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Chemistry: Generation 2+ LICA

Olezarsen, formerly known as IONIS-APOCIII-LRx and AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to inhibit the production of apoC-III in the liver. ApoC-III is a protein that regulates triglyceride metabolism in the blood by inhibiting lipoprotein lipase that breakdown triglycerides and by preventing clearance of triglyceride-rich lipoproteins. ApoC-III is an independent risk factor for both pancreatitis and cardiovascular disease.

About Severe Hypertriglyceridemia (SHTG)

Severe Hypertriglyceridemia (triglyceride levels ≥  500 mg/dL) can be caused by diet-derived chylomicronemia and excessive liver triglyceride production, often superimposed on genetic predisposition. Its primary manifestation is acute pancreatitis, particularly if triglyceride levels are > 880 mg/dL. A subset of patients with triglyceride levels 500-880 are also at risk for cardiovascular disease. Lowering of plasma triglycerides is desired. Treatment regimens include nutritional intervention, the use of antihyperlipidemic drugs, and therapeutic apheresis. SHTG is a common condition that affects millions of people around the world, including more than 3 million in the U.S.