Elias Jabbour, MD, MD Anderson Cancer Center at the University of Texas, discusses the cAMeLot-2 study design of bleximenib combination therapy for patients with acute myeloid leukemia (AML).
AML is a hematologic malignancy. Early signs and symptoms of AML vary but may include easy bruising, bone pain or tenderness, fatigue, fever, frequent nosebleeds, bleeding from the gums, shortness of breath, and/or weight loss. AML is one of the most common types of leukemia among adults and is rarely diagnosed in people under age 40. There are many potential causes of AML such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop AML have no identifiable risk factor.
Bleximenib is a menin inhibitor designed to target specific genetic subtypes of AML, namely patients with KMT2Ar and NPM1m AML. By potently and selectively disrupting KMT2A from binding to menin, bleximenib induces leukemia cell differentiation and cell death.
In the phase 1 ALE1002 (NCT05453903) clinical trial, high rates of response were observed at the bleximenib recommended phase 2 dose of 100 mg BID in combination with venetoclax and azacitidine (VEN + AZA) in patients with KMT2Ar or NPM1m AML. The safety profile of bleximenib in combination with VEN + AZA was consistent, with no new drug-drug interactions observed or QTc prolongation signal identified.
cAMeLot-2 (NCT06852222) is a Phase 3, randomized, double-blind, placebo-controlled, global multicenter study evaluating the safety and efficacy of bleximenib, with VEN + AZA, in adults with newly diagnosed KMT2Ar or NPM1mAML who are ineligible for intensive chemotherapy. 600 patients will be randomized to bleximenib 100 mg BID or placebo, both in combination with VEN + AZA.
Primary endpoints for the cAMeLot-2 study are complete response rate and overall survival. Secondary endpoints include event-free survival, duration of complete response, time to complete response, rate of complete response without measurable residual disease, transfusion independence, allogeneic stem cell transplantation rate, incidence of adverse events, clinical laboratory parameter abnormalities, and serum concentration of bleximenib.
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