Kite's CAR-T gets a 'breakthrough' tag with pivotal data on the way

Kite Pharma ($KITE), developing souped-up T cells to fight cancer, won the FDA's coveted breakthrough therapy designation for its lead candidate as it outlines a path to regulatory submission next year.

Kite CEO Arie Belldegrun

Kite's top prospect, KTE-C19, is crafted by removing patients' T cells and genetically engineering them to home in on the protein CD19, expressed by a variety of blood cancers. The FDA conferred its breakthrough tag to KTE-C19's Phase II non-Hodgkin lymphoma program, promising the company access to key agency officials and a shot at early approval. That study, which began in May, is slated to report final data in 2016 and support an FDA application thereafter.

Meanwhile, Kite kicked off a fourth pivotal trial of KTE-C19 in children with advanced acute lymphoblastic leukemia, or ALL, looking to enroll 75 patients in Phase I/II trial that will first assess safety and then measure efficacy. Last week, Kite launched a Phase I/II study in adults with ALL, and, in November, the company started a Phase II KTE-C19 study in mantle cell lymphoma.

Kite's plan is to execute on each trial over the coming year, filing its cell therapy for lymphoma approval in 2016 and gradually broadening the treatment's indication as more data roll in. CEO Arie Belldegrun has been bullish about his company's future in the competitive CAR-T space, talking up plans to launch KTE-C19 in 2017.

Meanwhile, Novartis ($NVS) is pressing forward with a CD19-targeting CAR-T of its own, posting positive non-Hodgkin lymphoma data over the weekend at the American Society of Hematology meeting and promising to file its lead candidate by 2017. Juno Therapeutics ($JUNO) is in the midst of a Phase II trial with a similar cell therapy, studying how well its JCAR015 can produce remissions in ALL. Both Novartis and Juno won breakthrough designations last year for their ALL programs.

CAR-T immunotherapies are made through a process in which scientists extract T cells from a patient's blood and equip them with targeting mechanisms called chimeric antigen receptors, which seek out and bind to proteins expressed by cancer cells.

- read the breakthrough announcement
- here's the ALL release