetic complaints in patients. However, when inflamed, it is known as pingueculitis. When a pinguecula is inflamed, the dilated blood vessels release histamine, serotonin, bradykinin, and prostaglandins to result in the symptoms of pingueculitis. The surface of the conjunctiva overlying the pinguecula will interfere with the normal spreading of the tear film. Dry eye symptoms such as burning sensation, itching, and foreign body sensation occur.²

The company noted that based on these positive phase 2 results, it plans to advance CBT-004 into phase 3 development and initiate discussions with the US Food and Drug Administration (FDA) to establish the regulatory pathway toward potential approval. Additional phase 3 study design and timing information is expected in the coming months.¹

References:

1. Cloudbreak Pharma Inc. Announces Positive Phase 2 Results for CBT-004 in Patients with Vascularized Pinguecula. Published July 20, 2025. Accessed July 21, 2025. https://www.businesswire.com/news/home/20250717640231/en/Cloudbreak-Pharma-Inc.-Announces-Positive-Phase-2-Results-for-CBT-004-in-Patients-with-Vascularized-Pinguecula

2. Somnath A, Tripathy K. Pinguecula. [Updated 2023 Aug 25]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK558965/