Promising Phase 1b Results for Johnson & Johnson's Bleximenib in Combating Acute Myeloid Leukemia

Johnson & Johnson Reports Encouraging Phase 1b Data on Bleximenib

Johnson & Johnson (NYSE: JNJ) has shared exciting new findings regarding its investigational drug, Bleximenib (JNJ-75276617), which exhibits significant antileukemic potential for patients battling acute myeloid leukemia (AML). This data comes following a Phase 1b trial assessing the drug's effectiveness in conjunction with venetoclax (VEN) and azacitidine (AZA) for treating AML, particularly among patients ineligible for intensive chemotherapy.

The study results were recently highlighted during an oral presentation at the European Hematology Association (EHA) Congress in 2025, drawing attention to Bleximenib's promising safety profile and effectiveness against AML with KMT2A gene rearrangements or NPM1 mutations.

Understanding AML and Its Challenges

Acute myeloid leukemia is recognized as one of the most daunting forms of leukemia, notoriously aggressive and fast-spreading. It primarily results from the uncontrolled growth of myeloblasts originating in the bone marrow. Despite advancements in treatment, AML continues to pose a significant threat, particularly to older patients or those with high-risk genetic profiles, resulting in a grim five-year survival rate.

Dr. Andrew M. Wei from Peter MacCallum Cancer Centre notes, "AML encompasses a spectrum of genetically diverse cancers affecting 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 newly diagnosed with AML who are ineligible for intensive chemotherapy or have relapsed after prior therapy."

Study Overview

The Phase 1b study, which enrolled 125 patients, compared various dosage levels of Bleximenib in combination with VEN + AZA, without requiring step-up dosing. Among the participants, a substantial proportion had previously failed multiple lines of treatment, with 47% having received venetoclax.

Notably, Bleximenib—when administered at a dose of 100 mg twice daily—demonstrated superior efficacy, achieving an impressive overall response rate (ORR) of 82% and a composite complete response (cCR) rate of 59% for relapsed or refractory patients, while newly diagnosed patients saw an ORR of 90% and a cCR rate of 75%. These outcomes underscore the compound's potential as a robust therapeutic option for AML, marking a hopeful advancement in the field.

Safety Profile

The safety data from the trial revealed a commendable profile, with differentiation syndrome events occurring in only 4% of patients, and no significant QTc prolongation reported. While common treatment-emergent adverse events included nausea, thrombocytopenia, neutropenia, and anemia, the management of these issues continues to support Bleximenib's viability as a treatment avenue in AML.

Dr. Jeffrey Infante, Vice President of Early Clinical Development at Johnson & Johnson, remarked, "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."

Future Directions

Johnson & Johnson's commitment extends beyond the current findings, as they continue to explore Bleximenib's role as both a monotherapy and in combination with other standard treatments in future Phase 2 and 3 studies.

This ongoing work underscores the company's dedication to pioneering effective treatments for complex diseases, aiming to carve a pathway for better patient outcomes in the challenging landscape of aggressive blood cancers like AML.

In conclusion, Bleximenib's promising Phase 1b results not only reflect significant therapeutic potential but also mark a pivotal moment in the continued fight against acute myeloid leukemia. As Johnson & Johnson forges ahead with further investigative studies, the hope for improved survival rates in this high-risk patient population remains bright.