Fate Therapeutics, Inc. · NASDAQ:FATE
Fate Therapeutics — Investor Presentation (October 2018)
44 slides from Fate Therapeutics's investor presentation, October 2018
Slide 1: Programmed Cellular Immunotherapies: Corporate Overview
Better Cells For Better Therapies™ Programmed Cellular Immunotherapies Corporate Overview October 2018 -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: Early Innings of Cell Therapy Development
Early Innings of Cell Therapy Development Patient-derived CAR-T Cell Immunotherapy Patient ~2-3 weeks / single dose How do we Build on Early Successes and Transition From a Personalized Process to the Delivery of Optimized Cell Products? -3-
Slide 4: Our Approach to Next-Generation Cellular Immunotherapy
Fate Therapeutics Our Approach to Next-Generation Cellular Immunotherapy Donor-derived Cell Products 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 modulation Ex viv
Slide 5: First-in-Class Cellular Immunotherapy Pipeline
First-in-Class Cellular Immunotherapy Pipeline PROGRAM PRECLINICAL CLINICAL RIGHTS IMMUNO-ONCOLOGY FATE-NK100 – AML Phase 1 Worldwide FATE-NK100 – Ovarian Phase 1 Worldwide FATE-NK100 – Solid Tumor mAb Combo Phase 1 Worldwide FT500 (iNK Cell) OTS Checkpoint Inhibitor Combination Worldwide FT516 (Eng
Slide 6: ProTmune: Next-Generation Graft for Allogeneic HCT
ProTmune™ Next-Generation Graft to Enhance Curative Potential of Allogeneic HCT • Allogeneic HCT is performed with curative intent – Orphan hematologic malignancies (e.g., AML, ALL, MDS) • The donor cell graft is the drug – Donor stem cells engraft and reconstitute the patient’s blood and immune sys
Slide 7: A Next-Generation Hematopoietic Cell Graft for Allogeneic HCT
ProTmune™ A Next-Generation Hematopoietic Cell Graft for Allogeneic HCT ProTmune™ FT1050 + FT4145 Small molecule programmed mobilized peripheral blood graft ➢ Ex vivo programming to promote stem cell engraftment and attenuate T-cell signaling ➢ On-site manufacture integrates into current clinical pr
Slide 8: PROTECT Phase 2 Study Design
ProTmune™ PROTECT Phase 2 Study Fast Track Designation 60 Subjects, Double-blinded, Randomized, Controlled Controlled, Blinded ProTmune Cell Graft (n=30) Randomized, Standard-of-care mPB Cell Graft (n=30) Day 100 Day 365 Primary Endpoint Secondary Endpoint Cumulative Incidence of GvHD-free, Relapse-
Slide 9: PROTECT Phase 1 Study: Neutrophil Engraftment
ProTmune™ PROTECT Phase 1 Study: Neutrophil Engraftment Age / Neutrophil Day 100 Steroid Days to Cancer Overall Days on Subject Disease Sex Engraftment aGvHD Responsive Resolution † Relapse Survival Study 1 MDS 66 / F Day 14 None --- --- No No* 228 2 AML 56 / F Day 18 None --- --- No Yes 343 3 AML 6
Slide 10: PROTECT Phase 1 Study: Day 100 Acute GvHD
ProTmune™ PROTECT Phase 1 Study: Day 100 Acute GvHD Age / Neutrophil Day 100 Steroid Days to Cancer Overall Days on Subject Disease Sex Engraftment aGvHD Responsive Resolution † Relapse Survival Study 1 MDS 66 / F Day 14 None --- --- No No* 228 2 AML 56 / F Day 18 None --- --- No Yes 343 3 AML 66 /
Slide 11: PROTECT Phase 1 Study: Cancer Relapse
ProTmune™ PROTECT Phase 1 Study: Cancer Relapse Age / Neutrophil Day 100 Steroid Days to Cancer Overall Days on Subject Disease Sex Engraftment aGvHD Responsive Resolution † Relapse Survival Study 1 MDS 66 / F Day 14 None --- --- No No* 228 2 AML 56 / F Day 18 None --- --- No Yes 343 3 AML 66 / F Da
Slide 12: FATE-NK100: 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 13: 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 - 13 -
Slide 14: Multi-pronged Clinical Development Strategy
FATE-NK100 Multi-pronged Clinical Development Strategy VOYAGE • Single IV infusion; accelerated dose-escalation; up to 3 dose levels Refractory / • 10-patient expansion at MTD Relapsed AML • Key read-outs: NK cell persistence; anti-leukemia activity; CRs • Single IP infusion; accelerated dose-escala
Slide 15: The VOYAGE Study: Initial Clinical Observations
FATE-NK100 The VOYAGE Study: Initial Clinical Observations VOYAGE • Single IV infusion; accelerated dose-escalation; up to 3 dose levels Refractory / • 10-patient expansion at MTD Relapsed AML • Key read-outs: NK cell persistence; anti-leukemia activity; CRs Dose Cohort 1 (1x107 TNC/kg) Dose Cohort
Slide 16: The VOYAGE Study: Initial Clinical Observations
FATE-NK100 The VOYAGE Study: Initial Clinical Observations VOYAGE • Single IV infusion; accelerated dose-escalation; up to 3 dose levels Refractory / • 10-patient expansion at MTD Relapsed AML • Key read-outs: NK cell persistence; anti-leukemia activity; CRs Dose Cohort 1 (1x107 TNC/kg) Dose Cohort
Slide 17: The APOLLO Study: Initial Clinical Observations
FATE-NK100 The APOLLO Study: Initial Clinical Observations • Single IP infusion; accelerated dose-escalation; up to 3 dose levels APOLLO • 10-patient expansion at MTD Recurrent Ovarian • Key read-outs: NK cell persistence; ORR by RECIST Dose Cohort 1 (1x107 TNC/kg) Dose Cohort 2 (2x107 TNC/kg) Age /
Slide 18: The APOLLO Study: Initial Clinical Observations
FATE-NK100 The APOLLO Study: Initial Clinical Observations • Single IP infusion; accelerated dose-escalation; up to 3 dose levels APOLLO • 10-patient expansion at MTD Recurrent Ovarian • Key read-outs: NK cell persistence; ORR by RECIST Dose Cohort 1 (1x107 TNC/kg) Dose Cohort 2 (2x107 TNC/kg) Age /
Slide 19: Changing the Game in Cell Therapy
Changing the Game in Cell Therapy Key Features Cell Therapy 1.0 Cell Therapy 2.0 Cell Source Patient Cells Master Cell Line Genetic Engineering Random & Variable Uniform & Complete Product Consistency Heterogeneous Homogeneous Characterization Imprecise Well-defined Manufacturing Personalized Off-th
Slide 20: Changing the Game in Cell Therapy
Changing the Game in Cell Therapy Key Features Cell Therapy 1.0 Cell Therapy 2.0 Cell Source Patient Cells Master Cell Line Genetic Engineering Random & Variable Uniform & Complete Product Consistency Heterogeneous Homogeneous Characterization Imprecise Well-defined Manufacturing Personalized Off-th
Slide 21: Foundational Role of Fate Therapeutics' Scientific Founders
Human Induced Pluripotent Stem Cells Foundational Role of Fate Therapeutics’ Scientific Founders Rudolf Jaenisch Sheng Ding Fate Therapeutics’ iPSC product platform is supported by an IP portfolio of 100+ issued patents and 100+ pending patent applications - 21 -
Slide 22: Pioneering the Use of Master iPSC Lines
Human Induced Pluripotent Stem Cells Pioneering the Use of Master iPSC Lines for Making Cell Products 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 Compositi
Slide 23: Universal Off-the-Shelf Cell Products
Universal Off-the-Shelf Cell Products Pioneering the Use of Master iPSC Lines for Making Cell Products Human Induced Multiplexed Engineering Renewable Pluripotent Stem Cells Single-cell Clonal Selection Master iPSC Line Universal | Off-the-Shelf | Cell Products iT Cells iNK Cells iCD34+ Cells Other
Slide 24: Collaborations with Top Investigators and Leading Centers
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 - 24 -
Slide 25: Properties of Natural Killer Cells for Cancer Immunotherapy
Universal Off-the-Shelf NK Cell Products Properties of Natural Killer Cells for Cancer Immunotherapy • Use of mismatched donor cells has been shown to be safe and well-tolerated ➢ No reports of GvHD, cytokine release syndrome or neurotoxicity • Multi-faceted effector function ➢ Anti-tumor activity i
Slide 26: Robust Differentiation Protocol to Create iPSC-derived NK Cells
Universal Off-the-Shelf NK Cell Products Robust Differentiation Protocol to Create iPSC-derived NK Cells 1 iPSC Hundreds of doses of homogeneous NK cells for off-the-shelf delivery to patients - 26 -
Slide 27: Potency of iPSC-derived vs. Peripheral Blood NK Cells
Universal Off-the-Shelf NK Cell Products Potency of iPSC-derived vs. Peripheral Blood NK Cells SKOV3 (Ovarian Cancer) Killing Assay Peripheral Blood NK Cells Off-the-Shelf iPSC-derived NK Cells Off-the-Shelf iPSC-derived NK Cells Tumor Killing - 27 -
Slide 28: Collaboration with MCT for GMP Manufacture
Universal Off-the-Shelf NK Cell Products Collaboration with MCT for GMP Manufacture > 95% pure for CD56+ cells Cryopreserved for off- Molecular and Cellular Therapeutics the-shelf utilization 33,000 SF State-of-Art GMP Facility - 28 -
Slide 29: Three Product Candidates Undergoing IND-Enabling Activities
Universal Off-the-Shelf NK Cell Products Three Product Candidates Undergoing IND-Enabling Activities ► IND Submitted to FDA • Clonal master iPSC line • NK differentiation FT500 • Homogenous cell product Master iPSC Line + hnCD16 • hnCD16 insertion • Clonal master iPSC line • NK differentiation FT516
Slide 30: FT500: Combination with Checkpoint Inhibitors
FT500: Combination with Checkpoint Inhibitors Rescue Therapy in Patients with B2M Mutations • B2M mutations are enriched in patients who are resistant to checkpoint therapy Survival (~30%) • Loss of B2M associated with poor survival • MHC Class 1 null tumor cells are highly susceptible to killing by
Slide 31: FT500: Proposed First-in-Human Study
FT500: Proposed First-in-Human Study Multiple Cycles of FT500 + Checkpoint Inhibitor Screening Window: Refractory Solid Tumors -14 --------One Cycle-------- - 31 -
Slide 32: FT516: Engineered hnCD16+ NK Cell Product
FT516: Engineered hnCD16+ NK Cell Product From a Single iPSC Clone to a Homogeneous Effector Cell Population Renewable Engineered Engineered hnCD16 iNK Pluripotent Cell Line Cells for ADCC CD16 CD16 hiPSCs CD56 (NK cells) 1 iPSC 1x106 hnCD16 iNKs Clonal Expansion 1M iPSCs 1x1012 hnCD16 iNKs - 32 -
Slide 33: FT516: Universal Cell Engager of Monoclonal Antibody
FT516: Engineered hnCD16+ NK Cell Product Universal Cell Engager of Monoclonal Antibody in Liquid and Solid Tumors Engineered high-Affinity non-Cleavable CD16 Fc Receptor FDA-approved Monoclonal Antibodies Modified form of CD16a IgG antibody-binding receptor resists shedding upon activation hnCD16 +
Slide 34: FT516: Combination with Herceptin
FT516: Combination with Herceptin In Vivo POC Untreated Herceptin Herceptin + FT516 Luc-SKOV3 (Ovarian Cancer) Tumor Image Analysis D4 Initiation of treatment @ Day 5 D18 D39 - 34 -
Slide 35: FT516: Combination with Rituxan
FT516: Combination with Rituxan Enhanced In Vivo Survival CD20+ Raji Tumor implantation 1st NK cells 2nd NK cells 3rd NK cells 4th NK cells Days -1 0 3 4 7 11 14 18 21 25 28 - 35 -
Slide 36: FT538: Engineered hnCD16+ / CD38-null NK Cell Product
FT538: Engineered hnCD16+ / CD38-null NK Cell Product For Combination with Darzalex for Multiple Myeloma FT538 • hnCD16 insertion • CD38 knock-out CD16 • Single cell selected • Master iPSC line generated • NK differentiation Renewable Engineered Master Pluripotent Cell Line CD38 • CD38 is expressed
Slide 37: Universal Off-the-Shelf CAR T-Cell Products: MSK Collaboration
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 38: FT819: TRAC-encoded CAR Expression
FT819: TRAC-encoded CAR Expression Engineering Primary T Cells vs. Single iPSC Clone 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 Clone ✓ Complete elimin
Slide 39: FT819: CAR19 TCR-null T-Cell Product Candidate
FT819: CAR19 TCR-null T-Cell Product Candidate In Vitro Cytokine Production and CD19 Antigen Specificity - 39 -
Slide 40: 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 • Propensity to differentiate into 200+ cell types Expansion & Banking Working Cell Banks Unlimited Supply of Master Cell Working Cell Banks Clonal M
Slide 41: Off-the-Shelf CAR T-Cell Collaboration with ONO Pharmaceutical
iPSC Product Platform Off-the-Shelf CAR T-Cell Collaboration with ONO Pharmaceutical • 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 joint
Slide 42: Financial Summary
Financial Summary Three Months Ended June 30, 2018 Revenue $1.0M Operating Expense, Adjusted 1 $13.3M Cash & Cash Equivalents 2 $228.0M Employees 90 Total Shares Outstanding 2, 3 77.5M [1] Excludes one-time expense of $5.3m associated with the in-license of additional IP from Memorial Sloan Ketterin
Slide 43: Projected Milestones: Next Six Months
Projected Milestones: Next Six Months • ProTmune: Next-Generation Hematopoietic Cell Graft – Present 1-year follow-up data from PROTECT Phase 1 study at 2018 ASH Annual Meeting – Continue to enroll randomized, controlled and double-blinded PROTECT Phase 2 study • NK100: First-in-class Adaptive Memor
Slide 44: Better Cells For Better Therapies
Better Cells For Better Therapies™ - 44 -
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