Jefferies Global Healthcare Conference June 2, 2015

Transcription

1 Jefferies Global Healthcare Conference June 2, 2015

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. Forwardlooking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry s 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-Q for the quarter ended March 31, 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. 2 K I T E P H A R M A, I N C.

3 30 Years of Cancer Cellular Immunotherapy Before Treatment 6 Months After 1985 Before After 3 Treated with anti-cd19 CAR Treated with LAK cells + IL-2 3 K I T E P H A R M A, I N C.

4 Dramatic Response with Anti-CD19 CAR Before Treatment Post Treatment A patient with recurrent DLBCL post-sct treated with anti-cd19 CAR T cells Ongoing Complete Response 15+ months in a patient with chemo-refractory PMBCL 4 Scans from Dr. Rosenberg NCI 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 Ongoing Response 4+ years in a patient with Follicular Lymphoma Proprietary, low-cost and rapid manufacturing process 5 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 6 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 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 James Economou, MD, PhD Professor of Surgery, Microbiology, Immunology and Molecular Genetics and Molecular and Medical Pharmacology, UCLA Vice Chancellor of Research, UCLA 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 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 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 7 K I T E P H A R M A, I N C.

8 Building a Robust IP Estate with Scientific Leaders in Gene-Based Cellular Immunotherapy Inventors Clinical Pioneer 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 8 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 9 clinical programs Technology platform US and EU R&D capabilities Clinical and commercial manufacturing Strategic Collaboration Rights to Next Generation TCR Access to Manufacturing Early clinical evaluation 9 K I T E P H A R M A, I N C.

10 Engineered Autologous T Cell Therapy (eact ) Elegant Science, Streamlined Manufacturing 1. Cell Harvest 2. Patient Conditioning 3. Single CAR Cell Infusion Kochenderfer and Rosenberg, Nature Reviews Clinical Oncology, 2013 K I T E P H A R M A, I N C.

11 Multiple CAR and TCR programs in Clinical Testing PROGRAM INDICATION PRE-IND PHASE 1 PHASE 2/3 Anti-CD19 CAR B Cell Malignancies KTE-C19 CAR EGFRvIII CAR NY-ESO-1 TCR MAGE A3/A6 TCR MAGE A3 TCR HPV-16 E6 TCR HPV-16 E7 TCR SSX2 TCR Neo-Antigen TCRs Amgen Collaboration NHL (DLBCL) NHL (MCL) CLL ALL Glioblastoma Solid tumors Solid tumors Solid tumors Cervical and Head & Neck Cancer Solid tumors Solid Tumors 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. K I T E P H A R M A, I N C.

12 IP Portfolio of Greater than 100 Patent Assets Worldwide * Broad CAR IP Proprietary CARs Proprietary TCRs Proprietary scfvs Proprietary Manufacturing Techniques Proprietary T-Cell Selection Techniques *Includes in-house and in-licensed granted patents and pending patent applications K I T E P H A R M A, I N C.

13 Kite CAR & TCR Platforms Redirecting Immune Cells Against Cancer Chimeric Antigen Receptor (CAR) Products T Cell Receptor (TCR) Products Targets molecules on the cell surface Targets molecules at or below the cell surface 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) K I T E P H A R M A, I N C.

15 Franchise Approach to TCR Platform and Pipeline Cancer Testis Antigens Uniquely expressed only on cancer cells NY-ESO-1 MAGE A3 SSX2 MAGE A3/A6 Targets Viral Antigens Viral antigens are unique High-risk oncogenic viruses HPV-16 E6 HPV-16 E7 Neoantigens Specific to a patient s own tumor Personalized Proprietary Platform Allowing Rapid, High-throughput Identification of TCR-Based Product Candidates 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 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 K I T E P H A R M A, I N C.

17 Actively Investigating CARs and TCRs that Target Multiple Tumor Types TARGET TUMOR TYPES CD19 EGFRvIII NY-ESO-1 HPV-16 E6 HPV-16 E7 MAGEA3/A6 MAGE A3 SSX2 Neo-Antigens Amgen Collaboration Targets Non-Hodgkin Lymphoma and Leukemias Glioblastoma, Head and neck cancer, Melanoma Urothelial carcinoma, Sarcoma, Non-small cell lung 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 Solid Tumors Potentially all carcinomas Heme Malignancies and Solid Tumors (AML, renal cell carcinoma and MM) Heme Malignancy Solid Tumors 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 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 Intention-to-Treat Analysis ALL (N=20) 78.8% Complete Response 14 (70%) MRD negative Complete Response 12 (60%) Allogeneic Transplant 10 (50%) Relapse Post Allogeneic Transplant 0 (0%) 51.6% Median follow up = 10 mo 19 Lee et al Lancet K I T E P H A R M A, I N C.

20 KTE-C 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 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 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 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 POPULATION FIRST SUBJECT ENROLLED DEATHS/YEAR* DLBCL PMBCL 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 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 PROCESS CAPABILITIES Apheresis product T Cell Activation cgmp facility in Santa Monica, CA and in Amsterdam Commercial facility near LAX airport Gene Transduction Cell Expansion Cryopreservation Ready for bedside use K I T E P H A R M A, I N C.

23 Commercial Manufacturing Facility Kite Pharma K I T E P H A R M A, I N C.

24 Commercial Manufacturing Facility Kite Pharma K I T E P H A R M A, I N C.

25 Advancing Next Generation eact Technology Target Selection CAR/TCR Optimization 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 Manufacturing Limited ex vivo T cell expansion Selected expansion of T cell subtypes Tumor Microenvironment Conditioning Chemotherapy Combination therapy CRITICAL for T cell expansion 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 K I T E P H A R M A, I N C.

27 Expected Data Flow Program 1 Indication Data Availability 2 Anti-CD19 CAR B Cell Malignancies periodic update DLBCL P P KTE-C19 CAR MCL ALL CLL 1 Other than KTE-C19, clinical trials being conducted by the NCI pursuant to the CRADA 2 Anticipated dates for initial data availability are provided K I T E P H A R M A, I N C. TBD TBD 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

28 Kite Pharma s Competitive Advantages for Building the Future of Cancer Immunotherapy BEST TEAM ROBUST PIPELINE (CAR & TCR) BREAK- THROUGH EFFICACY SOLID IP THROUGH 2027 WELL FINANCED STRATEGIC PARTNERS (NCI/Amgen) FIRST NHL MULTICENTER TRIALS PROPRIETARY MANU- FACTURING PROCESS K I T E P H A R M A, I N C.

29 Join us on June 23 rd for Kite Investor Day K I T E P H A R M A, I N C.

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