Kazia Therapeutics Ltd · NASDAQ:KZIA
Kazia Therapeutics — Investor Presentation (March 2026)
55 slides from Kazia Therapeutics's investor presentation, Mar 31, 2026
Slide 1: 2026 Corporate Overview
NASDAQ: KZIA | X: @KaziaTx Beyond Inhibition. Reprogramming Cancer Control. 2026 Corporate Overview March 2026
Slide 2: Forward-looking statements
1 This presentation contains forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," "plan," "expect," "explore," "potential" or other similar words. Any statement describing Kazia's future plans,
Slide 3: Kazia Therapeutics company description
2 Kazia Therapeutics Limited is a Sydney-based clinical-stage oncology company focused on high-need cancers of the central nervous system and select aggressive solid tumors. Founded in 2016, the company is dedicated to advancing transformative therapies by harnessing next- generation science, discip
Slide 4: Our mission
3 Our mission is to redefine cancer treatment by moving beyond conventional pathway inhibition toward therapeutic reprogramming of cancer biology by targeting regulatory drivers of growth, immune escape, and relapse to deliver more durable outcomes for patients with the greatest unmet need.
Slide 5: Company Highlights
Company Highlights Clinical-stage oncology company advancing next-generation differentiated therapies designed to overcome resistance in aggressive and immunotherapy-refractory cancers Lead asset paxalisib is an oral, brain-penetrant dual PI3K / mTOR inhibitor for solid tumor indications, including
Slide 6: Our Approach: A New Approach to Cancer
Our Approach 5 A New Approach to Cancer o Cancer progression, metastasis, and treatment resistance are increasingly understood not as single-gene problems, but as failures of regulatory control. o Transcriptional programs, chromatin states, and immune checkpoints adapt dynamically to evade therapy.
Slide 7: Complementary Layers of Cancer Control
Complementary Layers of Cancer Control Paxalisib Brain-penetrant dual PI3K / mTOR inhibitor with epigenetic and immunomodulatory activity PD-L1 Protein Degrader Platform First-in-class optimized PD-L1 protein degrader Epigenetic Pathway Modulation – Inhibits PI3K-driven epigenetic regulators – Repro
Slide 8: Pipeline Overview
Pipeline Overview 7 High-value indications with strong partnering potential
Slide 9: Development Plan At a Glance
Development Plan At a Glance 8
Slide 10: Market Landscape: Growth & Opportunity
Market Landscape: Growth & Opportunity 9 1. Epigenetic Drugs Market 2. Immuno-Oncology Market 3. Targeted Protein Degradation Market o Current Market: $15–20B o Projected 2034: $80B+ o Growth Driver: Oncology applications Immuno-Oncology o Current Market: $83.34B o Projected 2035: $409.44B o Growth
Slide 11: Paxalisib
10 Paxalisib
Slide 12: Paxalisib Overview
Paxalisib Overview 11 Asset Overview Brain-penetrant, oral, once daily small molecule dual PI3K / mTOR inhibitor with epigenetic and immunomodulatory activity Origin Discovered by Genentech (GDC-0084); In-licensed by Kazia Therapeutics Development Stage Clinical Lead Indications Advanced Breast Canc
Slide 13: Paxalisib Developmental History & Milestones
Paxalisib Developmental History & Milestones 12 Early 2000's Genentech develops GDC-0084 as a potential new therapeutic for glioblastoma 2012-2015 Genentech Phase 1 clinical study in 47 patients with advanced, high-grade glioma. Study demonstrated a favorable safety profile and provided efficacy sig
Slide 14: Paxalisib vs other PI3K inhibitors
Paxalisib Paxalisib (GDC-0084) EC: endometrial, OC: ovarian cancer, ROA: route of administration 1. Layman et al 2024 2. GlobalData 3. Piqray Five PI3K inhibitors have been approved by the US FDA. Chronic lymphocytic leukemia Follicular lymphoma Follicular lymphoma Chronic lymphocytic leukemia Folli
Slide 15: Unlocking the Potential of PI3K / mTOR Pathway Inhibition
Paxalisib Unlocking the Potential of PI3K / mTOR Pathway Inhibition 14 Brain penetrant PI3K / mTOR inhibitor with epigenetic and immunomodulatory activity While traditionally classified as a kinase inhibitor, emerging clinical and translational data support a broader and more differentiated mechanis
Slide 16: Paxalisib Mechanism of Action
Paxalisib Paxalisib Mechanism of Action Paxalisib Dual Inhibition PI3K AKT mTOR Growth factor receptor Metastasis Inflammation Immune re- invigoration Cancer immune visibility Primary tumor burden + PARP Inhibition + Immunotherapy PI3K oral inhibitor clinically proven to cross the blood-brain barrie
Slide 17: Clinical & Translational Manifestations
Paxalisib Clinical & Translational Manifestations 16 In clinical settings, there is evidence that paxalisib's downstream epigenetic effects manifest as: Paxalisib is positioned as a backbone therapy within the emerging category of functional epigenetic reprogramming therapeutics. Reduction of Aggres
Slide 18: Licensing & Collaborations
Paxalisib Cutting-edge preclinical program to evaluate paxalisib in combination with immuno-therapies for advanced breast cancer • Paxalisib alone and in combination with other targeted agents is active in preclinical models of DIPG and AT / RT. • US FDA has awarded Orphan Drug Disease and Rare Pedi
Slide 19: Paxalisib Potential in Breast Cancer
Paxalisib: Advanced Breast Cancer Paxalisib Potential in Breast Cancer 1. Cancer.org 2024 2. Data Bridge Market Research Paxalisib inhibits PI3K / mTOR, a central oncogenic pathway driving cell survival and proliferation, resistance to endocrine, HER2-targeted, and cytotoxic therapies, and immune su
Slide 20: Current Treatment Landscape in Advanced Breast Cancer
Paxalisib: Advanced Breast Cancer Current Treatment Landscape 19 * Potential future regimen, depending on Keynote B49 readout (NCT04895358); CPS = Combined Positive Score; wt = wildtype; NS = not statistically significant 1. BC subtypes and distribution: (7% unknown subtype) Cancer.org; 2. Treatment
Slide 21: Consistent & Statistically Significant Signals
Paxalisib: Advanced Breast Cancer Consistent & Statistically Significant Signals 20 * αPD1: pembrolizumab or "pembro" Checkpoint Inhibitor PARP Inhibitor o Reduced tumor volume o ↓ Lung metastases o ↓ Lymph node metastases o ↓ Liver inflammation o ↓ Lung inflammation o ↓ Liver and spleen EMH o No ob
Slide 22: Paxalisib in Triple Negative Breast Cancer
Paxalisib: Advanced Breast Cancer Paxalisib in Triple Negative Breast Cancer 21 "In treatment -resistant preclinical models of breast cancer, paxalisib (4T1 mouse model, TNBC) has shown encouraging results in inhibiting both the primary tumor burden and metastasis by reinvigorating the immune system
Slide 23: Phase 1b Study Overview
Paxalisib: Advanced Breast Cancer Kazia-Sponsored Clinical Phase 1b Study Overview Multi-center, open-label, randomized trial Inclusion Criteria Endpoints Treatment Arm B • Recurrent, unresectable or metastatic TNBC • Confirmed that tumors express PD-L1 with a combined positive score (CPS) ≥ 10 • Me
Slide 24: Early Clinical Trial Epigenetic Effects
Paxalisib: Advanced Breast Cancer Early Clinical Trial Epigenetic Effects Trial ID: ACTRN12624001340527 Kazia Press Release 10 July 2025 SABCS 2025 poster presentation. First-in-human data reflects mechanistic synergy consistent with the preclinical data. Combination regimen results in >50% reductio
Slide 25: Early Evidence of Tumor Regression
24 Trial ID: ACTRN12624001340527 Kazia Press Release 27 January 2026 • Generally well tolerated at 30mg daily • Majority of adverse events assessed as unlikely related to paxalisib • All paxalisib-related events were mild to moderate to date 1 CR in mTNBC patient treated under expanded access, and 2
Slide 26: Paxalisib Potential in Glioblastoma
Paxalisib: Glioblastoma (GBM) Paxalisib Potential in Glioblastoma 25 Data on file. Market research performed 2021. Glioblastoma Multiforme Treatment Market Size Report, 2034 PI3K / mTOR signaling pathway is a central regulator of cell growth and is hyperactivated in approximately ~85% to 90% of glio
Slide 27: Paxalisib in Newly Diagnosed Unmethylated GBM
Paxalisib: Glioblastoma (GBM) Paxalisib in Newly Diagnosed Unmethylated GBM Patients 26 NDU: Newly Diagnosed Unmethylated, OS: Overall Survival Consistent median overall survival data Phase 2 (NCT03522298) GBM-AGILE (Phase 2/3) (NCT03970447) Study Type Open-label, single arm Adaptive, multi-arm rand
Slide 28: US FDA Type C Meeting
27 Paxalisib: Glioblastoma (GBM) US FDA Type C Meeting FDA position: o Data on OS would generally not be appropriate for accelerated approval but could be considered to support a traditional / standard approval o Secondary endpoint OS data from the GBM-AGILE study may be supportive and informative f
Slide 29: Paxalisib Potential in Childhood Brain Cancer
Paxalisib: Childhood Brain Cancer Paxalisib Potential in Childhood Brain Cancer 28 1. NCI: Childhood Brain and Other Nervous System Cancer 2. NIH: PI3K Pathway in Pediatric Brain Tumors 3. Strategic Market Research: Pediatric Brain Tumor Market Report Childhood Brain Cancer • The most common maligna
Slide 30: Paxalisib in Childhood Brain Cancer
Paxalisib: Childhood Brain Cancer Paxalisib in Childhood Brain Cancer 29 Kazia is actively pursuing three forms of childhood brain cancer: Diffuse Midline Gliomas (DMG, DIPG) Atypical Teratoid / Rhabdoid Tumors (AT / RT) Advanced Childhood Cancer (PI3K / mTOR activated) Preclinical Research Positive
Slide 31: Paxalisib in Diffuse Midline Gliomas
Paxalisib: Childhood Brain Cancer Paxalisib in Diffuse Midline Gliomas (DMG) 30 Trial ID: NCT05009992 1. Hargrave, D., Bartels, U. & Bouffet, E. Diffuse brainstem glioma in children: critical review of clinical trials. Lancet Oncol 7, 241-8 (2006) In spite of research that has helped improve treatme
Slide 32: Paxalisib in Brain Metastasis
Paxalisib: Brain Metastases Paxalisib in Brain Metastasis 31 Trial ID: NCT04192981 Interim analysis showed encouraging clinical activity of paxalisib in combination with radiation therapy. August 2022 July 2023 February 2024 Data from first stage presented at 2022 Annual Conference on CNS Clinical T
Slide 33: Paxalisib in Brain Metastasis: Phase 1 Results
Paxalisib: Brain Metastases Paxalisib in Brain Metastasis 32 Trial ID: NCT04192981 RANO-BM: Response assessment in neuro-oncology brain metastases, WBRT: whole-brain radiation therapy, SRS: stereotactic radiosurgery 1. Zhou et al. 2021, Kim et al. 2020 Response signal seen for concurrent paxalisib a
Slide 34: Paxalisib Summary & Next Steps
Paxalisib Summary & Next Steps 33 Summary o Brain-penetrant dual pan-PI3K / mTOR inhibitor in development (Only 2% of small-molecule drugs are brain-penetrant) o Designed to be best-in-class pan-PI3K pathway inhibitor with modest mTOR activity (dual targeting required to inhibit cancer cell prolifer
Slide 35: PD-L1 Protein Degrader Platform
34 PD-L1 Protein Degrader Platform
Slide 36: Platform Overview
PD-L1 Protein Degrader Platform Platform Overview 35 Asset Overview Lead candidate, NDL2, is an optimized bicyclic peptide PD-L1 degrader designed to target intracellular PD- L1, thereby restoring anti-tumor immune responses even in cases of immune evasion or checkpoint inhibitor resistance Origin D
Slide 37: Current Anti-PD-1 / L1 Therapy Landscape
PD-L1 Protein Degrader Platform Current Anti-PD-1 / L1 Therapy Landscape 36 Asare-Werehene et al., Front. Immunol., 2025, DOI: 10.3389/fimmu.2025.1543529; Liu et al., Biochemical Pharmacology, 2024, DOI: 10.1016/j.bcp.2023.115984 Healthy Cell PD-L1 expressed on cell surface (sPD-L1) • Binds to PD-1
Slide 38: NDL2: First-In-Class PD-L1 Degrader
PD-L1 Protein Degrader Platform NDL2: First-In-Class PD-L1 Degrader 37 Targeted Protein Degradation (TPD) therapies opened a new frontier for treating cancer by exploiting naturally occurring protein degradation pathways to disrupt critical cellular processes. NDL2 is an optimized bicyclic peptide P
Slide 39: Key Differentiator
PD-L1 Protein Degrader Platform Key Differentiator 38 Sullivan et al., PLoS ONE 2023, doi.org/10.1371/journal.pone.0286724 NDL2 Peptide Degrader of PD-L1 PD-(L)1 mAbs Primary Mechanism Targeted protein degradation via intracellular E3 ubiquitin proteasome system Block the PD-1:PD-L1 interaction at t
Slide 40: Preclinical & Translational Manifestations
PD-L1 Protein Degrader Platform Preclinical & Translational Manifestations 39 Across multiple preclinical models and patient-derived samples, NDL2 demonstrated: Data generated collectively supports intracellular PD-L1 as a mechanistically distinct and therapeutically actionable driver of immune evas
Slide 41: Efficacy in 4T1 Mouse Model
PD-L1 Protein Degrader Platform Efficacy in 4T1 Mouse Model 40 • Gold Standard model for TNBC • Immunocompetent BALB/c mice • Highly tumorigenic and invasive Intermitted dosing 0 Tumor Inoculation NDL2 IP injection Day -10 Tumors approx. 100mm3 1 2 3 4 5 6 Harvest 7 8 +/- antiPD1 IP injection 1x105
Slide 42: Efficacy in 4T1 Mouse Model (Cont.)
PD-L1 Protein Degrader Platform 41 Method: FFPE sections from Control (vehicle), anti-PD1, NDL2 or NDL2 + anti-PD1 (10mg/kg) tumors were stained using the BondRX automatic FFPE staining platform and the change in expression characterized with high resolution digital pathology. Significant difference
Slide 43: Safety in 4T1 Mouse Model
PD-L1 Protein Degrader Platform 42 Body Weight Vehicle aPD1 NDL2 NDL2+aPD1 90 100 110 120 % Change in body weight (versus Day 0) ns ns ns * ns, not significant (verus Vehicle), One-way ANOVA, Dunnett's post test • No toxicity observed for NDL2 • Normal body weight • Normal liver weight for treatment
Slide 44: Summary & Next Steps
PD-L1 Protein Degrader Platform Summary & Next Steps 43 Innovation Breakthrough o Selectively targets cancer intracellular enriched PD-L1 protein o Amongst the first therapeutics to address metastasis-seeding cancer cells resistant to traditional immunotherapy o Precisely degrades the resistance-ass
Slide 45: EVT801
EVTSOl ESSKAZIA
Slide 46: EVT801 Overview
EVT801 45 Overview Asset Overview A highly selective, potential first-in-class small molecule Vascular Endothelial Growth Factor Receptor-3 (VEGFR3) inhibitor, primarily inhibiting lymphangiogenesis Origin Discovered by Sanofi; Early development by Evotec SE; In-licensed by Kazia in 2021 Development
Slide 47: EVT801 Selectively Inhibits VEGFR3
EVT801 EVT801 Selectively Inhibits VEGFR3 46 Cell Survival (Lymph)- angiogenesis T-Cell Migration Avastin® (bevacizumab) VEGFR1 VEGFR2 VEGFR3 VEGF-A VEGF-B VEGF-C VEGF-D VEGF-E Angiogenesis Vasculogenesis T-Cell Migration Signal Modulation LUCENTIS® (ranibizumab) SUTENT® (sunitinib) NEXAVAR® (sorafe
Slide 48: A Differentiated Anti-Tumor Approach
EVT801 A Differentiated Anti-Tumor Approach 47 Tumor Killing • Direct effect on VEGFR3-expressing tumor cells (typically from endothelial origin, e.g. sarcoma) Increase in anti-tumor immune activity • No impact on T-cells viability • Increased infiltration of effector T-cells • Reduction in immunosu
Slide 49: Phase 1 Dose-Finding Trial Completed
EVT801 Phase 1 Dose-Finding Trial Completed 48 Design Monotherapy dose escalation N=26 Dose cohort 3 EVT801 monotherapy Dose cohort 2 EVT801 monotherapy Dose cohort 1 EVT801 monotherapy • 6 cohorts • Single-patient cohorts initially; expand to 3+3 when toxicity is encountered • Mixed population of a
Slide 50: EVT801 Summary & Next Steps
EVT801 Summary & Next Steps 49 Well-understood mechanism (anti-angiogenesis) with unique differentiating feature (high VEGFR3 selectivity) Strong preclinical data package, with observed activity in multiple tumors and favourable toxicology Potential for combination use with immuno-oncology therapies
Slide 51: Corporate Highlights
50 Corporate Highlights
Slide 52: Near-Term Milestones & Anticipated Catalysts
Near-Term Milestones & Anticipated Catalysts 51 Q1 2026 Complete metabolic response in stage IV TNBC expanded access patient and 2 partial responses in metastatic TNBC Phase 1b study Advancement of NDL2 PD-L1 degrader platform toward IND-enabling studies Anticipated update from FDA discussion regard
Slide 53: Investment Drivers
Investment Drivers 52 Kazia is helping to redefine oncology development by advancing a cohesive therapeutic approach aimed at reprogramming tumor biology. Resistance biology is well documented and combination strategies are actively sought by strategic partners. Our strategy is aligned with where bo
Slide 54: Leadership
Leadership 53 Management Dr. John Friend Chief Executive Officer Jeffrey J. Kraws Head of Corporate Strategy & Development Jeffrey Bonacorda VP Finance & Controller Robert Apple Chairman Steven Coffey Non-Executive Director Ebru Davidson Non-Executive Director Board of Directors Elissa Hansen Corpor
Slide 55: Contact
www.kaziatherapeutics.com [email protected]
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