The U.S. Food and Drug Administration granted full approval of Kura Oncology’s oral menin inhibitor, KOMZIFTI (ziftomenib), for adult patients with relapsed or refractory NPM1‑mutated acute myeloid leukemia (AML). The approval, announced on November 13, 2025, makes ziftomenib the first once‑daily, oral therapy for this genetically defined AML subset.
The pivotal KOMET‑001 Phase 2 study enrolled 112 patients and reported a complete remission plus CR with partial hematologic recovery (CR + CRh) rate of 21.4 % (23 % in the Phase 2 cohort). Median duration of CR + CRh was 5.0 months, and 88 % of responders achieved remission within six months of starting treatment. These data demonstrate meaningful disease control in a population with limited options.
Safety data were favorable. No boxed warning for QTc prolongation or torsades de pointes was issued, and the only boxed warning was for differentiation syndrome, a known class effect of menin inhibition. The absence of cardiac risk signals supports the drug’s suitability for outpatient use.
The approval unlocks a $350‑$400 million annual opportunity in the U.S. market for NPM1‑mutated AML. Kyowa Kirin’s partnership, which includes a $330 million upfront payment and co‑commercialization rights, provides Kura with capital and a distribution network that accelerates market entry. The partnership also signals confidence in the product’s commercial potential.
Kura’s Q3 2025 earnings reflected the impact of the new partnership. Collaboration revenue rose to $20.8 million, a sharp increase from zero in the prior year, but the company reported a net loss that widened to $0.85 per share versus an analyst estimate of $0.57. The earnings miss was driven by higher research and development and administrative expenses that offset the collaboration revenue, illustrating the company’s continued investment in pipeline development.
The company is advancing ziftomenib into frontline AML with the Phase 3 KOMET‑017 trial, which began enrolling patients in September 2025. Success in this trial could expand the drug’s indication to a larger patient population and further strengthen its market position.
CEO Troy Wilson emphasized that the approval “positions Kura to advance ziftomenib toward commercialization” and highlighted the company’s focus on leveraging the partnership to bring the therapy to patients quickly. Management also noted that the collaboration revenue provides a runway to fund ongoing clinical programs and support the company’s long‑term growth strategy.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.