Key Takeaways

  • Cloudbreak Pharma reported that the final patient has completed the 12-month assessment in its global phase 3 trial of CBT-001 for pterygium
  • Topline 12-month data are expected in the third quarter of 2026 and could support regulatory submissions 
  • CBT-001 is a multi-kinase inhibitor designed to target the underlying fibrovascular mechanisms of disease by inhibiting PDGF, VEGF and FGF signaling pathways

Cloudbreak Pharma has reached a development milestone for CBT-001, its investigational therapy for pterygium, with the final patient completing the 12-month assessment in the company's global phase 3 clinical trial.

The milestone marks completion of the primary efficacy evaluation period and sets the stage for a topline data readout expected in the third quarter of 2026. The study is evaluating CBT-001's ability to reduce pterygium lesion size and improve patient-reported ocular symptoms.

Positive results could position CBT-001 as a potential first approved drug therapy for pterygium, a common ocular surface disease characterized by fibrovascular tissue growth onto the cornea. Currently, surgery remains the primary treatment option for patients with progressive disease, despite recurrence rates that can remain significant following excision.

"Pterygium remains an area of significant unmet need in ophthalmology," said Abu Abraham, MD, Chief Medical Officer at Cloudbreak Pharma. "Our approach targets multiple receptor tyrosine kinases implicated in the disease process, including PDGF, VEGF and FGF, with the goal of modifying the underlying fibrovascular pathology rather than simply managing symptoms."

CBT-001 is derived from Cloudbreak's proprietary Multi-Kinase Inhibitor (MKI) platform and is designed to simultaneously inhibit pathways involved in angiogenesis, fibrosis and inflammation—mechanisms believed to contribute to pterygium growth and progression.

The phase 3 study enrolled 660 patients across sites in the United States, China, New Zealand, Australia and India. The multicenter, double-masked, randomized, vehicle-controlled trial is evaluating two dose levels of CBT-001 emulsion administered twice daily over 24 months. Key efficacy assessments occur at Months 3 and 12, with endpoints focused on reducing conjunctival hyperemia associated with the vascularized lesion and preventing disease progression.

Enrollment began in the United States in June 2022 and expanded internationally over the following 2 years. The company completed patient enrollment in May 2025. Topline results from the 12-month analysis are expected in the third quarter of 2026.