"The end result was cholesterol levels came down and down and down, and we've seen cholesterol levels lower than we have ever seen before in the practice of medicine".
Some patients who are unable to take traditional cholesterol-lowering drugs known as statins, due to their side effects, may be able to take PCSK9 inhibitors instead. In the research paper, the scientists show how one heart attack or stroke was prevented for every 74 patients taking the drug in the two-year trial. All of them were already receiving statin therapy.
FOURIER is the first outcomes trial to read out for the PCSK9 class. Sanofi and Regeneron are conducting their own study, dubbed ODYSSEY, to test Praluent's cardiovascular benefit. The detailed results from EBBINGHAUS were presented at a Late-Breaking Clinical Trials Session at the American College of Cardiology 66th Annual Scientific Session (ACC.17) in Washington, D.C. People with naturally-occurring mutations in the gene encoding PCSK9 have lower levels of LDL cholesterol, and are protected from heart disease, strongly suggesting that an antibody against the PCSK9 protein should have the same beneficial effects - a story that remains the poster-child for exploiting human genetics to understand the biology of common diseases (such as heart disease) in the same way it revolutionised the discovery of new treatments for rare diseases a decade earlier.
The results are rooted in the drug's ability to cut levels of "bad" LDL cholesterol, which furs arteries and causes heart problems.
There are approximately 2.3 million people living with coronary heart disease in the United Kingdom, according to the NHS. This drug would cost $14,000 per year, which means that everyone would not be able to acquire it. JP Morgan's view is that, given the data and the current payer environment, physicians will choose to reserve treatment for patients with multiple comorbidities and relatively higher LDLs than the cutoff for FOURIER ( 70mg/dL).
Some doctors hailed the results as major progress against heart disease.
The FDA approved the use of the Repatha injection in August 2015 as a Proprotein convertase subtilisin/Kexin type 9 (PCSK9) inhibitor.
Now, this new phase 2 clinical trial has confirmed the effectiveness of injecting inclisiran for reducing cholesterol that can be taken alone or potentially combined with statins for maximum effect.
The best dose turned out to be 300 mg given three months apart, which cut LDL-c by around 45% at nine months, and there have been no additional concerns about safety after the fatal heart attack in one patient reported previous year. It also blocks the capacity of the PCSK9, so there is a high number of receptors to break up cholesterol LDL.
"After an average of 19 months of treatment, our data show that changes in memory and cognitive function were very small and similar between patients treated with evolocumab and those treated with placebo", said Robert Giugliano, a cardiac doctor at BWH.
Result of study involving Repatha were shared on Friday at a meeting of the American College of Cardiology.
After about two years, Repatha, used along with statins, reduced LDL from a median of 92 to 30.
"With this trial, we now have definitive data that by adding evolocumab to a background of statin therapy, we can significantly improve cardiovascular outcomes and do so safely", said lead author Marc S. Sabatine from Brigham and Women's Hospital in Boston.