Kura Oncology says ‘positive’ results from KOMET-001 Phase 2 trial of ziftomenib

Kura Oncology (KURA) announced the presentation of positive pivotal results from the KOMET-001 Phase 2 registration-directed trial of ziftomenib, a once-daily, oral investigational menin inhibitor, in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia in an oral session at the 2025 American Society of Clinical Oncology Annual Meeting being held in Chicago, IL from May 30 – June 3, 2025. “We are delighted to announce positive pivotal data from the KOMET-001 trial in R/R NPM1-mutated AML patients treated with ziftomenib,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this patient population. With these encouraging results and a PDUFA target action date of November 30, 2025, we and our partners at Kyowa Kirin look forward to supporting FDA with its review of the ziftomenib New Drug Application and are well-positioned to meaningfully impact relapsed or refractory patients with NPM1 mutations.” The KOMET-001 Phase 2 population included 92 adult patients with R/R NPM1-m AML. The median age was 69. Patients were heavily pretreated, with 33% having received three or more prior lines of therapy (median prior lines: 2) and 59% having been previously treated with venetoclax. A complete remission plus CR with partial hematological recovery rate of 23% was observed among patients with R/R NPM1-m AML in the Phase 2 portion of the KOMET-001 trial. Among those 21 patients who achieved CR/CRh, 13 had a CR and 8 had a CRh. The median duration of CR/CRh responses was 3.7 months and the restricted mean duration of response was 4.3 months at the time of the data cutoff. MRD status was assessed in 19 of 21 patients who achieved CR/CRh, and 63% of these patients were MRD-negative. Comparable CR/CRh rates were observed across pre-specified subgroups, regardless of prior HSCT, prior venetoclax or FLT3/IDH co-mutations. Additional patient benefit beyond CR/CRh was observed with a rate of transfusion conversion of 21% and a rate of maintenance of transfusion independence of 20%. A median OS of 16.4 months was observed for responders and a median overall survival of 3.5 months was observed among non-responders. The safety population included 112 adult patients with R/R NPM1-m AML from the pooled Phase 1b and Phase 2 portions of the KOMET-001 trial. The safety profile observed with ziftomenib in this population was consistent with previously reported data. Treatment-related adverse events led to treatment discontinuations in 3% of patients. TRAEs of Grade greater than or equal to3 which occurred in more than 10% of patients were limited to differentiation syndrome, which was well managed by protocol-specified mitigation strategies and no Grade 4/5 treatment-related DS was observed. Although QTc prolongation was reported in three patients per investigator assessment, all three patients were on concomitant medications associated with QTc prolongation, two had electrolyte abnormalities and one had a prior diagnosis of atrial fibrillation.