Kazia Therapeutics Limited (“Kazia” or “the Company”) is a Sydney-based clinical-stage oncology company focused on high-need cancers of the central nervous system (CNS) and select hard-to-treat solid tumors. The portfolio includes two small-molecule programs: (1) paxalisib, a brain-penetrant inhibitor of the PI3K/AKT/mTOR pathway, a critical signaling route that helps cancer cells grow and multiply, licensed from Genentech, and (2) EVT801, a selective vascular endothelial growth factor receptor 3 (VEGFR3) inhibitor licensed from Evotec SE. Paxalisib is designed to achieve reliable drug levels in the brain, addressing a central barrier to treatment. It is advancing toward a single pivotal study in newly diagnosed, MGMT-unmethylated glioblastoma (GBM) and is being studied across additional settings, including brain metastases, select gliomas, such as Diffuse Intrinsic Pontine Glioma (DIPG), primary CNS lymphoma, and certain advanced breast cancer subtypes. Paxalisib has secured multiple FDA designations (Orphan Drug, Fast Track, Rare Pediatric Disease), enhancing regulatory engagement speed and providing potential market exclusivity, priority review, and other incentive benefits. EVT801 targets VEGFR3 to limit lymphangiogenesis—the formation of new lymphatic vessels that can enable tumor growth and metastasis—and favorably conditions the tumor microenvironment. Stage 1 of the first-in-human study is complete, with final Phase 1 readout expected in CY2025 and Phase 2 planning underway, initially in high-grade serous ovarian cancer (HGSOC) with potential immunotherapy combinations under evaluation.
* Updated on November 21, 2025.
