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Kura Oncology presented early clinical trial results Saturday showing for the first time that its genetically targeted cancer drug can induce responses — including complete remission — in patients with advanced leukemia.

The data are preliminary, but suggest that the Kura drug, a once-daily pill called KO-539, could become an effective treatment for certain types of genetically defined acute myeloid leukemia (AML), a rapidly progressing bone marrow cancer that affects adults. The drug could also be used eventually to treat children with a different form of leukemia.


“What’s encouraging is that the drug clearly has some activity as a monotherapy. They saw complete remissions, so these data are something to build on,” said David Steensma, a leukemia expert who recently left Dana-Farber Cancer Institute to take a job with a pharmaceutical company. Steensma has no relationship with or financial ties to Kura. He reviewed the data at STAT’s request ahead of Kura’s presentation at the annual meeting of the American Society of Hematology.

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