Investigational KTE-X19 To Be First Chimeric Antigen Receptor (CAR) T Cell Therapy for Mantle Cell Lymphoma in Europe if Approved

Kite, a Gilead Company, today announced that the company’s Marketing Authorization Application (MAA) for KTE-X19, an investigational chimeric antigen receptor (CAR) T cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL), has been fully validated and is now under evaluation by the European Medicines Agency (EMA).

The MAA is supported by data from the single arm, open-label, Phase 2 ZUMA-2 trial, which demonstrated an overall response rate of 93 percent, including 67 percent with complete response, as assessed by an Independent Radiologic Review Committee (IRRC) following a single infusion of KTE-X19 (median follow-up of 12.3 months). In the safety analysis, Grade 3 or higher cytokine release syndrome (CRS) and neurologic events were seen in 15 percent and 31 percent of patients, respectively. No Grade 5 CRS or neurologic events occurred. Detailed findings from this trial were recently presented during an oral session at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando.

“Relapse rates in mantle cell lymphoma remain overwhelmingly high and there is a significant need for new therapies that may improve patients’ prognosis,” said Ken Takeshita, MD, Kite’s Global Head of Clinical Development. “The EMA validation of our marketing application brings us closer to delivering on the promise of our industry-leading cell therapy development program, with the hope that we can bring KTE-X19 to appropriate patients in Europe as quickly as possible.”

Kite submitted a Biologics License Application (BLA) for KTE-X19 to the U.S. Food and Drug Administration (FDA) on December 11, 2019 for the treatment of adult patients with relapsed or refractory MCL. KTE-X19 has been granted Breakthrough Therapy Designation (BTD) by the FDA and Priority Medicines (PRIME) by the EMA.

KTE-X19 is investigational and not approved anywhere globally. Its efficacy and safety have not been established. More information about clinical trials with KTE-X19 is available at www.clinicaltrials.gov.

About MCL

MCL is a rare form of non-Hodgkin lymphoma (NHL) that arises from cells originating in the “mantle zone” of the lymph node and typically affects men over the age of 60.

About ZUMA-2

ZUMA-2 is a single-arm, multicenter, open-label Phase 2 study involving 74 enrolled/leukapheresed adult patients (≥18 years old) with MCL whose disease is refractory to or has relapsed following up to five prior lines of therapy, including anthracycline or bendamustine-containing chemotherapy, anti-CD20 monoclonal antibody therapy and the BTK inhibitors ibrutinib or acalabrutinib. The objectives of the study are to evaluate the efficacy (60 patients) and safety (68 patients) after a single infusion of KTE-X19 in this patient population. The primary endpoint for the study is objective response rate (ORR). ORR in this trial is defined as the combined rate of complete responses and partial responses as assessed by an IRRC.

Secondary endpoints include duration of response, progression-free survival, overall survival, incidence of adverse events, incidence of anti-CD19 CAR antibodies, levels of anti-CD19 CAR T cells in blood, levels of cytokines in serum, and changes over time in the EQ-5D scale score and visual analogue scale score. The study is ongoing.

About KTE-X19

KTE-X19 is an investigational, autologous, anti-CD19 CAR T cell therapy. KTE-X19 uses the XLP™ manufacturing process that includes T-cell selection and lymphocyte enrichment. Lymphocyte enrichment is a necessary step in certain B-cell malignancies in which circulating lymphoblasts are a common feature. KTE-X19 is currently in Phase 1/2 trials in acute lymphoblastic leukemia (ALL), mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).