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Asciminib add-on

Phase 2

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
RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment104
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03578367Study of Efficacy and Safety of Asciminib in Combination With Imatinib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Who Have Been Previously Treated With Imatinib and Have Not Achieved Deep Molecular Response.PHASE2 COMPLETED 104Nov 22, 2018Feb 26, 2025Apr 21, 202630 United States, Austria +13
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Study Endpoints
Primary Endpoints
Molecular Response (MR)^4.5 Rate at 48 Weeks and Difference in Rate Between Asciminib + Imatinib and Imatinib Alone
at Week 48

Percentage of participants still treated with the randomized treatment at 48 weeks and are in MR\^4.5 (BCR::ABL1 ratio of ≤ 0.0032%) at 48 weeks (± assessment window), among all participants in the asciminib add-on arms vs imatinib arm.

Secondary Endpoints
Rate of MR^4.5 at 48 Weeks (Asciminib add-on Arms vs Nilotinib)
at Week 48
Rate of MR^4.5 by 48 Weeks (Randomized Arms)
by 48 weeks
Rate of MR^4.5 at 96 Weeks (Randomized Arms) and Difference in Rate Between Asciminib + Imatinib and Nilotinib Alone
at Week 96
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Asciminib 60mg QD + Imatinib 400mg QDEXPERIMENTALAsciminib 60 mg taken once daily in combination with Imatinib 400 mg taken once daily
Asciminib 40mg QD + Imatinib 400mg QDEXPERIMENTALAsciminib 40 mg taken once daily in combination with Imatinib 400 mg taken once daily
Imatinib 400mg QDACTIVE_COMPARATORImatinib 400 mg taken once daily
Nilotinib 300mg BIDACTIVE_COMPARATORNilotinib 300 mg taken twice daily
Asciminib 80mg QD (ASAC)EXPERIMENTALAsciminib 80 mg taken once daily
Interventions
NameTypeDescription
Asciminib add-onDRUGAsciminib was supplied as 40 mg and 20 mg tablets and taken orally once daily.
ImatinibDRUGImatinib was supplied as 400 mg and 100 mg tablets and taken orally once daily.
NilotinibDRUGNilotinib was supplied as 150 mg and 200 mg hard gelatin capsules and taken orally twice daily.
Asciminib 80mg QD (asciminib single agent (ASAC))DRUGAsciminib was supplied as 40 mg and 20 mg tablets and taken orally once daily (in the fasted state) on a continuous schedule (QD).
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Eligibility Criteria
Age Range18 Years — 100 Years
SexALL
Healthy VolunteersNo
Study Sites30

Inclusion Criteria: * Male or female patients ≥ 18 years of age with a confirmed diagnosis of CML-CP. * Minimum of one year (12 calendar months) treatment with imatinib first line for CML-CP (patients have to be on imatinib 400 mg QD at randomization and had no dose change in the past three months)...

Countries:United StatesAustriaCanadaCzechiaDenmarkFranceGermanyItalyPolandPortugalRussiaSouth KoreaSpainTaiwanUnited Kingdom
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