Prescient Therapeutics Limited · ASX:PTX
Prescient Therapeutics — Investor Presentation (August 2022)
59 slides from Prescient Therapeutics's investor presentation, Aug 23, 2022
Slide 1: In Front of the Biggest Wave in Oncology
For personal use only IN FRONT OF THE BIGGEST WAVE IN ONCOLOGY August 2022 1
Slide 2: Disclaimer and safe harbor
Disclaimer and safe harbor 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 but rather are based on
Slide 3: Corporate snapshot
Corporate snapshot For personal use only ASX Ticker PTX Total Issued Capital 648 M shares Listed Options 95.4 M Unlisted Options 12.1 M Share Price1 A$0.21 (US$0.15) Market A$141 M Capitalisation1 (US$99 M) Market Cap fully A$162 M diluted1 (US$114 M) Cash Position2 A$14.7M (US$11M) Top 20 Own 16% 1
Slide 4: Investment Highlights
Investment Highlights For personal use only World class pedigree. We license from the best; and work with the best Many shots on goal for 2 Cell Therapy platforms substantial value creation Internal & external opportunities 2 Targeted Therapies Upcoming newsflow in clinic trials, showing activity fr
Slide 5: Innovative Personalised Oncology Assets
4 Innovative Personalised Oncology Assets For personal use only Cell therapy Next-gen Targeted therapies platforms CAR-T therapies CD33/CLL-1 Acute Myeloid PTX-100 Leukaemia Ras / Rho inhibitor Her2/EGFRviii Glioblastoma Multiforme PTX-200 Novel AKT Her2 inhibition Breast, Ovarian & Gastric cancers
Slide 6: Innovative pipeline in personalised medicine
Innovative pipeline in personalised medicine Platform Program Screening Preclinical Phase IB Phase II For personal use only CD33/CLL-1 AML Her2 Breast, Ovarian & Gastric cancers Her2/EGFRviii GBM Platform extensions Various CellPryme-M Cell manufacturing enhancement CellPryme-A Undisclosed PTX-100 P
Slide 7: Targeted Therapies
For personal use only TARGETED THERAPIES 7
Slide 8: PTX-100 First in Class Ras Pathway Inhibitor
For personal use only PTX-100 FIRST IN CLASS RAS PATHWAY INHIBITOR 8
Slide 9: PTX-100 Phase 1B Summary
PTX-100 Phase 1B Summary Licensed from For personal use only • Phase 1b PK/PD safety study • Targeting cancers predisposed to Ras & Rho mutations • Basket trial of: Principal Investigator • Gastric cancer • Myeloma • Pancreatic cancer • T-cell lymphomas • Colorectal cancer • Encouraging signal in TC
Slide 10: Favourable safety profile compared to peers
Favourable safety profile compared to peers For personal use only Approved PTCL drugs have troublesome safety profiles % Serious Adverse Events (≥Grade 3) PTX-100 HAS AN for Approved PTCL Drugs EXCELLENT SAFETY PROFILE Mogamulizumab • No serious adverse events Chidamide related to PTX-100 Brentuxima
Slide 11: Encouraging activity in TCL
Encouraging activity in TCL For personal use only Early clinical activity Time on Treatment with PTX-100 • PRs in 2 patients with aggressive refractory TCL • Expected PFS of <4 months on SoC • r/r CTCL: 12 months (19 cycles) • r/r PTCL: >32 months so far (37 cycles, still on therapy) • Expansion coh
Slide 12: Now in Expansion Cohort for TCL
Now in Expansion Cohort for TCL For personal use only Case Study • 8 – 12 patients with r/r T cell lymphoma • pralatrexate (Folotyn®) (esp PTCL) • Approved for PTCL • Potential bridge to registration study • 5,600 cases/year in US • Focussing on sweet spot in an area of • US$450,540 per patient, per
Slide 13: PTX-200 Novel AKT Inhibition
For personal use only PTX-200 NOVEL AKT INHIBITION 13
Slide 14: Phase 1B trial underway: Acute Myeloid Leukemia
Phase 1B trial underway: Acute Myeloid Leukemia For personal use only • Building upon encouraging Phase 1 results with PTX-200 (monotherapy) Principal Investigator • PI Professor Jeff Lancet at Moffitt, Key Opinion Leader in AML • 24 patients with cytarabine held constant at 200-400 mg/m 2 as contin
Slide 15: Platform Technologies
For personal use only PLATFORM TECHNOLOGIES 15
Slide 16: How does the CAR-T process work?
How does the CAR-T process work? For personal use only 1 2 3 4 5 Blood is collected T-Cells are separated T-Cells are Millions of CAR-T CAR – T cells are from the patient and removed genetically altered to cells are grown delivered to the have special cancer- patient recognising receptors called Chi
Slide 17: Cell Therapy is the future of oncology
Cell Therapy is the future of oncology For personal use only Immune system Powerful cancer Highly encouraging Long-term Still a lot to focus fighting potential data remissions discover 17
Slide 18: CAR-T is booming
CAR-T is booming GlobalData forecasts CAR-T sales to 2028 exceed US$37bn TODAY For personal use only • 10y+ remissions 2023 GlobalData forecasts • Continued positive data flow Kymriah® sales to exceed US$1bn • Some outstanding results • 100s of companies targeting different antigens to target 2017 m
Slide 19: Penn is a pioneer and world leader in CAR-T
Penn is a pioneer and world leader in CAR-T For personal use only • Novartis licensed CAR-T technology from Penn in 2012 • Kymriah® became the first CAR-T therapy approved by the FDA • Used for certain blood cancers • Cost of treatment in excess of $500,000 per treatment • GlobalData forecasts Kymri
Slide 20: CAR-T's key challenges
CAR-T’s key challenges Challenge For personal use only Safety / Control No control post infusion Difficulties with targeting, Unsafe Targeting antigen heterogeneity Escape Difficulties with mutating antigens Less effective Production efficiency Cost prohibitive & slow Not sustainable Exhaustion Cell
Slide 21: CAR-T's key challenges
CAR-T’s key challenges Challenge For personal use only Safety / Control No control post infusion - Difficulties with targeting, Targeting antigen heterogeneity - Safe Escape Difficulties with mutating antigens - Effective Production efficiency Cost prohibitive & slow - Sustainable Exhaustion
Slide 22: Universal, Next Gen CAR-T Platform
For personal use only Universal, Next Gen CAR-T Platform 22
Slide 23: OmniCAR: flexible, modular CAR platform
OmniCAR: flexible, modular CAR platform For personal use only Targeting ligand Using any targeting SpyTag Covalent ligand… bond Spy Catcher Associate Professor Professor Daniel J. Powell, Jr Andrew Tsourkas Signalling domain …with any immune UNARMED ARMED CAR-T cell CAR-T cell cell 23
Slide 24: How OmniCAR works
For personal use only How OmniCAR works 24
Slide 25: Complementary platforms to address CAR-T challenges
Complementary platforms to address CAR-T challenges Challenge For personal use only Tune activity up/down; Safety / Control No control post infusion - On/off Difficulties with targeting, Targeting Target multiple antigens - antigen heterogeneity Escape Difficulties with mutating antigens Sequential
Slide 26: OmniCAR can do what conventional CAR-T cannot
OmniCAR can do what conventional CAR-T cannot For personal use only Conventional CAR-T Can be given any map; Armed with any weapon Multiple deployments Including several at once • Soldier with only one map • Single weapon Full communication • Only trained to hit one target and control at all times,
Slide 27: OmniCAR: Control Features
OmniCAR: Control Features For personal use only Modular and covalent architecture of OmniCAR enables true post-infusion control of CAR functionality Dose Titration On/off switch Target Re-direction Multi-Antigen Targeting Control activity to safe Turn therapy on/off/on Re-direct cells from Target mu
Slide 28: Control: Dose-dependent CAR-T activity
Control: Dose-dependent CAR-T activity For personal use only • Ovarian cancer model, using anti-HER2 OmniCAR • Loading more binder results in proportionate killing of cancer… • …and proportionate survival • Lasting effects even when cease dosing of binder 28 Powell, DJ et al, JACS; 2020 = dosing win
Slide 29: OmniCAR cells can be Re-Armed
OmniCAR cells can be Re-Armed T cell activation Transduction Armed Cytotoxicity assay For personal use only Day 0 Day 3 Day 7 Days 8–10 Re-armed Cytotoxicity assay Wash; Rest 2:1 OmniCARDay Her2 12 normalised post-REP Days 13–14 2:1 OmniCAR Her2Anormalised pre-REP B 150 150 Tumour alone Washed Omn %
Slide 30: OmniCAR cells can be Redirected
OmniCAR cells can be Redirected Coculture of U251 GBM Cells For personal use only expressing HER2 or EGFRviii • Rapid cytotoxicity to EGFRviii • Rapid switching and cytotoxicity against HER2+ tumours upon administration of new binder • OmniCAR cells can be re-directed to different antigens upon admi
Slide 31: OmniCAR manufacturing & COGS advantages
OmniCAR manufacturing & COGS advantages For personal use only Conventional CAR-T 6 INDs 1 IND 6 Manufacturing runs 1 Optimized Manufacture 6 Vectors/Transductions 1 Vector/Transduction 6 Sets of release testing 1 Release program No Flexibility Multiple binders off the shelf = 6 X ($$$$) No time dela
Slide 32: Next Gen CAR-T Programs
For personal use only Next Gen CAR-T Programs 32
Slide 33: OmniCAR internal program summary
OmniCAR internal program summary Targets Indications OmniCAR features Comments For personal use only • Titration for improved safety • Validated targets; expressed on Acute Myeloid 90%+ of AML blasts & LSCs CD33 + Leukemia • Co-arming against CD33 & CLL-1 CLL-1 (AML) • 1 of 5 programs worldwide; the
Slide 34: OmniCAR progressing towards clinic
OmniCAR progressing towards clinic For personal use only • Steady progress across all programs • OmniCAR AML likely the first program in clinical trials • Q-Gen Cell Therapeutics appointed as cell manufacturer • Clinical grade cells • Autologous T cells expressing SpyCatcher • Incorporating CellPrym
Slide 35: Thermo Fisher agreement for next version OmniCAR
Thermo Fisher agreement for next version OmniCAR For personal use only Research agreement (MTA) to extend OmniCAR platform: • Non-viral methods of transduction • Greater transduction efficiency • Global leader in scientific • Faster instrumentation & services • Lower COGS • US$40 billion revenue • A
Slide 36: Aims & Outcomes for OmniCAR
Aims & Outcomes for OmniCAR For personal use only Further future-proofing Positioning OmniCAR for OmniCAR platform technical & commercial success • V2 OmniCAR cells that: • Can be made in an automated process • Manufacturing can be easily tech transferred to 3rd parties • Unmatched reproducibility •
Slide 37: Additional potential benefits of the Thermo Fisher agreement
Additional potential benefits of the Thermo Fisher agreement For personal use only • Early access to Thermo Fisher’s new, state-of-the-art technologies • Protocol and process optimization from Thermo Fisher’s technical experts • Regulatory support to enable Prescient’s regulatory filings • Ongoing s
Slide 38: Cell therapy enhancements
For personal use only Cell therapy enhancements 38
Slide 39: CellPryme: Prescient's newest family member
CellPryme: Prescient’s newest family member PROCESS TO ENHANCE 2 SYNERGISTIC For personal use only CELL THERAPIES COMPONENTS • Current gen and next gen • CellPryme-M • Complementary to OmniCAR • CellPryme-A (coming soon) CellPryme-M • Produces superior cells READY FOR • Use with any existing CAR-T C
Slide 40: What does CellPryme-M do?
What does CellPryme-M do? For personal use only CellPryme-M is a single, rapid manufacturing step that produces a better, more effective cell type: LONGER LASTING CELLS FOR CELLS THAT CAN BETTER SUSTAINED TUMOUR KILLING LOCATE THE TUMOUR • 50% more memory T cells • Significantly more chemokine recep
Slide 41: CellPryme-M complements OmniCAR
CellPryme-M complements OmniCAR For personal use only • Multi-targeting • Redirection • Control & safety • Any target; any cell Process that produces a better cell type • Persistence • Trafficking Next generation Current generation Cell therapies cell therapies 41
Slide 42: Complementary platforms to address CAR-T challenges
Complementary platforms to address CAR-T challenges Challenge For personal use only Safety / Control No control post infusion - Difficulties with targeting, Targeting antigen heterogeneity - Escape Difficulties with mutating antigens - Production efficiency Cost prohibitive & slow Superior c
Slide 43: More memory cells required for clinical efficacy
More memory cells required for clinical efficacy • Clinical efficacy of CAR-T therapy remains dependent on the T cell phenotype For personal use only • It is possible to control this during the manufacturing step We need more of Current CAR-T makes these cells too many of these cells CXCR3 CXCR3 Naï
Slide 44: Greater Persistence: 50% more central memory cells
Greater Persistence: 50% more central memory cells than conventional CAR-T For personal use only Tem (CD44 Tem (CD44 hiCD62L hiCD62L lo) lo) Tcm (CD44 Tcm (CD44 hiCD62L hiCD62L hi) hi) CellPryme-M increases central memory T cells 1.5-fold within 24hrs 100 100 CD8+CAR+ 80 80 %+% CD8+NGFR+% Convention
Slide 45: CellPryme-M doubles tumour control and survival
CellPryme-M doubles tumour control and survival For personal use only CellPryme-M nearly doubles CellPryme-M CAR-T tumour control doubles survival 150 100 Probability of Survival Tumour area (mm2 ) 80 100 60 40 50 20 0 0 0 5 10 15 20 25 0 10 20 30 40 Time post treatment (d) Days post tumour inoculat
Slide 46: Greater Persistence/Less Exhaustion
Greater Persistence/Less Exhaustion For personal use only Sustained increase in TCM Sustained decrease in TEM for both cytotoxic CD8+ and helper CD4+ for both cytotoxic CD8+ and helper CD4+ CD8+ CAR TCM CD4+ CAR TCM CD8+ CAR TEM CD4+ CAR TEM 100 p<0.0001 100 100 100 % CAR CD4+ T cells % CD8+ CAR T c
Slide 47: Synergy: CellPryme-M doubles proportion of helper T cells
Synergy: CellPryme-M doubles proportion of helper T cells For personal use only 100 100 helper CD4+CD4+ CARCAR T T CD8+CD8+ T • TShift towards dominant helper CD4+ CAR T cells CARCAR 80 80 %CD4/CD8 %CD4/CD8 100 CD4+ CAR T 60 60 80 CD8+ CAR T • Helper T cells are known to prevent the exhaustion %CD4/
Slide 48: Trafficking: greater chemokine receptor expression
Trafficking: greater chemokine receptor expression For personal use only Chemokine receptor expression on CD8+ cytotoxic CAR-T cells • Effector T cells can downregulate chemokine 1.3 receptors (CXCR3), limiting the ability of p<0.0001 conventional CAR-T cells to locate tumours CXCR3 expression 1.2 •
Slide 49: CellPryme-M produces CAR-T cell types with ideal characteristics
CellPryme-M produces CAR-T cell types with ideal characteristics and attributes For personal use only Immune memory Persistence Central memory T cells typically persist For longevity of effects and 10-20 years and as long as 75 years continued tumour control Trafficking Tumour penetrance CAR-T cells
Slide 50: Scope, Model & Business
For personal use only SCOPE MODEL & BUSINESS
Slide 51: Prescient's CAR-T platform business model
Prescient’s CAR-T platform business model PTX therapies For personal use only PTX CD33 + CLL-1 PTX platforms PTX HER2 • Huge market PTX HER2 + EGFRviii • “Shovels to CAR-T External partners goldrush” Partner 1 Partner 2 • Diversified risk Partner 3 Partner 4 • Highly scalable Partner 5 • Earlier rev
Slide 52: Commercial models - Partners
Commercial models - Partners For personal use only Partner 1 Agreement Development Sales Upfront fee Milestone fees Royalties & milestone fees Partner 2 Agreement Development Sales Upfront fee Milestone fees Royalties & milestone fees Partner 3 Development Sales Agreement Upfront fee Milestone fees
Slide 53: OmniCAR Platform business model
OmniCAR Platform business model COLLABORATE & For personal use only NEXT GENERATION LICENSE TO 3RD PRODUCTS PARTIES IN-HOUSE Partners for TARGETS & BINDERS OmniCAR CD33/CLL1 for AML Partners for CELL TYPES OmniCAR Her2 For Solid Tumours Partners for SPECIFIC OmniCAR Her2/EGFRviii CONSTRUCTS for GBM
Slide 54: CellPryme-M Next steps and future applications
CellPryme-M Next steps and future applications For personal use only IN-HOUSE DEVELOPMENT EXTERNAL OPPORTUNITIES • Incorporate into 3rd party programs • PTX will be its own first customer • Attractive option for improving existing • Incorporate into internal OmniCAR suboptimal CAR T products program
Slide 55: The End Game: Personalized Plug & Play Cell Therapy Ecosystem
The End Game: Personalized “Plug & Play” Cell Therapy Ecosystem For personal use only Step 4: Step 3: Bespoke therapy with Pair binders with OmniCAR cells post-infusion control (T cells; NK; auto/allo) Step 2: Match patient’s antigens to corresponding binders Step 1: Patient sample to determine indi
Slide 56: Summary
For personal use only Summary 56
Slide 57: Top-down analysis is sensible for investors
Top-down analysis is sensible for investors For personal use only Oncology* 1 Biggest Market in • 2021: US$ 280bn Healthcare? • 2029: US$ 536bn (8.2% CAGR) 2 Fastest growing area Cell Therapies (CAR-T) in oncology? • >US$37bn by 2028^ Prescient Therapeutics 3 Forefront of • Next gen platforms Cell T
Slide 58: Investment Thesis Summary
Investment Thesis Summary 4 blue chip Superior Huge & For personal use only oncology assets positioning & model growing market 2 next gen platforms Internal products + external partnering $280bn industry PTX-100 & PTX-200 Shovels to goldrush Growing demand in clinic Top pedigree Highly scalable Cell
Slide 59: Thank you!
For personal use only Thank you! ASX code: PTX www.ptxtherapeutics.com
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