Cloudbreak Pharma Reports Successful End-of-Phase 2 FDA Meeting For CBT-004, Paving Path For Phase 3 Trials In Pinguecula Treatment

Cloudbreak Pharma Inc., a clinical-stage ophthalmic drug company leveraging its proprietary Multi Kinase Inhibitor (MKI) discovery platform to develop first-in-class and best-in-class therapies, announced the outcome of a successful End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) regarding the late-stage development of CBT-004 for the treatment of pinguecula.

Pinguecula is a common ocular condition characterized by a growth near the cornea, often leading to inflammation and abnormal vascularization. This can create a persistent “foreign body” sensation in the eye, significantly affecting patient quality of life. Current standard-of-care treatments, such as corticosteroids, primarily address symptoms without targeting the underlying disease and present safety concerns with long-term use. 

Francis Mah, MD, Director of Cornea and External Disease at Scripps Clinic Medical Group, commented that CBT-004 has demonstrated the potential not only to relieve the most bothersome symptoms of pinguecula but also to modify the course of the disease. He expressed optimism about the possibility of incorporating CBT-004 into clinical practice if approved.

Abu Abraham, MD, Chief Medical Officer of Cloudbreak, said the successful EOP2 meeting with the FDA establishes a clear path forward for CBT-004. The meeting confirmed that the endpoints achieved in the Phase 2 study are aligned with the Agency’s expectations for pivotal studies. Dr. Abraham thanked the patients and investigators who participated in the Phase 2 program and emphasized the potential to replicate the compelling results in upcoming Phase 3 trials. He also highlighted the opportunity to address the needs of millions of patients in the U.S. while creating substantial value for the Company in this multi-billion-dollar market.

The Phase 2 study was a multicenter, randomized, double-masked, vehicle-controlled trial enrolling 88 adult patients with vascularized pinguecula and associated conjunctival hyperemia. Patients received one of two concentrations of CBT-004 or vehicle. Both CBT-004 concentrations produced statistically significant improvements in conjunctival hyperemia compared to vehicle at Day 28, the primary endpoint. 

The higher dose showed improvements as early as Day 7, with benefits persisting throughout the 28-day treatment period. Statistically significant improvements were observed in five key patient-reported symptoms, including burning/stinging, itching, foreign body sensation, eye discomfort, and pain. No treatment-related adverse events were reported, and there were no clinically meaningful changes in visual acuity or intraocular pressure.

For the Phase 3 program, the primary efficacy endpoints will focus on both signs and symptoms of pinguecula, replicating the statistically significant findings from Phase 2. Efficacy will be evaluated at three months, and safety at twelve months. Cloudbreak plans to complete remaining toxicity studies in 2026 and initiate Phase 3 trials in the first quarter of 2027. The Company continues discussions with the FDA to explore the possibility of conducting a single, sufficiently powered pivotal study in line with recent guidance from the FDA Commissioner.