Ionis shares phase 3 results for olezarsen in moderate hypertriglyceridaemia

The drug was recently approved in the US for familial chylomicronaemia syndrome

Ionis Pharmaceuticals has shared positive top-line results from a late-stage study of olezarsen in hypertriglyceridaemia.

The phase 3 Essence trial, conducted with Ionis’ research partner the TIMI Study Group, has been evaluating the RNA-targeted drug in adults aged 18 years and older with moderate hypertriglyceridaemia.

All patients were diagnosed with, or were at risk for, atherosclerotic cardiovascular disease (ASCVD), and the vast majority were on current standard-of-care lipid-lowering medicines.

The study met its primary endpoint, with a statistically significant placebo-adjusted 61% and 58% reduction in triglyceride (TG) levels with the 80mg and 50mg monthly doses of olezarsen, respectively, after six months of treatment.

All key secondary endpoints were also achieved, including percentage changes in triglyceride levels at 12 months, and olezarsen demonstrated a favourable safety and tolerability profile.

Hypertriglyceridaemia is a condition where the blood contains abnormally high levels of triglycerides, a type of fat the body uses as a source of energy. Patients are at an increased risk of developing ASCVD, as well as acute pancreatitis.

Given as a subcutaneous injection, olezarsen is designed to lower the production of a protein that regulates triglyceride metabolism in the blood and is already approved in the US under the brand name Tryngolza to help reduce triglycerides in adults with familial chylomicronaemia syndrome (FCS).

Sam Tsimikas, Senior Vice President, Global Cardiovascular Development at Ionis, said: “The positive results of this study are an important step in bringing forward a potential new treatment for people with severely elevated triglycerides.

“Following the [US Food and Drug Administration] approval and encouraging launch of [Tryngolza] for people living with FCS, a rare, genetic form of severely elevated TGs, this data supports olezarsen’s potential to benefit the much broader population of people living with severe hypertriglyceridaemia.”

The drug is also currently being evaluated in two phase 3 clinical trials as a treatment for severe hypertriglyceridaemia, with data from these studies expected in the third quarter of this year.