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.
