Ionis Pharmaceuticals has reported positive topline outcomes from the randomised, global Phase III Essence study of Tryngolza (olezarsen) in individuals with moderate hypertriglyceridemia and, or at risk for, atherosclerotic cardiovascular disease (ASCVD).
This condition is characterised by fasting triglyceride (TG) levels between ≥150mg/dL and <500mg/dL.
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The multicentre, double-blind, placebo-controlled Essence study was carried out with the TIMI Study Group, the company’s research partner.
It enrolled 1,478 subjects aged 18 years and above diagnosed with or at risk for ASCVD, and 9% of them had fasting triglycerides of ≥500mg/dL at baseline.
Subjects received either 50mg or 80mg of olezarsen or a placebo every four weeks via subcutaneous injection for 12 months.
The primary endpoint focused on the percentage change from baseline in fasting TG levels at the six-month mark, compared to the placebo.
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By GlobalDataKey secondary endpoints were changes in TG levels at 12 months, the proportion of subjects achieving fasting TG levels of <150mg/dL, and the percentage variations in other lipid parameters against the placebo over the treatment period.
The trial, which included subjects already on standard of care, lipid-lowering medications, achieved its primary endpoint.
A significant decrease in TG levels was observed, with a placebo-adjusted 61% decrease at the 80mg monthly dose and a 58% decrease at the 50mg dose over a six-month period, compared to the placebo.
In addition, the therapy met all key secondary endpoints, with the majority of subjects achieving normal TG levels of <150mg/dL.
Olezarsen also demonstrated a favourable tolerability and safety profile in the trial.
Ionis Pharmaceuticals global cardiovascular development senior vice-president Sam Tsimikas said: “Following the Food and Drug Administration (FDA) approval and encouraging launch of Tryngolza (olezarsen) for people living with familial chylomicronaemia syndrome (FCS), a rare, genetic form of severely elevated TGs, these data support olezarsen’s potential tobenefit the much broader population of people living with severe hypertriglyceridemia (sHTG).”
The investigational RNA-targeted medicine is designed to decrease the body’s apolipoprotein C III (apoC-III) production, a protein for regulating the metabolism of triglyceride in the blood.
Last November, Ionis announced the design for a pivotal Phase III trial of ION582, an investigational treatment for Angelman syndrome (AS), following discussions with the US FDA.
