PASADENA, Calif. - Arrowhead Pharmaceuticals Inc. (NASDAQ: NASDAQ:ARWR) today announced significant results from its Phase 3 PALISADE study of plozasiran, a treatment for familial chylomicronemia syndrome (FCS), a rare genetic disorder without FDA-approved therapies.
The study met its primary endpoint, showing substantial reductions in triglycerides and a decrease in acute pancreatitis incidents compared to a placebo.
Plozasiran, an investigational RNAi-based therapy, demonstrated median triglyceride reductions of up to 80% and mean reductions in Apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism, by up to 94% at month 10. The study also observed a significant reduction in the incidence of acute pancreatitis, a severe complication of FCS.
The PALISADE study, which included patients with genetically confirmed or clinically diagnosed FCS, showed that plozasiran has a favorable safety profile, with fewer severe and serious adverse events reported compared to the placebo group. Common adverse events included abdominal pain, COVID-19, nasopharyngitis, headache, and nausea.
Dr. Bruce Given, chief medical scientist at Arrowhead, expressed optimism in the potential of plozasiran for treating various cardiometabolic disorders. The company plans to discuss the results at the upcoming Cardiometabolic event on June 25, 2024, and to present full data at future medical conferences.
Christopher Anzalone, president and CEO of Arrowhead, highlighted the data as potentially best in class, with the ability to address multiple diseases with substantial unmet needs. Arrowhead intends to communicate these results to the FDA and explore a New Drug Application for FCS.
The PALISADE study is part of a broader clinical program for plozasiran, which includes ongoing Phase 3 studies in severe hypertriglyceridemia (SHTG) and mixed hyperlipidemia. The drug has received Orphan Drug and Fast Track Designations from the U.S. FDA and Orphan Drug Designation from the European Medicines Agency.
Arrowhead has also established an early access program (EAP) for plozasiran, offering hope to individuals living with FCS as it progresses through the regulatory process.
This news is based on a press release statement from Arrowhead Pharmaceuticals, Inc. and has not been independently verified. The company is known for developing RNAi-based therapeutics aimed at silencing genes responsible for intractable diseases.
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