kite corporate presentation 4-15-15 - jefferies immuno-oncology summit

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Jefferies Immuno - Oncology Summit April 15, 2015

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Kite Corporate Presentation 4-15-15 - Jefferies Immuno-Oncology Summit - FINAL

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  • Jefferies Immuno-Oncology SummitApril 15, 2015

  • 2 K I T E P H A R M A , I N C .

    Forward Looking Statements / Safe Harbor

    To the extent statements contained in this presentation are not descriptions of historical facts regarding Kite Pharma, Inc. (Kite, we, us, or our), they are forward-looking statements reflecting managements current beliefs and expectations. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industrys actual results, levels or activity, performance, or achievements to be materially different from those anticipated bysuch statements. You can identify forward-looking statements by words such as anticipate, believe, could, estimate, expect, intend, may, plan, potential, predict, project, should, will, would or the negative of those terms, and similar expressions that convey uncertainty of future events or outcomes. Forward-looking statements contained in this presentation include, but are not limited to, statements regarding: (i) the success, cost and timing of our product development activities and clinical trials; (ii) the ability and willingness of the National Cancer Institute (NCI) to continue research anddevelopment activities relating to our product candidates; (iii) our ability to obtain and maintain regulatory approval of KTE-C19 and any other product candidates; (iv) our ability to obtain funding for our operations and further development and commercialization of our product candidates; (v) our plans to research and discover additional product candidates, including through our acquired subsidiary in Amsterdam; (vi) our ability to develop, manufacture and commercialize our product candidates; (vii) the size and growth potential of the markets for our product candidates, and our ability to serve those markets; (viii) the rate and degree of market acceptance of our product candidates; (ix) our ability to attract and retain key scientific or management personnel; (x) the anticipated timing of clinical data availability; and (xi) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates. Various factors may cause differences between Kite's expectations and actual results as discussed in greater detail in Kite's filings with the Securities and Exchange Commission, including without limitation in its Form 10-K for the year ended December 31, 2014. Except as required by law, we undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy securities, nor shall there be any sale of securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

  • 3 K I T E P H A R M A , I N C .

    Kite Pharma Overview

    Corporate Update

    CAR & TCR Pipeline

    KTE-C19 in B Cell Malignancy

    Manufacturing

    Advancing eACT

  • 4 K I T E P H A R M A , I N C .

    Redefining Cancer Therapy with Gene-based Cellular Immunotherapy

    Headquartered in Santa Monica, CA with a European facility in Amsterdam, NL

    Fully integrated R&D and Manufacturing capabilities

    Strong IP for Chimeric Antigen Receptor (CAR) and T Cell Receptor (TCR) products

    Strategic partnerships

    Robust pipeline of CAR and TCR products with multiple products in clinical trials

    KTE-C19 entering pivotal trials in B-cell malignancies under Kite-sponsored IND

    Proprietary, low-cost and rapid manufacturing process

    Ongoing Complete Response 15+ months in a patient with

    chemo-refractory PMBCL

  • 5 K I T E P H A R M A , I N C .

    Experienced Senior Leadership with Proven Track Records for Success

    Arie Belldegrun, MD, FACSFounder, Chairman, President, CEO

    UCLA, Teva, Arno, Cougar, Agensys, NCI

    Cynthia M. Butitta, MBACOO and CFO

    NextWave, Telik, Connetics

    David D. Chang, MD, Ph.DEVP R&D, CMO

    Amgen, UCLA

    Helen KimEVP, Business Development

    NGM Biopharmaceuticals, Kosan, Onyx, Chiron

    Margo R. Roberts, Ph.DChief Scientific Officer

    University of Virginia, Cell Genesys

    Ton N. M. Schumacher, Ph.DChief Scientific Officer Kite Pharma EU

    Netherlands Cancer Institute (NKI)

    Salah D. Kivlighn, Ph.DVP, Marketing

    MedImmune, NABI Biopharmaceuticals, Merck

    Marc Better, Ph.DVP, Product Sciences

    Boehringer Ingelheim, Amgen, Abgenix, XOMA

    Jeffrey Wiezorek, MD, MSVP, Clinical Development

    Amgen, UCLA, California Institute of Technology

    Rizwana F. Sproule, Ph.DVP, Regulatory Affairs

    Amgen, SmithKline Beecham

  • 6 K I T E P H A R M A , I N C .

    Scientific Advisory Board: World Leaders in Cancer Immunotherapy

    Owen Witte, M.D. Chair Distinguished Professor of Microbiology,

    Immunology and Molecular Genetics, UCLA

    Howard Hughes Investigator

    Member, National Academy of Sciences

    Inder Verma, Ph.D. Irwin and Joan Jacobs Chair of Exemplary

    Science and American Cancer Society Professor of Molecular Biology, The Salk Institute

    Member, National Academy of Sciences

    James Economou, M.D., Ph.D. Professor of Surgery, Microbiology,

    Immunology and Molecular Genetics and Molecular and Medical Pharmacology, UCLA

    Vice Chancellor of Research, UCLA

    Antoni Ribas, M.D., Ph.D. Director, Tumor Immunology Program,

    Jonsson Comprehensive Cancer Center, UCLA

    Professor of Medicine, Professor of Surgery and Professor of Molecular and Medical Pharmacology, UCLA

    Ronald Levy, M.D. Robert K Summy and Helen K Summy

    Professor of Medicine, Stanford University

    Director, Lymphoma Program, Stanford University

    Member, National Academy of Sciences

    Donald Kohn, M.D. Professor of Microbiology, Immunology and

    Molecular Genetics & Pediatric Hematology/Oncology, UCLA

    Director, Human Gene and Cell Therapy Program, UCLA

    Member, Broad Stem Cell Research Center & Jonsson Comprehensive Cancer Center

  • 7 K I T E P H A R M A , I N C .

    Building a Robust IP Estate with Scientific Leadersin Gene-Based Cellular Immunotherapy

    Broadest claims for all scFv-based CAR constructs

    Expanding portfolio of TCR and CAR products

    Pioneering neo-antigen TCR products

    Proprietary manufacturing processes

  • 8 K I T E P H A R M A , I N C .

    Expanding Pipeline Through Strategic Collaborations and Internal R&D

    CRADA Pioneering research

    Product & process design

    Early clinical evaluation

    Internal R&D and Business Dev. R&D and Manufacturing in SM, CA

    TCR Discovery Research in Amsterdam

    Strong relationship with the Netherlands Cancer Institute provides access to investigators, clinical sites and manufacturing facilities in Europe.

    Multi-Target Partnership Amgens validated oncology targets

    Kites CAR design, IND enabling research & eACTTM manufacturing

  • 9 K I T E P H A R M A , I N C .

    Actively Investigating CARs and TCRs that Target Multiple Tumor Types

    TARGET TUMOR TYPES

    CD19Non-Hodkins Lymphoma, including DLBCL, PMBCL, TFL, and

    Leukemias, including MCL, CLL and ALL

    EGFRvIII Glioblastoma, head and neck cancer, melanoma

    NY-ESO-1

    Sarcoma, urothelial carcinoma, esophageal carcinoma, non-small cell lung cancer, breast

    carcinoma, ovarian carcinoma, prostate carcinoma, multiple myeloma, hepatocellular

    carcinoma, gastric cancer, head and neck cancer, pancreatic carcinoma, brain cancer,

    colorectal carcinoma and melanoma

    HPV-16 E6Head and neck cancer, cervical carcinoma, anal cancer, penile cancer, and various

    aerodigestive tumorsHPV-16 E7

    MAGE A3/A6 Urothelial carcinoma, non-small cell lung cancer, breast carcinoma, ovarian carcinoma,

    prostate carcinoma, hepatocellular carcinoma, gastric cancer, head and neck cancer,

    pancreatic carcinoma and melanomaMAGE A3

    SSX2 Head and neck cancer, hepatocellular carcinoma, melanoma, prostate cancer, and sarcoma

    Tumor Specific

    Neo-AntigensSolid Tumors Potentially all carcinomas

    Amgen

    Collaboration

    Targets

    Heme Malignancies and Solid Tumors, such as AML, clear cell renal cell carcinoma and

    multiple myeloma

    Heme Malignancies Solid Tumors

  • 10 K I T E P H A R M A , I N C .

    Advancing eACT on Multiple FrontsSeven CAR and TCR programs in clinical testing

    K I T E P H A R M A , I N C .

    PROGRAM INDICATION PRE-IND PHASE 1 PHASE 2/3

    Anti-CD19 CAR B Cell Malignancies

    KTE-C19 CAR

    NHL (DLBCL)

    NHL (MCL)

    CLL

    ALL

    EGFRvIII CAR Glioblastoma

    NY-ESO-1 TCR Solid tumors

    HPV-16 E6 TCR Cervical and

    Head & Neck CancerHPV-16 E7 TCR

    MAGE A3/A6

    TCRSolid tumors

    MAGE A3 TCR Solid tumors

    SSX2 TCR Solid tumors

    Amgen

    Collaboration

    Heme Malignancies

    and Solid Tumors

    Pivotal studies across 4 indications in 2015

    Heme Malignancy

    Solid Tumors

    Other than the KTE-C19 and Amgen related programs, the clinical trials are being conducted by the NCI pursuant to our CRADA.

  • 11 K I T E P H A R M A , I N C .

    Laying the Foundation to Transform Cancer Therapy

    Solid IP

    First Product Launch in

    2017 (anticipated)

    Strategic Partnership

    US/EU Presence

    Proprietary Manufacturing

    ProcessExperienced Team with

    Proven Record of Success

    RobustPipeline

    (CAR & TCR)

    Breakthrough Efficacy in KTE-C19

  • 12 K I T E P H A R M A , I N C .

    Kite Pharma Overview

    Corporate Update

    CAR & TCR Pipeline

    KTE-C19 in B Cell Malignancy

    Manufacturing

    Advancing eACT

  • 13 K I T E P H A R M A , I N C .

    Kite CAR & TCR PlatformsRedirecting Immune Cells Against Cancer

    Chimeric Antigen Receptor (CAR) Products T Cell Receptor (TCR) Products

    Targets moleculeson the cell

    surface

    Targets molecules at or below the

    cell surface

  • 14 K I T E P H A R M A , I N C .

    Chimeric Antigen ReceptorsModular Approach to Harness T Lymphocytes

    Single-Chain Variable Fragment Specific for cell surface protein Target selection is critical

    Co-Stimulatory DomainCD28 Induces IL-2 production and proliferation Protects T cells from activation induced cell

    death and anergy Promotes Tcm expansion and survival

    Transmembrane & Hinge Domains Impacts CAR expression Important for optimal CAR

    function at immunologic synapse

    Signaling Domain From TCR CD3z chain Provides essential activation

    signal via ITAMs

    1. Boesteanu and Katsikis, Seminars in immunology. Vol. 21, 20092. Volpin et al., doi: 10.3389/conf. fimmu. Vol. 999. 2013..

  • 15 K I T E P H A R M A , I N C .

    TCRs Aim at a Broad Spectrum of Therapeutic Tumor Targets

    Kite Pharma is uniquely positioned to address an unmet need and target a large hidden opportunity

  • 16 K I T E P H A R M A , I N C .

    Building TCR FranchiseAddressing Both the Targets and HLA Haplotypes

    NY-ESO-1 TCR (HLA-A2)

    HPV-16 E6 TCR(HLA-A2)

    Neo-antigens

    MAGE A3 TCR(HLA-A1)

    MAGE A3/A6 TCR

    (HLA-DP4)

    Targets

    HLA

    HLA..

    Aberrantly expressed & elicit autologous T cell responses

    MAGE, NY-ESO-1, SSX

    Cancer Testis Antigens

    (CTAg)

    Viral antigens are unique

    High-risk oncogenic viruses(e.g., HPV, EBV, HBV)

    Viral Antigens

    (VAg)

    From acquired mutations in cancer

    NOT present in normal cells

    Neo-antigens

    (NAg)

    Kite Pharma EUs TCR-GENErator is an industry-leading R&D engine for rapid, high-throughput identification of TCR-based product candidates,

    which could enter the clinic as early as 2017

  • 17 K I T E P H A R M A , I N C .

    Kite Pharma Overview

    Corporate Update

    CAR & TCR Pipeline

    KTE-C19 in B Cell Malignancy

    Manufacturing

    Advancing eACT

  • 18 K I T E P H A R M A , I N C .

    Compelling Preliminary Evidence of Broad Anti-Tumor Activity in B-cell Malignancies

    32 patients enrolled (29 evaluable), including largest dataset of anti-CD19 CAR in lymphoma

    Emerging AE profile includes: Transient cytokine release syndrome Reversible neurotoxicity B cell aplasia

    Response Rate 76% overall, 65% in DLBCL/PMBCL (n=17)

    16 patients with ongoing response

    12 patients with ongoing response over 1 year

    3 patients were re-treated after progression; all in ongoing response (17+ to 52+ months)

    Kochenderfer Blood 2012; Kochenderfer JCO 2014; Kochenderfer ASH 2014

    Updated 12/2014

    A patient with recurrent DLBCL post-SCT treated with CD-19 CAR T cells

  • 19 K I T E P H A R M A , I N C .

    Anti-CD19 CAR Treatment Achieves Complete Responses in Heavily Pretreated Patients with ALL

    Lee et al Lancet 2014

    Intention-to-TreatAnalysis

    ALL (N=20)

    Complete Response 14 (70%)

    MRD negative Complete Response

    12 (60%)

    Allogeneic Transplant 10 (50%)

    Relapse Post Allogeneic Transplant

    0 (0%)

    78.8%

    51.6%Median follow

    up = 10 mo

  • 20 K I T E P H A R M A , I N C .

    KTE-C19-101Phase 1/2 Trial in Refractory Aggressive NHL

    Cohort 2: PMBCL and TFL(n=40)

    Cohort 1: DLBCL(n=72)

    DLBCL=Diffuse Large B-cell LymphomaPMBCL=Primary Mediastinal B-cell LymphomaTFL=Transformed Follicular Lymphoma

    Pivotal Phase 2 Key Eligibility Criteria

    Refractory DLBCL, PMBCL, TFL Measurable Disease ECOG 0-1

    Primary Endpoint Objective Response Rate

    Operations First patient enrolled H1 2015 Multi-center study (~25 sites) Interim analysis (cohort 1) after 50

    patients Plan to file for accelerated

    approval if compelling data

  • 21 K I T E P H A R M A , I N C .

    Lead Product Candidate, KTE-C19, Could Address Significant Unmet need in B Cell Malignancies

    Indication Population PhaseFirst Subject

    Enrolled

    DLBCLPMBCL

    TFL

    Refractory or relapsed post

    transplant

    2(n=112)

    1H 2015

    MCL Relapsed/refractory 2 2015

    ALL Relapsed/refractory 2 2015

    CLL Relapsed/refractory 2 2015

    If Phase 2 data are compelling, potential for Accelerated Approvals and product launch in 2017 (DLBCL)

    New Cases per Year (US)1,2

    *Deaths per Year (US)2,3

    1) American Cancer Society, 2014 Facts and Figures; 2) The Leukemia and Lymphoma Society, Facts 2013 3) Adv Immunol. 2005; 87: 163208.

    10,00022,000

    4,600 15,700

    1,400 6,000

  • 22 K I T E P H A R M A , I N C .

    Streamlined and Rapid eACT Manufacturing Process for KTE-C19

    Apheresis product shipped to CMO

    Gene Transduction

    Cell Expansion

    Cryopreservation

    T Cell Activation

    Ready for bedside use

    Single T cell stimulation of PBMC in human serum-free media

    Streamlined process amenable to cGMP

    Short duration of cell expansion enriches TCM population

    Transportation logistics in place for multi-center clinical trial

    CD45RA+CCR7+(Naive)

    CD45RA-CCR7+(CM)

    CD45RA-CCR7-(EM)

    CD45RA+CCR7-(Eff)

    0

    20

    40

    60

    80

    All pts (1-15 inc)

    * Differences between subsets statistically signifcant except for naive versus eff

    % o

    f C

    AR

    + T

    ce

    lls

    increasing differentiation

    CD45

    RA+

    CCR7+

    CD45

    RA-

    CCR7+

    CD45

    RA-

    CCR7-

    CD45

    RA+

    CCR7-

    0

    20

    40

    60

    80

    Pts 1-15, minus 8,9

    % o

    f C

    AR

    + T

    ce

    lls

    CD45

    RA+

    CCR7+

    CD45

    RA-

    CCR7+

    CD45

    RA-

    CCR7-

    CD45

    RA+

    CCR7-

    0

    20

    40

    60

    Pts 8,9 only

    % o

    f C

    AR

    + T

    ce

    lls

    N/Tscm CM EM Eff

    N=13

  • 23 K I T E P H A R M A , I N C .

    Establishing Manufacturing Capabilities in Anticipation of KTE-C19 Launch in 2017

    Developed a proprietary, cost-efficient manufacturing process for KTE-C19

    Secured clinical supply with contract manufacturing

    Established a GMP facility in Santa Monica, CA for additional clinical supply

    Building out commercial facility in El Segundo, CA

    Staffing fully integrated Manufacturing Operations

  • 24 K I T E P H A R M A , I N C .

    Kite Pharma Overview

    Corporate Update

    CAR & TCR Pipeline

    KTE-C19 in B Cell Malignancy

    Manufacturing

    Advancing eACT

  • 25 K I T E P H A R M A , I N C .

    Advancing eACT on All Fronts

    Lineage-specific targets (Heme malignancies)

    Cancer-specific (CTAg, VAg, NAg) vs. Cancer-associated antigens

    Target Selection

    Human scFv; linker-spacer; co-stimulatory domain

    High affinity TCR

    Additional stimulatory signals

    CAR/TCR Optimization

    Limited ex vivo T cell expansion

    Selected expansion of T cell subtypesManufacturing

    Combination therapyTumor

    Microenvironment

    CRITICAL for T cell expansionConditioning

    Chemotherapy

  • 26 K I T E P H A R M A , I N C .

    Maintaining the Momentum: Projected Milestones for the Next 12 Months

    KTE-C19 IND accepted by FDA

    Secure in-house clinical manufacturing site

    Establish European presence

    Build out commercial manufacturing capacity

    Initiate KTE-C19 DLBCL pivotal study (1H) and additional studies

    Obtain Breakthrough Therapy Designation in DLBCL

    Present KTE-C19 data

    File an IND relating to a TCR product

  • 27 K I T E P H A R M A , I N C .

    Multiple Clinical Trials Will Generate Meaningful Data that Will Drive Kite Global Development

    Program1 Indication FSE2 Data Availability3

    1 Anti-CD19 CAR B Cell Malignancies 2009 periodic update

    2 KTE-C19 CAR

    DLBCL H1 2015P1 2015P2 2016

    MCL 2015 TBD

    ALL 2015 TBD

    CLL 2015 TBD

    3 EGFRVIII CAR GBM 2011 2016

    4 NY-ESO-1 TCR Solid Tumors 2013 2016

    5 MAGE A3/A6 TCR Solid Tumors 2014 2016

    6 MAGE A3 TCR Solid Tumors 2014 2016

    7 HPV-16 E6 TCR Cervical, H&N 2014 2016

    8 HPV-16 E7 TCR Cervical, H&N 2015 20161 Other than KTE-C19, clinical trials being conducted by the NCI pursuant to the CRADA2 FSE: first subject enrolled; expected for KTE-C19 and HPV-16 E73 Anticipated dates for initial data availability are provided