WAYLIVRA is designed to reduce the production of apolipoprotein C-III (apoC-III
), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides.
(NASDAQ: ARWR) has dosed the first subjects in a Phase 1 clinical study of ARO-APOC3, an RNAi-based investigational medicine targeting Apolipoprotein C-III (apoC-III
) being developed for the treatment of hypertriglyceridemia, the company said.
antisense oligonucleotides: a new option for the treatment of hypertriglyceridemia.
Volanesorsen, developed using Ionis' proprietary antisense technology, is designed to reduce the production of ApoC-III
, a protein that is produced in the liver and plays a central role in the regulation of plasma triglycerides.
Results showed a 66% drop in triglycerides, 68% in apoliprotein C-III (ApoC-III
), 35% in LDL cholesterol, and 36% in total cholesterol.
Apolipoprotein C-III (apoC-III
) is secreted with VLDL and becomes distributed among circulating lipoproteins  conferring harmful properties resulting in ACVD [3, 12].
The regulatory role of apoE and apoC-III
lipoproteins in the apoB metabolism was studied in a recent work on healthy subjects and patients with hypertriglyceridemia .
It was recently shown that apolipoprotein C-III (apoC-III
), which is highly correlative to plasma TG levels, inhibits the hepatic clearance of TRLs by both LRP1 and LDLR .
It is primarily due to increase in plasma ApoC-III
levels.8 There is also down regulation of lipoprotein lipase, hepatic lipase and the VLDL receptor and upregulation of hepatic acyl-CoA cholesterol acyltransferase (ACAT).2
The gene in question codes for the apoC-III
For example, in my lab we're looking at ApoC-III
, a small inflammatory protein on the surface of some HDL particles.