A new experimental cholesterol-lowering pill has produced groundbreaking results, reducing levels of low-density lipoprotein cholesterol (LDL-C) — often called “bad cholesterol” — by nearly 60%, according to early clinical trial data.

The drug, Enlicitide, developed by Merck, belongs to a new class of oral PCSK9 inhibitors, offering an alternative to existing PCSK9 treatments that require injections. The findings could reshape how millions of people manage high cholesterol, especially those with genetic disorders that make traditional treatments less effective.

Strong Results From Early Trials

Researchers studied nearly 300 participants living with heterozygous familial hypercholesterolemia (HeFH) — a genetic condition affecting 1 in 250 people worldwide, characterized by extremely high LDL levels from birth. All participants were already taking statins but were not seeing adequate results.

After 24 weeks of taking the once-daily Enlicitide pill:

  • LDL cholesterol dropped by 58.2%
  • The placebo group saw LDL levels increase slightly
  • 98% of participants adhered to the pill regimen, showing high compliance

Scientists noted that Enlicitide’s safety profile was similar to placebo, though long-term monitoring will be needed.

How the Drug Works

Enlicitide decanoate works by inhibiting PCSK9, a protein that blocks the liver’s ability to remove LDL cholesterol from the blood. By reducing PCSK9 activity, the liver can clear excess LDL more effectively.

PCSK9 inhibitors already exist, but they are injectable. An oral pill could dramatically improve patient adherence and accessibility.

Regulatory Approval Needed

Although the results from this Phase 3 trial are promising, the drug must undergo regulatory review before it can be prescribed. Scientists, however, are optimistic given the strong clinical outcomes.

Why LDL Cholesterol Matters

LDL is known as “bad cholesterol” because high levels lead to plaque buildup in arteries, increasing the risk of:

  • Heart attack
  • Stroke
  • Peripheral artery disease

Lowering LDL-C is one of the most effective ways to reduce long-term cardiovascular risk.

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