On June 12, 2025 Kura Oncology, Inc. (Nasdaq: KURA, "Kura") and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported positive updated clinical data from KOMET-007, a Phase 1a/1b trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care in patients with newly diagnosed NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML) (Press release, Kura Oncology, JUN 12, 2025, View Source [SID1234653844]). The data for the combination with cytarabine/daunorubicin (7+3) were presented as an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (EHA2025) being held in Milan, Italy from June 12-15, 2025.
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"The findings presented at EHA (Free EHA Whitepaper)2025 underscore the potential of ziftomenib in combination with 7+3 as an early intervention in the frontline setting of AML and could offer a meaningful opportunity to improve patient outcomes," said Harry Erba, M.D., Ph.D., Director of the Leukemia Program at the Duke Cancer Institute. "The high rates of complete remission and MRD negativity across the 7+3 cohorts are particularly encouraging. The continued rapid enrollment in the Phase 1b portion of this study underscores the urgency and enthusiasm for further evaluating this combination approach."
"We remain very encouraged by the updated clinical activity, safety and tolerability data from the KOMET-007 study evaluating ziftomenib with 7+3 in newly diagnosed AML patients with NPM1 mutations or KMT2A rearrangements," said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. "These updated data reinforce the combination potential of ziftomenib in the frontline setting, strengthening our confidence in its ability to provide a valuable treatment option for a significant portion of the AML population. We and our partners at Kyowa Kirin are working in earnest to prepare for the KOMET-017-IC and NIC pivotal Phase 3 studies, which will enable us to test ziftomenib-based combinations and their potential, if approved, to transform care for AML patients worldwide."
In the ongoing study, ziftomenib dosed once daily at 600 mg in combination with 7+3 continued to demonstrate robust and evolving clinical activity in patients with newly diagnosed AML. Among 71 response-evaluable patients, 92% (65/71) achieved a composite complete remission (CRc) (93% for NPM1-m, 89% for KMT2A-r patients) and 80% (57/71) achieved a complete remission (CR) (84% for NPM1-m, 74% for KMT2A-r patients) at the time of data cutoff. A rate of CR minimal residual disease (CR-MRD) negativity of 71% for NPM1-m with a median time to MRD negativity of 4.7 weeks and a rate of CR-MRD negativity of 88% for KMT2A-r patients with a median time to MRD negativity of 4.4 weeks were observed. Ziftomenib did not delay time to neutrophil and platelet count recovery, which was comparable to intensive chemotherapy regimens.
Median follow-up times for the two populations were 24.9 weeks (range 4.3-47.1) in NPM1-m patients and 15.7 weeks (range 1.1-40.3) in KMT2A-r patients. Among response-evaluable NPM1-m patients, neither a median duration of CR nor a median overall survival (OS) had been reached. Among response-evaluable KMT2A-r patients, a median duration of CR was determined to be 25.6 weeks (95% CI, range 8.3-NE), and a median OS had not been reached. Notably, 96% (47/49) of NPM1-m patients and 88% (29/33) of KMT2A-r patients remained alive and on study.
The safety population included 82 newly diagnosed adult patients with NPM1-m or KMT2A-r AML from the pooled Phase 1a/1b portions of the trial at the 600 mg QD dose of ziftomenib. The safety profile observed with ziftomenib was consistent with previously reported data. Ziftomenib-related adverse events (TRAEs) of ≥ Grade 3 (Gr3), which occurred in more than 10% of patients were febrile neutropenia (15%), decreased platelet count (15%), anemia (11%) and decreased neutrophil count (11%). One case of differentiation syndrome (KMT2A-r, Gr3) was successfully managed by protocol-specified mitigation strategies. Two cases of investigator-assessed QTc prolongation (both KMT2A-r, Gr3) were reported; both patients were on other medications (posaconazole and/or piperacillin/tazobactam), which have been identified as potentially causing QT prolongation at the time of QT assessment. No dose-limiting toxicities, drug-drug interactions, clinically meaningful ziftomenib-associated QTc prolongation or additive myelosuppression were observed.
"Despite the availability of approved therapies for AML, up to 70% of patients who initially achieve a complete response relapse within three years – highlighting a substantial unmet need," said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. "The data presented at EHA (Free EHA Whitepaper)2025 suggest a favorable safety, tolerability, and efficacy profile for ziftomenib. We are encouraged by its potential as a future first-line treatment option and are committed to advancing the KOMET-017 Phase 3 trial, expected to begin later this year, to further evaluate its value in AML care."
The EHA (Free EHA Whitepaper)2025 oral presentation highlighting ziftomenib combined with 7+3 in newly diagnosed (1L) NPM1-m and KMT2A-r AML, and an encore presentation of results from the KOMET-001 registrational trial of ziftomenib in relapsed/refractory (R/R) NPM1-m AML (also presented during EHA (Free EHA Whitepaper)2025) are available in the Posters and Presentations section of the Kura website. The KOMET-017 protocol consists of 2 separate Phase 3 studies, which will investigate the benefits and risks of adding ziftomenib to standards of care treatments in patients newly diagnosed NPM1-m or KMT2A-r AML and which is registered at www.clinicaltrials.gov as NCT07007312.
Virtual Investor Event
Kura will host a virtual investor event featuring company management and investigators from the KOMET-007 trial of ziftomenib in combination with 7+3 in patients with NPM1-m and KMT2A-r AML at 4:30pm ET / 1:30pm PT on Wednesday, June 18, 2025. Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The event can also be accessed on the Investors section of Kura’s website at www.kuraoncology.com. An archived replay will be available shortly after the conclusion of the live event.