An entirely new cholesterol-lowering drug is on track for broad clinical approval after the latest trial results to be published reveal it is both safe and effective when administered in conjunction with commonly prescribed statins.

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

Bempedoic acid has been in development for several years. Much like commonly prescribed statins, the drug works by blocking production 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 efficacy of bempedoic acid administered in conjunction with statin therapy for over a year in more than 2,000 subjects. The results were incredibly promising, with no more adverse effects reported in the treatment group compared to a placebo. The combination therapy was also found to 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 efficacy of bempedoic acid in conjunction with statins, broader trials are ongoing to examine the efficacy of the new drug when administered on its own. A second, newly published study into the likely long-term effects of bempedoic acid also delivered promising results.

This study tracked genetic markers in over half a million people to compare the potential effects of inhibiting the enzyme that bempedoic acid works on (called ATP citrate), compared to the effects of the enzyme that statins traditionally inhibit. These results confidently suggest blocking ATP citrate should be just as safe and effective as statins over a long period of time. The study also claims bempedoic acid may avoid some of the more negative, muscle-related side effects often associated with statins, due to a different mechanism of action.

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

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

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