Arrowhead Presents Interim Data from ARO-ANG3 Phase 2 GATEWAY Study in Patients with HoFH - Seite 2

Homozygous familial hypercholesterolemia (HoFH) is the most serious and rare form of familial hypercholesterolemia, which if left untreated leads to early clinical manifestations of coronary artery disease. Most cases of HoFH are due to mutations in the low-density lipoprotein receptor (LDLR) gene coding for the LDL receptor. Thus, patients with HoFH due to dysfunctional or absent LDLR can be resistant to standards of care such as statins and even resistant to alternatives such as PCSK9 inhibitors. Patients with HoFH are therefore a population with a particularly high need for additional therapy with a mechanism working outside of the LDL receptor, such as therapeutic ANGPTL3 inhibition.

About the GATEWAY Phase 2 Study

The AROANG3-2003 GATEWAY study (NCT05217667) is a Phase 2 open-label study to evaluate the efficacy, safety, and tolerability of 200 mg and 300 mg of ARO-ANG3 administered once every 12 weeks in 18 patients with HoFH. The initial treatment period is up to 36 weeks with an optional 24-month extension treatment period. The primary endpoint of the study is the percent change from baseline in LDL-cholesterol.

About ARO-ANG3

ARO-ANG3 is an investigational RNAi therapeutic designed to reduce expression of angiopoietin-like protein 3 (ANGPTL3), a hepatocyte expressed regulator of lipid and lipoprotein metabolism with multiple potential modes of action, including inhibition of lipoprotein lipase and endothelial lipase. Given the inhibitory role of ANGPTL3 in the metabolism of various lipoproteins and triglycerides, reduced expression and reduced circulating levels of ANGPTL3 may increase clearance of LDL-cholesterol, HDL-cholesterol, and triglycerides.

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.