Experimental Drug Could Lower Blood Lipids More Than Available Treatments

An experimental drug may be able to lower levels of lipids in the blood to a greater degree than available treatments, helping to reduce the risk of heart health issues in high-risk patients, according to a new study.

Those who suffer from hypertriglyceridemia, or high levels of lipids (triglycerides or fats) in the blood, face a greater risk of heart attack, stroke, and acute pancreatitis — but an experimental treatment called olezarsen may be able to help.

That’s according to a new study by researchers at Brigham and Women’s Hospital, published in the New England Journal of Medicine.

Currently, patients with hypertriglyceridemia receive medications such as statins, ezetimibe, fibrates, and prescription omega-3 fatty acids to combat the condition. These drugs usually lower triglyceride levels from less than 10% to up to 40%.

While beneficial, these available treatments aren’t necessarily enough to prevent all cardiovascular events that stem from high lipids in the blood. That’s why researchers set out to determine whether olezarsen — a type of genetic therapy called antisense oligonucleotide that inhibits APOC3, a gene associated with higher levels of triglycerides, by targeting its mRNA — may be able to further reduce triglyceride levels and, subsequently, adverse outcomes.

The researchers conducted the placebo-controlled, double-blind trial on a randomized group of 154 adults on lipid-lowering therapy with moderate or severe hypertriglyceridemia. Participants received either 50 mg of olezarsen, 80 mg of olezarsen, or a placebo every four weeks for one year.

Ultimately, they found that the 50 mg dose of olezarsen reduced triglyceride levels by 49% and the 80 mg dose by 53%, compared with the placebo. It also reduced apolipoprotein B and non-HDL cholesterol, which contribute to plaque formation, by 18 to 18.5% and 23% to 25%, respectively.

“These findings indicate that targeting APOC3 mRNA is a promising new pathway for lowering triglycerides and potentially reducing the risk of heart attack and stroke,” said corresponding author Brian Bergmark, M.D., of the Division of Cardiovascular Medicine at Brigham and Women’s Hospital, in a news release.

No major safety concerns were identified throughout the trial. While more research is needed on the drug, the researchers said this new treatment may present a more effective, reliable course of action to prevent cardiovascular events in those with hypertriglyceridemia.