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← Chronic Myeloid Leukemia

HEALTH PROFESSIONAL · SOURCE READING

Asciminib

Source: Chronic Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: March 13, 2025 · Captured 2026-09-09.

Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.

Context: Treatment of Relapsed CML / Treatment Options for Relapsed CML / Targeted therapy with TKIs

Asciminib is an allosteric inhibitor of BCR::ABL1 at the ABL1 myristoyl pocket, a site unique from those used by TKIs.

Evidence (asciminib):

An open-label randomized clinical trial compared asciminib with bosutinib. With a median follow-up of 14.9 months, 233 patients with refractory or resistant disease were randomly assigned in a 2:1 ratio to receive either asciminib or bosutinib.[23]

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An open-label randomized clinical trial compared asciminib with bosutinib. With a median follow-up of 14.9 months, 233 patients with refractory or resistant disease were randomly assigned in a 2:1 ratio to receive either asciminib or bosutinib.[23]

The major molecular response (MMR) rate at week 24 was 25.5% for patients who received asciminib versus 13.2% for patients who received bosutinib. The difference in response (adjusted for major cytogenetic response at baseline) was 12.2% (95% CI, 2.19%–22.30%; P = .029).[23][Level of evidence B3]

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An open-label randomized clinical trial compared asciminib with bosutinib. With a median follow-up of 14.9 months, 233 patients with refractory or resistant disease were randomly assigned in a 2:1 ratio to receive either asciminib or bosutinib.[23]

Grade 3 or 4 adverse events were experienced by 50.6% of patients who received asciminib and 60.5% of patients who received bosutinib.

A phase I trial of asciminib included heavily pretreated patients who experienced resistance or unacceptable side effects after standard TKIs. Patients with a T315I variant and those in whom ponatinib failed were included.[24][Level of evidence C3]

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A phase I trial of asciminib included heavily pretreated patients who experienced resistance or unacceptable side effects after standard TKIs. Patients with a T315I variant and those in whom ponatinib failed were included.[24][Level of evidence C3]

Of 141 patients, 48% achieved an MMR by 12 months.

A phase II trial included 31 patients who received asciminib.[25][Level of evidence C3]

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A phase II trial included 31 patients who received asciminib.[25][Level of evidence C3]

An MMR rate of 41% was reported by 12 months.

A phase II trial included 31 patients who received asciminib.[25][Level of evidence C3]

Three of nine patients with disease that failed to respond to previous ponatinib responded to asciminib.

Publication references

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Preserved source evidence · Not medical advice