Inclisiran, a New Type of PCSK9 Inhibitor, Passes Phase 2

Inclisiran — a long-acting, subcutaneously delivered, synthetic, small-interfering RNA molecule directed against proprotein convertase subtilisin–kexin type 9 (PCSK9) — significantly lowered LDL-cholesterol levels in a phase 1 trial involving people with baseline LDL levels ≥100 mg/dL (NEJM JW Cardiol Jan 2017 and N Engl J Med 2017 376; 41). Now, in a manufacturer-funded phase 2 trial, researchers randomized 501 adults with elevated LDL levels (>70 mg/dL plus a history of atherosclerotic cardiovascular disease [CVD] or >100 mg/dL without a history of atherosclerotic CVD) to receive a single dose of placebo or inclisiran (200, 300, or 500 mg on day 1) or two doses of placebo or inclisiran (100, 200, or 300 mg on days 1 and 90). All participa…