Drug intelligence / Profile preview

AZD9829

Development stage
Phase 2
Lead developer
AstraZeneca
Modality
Small Molecules, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, Monoclonal Antibodies → Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

AZD9829 is a first-in-class antibody-drug conjugate (ADC) developed by AstraZeneca for the treatment of hematological malignancies, particularly relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes. The drug consists of a monoclonal antibody targeting the interleukin-3 receptor subunit alpha (CD123), which is overexpressed on certain cancer cells, conjugated to a topoisomerase I inhibitor payload. Upon binding to CD123-expressing cells, AZD9829 delivers the cytotoxic topoisomerase I inhibitor directly into malignant cells, leading to DNA damage and apoptosis. Preclinical studies have demonstrated its efficacy in vitro and in vivo against CD123-positive AML cell lines and patient-derived xenograft models[1][2][5][6][8].

Other names
anti-CD123/TOP1i antibody-drug conjugate
02

Targets

TOP1 (DNA Topoisomerase I)IL3RA (Interleukin 3 Receptor)

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