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Liposome-encapsulated Daunorubicin-Cytarabine

Phase 2

Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Dec 23, 2025

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment21
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04269213CPX-351 for the Treatment of Secondary Acute Myeloid Leukemia in Patients Younger Than 60 Years OldPHASE2 ACTIVE NOT_RECRUITING 21Jul 29, 2021Jan 29, 2027Dec 23, 20254 United States
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Study Endpoints
Primary Endpoints
Complete response rate (morphological complete remission [CR] and incomplete blood count recovery [CRi])
At day 45

Defined by the International Working Group Criteria. Will be summarized using frequencies and relative frequencies.

Secondary Endpoints
CR + CRi duration
Time from CR or CRi until relapse or last follow-up, assessed up to 5 years
Event free survival
Time from treating until disease progression/relapse, death due to disease, or last follow-up, assessed up to 5 years
Overall survival
Time from treatment until death due to any cause or last follow-up, assessed up to 5 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment (CPX-351)EXPERIMENTALINDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.
Interventions
NameTypeDescription
Liposome-encapsulated Daunorubicin-CytarabineDRUGGiven IV
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Eligibility Criteria
Age Range18 Years — 59 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Newly diagnosed: * Therapy-related acute myeloid leukemia (AML) * AML with antecedent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) * AML with MDS-related changes (as per World Health Organization \[WHO\]) * Have an Eastern Cooperative Oncolog...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04269213studyFirstPostDate: changed