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ABL001
ABL001 is an allosteric inhibitor of BCR-ABL that binds to a pocket distinct from the ATP-binding site, allowing it to overcome certain resistance mutations.
ABL001 is an allosteric inhibitor of BCR-ABL that binds to a pocket distinct from the ATP-binding site, allowing it to overcome certain resistance mutations. Used for Chronic myeloid leukemia (CML) with BCR-ABL mutations resistant to standard tyrosine kinase inhibitors.
At a glance
| Generic name | ABL001 |
|---|---|
| Sponsor | Marlise Luskin, MD |
| Drug class | BCR-ABL allosteric inhibitor |
| Target | BCR-ABL |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
ABL001 targets the BCR-ABL tyrosine kinase through an allosteric mechanism, binding to a regulatory site rather than competing for ATP binding. This approach enables activity against BCR-ABL variants that have developed resistance to ATP-competitive inhibitors like imatinib and dasatinib. The allosteric binding preserves normal ABL kinase regulation while selectively inhibiting the oncogenic BCR-ABL fusion protein.
Approved indications
- Chronic myeloid leukemia (CML) with BCR-ABL mutations resistant to standard tyrosine kinase inhibitors
Common side effects
- Musculoskeletal pain
- Fatigue
- Nausea
- Diarrhea
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)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |