Plozasiran Notches Big Wins in Severe Hypertriglyceridemia

Two phase III studies have shown that the small interfering RNA therapy plozasiran (Redemplo) significantly reduces triglycerides in patients with severe hypertriglyceridemia, compared to placebo. After three months, triglyceride levels dropped by 79% and remained reduced by 81% after 12 months. At 12 months, 91-93% of plozasiran-treated patients had triglyceride levels below 500 mg/dL, the threshold for severe hypertriglyceridemia, while only 50-51% of placebo recipients achieved this.

Over 50% of plozasiran patients had triglyceride levels below 150 mg/dL, compared to under 10% of placebo recipients. The drug targets apolipoprotein C-III, which regulates triglyceride-rich lipoprotein metabolism in the liver. Arrowhead, the company behind plozasiran, plans to submit SHASTA trial data to the FDA for broader approval.

The drug also showed a 78% reduction in acute pancreatitis events and slight increases in HbA1c, which stabilized by 12 months. More research is needed on the impact of plozasiran on atherosclerotic cardiovascular disease risk.