Last reviewed 2026-04-20 · How we verify

Scemblix (ASCIMINIB)

Scemblix works by blocking the ABL1 enzyme, which is involved in the growth and survival of cancer cells.

At a glance

Mechanism of action

Asciminib is an ABL/BCR-ABL1 tyrosine kinase inhibitor. Asciminib inhibits the ABL1 kinase activity of the BCR-ABL1 fusion protein, by binding to the ABL myristoyl pocket. In studies conducted in vitro or in animal models of CML, asciminib showed activity against wild-type BCR-ABL1 and several mutant forms of the kinase, including the T315I mutation.

Approved indications

• Newly diagnosed Ph+ CML in CP
• Previously treated Ph+ CML in CP
• Ph+ CML in CP with T315I mutation

Common side effects

• Upper respiratory tract infections
• Musculoskeletal pain
• Thrombocytopenia
• Neutropenia
• Fatigue
• Rash
• Hypertension
• Diarrhea
• Nausea
• Abdominal pain
• Pyrexia
• Cardiac failure congestive

Key clinical trials

• Asciminib Maintenance Therapy Following alloHCT or CAR T to Prevent Relapse in Adults With Ph+ALL (PHASE1)
• Asciminib & Standard-of-Care Integration in Maintenance Therapy for POST Allogeneic Stem Cell Transplant (Allo-HSCT) of Patient With Ph+ B-ALL or Blastic Transformed CML (PHASE2)
• Full-Course Immunotherapy Consolidation for Unfit or Fit B-ALL Who Decline Chemotherapy (PHASE2)
• Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP (PHASE2)
• Asciminib as Initial Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase (PHASE2)
• Asciminib Treatment Optimization in ≥ 3rd Line CML-CP (PHASE3)
• Asciminib With or Without Sildenafil for Brain Tumors (EARLY_PHASE1)
• A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP (PHASE3)

Patents

Primary sources

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