Cloudbreak Pharma completes FDA end-of-phase-2 meeting for CBT-004 in pinguecula

Cloudbreak Pharma and the US Food and Drug Administration have completed an end-of-phase 2 meeting regarding the late-stage development of CBT-004 for the treatment of pinguecula. According to the company, this meeting helped to provide a “clear path forward” for CBT-004.¹

The meeting was supported by positive phase 2 results from the company’s phase 2 trial evaluating CBT-004 ophthalmic solution in patients with vascularized pinguecula and associated conjunctival hyperemia. The results were reported in July 2025.

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.^1,2

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.^1,2

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 results, both investigated concentrations of CBT-004 demonstrated statistically significant improvements in conjunctival hyperemia compared to the vehicle at day 28. Additionally, significant improvements were observed with the higher dose of CBT-004 as early as the first scheduled study visit on day 7.^1,2

Francis Mah, MD, director of cornea and external disease at Scripps Clinic Medical Group in La Jolla, CA, commented on the meeting, saying, “The current standard of care [for pinguecula], corticosteroids, addresses only symptoms and not the underlying disease and presents safety challenges with longer-term use. In a prior rigorously designed Phase 2 study, CBT-004 addressed the most bothersome symptoms of pinguecula while also demonstrating an ability to modify the trajectory of the disease. I look forward to one day incorporating it into my own practice, if approved.”¹

The company also outlined projected phase 3 study designs, including primary efficacy endpoints, including a “sign and symptom of pinguecula,” both of which were statistically significant findings in the prior phase 2 trial. The company plans to assess efficacy at 3 months and safety at 12 months in the phase 3 trial. Cloudbreak plans to complete remaining toxicity studies in 2026 and initiate the Phase 3 program in Q1 2027.¹

References:

1. Cloudbreak Pharma Announces Successful End-of-Phase-2 Meeting and Alignment with FDA on Phase 3 Path Forward for CBT-004. Published January 27, 2026. Accessed January 27, 2026. https://www.globenewswire.com/news-release/2026/01/27/3226454/0/en/Cloudbreak-Pharma-Announces-Successful-End-of-Phase-2-Meeting-and-Alignment-with-FDA-on-Phase-3-Path-Forward-for-CBT-004.html

2. Harp MD. Cloudbreak Pharma reports positive phase 2 results for CBT-004 in vascularized pinguecula. Published July 21, 2025. Accessed January 27, 2026. https://www.ophthalmologytimes.com/view/cloudbreak-pharma-reports-positive-phase-2-results-for-cbt-004-in-vascularized-pinguecula