Jefferies Immuno-Oncology Summit

April 15, 2015

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. Forwardlooking 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 by
such 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 and
development 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.

KITE PHARMA, INC.

Kite Pharma Overview

Corporate Update
CAR & TCR Pipeline

KTE-C19 in B Cell Malignancy

Manufacturing
Advancing eACT
3

KITE PHARMA, INC.

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
4

Ongoing Complete Response

15+ months in a patient with
chemo-refractory PMBCL

KITE PHARMA, INC.

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, Ph.D

EVP R&D, CMO

Helen Kim
EVP, Business Development

Margo R. Roberts, Ph.D

Chief Scientific Officer

Ton N. M. Schumacher, Ph.D

Chief Scientific Officer Kite Pharma EU

Salah D. Kivlighn, Ph.D

VP, Marketing

Marc Better, Ph.D

VP, Product Sciences

Jeffrey Wiezorek, MD, MS

VP, Clinical Development

Rizwana F. Sproule, Ph.D

VP, Regulatory Affairs
5

UCLA, Teva, Arno, Cougar, Agensys, NCI

NextWave, Telik, Connetics
Amgen, UCLA
NGM Biopharmaceuticals, Kosan, Onyx, Chiron
University of Virginia, Cell Genesys

Netherlands Cancer Institute (NKI)

MedImmune, NABI Biopharmaceuticals, Merck
Boehringer Ingelheim, Amgen, Abgenix, XOMA
Amgen, UCLA, California Institute of Technology
Amgen, SmithKline Beecham
KITE PHARMA, INC.

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

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

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
6

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

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

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
KITE PHARMA, INC.

Building a Robust IP Estate with Scientific Leaders

in 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

KITE PHARMA, INC.

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

KITE PHARMA, INC.

Actively Investigating CARs and TCRs that Target

Multiple Tumor Types
TARGET

TUMOR TYPES

CD19

Non-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 E6
HPV-16 E7

MAGE A3

Urothelial carcinoma, non-small cell lung cancer, breast carcinoma, ovarian carcinoma,
prostate carcinoma, hepatocellular carcinoma, gastric cancer, head and neck cancer,
pancreatic carcinoma and melanoma

SSX2

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

Tumor Specific
Neo-Antigens

Solid Tumors Potentially all carcinomas

Amgen
Collaboration
Targets

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

MAGE A3/A6

Head and neck cancer, cervical carcinoma, anal cancer, penile cancer, and various
aerodigestive tumors

Heme Malignancies

Solid Tumors

KITE PHARMA, INC.

Advancing eACT on Multiple Fronts

Seven CAR and TCR programs in clinical testing
PROGRAM
Anti-CD19 CAR

INDICATION

PRE-IND

PHASE 1

PHASE 2/3

B Cell Malignancies
NHL (DLBCL)
NHL (MCL)

KTE-C19 CAR
CLL

Pivotal studies across 4

indications in 2015

ALL
EGFRvIII CAR

Glioblastoma

NY-ESO-1 TCR

Solid tumors

HPV-16 E6 TCR

Cervical and
Head & Neck Cancer

HPV-16 E7 TCR
MAGE A3/A6
TCR

Solid tumors

MAGE A3 TCR

Solid tumors

SSX2 TCR

Solid tumors

Heme Malignancy

Heme Malignancies
and Solid Tumors

Solid Tumors

Amgen
Collaboration
10

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

KITE PHARMA, INC.

Laying the Foundation to Transform Cancer Therapy

Proprietary
Manufacturing
Process

Experienced
Team with
Proven
Record of
Success

US/EU
Presence

Solid IP

11

Robust
Pipeline
(CAR & TCR)
Strategic
Partnership
Breakthrough
Efficacy in
KTE-C19

First Product
Launch in
2017
(anticipated)

KITE PHARMA, INC.

Kite Pharma Overview

Corporate Update
CAR & TCR Pipeline

KTE-C19 in B Cell Malignancy

Manufacturing
Advancing eACT
12

KITE PHARMA, INC.

Kite CAR & TCR Platforms

Redirecting Immune Cells Against Cancer

Chimeric Antigen Receptor (CAR) Products

Targets molecules
on the cell
surface
13

T Cell Receptor (TCR) Products

Targets molecules
at or below the
cell surface
KITE PHARMA, INC.

Chimeric Antigen Receptors

Modular Approach to Harness T Lymphocytes

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

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

Co-Stimulatory Domain
CD28
Induces IL-2 production and proliferation
Protects T cells from activation induced cell
death and anergy
Promotes Tcm expansion and survival

1. Boesteanu and Katsikis, Seminars in immunology. Vol. 21, 2009

2. Volpin et al., doi: 10.3389/conf. fimmu. Vol. 999. 2013..
14

KITE PHARMA, INC.

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
15

KITE PHARMA, INC.

Building TCR Franchise

Addressing Both the Targets and HLA Haplotypes

Cancer Testis Aberrantly expressed & elicit
autologous T cell responses
Antigens
MAGE, NY-ESO-1, SSX
(CTAg)

NY-ESO-1
TCR (HLA-A2)

MAGE A3 TCR
(HLA-A1)

Targets

Viral
Antigens
(VAg)

Viral antigens are unique

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

MAGE A3/A6
TCR
(HLA-DP4)

HLA..

HPV-16 E6 TCR
(HLA-A2)

HLA
Neoantigens
(NAg)

From acquired mutations in

cancer
NOT present in normal cells

Neo-antigens

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
16

KITE PHARMA, INC.

Kite Pharma Overview

Corporate Update
CAR & TCR Pipeline

KTE-C19 in B Cell Malignancy

Manufacturing
Advancing eACT
17

KITE PHARMA, INC.

Compelling Preliminary Evidence of Broad AntiTumor 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)
18

Kochenderfer Blood 2012; Kochenderfer JCO 2014; Kochenderfer ASH 2014

A patient with recurrent DLBCL post-SCT

treated with CD-19 CAR T cells
Updated 12/2014

KITE PHARMA, INC.

Anti-CD19 CAR Treatment Achieves Complete

Responses in Heavily Pretreated Patients with ALL

19

Intention-to-Treat
Analysis

ALL (N=20)

Complete Response

14 (70%)

MRD negative
Complete Response

12 (60%)

Allogeneic Transplant

10 (50%)

Relapse Post Allogeneic

Transplant

0 (0%)

Lee et al Lancet 2014

78.8%

51.6%
Median
follow
up = 10
mo

KITE PHARMA, INC.

KTE-C19-101
Phase 1/2 Trial in Refractory Aggressive NHL
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
20

Pivotal Phase 2
Cohort 1: DLBCL
(n=72)
Cohort 2: PMBCL and TFL
(n=40)
DLBCL=Diffuse Large B-cell Lymphoma
PMBCL=Primary Mediastinal B-cell Lymphoma
TFL=Transformed Follicular Lymphoma

KITE PHARMA, INC.

Lead Product Candidate, KTE-C19, Could Address

Significant Unmet need in B Cell Malignancies

10,000
22,000

4,600
15,700

1,400
6,000

Indication

Population

Phase

First Subject
Enrolled

DLBCL
PMBCL
TFL

Refractory or
relapsed post
transplant

2
(n=112)

1H 2015

MCL

Relapsed/refractory

2015

ALL

Relapsed/refractory

2015

CLL

Relapsed/refractory

2015

*Deaths per Year (US) 2,3

New Cases per Year (US)1,2

If Phase 2 data are compelling, potential for Accelerated Approvals

and product launch in 2017 (DLBCL)
21

1) American Cancer Society, 2014 Facts and Figures; 2) The Leukemia and Lymphoma Society, Facts 2013 3) Adv Immunol. 2005; 87: 163208.K I T E P H A R M A , I N C .

except for naive versus eff

80
% of CAR+ T cells

Streamlined and Rapid eACT Manufacturing

Process for KTE-C19
60
40
20
0

Apheresis product shipped to CMO

CD45RA+ CD45RA- CD45RACD45RA+ in

Single T cell stimulation
of PBMC
CCR7+ CCR7+ CCR7- CCR7(Naive) media
(CM)
(EM)
human serum-free

(Eff)

increasing differentiation
Streamlined process
amenable to cGMP

TCM population

Pts 1-15, minus 8,9

60
40
20
0

Eff

CD
C C 45 R
R7 A CD
CC 45R
R 7 A+
-

CD
CC 45
R7 RA
+ -

CD
CC 45R
R7 A+
+

20

N/Tscm CM EM

Ready for bedside use

40

CD
C C 45 R
R7 A CD
CC 45R
R 7 A+
-

Cryopreservation

60

N=13

CD
CC 45
R7 RA
+ -

Cell Expansion

% of CAR+ T cells

80

Pts 8,9 only

% of CAR+ T cells

Gene Transduction

Short duration of cell expansion enriches

CD
CC 45R
R7 A+
+

T Cell Activation

Transportation logistics in place for multi-

center clinical trial

22

KITE PHARMA, INC.

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

23

KITE PHARMA, INC.

Kite Pharma Overview

Corporate Update
CAR & TCR Pipeline

KTE-C19 in B Cell Malignancy

Manufacturing
Advancing eACT
24

KITE PHARMA, INC.

Advancing eACT on All Fronts

Target Selection
CAR/TCR
Optimization

Manufacturing
Tumor
Microenvironment
Conditioning
Chemotherapy
25

Lineage-specific targets (Heme malignancies)

Cancer-specific (CTAg, VAg, NAg) vs. Cancerassociated antigens
Human scFv; linker-spacer; co-stimulatory domain
High affinity TCR
Additional stimulatory signals
Limited ex vivo T cell expansion
Selected expansion of T cell subtypes

Combination therapy

CRITICAL for T cell expansion

KITE PHARMA, INC.

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

26

KITE PHARMA, INC.

Multiple Clinical Trials Will Generate Meaningful

Data that Will Drive Kite Global Development
1

Program1

Indication

FSE2

Data Availability3

Anti-CD19 CAR

B Cell Malignancies

2009

periodic update

DLBCL

H1 2015

P1 2015
P2 2016

MCL

2015

TBD

ALL

2015

TBD

CLL

2015

TBD

KTE-C19 CAR

EGFRVIII CAR

GBM

2011

2016

NY-ESO-1 TCR

Solid Tumors

2013

2016

MAGE A3/A6 TCR

Solid Tumors

2014

2016

MAGE A3 TCR

Solid Tumors

2014

2016

HPV-16 E6 TCR

Cervical, H&N

2014

2016

HPV-16 E7 TCR

Cervical, H&N

2015

2016

1 Other than KTE-C19, clinical trials being conducted by the NCI pursuant to the CRADA
2 FSE: first subject enrolled; expected for KTE-C19 and HPV-16 E7
3 Anticipated dates for initial data availability are provided
27

KITE PHARMA, INC.