Asciminib is a medication used to treat patients with chronic myeloid leukemia (CML). It works by targeting specific genetic mutations in leukemia cells, particularly in patients who have developed a specific mutation called T315I. This medication is designed for patients who cannot take or have not responded well to another medication called ponatinib.

Ponatinib is a reference medication in this trial, though not actively used. It is a type of cancer medication that is typically used to treat chronic myeloid leukemia, but some patients may be resistant to it, unable to tolerate it, or not eligible to receive it.

Chronic Myeloid Leukemia (CML) – A type of blood cancer that begins in the bone marrow, causing the body to produce too many white blood cells. The disease typically progresses through three phases: chronic phase (CP), accelerated phase (AP), and blast phase. In the chronic phase, there are increased numbers of white blood cells, but they still function normally. The accelerated phase shows more immature blood cells and more severe symptoms. The T315I mutation is a specific genetic change that makes some CML cases resistant to certain treatments. Patients with this mutation have a distinct form of CML that requires specialized medical attention.