Arrowhead files for approval of dual-target RNAi therapy for heart disease

PASADENA, Calif. - Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR), a $5.2 billion market cap biotechnology company whose stock has surged over 265% in the past six months, has filed for regulatory clearance to begin a Phase 1/2a clinical trial of ARO-DIMER-PA, its investigational RNA interference (RNAi) therapeutic for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia, according to a company press release. InvestingPro analysis shows the company maintains a GOOD overall financial health score, with 14+ additional insights available to subscribers.

The experimental therapy represents a significant advancement in RNAi technology as the first clinical candidate designed to target two genes simultaneously with a single molecule. ARO-DIMER-PA aims to silence the expression of both proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes using Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform. The company’s strong liquidity position, with a current ratio of 4.87, suggests robust financial flexibility to support its research initiatives.

The application was submitted to New Zealand’s Medicines and Medical Devices Safety Authority. If approved, the company plans to conduct a randomized, double-blind, placebo-controlled study evaluating single and multiple doses in up to 78 adult patients with mixed hyperlipidemia.

Mixed hyperlipidemia, characterized by elevated low-density lipoprotein cholesterol (LDL-C) and triglyceride levels, is a major risk factor for ASCVD, the leading cause of mortality worldwide.

In preclinical studies, ARO-DIMER-PA demonstrated the ability to lower serum PCSK9 and APOC3, while reducing non-HDL-cholesterol, LDL-cholesterol, and triglycerides in dyslipidemic nonhuman primates.

"ARO-DIMER-PA silences the PCSK9 and APOC3 genes, which both together have substantial clinical validation as important targets for reducing LDL-cholesterol, triglycerides, and total atherogenic lipoproteins," said Chris Anzalone, President and CEO at Arrowhead.

The company expects the clinical study may provide insights into ARO-DIMER-PA’s potential by 2026. This development aligns with Arrowhead’s expanding cardiometabolic portfolio, which includes plozasiran with a PDUFA date of November 18, 2025, and zodasiran, currently in Phase 3 trials for homozygous familial hypercholesterolemia. With analysts forecasting revenue growth of over 200% for the current fiscal year, investors seeking detailed analysis can access the comprehensive Pro Research Report available exclusively on InvestingPro, which covers this and 1,400+ other top US stocks with expert insights and actionable intelligence.

In other recent news, Arrowhead Pharmaceuticals has filed for regulatory clearance to initiate a Phase 1/2a clinical trial for its investigational drug ARO-MAPT, aimed at treating Alzheimer’s disease. The application was submitted to New Zealand’s Medicines and Medical Devices Safety Authority and includes plans to evaluate the drug in 64 healthy subjects and 48 individuals with early Alzheimer’s. Additionally, Arrowhead has entered into a significant collaboration with Novartis, securing a $200 million upfront payment for a global licensing agreement on their siRNA therapy targeting Parkinson’s Disease. This agreement could potentially bring Arrowhead up to $2 billion in milestone payments and royalties on commercial sales.

In other developments, H.C. Wainwright has reaffirmed its Buy rating on Arrowhead Pharmaceuticals, maintaining a price target of $80.00. This follows promising data from Ionis Pharmaceuticals on a related therapy, which H.C. Wainwright views positively for Arrowhead. Meanwhile, Sarepta Therapeutics has sold over 9.2 million shares of Arrowhead, generating at least $174 million in gross proceeds. As part of a milestone payment obligation, Sarepta will also transfer approximately 2.66 million Arrowhead shares back to Arrowhead, representing a $50 million stock buyback. Arrowhead plans to place these shares into its treasury, effectively reducing the number of outstanding shares.

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