Arrowhead Pharmaceuticals Reports Successful Topline Results for Plozasiran from the Pivotal Phase 3 PALISADE Study in Patients with Familial Chylomicronemia Syndrome
- Plozasiran achieved statistically significant median reductions in triglycerides up to 80% and mean reductions in APOC3 up to 94% at month 10
- Plozasiran achieved a statistically significant reduction in incidence of acute pancreatitis versus placebo
- Plozasiran is the company’s first investigational RNAi-based therapy to show clinical efficacy in a Phase 3 study
- Arrowhead plans to highlight recent data for its cardiometabolic pipeline at its
“The strong results from the Phase 3 PALISADE study, evaluating plozasiran in patients with
The primary endpoint for the PALISADE study was placebo adjusted median change in triglycerides at Month 10. At that timepoint, patients treated with quarterly doses of 25 and 50 mg plozasiran achieved median triglyceride reductions of -80% and -78%, respectively, with a maximal reduction of -98%. At month 12, patients treated with 25 and 50 mg plozasiran achieved median triglyceride reductions of -78% and -73%, respectively, with a maximal reduction of -99%. These compared with median triglyceride reductions in placebo-treated patients of -17% at month 10 (primary endpoint, p<0.001) and -7% at month 12. Mean reductions in Apolipoprotein C-III (APOC3) at month 10 were -88% and -94% at 25 and 50 mg plozasiran, respectively.
In addition to meeting the primary endpoint, plozasiran met all key secondary endpoints and demonstrated statistical significance versus placebo.
There were 4 multiplicity-controlled key secondary endpoints: 1) percent change from baseline at Months 10 and 12 (averaged) in fasting triglycerides; 2) percent change from baseline at Month 10 in fasting APOC3; 3) percent change from baseline at Month 12 in fasting APOC3; 4) incidence of positively adjudicated events of acute pancreatitis during the randomized period.
Plozasiran demonstrated a favorable safety profile in the PALISADE study. The number of subjects reporting treatment emergent adverse events (AEs) were similar in plozasiran and placebo groups. Severe and serious AEs were less common with plozasiran than with placebo. The most common AEs reported were abdominal pain, COVID-19, nasopharyngitis, headache and nausea.
Arrowhead plans to highlight recent data for its cardiometabolic pipeline at its
About PALISADE Phase 3 Study
The PALISADE study (NCT05089084) is a Phase 3 placebo controlled study to evaluate the efficacy and safety of plozasiran in adults with genetically confirmed or clinically diagnosed
About Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome (FCS) is a severe and ultrarare genetic disease often caused by various monogenic mutations.
About Plozasiran
Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of Apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of APOC3, thereby reducing triglycerides and restoring lipids to more normal levels.
In multiple clinical studies, investigational plozasiran demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. Plozasiran has demonstrated a favorable safety profile to date with treatment emergent adverse events reported that reflect the comorbidities and underlying conditions of the study populations. Plozasiran is currently being investigated in the PALISADE Phase 3 clinical study in patients with
Plozasiran has been granted Orphan Drug Designation and Fast Track Designation by the
About Plozasiran EAP
Arrowhead is committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. The company has established an early access program (EAP) for some individuals living with
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