Merck kicks off Phase 3 tests for PCSK9 cholesterol pill

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Pharmaceutical giant Merck & Co. has begun late-stage testing of a cholesterol pill it hopes could be its next big heart medicine.

The company on Friday said it had started two Phase 3 studies of the pill, which works by blocking a protein called PCSK9 that regulates levels of LDL, or “bad,” cholesterol. While Amgen, Regeneron and Novartis sell injectable PCSK9 inhibitors, Merck’s drug, called MK-0616, would be the first pill.

Merck plans to test MK-0616 in three Phase 3 trials in total, studying the pill in adults with high cholesterol and measuring how well it reduces their LDL cholesterol levels and heart risk.

The company chose to advance the drug after positive results from a small mid-stage study of 381 adults. Results, which were published in the Journal of the American College of Cardiology in April, showed the drug lowered LDL cholesterol between 40% and 60% after eight weeks of treatment. Treatment was not associated with a greater rate of side effects, either.

“With its novel modality among PCSK9 inhibitors, MK-0616 could provide an important option for patients for the treatment of hypercholesterolemia,” said Marc Sabatine, chair of cardiovascular medicine at Brigham and Women’s Hospital, and professor of medicine at Harvard Medical School, in a statement provided by Merck.

The company is now enrolling participants in the first two of the Phase 3 studies, dubbed CORALreef Lipids and CORALreef HeFH. The first will include people with a history of at least one major heart event or are at risk of their first, and are on lipid-lowering therapies already. The second will involve people with heterozygous familial hypercholesterolemia, a genetic condition that causes high cholesterol.

The third study, CORALreef Outcomes, will begin by the end of the year and test whether MK-0616 increases the time to first occurrence of heart attack, stroke, heart disease death or other serious cardiovascular events.

The first two studies are expected to finish in 2025, Merck said, while the heart outcomes trial will continue to 2029.