Medical

Positive human trial results for promising new cholesterol-lowering drug

Positive human trial results f...
A new drug for lowering cholesterol is hoped to be available within the next one to two years
A new drug for lowering cholesterol is hoped to be available within the next one to two years
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A new drug for lowering cholesterol is hoped to be available within the next one to two years
1/1
A new drug for lowering cholesterol is hoped to be available within the next one to two years

An entirely new cholesterol-lowering drug is on track for broadclinical approval after the latest trial results to be publishedreveal it is both safe and effective when administered in conjunctionwith commonly prescribed statins.

Levels of LDL cholesterol in the blood are intimately connected to heart disease andstroke, so lowering those levels can be a vital strategy inmaintaining cardiovascular health. Statins, the big class ofcholesterol-lowering drugs currently prescribed, are relativelyeffective but not without side effects. In many cases the benefits of the drug outweigh any potential side effects, but some patients areentirely unable to safely take statins so there is a pressing need for newcholesterol-lowering drugs.

Bempedoic acid has been in development for several years. Muchlike commonly prescribed statins, the drug works by blockingproduction of a key enzyme the body needs to build cholesterol,however bempedoic acid targets a very different enzyme to statins.

This latest study reports the results of the safety and efficacyof bempedoic acid administered in conjunction with statin therapy forover a year in more than 2,000 subjects. The results were incrediblypromising, with no more adverse effects reported in the treatmentgroup compared to a placebo. The combination therapy was also foundto significantly lower levels of low-density lipoprotein (LDL)cholesterol compared to patients solely on statin treatments.

While this particular study was evaluating the safety and efficacyof bempedoic acid in conjunction with statins, broader trials areongoing to examine the efficacy of the new drug when administered onits own. A second, newly published study into the likely long-termeffects of bempedoic acid also delivered promising results.

This study tracked genetic markers in over half a million peopleto compare the potential effects of inhibiting the enzyme thatbempedoic acid works on (called ATP citrate), compared to the effectsof the enzyme that statins traditionally inhibit. These resultsconfidently suggest blocking ATP citrate should be just as safe andeffective as statins over a long period of time. The study alsoclaims bempedoic acid may avoid some of the more negative, muscle-related sideeffects often associated with statins, due to a differentmechanism of action.

"One of the key advantages of bempedoic acid is supposed to bethat it shouldn't cause the muscle side effects reported by somestatins users, as it taken up by the liver and needs to be convertedinto its active form via an enzyme only found in the liver,"explains Kausik Ray, lead on the new research from Imperial CollegeLondon. "Once converted to the active form the drug cannot leavethe liver, so it can't enter muscles and hence could be ofconsiderable advantage for some."

Ray suggests this makes the new drug potentially useful topatients who have tried statins and either found them ineffective, orsuffered from major side effects. It is unclear at this stage how faroff bempedoic acid is from being approved and implemented in clinicaluses. Esperion, the pharmaceutical company working with the drug, hopes to push for approvals in Europe and the US later this year butrealistically it probably will be at least 2020 before the new drugis broadly available to patients around the world.

The new study was published in The New England Journal of Medicine.

Source: Imperial College London

2 comments
guzmanchinky
"Levels of LDL cholesterol in the blood are intimately connected to heart disease and stroke" - what's interesting is several other studies have shown there isn't much of a statistically significant correlation between the two. But new drugs that are better than Statins, which have terrible side effects for many people, is a step in the right direction.
JohanJ
When will these people learn that cholesterol is not the problem? When they stop making money from it. It's a racket and more people die from medicine/medical intervention in America than from any other cause - in about half the cases from 'correctly' prescribed medicine. But they will persist with this witch-hunt on cholesterol as long as they make money from it.