Drug intelligence / Profile preview

AZD0780 + ezetimibe

Development stage
Phase 1
Lead developer
AstraZeneca
Modality
Small Molecules
Administration
Oral
01

Overview

AZD0780 + ezetimibe is an oral combination of two small molecule drugs investigated for the treatment of dyslipidemia and elevated low-density lipoprotein cholesterol (LDL-C). AZD0780 is a novel, oral small molecule inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9), developed by AstraZeneca to lower LDL-C by preventing PCSK9-mediated degradation of LDL receptors, thereby enhancing LDL clearance from plasma. Ezetimibe is a cholesterol absorption inhibitor that acts at the intestinal brush border by inhibiting Niemann-Pick C1-Like 1 (NPC1L1), reducing intestinal cholesterol uptake. The combination is being studied in phase 1 trials for the safety, pharmacokinetics, tolerability, and efficacy in patients with elevated LDL-C who may not achieve goals with statins or current therapies. The goal is to provide an alternative or additive, oral cholesterol-lowering strategy for patients not reaching LDL-C targets with standard therapy[1][3][4][6].

02

Targets

NPC1L1 (Niemann-pick C1-Like 1 transporter)PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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