Drug intelligence / Profile preview

gemtuzumab ozogamicin + daunorubicin + cytarabine + CPX-351

Development stage
Preclinical
Lead developer
Pfizer
Modality
Small Molecules, Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

This is a combination therapy that includes gemtuzumab ozogamicin (a monoclonal antibody-drug conjugate targeting CD33), along with daunorubicin and cytarabine (standard chemotherapy agents), and CPX-351 (a liposomal formulation of daunorubicin and cytarabine at a fixed molar ratio of 1:5). The combination is being investigated for the treatment of acute myeloid leukemia (AML). Gemtuzumab ozogamicin targets the CD33 protein on the surface of leukemia cells. The antibody component (gemtuzumab) binds to CD33, allowing the cytotoxic component (ozogamicin) to enter and kill the cells. Daunorubicin is antimitotic and cytotoxic, forming complexes with DNA, inhibiting topoisomerase II activity, inhibiting DNA polymerase activity, affecting gene expression regulation, and producing DNA-damaging free radicals. Cytarabine affects cells during the synthesis phase of cell division by inhibiting DNA polymerase. CPX-351 is a liposomal formulation of daunorubicin and cytarabine at a fixed 1:5 molar ratio, which has shown synergistic effects in killing leukemia cells.

02

Targets

TOP2A (DNA topoisomerase II)DNA polymerase familyCD33 (Myeloid cell surface antigen CD33)

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