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Jefferies Global Healthcare Conference June 2, 2015

Jefferies Global Healthcare Conference · 2015. 6. 11. · KTE-C19 CAR NHL (DLBCL) NHL (MCL) CLL ALL EGFRvIII CAR Glioblastoma NY-ESO-1 TCR Solid tumors MAGE A3/A6 TCR Solid tumors

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  • Jefferies Global Healthcare ConferenceJune 2, 2015

  • 2 K I T E P H A R M A , I N C .2

    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 management’s current beliefs and expectations. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or ourindustry’s 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, andsimilar expressions that convey uncertainty of future events or outcomes. Forward-looking statements contained in thispresentation include, but are not limited to, statements regarding: (i) the success, cost and timing of our product developmentactivities 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-C19and any other product candidates; (iv) our ability to obtain funding for our operations and further development andcommercialization of our product candidates; (v) our plans to research and discover additional product candidates, includingthrough our acquired subsidiary in Amsterdam; (vi) our ability to develop, manufacture and commercialize our productcandidates; (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 ormanagement personnel; (x) the anticipated timing of clinical data availability; and (xi) our expectations regarding our ability toobtain and maintain intellectual property protection for our product candidates. Various factors may cause differences betweenKite'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-Q for the quarter ended March 31, 2015. Except as required by law, we undertake noobligation to publicly update any forward-looking statements, whether as a result of new information, future events orotherwise. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy securities, nor shall there beany sale of securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration orqualification under the securities laws of any such state or jurisdiction.

  • 3 K I T E P H A R M A , I N C .3

    Before Treatment 6 Months After

    Treated with anti-CD19 CAR

    1985

    1995

    2005

    2015

    Before After

    Treated with LAK cells + IL-2

    30 Years of Cancer Cellular Immunotherapy

  • 4 K I T E P H A R M A , I N C .4 Scans from Dr. Rosenberg NCI

    Before Treatment Post Treatment

    Ongoing Complete Response 15+ months in a patient with

    chemo-refractory PMBCL

    A patient with recurrent DLBCL post-SCT treated

    with anti-CD19 CAR T cells

    Dramatic Response with Anti-CD19 CAR

  • 5 K I T E P H A R M A , I N C .5

    Kite is Redefining Cancer Therapy with Gene-based Cellular Immunotherapy

    • Corporate Headquarters in Santa Monica, CA and EU R&D facility in Amsterdam, NL

    • First product, KTE-C19 projected to launch in 2017

    • Robust pipeline of Chimeric Antigen Receptor (CAR) and T Cell Receptor (TCR) products with multiple products in clinical trials

    • Strong IP Estate for CAR and TCR products

    • Strategic partnerships providing access to targets, technologies and faster time to human POC data

    • Proprietary, low-cost and rapid manufacturing process

    Ongoing Response 4+ years in a patient with Follicular Lymphoma

  • 6 K I T E P H A R M A , I N C .6

    Experienced Senior Leadership with Proven Track Records for Success

    Arie Belldegrun, MD, FACS Founder, Chairman, President, CEO

    Cynthia M. Butitta, MBA COO and CFO

    David D. Chang, MD, PhD CMO and EVP, R&D

    Helen S. Kim, MBA EVP, Business Development

    Margo R. Roberts, PhD Chief Scientific Officer

    Ton N. M. Schumacher, PhD Chief Scientific Officer, Kite EU

  • 7 K I T E P H A R M A , I N C .7

    Scientific Advisory Board: World Leaders in Cancer Immunotherapy

    Owen Witte, MD – Chair• Distinguished Professor of Microbiology,

    Immunology and Molecular Genetics, UCLA

    • Howard Hughes Investigator

    • Member, National Academy of Sciences

    James Economou, MD, PhD• Professor of Surgery, Microbiology,

    Immunology and Molecular Genetics and Molecular and Medical Pharmacology, UCLA

    • Vice Chancellor of Research, UCLA

    Donald Kohn, MD• 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

    Ronald Levy, MD• Robert K. Summy and Helen K. Summy

    Professor of Medicine, Stanford University

    • Director, Lymphoma Program, Stanford University

    • Member, National Academy of Sciences

    Antoni Ribas, MD, PhD• Director, Tumor Immunology Program,

    Jonsson Comprehensive Cancer Center, UCLA

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

    Inder Verma, PhD• Irwin and Joan Jacobs Chair of Exemplary

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

    • Member, National Academy of Sciences

  • 8 K I T E P H A R M A , I N C .8

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

    Inve

    nto

    rsC

    linic

    al P

    ion

    ee

    r Steven Rosenberg, MD, PhD

    • Chief of Surgery, NCI

    • Professor of Surgery, Uniformed Services University of Health Sciences and George Washington University School of Medicine and Health Sciences

    Margo R. Roberts, PhD

    • Chief Scientific Officer, Kite Pharma, Inc.

    • Inventor on 16 US patents and patent applications related to CAR T cell technology and tumor vaccine therapies

    Zelig Eshhar, PhD

    (Member, Kite Scientific Advisory Board)

    • Chairman of Immunology Research, Sourasky Medical Center, Tel Aviv

    • Professor Emeritus, Weizmann Institute of Science, Israel

  • 9 K I T E P H A R M A , I N C .9

    Expanding Pipeline Through Strategic Collaborations and Internal R&D

    CRADA•Pioneering research/pipeline

    •Product & process design

    •Early clinical evaluation

    Fully Integrated Company

    Strategic Collaboration•Rights to Next Generation TCR

    •Access to Manufacturing

    •Early clinical evaluation

    •9 clinical programs

    •Technology platform

    •US and EU R&D capabilities

    •Clinical and commercial manufacturing

  • 10 K I T E P H A R M A , I N C .10

    Engineered Autologous T Cell Therapy (eACT™)

    Elegant Science, Streamlined Manufacturing

    Kochenderfer and Rosenberg, Nature Reviews Clinical Oncology, 2013

    1. Cell Harvest

    2. Patient Conditioning

    3. Single CAR Cell Infusion

  • 11 K I T E P H A R M A , I N C .11

    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

    MAGE A3/A6 TCR Solid tumors

    MAGE A3 TCR Solid tumors

    HPV-16 E6 TCRCervical and Head

    & Neck CancerHPV-16 E7 TCR

    SSX2 TCR Solid tumors

    Neo-Antigen TCRs Solid Tumors

    Amgen

    Collaboration

    Heme Malignancies

    and Solid Tumors

    Multiple CAR and TCR programs in Clinical Testing

    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.

  • 12 K I T E P H A R M A , I N C .12

    IP Portfolio of Greater than 100 Patent Assets Worldwide*

    *Includes in-house and in-licensed granted patents and pending patent applications

    Broad CAR IPProprietary CARsProprietary TCRsProprietary scFvs

    Proprietary Manufacturing TechniquesProprietary T-Cell Selection Techniques

  • 13 K I T E P H A R M A , I N C .13

    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 .14

    TCR Platform Provides Access to a Broad Spectrum of Therapeutic Tumor Targets

    Potential CAR targets(~27% Human Proteome)

    Potential TCR targets(~73% Human Proteome)

    Sallman et al, BMC Biology (2009)

  • 15 K I T E P H A R M A , I N C .15

    Franchise Approach to TCR Platform and Pipeline

    NY-ESO-1

    HPV-16 E6

    Personalized

    MAGE A3 MAGE A3/A6

    •Uniquely expressed only on cancer cells

    Cancer Testis

    Antigens

    •Viral antigens are unique

    •High-risk oncogenic virusesViral

    Antigens

    • Specific to a patient’s own tumor

    Neo-antigens

    Proprietary Platform Allowing Rapid, High-throughput Identification of TCR-Based Product Candidates

    SSX2

    HPV-16 E7

    Targets

  • 16 K I T E P H A R M A , I N C .16

    TCF Acquisition Expands TCR Product Pipeline in Solid Tumors

    HIGHLIGHTS OF ACQUISITION

    • TCR-GENErator™: industry-leading R&D engine for rapid, high-throughput identification of TCR-based product candidates, which could enter the clinic as early as 2017.

    • NKI Relationship: provides important operational platform, access to investigators, clinical sites and manufacturing facilities in Europe.

    • Professor Dr. Schumacher: preeminent scientist in immunotherapy to lead Kite Pharma EU as CSO

    • European presence

  • 17 K I T E P H A R M A , I N C .17

    Actively Investigating CARs and TCRs that Target Multiple Tumor Types

    CD19

    EGFRvIII

    HPV-16 E6

    NY-ESO-1

    HPV-16 E7

    MAGE A3

    MAGEA3/A6

    SSX2

    Neo-Antigens

    Non-Hodgkin Lymphoma and Leukemias

    Glioblastoma, Head and neck cancer, Melanoma

    Head and neck cancer, Cervical carcinoma, Anal cancer

    Non-small cell lung cancer, Breast carcinoma, Ovarian carcinoma, Prostate carcinoma, Gastric cancer, Pancreatic carcinoma, Melanoma

    Hepatocellular carcinoma, Melanoma, Prostate cancer, Sarcoma

    Urothelial carcinoma, Sarcoma, Non-small cell lung cancer, Melanoma

    Solid Tumors – Potentially all carcinomas

    Amgen Collaboration Targets

    Heme Malignancies and Solid Tumors (AML, renal cell carcinoma and MM)

    TARGET TUMOR TYPES

    Heme Malignancy Solid Tumors

  • 18 K I T E P H A R M A , I N C .18

    Compelling Evidence of Broad Anti-Tumor Activity in B Cell Malignancies

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

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

    • 16 patients with ongoing response

    • 12 patients with ongoing response over 1 year

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

    • Ongoing clinical studies to optimize cell number and conditioning regimen

    Kochenderfer Blood 2012; Kochenderfer JCO 2015; Kochenderfer ASH 2014

  • 19 K I T E P H A R M A , I N C .19

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

    Lee et al Lancet 2015

    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 .20

    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 treated with KTE-C19

    1H 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 .21

    KTE-C19 Will Address Significant Unmet Needs in B Cell Malignancies

    INDICATION POPULATIONFIRST SUBJECT

    ENROLLEDDEATHS/YEAR*

    DLBCLPMBCL

    TFL

    Refractory or relapsed post transplant

    1H 2015 ~10,000

    MCL Relapsed/refractory 2015 ~1,200

    ALL Relapsed/refractory 2015 ~1,400

    CLL Relapsed/refractory 2015 ~4,600

    *US death per year

  • 22 K I T E P H A R M A , I N C .22

    Rapid and Efficient eACT ™ Manufacturing for KTE-C19 at Clinical and Commercial Scale

    Apheresis product

    Gene Transduction

    Cell Expansion

    Cryopreservation

    T Cell Activation

    Ready for bedside use

    • cGMP facility in Santa Monica, CA and in Amsterdam

    • Commercial facility near LAX airport

    PROCESS CAPABILITIES

  • 23 K I T E P H A R M A , I N C .23

    Commercial Manufacturing Facility – Kite Pharma

  • 24 K I T E P H A R M A , I N C .24

    Commercial Manufacturing Facility – Kite Pharma

  • 25 K I T E P H A R M A , I N C .25

    Advancing Next Generation eACT™ Technology

    • 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 .26

    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

    Treat first patient with KTE-C19 in a pivotal phase 1/2 clinical trial for refractory DLBCL

    • Initiate additional KTE-C19 studies in MCL, ALL, and CLL

    • 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 .27

    Expected Data Flow

    Program1 Indication Data Availability2

    Anti-CD19 CAR B Cell Malignancies periodic update

    KTE-C19 CAR

    DLBCLP1 2015P2 2016

    MCL TBD

    ALL TBD

    CLL TBD

    EGFRVIII CAR GBM 2016

    NY-ESO-1 TCR Solid Tumors 2016

    MAGE A3/A6 TCR Solid Tumors 2016

    MAGE A3 TCR Solid Tumors 2016

    HPV-16 E6 TCR Cervical, H&N 2016

    HPV-16 E7 TCR Cervical, H&N 2016

    1Other than KTE-C19, clinical trials being conducted by the NCI pursuant to the CRADA2Anticipated dates for initial data availability are provided

  • 28 K I T E P H A R M A , I N C .28

    Kite Pharma’s Competitive Advantages for Building the Future of Cancer Immunotherapy

    STRATEGIC PARTNERS

    (NCI/Amgen)

    FIRST NHL MULTICENTER

    TRIALS

    PROPRIETARY MANU-

    FACTURINGPROCESS

    SOLID IPTHROUGH 2027

    WELL FINANCED

    BEST TEAM

    BREAK-THROUGH EFFICACY

    ROBUSTPIPELINE

    (CAR & TCR)

  • 29 K I T E P H A R M A , I N C .29

    Join us on June 23rd for Kite Investor Day

  • 30 K I T E P H A R M A , I N C .30

    THANK YOU