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ABL001/Asciminib

Phase 2

Chronic Myeloid Leukemia (CML) | Small molecule | Oncology |Novartis AG|Last Updated: Apr 21, 2026

Target and mechanism

Molecular targetBCR-ABL1
Target classProtein
ModalitySmall molecule

Also known as ABL001

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment20

FDA Designations

No designations recorded

Clinical trial landscape

ABL001/Asciminib · 1 trial · 1 indication

Phase 2 1
NCT06514534Open-label Study of Asciminib for CML-CP or CML-AP Patients With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.Chronic Myeloid Leukemia (CML)
RECRUITING20 Analytics
PHASE2RECRUITING
Open-label Study of Asciminib for CML-CP or CML-AP Patients With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.
Chronic Myeloid Leukemia (CML)Unlock trial analytics

Study Endpoints

Primary Endpoints

Rate of BCR::ABL1 (Breakpoint Cluster Region Gene::Abelson proto-oncogene) IS (International Scale) ≤ 1% [MR2 (Molecular Response 2)]
Month 12

Evaluation of asciminib efficacy : proportion of patients with MR2 (BCR::ABL1 IS ≤1%) level of response at 12 months.

Secondary Endpoints

Kinetics of response: BCR::ABL1 IS (MR2, MMR, MR4.0, MR4.5, undetectable MR4.5)
At 3, 6, 9, 12, 18 and 24 months
Estimate response to treatment MR2 at 12 months in participants with BCR::ABL1 IS > 1% at treatment initiation or maintenance of MR2 at 12 months in participants with MR2 at treatment initiation
At 12 months
Time to Major Molecular Response (MMR) (for participants not in MMR at treatment initiation)
up to 24 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Asciminib (Scemblix®)EXPERIMENTALAsciminib will be administered 200 mg twice a day orally. The minimum dose is 200 mg, and maximum dose is 400 mg.

Interventions

NameTypeDescription
ABL001/AsciminibDRUGThe study treatment for this clinical trial is an investigational drug called asciminib, which is marketed under the brand name Scemblix®. Asciminib is a compound that is being evaluated for its efficacy and safety in the treatment of the target condition. The minimum dose of asciminib to be administered in this study is 200 mg, while the maximum dose is 400 mg. The dose is planned as 200 mg twice a day (BID). The drug will be administered orally, allowing for convenient and non-invasive administration.
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Eligibility Criteria

Age Range18 Years to 99 Years
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Signed informed consent must be obtained prior to participation in the study. * Male or female participants with a diagnosis of CML-CP or CML-AP ≥ 18 years of age. * Patients with CML-CP or CML-AP with history of documented T315I mutation after at least one TKI and are resista...

Countries:France
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Frequently asked questions about ABL001/Asciminib

What is ABL001 used for?

ABL001, also known as asciminib, is an investigational small molecule being studied for the treatment of chronic myelogenous leukemia (CML), including chronic phase CML, B-cell acute lymphoblastic leukemia, and Philadelphia chromosome positive acute lymphoblastic leukemia. It is also being evaluated in patients with hepatic impairment.

What does ABL001 target?

ABL001 targets the BCR-ABL1 fusion protein, which drives the growth of cancer cells in chronic myeloid leukemia and Philadelphia chromosome positive acute lymphoblastic leukemia. By inhibiting this protein, the drug aims to block the signaling pathways that promote cancer cell proliferation.

Who makes ABL001?

ABL001 is being developed by Novartis AG, a global pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy in multiple oncology indications.

What phase is ABL001 in?

ABL001 is in clinical development across multiple phases. It is being studied in Phase 1, Phase 2, and Phase 3 trials for various indications including chronic myeloid leukemia and acute lymphoblastic leukemia. The drug is investigational and has not been approved by regulatory authorities.

What clinical trials is ABL001 in?

ABL001 is being evaluated in several clinical trials, including NCT03595917, a Phase 1 study combining ABL001 with dasatinib, prednisone, and blinatumomab in BCR-ABL+ B-ALL or CML. Other trials include NCT04666259, NCT04948333, and NCT06514534, which assess asciminib in different CML patient populations.

Is ABL001 the same as asciminib?

Yes, ABL001 is the same as asciminib. The drug is referred to by both names in clinical research and development. Asciminib is the international nonproprietary name, while ABL001 is the compound code used by the developer, Novartis.