Komzifti Gains FDA Approval, Giving Kura Oncology and Kyowa Kirin a Key Strategic Win in NPM1-Mutated AML

The FDA has granted full approval to Kura Oncology and Kyowa Kirin’s Komzifti (ziftomenib), marking the first FDA-approved once-daily, oral menin inhibitor for adults with relapsed or refractory (R/R) NPM1-mutated acute myeloid leukemia.

How Komzifti’s FDA Approval Advances Targeted Therapy in AML

The regulatory action holds particular significance for a patient population with poor outcomes and limited survival rates at relapse.¹

“Komzifti combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options. These features highlight Komzifti’s potential to serve as the menin inhibitor of choice in its approved indication,” Troy Wilson, PhD, JD, president and CEO of Kura Oncology, said in a press release. “Together with our partner, Kyowa Kirin, we remain committed to advancing development of Komzifti across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy. We are fully prepared to launch Komzifti today and deliver this new medicine to patients in need.”¹

What the Approval Means for Kura Oncology and Kyowa Kirin’s Commercial Strategy

• Kura Oncology and Kyowa Kirin reached an agreement in November 2024 to develop and commercialize Komzifti.
• As part of the agreement, Kyowa Kirin paid $330 million upfront, with Kura eligible to achieve up to $1.16 billion in milestones, with a payment now due from the approval of Komzifti.²
• The collaboration will involve Kura manufacturing the drug as well as leading the development, regulatory, and commercial strategy in the United States, while Kyowa Kirin is leading the development, regulatory, and commercialization strategy outside of the United States.

The approval of Komzifti gives both companies a pivotal foothold in a defined segment of AML with high relapse rates and limited treatment options.

“Komzifti addresses a critical need for adult patients with R/R NPM1-m AML, many of whom are older and unable to tolerate intensive chemotherapy or transplant,” Eunice Wang, MD, chief of the Leukemia Service and professor of Oncology at Roswell Park Comprehensive Cancer Center, said in the press release. “The clinical data demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions and no Boxed Warnings for QTc prolongation or Torsades de Pointes—key advantages for patients on multiple concurrent medications. This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population.”¹

The Role of Menin Inhibition and NPM1 Mutations in AML Treatment Advances

• AML is the most commonly diagnosed form of acute leukemia and has a patient population with significant unmet needs and a poor prognosis.
• The menin pathway is a key driver of multiple genetic AML alterations, of which NPM1 is the most common, comprising approximately 30% of total AML cases.
• Despite the high response rates observed with frontline therapy, relapse rates and survival outcomes have remained low.
• Further, co-occurring mutations have been found to worsen prognosis and R/R patients with NPM1-mutant AML and additional mutations have low survival rates.³
• Komzifti targets the menin-KMT2A/MLL protein-protein interaction in genetically defined patients with AML.

In April 2024, the drug was granted Breakthrough Therapy Designation by the FDA for heavily pretreated patients with R/R NPM1-mutant AML based on data from the Phase I/II KOMET-001 trial (NCT04067336).³ Additionally, Komzifti was granted Orphan Drug Designation in 2019.⁴

“The approval of Komzifti underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology,” Takeyoshi Yamashita, PhD, executive vice president and chief medical officer of Kyowa Kirin, said in the release. “In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as Komzifti represents a major step forward and offers potential to transform existing standards of care. We are proud to partner with Kura Oncology in bringing this important therapy to patients and their families.”¹

References

1. Kura Oncology and Kyowa Kirin Announce FDA Approval of KOMZIFTI™ (ziftomenib), the First and Only Once-Daily Targeted Therapy for Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia. News release. Kura Oncology. November 13, 2025. Accessed November 14, 2025. https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-fda-approval-komziftitm

2. Kura Oncology and Kyowa Kirin Announce Global Strategic Collaboration to Develop and Commercialize Ziftomenib in Acute Leukemias. News release. Kura Oncology. November 20, 2024. Accessed November 14, 2025.

3. Kura Oncology receives breakthrough therapy designation for ziftomenib in NPM1-mutant AML. News release. Kura Oncology. April 22, 2024. Accessed November 14, 2025. https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-receives-breakthrough-therapy-designation

4. Kura Oncology’s menin-MLL inhibitor KO-539 receives orphan drug designation from FDA for treatment of acute myeloid leukemia. News release. Kura Oncology. July 24, 2019. Accessed November 14, 2025. https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-doses-first-patient-phase-1-clinical-trial-menin