Ziftomenib联合Venetoclax/Azacitidine治疗复发/难治性NPM1突变型急性髓系白血病显示出显著疗效——临床试验先锋

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Nearly two-thirds of patients with relapsed or refractory NPM1-mutated AML achieved clinically meaningful responses on a three-drug regimen — but the 39-percentage-point gap in composite complete remission rates between venetoclax-naïve (70% CRc) and venetoclax-experienced (24% CRc) patients at the 600 mg dose tells the more important story. Prior venetoclax exposure does not simply attenuate response; it nearly eliminates it, and that stratification will define where this combination lands in the treatment sequence. The KOMET-007 Phase 1a/b data published in Blood cover 64 response-evaluable R/R patients treated with ziftomenib plus venetoclax and azacitidine. In the venetoclax-naïve cohort at 600 mg, 87% ORR and 75% central MRD negativity among CRc responders are numbers that command attention in a setting where ven/aza alone notoriously underperforms. Median OS was not reached after 10.7 months of follow-up in that group. Response was also fast: median time to CRc was 3.9 weeks, a cadence that matters in a disease where patients deteriorate quickly. Safety held up under scrutiny — differentiation syndrome appeared in just 3% of the 67-patient safety population, a rate the investigators attribute to a protocol-specified staggered dosing schedule that initiates ven/aza before menin inhibition, and QTc prolongation resolved without dose modification. The venetoclax-experienced population is the harder problem. A 48% ORR sounds tolerable until the CRc rate of 24% and median OS of 7.4 months are placed beside the naïve-patient data. Half of CRc responders in that subgroup still achieved MRD negativity, which suggests the combination can generate deep responses even through prior exposure — just far less reliably. Whether the staggered dosing schedule or dose optimization could rescue more of that population is not answered here. What the data do establish is that ziftomenib’s monotherapy FDA approval as a salvage agent and this triplet’s emerging combination profile are solving overlapping but distinct problems. The result that will most directly determine ziftomenib’s commercial trajectory is enrollment completion and interim readout from the pivotal KOMET-017 Phase 3 trials in newly diagnosed NPM1-mutated and KMT2A-rearranged AML, where the venetoclax-naïve effect size seen in KOMET-007 will face a randomized test against standard of care. Source link: https: //www. globenewswire. com/news-release/2026/06/02/3305195/0/en/Kura-Oncology-and-Kyowa-Kirin-Announce-Publication-in-Blood-of-Ziftomenib-plus-Venetoclax-Azacitidine-Combination-in-Patients-with-R-R-NPM1-m-AML. html Obexelimab Reduces IgG4-RD Flare Risk 56% in Phase 3 INDIGO Trial Silence Therapeutics Nabs $10M From AstraZeneca Phase 1 Start Kymera Launches Parallel Phase 2b Trials for STAT6 Degrader KT-621 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. Jon NapitupuluFDA Clears IND for EOM613 Trial in Cancer Cachexia Patients Jon NapitupuluZenas BioPharma Submits BLA for Obexelimab in IgG4-Related Disease Jon NapitupuluSenti Bio Advances SENTI-202 to Pivotal Trial With Donor X Selection Strategy Jon NapitupuluLifestyle Program Improves Brain Health in Cognitive Decline