Kite Pharma Presents Updated Phase 1 Results from ZUMA-1 at AACR

Kite Pharma, Inc.

("Kite") today announced updated clinical results from the phase 1 portion of Kite's ZUMA-1 trial of its lead product candidate, KTE-C19, in patients with chemorefractory, aggressive non-Hodgkin lymphoma (NHL). KTE-C19 is an investigational therapy in which a patient's T cells are genetically modified to express a chimeric antigen receptor (CAR) that is designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias. David Chang, M.D., Ph.D., Kite's Executive Vice President, Research and Development, and Chief Medical Officer, commented, "Today's report affirms the early safety and efficacy profile of KTE-C19 in chemorefractory, aggressive NHL. We are encouraged by the ongoing complete remissions in patients with significant unmet need for new therapies. We remain on track to provide interim data from the pivotal phase 2 portion of the study later this year and to submit the KTE-C19 registration filing to the U.S. Food and Drug Administration (FDA) by the end of 2016." "The data reported today are important because refractory DLBCL is incurable. Median survival for these patients is short and there is no standard therapy," noted Ronald Levy, M.D., Robert K. Summy and Helen K. Summy Professor of Medicine and Director of the Lymphoma Program at Stanford University School of Medicine and Associate Director of Translational Science for the Stanford Cancer Institute. "A rate of complete response and durability of response in the ranges of those reported today would be of profound clinical importance if replicated in the phase 2 portion of the ZUMA-1 study. Adoptive transfer of engineered T cells has the potential to become standard of care for patients with refractory NHL in the near future." Updated Phase 1 Results from ZUMA-1: A Phase 1-2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-C19 (Anti-CD19 CAR T Cells) in Subjects with Refractory Aggressive Non-Hodgkin Lymphoma (NHL) Session: Early Clinical Trials Evaluating Cell-based, Checkpoint Inhibitors, and Novel Immunotherapeutics; Abstract Number: CT135; Presenter: Armin Ghobadi, M.D., Washington University, St. Louis, MO Phase 1 of ZUMA-1 treated a total of 7 patients with chemorefractory, diffuse large B cell lymphoma (DLBCL) KTE-C19 related adverse events consisted predominantly of cytokine release syndrome (CRS) and neurotoxicity which were generally reversible Grade 3 or higher CRS was observed in 14% and neurotoxicity in 57%; all were reversible except in one patient with dose-limiting toxicity KTE-C19 achieved rapid and durable responses in patients with chemorefractory disease (objective response rate 71%, complete response rate 57%) Ongoing complete response (CR) observed in 3 of 7 patients. One ongoing CR as of 9-month study follow-up and 2 ongoing CRs as of 6-month study follow-up. In addition, two posters on KTE-C19 engineered cell manufacturing and characteristics were presented at AACR on April 18, 2016. Manufacturing and Characterization of KTE-C19 in a Multicenter Trial of Patients with Refractory Aggressive Non-Hodgkin Lymphoma (NHL) (ZUMA-1) Session: Adoptive Cell Therapy; Abstract Number: 2308; Presenter: John Rossi, M.S., Kite Pharma The optimized GMP-manufacturing process generated anti-CD19 CAR T cells rapidly and without the need for pre-selection of a defined composition of T cells Biologically active anti-CD19 CAR T cells were manufactured for all patients enrolled in the multicenter phase 1 ZUMA-1 trial. Comparative Evaluation of Peripheral Blood T Cells and Resultant Engineered Anti-CD19 CAR T Cell Products from Relapsed/Refractory Non-Hodgkin's Lymphoma (NHL) Patients Session: Adoptive Cell Therapy; Abstract Number: 2305; Presenter: Timothy J. Langer, Kite Pharma CAR T cells were successfully manufactured for all patients enrolled in the study at the National Cancer Institute, Surgery Branch CAR T cell products were composed of both CD4+ and CD8+ T cells with a less differentiated phenotype than the starting leukapheresis products CAR T cells were polyfunctional and produced a wide range of immune homeostatic, modulating and effector cytokines/chemokines in response to antigen-positive target cells.