Kite Pharma announces results of ZUMA-1 trial of axicabtagene ciloleucel (KTE-C19)for chemorefractory aggressive non-Hodgkin lymphoma (NHL).

Kite Pharma, Inc. presented results from the ZUMA-1 trial of axicabtagene ciloleucel (KTE-C19) in patients with chemorefractory aggressive non-Hodgkin lymphoma (NHL) in two oral presentations at the American Society of Hematology (ASH) 58th Annual Meeting in San Diego, California.

ZUMA-1 enrolled 111 patients with diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL), or transformed follicular lymphoma (TFL). Patients were required to have chemorefractory disease, defined as progressive or stable disease as best response to last line of therapy, or disease progression morre than 12 months after autologous stem cell transplant. Manufacturing was successful for 110 patients, and 101 patients were treated. The pre-specified interim analysis was triggered when 51 patients with DLBCL had a minimum of three months of follow-up. At the time of interim analysis, 11 patients with PMBCL/TFL had been followed for three months. An additional 31 patients with one month of follow-up were included in the late breaker presentation.

ZUMA-1 met the primary endpoint of objective response rate (ORR), p < 0.0001. Response rates by disease subtype are shown below. Responses were observed across key subgroups, including 75 percent CR in patients who relapsed in more than 12 months after autologous stem cell transplant and 47 percent CR in patients refractory to second line or later chemotherapy. At the month three assessment, 39 percent of patients were in CR.

Overall response for more than 3 months follow up: DLBCL (n=51) ORR 76%.CR 47%. TFL/PMBCL (n=11) ORR 91%. CR 73%. Total (n=62) ORR 79%. CR 52%.

In 93 patients with a minimum one month follow-up, the most common grade 3 or higher adverse events included neutropenia (63 percent), anemia (42 percent), leukopenia (40 percent), febrile neutropenia (29 percent), thrombocytopenia (26 percent), encephalopathy (19 percent), hypophosphatemia (17 percent), and decreased lymphocyte count (17 percent). Grade 3 or higher CRS and NE were observed in 13 percent and 29 percent of patients, respectively. Three patients died from treatment-emergent adverse events (hemophagocytic lymphohistiocytosis, cardiac arrest in the setting of CRS and pulmonary embolism). There were no cases of cerebral edema. The primary analysis of ZUMA-1 will include a minimum of 6 months of follow-up. Kite intends to seek regulatory approval of axicabtagene ciloleucel in refractory aggressive NHL and plans to complete its rolling submission of the Biologics License Application (BLA) in the first quarter of 2017.