Kazia Therapeutics Ltd · NASDAQ:KZIA
Kazia Therapeutics — Investor Presentation (January 2025)
43 slides from Kazia Therapeutics's investor presentation, January 2025
Slide 1: A Diversified Oncology Drug Development Company
A Diversified Oncology Drug Development Company Kazia Corporate Overview January 2025 NASDAQ: KZIA | X: @KaziaTx
Slide 2: Forward Looking Statements
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 descr
Slide 3: Company Overview
Company Overview A late-clinical-stage oncology drug development company Corporate Highlights Paxalisib EVT801 Licensing-driven business model focused on high quality, differentiated clinical- stage assets sourced from Genentech Brain-penetrant pan-PI3K / mTOR inhibitor Selective VEGFR3 inhibitor (P
Slide 4: Pipeline – Two Differentiated Assets
Pipeline – Two Differentiated Assets CY2025 positive clinical data updates driving strong interest in oncology community Paxalisib licensed from: Investigational, small molecule, potent, brain-penetrant inhibitor of PI3K / mTOR Preclinical PHASE 1 PHASE 2 PHASE 3 MARKET Collaborators / Sponsors Anti
Slide 5: Paxalisib Mechanism of Action
Paxalisib Mechanism of Action Only brain-penetrant drug in development within the dual PI3K/mTOR inhibitor class 1 The PI3K pathway is activated in 2 Five PI3K inhibitors have already 3 Paxalisib is the only brain-penetrant dual many forms of cancer been approved by FDA PI3K/mTOR inhibitor in develo
Slide 6: Paxalisib – Development History
Paxalisib – Development History Growing Body of Clinical Evidence Demonstrating Activity in GBM 2012-2015 February 2018 August 2019 7 January2021 1 August2022 Genentech Phase 1 clinical GDC-0084 awarded Orphan GDC-0084 becomes ‘paxalisib’ GBM AGILE Kazia provides progress study in 47 patients with D
Slide 7: Glioblastoma Background & Market potential
Glioblastoma Background & Market potential 6
Slide 8: Glioblastoma Overview
Glioblastoma Overview The most aggressive malignant brain cancer Lung Any age, No clear No clear but most improvement in cause common in prognosis for Breast or strong risk factors 60s 20 years 14 million cancer cases per annum Colon 3-4 months Five-year survival Prostate 3 – 5% Stomach Survival, (b
Slide 9: Primary Market Research Outcomes
Primary Market Research Outcomes Physicians indicated a 2-month minimum and 12-month optimum increase in efficacy for newly diagnosed unmethylated GBM treatments, but adoption would be high regardless Key Takeaway • Due to the high unmet need for a more efficacious therapy for Newly Diagnosed Unmeth
Slide 10: GBM AGILE study data – Primary and secondary analysis
GBM AGILE study data – Primary and secondary analysis 9
Slide 11: Randomization and GBM-Agile
Paxalisib and GBM-Agile International, multi-center, adaptive, phase 2/3 study evaluating promising therapeutics in patients with glioblastoma Key Points Regorafenib Completed Negative outco me Bayer (Sep 2022) • A ‘platform study’, sponsored by GCAR run independently of Paxalisib 3 Final Top Line D
Slide 12: Paxalisib & GBM-Agile
Paxalisib & GBM-Agile Study schema; Paxalisib arm (n=154) enrolled Newly Diagnosed Unmethylated GBM patients (NDU) and Recurrent GBM patients Paxalisib stage Paxalisib stage 1 2 Evaluate for graduation Evaluate for futility Paxalisib Study completed enrolment stops 12 months after last patient enrol
Slide 13: Paxalisib & GBM-Agile: Concurrent vs Cumulative Control
Paxalisib & GBM-Agile Concurrent Control versus Cumulative Control Cumulative controls: Patients randomized to control from the start of the entire GBM AGILE study (June 2019) to the date the last patient was randomized to paxalisib. Cumulative control patients are censored one year after the last p
Slide 14: Summary of OS in NDU Patients: Primary and Secondary Analyses
Paxalisib and GBM-Agile Summary of OS in NDU Patients: Primary and Secondary Analyses Prespecified Secondary OS analysis Primary OS analysis Main analysis Sensitivity analysis I Sensitivity analysis II Method Bayesian piecewise Frequentist methods and Frequentist methods and Frequentist methods and
Slide 15: Treatment Emergent Adverse Events NDU Patients
Paxalisib and GBM-Agile Treatment Emergent Adverse Events (TEAE) NDU Patients Common Adverse Events Summary TEAEs reported by ≥ 20% of patients Concurrent • In the paxalisib arm, the most frequently Paxalisib SOC: reported treatment emergent adverse events Preferred Term Patients with any TEAE (N=52
Slide 16: Recap and next steps for Paxalisib in glioblastoma
Recap and next steps for Paxalisib in glioblastoma 15
Slide 17: Paxalisib in Glioblastoma Phase 2 Clinical Study
Paxalisib in Glioblastoma Phase 2 Clinical Study Encouraging median OS (mOS) in Newly Diagnosed Unmethylated GBM patients Overall Survival (OS) (n=30) Median OS: 15.7 months (11.1-19.1) Historical mOS for existing therapy: 12.7 months (Hegi et al. 2005) Note: Figures for existing therapy are for tem
Slide 18: Paxalisib Phase 2: Safety Profile
Paxalisib in Glioblastoma Phase 2 Clinical Study Encouraging safety profile Number of Patients at Any Dose (n=30) Experiencing AEs ‘Possibly’ or ‘Likely’ Related to Paxalisib (affecting ≥10% of patient s) Term Gr 1 Gr 2 Gr 3 Gr 4 Total (%) Fatigue 3 13 2 18 (60%) Stomatitis 4 7 3 14 (47%) Decreased
Slide 19: Paxalisib in Glioblastoma: Consistent Median OS
Paxalisib in Glioblastoma Consistent median Overall Survival data in two studies of NDU glioblastoma patients Compelling Paxalisib data in NDU patients when Standard of Care data GBM AGILE study (left) and compared to SOC STUPP historical controls (right) in NDU patients Paxalisib in Paxalisib in Co
Slide 20: Paxalisib in NDU GBM Next Steps
Paxalisib in Newly Diagnosed Unmethylated GBM Next Steps FDA Type C meeting was held in December 2024 to discuss next steps with key highlights of the discussion below: • The FDA's current position is that data on Overall Survival would generally not be appropriate for accelerated approval but could
Slide 21: Childhood Brain Cancers
Childhood Brain Cancers 20
Slide 22: Paxalisib in Childhood Brain Cancer
Paxalisib in Childhood Brain Cancer High unmet need especially in patients with diffuse midline gliomas (DMGs) 1 Brain cancer is the most common 2 Brain cancer represents about one third 3 Prognosis of childhood brain cancer, malignancy of childhood of childhood cancer deaths especially DMGs, has im
Slide 23: Summary of Paxalisib in Childhood Brain Cancer
Summary of Paxalisib in Childhood Brain Cancer Kazia is actively pursuing three forms of childhood brain cancer Diffuse Midline Gliomas (DMG, Atypical Teratoid / Advanced Childhood Cancer DIPG) Rhabdoid Tumors (AT/RT) (PI3K/mTOR activated) Positive preclinical data as Preclinical Positive preclinica
Slide 24: Paxalisib in Diffuse Midline Gliomas
Paxalisib in Diffuse Midline Gliomas Follow-up Phase 2 data presented at ISPNO 2024 Annual Meeting In spite of research that has helped improve treatment for DIPG patients, the prognosis remains poor– with the median survival range being from 8-11 months1 • 68 patients with biopsy-proven DMG were en
Slide 25: Brain Metastases
Brain Metastases 24
Slide 26: Paxalisib in Brain Metastasis
Paxalisib in Brain Metastasis MSKCC-sponsored Phase 1 trial’s interim analysis showed encouraging clinical activity of paxalisib in combination with radiation therapy (NCT04192981) 12-13 August 2022 July 2023 February 2024 Data from first stage presented at 2022 Fast Track Designation granted by US
Slide 27: Paxalisib in Brain Metastasis: Interim Analysis
Paxalisib in Brain Metastasis MSKCC-sponsored Phase 1 trial’s interim analysis presented at 2024 ASTRO* & Society of Neuro-oncology meetings showed encouraging clinical activity of paxalisib in combination with radiation therapy (NCT04192981) Robust response signal seen for concurrent paxalisib and
Slide 28: Other Solid Tumors: Triple Negative Breast Cancer
Other Solid Tumors Triple Negative Breast Cancer 27
Slide 29: Triple Negative Breast Cancer Treatment Landscape
Triple Negative Breast Cancer Treatment Landscape Projected TNBC market to exceed $1.5 Billion by 2030 2.3 million1 American Cancer Triple negative Society says breast cancer TNBC market growth forecast2 Cases / year nearly Breast cancer TNBC USD $1.5b most commonly 300,000 most aggressive diagnosed
Slide 30: Paxalisib in Triple Negative Breast Cancer
Paxalisib in Triple Negative Breast Cancer QIMR Berghofer Medical Institute collaboration “In treatment-resistant pre-clinical models of breast cancer, paxalisib (4T1 mouse model, TNBC1) has shown encouraging results in inhibiting both the primary tumor burden and metastasis by reinvigorating the im
Slide 31: Current challenges with immunotherapy in advanced breast cancer
Current challenges with immunotherapy in advanced breast cancer Cancer Stem Cells and a functioning immune system play a critical role in recurrence and metastatic spread of cancers Potential Solution: Targeting BOTH PI3K & mTOR inhibit critical cancer inflammation switch signature; induces “viral m
Slide 32: Pre-clinical studies combining Paxalisib with checkpoint or PARP inhibitor
Pre-clinical studies combining Paxalisib with either checkpoint inhibitor or PARP inhibitor resulted in highly consistent and statistically significant signals of efficacy 4T1 mouse model: • Standard model for TNBC • Immunotherapy resistant model • Highly tumorigenic and invasive Checkpoint Inhibito
Slide 33: Kazia-sponsored Clinical Study Overview
Kazia-sponsored Clinical Study Overview KZA-0084-ABC001 Phase 1b, multi-center, open-label, randomized study to evaluate the safety, tolerability, and clinical activity of combining paxalisib with olaparib or pembrolizumab/chemotherapy in approximately 24 patients with advanced breast cancer Primary
Slide 34: Kazia-sponsored Clinical Study Overview: Study Arms
Kazia-sponsored Clinical Study Overview KZA-0084-ABC001 Arm B: Paxalisib plus Arm A: Paxalisib plus Olaparib Pembrolizumab/chemotherapy 28 Day Cycle 21 Day Cycle 1 Participant disease characteristics: Participant disease characteristics: • HER2-negative stage IV (metastatic) breast cancer • Recurren
Slide 35: EVT801
EVT801 34
Slide 36: EVT801 is a highly selective VEGFR3 inhibitor
EVT801 is a highly selective VEGFR3 inhibitor, primarily inhibiting lymphangiogenesis (formation of new lymphatic vessels) Highly selective for VEGFR3 Expected to minimise off-target toxicities Oral Presentation Administered by mouth once or twice daily Strong IP Protection Inhibits lymphangiogenesi
Slide 37: EVT801 Mechanism of Action
EVT801 Mechanism of Action By targeting VEGFR3 pos tumor blood vessels, EVT801 may induce tumor blood vessel normalization, reduce hypoxia, and improve CD8 T-cells infiltration Schematic overview based on pre-clinical data EVT801 activity on tumor microenvironment Tumor metastasis Normalization of t
Slide 38: EVT801: Phase 1 dose-finding trial
EVT801: Phase 1 dose-finding trial; KZA 0801-101 (NCT05114668) Staged development in patients with advanced cancer l a i t n e d i f n Phase 1 study in advanced cancer patients completed STAGE 1 • Primary objective of stage one of the study was successfully met: Monotherapy dose escalation o MTD has
Slide 39: EVT801 Key Points
EVT801 Key Points 1 Well-understood mechanism (anti-angiogenesis) with unique differentiating feature (high VEGFR3 selectivity) 2 Strong preclinical data package, with observed activity in multiple tumours and favourable toxicology Potential for combination use with immuno-oncology therapies 3 Phase
Slide 40: 2024 Corporate Update
2024 Corporate Update 39
Slide 41: Paxalisib Licensing and Collaborations
Paxalisib Licensing and Collaborations Opportunistic partnering and strategic collaborations continue to add value Licensing Key Collaborations Summary To develop, manufacture and commercialize Paxalisib as a To develop and commercialize Cutting edge preclinical program to evaluate Paxalisib in pote
Slide 42: Kazia Therapeutics: 2025 Corporate Focus
Kazia Therapeutics: 2025 Corporate Focus Objectives for value creation • FDA meeting in Dec2024 confirmed standard approval pathway with single pivotal registrational study Progress paxalisib in NDU GBM patients glioblastoma program • Finalize protocol, assess costs/timelines and select strategic CR
Slide 43: www.kaziatherapeutics.com info@kaziatherapeutics.com
www.kaziatherapeutics.com info@kaziatherapeutics.com
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