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Asciminib Hydrochloride is the hydrochloride form of asciminib, an orally bioavailable variant Bcr-Abl1 tyrosine kinase inhibitor with antitumour activity. Upon administration, aximinib targets and binds to the myristoyl pocket of the Bcr-Abl1 fusion protein that is distinct from the ATP-binding domain, thereby inhibiting the activity of wild-type Bcr-Abl and certain mutants, including the T315I mutation. This binding inhibits Bcr-Abl1-mediated proliferation and enhances apoptosis in Philadelphia chromosome-positive (Ph+) haematological malignancies.The Bcr-Abl1 fusion protein tyrosine kinase is an aberrant enzyme produced by Philadelphia chromosome-containing leukaemia cells.

Asciminib (ABL001) hydrochloride is a potent and selective allosteric BCR-ABL1 inhibitor, which inhibits Ba/F3 cells grown with an IC50 of 0.25 nM[1].

Asciminib hydrochloride is approved for the treatment of adult patients with chronic myeloid leukaemia (CML) who are in the chronic phase and are Philadelphia chromosome positive. This includes:
Patients who have been treated with at least two tyrosine kinase inhibitors;
Patients with CML who have the T315I mutation;
Newly diagnosed CML patients

[1] Wylie AA, et al. The allosteric inhibitor ABL001 enables dual targeting of BCR-ABL1. Nature. 2017 Mar 30;543(7647):733-737. DOI:10.1038/nature21702