Kite Pharma Reports Positive Data From ZUMA-1, Evaluating a CAR-T Treatment

Article

Median overall survival was not yet reached at a median follow-up of 8.7 months on the company’s ZUMA-1 trial.

Kite Pharma, a pioneer in developing the chimeric antigen receptor (CAR)-T cell treatment, has announced that median overall survival (OS) was not yet reached at a median follow-up of 8.7 months on the company’s ZUMA-1 trial.

The trial, being conducted in patients with chemotherapy-resistant aggressive B-cell non-Hodgkin lymphoma (NHL), is testing axicabtagene ciloleucel (previously referred to as KTE-C19). Interim analysis of the data showed that the trial met its primary endpoints of objective response rate (ORR), complete response (CR), and partial response (PR), following a single infusion of axicabtagene ciloleucel.

Of the 101 trial participants, 41% achieved ORR at 6 months (P <.0001), 36% had a CR, and 5% of patients had a durable PR. One of the partial responders became a complete responder at 9 months following infusion. More importantly, the trial was yet to reach a median OS at nearly 9 months of follow-up.

The following grade 3 or higher adverse events were reported from the trial:

  • Anemia (43%)
  • Neutropenia (39%)
  • Decreased neutrophil count (32%)
  • Febrile neutropenia (31%)
  • Decreased white blood cell count (29%)
  • Thrombocytopenia (24%)
  • Encephalopathy (21%)
  • Decreased lymphocyte count (20%)

No cases of cerebral edema were reported, unlike the JCAR015 trial. Grade 3 or higher cytokine release syndrome, very commonly observed with CAR-T treatment, decreased from 18% to 13%.

“Several patients we treated at Moffitt Cancer Center experienced a rapid and durable complete response with this first-of-its kind therapy,” Frederick L. Locke, MD, ZUMA-1 co-lead investigator, and director of research for the Immune Cell Therapy Program at Moffitt Cancer Center, said in a statement. "The ZUMA-1 study results suggest that axicabtagene ciloleucel could become a new standard of care for patients with refractory aggressive lymphoma."

The company is planning to submit a rolling Biologics License Application based on these results by the end of the first quarter of 2017.

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Recent Videos
Prerna Mewawalla, MD, medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network, as well as an associate professor at the Drexel University College of Medicine.
Surbhi Sidana, MD, an assistant professor of medicine, bone marrow transplantation, and cellular therapy at Stanford
Alexey Danilov, MD, PhD, the Marianne and Gerhard Pinkus Professor of Early Clinical Therapeutics, medical director of the Early Phase Therapeutics Program for the Systems Clinical Trials Office, codirector of the Toni Stephenson Lymphoma Center, and a professor in the Division of Lymphoma in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope in Duarte, California
© 2025 MJH Life Sciences

All rights reserved.