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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