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

Summary

To confirm the efficacy of CPX-351 compared to 7+3 as first line therapy in elderly patients (60-75 yrs) with high risk (secondary) Acute Myeloid Leukemia. The primary efficacy endpoint will be overall survival.

Timeline

Start

2012-12-13

Primary completion

2015-12-31

Completion

2015-12-31

Outcome

Met primary endpoint

paper CPX-351 significantly improved median overall survival versus 7+3 (9.56 v 5.95 months; hazard ratio, 0.69; 95% CI, 0.52 to 0.90; one-sided P = .003). PMID 30024784 ↗

release “The NDA submission includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which met its primary endpoint” jazzpharma.com ↗

release “For the primary endpoint of overall survival, Vyxeos demonstrated an improvement that was superior to the 7+3 treatment regimen.” jazzpharma.com ↗

release “Data from the Phase 3 study, which met its primary endpoint, were presented at the American Society of Clinical Oncology Annual Meeting in June 2016” jazzpharma.com ↗

release “309 adult patients, aged 60-75, with newly diagnosed t-AML or AML-MRC. 1 The study met its primary endpoint as Vyxeos demonstrated a superior improvement” jazzpharma.com ↗

release “Phase 3 study of 309 adult patients, aged 60-75, with newly diagnosed t-AML or AML-MRC. The study met its primary endpoint” jazzpharma.com ↗

Publications

• Shimony S, Murdock HM, Keating J, Tsai HK, Sasi A, Gibson CJ, Faderl S, Wagner A, Dronamraju N, Lin TL, Prebet T, Cortes JE, Uy GL, Lancet JE, Reilly CR, Neuberg D, Stone RM, Lindsley RC. CPX-351 selectively benefits patients with AML and myelodysplasia-related mutations in the pivotal randomized trial. Blood Adv. 2026 Apr 28;10(8):2854-2864. doi: 10.1182/bloodadvances.2025019378.
• Guolo F, Fianchi L, Martelli MP, Lussana F, Grimaldi F, Pilo F, Rondoni M, Fili C, Minetto P, Capelli D, Chiusolo P, Breccia M, Mastaglio S, Bernardi M, Bocchia M, Fumagalli M, Galimberti S, Mancini V, Piccioni AL, Maurillo L, Fracchiolla NS, Palmieri R, Vetro C, Papayannidis C, Brunetti L, Sperotto A, Gigli F, Zappasodi P, Mule A, Patti C, Borlenghi E, Dargenio M, Lessi F, Cerrano M, Cilloni D, Isidori A, Lunghi M, Alati C, Gurrieri C, Riva C, Marconi G, Lotesoriere I, Gatani S, Scattolin AM, Caizzi M, Perrone S, Billio A, Gherlinzoni F, Mannelli F, Gottardi M, Cairoli R, Candoni A, Ferrara F, Pagano L, Lemoli RM, Venditti A, Todisco E. Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation. Am J Hematol. 2025 Dec;100(12):2293-2304. doi: 10.1002/ajh.70083. Epub 2025 Sep 24.
• Cortes JE, Lin TL, Asubonteng K, Faderl S, Lancet JE, Prebet T. Efficacy and safety of CPX-351 versus 7 + 3 chemotherapy by European LeukemiaNet 2017 risk subgroups in older adults with newly diagnosed, high-risk/secondary AML: post hoc analysis of a randomized, phase 3 trial. J Hematol Oncol. 2022 Oct 26;15(1):155. doi: 10.1186/s13045-022-01361-w.
• Cortes JE, Lin TL, Uy GL, Ryan RJ, Faderl S, Lancet JE. Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML. J Hematol Oncol. 2021 Jul 13;14(1):110. doi: 10.1186/s13045-021-01119-w.
• Lancet JE, Uy GL, Newell LF, Lin TL, Ritchie EK, Stuart RK, Strickland SA, Hogge D, Solomon SR, Bixby DL, Kolitz JE, Schiller GJ, Wieduwilt MJ, Ryan DH, Faderl S, Cortes JE. CPX-351 versus 7+3 cytarabine and daunorubicin chemotherapy in older adults with newly diagnosed high-risk or secondary acute myeloid leukaemia: 5-year results of a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2021 Jul;8(7):e481-e491. doi: 10.1016/S2352-3026(21)00134-4.
• Lin TL, Rizzieri DA, Ryan DH, Schiller GJ, Kolitz JE, Uy GL, Hogge DE, Solomon SR, Wieduwilt MJ, Ryan RJ, Faderl S, Cortes JE, Lancet JE. Older adults with newly diagnosed high-risk/secondary AML who achieved remission with CPX-351: phase 3 post hoc analyses. Blood Adv. 2021 Mar 23;5(6):1719-1728. doi: 10.1182/bloodadvances.2020003510.
• Kolitz JE, Strickland SA, Cortes JE, Hogge D, Lancet JE, Goldberg SL, Villa KF, Ryan RJ, Chiarella M, Louie AC, Ritchie EK, Stuart RK. Consolidation outcomes in CPX-351 versus cytarabine/daunorubicin-treated older patients with high-risk/secondary acute myeloid leukemia. Leuk Lymphoma. 2020 Mar;61(3):631-640. doi: 10.1080/10428194.2019.1688320. Epub 2019 Nov 25.
• Lancet JE, Uy GL, Cortes JE, Newell LF, Lin TL, Ritchie EK, Stuart RK, Strickland SA, Hogge D, Solomon SR, Stone RM, Bixby DL, Kolitz JE, Schiller GJ, Wieduwilt MJ, Ryan DH, Hoering A, Banerjee K, Chiarella M, Louie AC, Medeiros BC. CPX-351 (cytarabine and daunorubicin) Liposome for Injection Versus Conventional Cytarabine Plus Daunorubicin in Older Patients With Newly Diagnosed Secondary Acute Myeloid Leukemia. J Clin Oncol. 2018 Sep 10;36(26):2684-2692. doi: 10.1200/JCO.2017.77.6112. Epub 2018 Jul 19.
• Stein EM, Tallman MS. Emerging therapeutic drugs for AML. Blood. 2016 Jan 7;127(1):71-8. doi: 10.1182/blood-2015-07-604538. Epub 2015 Dec 10.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CPX-351	Other / unclassified	65 unknown	Intravenous
Subject	CPX-351	Other / unclassified	100 unknown	Intravenous
Comparator	Cytarabine	Small molecule	100 mg/m2	Intravenous
Comparator	Daunorubicin	Small molecule	60 mg/m2	Intravenous

Indications