What's Happening?
Arrowhead Pharmaceuticals has filed a request for regulatory clearance to begin a Phase 1/2a clinical trial of ARO-DIMER-PA, an investigational RNA interference (RNAi) therapeutic. This drug is designed to treat atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia by silencing the expression of two genes, PCSK9 and APOC3, using Arrowhead's proprietary TRiMTM technology. The trial aims to evaluate the safety, tolerability, and effects on LDL-C and triglycerides in patients with mixed hyperlipidemia. This development marks a significant step in RNAi therapeutics, as ARO-DIMER-PA is the first clinical candidate to target two genes simultaneously with a single molecule.
Why It's Important?
The advancement of ARO-DIMER-PA could have significant implications for the treatment of cardiovascular diseases, particularly ASCVD, which is a leading cause of mortality worldwide. By targeting both PCSK9 and APOC3, the drug has the potential to address the substantial residual risk in patients with mixed hyperlipidemia, even after LDL-C-lowering therapies. This could lead to a reduction in healthcare costs and improve patient outcomes. Arrowhead's progress in RNAi therapeutics reinforces its leadership in the field and could pave the way for more innovative treatments targeting multiple genes.
What's Next?
Pending regulatory clearance, Arrowhead plans to proceed with the clinical trial, which will be conducted in New Zealand. The trial will be a randomized, double-blind, placebo-controlled study involving up to 78 adult subjects. The results of this trial could provide important insights into the potential of ARO-DIMER-PA and influence future regulatory and commercial strategies. Arrowhead is also preparing for a PDUFA date for another product, plozasiran, and is conducting a Phase 3 trial for zodasiran, indicating a strategic focus on expanding its RNAi therapeutic portfolio.
