Definition

An orally bioavailable, selective, active-form inhibitor of tyrosine kinase BCR-ABL fusion oncoprotein, with potential antineoplastic activity. Upon oral administration, BCR-ABL inhibitor ELVN-001 specifically targets and binds to the ATP-binding site in the kinase domain of activated BCR-ABL1, thereby inhibiting the activity of both wild-type BCR-ABL and certain mutation forms, including the T315I mutation. This binding results in the inhibition of BCR-ABL-mediated proliferation and enhanced apoptosis of Philadelphia chromosome-positive (Ph+) hematological malignancies. The BCR-ABL fusion protein tyrosine kinase is an abnormal enzyme produced by leukemia cells that contain the Philadelphia chromosome. The T315I mutation, the most common BCR-ABL mutation, shows resistance to nearly all commonly used tyrosine kinase inhibitors (TKIs).