Jefferies Immuno - Oncology Summit April 15, 2015
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