Kite Pharma on Tuesday reported positive six-month results from a mid-stage study investigating its experimental CAR-T therapy axicabtagene ciloleucel in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma. Data showed that in 77 patients with diffuse large B-cell lymphoma, the complete response rate was 31 percent at six months, versus 33 percent at three months, as disclosed in September last year.

Results from the ZUMA-1 trial also showed that at six months, the objective response rate (ORR) was 36 percent, compared to 39 percent at three months. "These results...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," commented Jeff Wiezorek, senior vice president of clinical development.

In the study, 101 patients with multiple forms of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma and transformed follicular lymphoma, were assigned to treatment with a single infusion of axicabtagene ciloleucel, previously known as KTE-C19. The primary endpoint of the study was ORR.

Further results from the study illustrated that the ORR for the entire patient population at six months was 41 percent, including a complete response rate of 36 percent. Meanwhile, the six-month ORR in patients with primary mediastinal B-cell lymphoma or transformed follicular lymphoma was 54 percent, with 50 percent of patients exhibiting complete responses. Kite Pharma stated that the median overall survival (OS) of the entire study population has not been reached after a median follow-up of 8.7 months. The drugmaker explained that the OS in a similar patient population was 6.6 months.

Additionally, Kite Pharma said that no new patient deaths occurred in the study. The company had previously reported the deaths of three patients in the trial that were not attributable to disease progression, including two deaths due to haemophagocytic lymphohistiocytosis and cardiac arrest in the setting of cytokine release syndrome (CRS). Kite Pharma added that at six months, grade 3 or higher CRS was 13 percent, down from 18 percent at three months, while neurologic events decreased from 34 percent to 28 percent. The drugmaker said that full data from the primary analysis will be presented at the American Association for Cancer Research (AACR) annual meeting in April.

Kite Pharma noted that it plans to complete its rolling submission for a biologics licence application to the FDA by the end of the first quarter. The drugmaker also expressed plans to pursue a European submission later this year. Axicabtagene ciloleucel consists of a patient's T-cells engineered to express a chimaeric antigen receptor (CAR) to target the antigen CD19 and redirect the T-cells to kill cancer cells.

Commenting on the news, RBC Capital Markets analyst Michael Yee remarked the "data looks very good and above expectations, demonstrating a solid risk/benefit efficacy profile that should support rapid FDA approval, possibly by the end of this year." 

For additional analysis, see KOL Views: CAR-T cell therapies poised to make commercial debuts but one company is best positioned for initial success, according to leading medical oncologist.