Kura Oncology and Kyowa Kirin announced positive Phase 2 trial results for ziftomenib, an oral menin inhibitor, in patients with relapsed/refractory (R/R) •NPM1•-mutated acute myeloid leukemia (AML). The trial achieved a 23% composite complete remission rate and demonstrated a favorable safety profile with limited myelosuppression and no clinically significant QTc prolongation. The companies have submitted a New Drug Application (NDA) to the FDA, with a target action date of November 30, 2025.
This news is potentially groundbreaking for AML patients with •NPM1• mutations, a common genetic abnormality for which there are currently no targeted therapies. The positive results offer a potential new treatment option for this patient population, particularly those with R/R disease who often have limited options and poor prognoses. The data suggest ziftomenib could significantly improve outcomes, offering hope for extended survival and improved quality of life.
In the pivotal Phase 2 cohort of the KOMET-001 trial, ziftomenib demonstrated a 23% CR/CRh rate in heavily pre-treated patients. Importantly, consistent efficacy was observed across various subgroups, irrespective of prior treatments like hematopoietic stem cell transplantation or venetoclax, or the presence of •FLT3/IDH• co-mutations. The safety profile was favorable, with only 3% of patients discontinuing treatment due to related adverse events. Differentiation syndrome, a known side effect of menin inhibitors, was observed but managed effectively with protocol-specified mitigation strategies.
The positive Phase 2 data and impending FDA decision represent a crucial step toward potential approval for ziftomenib. If approved, ziftomenib could become the first oral menin inhibitor specifically indicated for R/R •NPM1•-mutated AML, significantly altering the treatment landscape for this challenging disease. This approval could also pave the way for further investigation of ziftomenib in combination with other therapies and in earlier lines of treatment, potentially benefiting a broader range of AML patients.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
