What's Happening?
Arrowhead Pharmaceuticals has filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-DIMER-PA, an investigational RNA interference (RNAi) therapeutic. This therapeutic is designed to silence the expression of two genes, PCSK9 and APOC3, using Arrowhead's proprietary TRiMTM technology. The trial aims to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on LDL-C and triglycerides in patients with mixed hyperlipidemia. Mixed hyperlipidemia is characterized by elevated LDL-C and triglyceride levels, posing a significant risk for atherosclerotic cardiovascular disease (ASCVD). Arrowhead's approach represents a novel advancement in RNAi therapeutics, targeting two genes simultaneously to potentially reduce residual ASCVD risk.
Why It's Important?
The development of ARO-DIMER-PA is significant as it addresses the substantial residual risk of ASCVD in patients with mixed hyperlipidemia, despite existing LDL-C-lowering therapies. Arrowhead's innovative approach could lead to more effective treatments for cardiovascular diseases, which are a leading cause of mortality worldwide. The success of this trial could reinforce Arrowhead's leadership in the RNAi field and expand its commercial focus on cardiometabolic therapeutics. This advancement may also influence the pharmaceutical industry's approach to treating complex lipid disorders, potentially improving patient outcomes and reducing healthcare costs associated with cardiovascular diseases.
What's Next?
Pending regulatory clearance, Arrowhead plans to proceed with a Phase 1/2a clinical trial in New Zealand. The trial will be randomized, double-blind, placebo-controlled, and dose-escalating, involving up to 78 adult subjects with mixed hyperlipidemia. The study will provide insights into the potential of ARO-DIMER-PA, with results expected in 2026. Arrowhead is also anticipating a PDUFA date for plozasiran, its first potential commercial product, and is conducting a Phase 3 trial for zodasiran in homozygous familial hypercholesterolemia. These developments align with Arrowhead's strategic focus on RNAi therapeutics in the cardiometabolic area.
