Fate Therapeutics, Inc. · NASDAQ:FATE
Fate Therapeutics — Investor Presentation (May 2024)
42 slides from Fate Therapeutics's investor presentation, May 2024
Slide 1: Programmed Cellular Immunotherapies
Better Cells For Better Therapies® Programmed Cellular Immunotherapies Transforming the Treatment of Cancer and Autoimmune Diseases with Off-the-shelf, Multiplexed-engineered, iPSC-derived Cellular Immunotherapy May 2024 -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 safety and therapeutic potential of the Company’s product candidates, the advancement of and plans and tim
Slide 3: Fate Therapeutics
Fate Therapeutics Pioneering Off-the-Shelf iPSC-derived Cell Therapies Induced Pluripotent Highly differentiated approach to cell therapy with unmatched engineering capability, Stem Cell Platform manufacturing scale, and product quality and consistency Eliminate Conditioning Proprietary ADR technolo
Slide 4: Changing the Game of Cell Therapy
Changing the Game of Cell Therapy Mass Produced, Multiplexed-engineered Cell Products for Off-the-shelf Patient Treatment Induced Pluripotent Clonal Master iT Cells iNK Cells Stem Cells iPSC Bank Multiplex Gene Clonal Sorting iPSC Expansion Scalable or Engineering & Selection & Banking Manufacturing
Slide 5: Disruptive iPSC Product Platform
Disruptive iPSC Product Platform Creating Multiplexed-engineered Clonal Master iPSC Banks Single iPSC Clone Unprecedented Resolution in Clonal iPSC Characterization and Selection Round 1 of Engineering: Round 2 of Engineering: TRAC KO / Transgene A KI CD38 KO / Transgene B KI Multiplexed-engineered
Slide 6: First-in-class Product Pipeline
First-in-class Product Pipeline Multiplexed-engineered, iPSC-derived CAR NK Cell and CAR T-cell Product Candidates Program Indication CAR Target(s) Research Preclinical Phase 1 Partner CAR T-cell Product Candidates FT819 Systemic Lupus Erythematosus CD19 FT825 Solid Tumors HER2 Undisclosed Solid Tum
Slide 7: ADR
ADR -7-
Slide 8: Proprietary ADR Technology
Proprietary ADR Technology Synthetic ADR Receptor Designed to Eliminate Requirement for Cy / Flu Conditioning “The best defense is a good offense” ADR-armed CAR T cells specifically target activated host immune cells expressing 4-1BB, avoid host immune cell rejection, and retain potent and durable a
Slide 9: Synthetic ADR Receptor
Synthetic ADR Receptor Unique Attributes in Comparison to Other Immune Evasion Strategies Combination with Knockout of HLA-I & -II Knockout of HLA-I & -II Knockout of HLA-I & -II Fate’s Approach Strategy Intense Conditioning + HLA-E & -G + CD47 ADR Expression Chemotherapy CD58 Knockout Avoidance of
Slide 10: FT819 Program
FT819 Program Off-the-shelf, CD19-targeted CAR T-cell Product Candidate - 10 -
Slide 11: FT819 iPSC-derived, CD19-targeted CAR T-Cell Product Candidate
FT819 iPSC-derived, CD19-targeted CAR T-Cell Product Candidate Product Design Derived from Clonal Master Engineered iPSC Line Incorporating A Novel 1XX CAR Targeting CD19 and a TCR Knock-out 1XX CAR19: Novel chimeric antigen receptor consisting of CD28 costimulatory domain and modified CD3z signalin
Slide 12: Ex Vivo B-cell Depletion
Ex Vivo B-cell Depletion Preclinical Data in B Cell-mediated Autoimmune Disease FT819 demonstrates rapid, deep, and dose-dependent depletion of CD19+ B cells sourced from the peripheral blood of unmatched SLE donors in preclinical studies* * Rapid and deep depletion of CD19+ B cells was observed in
Slide 13: Pharmacokinetics
Pharmacokinetics Phase 1 Translational Data FT819 demonstrates dose-dependent CAR T-cell expansion and exposure* * FT819 PK (mean ± SEM) at 90 and 360 million cells in patients with r/r B-cell lymphoma (BCL) (n=11). In vivo CAR T-cell expansion and persistence were measured by a ddPCR assay accordin
Slide 14: B-cell Depletion in the Periphery
B-cell Depletion in the Periphery Phase 1 Translational Data FT819 demonstrates rapid, deep, and sustained CD19+ B cell depletion during 30-day treatment cycle* * Rapid and deep B cell depletion, with sustained suppression of B cells, in the peripheral blood during FT819 treatment cycle in patients
Slide 15: B-cell Depletion by FT819 Independent of Cy / Flu Conditioning
B-cell Depletion by FT819 Independent of Cy / Flu Conditioning Phase 1 Translational Data Cy / Flu conditioning did not result in B-cell depletion in the periphery Single dose of FT819 achieved rapid and deep depletion of supraphysiological B-cell burden* * Case study of two patients with r/r chroni
Slide 16: Reconstitution of Naïve B Cells after FT819 Treatment
Reconstitution of Naïve B Cells after FT819 Treatment Phase 1 Translational Data FT819 demonstrated depletion of low-frequency CD19+ plasmablast population Post-treatment reconstitution comprised of naïve B cells* * Case study of plasma cell depletion and B cell reconstitution in 3 patients with div
Slide 17: Tissue Homing, Infiltration & B Cell Clearance
Tissue Homing, Infiltration & B Cell Clearance Phase 1 Translational Data FT819 was detected in bone marrow at Day 9, with complete elimination of CD19+ cells in the bone marrow at the end of 30-day treatment cycle* * Case study of chronic lymphocytic leukemia patient treated with conditioning chemo
Slide 18: FT819 for B Cell-mediated Autoimmune Diseases
FT819 for B Cell-mediated Autoimmune Diseases Phase 1 Study for System Lupus Erythematosus: Design First SLE Patient Treated Pharmacodynamics Clinical activity at 3 months Single-dose • Auto-antibodies • SLEDAI • B-cell reconstitution • Discontinuation / need to re-initiate immunomodulating therapy
Slide 19: FT819 Ex Vivo Cytotoxicity Assay, First SLE Patient
FT819 for B Cell-mediated Autoimmune Diseases Ex Vivo Cytotoxicity Assay Using Pre-treatment Blood Sample from First SLE Patient Potent CD19+ B-cell depletion with FT819 using pre-treatment sample of first patient’s peripheral blood in ex vivo killing assay* * Using a pretreatment sample of the firs
Slide 20: FT522 Program
FT522 Program Off-the-shelf, CD19-targeted CAR NK Cell Product Candidate - 20 -
Slide 21: FT522 iPSC-derived, CD19-targeted CAR NK Cell Product Candidate
FT522 iPSC-derived, CD19-targeted CAR NK Cell Product Candidate Product Design hnCD16 CD20-targeted Multi-antigen targeting monoclonal antibody • CD19: CAR construct targeting CD19 ADR • CD20: proprietary hnCD16 receptor designed to augment CAR-CD19 antibody-dependent cellular cytotoxicity in combin
Slide 22: ADR-armed NK Cells Uniquely Proliferate and Persist
ADR-armed NK Cells Uniquely Proliferate and Persist Preclinical Data in Ex Vivo Allogeneic System Williams et al. 2022 ASH Annual Conference - 22 -
Slide 23: ADR-armed NK Cells Demonstrate Potent Anti-tumor Activity
ADR-armed NK Cells Demonstrate Potent Anti-tumor Activity Preclinical Data in Ex Vivo Alloreactive T-cell System In a tumor cell restimulation assay, FT522 maintains potent CAR activity alone and in the presence of allogeneic T cells ADR-null CAR19 iNK ± Allo T Cells ADR-armed CAR19 iNK ± Allo T Cel
Slide 24: FT522 In Vivo Trafficking and Tissue Residency
FT522 In Vivo Trafficking and Tissue Residency Preclinical Data Show Broad Distribution across Primary, Secondary and Tertiary Tissues Dose-dependent trafficking, infiltration, & residency in primary, secondary & tertiary tissues without cytokine support at human dose equivalency levels of 250 milli
Slide 25: FT522 Unique Functional Profile in Unmatched Donor SLE System
FT522 Unique Functional Profile in Unmatched Donor SLE System Preclinical Data Show B-cell Depletion, Alloreactive T Cell Elimination, and Functional Persistence In vitro activity in unmatched donor SLE PBMCs suggest unique functional profile in the presence of an unmatched host immune system* Witty
Slide 26: FT522 Phase 1 Study in Relapsed / Refractory B-cell Lymphoma
FT522 Phase 1 Study in Relapsed / Refractory B-cell Lymphoma Two-arm Study Designed to Assess Safety & Activity with and without Cy / Flu Conditioning Potential to demonstrate early clinical POC of FT522 without administration of conditioning chemotherapy to patients Conditioning Arm No Conditioning
Slide 27: FT522 Phase 1 Study: Enhanced Product PK
FT522 Phase 1 Study in Relapsed / Refractory B-cell Lymphoma Initial Clinical Data Indicate Enhanced Product PK in the Periphery Clinical data from first two patients in Conditioning Arm show improved PK and AUC with FT522 compared to prior-generation CAR NK cell program* * Phase 1 study in B-cell l
Slide 28: FT522 Phase 1 Study: B-cell Depletion in the Periphery
FT522 Phase 1 Study in Relapsed / Refractory B-cell Lymphoma Initial Clinical Data Show B-cell Depletion in the Periphery Clinical data from first two patients in Conditioning Arm show rapid, deep, and sustained CD19+ B cell depletion in the periphery* * Regimen A (FT522 combination with rituximab;
Slide 29: FT825 Program
FT825 Program Off-the-shelf, HER2-targeted CAR T-cell Product Candidate - 29 -
Slide 30: Off-the-shelf Cell-based Cancer Immunotherapies for Solid Tumors
Off-the-shelf Cell-based Cancer Immunotherapies for Solid Tumors Developing Multiplexed-engineered, iPSC-derived Synthetic Effector Cells for Solid Tumors • Next-generation cancer immunotherapies must address numerous challenges that limit the effectiveness of today’s agents in treating solid tumors
Slide 31: FT825 iPSC-derived, HER2-targeted CAR T-Cell Product Candidate
FT825 iPSC-derived, HER2-targeted CAR T-Cell Product Candidate Incorporates Seven Novel Synthetic Controls of Cell Function CAR-HER2: Novel 1XX CAR targeting HER2; controlled by hnCD16a IL7RF TRAC locus CXCR2 CAR-HER2 TCR KO: Complete loss of TCR surface expression to eliminate TGFβ Redirect potenti
Slide 32: FT825 Antigen Binder Preferentially Targets HER2 on Tumor Cells
FT825 iPSC-derived, HER2-targeted CAR T-Cell Product Candidate Novel Cancer-specific Antigen Binder Preferentially Targets HER2 on Tumor Cells Novel cancer-specific antigen binder preferentially targets HER2 on tumor cells with limited recognition of HER2 on healthy cells Hosking et al. 2023 SITC An
Slide 33: FT825 Binder Activity Across HER2 Expression Density
FT825 iPSC-derived, HER2-targeted CAR T-Cell Product Candidate Novel Cancer-specific Antigen Binder Exhibits Activity Across HER2 Expression Density Robust antigen-dependent targeting across HER2 expression levels, including HER2-low tumor cell lines FT825 Target cells Hosking et al. 2022 SITC Annua
Slide 34: ONO Pharmaceutical Collaboration
ONO Pharmaceutical Collaboration FT825 Plus Second Undisclosed Antigen Target for Solid Tumors Established September 2018 and expanded June 2022 Progress Innovation for Solid Tumors ✓ $12.5m Ono option exercise for • Multiplexed-engineered, CAR-targeted product candidates FT825 (2022) • Novel antige
Slide 35: T-cell Platform Innovation
T-cell Platform Innovation - 35 -
Slide 36: Developing a Portfolio of Engineered Features
Developing a Portfolio of Engineered Features Proprietary Functional Elements for Enhanced Cell Functionality & Synergizing with IO mAbs Direct Multi-Antigen Targeting CARs directed to CD19, BCMA, Combinations with mAbs / Engagers MICA/B, B7H3, others hnCD16 and synthetic CD3 receptors to uniquely I
Slide 37: Next-generation iPSC-derived CD8αβ CAR T cells
Next-generation iPSC-derived CD8αβ CAR T cells Creating Differentiated iPSC-derived CAR T Cells 1250 1250 1250 Tumor only Primary CAR T cell CAR iT cell Subcutaneous model 1000 1000 1000 Tumor Volume (mm3) Tumor Volume (mm3) Tumor Volume (mm3) of prostate cancer 750 750 750 500 500 500 250 250 250 6
Slide 38: Integration of ADR Technology Enables Resistance to Allogeneic Immune Cells
Next-generation iPSC-derived CD8αβ CAR T cells Integration of ADR Technology Enables Resistance to Allogeneic Immune Cells In vitro allo-resistance In vivo allo-resistance and function and function or + ADR- ADR+ Primed CAR-iT CAR-iT Allogeneic T cell CAR-iT cell count Allo T cell count Target cell
Slide 39: MICA/B-targeted CAR T Cells
MICA/B-targeted CAR T Cells α3 Domain Targeting Uniquely Eliminates Broad Array of Tumors in Preclinical Studies MICA and MICB are stress ligands expressed on many cancers CAR targeting the alpha 3 domain is uniquely resistant to shedding and interference by soluble MICA/B Cytotoxicity post MICA/B c
Slide 40: 2024 Milestones
2024 Milestones - 40 -
Slide 41: Upcoming 2024 Key Milestones
Upcoming 2024 Key Milestones • Present initial clinical data for FT819 in SLE Autoimmunity • Dose first SLE patient as add-on to commonly-used treatment regimen • Clear multi-indication IND for FT522 and initiate enrollment without conditioning chemotherapy • Present clinical POC data for FT522 with
Slide 42: Better Cells For Better Therapies
Better Cells For Better Therapies® - 42 -