Johnson & Johnson reported encouraging new results from a Phase 1b trial that found its experimental oral medicine, bleximenib, given with the routine treatment regimen of venetoclax plus azacitidine, exhibited strong antileukemic activity in adults with acute myeloid leukemia (AML). The findings, reported at the 2025 European Hematology Association (EHA) Congress, bring new hope to patients with particular genetic mutations that render the disease particularly resistant to treatment.
It targeted patients with de novo AML who are not candidates for induction chemotherapy and R/R AML. The three-drug combination achieved a remarkable 82 percent overall response rate (ORR) and 59 percent composite complete response (cCR) rate in R/R patients at the Phase 2 recommended dose. The outcomes were even more impressive in the recently diagnosed, intensive chemo-ineligible patients, who had a 90 percent ORR and a 75 percent cCR.
AML is a fast-evolving blood and bone marrow cancer and possesses one of the lowest survival rates for any form of leukemia. A high unmet need is present for novel treatments, particularly for patients with KMT2A or NPM1 gene mutations, which portend poor prognoses.
AML encompasses a spectrum of genetically diverse cancers affecting the bone marrow and blood, which progress rapidly, making it an extremely challenging cancer to treat,
These data highlight the potential of this targeted therapy in combination with VEN + AZA for patients with newly diagnosed AML who are ineligible for intensive chemotherapy or with disease that has relapsed after prior therapy.
Andrew M. Wei
Bleximenib is a first-in-class, orally active and highly selective menin inhibitor. It inhibits the essential interaction between menin and KMT2A, a protein complex that induces the proliferation and growth of leukemia cells in patients with KMT2A rearrangements or NPM1 mutations. This targeted mechanism is thought to act synergistically with venetoclax and azacitidine to effectively destroy the cancer cells.
The safety profile of the combination bleximenib was also consistent with each drug’s known profiles. The most frequent treatment-emergent adverse events were nausea, thrombocytopenia, neutropenia, and anemia. Notably, the rate of differentiation syndrome, a recognized complication of drugs in this class, was low.
Building on our heritage of leadership and innovation in hematologic malignancies, we are committed to delivering transformative treatment options that address the significant unmet needs of patients with acute myeloid leukemia,
We continue to explore the potential of this compound as a monotherapy and in combination with standard of care regimens in additional Phase 2 and 3 studies, which are currently enrolling patients.
Dr. Jeffrey Infante
The Phase 1b study (NCT05453903) has already enrolled 125 patients to date and is intended to identify the recommended Phase 2 dose and continue to evaluate the safety and tolerability of the combination. The robust efficacy signals seen present a sound basis for further development of bleximenib as a next-generation cornerstone therapy for genetically well-defined AML patients.
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