• Company hosting a virtual analyst and investor event on September 3, 2024, at 8:00 am EDT to discuss results

PASADENA, Calif. / Aug 21, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today 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, being held in London August 30 through September 2, 2024. The company will also host a virtual analyst and investor event on September 3, 2024, at 8:00 am EDT.

Details about the ESC presentation are listed below.

European Society of Cardiology Congress 2024 – August 30 - September 2, 2024

Title: A phase 3 study to evaluate the efficacy and safety of ARO-APOC3 in adults with familial chylomicronemia syndrome
Date/Time: September 2, 2024, 11:36 am BST
Presenter: Professor Gerald Watts, University of Western Australia
Session: Small trials, trial updates, and other studies on lipid therapy
Session Type: Late Breaking Science

The analyst and investor event on September 3, 2024, at 8:00 am EDT will feature an encore presentation of the ESC 2024 data by Professor Watts, and will include discussion by Arrowhead management. To register for the event, please visit: https://lifescievents.com/event/arrowheadpharma/. The live event and an archived webcast may also be accessed on the Events and Presentations page in the Investors section of the Arrowhead website.

Expert Bio

Gerald F Watts, DSc, DM, PhD, FRCP, FRACP, FCSANZ, is Winthrop Professor of Cardio-metabolic Medicine in the University of Western Australia. Dr. Watts trained at Imperial & King’s College, London University, and was a scholar at Wolfson College, Oxford University. He is a senior consultant physician, specializing in the rapidly developing field of cardio-metabolic medicine, and current chair of the Familial Hypercholesterolemia Australasia Network. He leads the Cardio-metabolic Service in the Departments of Cardiology and Internal Medicine at Royal Perth Hospital and is Winthrop Professor of Cardio-metabolic Medicine in the University of Western Australia. Research interests include fundamental and applied aspects of lipid disorders and cardiovascular prevention and improving healthcare delivery for patients with high-risk dyslipidemias, in particular familial hypercholesterolemia and hyperchylomicronemia. He has supervised several Masters, MD and PhD students and post-doctoral research fellows, and holds several research grants, with multiple collaborations.

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 FCS. The primary endpoint of the study is percent change from baseline in fasting TG versus placebo at Month 10. A total of 75 subjects distributed across 39 different sites in 18 countries were randomized to receive 25 mg plozasiran, 50 mg plozasiran, or matching placebo once every three months. Participants who completed the randomized period were eligible to continue in a 2-part extension period, where all participants receive plozasiran.

About Familial Chylomicronemia Syndrome

Familial chylomicronemia syndrome (FCS) is a severe and ultrarare genetic disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, the therapeutic options that can adequately treat FCS are limited.

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 FCS, which recently completed, and the Phase 3 SHASTA-3 and SHASTA-4 studies in patients with 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.

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 FCS. As with any investigational medicine that has not been approved by regulatory authorities, investigational plozasiran may or may not be effective in treating your diagnosis or condition, and there may be risks associated with its use. If you are a patient or caregiver wishing to know more about this plozasiran EAP for FCS, please discuss this EAP and all treatment options with your treating physician. If you are a treating physician and are seeking information about the plozasiran EAP or would like to request access for a patient, please contact This email address is being protected from spambots. You need JavaScript enabled to view it..

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

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