An Oral PCSK9 Inhibitor for Lowering LDL-C?

Subcutaneously administered inhibitors of proprotein convertase subtilisin/kexin type 9 (PCSK9) have been shown to significantly lower serum LDL cholesterol levels and reduce the incidence of cardiovascular events (NEJM JW Cardiol Mar 17 2017 and N Engl J Med 2017; 376:1713). Currently, however, no oral PCSK9 inhibitors are available. Now, in a manufacturer-funded phase 2 trial, researchers randomized 426 patients (mean age, 62 years; 52% men) with hypercholesterolemia (fasting LDL cholesterol ≥70 mg/dL but <190 mg/dL) already taking moderate- to high-intensity statin therapy, with or without ezetimibe, to receive once-daily oral AZD0780 (1, 3, 10, or 30 mg), a non–FDA-approved, small-molecule PCSK9 inhibitor, or matching placebo for 12 wee…