Arrowhead Pharmaceuticals has dosed the first participants in its Phase I/IIa clinical trial of ARO-DIMER-PA, an investigational dual functional RNA interference (RNAi) therapy, for mixed hyperlipidaemia.
The placebo-controlled dose-escalating ARO-DIMER-PA-1001 study aims to assess the therapy as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) associated with this condition.
It will assess the tolerability, pharmacokinetics, safety, pharmacodynamics, and effects on low-density lipoprotein cholesterol (LDL-C) and triglycerides (TGs) of single-dose and multiple doses involving up to 78 adult participants with mixed hyperlipidaemia.
ARO-DIMER-PA is designed to silence both proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes simultaneously.
Mixed hyperlipidaemia is characterised by increased levels of LDL-C and TGs and remains major risk factor for ASCVD.
The initiation of this clinical study represents Arrowhead’s continued expansion in cardiometabolic RNAi therapeutics.
Arrowhead president and CEO Chris Anzalone said: “Arrowhead is at the forefront of innovation in the RNAi field, and we’re proud of the versatile capabilities of our TRiM platform, now including the first-ever clinical candidate that can potentially silence expression of two genes in one RNAi molecule.
“ARO-DIMER-PA is designed to silence both the PCSK9 and APOC3 genes, which together have substantial clinical validation as important targets for reducing LDL-cholesterol, triglycerides, and total atherogenic lipoproteins.
“We see ARO-DIMER-PA as having the potential to reduce the risk of ASCVD for people living with mixed hyperlipidaemia, and we are excited to see what this study may reveal about the possibility of creating other dual-functional RNAi molecules for potentially treating complex genetic diseases.”
Earlier this month, Arrowhead’s investigational RNAi obesity candidate, when combined with Eli Lilly’s Zepbound (tirzepatide), demonstrated benefits according to interim results from a Phase I/IIa trial.
