Arrowhead Pharmaceuticals has announced interim topline data from a phase 1/2a clinical trial of ARO-DIMER-PA, the company’s first investigational RNA interference (RNAi) therapeutic dimer being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia.

Chris Anzalone, PhD, President and CEO, Arrowhead Pharmaceuticals, said, “With these interim topline results, Arrowhead’s innovative and proprietary Targeted RNAi Molecule (TRiM) platform has achieved clinical validation of its ability to target and silence 2 genes simultaneously in 1 molecule. This represents an important step forward and a first for the field of RNAi therapeutics. We look forward to presenting additional details on this groundbreaking advance at an upcoming medical congress. This validation potentially expands the number of diseases and patients worldwide that may be helped by Arrowhead’s RNAi therapies. ARO-DIMER-PA also fits well strategically with Arrowhead’s growing focus on cardiometabolic diseases. It is complemented by our existing portfolio, which includes our first commercial product REDEMPLO (plozasiran), now approved in the US, European Union (EU), Canada, Australia, and China for the treatment of Familial Chylomicronemia Syndrome (FCS), and investigational zodasiran, which is currently being studied in the global phase 3 YOSEMITE clinical trial in patients with Homozygous Familial Hypercholesterolemia (HoFH).”

ARO-DIMER-PA is designed to silence expression of both Proprotein Convertase Subtilisin Kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes. Additional results will be submitted for presentation at an upcoming medical congress.

Steven Nissen, MD, Chief Academic Officer for the Heart and Vascular Institute, Cleveland Clinic, the Lewis and Patricia Dickey Chair in Cardiovascular Medicine and Professor of Medicine, Lerner College of Medicine, added, “Mixed hyperlipidemia is not adequately addressed by treating LDL-C alone. Even with intensive statin therapy and PCSK9 inhibitors, substantial ASCVD risk remains, and triglyceride-rich remnant lipoproteins may be an important part of that residual risk. This medication represents a new approach to targeting multiple lipid abnormalities with a single therapy.”