Kura Oncology announced that its oral menin inhibitor, KOMZIFTI (ziftomenib), has been added to the National Comprehensive Cancer Network (NCCN) Guidelines as a Category 2A recommended therapy for adults with relapsed or refractory NPM1‑mutated acute myeloid leukemia (AML). The guideline endorsement follows the drug’s U.S. FDA approval on November 13, 2025 and positions KOMZIFTI as the first‑in‑class, once‑daily oral therapy for this genetically defined AML subset.
The FDA approval was based on the Phase 2 KOMET‑001 study, which reported a 21.4 % complete remission or complete remission with partial hematologic recovery (CR/CRh) rate and a median duration of response of five months. Safety data showed no clinically significant QTc prolongation, but a boxed warning for differentiation syndrome—occurring in 26 % of patients and fatal in two cases—remains a critical consideration for clinicians.
Kura’s Q3 2025 financial results provide context for the commercial launch. The company posted a net loss of $74.1 million, up from $54.4 million in Q3 2024, while revenue of $20.8 million fell short of analyst estimates. Cash, cash equivalents, and short‑term investments stood at $609.7 million as of September 30, giving the company a runway that extends into 2027 and supporting the planned commercialization of KOMZIFTI across multiple AML indications.
The competitive landscape is shaped by Syndax’s revumenib, which received FDA approval for the same indication on October 24, 2025. Kura’s first‑in‑class status and once‑daily dosing may offer a clinical advantage, but the presence of an established competitor underscores the importance of payer coverage and clinical adoption for market share capture.
Analyst reactions to the FDA approval and subsequent NCCN endorsement were positive. UBS raised its price target for Kura to $16 from $14, while Wedbush lifted its target to $38 from $36, citing the drug’s strong efficacy profile and the potential for accelerated market penetration. These adjustments reflect confidence in Kura’s ability to translate regulatory milestones into commercial success.
Chief Medical Officer Mollie Leoni emphasized the significance of the guideline inclusion, stating, “The addition of KOMZIFTI to the NCCN Guidelines underscores the drug’s potential impact for patients with relapsed or refractory NPM1‑mutated AML and supports our commitment to ensuring that patients have access to this important treatment option.”
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