LDL cholesterol reduced with experimental evolocumab drug

The Amgen Inc experimental drug evolocumab met its trial goal of reducing LDL cholesterol levels in patients who tend to have high levels of the "bad" cholesterol due to a genetic predisposition.

Amgen announced on Monday that participants who were injected with evolocumab, also known as AMG-145, once a month, on top of standard daily statin treatments, showed "clinically meaningful" improvement compared with taking statins alone after 12 weeks of treatment.

In mid-stage studies, evolocumab cut LDL levels by as much as 60 percent more than with statins alone.

The Phase 3, or late-stage study, called TESLA, looked at 49 people with a rare condition called homozygous familial hypercholesterolemia, which affects more than 1 million people worldwide. It can cause a four-fold increase in LDL cholesterol levels, greatly raising the risk of heart disease.

Evolocumab works by blocking PCSK9, a naturally occurring protein that increases LDL levels in the bloodstream. If approved, it "would be used mainly for very high-risk patients, who have frustratingly high LDL despite statin treatment," Richard Purkiss, an analyst with Atlantic Equities, told Reuters.

Other drug-making companies, including Pfizer Inc and a partnership between Regeneron Inc and Sanofi, are competing with Amgen to complete trials of anti-PCSK9 antibodies, which can potentially be very profitable.

"You could have two or three drugs here, each with upwards of $3 billion to $4 billion in annual sales," Purkiss said.

After the completed TESLA study, Amgen says the most common adverse side effects are upper respiratory tract infection, gastrointestinal inflammation and a stuffy nose, regardless of whether a patient takes statins with or without the evolocumab drug.

Trials were conducted not just on participants with a genetic tendency toward the artery-clogging fat. Amgen looked at populations whose high cholesterol is not due to serious genetic causes, as well as trials among those who take a different cholesterol-fighting drug, Zetia (ezetimibe) from Merck & Co.

Amgen, the world's largest biotechnology company, noted that the data will be presented at a future medical conference.