JNJ-75276617 for Acute Leukemia

(cAMeLot-1 Trial)

This trial tests a new treatment called bleximenib (also known as JNJ-75276617) for individuals with specific types of acute leukemia, a cancer of the blood and bone marrow. The research aims to determine the optimal dose of bleximenib that is both safe and effective in slowing or stopping leukemia. Participants will receive the treatment in different phases to evaluate its efficacy and safety. This trial may suit those with relapsed or resistant acute leukemia who have specific genetic changes, such as KMT2A or NPM1 mutations, and have not succeeded with other treatments. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

The trial protocol does not specify if you must stop taking your current medications. However, you cannot have had prior cancer immunotherapy within 4 weeks before joining the trial, and other cancer therapies must not be given within 4 weeks or 5 half-lives of the agent, whichever is shorter.

Research has shown that JNJ-75276617, also known as bleximenib, has been safe in previous studies. Earlier research found no serious heart rhythm problems and no severe side effects. Only a few mild side effects were reported, which encourages those considering participation. Overall, the safety data suggest that bleximenib is well-tolerated, making it a good option for participants in this study.¹²³⁴⁵

Unlike the standard chemotherapy and targeted therapies for acute leukemia, bleximenib is unique because it targets specific proteins involved in the growth and survival of leukemia cells. Researchers are excited about bleximenib because it offers a novel mechanism of action that could potentially overcome resistance seen with current treatments. Additionally, bleximenib is administered orally, providing a more convenient option compared to traditional intravenous therapies. These features make bleximenib a promising candidate for improving outcomes in acute leukemia patients.

Research has shown that bleximenib, the investigational treatment in this trial, yields promising results for acute leukemia. In one study, 82% of patients with relapsed or hard-to-treat acute myeloid leukemia (AML) improved with the recommended dose of bleximenib, indicating that most patients experienced better outcomes. Earlier animal studies also support these findings, demonstrating that bleximenib reduced leukemia and extended survival. These results suggest that bleximenib may work by targeting a specific protein interaction in cancer cells.¹²⁵⁶⁷