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Ponatinib

Phase 2

Philadelphia Positive | Small molecule | Oncology |Incyte Corporation|Last Updated: Jul 23, 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

UNCONTROLLED
Total Trials1
Total Enrollment44

FDA Designations

No designations recorded

Clinical trial landscape

Ponatinib · 2 trials · 11 indications

Phase 2 1Phase 1 1
NCT01641107Phase II Front-line Ponatinib in Adult Philadelphia+/BCR-ABL+ Acute Lymphoblastic Leukemia.Philadelphia Positive
COMPLETED44 Analytics
PHASE2COMPLETED
Phase II Front-line Ponatinib in Adult Philadelphia+/BCR-ABL+ Acute Lymphoblastic Leukemia.
Philadelphia PositiveUnlock trial analytics

Study Endpoints

Primary Endpoints

Proportion of patients who are in Complete Hematological Response (CHR).
At 6 months from study entry.

The primary endpoint is the proportion of patients who are in CHR at 6 months, calculated on the total number of patients who have been enroled and have received at least one dose of the first drug (prednisone).

Phase 1: Number of dose-limiting toxicities
28 days

Defined as the occurrence of any protocol-defined toxicities occurring after dosing and up to and including Day 28, except those toxicities with a clear alternative explanation.

Phase 2: Efficacy of ponatinib assessed by major cytogenetic response (MCyR) in participants with chronic-phase chronic myeloid leukemia (CP-CML)
12 months

Defined as complete cytogenetic response (CCyR) or partial cytogenetic response (PCyR) as assessed by conventional cytogenetics or fluorescence in situ hybridization (FISH).

Phase 2: Efficacy of ponatinib assessed by major hematologic response (MaHR) or major molecular response (MMR) in participants with BCR-ABL-positive leukemias
3 months

Assessed by polymerase chain reaction (PCR).

Phase 2: Efficacy of ponatinib assessed by complete response (CR) in participants with leukemias other than BCR-ABL-positive leukemias to determine the efficacy of ponatinib
6 months
Phase 2: Efficacy of ponatinib assessed by incomplete complete response (iCR) in participants with leukemias other than BCR-ABL-positive leukemias
6 months

Assessed by conventional cytogenetics, FISH, or PCR.

Phase 2: Efficacy of ponatinib assessed by CR in participants with lymphoma
6 months

According to Lugano criteria based on computed tomography (CT) or magnetic resonance imaging (MRI) (or positron emission tomography \[PET\]).

Phase 2: Efficacy of ponatinib assessed by overall response rate in participants with solid tumors
6 months

Defined as the percentage of participants having CR or PR, as determined by investigator assessment of radiographic disease per tumors per RANO for central nervous system (CNS) tumors or Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) for other solid tumors based on CT or MRI (or PET).

Secondary Endpoints

The rate of Complete Hematological Response (CHR).
At 6, 12, 24, 36 and 48 weeks from study entry.
The rate of complete Cytogenetic Response (CgR).
At 6, 12, 24, 36 and 48 weeks from study entry.
Duration of Complete Cytogenetic Response (CCgR).
After four years from study entry.
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PonatinibEXPERIMENTAL -

Interventions

NameTypeDescription
PonatinibDRUGTreatment: Patients will receive daily oral administration of Ponatinib at a dose of 45 mg/day for 6 weeks (defined as one course) for 8 courses, same dose and schedule, for a total of 48 weeks. Each patient will be followed for the subsequent 24 months, every 3 month, providing survival information and monitoring serious adverse event. Each patient should be treated for a minimum of 6 weeks. Patients must be discontinued from the trial in the event of myocardial infarction or stroke, or for development or progression of arterial disease necessitating revascularization. Once a complete hematologic response has been achieved, with a platelet count ≥ 50x109/L, patients can be treated with aspirin and/or a statin as clinically indicated, at investigator discretion.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites42

Inclusion Criteria: 1. To be classified as having Ph+ ALL, patients must have \>20% blasts in bone marrow at the time of diagnosis and no prior history of CML. 2. Patients with previously untreated Ph+ and/or BCR/ABL + ALL: * age ≥ 60 years old or * age ≥ 18 years old, but unfit for program ...

Countries:ItalyBelgiumFranceNetherlandsSpainSwedenUnited Kingdom
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Recent Changes (Last 90 Days)

LOWJul 24, 2026NCT03934372lastUpdatePostDate: changed
LOWJul 24, 2026NCT03934372lastUpdatePostDate: changed
LOWJul 8, 2026NCT03934372lastUpdatePostDate: changed
LOWJul 8, 2026NCT03934372lastUpdatePostDate: changed

Frequently asked questions about Ponatinib

What is Ponatinib used for?

Ponatinib is an investigational small molecule being studied for use in Philadelphia Positive Acute Myeloid Leukemia. It is also being evaluated in clinical trials for other leukemias, lymphomas, and solid tumors. Ponatinib is in Phase 1 development and is not yet approved by the FDA.

What does Ponatinib target?

Ponatinib is a kinase inhibitor, belonging to the -tinib class of drugs. It is being studied for its activity against BCR-ABL positive leukemias, including Philadelphia Positive Acute Myeloid Leukemia. The drug is designed to inhibit certain kinases involved in cancer cell growth.

Who makes Ponatinib?

Ponatinib is being developed by Incyte Corporation, a biopharmaceutical company traded on the NASDAQ under the ticker symbol INCY. Incyte is conducting clinical trials to evaluate the safety and efficacy of Ponatinib in various cancer indications.

What phase is Ponatinib in?

Ponatinib is currently in Phase 1 clinical development. It is being studied in a Phase 1 trial for pediatric patients with recurrent or refractory leukemias, lymphomas, or solid tumors. Ponatinib is an investigational drug and has not received FDA approval.

What clinical trials is Ponatinib in?

Ponatinib is being studied in a Phase 1 clinical trial with the identifier NCT03934372. This trial is recruiting pediatric patients with acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, lymphoma, and solid tumors. The trial is enrolling participants in multiple European countries.

Is Ponatinib the same as Iclusig?

Ponatinib is also known by the brand name Iclusig. In clinical trials, it is referred to as ponatinib, and it is being investigated for use in Philadelphia chromosome positive leukemias. The drug is a kinase inhibitor developed by Incyte Corporation.