Arrowhead Pharmaceuticals’ New Drug Plozasiran Shows Promise in Treating Rare Genetic Disease

Arrowhead Pharmaceuticals announced positive results from its Phase 3 PALISADE study on plozasiran, a new drug for treating familial chylomicronemia syndrome (FCS). FCS is a rare genetic condition that causes extremely high triglyceride levels, leading to serious health issues like acute pancreatitis. Currently, there are no approved treatments for FCS in the United States or Canada. The study showed that plozasiran significantly reduced triglyceride levels and the risk of acute pancreatitis in patients with FCS. 

Plozasiren is also being trialed as a potential treatment option for hypertriglyceridemia, which is highly similar to FCS in that it is marked by high levels of triglycerides in a person’s blood. Elevated triglycerides may contribute to pancreatitis or hardening of the arteries, increasing the risk of stroke, heart attack, and heart disease.  

Join A Study Investigator Dr John O’Mahony will be enrolling volunteers with hypertriglyceridemia in the SHASTA-3 and MUIR-3 studies at his research site in Sarnia, ON. If you are interested in learning more about this opportunity to participate in research, please click the links at the bottom of this article and provide your contact information to learn more. 

The PALISADE study involved 75 patients, some with genetically confirmed FCS and others with persistent symptoms suggestive of the disease. Patients were given different doses of plozasiran or a placebo every three months for 12 months. Results showed that patients receiving plozasiran had an 80% reduction in triglyceride levels, compared to only 17% in those given a placebo. The drug also reduced levels of apolipoprotein C-III (APOC3) by up to 96%, compared to a 1% reduction in the placebo group. Most importantly, plozasiran significantly lowered the risk of acute pancreatitis by 83% compared to placebo. 

The drug was found to be generally safe, with most side effects, such as abdominal pain, headache, and nausea, being mild and similar between the plozasiran and placebo groups. Severe side effects were more common in the placebo group. 

Based on these promising results, Arrowhead plans to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) by the end of 2024 and seek approval from other regulatory bodies worldwide. If approved, plozasiran could become the first specific treatment for FCS, offering hope to patients and doctors who currently have limited options when addressing treatment of the disease.