Fate Therapeutics, Inc. · NASDAQ:FATE
Fate Therapeutics — Investor Presentation (February 2019)
43 slides from Fate Therapeutics's investor presentation, February 2019
Slide 1: Programmed Cellular Immunotherapies
Better Cells For Better Therapies™ Programmed Cellular Immunotherapies Overview of Immuno-Oncology Programs February 2019 www.fatetherapeutics.com -1-
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company's advancement of and plans related to the Company's product candidates, clinical studies, and rese
Slide 3: Proven Role of Cells in Cancer Immunotherapy
Proven Role of Cells in Cancer Immunotherapy Antibody-Dependent Cellular Immune Checkpoint Targeted / Activated Cytotoxicity (ADCC) Blockade Cell Products -3-
Slide 4: Cell-based Cancer Immunotherapy
Cell-based Cancer Immunotherapy Therapeutic Vision for Long-Term Durable Responses -4-
Slide 5: First Innings of Cell Therapy Development
First Innings of Cell Therapy Development Patient-derived CAR-T Cell Immunotherapy Patient ~2-3 week process Impaired Starting Material | Random & Variable Engineering | Complex Logistics Heterogeneous Drug Product | Expensive | Single-dose Limitation How do we Build on Early Successes and Transitio
Slide 6: Our Approach to Next-Generation Cellular Immunotherapy
Fate Therapeutics Our Approach to Next-Generation Cellular Immunotherapy "2.0" Donor-derived Cell Products "3.0" Off-the-Shelf Cell Products Cells from healthy donors with Renewable pluripotent cell lines selected traits with engineered functionality Donors iPSC Cell Line Ex vivo pharmacologic modul
Slide 7: Our First-in-Class Cellular Immunotherapy Pipeline
Fate Therapeutics Our First-in-Class Cellular Immunotherapy Pipeline Product Description Indication R&D Preclinical Clinical Partner Off-the-Shelf Cell Products derived from Clonal Master iPSC Line FT500 iNK +/- CPB in Solid Tumors IND Cleared by FDA FT516 hnCD16 iNK +/- mAb in Hematologic Malignanc
Slide 8: Unique Anti-Tumor Activity of Allogeneic NK Cells
Natural Killer Cells Unique Anti-Tumor Activity of Allogeneic NK Cells • Multi-faceted effector function ➢ Anti-tumor activity is not dependent on antigen recognition • Use of healthy donor cells has been shown to be safe and well-tolerated ➢ No reports of GvHD, cytokine release syndrome or neurotox
Slide 9: FATE-NK100: First-in-Class Adaptive Memory NK Cell Cancer Immunotherapy
FATE-NK100 First-in-Class Adaptive Memory NK Cell Cancer Immunotherapy NK NK NK NK NK NK NK NK NK NK NK NK NK NK NKG2C NK Jeffrey S. Miller, MD CD57+ Formation of Adaptive Memory NK Cells Correlated with reduced relapse risk and superior disease-free survival in HCT A Potent Subset of Activated NK C
Slide 10: Realizing the Potential of Adaptive Memory NK Cells
FATE-NK100 Realizing the Potential of Adaptive Memory NK Cells mono 7-Day NK Apheresis mono NK Ex Vivo Modulation NK NK NK NK NK NK NK mono NK NK NK NK T & B Cell Depletion FT1238 + Cytokine mono Feeder-free CMV+ Donor Patient-Derived Primary AML Blasts Luc-SKOV3 Tumor Image Analysis Day 21 - 10 -
Slide 11: Multi-pronged Clinical Development Strategy
FATE-NK100 Multi-pronged Clinical Development Strategy VOYAGE 1 • Single IV infusion; up to 3 dose levels (DLs) Refractory / • 10-patient expansion at MTD Relapsed AML • Key read-outs: NK cell persistence; anti-leukemia activity; CRs • Single IP infusion; up to 3 dose levels (DLs) APOLLO 1 • 10-pati
Slide 12: Initial Clinical Data: Dose-Escalation Phase
FATE-NK100 Initial Clinical Data: Dose-Escalation Phase 14 subjects treated with FATE-NK100 had one-month follow-up 1 All subjects refractory / failed multiple lines of therapy All subjects presented with progressive disease • Encouraging evidence of anti-cancer activity as a single-dose monotherapy
Slide 13: Changing the Game in Cell Therapy
Changing the Game in Cell Therapy Key Features Cell Therapy 1.0 & 2.0 Cell Therapy 2.0 Cell Source Patient / Donor Cells Master Cell Line Genetic Engineering Random, Variable & Repetitive Uniform & Complete Product Consistency Heterogeneous Homogeneous Characterization Imprecise Well-defined Manufac
Slide 14: Current Challenges of Cell Engineering
Current Challenges of Cell Engineering Cellular Chaos The Potential for Cellular Chaos A single dose of an engineered cell therapy is typically comprised of >10 million cells Not All Cells Are Engineered Source: Guggenheim Securities - 14 -
Slide 15: Current Challenges of Cellular Engineering
Current Challenges of Cellular Engineering Molecular Chaos The Potential for Molecular Chaos Each cell contains 23 pairs of chromosomes, with an estimated total of 20,000 to 25,000 genes and 6 billion nucleotides Each Cell Is Not Engineered The Same • Random integration of transgenes • Double strand
Slide 16: Changing the Game in Cell Therapy
Changing the Game in Cell Therapy Key Features Cell Therapy 1.0 & 2.0 Cell Therapy 2.0 Cell Source Patient / Donor Cells Master Cell Line Genetic Engineering Random, Variable & Repetitive Uniform & Complete Product Consistency Heterogeneous Homogeneous Characterization Imprecise Well-defined Manufac
Slide 17: Changing the Game in Cell Therapy
Changing the Game in Cell Therapy Key Features Cell Therapy 1.0 & 2.0 Cell Therapy 3.0 Cell Source Patient / Donor Cells Master Cell Line Genetic Engineering Random, Variable & Repetitive Specific, Uniform & One-time Product Consistency Heterogeneous Homogeneous Characterization Imprecise Well-defin
Slide 18: Human Induced Pluripotent Stem Cells
Human Induced Pluripotent Stem Cells Foundational Inventions of Fate Therapeutics’ Scientific Founders Induced Pluripotent Stem Cells (iPSCs) Rudolf Jaenisch Sheng Ding Fate Therapeutics’ iPSC product platform is supported by an IP portfolio of 100+ issued patents and 100+ pending patent application
Slide 19: Clonal Master iPSC Lines
Clonal Master iPSC Lines Single-cell Engineering, Selection, Characterization & Banking A Single Human Induced Pluripotent Cell Unlimited Self- Single iPSC Clone Potential to Differentiate Renewal into 200+ Cell Types Precise Master Cell Engineering Banks Uniform in Extensive Composition Characteriz
Slide 20: Clonal Master iPSC Lines
Clonal Master iPSC Lines Renewable Source for Manufacturing Off-the Shelf Cell Products Human Induced Multiplexed Engineering Clonal Pluripotent Stem Cells Single-cell Clonal Selection Master iPSC Line Universal | Off-the-Shelf | Cell Products iT Cells iNK Cells iCD34+ Cells Other iCells - 20 -
Slide 21: Universal Off-the-Shelf Cell Products
Universal Off-the-Shelf Cell Products Collaborations with Top Investigators and Leading Centers Engineered NK Cells CAR NK Cells CAR T Cells Jeffrey S. Miller, MD Dan Kaufman, MD PhD Michel Sadelain, MD, PhD - 21 -
Slide 22: Universal Off-the-Shelf NK Cell Products
Universal Off-the-Shelf NK Cell Products Robust Differentiation Protocol to Create iPSC-derived NK Cells Day 0 - iPSC Day 10 – iCD34 NK Cell Differentiation & Expansion NKp30 Renewable Clonal Master iPSC Line Hundreds of doses of homogeneous NK cells per each manufacturing campaign >95% pure for CD5
Slide 23: FT500: Universal Off-the-Shelf NK Cell Product
FT500: Universal Off-the-Shelf NK Cell Product Bridging Innate and Adaptive Immunity Landmark IND for FT500 Cleared by FDA in December 2018 Multi-faceted Functionality to Augment T-cell Activity 3-D Ovarian Cancer Tumor Model 1 5 0 0 0 _ ✓ Enhanced cytokine and chemokine production Target Cell Elimi
Slide 24: FT500: Combination with Checkpoint Blockade
FT500: Combination with Checkpoint Blockade Rescue Therapy in Patients with B2M Mutations Survival ✓ B2M mutations are enriched in patients who are resistant to checkpoint blockage (~30%) No B2M loss ✓ Loss of B2M associated with poor survival ✓ MHC Class 1 null tumor cells B2M loss are highly susce
Slide 25: FT500: FIH Study in Advanced Solid Tumors
FT500: FIH Study in Advanced Solid Tumors Multi-dose, Multi-cycle Combination with Checkpoint Blockade Cycle 2 - 25 -
Slide 26: NK Cell Expression of Naturally-Occurring CD16
NK Cell Expression of Naturally-Occurring CD16 Fc Receptor Mediates Antibody-Dependent Cellular Cytotoxicity (ADCC) • NK cells bind antibody-coated tumor cells via CD16 Fc receptor, initiating release of perforin and granzyme resulting in tumor cell death • ADCC contributes to anti-tumor activity of
Slide 27: FT516: Off-the-Shelf hnCD16 NK Cell Product
FT516: Off-the-Shelf hnCD16 NK Cell Product Enhanced ADCC for Combination with Monoclonal Antibody Therapy High-Affinity, Non-Cleavable CD16 Engineered High-affinity, Non-cleavable CD16 Fc Receptor Modified form of CD16a IgG antibody-binding receptor hnCD16 resists shedding upon activation High affi
Slide 28: FT516: Combination with Rituxan
FT516: Combination with Rituxan Enhanced In Vivo Survival IND Cleared by FDA in February 2019 for First-ever Engineered iPSC-derived Cell Therapy - 28 -
Slide 29: FT538: Off-the-Shelf hnCD16 / CD38- NK Cell Product
FT538: Off-the-Shelf hnCD16 / CD38- NK Cell Product Enhanced ADCC with Elimination of Fratricide for Anti-CD38 mAb Combo Serial Killing in the Presence of anti-CD38 mAb hnCD16 CD38 KO FT538 IL15/R Combination with anti-CD38 mAb for Multiple Myeloma daratumumab isatuximab - 29 -
Slide 30: Universal Off-the-Shelf CAR NK Cell Products
Universal Off-the-Shelf CAR NK Cell Products Developed Novel Construct Specifically Optimized for NK Cell Activity Identified novel NK cell-specific constructs by screening CARs consisting of a transmembrane domain and co- stimulatory domain(s) typically expressed in NK cells iPSC-derived CAR4 NK Ce
Slide 31: FT596: Off-the-Shelf hnCD16 / CAR19 NK Cell Product
FT596: Off-the-Shelf hnCD16 / CAR19 NK Cell Product Multi-modality Engineered Master iPSC Line Cell – CAR | CIP | USE “USE” hnCD16 Universal Secondary Engager CAR19 FT596 “CIP” IL15/R Cytokine- independent Persistence IND to be Submitted in 2019 - 31 -
Slide 32: FT596: Target-specific Enhanced Potency
FT596: Off-the-Shelf hnCD16 / CAR19 NK Cell Product Target-specific Enhanced Potency Target-specific Cytokine Production Target-specific Cytotoxicity - 32 -
Slide 33: FT596: In Vivo Anti-tumor Activity
FT596: Off-the-Shelf hnCD16 / CAR19 NK Cell Product In Vivo Anti-tumor Activity Control of Tumor Growth Survival - 33 -
Slide 34: Universal Off-the-Shelf CAR T-Cell Products
Universal Off-the-Shelf CAR T-Cell Products Memorial Sloan Kettering Collaboration “Engineering therapeutic attributes into pluripotent cell lines is a breakthrough approach to renewably generate potent T-cell immunotherapies. This unique approach offers the prospect for off-the-shelf delivery of T-
Slide 35: FT819: CAR19 TCR-null T-Cell Product Candidate
FT819: CAR19 TCR-null T-Cell Product Candidate TRAC-encoded CAR Expression: Primary T Cells vs. Single iPSC CRISPR Engineering: TCR Disruption + TRAC-encoded CAR Expression Primary T Cell Batch ✓ 20% of T cells express allo-reactive TCR ✓ Only 45% of T cells have TCR KO + CAR expression Single iPSC
Slide 36: FT819: Novel MSK Construct 'TRAC-1XX'
FT819: CAR19 TCR-null T-Cell Product Candidate Novel MSK Construct ‘TRAC-1XX’ Optimized for T-Cell Activity - 36 -
Slide 37: FT819: In Vitro Cytokine Production and Specificity
FT819: CAR19 TCR-null T-Cell Product Candidate In Vitro Cytokine Production and Specificity vs. Primary CAR T Cells Cytokine Production Antigen-specific Cytotoxicity - 37 -
Slide 38: FT819: In Vivo Anti-Tumor Activity vs. Primary CAR T Cells
FT819: CAR19 TCR-null T-Cell Product Candidate In Vivo Anti-Tumor Activity vs. Primary CAR T Cells Cryopreserved TRAC-CAR iT cells (6e6) and Primary CAR-T (1e6) were thawed and injected into NOD-SCID IL2Rgcnull mice inoculated with CD19+ NALM-6 expressing firefly luciferase. TRAC-CAR iT cohort were
Slide 39: iPSC Product Platform
iPSC Product Platform Clonal Master iPSC Lines for Off-the-Shelf Cell Products (Engineered) Single Pluripotent Stem Cell Single • Renewable iPSC Clone • Potential to differentiate into 200+ cell types Expansion & Banking Working Cell Banks Unlimited Supply of Master Cell Working Cell Banks Clonal Ma
Slide 40: ONO Pharmaceutical Collaboration
ONO Pharmaceutical Collaboration Two Off-the-Shelf CAR T-Cell Product Candidates • Partnership combines ONO’s immuno-oncology innovation with Fate’s industry-leading iPSC product platform – ONO was the originator of PD-1 checkpoint inhibitor Opdivo (nivolumab), licensed by BMS in 2011, and jointly d
Slide 41: Financial Summary
Financial Summary As of September 30, 2018 Three Months Ended September 30, 2018 Revenue $1.0M Operating Expense, Adjusted 1 $14.2M Cash & Cash Equivalents $211.2M Employees 92 Total Shares Outstanding 2 78.6M [1] Excludes one-time expense of $1.4M associated with the in-license of IP from J. David
Slide 42: Projected 2019 Milestones
Projected 2019 Milestones • ProTmune: Next-Generation Allogeneic Cell Graft – Complete enrollment in randomized, blinded, controlled PROTECT Phase 2 study • NK100: First-in-class Adaptive Memory NK Cell Cancer Immunotherapy – Additional clinical data in AML and Ovarian as monotherapy – Additional cl
Slide 43: Better Cells For Better Therapies
Better Cells For Better Therapies™ - 43 -
Fate Therapeutics — Investor Presentation (February 2019) on 6ix