By Betsy McKay

A statin isn't the only answer anymore to lowering cholesterol.

The lipid-reducing medicines, among the most widely prescribed drugs in the U.S., have been a mainstay of heart-disease prevention and treatment for decades. But they don't work for everyone, and can only reduce harmful "bad" cholesterol so much.

Now some patients have other cholesterol-busting medicines available as options -- and even more alternatives are on the horizon.

Certain patients already can take a twice-yearly injection, sold by Novartis as Leqvio, that uses an RNA-based technology, or a more frequent injection that targets a protein called PCSK9 that interferes with the body's ability to clear the bad form of cholesterol.

Biotech Amgen has been doing testing to expand use of its PCSK9 drug Repatha to more patients, while Merck is developing an easier-to-take pill version. Biotechs are working on therapies that use gene-editing technology to lower people's cholesterol, perhaps permanently.

Researchers presented the latest study results this weekend at the American Heart Association's annual meeting.

"This is an amazing time, a very, very exciting time for patients," said Dr. Leslie Cho, cardiologist and director of the Cleveland Clinic Women's Cardiovascular Center, who wasn't involved in the latest studies. She cautioned, however, that most patients can be helped by diet, exercise and statins, and some of the newer therapies can be very expensive.

In a late-stage study, Merck's PCSK9 pill reduced bad, or LDL, cholesterol up to 60% over six months in adults with or at risk of atherosclerotic cardiovascular disease, researchers reported.

Amgen's PCSK9 drug, Repatha, reduced the risk of heart disease, stroke and other cardiovascular events by 25% in a large Phase 3 study of subjects taking a statin and at high risk of an event but haven't had one.

An early, or Phase 1, study found that a gene-editing drug from CRISPR Therapeutics cut cholesterol levels by 49% in two months in patients who received the highest dose, study investigators reported. The study included 15 patients.

The therapy also reduced levels of triglycerides, a type of fat in the body, by 55%. The therapy uses so-called Crispr-Cas9 technology to knock out a cholesterol-promoting gene called ANGPTL3 that pushes up levels of cholesterol and triglycerides.

The goal is to offer a "one and done" therapy, said Dr. Luke Laffin, a preventive cardiologist at the Cleveland Clinic and lead author of the study, published in the New England Journal of Medicine.

While it is still early, "ultimately technologies like this will likely play a role in supplanting every two weeks injections and taking daily pills," he said.

High cholesterol is a major driver of heart disease. Statins, like Lipitor and Zocor, emerged as powerful antidotes starting in the 1980s. With blood-pressure medicines, antismoking campaigns and advances in treating heart attacks, the medicines contributed to a sharp decline in heart-disease deaths.

Yet those declines stalled nearly a decade-and-a-half ago, and heart disease is still the nation's biggest killer.

More than a quarter of Americans have levels of LDL cholesterol that are considered high -- 130 milligrams per deciliter -- according to the most recent data from AHA. Doctors generally recommend that people at high risk of a heart attack or stroke keep their LDL cholesterol less than 70 mg/dL, and those at very high risk keep it below 55 mg/dL. People at very high risk have had a heart attack or stroke and have multiple high-risk conditions like hypertension, diabetes or are age 65 or older.

"Often statins are not enough," said Dr. Jamal Rana, a cardiologist and professor of clinical science with Kaiser Permanente in Oakland, Calif.

Because statins don't reduce cholesterol levels low enough in certain high-risk patients, Rana adds other medicines to their prescriptions like the pill Zetia that also goes by the generic name ezetimibe, a PCSK9 drug or Leqvio, also known as inclisiran.

A little over half of people who could benefit from a cholesterol medicine are on one, according to the Centers for Disease Control and Prevention. Many quit within a year of starting a statin. Some drop off due to side effects like muscle pain or headaches.

Mary-Ellen Conway, 78, of Houston, has taken two statins over several years but got leg cramps taking one, while her LDL cholesterol levels were higher than she wanted on the other medicine.

Her LDL cholesterol has dropped significantly while a subject in the Phase 3 study of Merck's experimental PCSK9 pill. Her LDL cholesterol at last check was 35 mg/dL. It had been 166 mg/dL in 2003, and 62 mg/dL in 2024, when she took a statin and another drug.

The adults in Conway's trial also took statins. Merck said its drug, called enlicitide decanoate, is now being studied to see how effective it is at preventing heart attacks and strokes, with results expected in a couple of years. Merck said it plans to file for FDA approval early next year.

No serious side effects were reported among the 15 patients in the trial of CRISPR Therapeutics' experimental therapy, called CTX310.

Such gene-editing therapies must be tested in thousands more patients to assess their effectiveness and safety, said Dr. Kiran Musunuru, cardiologist, geneticist and professor for translational research at Penn Medicine, who wasn't involved in the CRISPR Therapeutics study.

A subject in a trial of a different gene-editing therapy died a few days ago, according to biotech Intellia Therapeutics. Studies testing the drug are now on hold.

Musunuru expects gene-editing therapies will become available by the 2030s and provide permanent and more targeted treatment to certain patients depending on the genetics of their conditions. "In my opinion, the more options, the better," he said.

Write to Betsy McKay at betsy.mckay@wsj.com

(END) Dow Jones Newswires

November 08, 2025 17:28 ET (22:28 GMT)

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