Novartis gets USFDA Breakthrough Therapy designations for STAMP inhibitor asciminib to treat chronic myeloid leukemia

Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP). As a STAMP inhibitor, asciminib is being studied in patients with chronic myeloid leukemia (CML) who experience resistance or intolerance to two or more tyrosine-kinase inhibitors (TKIs), and in several clinical trials in hopes of helping patients across multiple treatment lines of CML.

ASCEMBL is the first head-to-head clinical trial in chronic myeloid leukemia using a second-generation tyrosine-kinase inhibitor (TKI) as a comparator. As a Phase III, multicenter, open-label, randomized study, ASCEMBL was designed to evaluate superiority in major molecular response rate at 24 weeks of the oral investigational treatment asciminib (ABL001) versus bosutinib in patients with Philadelphia-chromosome positive CML in chronic phase previously treated with two or more TKIs. Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial.