Arrowhead reports RNAi trial results

• Arrowhead Pharmaceuticals reported interim Phase 1/2a results for its ARO-DIMER-PA RNAi therapy candidate.
• The treatment reduced PCSK9 by 72%, APOC3 by 88%, and LDL-C by 54% in study participants.
• The company is evaluating the therapy as a potential treatment for mixed hyperlipidemia linked to cardiovascular disease.

Arrowhead Pharmaceuticals (NASDAQ:ARWR) reported interim topline data from a Phase 1/2a clinical trial of ARO-DIMER-PA, an investigational RNA interference therapy designed to silence PCSK9 and APOC3 genes for patients with mixed hyperlipidemia.

The single-dose study showed dose-dependent reductions in PCSK9 and APOC3, with mean maximum reductions of 72% and 88%, respectively, among participants with mixed hyperlipidemia.

Arrowhead reported that ARO-DIMER-PA produced mean maximum reductions of 54% in LDL-C, 73% in triglycerides, 61% in non-HDL cholesterol, and 50% in apolipoprotein B, while no drug-related serious adverse events were reported.

Arrowhead said the study is continuing to evaluate safety and tolerability of multiple doses, with additional results planned for presentation at a future medical congress.

Arrowhead develops RNA interference medicines using its Targeted RNAi Molecule platform, which is designed to silence specific genes involved in disease pathways.

The company’s cardiometabolic pipeline includes REDEMPLO, an approved treatment for familial chylomicronemia syndrome, and investigational zodasiran, which is being studied in a Phase 3 trial for homozygous familial hypercholesterolemia.