ES-3000

ES-3000 is an orally bioavailable small molecule under development by Escend Pharmaceuticals for the treatment of myeloid malignancies, including acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndrome (MDS). It is also being investigated for idiopathic pulmonary fibrosis, hepatic fibrosis, dermal fibrosis, and was previously considered for triple negative breast cancer. The active ingredient is a plant-derived bis-benzylisoquinoline alkaloid (tetrandrine) that acts as an inhibitor of the Wnt/β-catenin pathway and calmodulin-dependent protein kinase II gamma (CaMKIIγ). By reducing β-catenin expression, ES-3000 ablates leukemic stem cells—cells implicated in disease resistance and relapse—offering a novel mechanism to potentially improve durable remission rates. The drug has received orphan drug designation from the FDA for AML and CML. Clinical studies have shown promising response rates in MDS patients as both monotherapy and in combination with decitabine/cedazuridine[1][2][4][5][6][7][8].

Other names

tetrandrine bis-benzylisoquinoline alkaloid