Akcea generates positive IONIS-ANGPTL3-LRx trial results
"These data provide solid support for the therapeutic potential of ANGPTL3 to reduce multiple lipid risk factors in patients with cardiovascular disease and to affect liver fat accumulation in patients with NASH," University of California, San Diego director of vascular medicine Sotirios Tsimikas said. "Pre-clinical studies in animal models of atherosclerosis and obesity demonstrate that lowering ANGPTL3 levels with antisense oligonucleotides significantly reduces progression of atherosclerosis and liver fat accumulation. The potential of ANGPTL3 reduction to improve cardiovascular risk is also supported by genetic studies in humans with both heterozygous and homozygous loss-of-function mutations in their ANGPTL3 gene showing that individuals with lower or absent plasma levels of ANGPTL3 protein exhibit low plasma levels of triglycerides and LDL cholesterol, which are both risk factors for cardiovascular disease."
The patients who took part in the study all had increased triglycerides, but all of them reached a large and noteworthy decrease in angiopoietin-like 3 (ANGPTL3) up to 83 percent, triglycerides up to 66 percent and LDL-cholesterol up to 35 percent.
The results of the trial were presented at the American Heart Association Scientific Sessions.