Kura Oncology and Kyowa Kirin Announce New Data for KOMZIFTI

Kura Oncology and Kyowa Kirin announced new data demonstrating a favorable safety profile and encouraging antileukemic activity for KOMZIFTI in combination with venetoclax and azacitidine for the treatment of acute myeloid leukemia harboring NPM1 mutations or KMT2A rearrangements. The ongoing KOMET-007 Phase 1a/1b trial evaluated patients in cohorts with newly diagnosed chemotherapy-ineligible AML and relapsed/refractory AML. The new data are being reported in two oral presentations at the 67th Annual Meeting of the American Society of Hematology. KOMZIFTI, the first and only once-daily oral menin inhibitor for adult patients with R/R AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options, has been approved by the U.S. Food and Drug Administration and is commercially available in the United States. Ziftomenib + Venetoclax/Azacitidine in Newly Diagnosed NPM1-m AML: The ongoing KOMET 007 Phase 1a/b trial evaluated 40 patients with newly diagnosed NPM1-m AML as of the September 24, 2025 data cutoff date. Of these, 58% had an ECOG performance status of 2 and 37 were response evaluable. Robust activity was observed in newly diagnosed NPM1-m AML, including high rates of durable morphologic complete responses. 68% of CRc responders achieved molecular MRD negativity by central next-generation sequencing. Median duration of CR and OS were not reached at median follow-up of 26.1 weeks as of the data cutoff. 68% of patients remained alive and on treatment or in long-term follow-up as of the data cutoff. Five chemotherapy-ineligible patients received HSCT; three received ziftomenib maintenance therapy thereafter. The triplet combination was generally well tolerated in newly diagnosed NPM1-m AML, with a safety profile consistent with that reported for ven/aza alone. Rates of ziftomenib-related myelosuppression were low, and the median times to neutrophil and platelet recovery were also consistent with those expected for ven/aza alone. One case each of grade 2 differentiation syndrome and grade 3 investigator-assessed QTc prolongation were successfully managed without treatment discontinuation. Ziftomenib + Venetoclax/Azacitidine in R/R AML The ongoing KOMET 007 Phase 1a/b trial evaluated 83 patients with R/R NPM1-m or KMT2A-r AML as of the September 24, 2025 data cutoff date. Of these, 58% had received prior venetoclax and 80 were response evaluable. Robust activity was observed in patients with R/R NPM1-m AML, including among those previously treated with venetoclax. ORR was 65% and CRc rate was 48%, with CRc median duration of 39.9 weeks. In venetoclax-naive patients, ORR was 83% and CRc rate was 70%, compared with 48% and 28%, respectively, in venetoclax-exposed patients. Median OS was 54.9 weeks.14 patients received HSCT, five proceeded to ziftomenib maintenance therapy, and five were pending maintenance at time of data cutoff. In patients with R/R KMT2A-r AML, encouraging activity was also observed. ORR was 41% and CRc rate was 28%, with CRc median duration of 12.4 weeks. In venetoclax-naive patients, ORR was 70% and CRc rate was 60%. Median OS was 21.1 weeks. Two patients received HSCT and both proceeded to ziftomenib maintenance therapy. The combination was generally well tolerated in both R/R NPM1-m and R/R KMT2A-r AML. Rates of ziftomenib-related myelosuppression were low, with neutrophil and platelet recovery consistent with expectations for ven/aza alone. No ziftomenib-related QTc prolongation was reported. One grade 3 differentiation syndrome case (in an NPM1-m patient) was successfully resolved with protocol-specified measures, and the patient resumed treatment with ziftomenib.