Merck & Co. presented the first results from its Phase 3 CORALreef HeFH trial for the investigational oral PCSK9 inhibitor enlicitide decanoate at the American Heart Association Scientific Sessions 2025. The study enrolled adults with heterozygous familial hypercholesterolemia and showed a 59.4% reduction in LDL‑C at week 24 versus placebo, with a 95 % confidence interval of –65.6 to –53.2 and a p‑value of less than 0.001. Safety data were comparable to placebo, with similar rates of adverse events and low discontinuation rates.
The magnitude of LDL‑C lowering is on par with the approved monoclonal‑antibody PCSK9 inhibitors, but enlicitide would be the first oral agent in this class. An oral formulation could dramatically improve patient adherence and convenience, addressing a substantial unmet need in a market that includes an estimated 31 million people worldwide with heterozygous familial hypercholesterolemia—roughly 1 in 250 of the global population.
For Merck, the data represent a significant expansion of its cardiovascular portfolio and a new revenue source beyond its oncology and vaccine businesses. The results reinforce the company’s strategy of diversifying its pipeline, which is particularly important as Keytruda—Merck’s blockbuster immunotherapy—generated $29.5 billion in 2024 and accounted for nearly 46 % of total revenue, with its U.S. patent set to expire in 2028.
Dean Y. Li, president of Merck Research Laboratories, said, “We look forward to sharing the totality of the results from the CORALreef program presented at AHA with regulatory authorities and progressing enlicitide’s ongoing clinical development program to bring forward the potential first approved oral PCSK9 inhibitor to help address the growing CV epidemic.”
The trial results were simultaneously published in the Journal of the American Medical Association, providing peer‑reviewed validation of the findings. The study’s safety profile, efficacy, and patient population details are now available to clinicians and regulators, setting the stage for potential regulatory submissions and market entry.
The 59.4% LDL‑C reduction, achieved in a well‑controlled, placebo‑controlled trial, positions enlicitide as a promising candidate to fill a therapeutic gap. If approved, the drug could capture a sizable share of the global HeFH market, offering a convenient once‑daily pill that could improve adherence and outcomes for patients who currently rely on injectable therapies.
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