Arrowhead to Present Phase 3 PALISADE Data on Plozasiran at ESC 2024

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has announced that it will present new Phase 3 clinical data from the PALISADE study of plozasiran (ARO-APOC3) at the European Society of Cardiology (ESC) Congress 2024. This event will be held in London from August 30 through September 2, 2024. Additionally, Arrowhead will organize a virtual analyst and investor event on September 3, 2024, at 8:00 am EDT to discuss the findings further.

The ESC presentation will focus on the efficacy and safety of ARO-APOC3 in adults with familial chylomicronemia syndrome (FCS). The presentation is scheduled for September 2, 2024, at 11:36 am BST, and will be delivered by Professor Gerald Watts from the University of Western Australia. Professor Watts is a distinguished expert in cardio-metabolic medicine and has extensive experience in lipid disorders and cardiovascular prevention.

The PALISADE Phase 3 study (NCT05089084) is a placebo-controlled trial that assesses the efficacy and safety of plozasiran in adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint of the study is the percent change from baseline in fasting triglycerides (TG) versus placebo at Month 10. A total of 75 participants across 39 sites in 18 countries were randomized to receive either 25 mg or 50 mg of plozasiran, or a matching placebo, once every three months. Participants who completed the randomized period were eligible to enter a 2-part extension period, where all participants received plozasiran.

Familial chylomicronemia syndrome (FCS) is a severe and ultra-rare genetic disease primarily caused by various monogenic mutations. This condition leads to extremely high levels of triglycerides, typically above 880 mg/dL, resulting in severe complications such as acute pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options available to treat FCS adequately.

Plozasiran, also known as ARO-APOC3, is an investigational RNA interference (RNAi) therapeutic designed to reduce the production of Apolipoprotein C-III (APOC3), a component of triglyceride-rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels by inhibiting the breakdown of TRLs and the uptake of TRL remnants by liver receptors. By reducing APOC3 levels, plozasiran aims to lower triglycerides and normalize lipid levels.

In various clinical studies, plozasiran has shown significant reductions in triglycerides and multiple atherogenic lipoproteins in patients with FCS, severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. The therapeutic has demonstrated a favorable safety profile, with treatment-emergent adverse events reflecting the comorbidities and underlying conditions of the study populations. Plozasiran is currently being investigated in the PALISADE Phase 3 clinical study for FCS, as well as the SHASTA-3 and SHASTA-4 Phase 3 studies for SHTG.

Plozasiran has been granted Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration and Orphan Drug Designation by the European Medicines Agency.

Arrowhead Pharmaceuticals is dedicated to the rapid and efficient development of investigational medicines for serious diseases. The company has initiated an early access program (EAP) for patients with FCS. This program provides access to investigational plozasiran for certain individuals, though its efficacy and safety are yet to be fully established. Patients and caregivers interested in the EAP should consult their treating physicians for more information.

Arrowhead Pharmaceuticals specializes in creating medicines that target and silence the genes causing intractable diseases. The company's RNAi-based therapeutics leverage the natural RNA interference mechanism to inhibit the expression of specific genes, thereby reducing the production of disease-causing proteins.

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