Efficacy and Safety of the First-in-Class STAMP-Inhibitor Asciminib in Patients With Chronic Myeloid Leukemia.

Abstract

The survival outcomes of patients with chronic myeloid leukemia (CML) have significantly improved due to the introduction of adenosine triphosphate (ATP) -competitive ABL1 tyrosine kinase inhibitors (TKIs). However, several patients with CML eventually develop treatment resistance or intolerance during the course of ATP-competitive ABL1 TKI treatment. ABL1 TKIs inhibit other tyrosine kinases via their off-target effects. This mechanism leads to the development of adverse events, which may result in treatment discontinuation. Asciminib is a first-in-class STAMP (specifically targeting the ABL myristoyl pocket) inhibitor used in patients with chronic-phase CML who exhibit resistance or intolerance to two prior TKI therapies. Asciminib was found to have excellent efficacy and safety therapeutic profiles. The lack of comprehensive reviews about asciminib, thus, the current study aimed to evaluate the clinical and preclinical evidence of the efficacy and safety of asciminib.