Promising Early Results for Ziftomenib Combo in Treating AML

Kura Oncology, Inc., a clinical-stage biopharmaceutical company specializing in precision cancer treatments, announced promising preliminary results from the KOMET-007 Phase 1 dose-escalation trial. This trial evaluated the menin inhibitor ziftomenib in combination with cytarabine/daunorubicin (7+3) and venetoclax/azacitidine (ven/aza) in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML).

Between July and November 2023, the trial enrolled 20 patients, including five newly diagnosed cases with adverse-risk NPM1-m or KMT2A-r AML and 15 patients with refractory/relapsed (R/R) NPM1-m or KMT2A-r AML. Ziftomenib at a 200 mg daily dose was well tolerated, showing no differentiation syndrome events or significant toxicities.

As of January 11, 2024, all newly diagnosed patients treated with ziftomenib and 7+3 achieved complete remission (CR) with full recovery. The CR rate was 100% for these patients, including four with NPM1-m AML and one with KMT2A-r AML. Among R/R patients treated with ziftomenib and ven/aza, the overall response rate (ORR) was 53%, with a CR/CRh rate of 56% among those not previously treated with a menin inhibitor.

Dr. Amer Zeidan from Yale Cancer Center highlighted the fast enrollment in this first-in-human study and noted the promising safety and efficacy profile of ziftomenib. The trial showed potential for this combination therapy to provide clinical benefits for difficult-to-treat AML patient populations.

The trial continues to evaluate higher doses, with 80% of patients remaining on trial as of the data cutoff date. Kura Oncology plans to proceed with a Phase 1b trial upon establishing the recommended Phase 2 dose of ziftomenib.

CEO Troy Wilson expressed optimism about the potential of ziftomenib to significantly impact AML treatment outcomes. The rapid enrollment in ongoing studies reflects strong investigator enthusiasm, and the company aims to complete enrollment for KOMET-001, their Phase 2 trial, by mid-year.

Ziftomenib, an investigational oral drug targeting the menin-KMT2A/MLL interaction, has demonstrated anti-leukemic effects in preclinical models. It has received Orphan Drug Designation from the FDA for AML treatment, targeting significant unmet needs in genetically defined AML patient populations.

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