Arrowhead Pharmaceuticals (NASDAQ:ARWR) reported positive Phase III results for plozasiran in severe hypertriglyceridemia (SHTG), accelerating plans for a supplemental New Drug Application and highlighting continued growth in the launch of its REDEMPLO therapy for familial chylomicronemia syndrome (FCS).
For the fiscal third quarter ended June 30, 2026, Arrowhead recorded a net loss of $194.3 million, or $1.36 per diluted share, compared with a loss of $175.2 million, or $1.26 per share, a year earlier. Revenue rose to approximately $75 million from $28 million in the prior-year period, driven by collaboration agreements and commercial REDEMPLO sales.
Phase III SHTG Results and Regulatory Plans
Median triglyceride reductions from baseline were 79% in SHASTA-3 and 81% in SHASTA-4. In the studies’ placebo groups, reductions were approximately 27%, according to Andy Davis, senior vice president and head of the Global Cardiometabolic Franchise.
Arrowhead also reported a statistically significant reduction in acute pancreatitis events in a prespecified pooled analysis of the trials. Davis said plozasiran reduced cumulative acute pancreatitis events by 78% versus placebo across the broader SHTG population. In a subgroup of patients with triglyceride levels above 880 milligrams per deciliter and a prior history of acute pancreatitis, the company reported a 100% reduction in events versus placebo.
The company said safety and tolerability findings were consistent with prior plozasiran studies. Anzalone said Arrowhead observed no new safety signals, no clinically meaningful adverse changes in liver enzymes, no hypersensitivity cases and no thrombocytopenia signal. In a prespecified MRI-PDFF subgroup, there was no statistically significant difference in mean liver fat content between plozasiran and placebo.
Detailed SHASTA results are scheduled for presentation at the European Society of Cardiology Congress on Aug. 30, followed by an Arrowhead webcast on Aug. 31. Chief Medical Officer and Head of R&D Dr. James Hamilton said the company remained under embargo regarding further trial details ahead of the conference.
Arrowhead intends to submit an sNDA to the Food and Drug Administration before the end of 2026. The company acquired an FDA priority review voucher that could shorten the agency’s review period from 10 months to six months. Chief Financial Officer Dan Apel said Arrowhead expects to pay $215 million for the voucher during its fiscal fourth quarter, following Hart-Scott-Rodino clearance.
Management said the initial focus of a potential SHTG launch would likely be patients at the highest risk, while emphasizing that the clinical data support treatment across patients with triglyceride levels above 500 milligrams per deciliter. Anzalone said the company expects SHTG to be an education-driven market because physicians have historically had limited options for substantially reducing triglycerides.
REDEMPLO Launch Expands in FCS
REDEMPLO prescription volume more than doubled during the fiscal third quarter and that momentum continued into the current quarter, Davis said. Arrowhead has supported more than 400 unique prescribers, led by preventive cardiologists and endocrinologists.
Davis said the company was receiving approximately 20 to 30 new prescriptions per week, consistent with its previously disclosed run rate. Arrowhead is working to improve the progression of prescriptions through prior authorization and appeals processes and expects an additional wave of field personnel to enter the market during August.
REDEMPLO has favorable policies in place with the most significant U.S. payers, according to Davis, and the company expects remaining coverage gaps to narrow in the coming months. Nearly all published payer policies allow clinicians to diagnose FCS through clinical criteria rather than genetic confirmation alone.
The therapy’s U.S. wholesale acquisition cost remains $45,000 per patient annually. Management said it does not intend to change the price following the SHASTA results, citing REDEMPLO’s efficacy, safety profile, quarterly dosing schedule and 25-milligram fixed dose.
REDEMPLO is approved for FCS in the United States, Canada, China, Australia and the European Union. Arrowhead said reimbursement processes in Europe will proceed country by country over approximately 12 months, beginning with Germany. Sanofi leads commercialization in Greater China.
Pipeline Readouts Ahead
Arrowhead expects several clinical data releases before year-end. The company plans to report top-line Phase I data for ARO-DIMER-PA in September. The candidate is designed to silence both APOC3 and PCSK9 to reduce LDL cholesterol and triglycerides in patients with mixed hyperlipidemia.
The company also expects September data from its Phase I healthy-volunteer study of ARO-MAPT, a subcutaneously administered RNA interference therapy targeting tau. Hamilton said the release will focus on safety and total tau knockdown, with Arrowhead targeting approximately 50% to 60% knockdown. The study’s second phase in Alzheimer’s disease patients is actively enrolling.
For obesity and metabolic dysfunction-associated steatohepatitis, Arrowhead plans to provide an update primarily focused on ARO-ALK7 in the fourth quarter. It has submitted an ARO-INHBE Phase IIb protocol to regulators. Earlier data showed a placebo-adjusted 44% reduction in liver fat in a small subgroup of patients with obesity and elevated baseline liver fat receiving at least a 200-milligram dose as monotherapy.
Separately, Arrowhead completed enrollment in the Phase III YOSEMITE trial of its therapy for homozygous familial hypercholesterolemia, enrolling 70 patients compared with a planned 60. The company expects study completion in mid-2027 and data in the second half of 2027.
Financial Position and Collaboration Revenue
Apel said the quarter’s revenue included approximately $26 million from the Sarepta collaboration, about $20 million from the Novartis collaboration and the full $25 million upfront payment from Madrigal for the ARO-PNPLA3 license and technology transfer.
Commercial REDEMPLO revenue was approximately $2.4 million for the quarter, more than double the roughly $1 million reported in the prior fiscal quarter. Arrowhead said it does not plan to separately highlight product sales until they become a more meaningful financial contributor.
Operating expenses increased to approximately $245 million from $193 million a year earlier, including research and development expense of $198 million and selling, general and administrative expense of $47 million. The increases reflected clinical development, manufacturing activity and commercialization investment.
Arrowhead ended the quarter with approximately $1.6 billion in cash and investments. Apel said the company believes its balance sheet provides flexibility to fund pipeline development, commercial activities and longer-term strategic priorities.
About Arrowhead Pharmaceuticals (NASDAQ:ARWR)
Arrowhead Pharmaceuticals, Inc is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Since its founding in 2008, Arrowhead has leveraged its proprietary delivery platform—known internally as the Advanced RNAi Compound (ARC) technology—to silence disease-causing genes in patients suffering from genetically defined diseases. The company’s approach aims to offer durable, targeted treatments across a range of therapeutic areas.
The company’s pipeline includes multiple candidates in various stages of development.
