Investment Highlights for Kazia Therapeutics Limited (KZIA-NASDAQ)

• Overview. Kazia Therapeutics is a clinical-stage oncology company focused on high-need cancers of the central nervous system (CNS) and select hard-to-treat solid tumors. Its portfolio centers on paxalisib, a brain-penetrant PI3K/mTOR inhibitor, and EVT801, a selective VEGFR3 inhibitor.
  
  
• Paxalisib program. After FDA Type C alignment, Kazia is advancing a single pivotal registrational study in newly diagnosed MGMT-unmethylated glioblastoma (GBM). Paxalisib is also in studies for advanced breast cancer, brain metastases, diffuse midline gliomas, and primary CNS lymphoma, and it holds FDA designations including Orphan Drug and Fast Track in GBM, Fast Track with radiation for PI3K-mutant solid-tumor brain metastases, and Rare Pediatric Disease plus Orphan Drug for DIPG and AT/RT.
  
  
• EVT801 program. EVT801 is designed to limit lymphangiogenesis and improve the tumor microenvironment. Stage 1 of the first-in-human study is complete, and Phase 2 planning is underway with high-grade serous ovarian cancer (HGSOC) as the initial focus and immunotherapy combinations under evaluation.
  
  
• Near-term catalysts. Upcoming events include initial efficacy signals from the Phase 1b breast-cancer study, GBM-AGILE-related regulatory updates and pivotal-design milestones, and completion of Stage 1 analyses for EVT801; each could be value-moving and guide the development plan.
  
  
• Validated CNS profile. Paxalisib originated at Genentech and completed a successful Phase 1 there, supported by extensive preclinical data. Its reliable blood-brain-barrier penetration differentiates it within the PI3K class and supports use in primary CNS tumors and brain metastases.
  
  
• Market opportunity. GBM is a multi-billion-dollar global market, and broader primary and metastatic brain cancers are often estimated near $10 billion. Expansion into breast cancer could further increase the opportunity.
  
  
• Early breast-cancer signals. In advanced breast cancer, including triple negative breast cancer (TNBC), early data showed about a 50% reduction in circulating tumor cells by Day 21, supporting combinations such as paxalisib plus olaparib or paxalisib plus pembrolizumab with chemotherapy per study arm.
  
  
• Partnerships. Simcere holds the exclusive Greater China license for paxalisib with $11 million upfront, up to $281 million in GBM milestones, and mid-teen royalties. Sovargen holds global (ex-Greater China) rights for paxalisib in FCD-T2/TSC epilepsy with $1.5 million upfront, up to $19 million in milestones, royalties, and a share of any sub-licensing revenue.
  
  
• Operating model. Kazia uses investigator-initiated trials to extend indications, deepen key opinion leader (KOL) engagement, and manage costs; through partnerships and Investigator-Initiated Trials (IITs), more than ten paxalisib trials have advanced while maintaining disciplined spend, with most operating expenses directed to clinical programs.
  
  
• 2025 priorities. In 2025, Kazia’s priorities are to finalize the pivotal GBM protocol, assess costs and timelines, and select a strategic clinical research organization (CRO); support PNOC PK/biomarker analyses with a 2Q CY2025 update; close out the Memorial Sloan Kettering Cancer Center (MSKCC) brain-metastasis study; launch the Company-sponsored Phase 1b breast-cancer study and provide QIMR updates; complete EVT801 Stage 1 analysis, plan a Phase 2 in ovarian cancer, and advance global and regional licensing.
  
  
• Focused portfolio and listing. The Company follows a licensing-driven strategy built on differentiated clinical-stage assets sourced from Genentech (paxalisib) and Sanofi/Evotec (EVT801). Kazia now trades solely on NASDAQ (KZIA) following its ASX delisting in November 2023.
  
  
• Leadership and governance. Kazia’s leadership and governance team includes experienced management, an active Board, and a strong Scientific Advisory Board.
  
  
  • In FY2024, John Friend, MD, moved from chief medical officer (CMO) to CEO and Managing Director; after Iain Ross resigned on August 11, 2023, Dr. Friend served as Interim Chairman until January 18, 2024, when longtime board member Bryce Carmine became Chairman.
    
    
  • Kazia’s Scientific Advisory Board member, Dr. Olivero, is an expert on intracellular signaling pathways and was the team leader for Genentech’s PI3K franchise. He has a specialist interest in brain cancer and is a coinventor of paxalisib (formally GDC-0084). He led the early development of paxalisib and was responsible for bringing the drug into human trials.
    
    
• Access to capital. As of December 31, 2024, cash and equivalents were about A$3.06 million. Subsequent activity included a $2.0 million registered direct offering (January 2025), $1.0 million from the Cantrixil IP sale (March 2025), and a $2.0 million premium private placement (August 2025), providing flexibility to use both dilutive and non-dilutive sources as late-stage studies progress.
  
  
  Updated on November 21, 2025.

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Kazia Therapeutics: Paxalisib and EVT801 Oncology Pipeline Overview

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