Arrowhead reports early data for dual-gene RNAi therapy ARO-DIMER-PA

Arrowhead Pharmaceuticals (NASDAQ: ARWR) released interim topline data from a Phase 1/2a clinical trial of ARO-DIMER-PA, an investigational RNA interference (RNAi) therapeutic being studied as a potential treatment for atherosclerotic cardiovascular disease due to mixed hyperlipidemia.

ARO-DIMER-PA is designed to silence two genes simultaneously — proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) — using a single RNAi molecule. According to the company, it is the first clinical candidate designed to selectively silence two genes with a single RNAi molecule.

In the single-dose portion of the trial, participants with mixed hyperlipidemia showed dose-dependent mean maximal reductions in serum PCSK9 of 72% and APOC3 of 88%. Additional lipid reductions included LDL-C of 54%, triglycerides of 73%, non-HDL cholesterol of 61%, and Apolipoprotein B of 50%.

Single-dose escalation has been completed through 400 mg. The most commonly reported treatment-emergent adverse events were injection site events and headaches. No drug-related serious adverse events were reported. The study is ongoing to assess safety and tolerability of multiple doses, with enrollment of up to 78 adult subjects.

Arrowhead said additional results will be submitted for presentation at an upcoming medical congress. The company also noted that ARO-DIMER-PA complements its existing cardiometabolic portfolio, which includes REDEMPLO (plozasiran), approved in the U.S., European Union, Canada, Australia, and China for familial chylomicronemia syndrome, and investigational zodasiran, currently in a global Phase 3 trial for homozygous familial hypercholesterolemia.

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