Ionis’ metabolic disorder drug lowers high levels of a type of fat in study

Ionis’ metabolic disorder drug lowers high levels of a type of fat in study

Sept 26 (Reuters) – Ionis Pharmaceuticals (IONS.O) said on Tuesday its experimental drug met the main goal of reducing abnormally high levels of the most common type of body fat in a late-stage trial in patients with a metabolic disorder.

The rare genetic disorder, known as familial chylomicronemia syndrome (FCS), prevents the body from breaking down fats and is characterized by extremely high levels of triglyceride.

FCS is caused by the deficiency or impaired function of lipoprotein lipase enzyme, responsible for breaking down triglycerides, which when accumulated in the blood leads to inflammation of the pancreas.

The National Pancreas Foundation estimates FCS to occur in 1 in 1-2 million people globally.

The drug candidate, olezarsen, showed statistically significant reduction in levels of triglyceride compared to placebo, as well as showed a 100% reduction in inflammation of the pancreas, Ionis said.

The company said it plans to file a marketing application with the U.S. Food and Drug Administration for olezarsen early next year.

Shares of the California-based drug developer rose 3% to $44 in premarket trading.

If approved, olezarsen will be the first available treatment for FCS in the U.S., Ionis said.

“We believe olezarsen has the potential to become the new standard of care for patients with FCS,” CEO Brett Monia said.

Olezarsen, if approved, is likely to become the first wholly owned Ionis drug to be sold. The company has partnered several major drugmakers including Biogen (BIIB.O) and Swiss-based Novartis (NOVN.S) to develop other treatments.