Amgen Inc’s Repatha drug cut the risk of heart attacks and strokes by over 20 percent in patients with heart disease, demonstrating a clinical benefit beyond its ability to slash “bad” LDL cholesterol levels, data from a huge study known as Fourier showed on Friday.
However, the overall results came in at the low end of investor expectations with no impact on heart-related deaths and Amgen shares fell 6.4 percent. Shares of Regeneron Pharmaceuticals, which sells a rival drug, Praluent, along with Sanofi were off about 4 percent. Sanofi shares were down 1 percent in Paris.
Amgen shares rose 3 percent last month when it announced that the study had succeeded. Results form a similar Praluent trial are expected later this year.
It remains to be seen whether results of the highly anticipated 27,564-patient Fourier study will help remove some barriers to patient access from health insurers and pharmacy benefit managers, who have been rejecting some 75 percent of prescriptions written for the medicine with a list price of over $14,000 a year before discounts.
Repatha cut the combined risk of heart attacks, strokes and heart-related death by 20 percent compared with a placebo in patients already on high doses of cholesterol-lowering statins, such at Lipitor. Most trial subjects had had a prior heart attack or stroke.
Separately, Repatha cut heart attacks by 27 percent and stroke by 21 percent. In the second year of the study, the results were more pronounced, with a 35 percent reduction in heart attack risk and a 24 percent decrease in stroke risk.
“Just like in statin trials the benefit appeared to grow over time,” said Dr. Marc Sabatine, the study’s lead researcher, who presented the data at the American College of Cardiology scientific meeting in Washington.
“As a clinician this is very big news. We have another tool to significantly reduce heart attacks and stokes. That’s a big win,” said Sabatine from Brigham and Women’s Hospital in Boston.
Dr. Steven Nissen, chief of cardiology at Cleveland Clinic, who was not involved in this study but has led other Repatha trials, said expectations for more dramatic results were unrealistic.
“The magnitude of benefit on heart attacks and strokes are about what thoughtful people would have expected,” he said, adding that he did not expect to see a death benefit in a trial that lasted only 26 months.
The trial’s primary composite goal included need for artery clearing procedures and hospitalization due to chest pains from angina in addition to heart attack, stroke and death. On that measure, the overall risk reduction of 15 percent was below analyst expectations. That was primarily due to no difference from placebo in angina hospitalizations.
Repatha, injected either once or twice a month, lowered LDL by about 60 percent to a median of 30, with a quarter of patients getting below 20, researchers reported. Earlier guidelines set an LDL target of 70 for such high risk patients.
There were no reports concerning safety issues. Incidence of cognitive decline, cataracts, new onset diabetes and muscle-related side effects were similar to the placebo group.
Amgen said it is taking steps to remove onerous barriers to patient access, including a plan under which it would offer to refund Repatha costs for all eligible patients who have a heart attack or stroke.
Sabatine said he would like to see Repatha tested in other high-risk patients, such as those with type 2 diabetes without prior history of heart attack, which could substantially increase the market.
“As a society we need to move to modifying these risk factors earlier in life to not only prevent recurrent events but to prevent first events,” he said.
(Editing by Bernadette Baum and Nick Zieminski)