Drug intelligence / Profile preview

CER-001

Development stage
Phase 3
Lead developer
ABIONYX Pharma
Modality
Recombinant Proteins and Enzymes, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

CER-001 is a recombinant high-density lipoprotein (HDL) mimetic designed to replicate the structure and function of nascent (pre-beta) HDL. It consists of human recombinant apolipoprotein A-I (ApoA1), the main protein component of natural HDL, combined with two phospholipids—sphingomyelin and dipalmitoyl-phosphatidylglycerol. The drug acts by stimulating cholesterol efflux from macrophages and promoting reverse lipid transport (reverse cholesterol transport), thereby capturing excess cholesterol and facilitating its elimination from the body. This mechanism is intended to address conditions characterized by low HDL or dysfunctional lipid metabolism. CER-001 has orphan drug status for lecithin-cholesterol acyltransferase deficiency and hypoalphalipoproteinaemia, and it has been investigated in clinical trials for acute coronary syndrome, acute kidney injury, atherosclerosis, uveitis, as well as being considered for hyperinflammatory states such as those seen in COVID-19[1][2][3][4][6].

Brand names
Cerenis HDL
Other names
Recombinant human apolipoprotein A-I/phospholipid complexRecombinant Human Apolipoprotein A-IRecombinant Human Apolipoprotein A-I/Phospholipids complex
02

Targets

ABCA1 (ATP-binding cassette transporter A1)LCAT (Lecithin-cholesterol acyltransferase)APOA1 (Apolipoprotein A-I)

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