Kite Pharma today reported positive 6-month data from its pivotal ZUMA-1 trial for its lead chimeric antigen receptor (CAR) T-cell candidate axicabtagene ciloleucel (formerly KTE-C19) in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL).

Axicabtagene ciloleucel met the trial’s primary endpoint with an 82% objective response rate (ORR), or rates of tumor response—complete response (CR) + partial response—recorded after a single infusion in 77 patients with diffuse large B-cell lymphoma (DLBCL) enrolled in Cohort 1. The ORR rose to 83% in 24 patients with primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) enrolled in Cohort 2.

For the combined 101-patient population, the ORR was 82%, Kite added, with a CR rate of 36% at 6 months. However, four of the 101 did not have a month-6 tumor assessment prior to the data cutoff and are therefore categorized as nonresponders. These patients could be counted as a month-6 CR in a follow-up analysis, which may increase the month-6 response and month-6 CR rate, Kite said.

Also at month 6, 41% of treated patients for the combined population achieved a response, including 36% that showed CR. Five of the 101 patients (5%) continue to experience highly significant and durable partial responses with what Kite said were “minimal” abnormalities in positron emission tomography (PET) scans.

Kite said no additional patients had died since the three that were reported in December at the American Society of Hematology Annual Meeting. All three deaths were unrelated to disease progression. One patient died of hemophagocytic lymphohistiocytosis and one cardiac arrest in the setting of cytokine release syndrome, both related to axicabtagene ciloleucel. A third death, due to a pulmonary embolism, was deemed unrelated to the treatment.

“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, M.D., Kite’s svp of clinical development, said in a statement.

Added Frederick L. Locke, M.D., ZUMA-1 co-lead investigator, and director of research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, FL: “The ZUMA-1 study results suggest that axicabtagene ciloleucel could become a new standard of care for patients with refractory aggressive lymphoma.”

Kite has begun a rolling Biologics License Application (BLA) seeking FDA approval for axicabtagene ciloleucel in aggressive NHL, to be based on combined data from all 101 patients in ZUMA-1. The company said it plans to complete that rolling submission by the end of the first quarter.

The success of that BLA will depend on 6-month data, Brad Loncar, CEO of Loncar Investments, told GEN in December: “It doesn’t really matter how you start a rolling application. What matters is how you finish it.”

Additionally, Kite said it plans to submit a marketing authorization application (MAA) with the European Medicines Agency (EMA) this year for axicabtagene ciloleucel for the treatment of relapsed or refractory DLBCL, PMBCL, and TFL.

Kite reported positive trial results on the same morning it released results for fourth-quarter and full-year 2016. The company finished the final 3 months of last year with a net loss of $84.9 million, and reported a net loss of $267.1 million for all of 2016, on revenues of $4.9 million for Q4 and $22.2 million for the full year.

For 2017, Kite issued guidance to investors projecting a GAAP net loss of between $450 million and $465 million, and full-year revenue of between $40 million and $50 million, which assumes no product revenue.








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