Kite Pharma has reported positive topline data for its lead product candidate KTE-C19 in the Phase II portion of a Phase I/II trial in patients with non-Hodgkin's lymphoma (NHL).

KTE-C19 met the ZUMA-1 trial’s primary endpoint of objective response rate (ORR) to the treatment, with a combined ORR of 79%, including 52% complete remission, in two cohorts of patients with chemorefractory aggressive NHL—one with chemorefractory diffuse large B-cell lymphoma (DLBCL), the other with transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma (PMBCL).

In patients with DLBCL, the ORR was 76%, including 47% complete remission. The ORR jumped to 91%, including 73% complete remission, in the TFL/PMBCL cohort.

The results, from a preplanned interim analysis, make ZUMA-1 the first multicenter pivotal trial of a chimeric antigen receptor (CAR) T-cell therapy to report a positive outcome, Kite said yesterday. ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program®.

The interim analysis of ZUMA-1 evaluated the ORR in the first 51 patients in the DLBCL cohort with at least 3 months of follow-up, as well as 11 patients in the TFL/PMBCL cohort.

Prospects for that filing should be even brighter, one analyst said, because KTE-C19 has the FDA’s Breakthrough Therapy Designation for DLBCL, TFL, and PMBCL.

“Breakthrough status enables rapid and frequent communication with FDA, and that agency has access to 100% of KITE's clinical data, far beyond what is disclosed to the Street. Therefore, KITE should have significant flexibility to update results at more than one time point before [year-end] ‘16, which should aid in successful BLA filing,” Canaccord Genuity healthcare analyst John Newman, Ph.D., said in a statement.

Across all 62 patients assessed in the interim analysis, 2 patients died from KTE-C19–related adverse events—hemophagocytic lymphohistiocytosis and cardiac arrest in the setting of cytokine release syndrome (CRS)—Kite acknowledged.

The most common grade 3 or higher adverse events included neutropenia (66%), anemia (40%), febrile neutropenia (29%), thrombocytopenia (29%), and encephalopathy (26%). Grade 3 or higher CRS and neurological toxicity was observed in 18% and 34% of patients, respectively.

Kite said it will submit additional data from this interim analysis for presentation “at an upcoming scientific meeting.”

Phase II interim outcomes in ZUMA-1 are largely consistent with results from the Phase I portion of the study, according to Kite. The primary analysis of 101 patients with chemorefractory aggressive NHL (both the DLBCL and TFL/PMBCL cohorts) will include approximately 6 months of follow-up and is expected in the first quarter of 2017, the company added.

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