Kura Oncology: Leukemia Drug FDA Filing Supported by ASCO Data
Kura Oncology’s ziftomenib, a potential first-in-class treatment, is poised for FDA approval to treat acute myeloid leukemia (AML) patients with NPM1 mutations. Data from the American Society of Clinical Oncology (ASCO) meeting supports the filing, revealing statistically significant improvements in disease signs and symptoms. The Phase 2 trial showed a 23% remission rate, offering a promising outlook for patients, many of whom had faced multiple prior therapies. The FDA has granted priority review to ziftomenib with a target decision date of november 30.This oral small molecule targets menin, a critical protein for leukemia growth, potentially offering a safer profile than current options. News Directory 3 provides regular updates on the latest breakthroughs. Discover what’s next for this innovative therapy and its impact on the future of AML treatment.
Kura Oncology’s Ziftomenib Poised for Potential AML Treatment Approval
Updated June 03, 2025
kura Oncology, in partnership with Kyowa Kirin, is seeking FDA approval for ziftomenib, a potential first-in-class treatment for acute myeloid leukemia (AML) patients with NPM1 mutations. Clinical data presented at the American Society of Clinical Oncology (ASCO) annual meeting in Chicago supports the request, demonstrating statistically meaningful improvements in disease signs and symptoms.
Ziftomenib, an oral small molecule, targets menin, a protein crucial for cancer growth in leukemias. Kura Oncology, based in San Diego, reports that NPM1 mutations characterize approximately 30% of all AML cases, making this a significant target for the new therapy.
The Phase 2 trial included 92 adults with relapsed or refractory AML harboring NPM1 mutations. A significant portion of participants had received multiple prior therapies, including Venclexta. The study also included patients ineligible for stem cell transplants and other standard treatments.
The trial showed a 23% remission rate, with 13 patients achieving complete remission and eight achieving complete remission with partial hematological recovery. The median duration of response was 3.7 months. Median overall survival was 16.4 months for responders and 3.5 months for non-responders. Anemia, neutropenia, and QTc prolongation were among the treatment-related adverse events reported.
None of the investigators deemed any of these QTc prolongations to be significant and none of the patients ended therapy as of QTc prolongation effects.
According to Dr. Eunice Wang, an investigator in the ziftomenib trial, the three patients who experienced QTc prolongation were also taking other medications known to cause this cardiovascular effect, and two had electrolyte abnormalities that could have contributed.
Kyowa Kirin invested $330 million upfront in late 2024 to collaborate on ziftomenib. Kura Oncology will lead commercialization efforts in the U.S., while Kyowa Kirin holds commercialization rights for the rest of the world. kura is eligible for up to $1.2 billion in milestone payments. The FDA granted priority review to ziftomenib with a target decision date of Nov. 30.
Syndax Pharmaceuticals’ Revuforj, approved last November, was the first menin inhibitor to reach the market, targeting KMT2A mutations. A Phase 3 trial is underway to test Revuforj in AML driven by NPM1 mutations.
What’s next
Analysts suggest ziftomenib may offer a more favorable safety profile compared to existing treatments, possibly solidifying its position in the menin inhibitor landscape pending FDA approval.