ION224 is an investigational ligand-conjugated antisense (LICA) medicine designed to reduce the production of diacylglycerol acyltransferase 2 (DGAT2) to treat patients with nonalcoholic steatohepatitis (NASH). DGAT2 is an enzyme that catalyzes the final step in triglyceride synthesis in the liver. Reducing the production of DGAT2 should therefore decrease triglyceride synthesis in the liver. In animal studies, antisense inhibition of DGAT2 significantly improved liver steatosis, lowered blood lipid levels and reversed diet-induced insulin resistance.
About Non-Alcoholic Steatohepatitis (NASH)
NASH is a common liver disease characterized by liver steatosis, inflammation and scarring. NASH is associated with an increased risk of cardiovascular disease and can lead to more severe diseases such as cirrhosis and liver failure.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.