Fate Therapeutics, Inc. · NASDAQ:FATE
Fate Therapeutics — Investor Presentation (January 2021)
53 slides from Fate Therapeutics's investor presentation, January 2021
Slide 1: Better Cells For Better Therapies: Programmed Cellular Immunotherapies
Better Cells For Better Therapies™ Programmed Cellular Immunotherapies Leading the Development of Off-the-Shelf Cell-based Cancer Immunotherapies using Clonal Master Engineered iPSC Lines January 2021 -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: A Remarkable 2-Year Journey of Firsts
A Remarkable 2-Year Journey of Firsts Building the Leading Off-the-Shelf NK Cell Cancer Immunotherapy Company Best-in-Class Lymphoma Best-in-Class Myeloma Solid Tumors FT500 FT516 FT596 FT538 FT576 • FT516 + mAbs • FT538 + mAbs • CAR MICA/B • ONO Product 2 • Janssen Targets July 2018 Sept 2020 1st I
Slide 4: Changing the Game in Cell Therapy: Necessary Hurdles
Changing the Game in Cell Therapy Necessary Hurdles to Overcome to Change the Game 1. Multiplexed Engineering. Embed multiple elements of synthetic biology to deliver multiple mechanisms of action, increase therapeutic efficacy and reduce toxicity 2. Uniform Product. Minimize sources of variability
Slide 5: Changing the Game in Cell Therapy: Universal, Off-the-Shelf Cell Products
Changing the Game in Cell Therapy 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 & Complete Characteriz
Slide 6: Unique Advantages of Human iPSCs
Unique Advantages of Human iPSCs Single-cell Isolation, Characterization & Selection A Single Human Induced Pluripotent Stem Cell (iPSC) A renewable source for making cell products Unlimited Clonal Single iPSC Clone Potential to Differentiate Expansion into 200+ Cell Types Multiplexed Master Cell Li
Slide 7: Creating a Clonal Master Engineered iPSC Line
Unique Advantages of Human iPSCs Creating a Clonal Master Engineered iPSC Line Single-cell iPSC Isolation, Clonal Master Engineered Cell Population Engineering Characterization and Selection iPSC Line ✓ Determination of copy number ✓ Confirmation of genomic stability ✓ Confirmation of transgene inte
Slide 8: The Making of Bona Fide NK Cells from Clonal Master iPSC Bank
The Making of Bona Fide NK Cells from Clonal Master Engineered iPSC Bank Robust cGMP Process iPSCs iCD34s iNKs Day 0 Day 10 Day 44a SSEA4 CD45 CD34 TRA181 CD43 CD56 ➢ Homogeneous cell product > 1 million-fold expansion ➢ 100s-1,000s doses per campaign ➢ Low-cost per dose cGMP production 106 iPSCs >
Slide 9: Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Pipeline
Off-the-Shelf, iPSC-derived 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 FT536 Multi-faceted Inn
Slide 10: Initial Clinical Validation
Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Franchise Initial Clinical Validation Clinical experience supports the transformative potential of iPSC Product Platform • Experience – 35+ patients dosed with 150+ doses of iPSC-derived NK cells (FT500, FT516, FT596, FT538) – Treated diseases
Slide 11: B-cell Malignancy Franchise
B-cell Malignancy Franchise - 11 -
Slide 12: FT516: hnCD16 NK Cell Product Candidate
FT516: 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 recognize, bind
Slide 13: FT516: High-Affinity, Non-Cleavable CD16a Fc Receptor
FT516: hnCD16 NK Cell Product Candidate Our Novel High-Affinity, Non-Cleavable CD16a Fc Receptor for Enhanced ADCC Proprietary High-affinity, Non-cleavable CD16a Resistance to Activation-induced Shedding as Prolonged Survival In Vivo as (hnCD16) Fc Receptor for Enhanced ADCC Compared to Healthy Dono
Slide 14: FT516-101: Phase 1 Study Design with Rituximab
FT516-101: B-Cell Lymphoma in Combination with Rituximab Phase 1 Study Design: Multiple Doses over Multiple Cycles Cyclophosphamide: 500 mg/m2 IV x 3 days Fludarabine: 30 mg/m2 IV x 3 days IL-2: 6M units sc with each FT516 dose Up to 6 doses of FT516 Rituximab: 1 dose at 375 mg/m2 IV per cycle Regim
Slide 15: FT516-101: Patient Baseline Characteristics
FT516-101: B-Cell Lymphoma in Combination with Rituximab Phase 1 Study: Patient Baseline Characteristics Prior Systemic Therapy FT516 Dose Lymphoma Cohort Subject # Age / Sex Type Regimen Best Response DoR 1) R-CHOP PR 1 month 2005 50 / M DLBCL 2) Flu/Cy → Yescarta CR 8 months 3) R-ICE PD NA 1) R-CE
Slide 16: FT516-101: Safety, Tolerability and Protocol-Defined Response
FT516-101: B-Cell Lymphoma in Combination with Rituximab Phase 1 Safety, Tolerability and Protocol-Defined Response Prior Systemic Therapy FT516-Related Safety Immunogenicity1 FT516 Dose Subject With Relapsed / Bridging FT516 Any Grade Any Grade Any Grade Grade ≥ 3 T-cell B-cell Post-C2 Cohort # Rit
Slide 17: FT516-101: Initial Clinical Observations
FT516-101: B-Cell Lymphoma in Combination with Rituximab Initial Clinical Observations • Up to 6 doses of FT516 were well-tolerated – No events of any grade of CRS, ICANS, or GvHD – No FT516-related Grade 3 or greater adverse events – No evidence of anti-product T- or B-cell mediated immunogenicity
Slide 18: FT596: Multi-antigen Targeted CAR19 NK Cell Product Candidate
FT596: Multi-antigen Targeted CAR19 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-cleavable C
Slide 19: FT596-101: Phase 1 Dose Escalation Schema
FT596-101: Phase 1 Dose Escalation Schema Parallel Escalation of Single-dose Mono and mAb Combo in BCL and CLL B-cell Malignancies Follicular Lymphoma CLL n = 3- 6/cohort n = 3- 6/cohort A: 9 x 108 cells B1: 9 x 108 cells B2: 9 x 108 cells A: 9 x 108 cells B3: 9 x 108 cells Day 1 Day 1 Day 1 Day 1 D
Slide 20: FT596-101: Patient 2002 Case Study – Patient History
FT596-101: Patient 2002 Case Study – Patient History Dose Cohort 1 Monotherapy (Single dose of FT596 at 30M cells) 76 y/o woman with DLBCL, GCB subtype Best Approximate Prior Regimen Response DOR • Stage II at study entry (mesenteric mass) Rituximab, cyclophosphamide, hydroxydaunomycin, • Medical hi
Slide 21: FT596-101: Patient 2002 Case Study – Clinical Course
FT596-101: Patient 2002 Case Study – Clinical Course Dose Cohort 1 Monotherapy (Single dose of FT596 at 30M cells) • First FT596 Single-dose Treatment Cycle − Single-dose monotherapy at 3 x 107 cells • Second FT596 Single-dose Treatment Cycle − Single-dose monotherapy at 3 x 107 cells − Administered
Slide 22: FT596-101: Patient 2002 Case Study – Safety
FT596-101: Patient 2002 Case Study – Safety Dose Cohort 1 Monotherapy (Single dose of FT596 at 30M cells) • No dose-limiting toxicities (DLTs) Adverse Events of Interest Cycle 1 Cycle 2 • No FT596-related serious adverse events (SAEs) Any Grade • Safety profile of adverse events (AEs) of interest Cy
Slide 23: FT596-101: Patient 2002 Case Study – Activity & Pharmacokinetics
FT596-101: Patient 2002 Case Study – Activity & Pharmacokinetics Dose Cohort 1 Monotherapy (Single dose of FT596 at 30M cells) • Partial response at Study Day 29 following first FT596 single-dose cycle • Deepening of response at Study Day 75 following second FT596 single-dose cycle • DoR = 3.7 month
Slide 24: Multiple Myeloma Franchise
Multiple Myeloma Franchise - 24 -
Slide 25: FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate
FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate First-ever CRISPR-edited iPSC-derived Cell Therapy Engineered with Three Components to Enhance Multiple Mechanisms of Innate Immunity hnCD16 “Universal Secondary Engager” hnCD16: High-affinity 158V, non-cleavable CD16 Fc receptor that has be
Slide 26: FT538: Enhancing Multiple Mechanisms of Innate Immunity
FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate Enhancing Multiple Mechanisms of Innate Immunity Enhanced NK Cell Persistence & Metabolic Fitness Enhanced NK Cell ADCCa a Bjordahl et al. ASH Annual Meeting 2019 - 26 -
Slide 27: FT538-101: Relapsed / Refractory Multiple Myeloma
FT538-101: Relapsed / Refractory Multiple Myeloma Multi-dose Combination with CD38-targeted and SLAMF7-targeted mAb DL1 = 100M cells / dose DL3 = 1.0B cells / dose DL2 = 300M cells / dose DL4 = 1.5B cells / dose - 27 -
Slide 28: FT576: Multi-antigen Targeted CAR-BCMA NK Cell Product Candidate
FT576: Multi-antigen Targeted CAR-BCMA NK Cell Product Candidate Potential Best-in-Class Cell-based Cancer Immunotherapy for Multiple Myeloma Engineered with Four Anti-tumor Modalities for Multiple Myeloma hnCD16: High-affinity 158V, non-cleavable CD16 Fc receptor that has been modified to augment a
Slide 29: FT576: BCMA Binding Domain with Differentiated Activation Threshold
FT576: Multi-antigen Targeted CAR-BCMA NK Cell Product Candidate BCMA Binding Domain with Differentiated Activation Threshold No Exogenous Cytokine No FT576 FT576 Tumor Untreated Batch #1 Batch #2 BCMA-CART ✓ Validated CAR BCMA in diffuse large B cell lymphoma, follicular lymphoma, mantle cell lymph
Slide 30: AML Franchise
AML Franchise - 30 -
Slide 31: Rationale for NK Cell Therapy in AML
Rationale for NK Cell Therapy in AML Clinical Precedent with Non-Engineered Allogeneic NK Cell Therapy • 300+ AML/MDS patients treated with allogeneic NK cellsa • Numerous clinical studies in relapsed / refractory AML have showna: – CR rates = 25-35% – No GvHD Jeffrey S. Miller, MD – Minimal CRS / n
Slide 32: Multiple Ongoing Phase 1 Studies in Relapsed / Refractory AML
Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Franchise Multiple Ongoing Phase 1 Studies in Relapsed / Refractory AML Program FT516 Monotherapy FT538 Monotherapy FT538 + anti-CD38 mAb Dose Three dose levels ranging from Four dose levels ranging from Four dose levels ranging from 90-900 mi
Slide 33: Solid Tumor Franchise
Solid Tumor Franchise - 33 -
Slide 34: The NK-Cell-Mediated Cancer Immunity Cycle
Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Franchise The NK-Cell-Mediated Cancer Immunity Cycle Bridging Innate and Adaptive Immunity - 34 -
Slide 35: Multi-antigen Targeting: Enhanced Innate Immunity + CAR
Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Franchise Multi-antigen Targeting: Enhanced Innate Immunity + CAR FT536 FT5xx FT5xx CAR-B7H3 Janssen CAR - 35 -
Slide 36: FT500-101: Phase 1 Dose Escalation in Advanced Solid Tumors
FT500-101: First-ever U.S. Clinical Study of iPSC-derived Cell Product Phase 1 Dose Escalation in Advanced Solid Tumors Up to 6 doses over 45 days Cy: 300 mg/m2 IV x 2 days Flu: 25 mg/m2 IV x 2 days Prior to Cycle 1 only FT500 • Regimen A: Monotherapy (n=9) – Salvage setting with patients having pro
Slide 37: FT500-101: Clinical Objectives
FT500-101: First-ever U.S. Clinical Study of iPSC-derived Cell Product Clinical Objectives Assessment of Safety & Tolerability as Monotherapy and in Combination with Checkpoint Inhibitor Assess Novel Paradigm Key Clinical Read-outs ➢ First-ever U.S. clinical study of iPSC-derived cell ➢ FT500 safety
Slide 38: FT500-101: Dose Escalation Clinical Results
FT500-101: Dose Escalation Clinical Results Phase 1 Dose Escalation in Advanced Solid Tumors Multi-dosing NK Cells • All 15 patients completed Cycle 1 (3 doses) 200 Doses Cy/Flu • 13 patients advanced to Cycle 2, with 11 of 150 Cells / L 13 patients completing Cycle 2 (3 additional 100 doses) 50 •
Slide 39: FT500-101: Dose Escalation Clinical Results, Safety and Case Study
FT500-101: Dose Escalation Clinical Results Phase 1 Dose Escalation in Advanced Solid Tumors Patient Case Study Safety (300M FT500 cells combined with ICI) • No dose-limiting toxicities, and no SAEs or Grade ≥ 3 AEs considered related to FT500, were observed • No cases of cytokine release syndrome,
Slide 40: FT500-101: Phase 1 Dose Expansion Ongoing
FT500-101: Phase 1 Dose Expansion Ongoing Targeting Solid Tumors Amendable to NK Cell Accessibility, Recognition, and Killing Overcoming Resistance to Checkpoint Inhibitor Therapy in Advanced Solid Tumors FT500 + IL2 + ICI FT500 mediates both direct tumor lysis Patient who progressed and T-cell recr
Slide 41: FT516-102: First Patient Treated with PD-L1-targeted mAb
FT516-102: hnCD16 NK Cell Product Candidate for Advanced Solid Tumors First Patient Treated in Combination with PD-L1-targeted mAb Cyclophosphamide: 500 mg/m2 IV x 3 days Fludarabine: 30 mg/m2 IV x 3 days IL-2: 6M units sc with each FT516 dose Up to 6 doses of FT516 Avelumab: 800 mg every 2 weeks IV
Slide 42: FT536: Multi-antigen Targeted CAR-MICA/B NK Cell Product Candidate
FT536: Multi-antigen Targeted CAR-MICA/B NK Cell Product Candidate Pan-tumor Targeting Strategy for Solid Tumors Engineered with Four Anti-tumor Modalities for Solid Tumors hnCD16: High-affinity 158V, non-cleavable CD16 Fc receptor that has been modified to augment antibody-dependent cellular cytoto
Slide 43: FT536: Novel Pan-tumor Targeting Strategy for Solid Tumors
FT536: Multi-Targeted CAR-MICA/B NK Cell Product Candidate Novel Pan-tumor Targeting Strategy for Solid Tumors ✓ MICA/B are induced by cellular stress and transformation, and their expression has been reported for many cancer types ✓ NKG2D, an activating receptor expressed on NK and T cells, targets
Slide 44: iPSC-derived CAR T Cells
iPSC-derived CAR T Cells - 44 -
Slide 45: FT819: Off-the-Shelf CAR19 T-Cell Product Candidate
FT819: Off-the-Shelf CAR19 T-Cell Product Candidate Collaboration with Memorial Sloan Kettering Cancer Center First-of-Kind Off-the-Shelf CAR T-cell Therapy Derived from Renewable Master iPSC Line Engineered to Uniformly Express Novel 1XX CAR19 and Knock-out TCR 1XX CAR19: Novel chimeric antigen rec
Slide 46: FT819: Enhanced Tumor Control vs. Primary CAR T Cells
FT819: Enhanced Tumor Control vs. Primary CAR T Cells Disseminated Xenograft Model of Lymphoblastic Leukemia Valamehr et al. Festival of Biologics Annual Meeting 2020 - 46 -
Slide 47: FT819-101: Phase I Dose Escalation Schema
FT819-101: Phase I Dose Escalation Schema Concurrent and Independent Dose Escalation in BCL, CLL and pre-B ALL 3 Indications x 3 Treatment Regimens Regimen A1 Regimen A Regimen B + IL-2 Day 1 Day 1 Day 1 Day 3 Day 5 MPAD or MAD DL4+IL2 MAD DL4 DL3 DL3 DL3 MPAD or DL3+IL2 DL3 DL2 DL2 DL2 DL1 = 3 × 10
Slide 48: Collaborations
Collaborations - 48 -
Slide 49: Janssen Cancer Immunotherapy Collaboration (April 2020)
Janssen Cancer Immunotherapy Collaboration (April 2020) Off-the-shelf, iPSC-derived CAR NK Cell and CAR T-Cell Collaboration Oncology Innovation • Proprietary antigen domains contributed by Janssen • Up to 4 targets including hematologic malignancies and solid tumors • Substantial investment in next
Slide 50: ONO Cancer Immunotherapy Collaboration (September 2018)
ONO Cancer Immunotherapy Collaboration (September 2018) Off-the-shelf, iPSC-derived CAR T-Cell Collaboration Oncology Innovation • Proprietary antigen domain contributed by Ono • Targeting solid tumors • Potential to include additional antigen binding domains Strategic Collaboration • FATE leads pre
Slide 51: Financials
Financials - 51 -
Slide 52: Financial Summary
Financial Summary As reported in Company’s Consolidated Financial Statements Three Months Ended September 30, 2020 Revenue $7.6M Operating Expense $39.0M Cash & Cash Equivalents1 $934M Employees 250+ Total Shares Outstanding2 100.9M 1 On an as adjusted basis to include January 2021 common stock offe
Slide 53: Better Cells For Better Therapies
Better Cells For Better Therapies™ - 53 -
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