Kazia Therapeutics Ltd · NASDAQ:KZIA
Kazia Therapeutics — Investor Presentation (April 2026)
59 slides from Kazia Therapeutics's investor presentation, Apr 27, 2026
Slide 1: 2026 Corporate Overview
NASDAQ: KZIA | X: @KaziaTx Beyond Inhibition. Reprogramming Cancer Control. 2026 Corporate Overview April 2026
Slide 2: Forward-looking statements
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: Sydney-based clinical-stage oncology company
• 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, disciplined development, and a
Slide 4: Our mission
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. 3
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, in
Slide 6: A Differentiated, Multi-Layered Approach
A Differentiated, Multi - Layered Approach Paxalisib Transcriptional Reprogramming o Alters gene expression programs that drive tumor growth and immune suppression o Exerts downstream effects on tumor transcriptional programs and immune microenvironment modulation o Activity extends beyond pathway i
Slide 7: Complementary Layers of Cancer Control
Complementary Layers of Cancer Control Paxalisib 6 Brain - penetrant dual PI3K / mTOR inhibitor with epigenetic and immunomodulatory activity PD - L1 Protein Degrader Platform First - in - class optimized PD - L1 protein degrader SETDB1 Inhibitor Platform First - in - class optimized SETDB1 inhibito
Slide 8: Pipeline Overview
Pipeline Overview High - value indications with strong partnering potential *Product candidates (not approved); IDH: Isocitrate dehydrogenase, DIPG: Diffuse Intrinsic Pontine Glioma, AT / RT: Atypical Teratoid Rhabdoid Tumor, CNS: central nervous system, TNBC: triple negative breast cancer, VEGFR3:
Slide 9: Development Plan At a Glance
Development Plan At a Glance 8
Slide 10: Market Landscape: Growth & Opportunity
Market Landscape: Growth & Opportunity 1. Epigenetic Drugs Market 2. I mmuno - Oncology Market 3. Targeted Protein Degradation Market o Current Market: $15 – 20B o Projected 2034: $80B+ o Growth Driver: Oncology applications o Current Market: $83.34B o Projected 2035 : $409.44B o Growth Driver : Inc
Slide 11: Paxalisib
Paxalisib 10
Slide 12: Paxalisib Overview
Paxalisib Overview Brain - penetrant, oral, once daily small molecule dual PI3K / mTOR inhibitor with epigenetic and immunomodulatory activity Asset Overview Discovered by Genentech (GDC - 0084); In - licensed by Kazia Therapeutics Origin Clinical Development Stage Advanced Breast Cancer (ABC, Phase
Slide 13: Paxalisib Developmental History & Milestones
Paxalisib Developmental History & Milestones Early2000’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
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. Piqray TOS - 358 (Totus Medicines) STX - 478 (Scorpion Therapeutics, acquired by Eli Lilly) OKI - 219 (OnKure Therapeutics) Zovegalisib (RLY - 2608) (Relay Therapeutics) PIKTOR
Slide 15: Pan-PI3K & mTOR Inhibition Advantage
Paxalisib Pan - PI3K & mTOR Inhibition Advantage Paxalisib is a comprehensive pathway and biology modulator, not simply a broader PI3K inhibitor. o Not built around a mutation - defined strategy – Narrows the addressable biology to tumors where a specific mutation is the dominant driver – Cancer bio
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 15 PI3K oral inhibitor clinically proven to cross the blood - brain
Slide 17: Clinical & Translational Manifestations
Paxalisib Clinical & Translational Manifestations 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 Aggressiv
Slide 18: Preclinical program, designations, 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
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
Slide 20: Current Treatment Landscape
Paxalisib: Advanced Breast Cancer Current Treatment Landscape * 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 PARP Inhibitor Checkpoint 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 observed toxicity o Reduced tumor volum
Slide 22: Kazia-Sponsored 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) ≥
Slide 23: Early Clinical Trial Epigenetic Effects
Paxalisib: Advanced Breast Cancer Early Clinical Trial Epigenetic Effects First - in - human data reflects mechanistic synergy consistent with the preclinical data. Combination regimen results in >50% reduction in CTCs Patient Profile • 61 - year - old woman with metastatic triple - negative breast
Slide 24: Early Evidence of Tumor Regression
• 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 Paxalisib: Advanced Breast Cancer Early Evidence of Tumor Regression 1 CR in mTNBC patient treated under expanded access, and
Slide 25: Paxalisib Potential in Glioblastoma
Paxalisib: Glioblastoma (GBM) Paxalisib Potential in Glioblastoma 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 gliobla
Slide 26: Glioblastoma overall survival data
Paxalisib: Glioblastoma (GBM) NDU: Newly Diagnosed Unmethylated, OS: Overall Survival GBM - AGILE (Phase 2/3) (NCT03970447 ) Phase 2 (NCT03522298 ) Adaptive, multi - arm randomized Open - label, single arm Study Type Newly diagnosed unmethylated GBM and recurrent GBM Newly diagnosed unmethylated GBM
Slide 27: US FDA Type C Meeting
Paxalisib: Glioblastoma (GBM) US FDA Type C Meeting December 2024 October 2025 FDA follow - up next steps: o Meeting anticipated for 1H 2026 o Anticipated outcome: Update from FDA discussion regarding paxalisib commercial and development path forward in GBM 26 202 6 Paxalisib in GBM commercial and d
Slide 28: Paxalisib Potential in Childhood Brain Cancer
Paxalisib: Childhood Brain Cancer Paxalisib Potential in Childhood Brain Cancer 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 malignancy
Slide 29: Paxalisib in Childhood Brain Cancer
Paxalisib: Childhood Brain Cancer Paxalisib in Childhood Brain Cancer Preclinical Research Phase 1 monotherapy clinical trial at St. Jude Children’s Research Hospital completed ( NCT03696355 ) PNOC022, Phase 2 clinical trial in combination with ONC201, ongoing ( NCT05009992 ) Orphan Drug Designation
Slide 30: Paxalisib in Diffuse Midline Gliomas
Paxalisib: Childhood Brain Cancer Paxalisib in Diffuse Midline Gliomas (DMG) Overall Survival - Cohort 2 (post RT) Median OS 15.6 months [CI 95%; 12.0, 22.4] Trial ID: NCT05009992 1. Hargrave, D., Bartels, U. & Bouffet, E. Diffuse brainstem glioma in children: critical review of clinical trials. Lan
Slide 31: Paxalisib in Brain Metastasis
Paxalisib: Brain Metastases Paxalisib in Brain Metastasis Trial ID: NCT0419298 1 Interim analysis showed encouraging clinical activity of paxalisib in combination with radiation therapy. August 2022 July 2023 February 2024 30 Data from first stage presented at 2022 Annual Conference on CNS Clinical
Slide 32: Response signal with paxalisib and brain RT
Paxalisib: Brain Metastases Paxalisib in Brain Metastasis Trial ID: NCT0419298 1 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 paxalis
Slide 33: Paxalisib Summary & Next Steps
Paxalisib Summary & Next Steps 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 ce
Slide 34: PD-L1 Protein Degrader Platform
PD - L1 Protein Degrader Platform 33
Slide 35: Platform Overview
PD - L1 Protein Degrader Platform Platform 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 Asset Overview Deve
Slide 36: PD-L1 distribution in healthy and cancer cells
PD - L1 Protein Degrader Platform 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 on cell surface • Prevents autoimm
Slide 37: NDL2: First-In-Class PD-L1 Degrader
PD - L1 Protein Degrader Platform Targeted Protein Degradation (TPD) therapies opened a new frontier for treating cancer by exploiting naturally occurring protein degradation pathways to disrupt critical cellular processes. 36 NDL2 is an optimized bicyclic peptide PD - L1 degrader • Designed to targ
Slide 38: NDL2 vs PD-(L)1 mAbs
PD - L1 Protein Degrader Platform Sullivan et al., PLoS ONE 2023, doi.org/10.1371/journal.pone.0286724 PD - (L)1 mAbs NDL2 Peptide Degrader of PD - L1 Block the PD - 1:PD - L1 interaction at the cell surface; Fc effector function varies by isotype / engineering Targeted protein degradation via intra
Slide 39: Preclinical & Translational Manifestations
PD - L1 Protein Degrader Platform Preclinical & Translational Manifestations 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
Slide 40: Efficacy in 4T1 Mouse Model
PD - L1 Protein Degrader Platform • Gold Standard model for TNBC • Immunocompetent BALB/c mice • Highly tumorigenic and invasive Intermitted dosing Tumor Inoculation NDL2 IP injection Day - 10 Tumors approx . 100 mm 3 Harvest 0 1 2 3 4 5 6 7 8 +/ - antiPD1 IP injection 1x10 5 4T1 cells right mammary
Slide 41: Efficacy in 4T1 Mouse Model (Cont.)
PD - L1 Protein Degrader Platform Method : FFPE sections from Control (vehicle), anti - PD 1 , NDL 2 or NDL 2 + anti - PD 1 ( 10 mg/kg) tumors were stained using the BondRX automatic FFPE staining platform and the change in expression characterized with high resolution digital pathology . Significan
Slide 42: Safety in 4T1 Mouse Model
PD - L1 Protein Degrader Platform Body Weight 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 duration Safety in 4T1 Mou
Slide 43: Summary & Next Steps
PD - L1 Protein Degrader Platform 42 Summary & Next Steps 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 resistan
Slide 44: SETDB1 Inhibitor Platform
SETDB1 Inhibitor Platform 43
Slide 45: Platform Overview
SETDB1 Inhibitor Platform 44 Platform Overview Lead candidate, MSETC is an optimized, bicyclic peptide designed to target SETDB1, a key epigenetic regulator of immune evasion. By inhibiting SETDB1, MSETC could restore anti - tumor immune responses, including in tumors resistant to checkpoint inhibit
Slide 46: SETDB1: Making Cancer Visible to the Immune System
SETDB1 Inhibitor Platform SETDB1: Making Cancer Visible to the Immune System Why is This a Big Opportunity ? Why Isn’t Everyone Doing This? • Access through leading research from QIMR Berghofer • First - in - class optimized approach to targeting SETDB1 • Complements Kazia’s existing focus on reprog
Slide 47: MSETC: First-In-Class Bicyclic Peptide Inhibitor
SETDB1 Inhibitor Platform MSETC: First - In - Class Bicyclic Peptide Inhibitor Target SETDB1, a key regulator of tumor immune evasion • SETDB1 is a key regulator of tumor immune evasion, driving resistance to immunotherapy in solid cancers • Overexpressed in aggressive solid tumors • Novel biology i
Slide 48: Summary & Next Steps
SETDB1 Inhibitor Platform 47 Summary & Next Steps Innovation Breakthrough o Targets SETDB1, a chromatin - level regulator that suppresses tumor immune recognition o Addresses immune evasion at its earliest control layer o Represents a novel approach to restoring immune signaling in tumors resistant
Slide 49: EVT801
EVT801 48
Slide 50: EVT801 Overview
EVT801 49 Overview A highly selective, potential first - in - class small molecule Vascular Endothelial Growth Factor Receptor - 3 (VEGFR3) inhibitor, primarily inhibiting lymphangiogenesis Asset Overview Discovered by Sanofi; Early development by Evotec SE; In - licensed by Kazia in 2021 Origin Pha
Slide 51: EVT801 Selectively Inhibits VEGFR3
EVT801 EVT801 Selectively Inhibits VEGFR3 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) NEXA
Slide 52: A Differentiated Anti-Tumor Approach
EVT801 A Differentiated Anti - Tumor Approach 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 i
Slide 53: Phase 1 Dose-Finding Trial Completed
EVT801 Phase 1 Dose - Finding Trial Completed Dose cohort 3 EVT801 monotherapy Dose cohort 2 EVT801 monotherapy Dose cohort 1 EVT801 monotherapy Design Monotherapy dose escalation N=26 • 6 cohorts • Single - patient cohorts initially; expand to 3+3 when toxicity is encountered • Mixed population of
Slide 54: EVT801 Summary & Next Steps
EVT801 Summary & Next Steps 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 Phase 1 completed demonstrating encouraging safety and tole
Slide 55: Corporate Highlights
Corporate Highlights 54
Slide 56: Near-Term Milestones & Anticipated Catalysts
Near - Term Milestones & Anticipated Catalysts 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 and MSETC SETDB1 inhibitor platforms toward IND - enabling studies Anticipated up
Slide 57: Investment Drivers
Investment Drivers 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 . • Build a portfolio that is greater tha
Slide 58: Leadership
Leadership Managemen t 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 Corp
Slide 59: www.kaziatherapeutics.com ir@kaziatherapeutics.com
www.kaziatherapeutics.com ir@kaziatherapeutics.com
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