The first patients have been dosed with a novel menin inhibitor in a phase 2 trial assessing the therapy in patients with NPM1-mutant relapsed or refractory acute myeloid leukemia (AML).
The phase 2 KOMET-001 trial will assess the efficacy of ziftomenib for the treatment of patients with NPM1-mutant relapsed or refractory AML, according to a press release from Kura Oncology, the drug’s manufacturer.
“Building on the strength of our phase 1 data, we remain committed to our mission to realize the full potential of ziftomenib as an important treatment option to patients with acute leukemia,” said Troy Wilson, president and chief executive officer of Kura Oncology, in the release.
Findings from the phase 1 clinical trial demonstrated that a 600-milligram dose of ziftomenib contributed to a 30% complete response rate (the disappearance of all signs of cancer as a result from treatment) in 20 patients with NPM1-mutant AML, according to the release. Ziftomenib also had a favorable safety profile and was tolerable at the dose given in the phase 1 trial, which led to the dose’s use in the phase 2 trial.
The main goal of this phase 2 trial is to assess complete response with hematologic recovery in patients with NPM1-mutant relapsed or refractory AML. Secondary goals include clinical benefit of ziftomenib, in addition to its safety and tolerability, according to the release.
NPM1 mutations are the most common genetic alterations in patients with AML, representing approximately 30% of all patients with the disease, according to the release.
Although patients with NPM1-mutation AML have high response rates to first-line therapy (the first treatment given for a disease), they often have high relapse rates and poor survival outcomes. In the relapsed or refractory setting, overall survival (the percentage of patients who are alive for a certain period of time after diagnosis or treatment initiation) at 12 months is 30%.
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