Kura Oncology, Inc., a clinical-stage biopharmaceutical innovator, and Japan-based pharmaceutical leader Kyowa Kirin Co., Ltd., have entered into a strategic partnership that could redefine treatment for acute myeloid leukemia (AML). The collaboration focuses on ziftomenib, an investigational oral menin inhibitor that has garnered attention as a potential breakthrough therapy for genetically defined AML patients.

This global agreement will see Kura Oncology receive $330 million upfront, supplemented by up to $420 million in near-term milestone payments. Totaling over $1.2 billion in potential payments, this partnership underscores the promise of ziftomenib as a transformative solution in oncology. Notably, the U.S. Food and Drug Administration (FDA) granted ziftomenib a coveted Breakthrough Therapy Designation, marking it as the first therapy targeting relapsed/refractory (R/R) NPM1-mutant AML patients.

Ziftomenib poised to redefine AML treatment

Ziftomenib stands out as a selective, oral inhibitor of menin, a protein linked to poor prognoses in NPM1-mutant AML cases. With its FDA designation, the drug is positioned as a critical solution for patients unresponsive to standard therapies. Kura Oncology has already completed enrollment in a pivotal Phase 2 trial, with plans for a New Drug Application (NDA) submission in 2025.

Clinical studies aim to expand ziftomenib’s application to frontline settings and combination therapies, targeting both fit and unfit AML patients. If successful, these efforts could unlock a U.S. market potential exceeding $3 billion annually, according to Kura’s leadership.

Strategic responsibilities

Under the agreement, Kura Oncology will spearhead U.S. development, regulatory approvals, and commercialization efforts, while sharing profits and losses equally with Kyowa Kirin. Outside the United States, Kyowa Kirin will manage regulatory and commercial strategies and retain sales rights, offering Kura tiered double-digit royalties on net sales.

The partnership will also share clinical trial responsibilities. Kura will fund research costs until 2028, with a 50:50 cost-sharing arrangement thereafter. Both companies plan to launch multiple Phase 2 and Phase 3 studies over the coming years, aiming to expand ziftomenib’s reach into other hematologic malignancies.

Expert perspectives on the collaboration

Kyowa Kirin’s Chief Strategy Officer, Yasuo Fujii, stated that ziftomenib aligns seamlessly with the company’s commitment to innovative cancer treatments. Fujii emphasized that the therapy complements their existing hemato-oncology portfolio and could pave the way for more effective combination regimens, enhancing outcomes for cancer patients.

Kura Oncology’s CEO, Troy Wilson, underscored the significance of this collaboration in fulfilling the company’s vision of delivering precision cancer therapies. Wilson expressed confidence in Kyowa Kirin’s global expertise, noting that the partnership provides the financial and strategic foundation needed to bring ziftomenib to patients worldwide.

Market impact and investor reactions

Despite the promising collaboration, Kura Oncology’s stock dropped 14% in after-hours trading following the announcement. Analysts attribute the decline to concerns over potential financial risks or uncertainties regarding the drug’s commercial success. This reflects broader market volatility, with Kura’s shares already down 20% over the past quarter.

The road ahead

Ziftomenib’s breakthrough designation and ambitious clinical pipeline place it at the forefront of AML innovation. By leveraging Kyowa Kirin’s global reach and Kura Oncology’s precision medicine expertise, the collaboration aims to meet urgent medical needs in hematologic oncology.

Looking forward, the companies plan to evaluate ziftomenib’s potential in solid tumors, such as gastrointestinal stromal tumors (GIST), further expanding its clinical impact. If Kyowa Kirin opts into these additional indications, Kura could secure an additional $228 million in milestone payments.

With this partnership, Kura and Kyowa Kirin set a new benchmark in biopharmaceutical collaboration, aiming to redefine AML treatment on a global scale.

  Acute myeloid leukemia, AML, biopharma collaboration, Kura Oncology, Kyowa Kirin, ziftomenib