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

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

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.

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Source: Arrowhead Pharmaceuticals, Inc.

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