Continued study of investigational lipid-lowering agent ETC-1002
The U.S. Food and Drug Administration (FDA) has removed the peroxisome proliferator-activated receptor (PPAR) partial clinical hold on ETC-1002. A partial clinical hold is a delay or suspension of part of the clinical work requested for the investigational new drug application.
ETC-1002 is a unique, first-in-class, orally available, once-daily small molecule designed to lower LDL-cholesterol levels and avoid the side effects associated with therapies currently available for lowering LDL-cholesterol. ETC-1002 is being developed primarily for patients with hypercholesterolemia and a history of statin intolerance.
As explained on the website of Esperion Therapeutics, ETC-1002 has a unique dual mechanism of action that has the potential to regulate both lipid and carbohydrate metabolism. ETC-1002 appears to work by inhibiting ATP citrate lyase (ACL), a key enzyme in the cholesterol biosynthetic pathway, and activating a complementary enzyme, 5′-adenosine monophosphate-activated protein kinase (AMPK). Both enzymes are known to play significant roles in the synthesis of cholesterol and glucose in the liver. By inhibiting cholesterol synthesis in the liver, ETC-1002 causes the liver to take up LDL particles from the blood, which reduces LDL-C levels.
To date, ETC-1002 has been studied in eight completed clinical trials.
The action by the FDA will now allow Esperion to conduct clinical trials exceeding six months in duration. A phase 3 clinical program for ETC-1002 will be initiated later this year.