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Severe hypertriglyceridemia increases risk for acute pancreatitis and atherosclerotic cardiovascular disease, yet currently available therapies are often inadequate to control it. A non–FDA-approved, small interfering RNA agent, plozasiran, which inhibits the endogenous protein apolipoprotein C3 (APOC3), is under investigation as a possible therapy in this setting.
To investigate the tolerability, efficacy, and dose of plozasiran, researchers conducted a phase 2b, double-blind, dose-ranging study in 229 patients (mean age, 55 years; 78% men) with fasting triglyceride levels of 500 to 4000 mg/dL while on lipid-lowering therapy (mean baseline triglyceride level, 897 mg/dL). Participants were randomized to receive subcutaneous plozasiran (10, 2…