Arrowhead reports phase 1/2a data for dual-gene RNAi therapy

PASADENA, Calif. - Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) disclosed interim topline results from a Phase 1/2a clinical trial of ARO-DIMER-PA, an experimental RNA interference therapy aimed at silencing two genes at once for mixed hyperlipidemia-related atherosclerotic cardiovascular disease. The study showed significant reductions in serum PCSK9 (72% decrease) and APOC3 (88% decrease) in participants with mixed hyperlipidemia.

By silencing both genes, ARO-DIMER-PA led to substantial mean reductions in LDL-C (54%), triglycerides (73%), non-HDL cholesterol (61%), and Apolipoprotein B levels. This marks the first clinical validation of targeting and silencing two genes simultaneously with a single RNAi molecule. The single dose escalation reached 400 mg, with the most frequently reported adverse events being injection site issues and headaches.

No serious drug-related adverse events were observed. The trial will continue to evaluate the safety and tolerability of various doses. ARO-DIMER-PA is the first in its class to specifically target two genes simultaneously using RNAi technology, addressing the major risk factor for atherosclerotic cardiovascular disease.