Merck & Co., Inc. (MRK.US) announces the results of Phase 3 clinical trials for oral PCSK9 inhibitors, showing significant efficacy in treating cholesterol.

Merck & Co., Inc. (MRK.US) announced today (June 10th) that its investigational oral PCSK9 inhibitor, enlicitide decanoate (MK-0616), has shown positive results in the first two of three Phase 3 clinical trials, demonstrating effectiveness in treating adults with hypercholesterolemia who are already on lipid-lowering therapy (including at least statin therapy). According to the press release, enlicitide decanoate is expected to be the first oral PCSK9 inhibitor approved by the U.S. FDA. Hypercholesterolemia is a disease characterized by elevated levels of low-density lipoprotein cholesterol (LDL-C) in the blood, and it is a major risk factor for atherosclerotic cardiovascular disease (ASCVD). Despite dietary adjustments and statin therapy, many hypercholesterolemic patients fail to reach or maintain the recommended LDL-C reduction targets. Participants had a history of ASCVD events or high ASCVD risk, and were already receiving moderate- or high-intensity statin therapy, with some possibly using other lipid-lowering medications concurrently. Primary endpoints included the average percentage change in LDL-C levels from baseline at Week 24, the number of participants experiencing one or more adverse events (AE), and the number of participants who discontinued the investigational drug due to an AE. Secondary endpoints included the average percentage change in LDL-C from baseline at Week 52, as well as the average percentage change in non-high-density lipoprotein cholesterol (non-HDL-C), apolipoprotein B (ApoB), and lipoprotein(a) levels at Week 24. Analysis showed that both the CORALreef HeFH and CORALreef AddOn trials successfully met their primary endpoints as well as all key secondary endpoints. Data revealed that enlicitide decanoate demonstrated statistically significant and clinically meaningful improvement in reducing patients' LDL-C levels compared to placebo (CORALreef HeFH) or other oral non-statin medications (CORALreef AddOn). Additionally, the occurrence rate of adverse events and serious adverse events (SAE) in patients using this therapy in both studies did not show any significant differences compared to the control group.