Fate Therapeutics, Inc. · NASDAQ:FATE
Fate Therapeutics — Investor Presentation (June 2021)
58 slides from Fate Therapeutics's investor presentation, June 2021
Slide 1: 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 June 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: Changing the Game in Cell Therapy
Changing the Game in Cell Therapy iPSC-derived, Off-the-Shelf Cell Therapies to Eradicate Cancer Multiplexed Engineering Incorporate multiple mechanisms of action to eradicate cancer Treatment Paradigm Mass Production Flexible out-patient treatment Reliable manufacturing process strategies to drive
Slide 4: Unique Challenges Confronting the Cell-based Cancer Immunotherapy Field
Unique Challenges Confronting the Cell-based Cancer Immunotherapy Field Key Considerations in Developing Best-in-Class Cell Products 1. Starting Cell Source. Whether from the patient (autologous) or a healthy donor (allogeneic), the starting cell source is variable and can create batch-to-batch inco
Slide 5: Master Cell Lines Enable Mass Production of Best-in-Class Cell Products
Master Cell Lines Enable Mass Production of Best-in-Class Cell Products Transitioning the Field from a Process-centric to a Product-centric Therapeutic Paradigm 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 R
Slide 6: Unique Biological Properties of Human iPSCs
Unique Biological Properties of Human iPSCs Single-cell Isolation, Characterization & Selection for Creation of Master Engineered Cell Lines A Single Human Induced Pluripotent Stem Cell (iPSC) A renewable source for making cell products Unlimited Clonal Single iPSC Clone Potential to Differentiate E
Slide 7: iPSC Product Platform
iPSC Product Platform Disruptive Approach Enabling 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 event) Renewable Starti
Slide 8: Robust In-house GMP Manufacture of Cryopreserved Off-the-Shelf Cell Products
iPSC Product Platform Robust In-house GMP Manufacture of Cryopreserved Off-the-Shelf Cell Products iPSCs iCD34s iNKs Day 0 Day 10 Day 44 SSEA4 CD45 CD34 TRA181 CD43 CD56 ➢ Homogeneous cell product ➢ 100s-1,000s doses per campaign > 1 million-fold expansion ➢ Low-cost per dose cGMP production 106 iPS
Slide 9: The Leading Developer of iPSC-derived Cell-based Cancer Immunotherapies
iPSC Product Platform The Leading Developer of iPSC-derived Cell-based Cancer Immunotherapies Disruptive Technology Platform: highly-edited master iPSC lines; worked closely with FDA to pioneer first-ever clinical investigation in U.S. of iPSC-derived cell therapy Scalable Manufacture: demonstrated
Slide 10: Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Franchise
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 FT573 Multi-facet
Slide 11: B-cell Malignancy Franchise
B-cell Malignancy Franchise - 11 -
Slide 12: Novel High-Affinity, Non-Cleavable CD16a Fc Receptor
Novel High-Affinity, Non-Cleavable CD16a Fc Receptor Optimizing Antibody-Dependent Cellular Cytotoxicity for Use with mAb Therapy Novel High-affinity, Non-cleavable CD16 (hnCD16) NK Cell Fc Receptor for Enhanced ADCC High Affinity Non-Cleavable Only 15% of the population TME causes shedding of has C
Slide 13: FT516 & FT596: First-in-Class NK Cell Cancer Immunotherapies
FT516 & FT596: First-in-Class NK Cell Cancer Immunotherapies Optimized Innate & Synthetic Biology hnCD16: High-affinity 158V, non- hnCD16 hnCD16 cleavable CD16 Fc receptor to augment ADCC CAR19 CAR19: Chimeric antigen receptor that targets B-cell antigen CD19 FT516 (optimized for NK cells) FT596 IL-
Slide 14: FT516-101: B-Cell Lymphoma in Combination with Rituximab
FT516-101: B-Cell Lymphoma in Combination with Rituximab Phase 1 Study – Multiple Doses over Multiple Cycles in Out-patient Setting 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 I
Slide 15: FT516-101: Interim Safety, Tolerability, and Response
FT516-101: B-Cell Lymphoma in Combination with Rituximab Phase 1 Study – Interim Safety, Tolerability, and Response Interim Data from FT516 Phase 1 Study in Relapsed / Refractory B-cell Lymphoma Prior Systemic Therapy # Prior # Prior CD20- Prior CD19 Most Recent FT516 Subject # Lymphoma Type Regimen
Slide 16: FT516-101: Interim Clinical Observations
FT516-101: B-Cell Lymphoma in Combination with Rituximab Interim Clinical Observations • Outpatient treatment paradigm of up to 6 doses was well-tolerated – No events of any grade of CRS, ICANS, or GvHD – No FT516-related SAEs or FT516-related Grade ≥3 AEs – No requirement for patient matching; no e
Slide 17: FT596: Multi-antigen Targeted CAR19 NK Cell Product Candidate
FT596: Multi-antigen Targeted CAR19 NK Cell Product Candidate Dual-Antigen Targeting of CD19 and CD20 B-cell Antigens for Best-in-class Potential hnCD16 Synergizes with CD20- Multiple Doses of FT596 Compares Favorably FT596 + CD20-targeted mAb Engage Multiple B-cell Antigens for targeted mAb to Sing
Slide 18: 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 19: FT596-101: Patient 2002 Case Study
FT596-101: Patient 2002 Case Study Dose Cohort 1 Monotherapy (Single dose of FT596 at 30M cells) Patient History Baseline SPD • 76 y/o woman with r/r DLBCL 1292 mm2 • Received 7 prior therapies SUV • Most recently refractory to experimental combo therapy comprised of expanded allogeneic 28 NK cells,
Slide 20: Multiple Myeloma Franchise
Multiple Myeloma Franchise - 20 -
Slide 21: FT538 & FT576: First-in-Class NK Cell Cancer Immunotherapies
FT538 & FT576: First-in-Class NK Cell Cancer Immunotherapies Optimized Innate & Synthetic Biology hnCD16: High-affinity 158V, non- hnCD16 cleavable CD16 Fc receptor to hnCD16 augment ADCC IL-15RF: Interleukin-15 receptor CAR-BCMA fusion to promote survival, proliferation and trans-activation of FT53
Slide 22: Multiple Myeloma Disease Franchise: Planned Phase 1 Studies
Multiple Myeloma Disease Franchise Planned Phase 1 Studies in Relapsed / Refractory MM FT576 FT538 (hnCD16 + IL15RF + CD38KO + CAR- Program (hnCD16 + IL15RF + CD38KO) BCMA) Treatment FT538 +/- daratumumab or FT576 +/- daratumumab elotuzumab Setting Relapsed / Refractory MM Relapsed / Refractory MM D
Slide 23: FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate
FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate A Uniform, Well-Characterized Cell Product Optimized for Innate Immunity - 23 -
Slide 24: FT538: Enhanced Persistence Without Cytokine Support
FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate Enhanced Persistence Without Cytokine Support Primary NK vs. FT538 in NSG Mouse FT516 vs. FT538 in NSG Mouse Primary NK Cells Day 16 Day 16 Cichocki et al. manuscript under review - 24 -
Slide 25: FT538: Enhanced Cytotoxicity vs. PB NK Cells
FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate Enhanced Cytotoxicity vs. PB NK Cells in a Serial Re-stimulation Cytotoxicity Assay Overcome Endogenous NK Cell Deficiencies for Optimized anti-CD38 Activity in Myeloma Transfer effector cells from MM.1R – Round 1 to MM.1R – Round 2 - 25 -
Slide 26: FT538: Enhanced ADCC in Combination with anti-CD38 mAb In Vivo
FT538: hnCD16 + IL-15RF + CD38KO NK Cell Product Candidate Enhanced ADCC in Combination with anti-CD38 mAb In Vivo - 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 BCMA Binding Domain with Differentiated Activation Threshold No Exogenous Cytokine No FT576 FT576 Tumor Untreated Batch #1 Batch #2 BCMA-CART ✓ Novel BCMA binding domain triggers target cell lysis at low levels of BCMA expression (~100
Slide 29: AML Franchise
AML Franchise - 29 -
Slide 30: Rationale for NK Cell Therapy in AML
Rationale for NK Cell Therapy in AML Clinical Precedent with Non-Engineered Allogeneic NK Cells • 300+ AML/MDS patients treated with allogeneic NK cellsa • Numerous clinical studies in relapsed / refractory AML have showna: – CR rates = 20-35% – No GvHD Jeffrey S. Miller, MD – Minimal CRS / neurotox
Slide 31: AML Disease Franchise: Multiple Ongoing Phase 1 Studies
AML Disease Franchise Multiple Ongoing Phase 1 Studies in Relapsed / Refractory AML FT516 FT538 FT538 (UMN IIT) Program (hnCD16) (hnCD16 + IL15RF + CD38 KO) (hnCD16 + IL15RF + CD38 KO) Treatment FT516 Monotherapy FT538 Monotherapy FT538 + daratumumab IL-2 cytokine support / dose No IL-2 cytokine sup
Slide 32: FT516-101: Monotherapy in R/R AML Patient Characteristics
FT516-101: Monotherapy in Relapsed / Refractory AML Patient Characteristics Reflect Extremely Poor Prognosis Risk Profile Last Line of Therapy # of Prior 2017 ELN Risk Subject # Age Lines Category PIF Regimen Response 1001 41 3 Intermediate Yes Ven + Dec Refractory 1003 64 3 Adverse No Ivosidenib Re
Slide 33: FT516-101: Safety, Tolerability & Immunogenicity
FT516-101: Monotherapy in Relapsed / Refractory AML Safety, Tolerability & Immunogenicity FT516-Related Safety Immunogenicity # Cells / # of Any Grade Any Grade Any Grade Grade ≥ 3 Subject # Dose Doses DLT CRS ICANS GvHD AEs SAEs T-cell B-cell 1001 90M 6 No No No No None None No No 1003 90M 6 No No
Slide 34: FT516-101: Best Overall Response
FT516-101: Monotherapy in Relapsed / Refractory AML Best Overall Response Baseline Best Overall Response (BOR) # Cells / # of % Bone Neutrophils Platelets % Bone % Change in Bone BOR Subject # Dose Doses Marrow Blasts (103/µl) (103/µl) Marrow Blasts Marrow Blasts (2017 ELN) 1001 90M 6 6% 0.8 12 0% -
Slide 35: FT516-101: Duration of Anti-leukemic Activity
FT516-101: Monotherapy in Relapsed / Refractory AML Duration of Anti-leukemic Activity Two Patients with CRi Remained in Remission with Ongoing DOR >6 months; No additional therapeutic intervention Evidence of Evolving Response from MLFS to CRi One Patient with CRi Proceeded to allo-HSCT CRi = Compl
Slide 36: FT538-101: Monotherapy in R/R AML Patient Characteristics
FT538-101: Monotherapy in Relapsed / Refractory AML Patient Characteristics Risk Profile Last Line of Therapy # of Prior 2017 ELN Risk Subject # Age Prior Therapy Lines Category PIF Regimen Response 1001 72 Ven + Aza 3 Unknown No Ven + Dec Refractory 7+3 Ven + Dec 1002 78 Ven + Aza 3 Adverse No GTB-
Slide 37: FT538-101: Safety, Tolerability, Immunogenicity & Response
FT538-101: Monotherapy in Relapsed / Refractory AML 100M Cells / Dose: Safety, Tolerability, Immunogenicity & Response FT538-Related Safety Immunogenicity Best Overall Subject % BM Blasts FT538 Any Grade Any Grade Any Grade Grade ≥3 Response # Baseline Doses DLT CRS ICANS GvHD AE T-cell B-cell (2017
Slide 38: FT538-101: Subject 1003 (100M cells)
FT538-101: Monotherapy in Relapsed / Refractory AML Subject 1003 (100M cells) • Patient Characteristics – 79 y.o. male diagnosed with de novo AML in 2017 – Multiple lines of prior therapy ▪ Idarubicin + Ara-C (CR; DOR = 20 months) ▪ Venetoclax + Azacitidine (PR; DOR = 1 month) ▪ GTB-3550 (investigat
Slide 39: FT516 / FT538: Initial Clinical Observations
FT516 / FT538: Monotherapy in Relapsed / Refractory AML Initial Clinical Observations • Phase 1 studies have enrolled an unfavorable patient population (n=12) – Median of 3 prior lines, with 11 patients refractory to their last prior therapy – 9 patients with adverse risk profile (with 1 patient unk
Slide 40: FT538 + daratumumab for Targeting of CD38 on Leukemic Blasts
Relapsed / Refractory Acute Myeloid Leukemia FT538 + daratumumab for Targeting of CD38 on Leukemic Blasts CD38 expression from bone marrow NK cells in combination with CD38-targeted FT538 uniquely elicits an anti-tumor response against samples was found in 239 of 241 mAb significantly enhances anti-
Slide 41: Solid Tumor Franchise
Solid Tumor Franchise - 41 -
Slide 42: Developing Multi-functional Off-the-Shelf Cell Products for Solid Tumors
Developing Multi-functional Off-the-Shelf Cell Products for Solid Tumors One Therapeutic Modality Incorporating Multiple Mechanisms in Fight Against Cancer CAR 1 Engager Secretion CAR 2 hnCD16 Immune Evasion + TCR - Endogenous TCR Off-the-Shelf Persistence / Multiplexed Engineered TME Redirect Expan
Slide 43: Evolution Toward Multiplexed-Engineered Cell-based Cancer Immunotherapies
Off-the-Shelf, iPSC-derived NK Cell Cancer Immunotherapy Franchise Evolution Toward Multiplexed-Engineered Cell-based Cancer Immunotherapies FT536 FT573 FT5xx CAR-B7H3 Janssen CAR - 43 -
Slide 44: FT538-102: First-Ever CRISPR-edited iPSC-derived Cell Therapy
FT538-102: First-Ever CRISPR-edited iPSC-derived Cell Therapy Incorporates Three Functional Components to Enhance Innate Immunity • Innate immunity is severely compromised in patients with cancer – Depleted / dysfunctional NK cell compartment – Inferior ADCC capacity due to naturally-occurring, low-
Slide 45: FT536: Multi-targeted CAR-MICA/B NK Cell Product Candidate
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 46: FT573: Multi-targeted CAR-B7H3 NK Cell Product Candidate
FT573: Multi-targeted CAR-B7H3 NK Cell Product Candidate Novel Pan-tumor Targeting Strategy for Oncogenic Cells and Prevention of Metastasis ✓ B7H3 (CD276) belongs to the B7 superfamily of immune checkpoint molecules and is overexpressed in a wide variety of cancers, often associated with poor progn
Slide 47: FT500-101: First-ever U.S. Clinical Study of iPSC-derived Cell Product
FT500-101: First-ever U.S. Clinical Study of iPSC-derived Cell Product Phase 1 Dose Expansion Ongoing in Advanced Solid Tumors FT500 Dose Expansion Program (non-engineered iPSC-derived NK cell) Rationale Assess direct tumor lysis and T-cell recruitment / activation to re-sensitize ICI- resistant tum
Slide 48: FT516-102: Combination with PDL1-targeted mAb for Advanced Solid Tumors
FT516-102: Combination with PDL1-targeted mAb for Advanced Solid Tumors Phase 1 Dose Escalation Ongoing 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 until disease progression
Slide 49: iPSC-derived CAR T Cells
iPSC-derived CAR T Cells - 49 -
Slide 50: 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 51: 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 - 51 -
Slide 52: 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 Max+IL2 MAD Max DL DL3+ DL3+ DL3+ MPAD or DL3+ DL3+ DL2 DL2 DL2 DL1 = 30
Slide 53: Collaborations
Collaborations - 53 -
Slide 54: 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 55: 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 56: Financials
Financials - 56 -
Slide 57: Financial Summary
Financial Summary As reported in Company’s Consolidated Financial Statements Three Months Ended March 31, 2021 Revenue $11.1M Operating Expense1 $57.3M Cash & Cash Equivalents $888M Employees 300+ Total Shares Outstanding2 107.9M 1 Includes $13m in stock-based compensation 2 Includes 14.0M shares of
Slide 58: Better Cells For Better Therapies
Better Cells For Better Therapies™ - 58 -
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