Arrowhead Pharmaceuticals (NASDAQ: ARWR), a provider of RNAi-based therapeutics, announced on Monday that it presented promising results from its pivotal Phase 3 PALISADE study at the European Society of Cardiology (ESC) Congress 2024.
The study evaluated plozasiran, an investigational RNA interference therapeutic, in patients with familial chylomicronemia syndrome (FCS), a severe and rare genetic disorder characterized by extremely high triglyceride levels and an increased risk of acute pancreatitis.
Key findings included: Triglyceride Reduction: Plozasiran achieved an 80% reduction in triglyceride levels from baseline, significantly outperforming the placebo group; Acute Pancreatitis Risk: The treatment reduced the risk of developing acute pancreatitis by 83%; and Consistent Efficacy: The drug was effective in both genetically confirmed and clinically diagnosed FCS patients.
These robust results pave the way for Arrowhead to file a New Drug Application (NDA) with the FDA by the end of 2024.
Results from the study were also simultaneously published in The New England Journal of Medicine.
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