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process
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Helen Kim
EVP, Business Development
TCR products
Proprietary manufacturing
processes
Multi-Target Partnership
Amgens validated oncology targets
Kites CAR design, IND enabling research &
eACTTM manufacturing
TUMOR TYPES
CD19
EGFRvIII
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
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
INDICATION
PRE-IND
PHASE 1
PHASE 2/3
B Cell Malignancies
NHL (DLBCL)
NHL (MCL)
KTE-C19 CAR
CLL
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
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Other than the KTE-C19 and Amgen related programs, the clinical trials are being conducted by the NCI pursuant to our CRADA.
Proprietary
Manufacturing
Process
Experienced
Team with
Proven
Record of
Success
US/EU
Presence
Solid IP
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Robust
Pipeline
(CAR & TCR)
Strategic
Partnership
Breakthrough
Efficacy in
KTE-C19
First Product
Launch in
2017
(anticipated)
Targets molecules
on the cell
surface
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Targets molecules
at or below the
cell surface
KITE PHARMA, INC.
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
NY-ESO-1
TCR (HLA-A2)
MAGE A3 TCR
(HLA-A1)
Targets
Viral
Antigens
(VAg)
MAGE A3/A6
TCR
(HLA-DP4)
HLA..
HPV-16 E6 TCR
(HLA-A2)
HLA
Neoantigens
(NAg)
Neo-antigens
DLBCL/PMBCL (n=17)
16 patients with ongoing response
12 patients with ongoing response over 1
year
3 patients were re-treated after
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Intention-to-Treat
Analysis
ALL (N=20)
Complete Response
14 (70%)
MRD negative
Complete Response
12 (60%)
Allogeneic Transplant
10 (50%)
0 (0%)
78.8%
51.6%
Median
follow
up = 10
mo
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
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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
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
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 .
80
% of CAR+ T cells
(Eff)
increasing differentiation
Streamlined process
amenable to cGMP
TCM population
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
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
% of CAR+ T cells
Gene Transduction
CD
CC 45R
R7 A+
+
T Cell Activation
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
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Manufacturing
Tumor
Microenvironment
Conditioning
Chemotherapy
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Combination therapy
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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
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
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