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Asciminib

Phase 3

Chronic Myelogenous Leukemia | Small molecule | Oncology |Novartis AG|Last Updated: Aug 26, 2026

Target and mechanism

Molecular targetABL1, BCR
Target classInhibitor
ModalitySmall molecule

Also known as Asciminib and Trastuzumab

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMC
Total Trials2
Total Enrollment559

FDA Designations

No designations recorded

Clinical trial landscape

Asciminib · 6 trials · 6 indications

Phase 3 2Phase 2 2Phase 1 2
NCT05456191A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)Philadelphia Chromosome-Positive Chronic Myeloid Leukemia
ACTIVE NOT_RECRUITING568 Analytics
NCT03106779Study of Efficacy of CML-CP Patients Treated With ABL001 Versus Bosutinib, Previously Treated With 2 or More TKIsChronic Myelogenous Leukemia
COMPLETED233 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)
Philadelphia Chromosome-Positive Chronic Myeloid LeukemiaUnlock trial analytics
PHASE3COMPLETED
Study of Efficacy of CML-CP Patients Treated With ABL001 Versus Bosutinib, Previously Treated With 2 or More TKIs
Chronic Myelogenous LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Time to Discontinuation of Study Treatment Due to Adverse Event (TTDAE) (From Interim Analysis)
From date of first dose to date of treatment discontinuation due to AE, assessed after 50 events had occurred, about 2 years since first patient first visit (FPFV).

TTDAE is defined as the interval from the date of first study treatment administration to the date of discontinuation of study treatment due to an adverse event (AE). The comparison between the asciminib and nilotinib treatment arms is performed using a cause-specific hazard model, in which discontinuations due to AE are considered the event of interest and discontinuations for reasons other than AE are treated as competing risks. The number of participants who discontinued study treatment due to AE within the specified timeframe is presented in the results table. The formal comparison between treatment arms is performed using a cause-specific hazard model, and the corresponding hazard ratio, confidence interval, and p-value are provided in the Statistical Analysis section. The TTDAE endpoint is event-driven by counting how many participants have experienced treatment discontinuations due to AE.

Time to Discontinuation of Study Treatment Due to Adverse Event (TTDAE) (From Primary Analysis)
From date of first dose to date of treatment discontinuation due to AE, assessed after 67 events had occurred, about 2.5 years since first patient first visit (FPFV),

TTDAE is defined as the interval from the date of first study treatment administration to the date of discontinuation of study treatment due to an adverse event (AE). The comparison between the asciminib and nilotinib treatment arms is performed using a cause-specific hazard model, in which discontinuations due to AE are considered the event of interest and discontinuations for reasons other than AE are treated as competing risks. The number of participants who discontinued study treatment due to AE within the specified timeframe is presented in the results table. The formal comparison between treatment arms is performed using a cause-specific hazard model, and the corresponding hazard ratio, confidence interval, and p-value are provided in the Statistical Analysis section. The TTDAE endpoint is event-driven by counting how many participants have experienced treatment discontinuations due to AE.

Major Molecular Response (MMR) Rate at 24 Weeks
24 weeks

MMR was 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 (IS) as measured by RQ-PCR.

Percentage of participants who achieve Major Molecular Response (MMR) in the 2L setting
Baseline up to 12 months

MMR is defined as BCR::ABL1IS ≤ 0.1%. A patient will be counted as having achieved MMR at 12 month if he/she meets the MMR criterion at 12 month.

Major molecular response rate of asciminib
week 24

Evaluate the major molecular response rate at 24 weeks in asciminib arm

Pharmacokinetics: Plasma concentration of asciminib by AUClast
pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, 48 and 72 hours post-dose

The AUC from time zero to the last measurable concentration sampling time (tlast) (ng\*h/mL)

Pharmacokinetics: Plasma concentration of asciminib by AUCinf
pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, 48 and 72 hours post-dose

The AUC from time zero to infinity (ng\*h/mL)

Pharmacokinetics: Plasma concentration of asciminib by Cmax
pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, 48 and 72 hours post-dose

The maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration (ng/mL)

Pharmacokinetics: Clearance of asciminib from plasma by CL/F
pre-dose (0 hour) and at 0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, 48 and 72 hours post-dose

The total body clearance of drug from the plasma (L/h)

Incidence of dose limiting toxicities (DLTs) during the first cycle of study treatment
First Cycle is 28 days

Determine the MTD and/or RDE of ABL001 as single agent in CML and Ph+ ALL, and in combination with either nilotinib or imatinib or dasatinib in CML patients

Secondary Endpoints

Percentage of Participants With Major Molecular Response (MMR) at Scheduled Data Collection Time Points
approximately 7.5 years
Percentage of Participants With Major Molecular Response (MMR) by Scheduled Data Collection Time Points
approximately 7.5 years
Percentage of Participants With MR4.0 at Scheduled Data Collection Time Points
approximately 7.5 years
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AsciminibEXPERIMENTALParticipants received asciminib 80 mg once a day (QD).
NilotinibACTIVE_COMPARATORParticipants will receive nilotinib 300 mg BID
BosutinibACTIVE_COMPARATORPatients were randomized to bosutinib 500mg QD
asciminb armEXPERIMENTALPatients will receive asciminib (40 mg BID continuous)
best available treatment armEXPERIMENTALPatients will receive best available therapy chosen by investigator
Normal renal functionEXPERIMENTALhealthy volunteers with normal renal function
Severe renal impairmentEXPERIMENTALsubjects with severe renal impairment
Moderate renal impairmentEXPERIMENTALsubject with moderate renal impairment
Mild renal impairmentEXPERIMENTALsubjects with mild renal impairment
Asciminib in CML patientsEXPERIMENTALDose escalation study estimated the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of asciminib in adult patients with chronic myeloid leukemia (CML).
Asciminib+Nilotinib in CML patientsEXPERIMENTALDose escalation study estimated the MTD and/or RDE of asciminib in combination with Nilotinib in adult CML patients
Asciminib in Ph+ ALL patientsEXPERIMENTALDose escalation study estimated the MTD and/or RDE of asciminib in adult patients with Ph positive ALL patients
Asciminib+Imatinib in CML patientsEXPERIMENTALDose escalation study to estimate the MTD and/or RDE of asciminib in combination with imatinib in adult CML patients
Asciminib+dasatinib in CML patientsEXPERIMENTALDose escalation study estimated the MTD and/or RDE of asciminib in combination with dasatinib in adult CML patients

Interventions

NameTypeDescription
AsciminibDRUGAsciminib 80 mg QD administered under fasting conditions.
NilotinibDRUGNilotinib 300 mg twice a day (BID) was administered under fasting conditions.
BosutinibDRUG500 mg tablets was taken orally once daily (QD)
best available treatmentOTHERBest available treatment will be based on investigator's choice identified prior to randomization. Dose and frequency will depend on label and institutional guidelines for various BAT
Asciminib (ABL001)DRUGAsciminib was be administered orally in a dose escalation schedule.
ImatinibDRUGAsciminib and imatinib was administered orally in CML patients
DasatinibDRUGAsciminib and dasatinib was administered orally in CML patients
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Eligibility Criteria

Age Range18 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites119

Key Inclusion Criteria: * Signed informed consent must be obtained prior to participation in the study. * Male or female patients ≥ 18 years of age. * Patients with CML-CP within 3 months of diagnosis. * Diagnosis of CML-CP (European Leukemia Network \[ELN\] 2020 criteria) with cytogenetic confirma...

Countries:United StatesArgentinaBulgariaCanadaCzechiaFranceGermanyGreeceHungaryIndiaItalyJordanMalaysiaNetherlandsOmanRomaniaSingaporeSlovakiaSouth AfricaSouth KoreaSwitzerlandTurkey (Türkiye)United Arab EmiratesUnited KingdomAustraliaBrazilIsraelJapanLebanonMexicoRussiaSaudi ArabiaSerbiaSpainChina
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Recent Changes (Last 90 Days)

LOWAug 26, 2026NCT05456191lastUpdatePostDate: changed
LOWAug 26, 2026NCT05456191lastUpdatePostDate: changed

Frequently asked questions about Asciminib

What is Asciminib used for?

Asciminib is an investigational small molecule being studied for chronic myelogenous leukemia, including Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, and for HER2+ metastatic breast cancer. It is also being studied in patients with renal impairment. Asciminib is not approved and remains in clinical development.

What does Asciminib target?

Asciminib is a kinase inhibitor, belonging to the -nib class of drugs. It is being studied in chronic myelogenous leukemia and other conditions where kinase signaling plays a role. The specific molecular target is not disclosed in available information.

Who makes Asciminib?

Asciminib is developed by Novartis AG, a pharmaceutical company listed on the New York Stock Exchange under the ticker NVS. Novartis is conducting clinical trials of Asciminib across multiple countries.

What phase is Asciminib in?

Asciminib has completed Phase 1, Phase 2, and Phase 3 trials. A Phase 3 study compared Asciminib to bosutinib in patients with chronic myelogenous leukemia previously treated with two or more tyrosine kinase inhibitors. Asciminib remains investigational and is not FDA approved.

What clinical trials is Asciminib in?

Asciminib has been studied in several completed trials. NCT02081378 was a Phase 1 study in chronic myelogenous leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia. NCT03106779 was a Phase 3 trial versus bosutinib. NCT03605277 examined pharmacokinetics in renal impairment, and NCT04795427 was a Phase 2 study in China.

Is Asciminib the same as Trastuzumab?

Asciminib is not the same as Trastuzumab. Asciminib is a small molecule kinase inhibitor, while Trastuzumab is a different type of agent. Asciminib is being studied for chronic myelogenous leukemia and HER2+ metastatic breast cancer, though the two drugs are distinct.