A genetic medicine from Arrowhead Pharmaceuticals has succeeded in two late-stage clinical trials focused on a condition that increases the risk of sudden, potentially life-threatening cases of pancreas swelling.
The medicine, known as plozasiran, is a small piece of synthetic RNA designed to lower fat levels in the bloodstream. It does this by degrading the instructions cells use to create a protein that slows down triglyceride metabolism. Plozasiran is already approved, and sold under the brand name Redemplo, as a treatment for a rare disease that leads to extremely high levels of triglycerides. Arrowhead, though, is trying to expand the drug’s use.
Positive results released Wednesday should help in that mission. Across the two trials, which enrolled adults with “severe hypertriglyceridemia,” an under-the-skin injection of Arrowhead’s drug once every three months respectively cut median triglyceride levels by 79% and 81% after one year. The reduction seen in placebo-treated patients, meanwhile, was around 27%.
The studies also looked at cases of acute pancreatitis, which often occurs because of too-high triglyceride levels. A pooled analysis found a significant reduction among the participants given plozasiran. Broadly, the number of acute pancreatitis events dropped 78% compared to the studies’ control arms.
