Kite Pharma's CAR-T Blood Cancer Candidate Succeeds in Major Study

Kite Pharma's CAR-T Blood Cancer Candidate Succeeds in Major Study February 28, 2017
By Alex Keown, Breaking News Staff

SANTA MONICA, Calif. – Shares of Kite Pharma are soaring more than 15 percent this morning after the company announced its lead CAR-T candidate axicabtagene ciloleucel (previously referred to as KTE-C19) met its primary endpoints in a critical study.

Trial results will pave the way for Kite to seek approval from the U.S. Food and Drug Administration ahead of other CAR-T rivals, particularly Swiss-based Novartis . Kite also plans to seek approval for its CAR-T treatment in Europe later this year.

Axicabtagene ciloleucel is being studied in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL). Kite said the ZUMA-1 study showed 82 percent of the 101 patients who received a single infusion of the drug saw the tumors shrink by half. The company said the results of the study show how effective the CAR-T therapy is for a patient population with multiple types of aggressive NHL, including diffuse large B-cell lymphoma (DLBCL), as well as primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL).

According to study data, six months following the one infusion, 41 percent of patients achieved a positive tumor response, while 36 percent were in full remission. Overall survival was estimated at 6.6 months, but Kite said so far more than half of the patients are still alive at nearly nine months following infusion.

“These results with axicabtagene ciloleucel are exceptional and suggest that more than a third of patients with refractory aggressive NHL could potentially be cured after a single infusion of axicabtagene ciloleucel,” Jeff Wiezorek, Kite’s senior vice president of clinical development, said in a statement. “The ZUMA-1 study was built on a foundation of support and commitment from Dr. Steven Rosenberg and the National Cancer Institute and our ZUMA-1 clinical trial investigators who believed in the potential for CAR-T therapy to change the paradigm of cancer treatment.”

Frederick Locke, the lead investigator of the study and director of research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida, said the results of Kite’s study “suggest that axicabtagene ciloleucel could become a new standard of care for patients with refractory aggressive lymphoma.”

While those responses are positive, there have been some problems with the treatment. There were three deaths associated with the study. Two deaths, one hemophagocytic lymphohistiocytosis and one cardiac arrest, were reported to be related to Kite’s axicabtagene ciloleucel. The third case, a pulmonary embolism, was deemed unrelated. Outside of the deaths, the most common grade 3 or higher adverse events included anemia (43 percent), neutropenia (39 percent), decreased neutrophil count (32 percent), febrile neutropenia (31 percent), decreased white blood cell count (29 percent), thrombocytopenia (24 percent), encephalopathy (21 percent) and decreased lymphocyte count (20 percent), Kite reported.

Chimeric Antigen Receptor T-Cell Therapies (CAR-T) are engineered in a laboratory to recognize a specific antigen in a cell and then administered into a cancer patient. The CAR-T cells should, if all goes as planned, multiply within the body and target the antigen and eliminate the threat. Axicabtagene ciloleucel is an investigational therapy in which a patient's T-cells are engineered to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T-cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for DLBCL, TFL, and PMBCL by the U.S. Food and Drug Administration and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

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