Drug intelligence / Profile preview

asciminib

Development stage
Approved
Lead developer
Novartis
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Irreversible Covalent Inhibitors → Covalent Small Molecules → Small Molecules, Allosteric Modulators → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Asciminib is a small molecule tyrosine kinase inhibitor used for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase, including patients who are newly diagnosed, those previously treated with two or more tyrosine kinase inhibitors (TKIs), and those with the T315I mutation. Unlike other TKIs that bind to the ATP-binding site of BCR-ABL1, asciminib uniquely acts as an allosteric inhibitor by binding to the myristoyl pocket of the BCR-ABL1 fusion protein, locking it into an inactive conformation. This mechanism allows it to overcome resistance seen with other TKIs and target both wild-type and mutant forms (including T315I) of BCR-ABL1. Asciminib was developed by Novartis and is marketed under the brand name Scemblix[2][4][6][8].

Brand names
Scemblix
Other names
asciminib hydrochloride
02

Targets

ABL1 (ABL proto-oncogene 1, non-receptor tyrosine kinase)

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