Akcea Therapeutics (NASDAQ:AKCA) reported positive topline results from its Phase 2 study of AKCEA-ANGPTL3-LRx in patients with hypertriglyceridemia, Type 2 diabetes (T2D) and non-alcoholic fatty liver disease (NAFLD).
AKCEA-ANGPTL3-LRx is an antisense oligonucleotide therapy designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, which is a key regulator of triglycerides, cholesterol, glucose and energy metabolism.
The study assessed 105 patients with hypertriglyceridemia, T2D and NAFLD who were administered AKCEA-ANGPTL3-LRx or placebo via subcutaneous injection for six months. Doses were administered weekly or monthly, with total monthly doses ranging from 40 mg to 80 mg. The trial met its primary endpoint of significant fasting triglyceride reduction compared with placebo, which was observed at all dose levels.
“Results from the Phase 2 study showed that antisense-mediated reduction of ANGPTL3 has the potential to address unmet needs in patients with cardiovascular diseases,” Louis O’Dea, Akcea’s CMO, said in a statement.
“This Phase 2 study further validates the advantages of the ligand conjugated antisense technology platform for large indications like cardiovascular disease,” he added.
