Prescient Therapeutics Limited · ASX:PTX
Prescient Therapeutics — Biotech Showcase, San Francisco 2017
35 slides from Prescient Therapeutics's conference presentation, Jan 11, 2017
Slide 1: Two Novel Targeted Therapies Yielding a Deep Oncology Clinical Pipeline
For personal use only TWO NOVEL TARGETED THERAPIES YIELDING A DEEP ONCOLOGY CLINICAL PIPELINE Prescient Therapeutics Limited (ASX: PTX) Biotech Showcase, San Francisco January 2017
Slide 2: Disclaimer and Safe Harbor
DISCLAIMER AND SAFE HARBOR For personal use only Certain statements made in this document are forward-looking statements within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. These forward-looking statements are not historical facts b
Slide 3: Company Overview
COMPANY For personal use only OVERVIEW 3 TARGETED THERAPIES YIELDING A DEEP CLINICAL PIPELINE
Slide 4: Company Overview
COMPANY OVERVIEW For personal use only • 2 novel drug candidates (small molecules) targeting key cancer Two Clinical Stage pathways Oncology Drugs • Akt (PTX-200) and Ras (PTX-100) One of Deepest Clinical • 3 clinical trials in cancer (AML, ovarian & breast cancers) Pipelines on ASX • All under INDs
Slide 5: Deep, Clinical Stage Product Pipeline
DEEP, CLINICAL STAGE PRODUCT PIPELINE For personal use only • PTX-200 currently in three clinical trials • Focus is now on further development of PTX-100 in Ras & Rho-mutant cancers DISCOVERY SCREENING PRE-CLINICAL PHASE 1 PHASE 1B PHASE 2 PHASE 3 PTX-200 BREAST CANCER Finishing PTX-200 OVARIAN CANC
Slide 6: Corporate Snapshot
CORPORATE SNAPSHOT For personal use only KEY METRICS ASX Ticker PTX Market Capitalisation1 A$19.2 M (US$13.8 M) Total Issued Capital 211.3 M shares Cash Position A$9.3 M (US$6.7 M) Options 57.8 M Top 20 Own 52% Share Price1 A$0.09 (US$0.065) 6 month turnover1 56.7 M shares; A$6.0 M (US$4.3 M) SHARE
Slide 7: World Class Centers & Collaborations
WORLD CLASS CENTERS & COLLABORATIONS For personal use only CONNECTICUT NEW YORK FLORIDA PREVIOUS CLINICAL TRIALS CONDUCTED AT: 7 TARGETED THERAPIES YIELDING A DEEP CLINICAL PIPELINE
Slide 8: Development Team with Bench to Bedside Success
DEVELOPMENT TEAM WITH BENCH TO BEDSIDE SUCCESS For personal use only Proven success from discover and clinical development, through to FDA approvals • Professor and Chair, Department of Drug Discovery - Moffitt Cancer Center Said Sebti, PhD • Co-Program Leader, Chemical Biology and Molecular Medicin
Slide 9: Clinical Advocates Driving Our Programs
CLINICAL ADVOCATE DRIVING OUR PROGRAMS For personal use only Acute Myeloid Leukemia Breast Cancer Ovarian Cancer Jeff Lancet, M.D. Joseph Sparano, M.D. Heather Han, M.D. Robert Wenham, M.D. • Chair of the • Prof. of Medicine & • Assistant Prof. of • Section Head, Department of Obstetrics, Gynecology
Slide 10: PTX-200: Novel Akt Inhibition
PTX-200 For personal use only NOVEL AKT INHIBITION OVERCOMING KINASE PROMISCUITY & LIMITATIONS OF PREVIOUS ATTEMPTS AT AKT INHIBITION 10 TARGETED THERAPIES YIELDING A DEEP CLINICAL PIPELINE
Slide 11: Akt Is a Master Switch for Cellular Growth & Survival
AKT IS A MASTER SWITCH FOR CELLULAR GROWTH & SURVIVAL For personal use only 11 ERSAHIN, T. ET AL; MOL. BIOSYST., 2015,11, 1946-1954
Slide 12: Akt Remains an Important Drug Target
AKT REMAINS AN IMPORTANT DRUG TARGET For personal use only • Akt pathway promotes cellular survival and growth • Hyperactive Akt signaling has two deleterious effects: » Plays key role in the development of many cancers including breast, ovarian, colorectal, prostate, pancreatic and hematologic canc
Slide 13: PTX-200 (Triciribine Phosphate Monohydrate) Introduction
PTX-200 (TRICIRIBINE PHOSPHATE MONOHYDRATE) INTRODUCTION For personal use only • PTX-200 (TCN-P) is a small molecule Akt activation inhibitor that is highly selective for killing tumors with hyperactivated Akt PTX-200 (TCN-P) • Inhibits Akt without the toxicity of other attempts • Anti-proliferative
Slide 14: PTX-200: Novel Akt Inhibition via PH Domain Binding
PTX-200: NOVEL AKT INHIBITION VIA PH DOMAIN BINDING For personal use only • PTX-200 is NOT an ATP mimic/direct kinase inhibitor, and therefore avoids off target effects associated with ATP mimic inhibitors. » By binding to the PH domain, PTX-200 prevents binding to the plasma membrane, thus inhibiti
Slide 15: Acute Myeloid Leukemia Market Overview
ACUTE MYELOID LEUKEMIA MARKET OVERVIEW For personal use only • AML is a type of cancer that affects the blood and bone marrow » Patient cannot produce normal blood cells » Blood cells cannot function properly nor fight disease • Progresses very quickly & 5 year survival is a dismal 25% • More common
Slide 16: PTX-200 in AML – Executive Summary
PTX-200 IN AML – EXECUTIVE SUMMARY For personal use only • Akt is highly relevant in AML • PTX-200 address the “phenotype, not the genotype” in AML mutations • Like other recent successful strategies in AML, PTX-200 is a targeted therapy complementing a “backbone” of standard chemotherapy • PTX-200
Slide 17: Mutational Complexity of AML
MUTATIONAL COMPLEXITY OF AML For personal use only • Although AML is a mutationally complex disease, many different types of mutations (and combinations of mutations) result in hyperphosphorylation of Akt » In fact, 72% of AML patients have high p-Akt • Previous Akt drugs have failed because of the
Slide 18: Relevance of Akt in AML
RELEVANCE OF AKT IN AML • Frequent constitutive Akt activation (phosphorylation) in AML For personal use only » Constitutive phosphorylation (Ser473 and Thr308) of Akt in AML compared to normal bone marrow cells in 44 out 66 of patients (72%) • Implications for Akt as a modulator of chemotherapy res
Slide 19: PTX-200 Synergizes Cytarabine in AML Cells
PTX-200 SYNERGIZES CYTARABINE IN AML CELLS For personal use only • PTX-200 highly synergistic with the current standard of care (cytarabine) in AML cells » PTX-200 + cytarabine = much more effective effect than the simple additive effect of either compound (i.e. 1 + 1 ≥ 2) • As cytarabine is the cur
Slide 20: PTX-200: Completed Phase 1 Monotherapy in Hematologic Malignancies
PTX-200: COMPLETED PHASE 1 MONOTHERAPY IN HEMATOLOGIC MALIGNANCIES (MAINLY AML) For personal use only Patients • 32 Trial Centers • MD Anderson & Moffitt Patient • Advanced hematologic malignancies (mainly AML) Inclusion Study • To establish dosing regime and biological dose Objectives • 1 hour IV i
Slide 21: PTX-200 Reduced p-Akt in AML Blasts
PTX-200 REDUCED P-AKT IN AML BLASTS For personal use only • Phase 1 trial demonstrated that blast cells with high p-Akt from AML patients are more sensitive to PTX-200’s ability to reduce p-Akt levels » p-Akt/Akt in AML » Action of PTX-200 on Akt samples before therapy phosphorylation in AML blasts
Slide 22: Compelling Evidence for PTX-200 in AML
COMPELLING EVIDENCE FOR PTX-200 IN AML For personal use only Efficacy hypothesis PTX-200 Evidence • High p-Akt is correlated with inferior survival in AML • PTX-200 decreased pAkt in AML blasts • Inhibiting p-Akt improves response to chemo in the clinical • PTX-200 decreased pAkt in AML blasts, sugg
Slide 23: Phase 1b Trial Underway: Acute Myeloid Leukemia
PHASE 1B TRIAL UNDERWAY: ACUTE MYELOID LEUKEMIA For personal use only • PTX-200 plus cytarabine in refractory or relapsed AML • Phase 1 results very encouraging • Phase 1b/2 IND allowed by FDA • 3+3 design, single arm Jeffrey E Lancet, M.D. • Jeff Lancet at Moffitt Cancer Center leading the trial Pr
Slide 24: Phase 1b Breast Cancer Trial Almost Completed
PHASE 1B BREAST CANCER TRIAL ALMOST COMPLETED For personal use only • PTX-200 in combination with paclitaxel, followed by doxorubicin and cyclophosphamide • Patients with metastatic and locally advanced HER2- breast cancer (mostly TNBC) » Recruiting at Albert Einstein College of Medicine Montefiore
Slide 25: Phase 1b Ovarian Cancer Trial Commenced
PHASE 1B OVARIAN CANCER TRIAL COMMENCED For personal use only • Significant need for new products to treat platinum-resistant ovarian cancer • Testing PTX-200 plus carboplatin in patients with platinum resistant ovarian cancer • PTX-200 already proven overcome cisplatin resistance and synergize with
Slide 26: Immediate PTX-200 Milestones & Indicative Timing
IMMEDIATE PTX-200 MILESTONES & INDICATIVE TIMING For personal use only 1H 2017 2H 2017 Phase 1b expansion cohort Breast Complete Phase 1b cancer Phase 1b results Phase 1b dose escalation & interim analysis Ovarian cancer Complete Phase 1b Acute Dose escalation & interim analysis Myeloid Leukemia Com
Slide 27: PTX-100: First in Class, First in Man GGT-1 Inhibitor of Ras Pathway
PTX-100 For personal use only FIRST IN CLASS, FIRST IN MAN GGT-1 INHIBITOR OF RAS PATHWAY 27 TARGETED THERAPIES YIELDING A DEEP CLINICAL PIPELINE
Slide 28: Ras Pathway Is an Important but Elusive Target
RAS PATHWAY IS AN IMPORTANT BUT AN ELUSIVE TARGET For personal use only • Ras mutated in 30% of all human cancers and 90% in certain cancers » A staggering 3 million new cancers diagnosed worldwide each year with Ras mutations • Mutant Ras tumors are often unresponsive to current treatments • Patien
Slide 29: PTX-100 (Geranylgeranyl Transferase) Introduction
PTX-100 (GERANYLGERANYL TRANSFERASE) INTRODUCTION For personal use only • PTX-100 (GGTI-2418) small molecule inhibitor of the GGT-1 enzyme PTX-100 (GGTI-2418) • Geranylgeranyl transferase (GGT-1) is key activator in the Ras pathway via Ral, Rac & Rho » Overcomes failures of Farnesyltransferase inhib
Slide 30: PTX-100 Is a Highly Effective Anti Tumor Agent in Pre-Clinical Models
PTX-100 IS A HIGHLY EFFECTIVE ANTI TUMOR AGENT IN PRE-CLINICAL MODELS AND PATIENT FRESH BIOPSIES For personal use only Lung tumor1 • PTX-100 inhibits tumor growth and metastasis, 1200 induces tumor regression, and increases 1000 Vehicle survival in various mouse models: 800 » Inhibits tumor growth i
Slide 31: PTX-100: Completed Phase 1 in Advanced Solid Tumors
PTX-100: COMPLETED PHASE 1 IN ADVANCED SOLID TUMORS Patients • 13 For personal use only Trial Centers • University of Pennsylvania & Indiana University • Heavily pre-treated patients with refractory, advanced solid tumors Patient » 5 rectal; 5 colon; 1 hepatocellular; 1 carcinoid; 1 esophageal Inclu
Slide 32: PTX-100: Cancer Stem Cell Opportunity
PTX-100: CANCER STEM CELL OPPORTUNITY For personal use only • Cancer stem cells (CSCs) are one of the reasons cancers rebound • CSCs resistant to conventional therapies • Targeting CSCs is crucial to treating malignant disease • PTX-100 reduces breast cancer stem cells both in vitro and in vivo in p
Slide 33: Many PTX-100 Development Opportunities
MANY PTX-100 DEVELOPMENT OPPORTUNITIES For personal use only • Mutated N- & K-Ras pathway is a feature of many cancers, including: » Pancreatic & other GI cancers » NSCLC » Multiple Myeloma • Considering Phase 1b/2 trial in Ras mutant all-comers with PTX-100 as monotherapy • RhoA (a direct target of
Slide 34: Investment Highlights
INVESTMENT HIGHLIGHTS For personal use only 2 DRUGS » 3 TRIALS » IMMINENT CATALYSTS » FUNDING IN PLACE » UNDISCOVERED VALUE • 2 targeted therapies with impeccable scientific pedigree • Multiple shots on goal with Akt and Ras pathway inhibitors in multiple trials • One of deepest clinical pipelines o
Slide 35: Contact
For personal use only CONTACT Steven Yatomi-Clarke e: steven@ptxtherapeutics.com CEO & Managing Director t: +61 417 601 440 Prescient Therapeutics Limited w: ptxtherapeutics.com 35 TARGETED THERAPIES YIELDING A DEEP CLINICAL PIPELINE
Prescient Therapeutics — Biotech Showcase, San Francisco 2017 on 6ix