Discovery of asciminib (ABL001), an allosteric inhibitor of the tyrosine kinase activity of BCR-ABL1

J Schoepfer, W Jahnke, G Berellini, S Buonamici… - 2018 - ACS Publications
J Schoepfer, W Jahnke, G Berellini, S Buonamici, S Cotesta, SW Cowan-Jacob, S Dodd…
2018ACS Publications
Chronic myelogenous leukemia (CML) arises from the constitutive activity of the BCR-ABL1
oncoprotein. Tyrosine kinase inhibitors (TKIs) that target the ATP-binding site have
transformed CML into a chronic manageable disease. However, some patients develop drug
resistance due to ATP-site mutations impeding drug binding. We describe the discovery of
asciminib (ABL001), the first allosteric BCR-ABL1 inhibitor to reach the clinic. Asciminib
binds to the myristate pocket of BCR-ABL1 and maintains activity against TKI-resistant ATP …
Chronic myelogenous leukemia (CML) arises from the constitutive activity of the BCR-ABL1 oncoprotein. Tyrosine kinase inhibitors (TKIs) that target the ATP-binding site have transformed CML into a chronic manageable disease. However, some patients develop drug resistance due to ATP-site mutations impeding drug binding. We describe the discovery of asciminib (ABL001), the first allosteric BCR-ABL1 inhibitor to reach the clinic. Asciminib binds to the myristate pocket of BCR-ABL1 and maintains activity against TKI-resistant ATP-site mutations. Although resistance can emerge due to myristate-site mutations, these are sensitive to ATP-competitive inhibitors so that combinations of asciminib with ATP-competitive TKIs suppress the emergence of resistance. Fragment-based screening using NMR and X-ray yielded ligands for the myristate pocket. An NMR-based conformational assay guided the transformation of these inactive ligands into ABL1 inhibitors. Further structure-based optimization for potency, physicochemical, pharmacokinetic, and drug-like properties, culminated in asciminib, which is currently undergoing clinical studies in CML patients.
ACS Publications