Ionis’ olezarsen shows new promise with strong Phase III results

US biotech Ionis Pharmaceuticals (Nasdaq: IONS) has reported positive top-line data from the Phase III ESSENCE trial of its antisense drug olezarsen in patients with moderate hypertriglyceridemia who are at risk of atherosclerotic cardiovascular disease.

The study met its primary endpoint, showing a placebo-adjusted reduction in triglyceride levels of 61% and 58% at six months with monthly doses of 80mg and 50mg, respectively. The drug also met all key secondary endpoints, with most participants reaching triglyceride levels under 150mg/dL - falling into the normal range. Ionis said the treatment was well tolerated and maintained a safety profile consistent with earlier studies.

Olezarsen is designed to inhibit the expression of the APOC3 gene, reducing production of apolipoprotein C-III, a protein that interferes with the metabolism of triglycerides. By promoting the breakdown of triglyceride-rich lipoproteins, the drug delivers significant reductions in plasma triglyceride levels.