Fate Therapeutics, Inc. · NASDAQ:FATE
Fate Therapeutics — Investor Presentation (November 2019)
39 slides from Fate Therapeutics's investor presentation, Nov 21, 2019
Slide 1: Off-the-shelf Cell-based Cancer Immunotherapy
Better Cells For Better Therapies™ Off-the-shelf Cell-based Cancer Immunotherapy Developing First-of-kind Cell Products using Clonal Master iPSC Lines November 2019 www.fatetherapeutics.com Page - 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 research and development activities and its progress, plans and timelines for its manufacture, p
Slide 3: First Innings of Cell Therapy Development
First Innings of Cell Therapy Development Patient-derived CAR-T Cell Immunotherapy "When factoring in all the costs associated with CAR T-cell therapy, hospitals may Patient charge as much as $1.5 million or more to 2-3 week manufacturing avoid losing money." $425,000 drug cost Richard T. Maziarz, M
Slide 4: Batch-to-Batch Engineering is Expensive and Results in Heterogeneity
First Innings of Cell Therapy Development Batch-to-Batch Engineering is Expensive and Results in Significant Product Heterogeneity Each manufacturing batch is comprised of millions of variable engineering events in primary T cells Healthy Donor or Patient Apheresis Product Patient Specificity (CAR)
Slide 5: Multiplexed CRISPR / Cas9 Gene Editing of Patient T Cells
First Innings of Cell Therapy Development Example: Multiplexed CRISPR / Cas9 Gene Editing of Patient T Cells • Patient-specific Engineered TCR T-cell Therapy (University of Pennsylvania) – Mulitplexed CRISPR / Cas9 editing ➢ Removal of TCRα (TRAC), TCRβ (TRBC) and PD-1 (PDCD1) ➢ Transduced to expres
Slide 6: Changing the Game in Cell-based Cancer Immunotherapy
Changing the Game in Cell-based Cancer Immunotherapy The Potential to Characterize, Select and Renewably Use a Single Cell What if we had the opportunity to engineer, characterize, select, and renewably use a single cell? Page - 6 -
Slide 7: Universal, Off-the-Shelf Cell Products Derived from Renewable Master Cell Lines
Changing the Game in Cell-based Cancer Immunotherapy Universal, Off-the-Shelf Cell Products Derived from Renewable Master Cell Lines Key Features Cell Therapy 1.0 and 2.0 Cell Therapy 3.0 Cell Source Patient and Donor Cells Renewable Master Cell Line Genetic Engineering Random & Variable Uniform & C
Slide 8: Human Induced Pluripotent Stem Cells (iPSCs)
Human Induced Pluripotent Stem Cells (iPSCs) Reprogramming Adult Somatic Cells to a Pluripotent State Generation of Human iPSCs Fate Scientific Founders Rudolf Jaenisch, MD Sheng Ding, PhD Page - 8 -
Slide 9: Unique Advantages of Human iPSCs
Unique Advantages of Human iPSCs Isolation, Characterization & Selection of a Single iPSC Clone A Single Human Induced Pluripotent Stem Cell (iPSC) A renewable source for making cells Unlimited Self- Single iPSC Clone Potential to Differentiate Renewal into 200+ Cell Types Multiplexed Master Cell Li
Slide 10: Off-the-Shelf Cell-based Cancer Immunotherapy
Off-the-Shelf Cell-based Cancer Immunotherapy iPSC Product Platform for Mass Production of Universal NK Cell and T-Cell Products Clonal Master Engineered Induced Pluripotent Single-Cell Sorting iPSC Bank Stem Cells & Clonal Selection Multiplexed Gene iPSC Expansion & Engineering Banking (one-time ev
Slide 11: Natural Killer (NK) Cells
Natural Killer (NK) Cells First Line of Defense against Tumors and Diverse Range of Pathogens Array of Activating and Inhibitory Surface Receptors Mediate NK Cell Activity • Activating receptors convey multi-faceted effector function against tumor cells – Unique ability to recognize stressed / trans
Slide 12: FT500 Off-the-Shelf NK Cell Product Candidate
FT500 Off-the-Shelf NK Cell Product Candidate First-ever iPSC-derived Cell Therapy to Advance to Clinical Investigation in the U.S. FT500 Product Candidate FT500 cGMP Manufacture (non-engineered) • High levels of expression of potent activating receptors (NKG2D, NKp30/40/46) • High levels of secreti
Slide 13: FT500 In Vitro Cytotoxicity vs Healthy Donor NK Cells
FT500 Off-the-Shelf NK Cell Product Candidate In Vitro Cytotoxicity – Head-to-Head Comparison against Healthy Donor NK Cells Enhanced Cytotoxicity vs. Healthy Donor NK Cells Target Cells Donor NK Cells FT500 (fresh) (cryopreserved) Page - 13 -
Slide 14: FT500 Phase 1 Dose Escalation
FT500 Off-the-Shelf NK Cell Product Candidate Phase 1 Dose Escalation – Monotherapy and Combination with Checkpoint Inhibitor Therapy Phase 1 Dose Escalation in Advanced Solid Tumors • Regimen A: Monotherapy – Salvage setting with patients having progressed or failed all FDA-approved therapies • Reg
Slide 15: FT500 Phase 1 Dose Escalation – Safety and Tolerability
FT500 Off-the-Shelf NK Cell Product Candidate Phase 1 Dose Escalation – Monotherapy and Combination with Checkpoint Inhibitor Therapy Phase 1 Dose Escalation in Advanced Solid Tumors Assess Novel Treatment Paradigm Key Read-outs: Safety and Tolerability • First-ever U.S. clinical investigation of iP
Slide 16: FT500 Phase 1 Dose Expansion
FT500 Off-the-Shelf NK Cell Product Candidate Phase 1 Dose Expansion – Overcome Resistance to Checkpoint Inhibitor Therapy Phase 1 Dose Expansion – Target Loss of MHC-I Tumor Escape Mechanism NK cells have the unique ability to recognize and kill cancer cells that have down-regulated MHC Class I, a
Slide 17: FT516 Off-the-Shelf hnCD16
FT516 Off-the-Shelf hnCD16 NK Cell Product Candidate CD16 Fc Receptor Mediates Antibody-Dependent Cellular Cytotoxicity (ADCC) • CD16 is an activating receptor expressed on NK cells NK Cell – Mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells re
Slide 18: FT516 High-Affinity, Non-Cleavable CD16 Fc Receptor
FT516 Off-the-Shelf hnCD16 NK Cell Product Candidate High-Affinity 158V, Non-Cleavable CD16 Fc Receptor for Enhanced ADCC hnCD16 Enhanced Survival In Vivo with Rituximab Mouse model of human lymphoma (Raji cells) FT516 Median survival time for FT516 + anti-CD20 Modified form of CD16a was not reached
Slide 19: FT516 Study Design: AML & Lymphoma
FT516 Off-the-Shelf hnCD16 NK Cell Product Candidate Phase 1 Study Design: Multiple Doses over Multiple Cycles for AML & Lymphoma First-ever Clinical Trial in World of Engineered iPSC-derived Cell Therapy Cyclophosphamide: 500 mg/m2 IV x 3 days Safety & Activity Fludarabine: 30 mg/m2 IV x 3 days IL-
Slide 20: FT516 Supported by Clinical POC
FT516 Off-the-Shelf hnCD16 NK Cell Product Candidate Supported by Clinical POC in Hematologic Malignancies and Solid Tumors Donor NK Cell Therapy for AML Monoclonal Antibody for Solid Tumors Phase 1 clinical trial in relapsed / refractory AML • Cytokine-primed, donor-derived NK cell therapy • Single
Slide 21: FT596 Off-the-Shelf Multi-Targeted CAR NK Cell
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate Potential Best-in-Class Cell-based Cancer Immunotherapy for B-cell Malignancies First-ever Cell Therapy Engineered with Three Active Anti-tumor Modalities Cleared for U.S. Clinical Investigation hnCD16: High-affinity 158V, non-cleavabl
Slide 22: FT596 IND Application Cleared by FDA
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate IND Application Cleared for Clinical Investigation by FDA Clonal Master iPSC Line Renewable source One-time iPSC engineering Scalable, cost-effective manufacture -------------------------------------- Off-the-Shelf Rapid time-to-patien
Slide 23: FT596 Uniformly Engineered with Three Anti-Tumor Components
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate Uniformly Engineered with Three Active Anti-Tumor Functional Components 3 Active Anti-Tumor Modalities Uniformly Engineered Product Profile CD16 CD56 CD45 CD56 IL15/R CAR19 CD56 CD56 Page - 23 -
Slide 24: FT596 In Vivo Activity vs Primary CAR T Cells
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate In Vivo Anti-Tumor Activity – Head-to-Head Comparison against Primary CAR T Cells Single-Antigen Targeting of CD19 in Humanized Mouse Model of Lymphoma Tumor Primary Control Only CART FT596 (3x) Primary CART FT596 Does not include addi
Slide 25: FT596 Overcoming Tumor Heterogeneity and Antigen Escape
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate Leveraging CAR + hnCD16 to Overcome Tumor Heterogeneity and Antigen Escape Targeting Multiple Tumor-Associated Antigens CD19+ targets 100 50 Co-culture 0 at 1:1 ratio 100 50 CD20+ targets + CD20-targeted mAb (rituximab) 0 Time of co-cu
Slide 26: FT596 Dual-Antigen Targeting in Lymphoma Xenograft
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate Leveraging CAR + hnCD16 to Overcome Tumor Heterogeneity and Antigen Escape Dual-Antigen Targeting of CD19 and CD20 in a Xenograft Model of Lymphoma FT596 (3x) Control Rituxan + Rituxan Page - 26 -
Slide 27: FT596 Phase 1 Study Design
FT596 Off-the-Shelf Multi-Targeted CAR NK Cell Product Candidate Phase 1 Study Design in Relapsed / Refractory B-cell Lymphoma and CLL Phase 1 Dose Escalation – Monotherapy and mAb Combination Combination with mAb in CLL @ MTD Combination with mAb @ MTD in B-cell Lymphoma 30M 90M 300M 900M cells cel
Slide 28: FT596 Supported by Clinical POC with Donor-derived CAR19 NK Cells
FT596 Supported by Clinical POC with Donor-derived CAR19 NK Cells M.D. Anderson Cancer Center, Katy Rezvani, M.D., Ph.D. (NCT03056339) • First-in-human clinical trial testing the safety and efficacy of donor-derived CAR NK cell therapy – Cord blood derived – Transduced with CAR19 (28z) / IL15 (secre
Slide 29: Off-the-Shelf NK Cell Cancer Immunotherapy Franchise
Off-the-Shelf NK Cell Cancer Immunotherapy Franchise Systematic Build of Industry-Leading iPSC-derived NK Cell Product Pipeline Universal, Off-the-Shelf NK Cell Cancer Immunotherapy Pipeline Clonal Master iPSC Line Synthetic Biology FT500 FT516 FT596 FT538 FT576 Multi-faceted Innate Immunity + High-
Slide 30: Memorial Sloan Kettering Collaboration
Off-the-Shelf CAR T-Cell Cancer Immunotherapy 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
Slide 31: FT819 TRAC-encoded CAR 1XX Expression
FT819 TRAC-encoded CAR 1XX Expression Engineering Primary T Cells vs. Single iPSC Clone for TCR Elimination 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 32: FT819 Off-the-Shelf CAR19 T-Cell Product
FT819 Off-the-Shelf CAR19 T-Cell Product Novel CAR19 Targeted to the TRAC Locus for Improved Safety and Efficacy First-of-Kind Off-the-Shelf CAR T-cell Therapy Derived from Renewable Master iPSC Line Engineered to Uniformly Lack TCR Expression and Express Novel 1XX CAR19 1XX CAR19: Novel chimeric an
Slide 33: FT819 In Vivo Tumor Clearance vs Primary CAR T Cells
FT819 Off-the-Shelf CAR19 T-Cell Product Novel CAR19 Targeted to the TRAC Locus for Improved Safety and Efficacy In Vivo Head-to-Head Tumor Clearance vs. Primary CAR T Cells Control Tumor Only FT819 (3x) Primary CART Acute Lymphoblastic Leukemia 10 11 10 10 10 9 FT819 10 8 Tumor only 10 7 Control Pr
Slide 34: Foundational IP – Recently Issued Composition of Matter Patents
Off-the-Shelf CAR T-Cell Franchise Foundational IP – Recently Issued Composition of Matter Patents • U.S. Patent Number 10,287,606 entitled “Genomic Engineering of Pluripotent Cells” – Issued May 2019 (FATE owned) – A cell or population thereof, wherein (i) the cell is an induced pluripotent stem ce
Slide 35: ONO Pharmaceutical Collaboration
ONO Pharmaceutical Collaboration Off-the-Shelf iPSC-derived CAR T-Cell Product Candidates Product 1 Product 2 CAR T-cell targeting Antigen “ND” CAR T-cell targeting Antigen “ND” Tumor Type Lymphoblastic leukemia Solid tumor Binding Domain FATE to contribute ONO to contribute Preclinical Funding Up t
Slide 36: Next-Generation CRISPR Editing Technologies
Next-Generation CRISPR Editing Technologies MAD7 CRISPR Nuclease • Patent-protected, RNA-guided, Class 2 Type V CRISPR nuclease isolated from Eubacterium rectale • Improved features over commonly-used CRISPR-Cas9 nucleases: – Different PAM recognition sequences and cut efficiencies – Reduced sizes a
Slide 37: 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 38: Financial Summary
Financial Summary As of September 30, 2019 Three Months Ended September 30, 2019 Revenue $2.4M Operating Expense, Adjusted 1 $25.0M Cash & Cash Equivalents $303.0M Employees 159 Total Shares Outstanding 2 89.6M [1] Excludes non-cash stock-based compensation expense of approximately $4.6M. [2] Includ
Slide 39: Better Cells For Better Therapies
Better Cells For Better Therapies™ Page - 39 -
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