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Kura Oncology Begins Phase II Ziftomenib Trial in NPM1-Mutant Acute Myeloid Leukemia

NEW YORK – Kura Oncology said on Thursday that it has treated the first acute myeloid leukemia patient in a Phase II trial of the investigational menin inhibitor ziftomenib.

In the registration-directed study, Kura is evaluating ziftomenib in relapsed or refractory AML patients whose cancers harbor NPM1 mutations. The firm plans to enroll 85 patients overall in the US and Europe and track patients' complete responses, or complete response with hematologic recovery, on ziftomenib. The trial's secondary endpoints include ziftomenib's clinical benefit, safety, and tolerability.

The Phase II trial follows encouraging results from Kura's Phase I ziftomenib trial, KOMET-001, in which 30 percent of 20 patients with NPM1-mutant AML experienced a complete response on the drug's recommended Phase II dose.

The San Diego-based firm said ziftomenib's safety profile was also encouraging, though in 2021, the US Food and Drug Administration did put a partial hold on the Phase I trial after a patient died. The agency lifted the hold once Kura incorporated a system to mitigate certain life-threatening adverse events.

Later this year, Kura plans to begin evaluating ziftomenib with standard-of-care therapies in earlier treatment settings and in patients with NPM1-mutant and KMT2A-rearranged AML.

"The speed with which we have begun enrolling in this registration-enabling Phase II study speaks to the significant interest in ziftomenib among investigators," Kura President and CEO Troy Wilson said in a statement.