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Ponatinib

Phase 2

Chronic Myeloid Leukemia, Chronic Phase | Small molecule | Oncology |Incyte Corporation|Last Updated: Dec 31, 2024

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment80
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04160546Study to Evaluate the Reinduction and Second Stop of TKI with Ponatinib in CML in Molecular Response (ResToP)PHASE2 ACTIVE NOT_RECRUITING 80Jan 17, 2020Dec 1, 2025Dec 31, 202413 Spain
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Study Endpoints
Primary Endpoints
Proportion of patients with a maintained MMR within 52 weeks following ponatinib Treatment-Free Remission (TFR)
52 weeks

This variable is defined as the number of patients who have a maintained MMR and have not restarted TKI therapy in the first 52 weeks after starting ponatinib TFR phase divided by the number of patients who entered ponatinib TFR phase.

Secondary Endpoints
Evaluate the toxicity and safety profile of 15 mg/24h dose treatment of ponatinib combined with ASA.
104 weeks
Evaluate thromboembolic events for study period.
104 weeks
Evaluate hemorrhagic events for study period.
104 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Ponatinib plus ASA treatmentEXPERIMENTALPatients will be treated with ponatinib 15 mg/day plus 100 mg/day ASA for 104 weeks. After that, ponatinib and ASA will be stopped.
Interventions
NameTypeDescription
Ponatinib 15 MGDRUGPatients will receive ponatinib 15 mg/day for 104 weeks orally. Ponatinib will be self-administered by the patient on a daily schedule. Acetyl salicylic acid (ASA) (100 mg) will be used such auxiliary medicinal product in order to prevent vascular occlusive events related with ponatinib.
Acetylsalicylic acid 100 MGDRUGPatients will receive acetylsalicylic acid 100 mg/day for 104 weeks orally.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: 1. Male or female patients ≥ 18 years of age. 2. ECOG Performance Status of 0, 1, or 2. 3. Patient with diagnosis of BCR-ABL positive and Ph+ CML-Chronic Phase. 4. Patients who failed the first attempt of TKI discontinuation and after TKI reintroduction they achieve again MR4 an...

Countries:Spain
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04160546studyFirstPostDate: changed