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New cholesterol drug a possible alternative to statins

March 15, 2019

A new class of drugs modestly reduced cholesterol and did not cause aching muscles in a Houston-led, year-long international study, potential help to patients who don’t tolerate high doses of statins.

The study, published Wednesday in the New England Journal of Medicine, reported the long-term positive data in patients receiving bempedoic acid, a small molecule that inhibits the body’s ability to create cholesterol. The patients got the drug in addition to varying doses of cholesterol-lowering regimens they were already taking.

“This is another tool in the toolbox, another way to individualize therapy for patients,” said Dr. Christie Ballantyne, chief of cardiology at Baylor and the study’s senior author. “It’s not going to replace statins, but in combination with other treatment, it could help some people reach their targets.”

On average, the drug reduced levels of bad cholesterol, LDL, 16.5 percent. That was 18 percent more than seen with a placebo.

The study, involving more than 2,200 patients, is the first to measure long term safety and effectiveness of the new treatment compared to a placebo in patients at high risk of heart attack and stroke. An ongoing complementary study of 12,000 people is looking at whether it reduces strokes and heart attacks.

The treatment, a daily pill, may be an option for people unable to sufficiently lower their LDL with statin therapy, the 1990s breakthrough that has reduced the risk of cardiovascular disease in millions of Americans but can cause side effects such as muscle pain, particularly in high doses. Studies show that about 10 percent of patients are unable to tolerate statins because of such side effects.

Cholesterol is crucial for the body, which uses it to make cell membranes. But the proteins that carry it around the bloodstream, particularly LDL, can build up in the walls of arteries, blocking blood flow to the heart and triggering heart attacks and strokes.

An estimated 71 million Americans have high LDL, including 28 million with levels of at least 160, an amount that puts them at high risk of heart attack or stroke. Nearly half aren’t taking medication to reduce the numbers, according to the Centers for Disease Control and Prevention.

Two cardiologists told the Chronicle bempedoic acid wouldn’t be a blockbuster but would fulfill a need for more non-statin cholesterol-lowering drugs.

“This seems like a great new alternative,” said Dr. Eric Yang, a Houston Methodist Hospital cardiologist. “It’s not groundbreaking but it looks like it’ll provide an incremental benefit in high-risk patients.”

Dr. Christopher Cannon, a cardiologist at Harvard and Brigham and Women’s Hospital, called bempedoic acid’s effect “moderate” but said “we’re always looking for ways to reduce LDL and this looks like another.”

Cannon said he is most enthusiastic about a pill combining bempedoic acid and ezetimibe, a non-statin that works by decreasing cholesterol absorption in the small intestine. A recent study found the combination pill reduced LDL by an average of 32 percent.

By comparison, statins reduce LDL by roughly 50 percent in high doses and 25 percent in lower doses. The greatest effect is from PCSK9 inhibitors, which reduce LDL by 60 percent but aren’t utilized at high levels because they’re so expensive.

It is unclear how much bempedoic acid would cost if approved for the U.S. market, but doctors said they expect it to be reasonably priced.

The study enrolled patients in the U.S., including Houston, Great Britain, Germany, Poland and Canada. They were randomly chosen to received either bempedoic acid or a placebo.

The bempedoic acid was well-tolerated, with some increased incidence of gout, due to slight increases in level uric acid in the blood. There were no side effects involving muscle pain, likely because the drug can’t penetrate muscles. Once it is converted into its active form in the liver, it is unable to leave liver cells.

The trial was funded by Esperion Therapeutics, a pharmaceutical company currently seeking Food and Drug Administration approval to market the drug in the U.S. The FDA should decide whether to grant the drug approval within the next 12 months, said Ballantyne.

todd.ackerman@chron.com

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