Kura Oncology disclosed that its oral menin inhibitor KOMZIFTI (ziftomenib) combined with venetoclax and azacitidine achieved an 86% composite complete remission (CRc) rate in newly diagnosed patients with NPM1‑mutated acute myeloid leukemia (AML). In the relapsed or refractory (R/R) NPM1‑mutated cohort, the triplet produced a 65% overall response rate (ORR), while 83% of venetoclax‑naïve R/R patients responded. For patients with KMT2A‑rearranged AML, the regimen yielded a 41% ORR. Among CRc responders, 68% achieved molecular minimal residual disease (MRD) negativity by central next‑generation sequencing.
The data come from the ongoing KOMET‑007 Phase 1a/1b trial, with a data cutoff of September 24 2025. These are the first published results for the triplet regimen, and the safety profile was comparable to venetoclax/azacitidine alone, with no new safety signals reported. The high response rates and deep MRD responses support the hypothesis that ziftomenib can enhance the efficacy of standard AML therapies without adding significant toxicity.
Strategically, the results expand the potential clinical use of ziftomenib beyond its current approval for R/R NPM1‑mutated AML. The data reinforce Kura’s plan to pursue a Phase 3 KOMET‑017 trial in frontline AML and may accelerate regulatory discussions and payer negotiations for broader access. By demonstrating robust activity in both newly diagnosed and R/R settings, the company positions ziftomenib as a foundational, best‑in‑class menin inhibitor that could become a standard component of AML treatment regimens.
"We’re truly encouraged by the consistent safety profile and the depth of responses observed with ziftomenib in combination with venetoclax and azacitidine across both newly diagnosed and relapsed/refractory NPM1‑mutated and KMT2A‑rearranged AML patients," said Mollie Leoni, M.D., Chief Medical Officer at Kura Oncology. "These compelling data reinforce our conviction that ziftomenib has the potential to become a foundational, best‑in‑class menin inhibitor for patients with AML." Dr. Gail J. Roboz, a leading hematologist, added, "The addition of ziftomenib to venetoclax and azacitidine has shown promising clinical activity, with 86% of newly diagnosed NPM1‑mutated AML patients achieving composite complete remission and 68% attaining deep molecular MRD negativity, although median duration of response and overall survival remain immature."
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