Arrowhead Pharmaceuticals Eyes SHTG Filing as RNAi Pipeline Expands

Arrowhead Pharmaceuticals Eyes SHTG Filing as RNAi Pipeline Expands Written by MarketBeat September 18, 2026 Add As Preferred Source Share Share Share This Article Link copied to clipboard. Close Image from MarketBeat Media, LLC.

Key Points Arrowhead plans to file for SHTG approval by year-end. The company expects a potential second-quarter 2027 launch of REDEMPLO (plozasiran) for severe hypertriglyceridemia, supported by approximately 80% triglyceride reductions and substantial reductions in acute pancreatitis risk. REDEMPLO’s initial launch for familial chylomicronemia syndrome is progressing, with Arrowhead expanding its sales force and estimating a potential $3 billion–$4 billion U.S. peak annual market for plozasiran. Arrowhead is broadening its RNAi pipeline beyond liver diseases, advancing programs in cholesterol, obesity and CNS disorders. Key upcoming milestones include zodasiran data in homozygous familial hypercholesterolemia, additional obesity data and early clinical results for the Alzheimer’s candidate ARO-MAPT. MarketBeat previews top five stocks to own in October . Want Diversified Upside in Biotechnology? Check out LABU

Arrowhead Pharmaceuticals NASDAQ: ARWR is expanding its commercial and clinical footprint around RNA interference therapies, with its recently launched triglyceride-lowering treatment REDEMPLO and several pipeline programs targeting cardiometabolic disease, obesity and central nervous system disorders. Speaking at H.C. Wainwright's 28th Annual Global Investment Conference, Chief Executive Officer Chris Anzalone said the company has evolved from its initial focus on liver-targeted RNAi therapies into a broader platform company. Arrowhead now has approximately 21 or 22 drug candidates in clinical studies, he said, with clinical candidates representing five different cell types. Get ARWR alerts: Sign Up “The first tissue that we could address is the liver, hepatocytes,” Anzalone said. “But we decided early on that in order to extract proper value from this technology and to treat as many patients as we could, we needed to get outside the liver.”

REDEMPLO Launch and Severe Hypertriglyceridemia Filing REDEMPLO, also known as plozasiran, is an APOC3 inhibitor designed to lower triglycerides. The drug is approved for familial chylomicronemia syndrome, or FCS, an ultra-orphan condition. Anzalone said the launch has progressed well, although contracting with payers required several quarters. The company is now able to serve patients with both genetic FCS and what it describes as clinically defined FCS. Anzalone estimated there could be 15,000 to 20,000 clinically defined FCS patients. Arrowhead has expanded its commercial organization from an initial sales force of roughly 20 representatives and expects another phase of sales-force growth in January and February as it prepares for a potential severe hypertriglyceridemia, or SHTG, launch in the second quarter of 2027. Arrowhead plans to submit a supplemental new drug application for plozasiran in SHTG by year-end. The company acquired a priority review voucher, which Anzalone said could reduce review timing by four months. Chief Medical Officer and Head of R&D James Hamilton said results from the Phase 3 SHASTA-3 and SHASTA-4 trials showed about an 80% reduction from baseline in triglycerides among patients with severe hypertriglyceridemia, defined in the studies as triglycerides above 500. The triglyceride reduction was associated with statistically significant reductions in acute pancreatitis, with risk reductions ranging from 78% to more than 80%, depending on the population, he said. In a subset of patients with triglycerides above 880 and a history of pancreatitis, Hamilton said the pooled SHASTA-3 and SHASTA-4 data showed a 100% risk reduction, with no events among patients receiving active treatment. Hamilton also described the safety profile as “pretty clean,” citing no evidence of thrombocytopenia or hypersensitivity reactions and no clinically...