SAN DIEGO and TOKYO, Dec. 8, 2025 — Kura Oncology and Kyowa Kirin reported new data showing a favorable safety profile and encouraging antileukemic activity for KOMZIFTI (ziftomenib) combined with venetoclax and azacitidine (ven/aza) in patients with acute myeloid leukemia (AML) harboring NPM1 mutations (NPM1-m) or KMT2A rearrangements (KMT2A-r). The results come from the ongoing KOMET-007 Phase 1a/1b trial (NCT05735184) and were presented orally at the 67th Annual Meeting of the American Society of Hematology.
In newly diagnosed, chemotherapy-ineligible NPM1-m AML, 40 patients were evaluated as of the Sept. 24, 2025 data cutoff; 37 were response-evaluable. Composite complete response (CRc) was 86% (32/37) and complete response (CR) was 73% (27/37). Of CRc responders, 68% (17/25) achieved molecular minimal residual disease (MRD) negativity by central next-generation sequencing. Median duration of CR and overall survival were not reached at a median follow-up of 26.1 weeks. Five patients proceeded to hematopoietic stem cell transplant (HSCT); three received ziftomenib maintenance. The regimen was generally well tolerated with a safety profile consistent with ven/aza alone; ziftomenib-related myelosuppression was low and one case each of grade 2 differentiation syndrome and grade 3 QTc prolongation were managed without treatment discontinuation.
Among 83 relapsed/refractory (R/R) patients with NPM1-m or KMT2A-r AML (80 response-evaluable) as of the data cutoff, overall response rate (ORR) across R/R NPM1-m patients was 65% (31/48) with a CRc rate of 48% and a median CRc duration of 39.9 weeks. In venetoclax-naïve R/R NPM1-m patients, ORR was 83% and CRc rate was 70%, compared with 48% and 28%, respectively, in venetoclax-exposed patients. Median overall survival in R/R NPM1-m patients was 54.9 weeks. Fourteen patients underwent HSCT; five received maintenance ziftomenib.
In R/R KMT2A-r AML, ORR was 41% (13/32) with a CRc rate of 28% and a median CRc duration of 12.4 weeks. Venetoclax-naïve KMT2A-r patients had an ORR of 70% and a CRc rate of 60%. Median overall survival in this cohort was 21.1 weeks. Two patients received HSCT and both proceeded to maintenance ziftomenib. The triplet was generally well tolerated in both R/R cohorts; rates of ziftomenib-related myelosuppression were low and neutrophil and platelet recovery were consistent with expectations for ven/aza. One grade 3 differentiation syndrome in an NPM1-m patient resolved with protocol-specified measures and treatment was resumed.
“The addition of ziftomenib to venetoclax and azacitidine has shown promising clinical activity, with 86% of newly diagnosed NPM1-mutated AML patients achieving composite complete remission and 68% attaining deep molecular MRD negativity, though median duration of response and overall survival remain immature,” said Gail J. Roboz, M.D., Weill Cornell Medicine. “In relapsed/refractory NPM1-m and KMT2A-r AML, overall response rates of 65% and 41% were observed, rising to 83% and 70% in venetoclax-naïve patients, underscoring ziftomenib’s potential benefit even in challenging settings. Importantly, inclusion of ziftomenib was generally well tolerated.”
Mollie Leoni, M.D., Chief Medical Officer at Kura Oncology, added that the data reinforce the potential for ziftomenib to become a foundational menin inhibitor in AML and support ongoing registration efforts, including site activation for the pivotal KOMET-017 trials.
KOMZIFTI (ziftomenib) is an FDA-approved, once-daily oral menin inhibitor for adult patients with relapsed or refractory AML with a susceptible NPM1 mutation and no satisfactory alternative treatment options. Ziftomenib is being developed across front-line and relapsed/refractory regimens in NPM1-m, KMT2A-r and FLT3-mutant AML subtypes, with company-sponsored registrational trials ongoing in both intensive chemotherapy-eligible and -ineligible front-line populations.
Slides from the ASH oral presentations will be available on Kura’s website under Posters and Presentations and in the ASH 2025 online program. Kura will host a virtual investor event on Dec. 8, 2025 at 12:30 p.m. ET / 9:30 a.m. PT; the live webcast and replay will be available on the company’s website.
Safety summary: KOMZIFTI carries a boxed warning for differentiation syndrome, which can be fatal and requires prompt interruption of therapy and corticosteroid treatment when suspected. KOMZIFTI can also cause QTc interval prolongation and embryo-fetal harm. The most reported adverse reactions include elevated liver enzymes, infections, electrolyte abnormalities, hemorrhage, gastrointestinal symptoms, fatigue, edema and differentiation syndrome. Dose interruptions, reductions and discontinuations occurred in the clinical program; serious adverse reactions were reported. Refer to the full U.S. prescribing information for complete safety details.
About Kura Oncology
Kura Oncology is a biopharmaceutical company developing precision medicines for cancer. Kura developed and is commercializing KOMZIFTI (ziftomenib) and is advancing programs in menin inhibition and farnesyl transferase inhibition. More information is available at https://kuraoncology.com.
About Kyowa Kirin
Kyowa Kirin is a global specialty pharmaceutical company focused on discovering and delivering novel medicines in areas including hematology, rare diseases and oncology. More information is available at www.kyowakirin.com.
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