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

Rhea-AI Summary

Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced the presentation of new Phase 3 clinical data from the PALISADE study of plozasiran (ARO-APOC3) at the European Society of Cardiology (ESC) Congress 2024 in London. The presentation, titled 'A phase 3 study to evaluate the efficacy and safety of ARO-APOC3 in adults with familial chylomicronemia syndrome,' will be given by Professor Gerald Watts on September 2, 2024. Arrowhead will host a virtual analyst and investor event on September 3, 2024, featuring an encore presentation of the ESC data and discussion by company management.

Positive

• Presentation of Phase 3 clinical data at a major cardiology conference
• Hosting a virtual analyst and investor event to discuss results

Negative

• None.

Insights

Medical Research Analyst

The upcoming presentation of Phase 3 data for plozasiran (ARO-APOC3) in Familial Chylomicronemia Syndrome (FCS) at ESC 2024 is a significant milestone for Arrowhead Pharmaceuticals. FCS is a rare genetic disorder causing severe hypertriglyceridemia, with treatment options. Plozasiran, targeting APOC3, could potentially offer a novel therapeutic approach.

Key points to watch for in the data presentation:

• Efficacy in reducing triglyceride levels
• Safety profile and tolerability
• Durability of effect
• Quality of life improvements for patients

If positive, these results could position Arrowhead as a leader in FCS treatment, potentially expanding their rare disease portfolio. However, investors should also consider the market size for FCS and potential regulatory hurdles ahead.

Financial Analyst

Arrowhead's strategic decision to present Phase 3 data at a major cardiology conference like ESC 2024 suggests confidence in their results. This exposure could significantly impact the company's market valuation and investor sentiment.

Key financial considerations:

• Potential for accelerated regulatory pathway given FCS's rare disease status
• Market opportunity in rare lipid disorders
• Possible partnerships or licensing deals if data is compelling
• Impact on Arrowhead's cash runway and R&D expenses

Investors should closely monitor the data presentation and subsequent analyst event for insights into Arrowhead's commercialization strategy and financial projections. Positive results could drive stock price appreciation, while disappointing data might lead to a significant downturn.

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

PASADENA, Calif.--(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 EAP@arrowheadpharma.com.

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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This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240821261066/en/

Arrowhead Pharmaceuticals, Inc.

Vince Anzalone, CFA

626-304-3400

ir@arrowheadpharma.com

Investors:

LifeSci Advisors, LLC

Brian Ritchie

212-915-2578

britchie@lifesciadvisors.com

Media:

LifeSci Communications, LLC

Kendy Guarinoni, Ph.D.

724-910-9389

kguarinoni@lifescicomms.com

Source: Arrowhead Pharmaceuticals, Inc.

FAQ

What is the PALISADE study that Arrowhead Pharmaceuticals (ARWR) is presenting at ESC 2024?

The PALISADE study is a Phase 3 clinical trial evaluating the efficacy and safety of plozasiran (ARO-APOC3) in adults with familial chylomicronemia syndrome.

When and where will Arrowhead Pharmaceuticals (ARWR) present the PALISADE study results?

Arrowhead will present the results at the European Society of Cardiology (ESC) Congress 2024 in London on September 2, 2024, at 11:36 am BST.

Is Arrowhead Pharmaceuticals (ARWR) hosting an event to discuss the PALISADE study results?

Yes, Arrowhead is hosting a virtual analyst and investor event on September 3, 2024, at 8:00 am EDT to discuss the PALISADE study results.

Who will be presenting the PALISADE study results for Arrowhead Pharmaceuticals (ARWR)?

Professor Gerald Watts from the University of Western Australia will be presenting the PALISADE study results at ESC 2024 and the virtual event.