Arrowhead Pharmaceuticals to Present Interim Phase 2 Clinical Data at AHA 2022 on SHASTA-2 Study of ARO-APOC3 and ARCHES-2 Study of ARO-ANG3

Rhea-AI Summary

Arrowhead Pharmaceuticals (NASDAQ: ARWR) will present interim clinical data on ARO-APOC3 and ARO-ANG3 at the American Heart Association Scientific Sessions 2022 in Chicago, from November 5-7, 2022. The SHASTA-2 and ARCHES-2 Phase 2 studies focus on patients with severe hypertriglyceridemia and mixed dyslipidemia, respectively. A virtual investor event is scheduled for November 9, 2022, to discuss these findings and future developments. Full data from ARCHES-2 is expected in the first half of 2023.

Positive

• Presentation of interim clinical data for ARO-APOC3 and ARO-ANG3 at AHA Scientific Sessions 2022.
• Interest from key opinion leaders in the potential of ARO-APOC3 and ARO-ANG3 to address lipid-related cardiovascular risks.
• Anticipation of full study results from ARCHES-2 in the first half of 2023.

Negative

• None.

- Company will host a virtual analyst and investor event on November 9, 2022

PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it will present interim clinical data from the ongoing SHASTA-2 Phase 2 study of ARO-APOC3 in patients with severe hypertriglyceridemia and the ongoing ARCHES-2 Phase 2 clinical study of ARO-ANG3 in patients with mixed dyslipidemia, in late-breaking oral presentations at the American Heart Association (AHA) Scientific Sessions 2022, being held in Chicago on November 5–7, 2022. The company will also host a virtual analyst and investor event on November 9, 2022, at 10:00 am ET to discuss these data and Arrowhead’s plans for future clinical development.

The analyst and investor event will feature presentations from key opinion leaders, Christie M. Ballantyne, M.D., (Baylor College of Medicine) and Robert S. Rosenson, M.D. (Icahn School of Medicine at Mount Sinai), who will discuss the data presented at AHA, the current treatment landscape for various lipid disorders with unmet medical need, and the potential of ARO-APOC3 and ARO-ANG3 to address lipids and lipoproteins that may contribute to the substantial residual risk of cardiovascular disease that remains even after low density lipoprotein cholesterol (LDL-C) is mostly well controlled with existing therapies. Details about the AHA presentations are listed below.

American Heart Association (AHA) Scientific Sessions 2022 – November 5-7, 2022

Title: ARO-ANG3, an Investigational RNAi Therapeutic, Decreases Serum Angiopoietin-Like Protein 3, Triglycerides, and Cholesterol in Patients With Mixed Dyslipidemia
Date/Time: November 7, 2022, 3:00 p.m. CST
Presenter: Robert S. Rosenson
Session: The Present and Future of Lipid Lowering

Title: ARO-APOC3, an Investigational RNAi Therapeutic, Decreases Serum Apolipoprotein C3, Triglyceride, and Non-HDL-C Concentrations While Increasing HDL-C in Patients With Severe Hypertriglyceridemia
Date/Time: November 7, 2022, 3:08 p.m. CST
Presenter: Christie M. Ballantyne
Session: The Present and Future of Lipid Lowering

ARO-ANG3 is the company’s investigational RNA interference (RNAi) therapeutic designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase, being developed as a treatment for patients with mixed dyslipidemia. In the Phase 2 ARCHES-2 clinical study (NCT04832971), eligible subjects (n=203) were randomized 3:1 to receive subcutaneous injections of 50, 100, or 200 mg ARO-ANG3 or placebo on day 1 and at week 12. Subjects were on a stable diet and optimal statin/lipid-lowering therapies. In subjects with hepatic steatosis, liver fat was assessed at baseline and week 24 by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF). The primary endpoint is the percent change from baseline in fasting triglycerides (TGs) at week 24. This interim analysis (data cutoff July 6, 2022) evaluated data when all subjects reached week 12. Serum lipid and lipoprotein levels, including LDL-C, were determined at week 16 after most subjects had received both doses.

ARO-APOC3 is the company’s investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3) being developed as a treatment for patients with hypertriglyceridemia (HTG), severe hypertriglyceridemia (sHTG), and familial chylomicronemia syndrome (FCS). In the Phase 2 SHASTA-2 clinical study (NCT04720534), eligible subjects (n=175/216 planned subjects) were randomized 3:1 to receive subcutaneous injections of 10, 25, or 50 mg ARO-APOC3 or placebo on day 1 and at week 12. Patients with familial chylomicronemia syndrome were excluded. The primary endpoint is percent change from baseline in fasting TGs at week 24. This interim analysis (data cutoff July 25, 2022) evaluated data when greater than 50% of subjects had reached week 12 and received both doses. Serum lipid, lipoprotein, and apolipoprotein levels were measured at week 16, four weeks after the second dose of ARO-APOC3 or placebo.

ARCHES-2 is fully enrolled and complete study data are expected in the first half of 2023. SHASTA-2 and MUIR, the Phase 2 study of ARO-APOC3 in patients with mixed dyslipidemia, are also both fully enrolled and complete study results are anticipated in the second half of 2023.

A copy of the presentation materials and a webcast link for the analyst and investor event will be available on the Events and Presentations page under the Investors section of the Arrowhead website.

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.

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

Safe Harbor Statement under the Private Securities Litigation Reform Act:

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,” or “continue” 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 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/20221011005313/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

www.lifesciadvisors.com

Media:

LifeSci Communications, LLC

Josephine Belluardo, Ph.D.

646-751-4361

jo@lifescicomms.com

www.lifescicommunications.com

Source: Arrowhead Pharmaceuticals, Inc.

FAQ

What are the upcoming events for Arrowhead Pharmaceuticals related to ARO-APOC3 and ARO-ANG3?

Arrowhead Pharmaceuticals will present interim clinical data at the American Heart Association Scientific Sessions from November 5-7, 2022, and will host a virtual analyst event on November 9, 2022, to discuss these findings.

What studies are being presented by Arrowhead Pharmaceuticals at the AHA 2022?

The SHASTA-2 Phase 2 study for ARO-APOC3 and the ARCHES-2 Phase 2 study for ARO-ANG3 will be presented.

When can we expect full results from the ARCHES-2 study?

Full results from the ARCHES-2 study are expected in the first half of 2023.

What is the focus of the ARO-APOC3 and ARO-ANG3 studies?

ARO-APOC3 targets severe hypertriglyceridemia, while ARO-ANG3 targets mixed dyslipidemia, both aiming to address lipid disorders linked to cardiovascular disease.