Phase 1b Study of Lower-Dose CPX-351 Plus Venetoclax As First-Line Treatment for Patients with AML Who Are Unfit for Intensive Chemotherapy: Preliminary Safety and Efficacy Results

Background: CPX-351 (United States: Vyxeos ®; Europe: Vyxeos ® Liposomal), a dual-drug liposomal encapsulation of daunorubicin and cytarabine in a synergistic 1:5 molar ratio, is approved for the treatment of newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in adults and pediatric patients aged ≥1 year in the United States and in adults in the European Union. In a phase 3 study in adults aged 60 to 75 years with newly diagnosed high-risk/secondary AML who were eligible for intensive chemotherapy (IC), after 5 years of follow-up CPX-351 significantly improved median overall survival versus conventional 7+3 cytarabine/daunorubicin, with a comparable safety profile. Venetoclax (VEN; BCL-2 inhibitor) + low-dose cytarabine has demonstrated efficacy in unfit patients with AML, and preclinical data support a rationale for combining CPX-351 + VEN. This study evaluates the safety and efficacy of lower-dose CPX-351 + VEN in adults with newly diagnosed AML who are considered unfit to receive IC.