“Kura Oncology and Kyowa Kirin have recently delivered an impactful presentation at the 2025 ASCO Annual Meeting, showcasing positive pivotal data on ziftomenib monotherapy. This promising treatment has shown significant potential in mitigating relapsed or refractory NPM1-mutant acute myeloid leukemia (R/R NPM1-m AML), a severe hematological malignancy.
What makes these findings noteworthy is the observed CR/CRh rate of 23% in the pivotal Phase 2 cohort of R/R NPM1-m AML patients. This represents a substantial achievement in the field of oncology, demonstrating the potential of ziftomenib as a transformative therapeutic option. Moreover, the treatment has shown consistent efficacy across pre-specified subgroups, regardless of prior hematopoietic stem cell transplantation (HSCT), previous usage of venetoclax, or the presence of FLT3/IDH co-mutations. This broad effectiveness underscores the potential of ziftomenib to significantly improve patient outcomes in a variety of clinical contexts.
In addition to its efficacy, ziftomenib has also exhibited a favorable safety and tolerability profile, particularly in patients who have been heavily pre-treated. The limited myelosuppression observed in these patients is particularly encouraging, as it suggests the treatment’s potential to minimize the debilitating side effects often associated with aggressive leukemia therapies. Importantly, ziftomenib also demonstrated no clinically meaningful QTc prolongation, a key cardiovascular safety assessment in the drug development process. Only 3% of patients discontinued the treatment due to adverse events related to the therapy, further highlighting its tolerability.
Ziftomenib could potentially become the first approved once-daily, oral menin inhibitor for the treatment of adult patients with relapsed or refractory NPM1-mutated AML. The treatment has already received Priority Review status, with a PDUFA target action date set for November 30, 2025. This expedited timeline underscores the urgency and potential impact of this novel therapeutic approach.
Despite these promising results, there are inherent uncertainties associated with the drug development process. Regulatory interactions, FDA approval timing, and patient benefits must all be thoroughly evaluated. Risks include compound safety and efficacy, potential approval challenges, clinical trial uncertainties, and reliance on external financing. Notwithstanding these challenges, Kura Oncology remains committed to the continuous evaluation and updating of information on ziftomenib’s safety and effectiveness, emphasizing transparency and patient safety as their guiding principles.
Kura Oncology, in partnership with Kyowa Kirin, is hosting a virtual investor event today to discuss these findings and the future directions of ziftomenib. Notably, this partnership between the San Diego and Tokyo-based biopharmaceutical companies demonstrates the global interest and investment in the development of novel cancer therapeutics. The success of ziftomenib thus far is a testament to the importance of international collaborations in advancing the field of oncology and improving patient outcomes worldwide.”
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