A November 2025 PDUFA date has been set for Kura Oncology’s oral acute myeloid leukaemia (AML) drug ziftomenib following the unveiling of positive Phase II data.
Results from the pivotal Phase I/II KOMET-001 trial (NCT04067336) were presented at the 2025 American Society of Clinical Oncology (ASCO) conference taking place over 30 May to 3 June in Chicago, Illinois. In a 2 June abstract, ziftomenib demonstrated positive disease response in relapsed or refractory AML patients with NPM1-mutation, regardless of prior venetoclax use or stem cell transplantation.
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Since the release of positive Phase II KOMET-001 results, the US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) from Kura and Kyowa Kirin, the company’s partner for non-US development and commercialisation of ziftomenib in relapsed or refractory AML. A PDUFA date is scheduled for 30 November 2025.
The NDA acceptance builds on a breakthrough designation the FDA awarded for ziftomenib, a menin inhibitor, in April 2024 for this patient population.
KOMET-001 enrolled 112 patients with NPM1-m relapsed or refractory AML, 92 of whom were assessed in its Phase II portion. Patients received 600mg of oral ziftomenib daily having been heavily pretreated previously; 60% had prior venetoclax exposure and 23% had undergone previous stem cell transplant.
Phase II KOMET-001 data show ziftomenib led to complete response (CR) or CR with partial haematological recovery (CRh) in 23% of participants with rates of 21% and 24% for venetoclax-naïve and exposed patients, respectively. Among 15 responsive patients, 67% demonstrated minimal residual disease (MRD) negativity at a median follow-up of 4.2 months.
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By GlobalDataAs per a 2 June release, Kura CEO Troy Wilson highlighted the drug’s potential to occupy a vacant niche in AML treatment. “NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this population,” he said.
Kura have also begun enrolling patients in its Phase I KOMET-015 trial of ziftomenib to treat advanced gastrointestinal stromal tumours (GIST) as of April 2025. Kyowa Kirin retain the option to collaborate with Kura for ziftomenib development in this and other solid tumour indications in return for payments to Kura of up to $228m. The companies’ current partnership grants Kura a potential $1.16bn from Kyowa Kirin for the rights to ziftomenib for AML outside of the US.
GlobalData predicts ziftomenib to generate total sales of $69m in 2026, though this hinges on a 2025 approval. These are expected to increase to $1.88bn annually by 2031.
GlobalData is the parent company of Pharmaceutical Technology.
