Multinational phase 2 trial of ziftomenib for relapsed or refractory NPM1-mutated AML

Ziftomenib is a selective oral menin inhibitor. The phase 2 part of the multinational KOMET-001 trial found that ziftomenib 600 mg once daily resulted in CR/CRh rate of 22% (95% CI 14-32) among patients with relapsed or refractory NPM1-mutated AML, meeting the primary endpoint. Overall response rate was 33% (95% CI 23-43), while median duration of response and median overall survival were 4.6 months (2.8-7.4) and 6.6 months (3.6-8.6), respectively. Three percent of patients discontinued treatment because of ziftomenib-related adverse events. The authors conclude that ziftomenib demonstrated significant clinical benefit and tolerability among patients with NPM1-mutated AML.