CPX-351

Phase 3Small molecule

Jazz Pharmaceuticals

CPX-351 is one of 159 small molecule programs targeting Myelodysplastic Syndrome. It is currently in Phase 3.

Indications

Total Trials

Active Trials

158

Competitors

Pipeline

Status by Indication[3][1]

Indication

Myelodysplastic SyndromeActive, not recruiting

Acute Myeloid LeukemiaTerminated

Older PatientsActive, not recruiting

PreventionRecruiting

Newly DiagnosedActive, not recruiting

Myelodysplastic SyndromePhase 1

Active, not recruiting

Acute Myeloid LeukemiaPhase 3

Terminated

Older PatientsPhase 2

Active, not recruiting

PreventionPhase 2

Recruiting

Newly DiagnosedPhase 3

Active, not recruiting

Competition

Competitive Landscape

235 competitors in Acute Myeloid Leukemia

#DrugCompanyPhaseTrials

Idamycin

Pfizer

Phase 3

60 2

Xospata

Astellas

Phase 3

24 3

Vepesid

AAvyxa

Phase 3

52 4

Mylotarg

WWyeth-Lederle Vaccines

Phase 3

38 5

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

32 6

Neupogen

EEastern Cooperative Oncology Group

Phase 3

30 7

Nexavar

Bayer

Phase 3

12 8

Cellcept

Roche

Phase 3

11 9

Cytoxan

BBaxter Healthcare Corporation

Busulfex

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1/2Recruiting

Prevention

NCT04990102

Q1 2027

Phase 2Not yet recruiting

Acute Myeloid Leukemia

NCT05628623

Phase 1Active, not recruiting

Myelodysplastic Syndrome

NCT03572764

Q2 2027

Phase 3Active, not recruiting

Newly Diagnosed

NCT03897127

Phase 2Active, not recruiting

Acute Myeloid Leukemia

NCT04231851

Q1 2028

PreclinicalRecruiting

Acute Myeloid Leukemia

NCT07008638

Clinical

Trials by Indication[1]

Acute Myeloid Leukemia12 trials

Patients Receiving Induction With Liposomal Daunorubicin and Cytarabine (CPX-351) for Acute Myeloid Leukemia

Terminated·MMedical College of Wisconsin·Phase 4·NCT03988205·3 enrolled

A Phase 1b Master Trial to Investigate CPX-351 in Subjects With Previously Untreated Acute Myeloid Leukemia

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 1·NCT04075747·57 enrolled

A Study of CPX-351 (Vyxeos™) With Quizartinib for the Treatment of FLT3-ITD Mutation-Positive Acute Myeloid Leukemia

Terminated·SSCRI Development Innovations, LLC·Phase 2·NCT04209725·1 enrolled

Show 9 more trials

Phase I/II Trial of CPX-351 + Palbociclib in Patients With Acute Myeloid Leukemia

Completed·SSudipto Mukherjee, MD, PhD, MPH·Phase 1/2·NCT03844997·35 enrolled

Trial of CPX-351 in Adult Patients With First Relapse Acute Myeloid Leukemia (AML)

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 2·NCT00822094·126 enrolled

Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm

Completed·FFrench Innovative Leukemia Organisation·Phase 2·NCT04992949·42 enrolled

Phase III Study of CPX-351 Versus 7+3 in Patients 60-75 Years Old With Untreated High Risk (Secondary) Acute Myeloid Leukemia

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 3·NCT01696084·309 enrolled

Investigator Initiated Trial of CPX-351 for Untreated Acute Myeloid Leukemia

Completed·WWeill Medical College of Cornell University·Phase 2·NCT03335267·30 enrolled

Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia

Withdrawn·GGuenther Koehne·Phase 1/2·NCT04982354

Testing the Use of Combination Therapy in Patients With Persistent Low Level Acute Myeloid Leukemia (AML) Following Initial Treatment

Not yet recruiting·EEastern Cooperative Oncology Group·Phase 2·NCT05628623·184 enrolled

HM2024-29: Phase I/II Clinical Trial of Proteasome Inhibitor in Combination With CPX-351 for the Treatment of Newly-Diagnosed TP53-mutated Acute Myeloid Leukemia (AML)

Recruiting·MMasonic Cancer Center, University of Minnesota·Preclinical·NCT07008638·32 enrolled

Phase II Study of the Combination of CPX-351 and Glasdegib in Previously Untreated Patients With Acute Myelogenous Leukemia With MDS Related Changes or Therapy-related Acute Myeloid Leukemia

Active, not recruiting·UUniversity of California, Irvine·Phase 2·NCT04231851·30 enrolled

Newly Diagnosed4 trials

Trial of CPX-351 in Newly Diagnosed Elderly AML Patients

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 2·NCT00788892·126 enrolled

Magrolimab Plus Intensive Chemotherapy in Newly Diagnosed AML or HR-MDS

Withdrawn·UUwe Platzbecker·Phase 2·NCT05829434

Study of Standard Intensive Chemotherapy Versus Intensive Chemotherapy With CPX-351 in Adult Patients With Newly Diagnosed AML and Intermediate- or Adverse Genetics

Active, not recruiting·UUniversity of Ulm·Phase 3·NCT03897127·882 enrolled

Show 1 more trial

A Phase II, Multicentre, Open Label Clinical Trial to Assess the Efficacy and Toxicity of Induction and Consolidation With CPX-351 for Patients Aged 60 to 75 Years With Secondary or High-risk Acute Myeloid Leukemia

Completed·PPETHEMA Foundation·Phase 2·NCT04230239·59 enrolled

Acute Leukemia2 trials

Pharmacokinetic and Pharmacodynamic Assessment of Treatment With CPX-351 (Cytarabine: Daunorubicin) Liposome for Injection in Acute Leukemias and MDS Patients With Moderate Hepatic Impairment

Withdrawn· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 2·NCT02269579

An Open Label Phase II Pharmacokinetic and Pharmacodynamic Assessment of the Potential for QTc Prolongation Following First Induction Treatment With CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in Acute Leukemias and MDS Patients

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 2·NCT02238925·26 enrolled

Myelodysplastic Syndrome2 trials

CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome

Active, not recruiting·WWashington University School of Medicine·Phase 1·NCT03572764·20 enrolled

CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome

Completed·WWeill Medical College of Cornell University·Phase 1·NCT03393611·14 enrolled

Leukemia2 trials

Safety Study of CPX-351 in Children With Relapsed Leukemia or Lymphoma

Completed·—·Phase 1·NCT01943682·27 enrolled

A Novel Sequential Treatment of Salvage and Reduced Intensity Conditioning (RIC) Chemotherapy for Allogeneic Stem-Cell Transplantation (SCT) for Primary Refractory and Relapsed Acute Myelogenous Leukemia (AML)

Completed·WWeill Medical College of Cornell University·Phase 1·NCT00875693·47 enrolled

13 more indications

MDS2 trials

CPX-351 Therapy for MDS After Hypomethylating Agent Failure

Terminated·—·Phase 2·NCT03957876·4 enrolled

Primary Comparison of Liposomal Anthracycline Based Treatment Versus Conventional Care Strategies Before Allogeneic Stem Cell Transplantation in Patients With Higher Risk MDS and Oligoblastic AML

Completed·GGWT-TUD GmbH·Phase 2·NCT04061239·150 enrolled

Effectiveness1 trial

CPX-351 Real-World Effectiveness and Safety Study

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals··NCT05169307·147 enrolled

Treatment1 trial

A Trial of CPX-351 Lower Intensity Therapy (LIT) Plus Venetoclax as First Line Treatment for Subjects With AML

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 1·NCT04038437·35 enrolled

AML1 trial

CPX-351+GO in Subjects 55 Years Old, or Older, With AML

Terminated·WWeill Medical College of Cornell University·Phase 1·NCT03878927·11 enrolled

Myelodysplastic Syndromes1 trial

CPX-351 in Patients Treated for Higher-risk Myelodysplastic Syndromes Experiencing Hypomethylating Agent Failure.

Withdrawn·YYale University·Phase 1/2·NCT04109690

Prevention1 trial

Phase IB/II of CPX-351 for Relapse Prevention in AML

Recruiting·GGeorgetown University·Phase 1/2·NCT04990102·24 enrolled

Renal Impairment1 trial

A Trial to Evaluate the Potential Impact of Renal Impairment on the Pharmacokinetics and Safety of CPX-351

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 1·NCT03555955·21 enrolled

Observational Study1 trial

A Post-Marketing Observational Study of VYXEOS™

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals··NCT03526926·52 enrolled

Chemotherapy1 trial

Trial to Test the Effects of Adding 1 of 2 New Treatment Agents to Commonly Used Chemotherapy Combinations

Unknown status·—·Phase 2/3·NCT02272478·1,600 enrolled

Older Patients1 trial

CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS

Active, not recruiting·—·Phase 2·NCT04668885·13 enrolled

Secondary AML1 trial

EAP of CPX-351 (VYXEOS) for Patients 60-75 Years of Age With Secondary AML

Approved for marketing· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals··NCT02533115

Acute Lymphoblastic Leukemia1 trial

Vyxeos(CPX-351) in Adults w R/R Acute Lymphoblastic Leukemia

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT03575325·11 enrolled

Myeloid Neoplasm1 trial

A Prospective, Single Site, Single-Arm Pilot Study of CPX-351 (VYXEOS) in Individuals < 22 Years With Secondary Myeloid Neoplasms

Active, not recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT05656248·25 enrolled

Sources

References

[1]
ClinicalTrials.gov — 36 trial records for CPX-351. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — CPX-351 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for CPX-351. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Jazz Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Jazz Pharmaceuticals

CPX-351

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q2 2027

Q1 2028

Trials by Indication[1]

Acute Myeloid Leukemia12 trials

Newly Diagnosed4 trials

Acute Leukemia2 trials

Myelodysplastic Syndrome2 trials

Leukemia2 trials

MDS2 trials

Effectiveness1 trial

Treatment1 trial

AML1 trial

Myelodysplastic Syndromes1 trial

Prevention1 trial

Renal Impairment1 trial

Observational Study1 trial

Chemotherapy1 trial

Older Patients1 trial

Secondary AML1 trial

Acute Lymphoblastic Leukemia1 trial

Myeloid Neoplasm1 trial

References

Drug Info

Quick Facts

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