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ABL001/Asciminib

Phase 2

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06514534Open-label Study of Asciminib for CML-CP or CML-AP Patients With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.PHASE2 RECRUITING 20Feb 18, 2025Feb 18, 2029Apr 21, 20266 France
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06514534primaryCompletionDate: changed
LOWMay 24, 2026NCT06514534studyFirstPostDate: changed