Kazia Therapeutics presented a corporate overview in November 2024, outlining its advancements in oncology drug development, particularly focusing on paxalisib and EVT801. The presentation underscored the company's licensing-driven business model, emphasizing high-quality, differentiated clinical-stage assets. Kazia Therapeutics delisted from the Australian Securities Exchange (ASX) in November 2023 and is now solely listed on NASDAQ (KZIA).
Paxalisib: A Brain-Penetrant PI3K/mTOR Inhibitor
Paxalisib, a small molecule inhibitor of the PI3K / Akt / mTOR pathway, is designed to penetrate the blood-brain barrier, making it a unique asset in the treatment of brain cancers. The PI3K pathway is activated in many forms of cancer, including glioblastoma (90%), breast cancer (80%), and lung cancer (75%). Five PI3K inhibitors have already been approved by the FDA, but paxalisib is the only brain-penetrant PI3K inhibitor in development.
Clinical trials are ongoing for paxalisib in brain metastases, childhood brain cancer, glioblastoma, IDH-mutant glioma, and primary CNS lymphoma. The drug has received Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the US FDA. Data from the GBM AGILE study showed clinically meaningful improvement in overall survival in paxalisib-treated, newly diagnosed unmethylated patients with glioblastoma.
EVT801: Selective VEGFR3 Inhibitor
EVT801 is a selective inhibitor of vascular endothelial growth factor receptor 3 (VEGFR3), primarily targeting lymphangiogenesis. It is designed to avoid the off-target toxicity associated with older, non-selective angiokinase inhibitors. A Phase 1 trial for advanced solid tumors has been completed, and preliminary data from an adaptive, biomarker study at cancer centers in France is expected to be presented at an upcoming 2024 meeting.
Potential indications for EVT801 include ovarian cancer, renal cell carcinoma, liver cancer, colon cancer, and sarcoma. Preclinical data suggests a strong synergy with immunotherapy, supporting potential monotherapy or combination use.
Glioblastoma Market and Unmet Needs
Glioblastoma is the most aggressive malignant brain cancer, with approximately 133,000 cases per year worldwide. The GBM treatment market was valued at US$2.5 billion in 2022. The current prognosis for glioblastoma patients remains poor, with a five-year survival rate of only 3-5%. Physicians have indicated that even a few months increase in overall survival would make a significant difference for patients, highlighting the high unmet need for more efficacious therapies.
Financial Overview
Kazia Therapeutics operates with a lean virtual pharma model, directing approximately 75% of cash flows to clinical trials. The company is also exploring opportunities for non-dilutive income through additional partnering activities.
