An experimental cancer therapy that re-activates the body’s immune system to fight cancer has shown remarkable success in a group of patients with a late-stage, aggressive blood cancer.

The drug was developed by US-based Kite Pharma which has seen its stock soar after encouraging results for its lead CAR-T drug, KTE-CI9.

The drug, now known as axicabtagene ciloceucil, met its primary endpoint in its ZUMA-1 trial, producing an objective response rate of 82% in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL).

The study includes 101 patients with non-Hodgkins lymphoma, including one group of patient with diffuse large B-cell lymphoma (DLBCL), and another group including patient with primary mediastinal B-cell lymphoma (PMBCL) or transformed follicular lymphoma (TFL).

Those patients in the second group responded better, with overall response rates reaching 71% compared to 49% in the first group.

The results build on the drug’s encouraging 6-month interim data from the same trial released last year, which showed 41% of patients responding to a single infusion of Kite’s therapy along with 36% of patients showing no signs of disease.

At the time, the data represented a breakthrough in aggressive lymphoma therapy which is now compounded by the release of the new data.

“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,” said Jeff Wiezorek, senior vice president of Clinical Development at Kite Pharma.

“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.”

One sticking point for the therapy continues to be the number of side effects experienced by patients, however, considering the lack of patient deaths in the study, the pharma – and any potential investors – will be happy with the drug’s progress.

In response to the new data, Kite Pharma’s stock soared, rising almost 25%. The company is now likely to seek approval from the FDA by the end of March this year and in Europe later this year.

The company is hoping it will be the first company ever to have a gene therapy approved for cancer treatment, although it will have to fight off competition from Novartis.

The Swiss pharma’s CTL019 is expected to be filed in the first half of this year for treatment of children with relapsed or refractory acute lymphoblastic leukaemia (ALL).

Regardless of which company gets its therapy to market first, they will have to resolve the big issue of gaining market access, considering the guide price for the therapy being an estimated $300,000 at least.

The company also needs to obtain longer term data to understand exactly how long responded patients remain in remission.

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