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Tech and Science

New Drug Lowers ‘Bad’ Cholesterol by 58% in Clinical Trial : ScienceAlert

Last updated: November 21, 2025 3:45 am
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New Drug Lowers 'Bad' Cholesterol by 58% in Clinical Trial : ScienceAlert
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A Breakthrough Treatment for High Cholesterol Levels

A groundbreaking new treatment has recently emerged as a potential solution for reducing high cholesterol levels and mitigating the associated risk of heart health issues. In a recent clinical trial, a drug known as enlicitide decanoate demonstrated remarkable efficacy in lowering ‘bad’ cholesterol by nearly 60 percent.

The study, conducted by an international team of researchers, focused on individuals with a genetic condition called heterozygous familial hypercholesterolemia (HeFH), which leads to dangerously high levels of low-density lipoprotein cholesterol (LDL-C) from birth.

The participants, totaling 293 individuals with HeFH who were already taking statins without achieving the desired results, were given a once-daily enlicitide decanoate pill. The results were astonishing, with LDL-C levels decreasing by an average of 58.2 percent after 24 weeks. In contrast, those in the placebo group experienced a slight increase in LDL-C levels.

According to the researchers, “In adults with HeFH, enlicitide is an effective and well-tolerated treatment for lowering the level of LDL-C.”

Positive Outcomes and Future Prospects

Not only were the side effects of enlicitide decanoate minimal and comparable to those in the placebo group, but the benefits of the drug remained consistent throughout the 52-week trial. Additionally, key cholesterol biomarkers associated with heart health showed improvement in the treatment group.

Enlicitide decanoate functions as a PCSK9 inhibitor, which helps regulate the activity of PCSK9, a protein that can impede the liver’s ability to eliminate LDL-C, leading to its accumulation. By targeting this mechanism, the drug facilitates the liver’s cholesterol-clearing function.

While existing PCSK9 inhibitors are administered via injections, enlicitide decanoate offers the convenience of oral administration. The high adherence rate among trial participants – 98 percent – highlights the potential of this treatment approach.

Implications for Heart Health

High LDL-C levels pose a significant health risk to millions of individuals worldwide, as they can contribute to the buildup of arterial plaques that restrict blood flow and increase the likelihood of heart attacks and strokes. The reduction in LDL-C levels achieved through enlicitide decanoate holds promise for mitigating these risks.

Future studies will assess whether the reduction in LDL-C translates to a decreased risk of cardiovascular events such as strokes and heart attacks, paving the way for broader applications of the drug. Regulatory approval is required before enlicitide decanoate can be prescribed, but the successful phase 3 trial results offer hope for individuals with HeFH.

The researchers caution that timely intervention to lower LDL-C levels is crucial for individuals with HeFH to reduce the risk of premature cardiovascular disease stemming from prolonged exposure to elevated cholesterol levels.

The findings of this research have been published in JAMA, marking a significant advancement in the treatment of high cholesterol levels and the promotion of heart health.

TAGGED:BadcholesterolclinicalDruglowersScienceAlertTrial
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