Arrowhead Pharmaceuticals Presents New Pivotal Phase 3 Data at ESC 2024 from PALISADE Study of Plozasiran in Patients with Familial Chylomicronemia Syndrome

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced results from the Phase 3 PALISADE study of investigational plozasiran in patients with familial chylomicronemia syndrome (FCS), a severe and rare genetic disease which currently has no approved treatments in the U.S. PALISADE successfully met its primary endpoint and all multiplicity-controlled key secondary endpoints, including statistically significant reductions in triglycerides (TGs), apolipoprotein C-III (APOC3), and the incidence of acute pancreatitis (AP). These data were presented today in a late-breaking oral presentation at the European Society of Cardiology (ESC) Congress 2024 and simultaneously published in The New England Journal of Medicine.

Based on these positive findings from the PALISADE study, Arrowhead intends to file a New Drug Application with the United States Food and Drug Administration (FDA) by year-end 2024 and plans to seek regulatory approval with additional global regulatory authorities thereafter.

“People living with extremely high triglyceride levels, like those in the PALISADE study, have a substantially higher risk of developing acute pancreatitis and associated long-term sequelae, including a poor quality of life. There are currently no approved therapies in the U.S. to specifically treat FCS, so as physicians we have very few options to help our patients other than various triglyceride-lowering medications which provide minimal benefit, and very strict diet restrictions that take a significant toll on patients and their families,” said Gerald F. Watts, D.Sc., M.D., Ph.D., Winthrop Professor of Cardio-metabolic Medicine at the University of Western Australia, Perth. “Plozasiran demonstrated very deep reductions in triglycerides in the PALISADE study and is the only investigational medicine to achieve a statistically significant reduction in the risk of developing acute pancreatitis in patients with genetically confirmed and clinically diagnosed FCS in a controlled study. These results are encouraging and offer hope to people living with FCS and their physicians who are in desperate need of new safe and effective treatment options.”

Bruce Given, M.D., chief medical scientist at Arrowhead, added, “We continue to be impressed by the promising results from the SUMMIT program of clinical studies of plozasiran in various patient populations, including SHASTA in patients with severe hypertriglyceridemia, MUIR in patients with mixed hyperlipidemia, and now PALISADE in patients with FCS. Based on the data generated to date, we view plozasiran as potentially best-in-class and supportive of development across the spectrum of triglyceride disorders. Specifically, today we showed that in PALISADE a high proportion of patients receiving plozasiran achieved triglyceride levels below guideline-directed risk thresholds associated with the risk of acute pancreatitis, which is a critical treatment goal that physicians communicate to us frequently. Further, PALISADE included patients with an established genetic diagnosis of FCS and patients with symptomatic, persistent chylomicronemia suggestive of FCS. The consistency of results in PALISADE suggests that plozasiran response may be independent of the presence of known FCS-associated genetic variants. This is supportive of the potential value of plozasiran in patients with clinically diagnosed disease, regardless of genetic status.”