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Asciminib single agent

Phase 2

Chronic Myelogenous Leukemia | Small molecule | Oncology |Novartis AG|Last Updated: May 26, 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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment50
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07354074Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CPPHASE2 RECRUITING 50Apr 28, 2026Feb 23, 2033May 26, 20264 Australia, Canada +1
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Study Endpoints
Primary Endpoints
MMR at Week 48
48 weeks

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.

Secondary Endpoints
MMR at Week 96
96 weeks
MMR at and by scheduled timepoints
Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Hematologic response at and by scheduled timepoints
Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Single arm studyEXPERIMENTALThis study will enroll pediatric patients (≥ 1 and \< 18 years of age) with newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation
Interventions
NameTypeDescription
Asciminib single agentDRUGAsciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)
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Eligibility Criteria
Age Range1 Year — 18 Years
SexALL
Healthy VolunteersNo
Study Sites4

Key Inclusion Criteria: Participants eligible for inclusion in this study must meet all of the following criteria: 1. Signed informed consent must be obtained prior to participation in the study. 2. Male or female participants 1 and \< 18 years of age at study enrollment 3. Diagnosis of CML-CP (Ap...

Countries:AustraliaCanadaSouth Korea
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Recent Changes (Last 90 Days)
LOWMay 27, 2026NCT07354074startDate: changed
LOWMay 27, 2026NCT07354074startDate: changed
LOWMay 26, 2026NCT07354074Status: NOT_YET_RECRUITING → RECRUITING
LOWMay 24, 2026NCT07354074studyFirstPostDate: changed