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

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.