Final safety and efficacy results from the CPX-351 early access program for older patients with high-risk or secondary acute myeloid leukemia

Abstract

CPX-351, a dual-drug liposomal encapsulation of cytarabine and daunorubicin at a synergistic 5:1 molar drug ratio, achieved superior efficacy compared with conventional chemotherapy in older adults with newly diagnosed, high-risk/secondary acute myeloid leukemia (AML) in phase 2 and 3 studies. Prior to CPX-351 commercialization, an expanded access program (EAP) provided CPX-351 access for this population in the United States. In this phase 4, single-arm, open-label study (NCT02533115), 52 patients were treated with CPX-351 for 1–2 induction cycles and ≤4 consolidation cycles. The primary endpoint was safety. The most common serious adverse events were febrile neutropenia (19%), pneumonia (10%), and infection (8%). The 30- and 60-d mortality rates were 0% and 6%, respectively. Remission was achieved by 44% of patients; 90% of patients were alive at study completion. Overall, these results support outcomes from prior studies and the use of CPX-351 in older adults with newly diagnosed, high-risk/secondary AML.

Keywords:

• Acute myeloid leukemia
• CPX-351
• expanded access program

Acknowledgments

The authors would like to thank Emaryn Mancino of Jazz Pharmaceuticals for her contributions to the study. Medical writing and editorial assistance were provided by William Perlman, PhD, and Diana Avery, PhD, of SciFluent Communications, Inc., and were financially supported by Jazz Pharmaceuticals.

Disclosure statement

G.J. Roboz has received research funding from Cellectis and consulting fees from AbbVie, Actinium, Agios, Aphivena Therapeutics, Argenx, Array Biopharma, Astellas, Astex, AstraZeneca, Bayer, Celgene, Celltrion, Daiichi Sankyo, Eisai, Epizyme, Helsinn, Janssen, Jasper Therapeutics, Jazz Pharmaceuticals, MEI Pharma, Novartis, Orsenix, Otsuka, Pfizer, Roche/Genentech, Sandoz, Takeda, and Trovagene. S.E. Rubenstein reports speaker’s bureau participation for Alexion and AstraZeneca. G.J. Schiller has received research funding from Jazz Pharmaceuticals and Pharmacyclics. Q. An is an employee of and holds stock ownership/options in Jazz Pharmaceuticals. M. Chiarella and A.C. Louie are former employees of and hold stock ownership/options in Jazz Pharmaceuticals; A.C. Louie additionally holds patents and royalties with Jazz Pharmaceuticals. M.L. Larson, S.R. Solomon, and T.L. Lin have no conflicts of interest to disclose.

Data availability

All relevant data are provided within the manuscript and supporting files.

Additional information

Funding

This study was sponsored by Celator Pharmaceuticals, a subsidiary of Jazz Pharmaceuticals.

This study was sponsored by Celator Pharmaceuticals, a subsidiary of Jazz Pharmaceuticals.