FDA Lifts Partial Clinical Hold on Trials of KO-539 in Acute Myeloid Leukemia

The U.S. Food and Drug Administration (FDA) has lifted its partial clinical hold on the phase Ib KOMET-001 study evaluating next-generation farnesyl transferase inhibitor KO-539 for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML). The trial was placed on hold after KO-539 manufacturer Kura Oncology reported a grade 5 serious adverse event (AE) resulting in a patient’s death, believed to be associated with differentiation syndrome.

This condition commonly occurs during treatment with differentiating agents used to treat AML. After reaching an agreement with the company on its mitigation strategy for differentiation syndrome, the FDA lifted the partial clinical hold. This decision allows Kura Oncology to continue enrolling patients in a phase Ib dose-expansion study. The company expects to enroll 12 patients with NPM1-mutant or KMT2A-rearranged relapsed or refractory AML in two dose cohorts to assess the drug’s safety, pharmacokinetics, and efficacy to determine the recommended phase II dose for KO-539.

Source: Kura Oncology news release, January 20, 2022.