Kazia Therapeutics Ltd · NASDAQ:KZIA
Kazia Therapeutics — Biotech Showcase 2020
36 slides from Kazia Therapeutics's conference presentation, Jan 13, 2020
Slide 1: A company developing innovative, high-impact drugs for cancer
A company developing innovative, high-impact drugs for cancer Presentation to Biotech Showcase San Francisco, CA 13 January 2020
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains “forward-looking statements” within the meaning of the “safe-harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such statements involve known and unknown risks, uncertainties and other factors that could cause the actual
Slide 3: Corporate Overview
Corporate Overview Company Description Oncology-focused, mid-clinical-stage, small-molecule biotechnology company, headquartered in Sydney, Australia Pipeline Paxalisib – brain-penetrant PI3K / mTOR inhibitor in phase II for glioblastoma Cantrixil – cancer stem cell-targeting agent in phase I for ov
Slide 4: Investment Rationale
Investment Rationale Our lead program, paxalisib, was designed by Genentech, the world’s most successful cancer drug developer, and has completed a successful phase 1 human trial, showing it to be generally safe and providing signals of efficacy Paxalisib has reported preliminary evidence of clinica
Slide 5: Six ongoing trials across two assets; lead program covers full range of brain cancers
Six ongoing trials across two assets; lead program covers full range of brain cancers Paxalisib (GDC-0084) Cantrixil (TRX-E-001) Glioblastoma most common primary brain cancer Secondary Brain Cancer (cancer that spreads from elsewhere in the body) DIPG highly aggressive childhood brain cancer Brain M
Slide 6: Kazia has delivered all milestones to date, with multiple data read-outs expected
Kazia has delivered all milestones to date, with multiple data read-outs expected over 6-12 months Paxalisib Brain Cancer Cantrixil Ovarian Cancer Initial phase 1 data (dose escalation) presented at AACR Completion of recruitment to phase 1 (expansion cohort) ü Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 2019 2020 Comm
Slide 7: Paxalisib (GDC-0084) Phase II Glioblastoma Multiforme & Other Brain Cancers
Paxalisib (GDC-0084) Phase II Glioblastoma Multiforme & Other Brain Cancers
Slide 8: Treatment of brain cancer has improved little in recent decades
Treatment of brain cancer has improved little in recent decades, unlike other cancers Lung Cancer Brain Cancer (glioblastoma) 1990s 1990s 2000s 2000s 2010s 2010s
Slide 9: Paxalisib was designed specifically to overcome challenges of brain cancer treatment
Paxalisib was designed specifically to overcome challenges associated with brain cancer treatment Challenge Approach Tumour Heterogeneity Brain tumours exhibit a wide range of genetic aberrations Rational Target Selection PI3K pathway is affected in 85-90% of GBM cases and many brain mets Blood-Brai
Slide 10: PI3K class is well-established, but paxalisib is unique in crossing the blood-brain barrier
PI3K class is well-established, but paxalisib is unique in its ability to cross the blood-brain barrier FDA Approved July 2014 (blood cancers) [accelerated approval] FDA Approved September 2017 (blood cancers) [accelerated approval] FDA Approved October 2018 (blood cancers) [accelerated approval] ü
Slide 11: Glioblastoma (GBM) is the most common and most aggressive form of primary brain cancer
Glioblastoma (GBM) is the most common and most aggressive form of primary brain cancer Lung 14 million cancer cases per annum Breast Colon Prostate Stomach No clear cause or strong risk factors 3-4 months untreated survival 12-15 months average survival with treatment Any age, but most common in 60s
Slide 12: Temozolomide is only FDA-approved drug for GBM; it is ineffective in ~65% of cases
Temozolomide is only FDA-approved drug for GBM; it is ineffective in ~65% of cases Source: ME Hegi, A-C Diserens, T Gorlia, et al. (2005). N Engl J Med 352:997-1003 Note: Temozolomide is only approved therapy for newly-diagnosed patients; Avastin (bevacizumab) is approved for use in recurrent settin
Slide 13: A phase 1 human trial of paxalisib in GBM showed favourable safety and efficacy signals
A phase 1 human trial of paxalisib in GBM showed favourable safety and multiple efficacy signals * Taal et al., Lancet Oncology (2015): ORR and mPFS of Lomustine in 2L GBM were 2/41 (5%) and 1 months, respectively (n = 46) † Schwarzenberg J, et al. Clin Cancer Res; 20(13); 3550–9 Efficacy Signals Sa
Slide 14: Best % change from baseline of target lesions
Best % change from baseline of target lesions CR / PR:0 SD:19 (40%) PD:26 (55%) Response rate showed dose-dependent reduction in tumor growth (consistent with mode of action)
Slide 15: Patients showed a metabolic partial response on FDG-PET
7 / 27 patients (26%) showed a ‘metabolic partial response’ on FDG-PET Analysis courtesy of Professor Ben Ellingson, UCLA Brain Tumor Imaging Laboratory Pre-Dosing On GDC-0084 Pre-Dosing On GDC-0084
Slide 16: Adverse events by dose level
* FATIGUE includes FATIGUE and ASTHENIA. ** Rash includes rash and rash maculo-paular ***MUCOSITIS includes MUCOSAL INFLAMMATION and STOMATITIS Dose level 30 mg N = 7 45 mg N = 8 65 mg N = 6 Grade 1 Grade 2 Grade 3 All Grade Grade 1 Grade 2 Grade 3 All Grade Grade 1 Grade 2 Grade 3 All Grade Any Adv
Slide 17: A broad-based clinical program is underway across multiple forms of brain cancer
A broad-based clinical program is underway across multiple forms of brain cancer Glioblastoma Most common and most aggressive brain tumour Phase II NCT03522298 Glioblastoma (planned pivotal study for approval [in set-up]) Phase II / III NCT03970447 Brain Metastases Cancer that has spread from any pr
Slide 18: Paxalisib is currently in a phase 2a study in newly-diagnosed GBM
Paxalisib is currently in a phase 2a study in newly-diagnosed GBM; aim to start pivotal study in 2020 Note: timelines are estimated and subject to periodic revision based on recruitment performance and treatment effect Step 1: Dose Optimisation 6 – 24 patients 12 months 20 patients 6 months Step 2:
Slide 19: Interim analysis of the phase 2 study shows evidence of delaying tumour progression
Interim analysis of the phase 2 study shows evidence of delaying tumour progression Note: figures for existing therapy are for temozolomide, per Hegi et al. (2005); comparison between different studies is never perfectly like-for-like Progression-Free Survival (PFS) Overall Survival (OS) 8.4 months
Slide 20: The NCI-funded Alliance study in brain metastases is a multi-drug clinical trial
The NCI-funded Alliance study in brain metastases is a cutting-edge, multi-drug clinical trial Tumour is genetically profiled paxalisib entrectinib abemaciclib 150 patients Secondary brain cancer from any primary tumour ‘Precision medicine’ study in which treatment is guided by the specific genetic
Slide 21: The St Jude study in DIPG has potential for breakthrough designation
The St Jude study in DIPG has the potential for breakthrough designation and early approval Step 1: Dose Escalation 6 – 24 patients 12 patients Step 2: Expansion Cohort All patients with DIPG or high-grade gliomas (2 – 22 years of age), following radiotherapy Paxalisib given once daily, orally, as m
Slide 22: GBM AGILE is an adaptive multi-drug registrational study, with strong FDA support
GBM AGILE is an adaptive multi-drug registrational study, with strong FDA support Randomization Drug #2 Drug #3, etc… Drug #1 Common Control Arm Comparison against a common control arm reduces overall subject numbers, saving time and cost (no drug vs. drug comparison) Adaptive study design limits nu
Slide 23: GBM AGILE features a two-stage design
GBM AGILE features a two-stage design Stage 1 (Phase II) upto 150 patients 50 patients Stage 2 (Phase III) Primary endpoint of both stages is overall survival (OS); final analysis performed on all patients from both stages, compared to all control patients recruited to date Stage 1 is the primary ef
Slide 24: Brain cancer represents a significant commercial opportunity for paxalisib
Brain cancer represents a significant commercial opportunity for paxalisib with limited competition ~$1.5B+ market opportunity INDICATIVE Glioblastoma 12,500 patients p.a. in the US Brain Metastases (secondary brain cancer) Childhood Brain Cancers Other Adult Primary Brain Cancers Other Cancers with
Slide 25: Cantrixil Phase I Ovarian Cancer
Cantrixil Phase I Ovarian Cancer
Slide 26: A second program, Cantrixil, is in a phase 1 study in ovarian cancer
A second program, Cantrixil, is currently in a phase 1 study in ovarian cancer, with data reporting this year Part A: Dose Escalation 3 to 42 patients in up to 8 cohorts Seeks to establish maximum tolerated dose and understand safety profile Part B: Dose Expansion 12 patients, all at MTD Seeks to pr
Slide 27: Ovarian cancer remains a disease of high unmet medical need
Ovarian cancer remains a disease of high unmet medical need Cause of death for 1 in 100 women 10% of cases are primarily genetic in origin Lung 14 million cancer cases per annum Breast Colon Prostate Stomach >60% of patients have disease spread at diagnosis 80% of patients are over 50 years of age F
Slide 28: Cantrixil has been developed to target cancer stem cells
Cantrixil has been developed to target ‘cancer stem cells’ which are often resistant to chemotherapy Development Rationale Tumor cancer stem cells daughter cells Post- Chemo Recurrence Data courtesy of Prof Gil Mor, Yale University Ovarian cancer tumours contain a mixture of different cell types Che
Slide 29: Part A has already shown evidence of activity with several partial responders
Part A has already shown evidence of activity with several partial responders to date Source: images courtesy of Professor Jim Coward, Icon Cancer Centre October 2017 (baseline) January 2018
Slide 30: Corporate Summary
Corporate Summary
Slide 31: Recent institutional placement leaves the company well funded for next data read-outs
Recent institutional placement leaves the company well funded for next round of data read-outs Market Capitalisation ~US$ 35 million Listing NASDAQ: KZIA (1:10 ratio) ASX: KZA Share Price (US$) KZIA Current AssetsAU$ 7.5 million (30 June 2019) Institutional PlacementAU$ 4.0 million (October 2019) Fu
Slide 32: A strong team brings international experience in big pharma and early-stage biotech
A strong team brings international experience in big pharma and early-stage biotech Board Scientific Advisory Board Iain Ross Chairman Executive and Board roles in pharma and small biotech Bryce Carmine Deputy Chairman 36 years executive experience in Eli Lilly Steven Coffey Non-Executive Director C
Slide 33: Kazia has implemented a strategy of developing high-quality assets from external sources
Kazia has implemented a strategy of developing high-quality assets from external sources Partner with big pharma for late-stage development to bring to market Bring in under-valued assets from other pharmaceutical companies Conduct focused clinical trials Identify optimal patient groups Understand s
Slide 34: The partnering market for new oncology drugs is active
The partnering market for new oncology drugs is active and driven by emerging data Licensee Licensor Stage Asset(s) Deal Value (US$) Discovery Lipid kinase inhibitors $470M Preclinical Anti-CD38 antibody $275M Preclinical RAS-RAF-MAPK inhibitors $207M Clinical MEK inhibitor $700M Discovery Complemen
Slide 35: The next six months will be an exciting period for Kazia
The next six months will be an exciting period for Kazia, and a crucial inflection point for our programs Note: all milestones are indicative and subject to periodic revision in light of operational factors and emerging data February 2020 Half-Year Financial Report 1Q CY2020 Completion of patient do
Slide 36: www.kaziatherapeutics.com
www.kaziatherapeutics.com