Drug intelligence / Profile preview

gemcabene + statin

Development stage
Unknown
Lead developer
Gemphire Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

Gemcabene (CI-1027) in combination with statins is an oral lipid-lowering therapy developed by Gemphire Therapeutics (now NeuroBo Pharmaceuticals). Gemcabene is a small molecule dicarboxylic acid derivative that acts through a dual mechanism: it inhibits acetyl-CoA carboxylase (ACC) and reduces the hepatic expression of apolipoprotein C-III (apoC-III) mRNA. These actions collectively decrease the hepatic production of cholesterol and triglycerides while enhancing the clearance of very low-density lipoproteins (VLDL). When administered as an add-on to stable statin therapy, gemcabene has demonstrated significant, dose-dependent reductions in low-density lipoprotein cholesterol (LDL-C) and high-sensitivity C-reactive protein (hsCRP) in clinical trials. The combination is primarily targeted at patients with hypercholesterolemia, including familial forms, who are unable to reach target lipid levels with statins alone, and it has also been explored for potential benefits in non-alcoholic steatohepatitis (NASH).

Other names
gemcabene calcium + statinCI-1027 + statingemcabene + HMG-CoA reductase inhibitor
02

Targets

HMGCR (3-hydroxy-3-methylglutaryl-coenzyme A reductase)APOC3 (Apolipoprotein C-III)ACC (Acetyl-CoA Carboxylase 1)

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