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Asciminib single agent

Phase 2

Chronic Myelogenous Leukemia | Small molecule | Oncology |Novartis AG|Last Updated: Sep 2, 2026

Target and mechanism

Molecular targetABL1, BCR
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment50

FDA Designations

No designations recorded

Clinical trial landscape

Asciminib single agent · 1 trial · 2 indications

Phase 2 1
NCT07354074Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CPChronic Myelogenous Leukemia
RECRUITING50 Analytics
PHASE2RECRUITING
Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP
Chronic Myelogenous LeukemiaUnlock trial analytics

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 to 18 Years
SexALL
Healthy VolunteersNo
Study Sites30

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:United StatesAustraliaBrazilCanadaChinaFranceGermanyItalyJapanNetherlandsPolandSouth KoreaSpain
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Recent Changes (Last 90 Days)

LOWSep 2, 2026NCT07354074lastUpdatePostDate: changed
LOWSep 2, 2026NCT07354074lastUpdatePostDate: changed
LOWSep 2, 2026NCT07354074lastUpdatePostDate: changed
LOWAug 20, 2026NCT07354074lastUpdatePostDate: changed
LOWAug 20, 2026NCT07354074lastUpdatePostDate: changed
LOWJul 24, 2026NCT07354074lastUpdatePostDate: changed
LOWJul 24, 2026NCT07354074lastUpdatePostDate: changed
LOWJul 9, 2026NCT07354074lastUpdatePostDate: changed
LOWJul 9, 2026NCT07354074lastUpdatePostDate: changed

Frequently asked questions about Asciminib single agent

What is Asciminib single agent used for?

Asciminib single agent is used for chronic myelogenous leukemia (CML), specifically in pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). It is an investigational small molecule being studied as a single agent for this indication.

What does Asciminib single agent target?

Asciminib single agent is a kinase inhibitor, belonging to the -nib class of targeted cancer therapies. It works by inhibiting kinase activity, which plays a role in the growth and survival of cancer cells in chronic myelogenous leukemia.

Who makes Asciminib single agent?

Asciminib single agent 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 drug's efficacy and safety in pediatric patients with Ph+ CML-CP.

What phase is Asciminib single agent in?

Asciminib single agent is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied in clinical trials to determine its safety and effectiveness.

What clinical trials is Asciminib single agent in?

Asciminib single agent is being studied in one active Phase 2 clinical trial with the identifier NCT07354074. This trial is recruiting participants to evaluate the efficacy and safety of asciminib in pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase.

Is Asciminib single agent the same as asciminib?

Asciminib single agent refers to the drug asciminib when used as a monotherapy, meaning it is administered alone rather than in combination with other treatments. In the context of the clinical trial, it is being studied as a single agent for pediatric patients with Ph+ CML-CP.