Arrowhead Pharmaceuticals, Inc.

Adults Living with Familial Chylomicronaemia Syndrome (FCS) in England and Wales Have REDEMPLO® (Plozasiran) as a New Treatment Option to Reduce Triglycerides

Arrowhead Pharmaceuticals won UK MHRA marketing authorization for REDEMPLO (plozasiran) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronaemia syndrome. NICE separately recommended NHS funding in England and Wales for genetically confirmed FCS under a commercial arrangement, making this a full approval-plus-reimbursement win in the company's first European market. Authorization was based on the Phase 3 PALISADE study (75 patients), where the 25 mg dose significantly reduced median fasting triglycerides vs placebo (p<0.0001) at month 10. Arrowhead says it will roll plozasiran out in several European countries over the coming months, though the ultra-rare FCS population caps near-term revenue potential.

•5d ago

Arrowhead Pharmaceuticals Announces ARO-MAPT Interim Phase 1/2a Clinical Data Demonstrating Deep and Durable Tau Reductions in Healthy Volunteers

Arrowhead announced interim Phase 1/2a data for ARO-MAPT showing mean CSF total tau reduction of 73% at Day 105, with a maximum individual reduction of 85%, in healthy volunteers. The results are the first clinical validation of subcutaneous siRNA delivery across the blood-brain barrier, supporting potential quarterly or less-frequent dosing; safety was clean with no serious adverse events. Alzheimer's and other tauopathy patient cohorts remain to be read out, with additional data updates expected in 2027 and regulatory discussions underway for frontotemporal dementia and Progressive Supranuclear Palsy studies.

•18d ago

Arrowhead Pharmaceuticals Granted Entry in the FDA STAR Pilot Program for Plozasiran in Severe Hypertriglyceridemia

The FDA granted Arrowhead's plozasiran entry into the STAR pilot program for the upcoming sNDA in severe hypertriglyceridemia. STAR entry qualifies the supplemental application for a 6-month Priority Review versus the standard 10 months, with a possible expedited action at least a month before the goal date — and Arrowhead retains its Priority Review Voucher since none is needed. Arrowhead remains on schedule to submit the sNDA by year-end 2026; preserving the PRV gives the company the option to redeploy it for a future application or monetize it.

•25d ago

Arrowhead Pharmaceuticals Announces Interim Topline Clinical Data for the First Dual-Functional RNAi Therapeutic for the Treatment of Mixed Hyperlipidemia

Arrowhead Pharmaceuticals reported interim topline Phase 1/2a data for ARO-DIMER-PA, its first dual-functional RNAi therapeutic, showing mean maximal single-dose reductions of 72% in serum PCSK9 and 88% in APOC3 in patients with mixed hyperlipidemia. The dual-gene silencing translated into broad lipid improvements: LDL-C down 54%, triglycerides down 73%, non-HDL cholesterol down 61%, and ApoB down 50%. No drug-related serious adverse events were reported, with dose escalation completed through 400 mg and the multi-dose portion of the up-to-78-subject study still ongoing. CEO Chris Anzalone called the results clinical validation of the TRiM platform's ability to silence two genes with a single molecule, a first for the RNAi field, with additional data to be presented at an upcoming medical congress. The asset fits Arrowhead's cardiometabolic focus alongside its first commercial product REDEMPLO (plozasiran) and zodasiran, which is in the global Phase 3 YOSEMITE trial in homozygous familial hypercholesterolemia.

•1mo ago

Arrowhead Pharmaceuticals Presents Phase 3 SHASTA-3 and SHASTA-4 Data Demonstrating Plozasiran Reduced Acute Pancreatitis Events in Patients with Severe Hypertriglyceridemia

Arrowhead Pharmaceuticals presented positive Phase 3 SHASTA-3 and SHASTA-4 data showing plozasiran reduced triglycerides by 79% and 81% and cut acute pancreatitis events by 78% versus placebo in patients with severe hypertriglyceridemia. More than 90% of treated patients achieved triglyceride levels below the 500 mg/dL risk threshold, with a favorable safety profile comparable to placebo and low discontinuation rates. Based on these results, the company plans to file a supplemental New Drug Application with the FDA by the end of 2026 and intends to utilize a Priority Review Voucher to accelerate approval.

•2mo ago

People Living with Familial Chylomicronaemia Syndrome (FCS) in Germany Have Access to REDEMPLO® (plozasiran) as a New Treatment Option to Reduce Triglycerides

Arrowhead announced that REDEMPLO (plozasiran) is now listed in the Lauer-Taxe database in Germany, making it available for prescription and reimbursement for patients with Familial Chylomicronaemia Syndrome. The marketing authorization is supported by Phase 3 PALISADE data showing significant reductions in fasting triglyceride levels at the 25 mg dose compared to placebo. Germany is the first EU market focus for the company, with plans to expand availability to other European countries in the coming months.

•2mo ago

Arrowhead Pharmaceuticals Reports Fiscal 2026 Third Quarter Results

Arrowhead delivered Q3 revenue of $75.3 million, up 171% year-over-year, driven by strong REDEMPLO commercial momentum with prescription volume roughly doubling during the quarter. Phase 3 SHASTA-3 and SHASTA-4 studies met all primary and secondary endpoints, demonstrating 79-81% median triglyceride reductions and 78% reduction in acute pancreatitis events versus placebo. The company secured EU and Australia marketing authorizations for REDEMPLO, acquired an FDA priority review voucher for plozasiran, and signed an exclusive license with Madrigal for ARO-PNPLA3 worth $25 million upfront with potential milestones of $975 million.

•2mo ago

Arrowhead Pharmaceuticals Acquires FDA Priority Review Voucher

Arrowhead Pharmaceuticals acquired a Rare Pediatric Disease Priority Review Voucher to accelerate the FDA review of a planned sNDA for plozasiran in severe hypertriglyceridemia. The company anticipates submitting the sNDA before the end of 2026, with the voucher reducing the standard review period from 10 months to six months. CEO Christopher Anzalone cited positive topline results from the SHASTA-3 and SHASTA-4 studies as the basis for confidence in the drug's potential to transform treatment for sHTG patients.

•2mo ago

Arrowhead Pharmaceuticals Completes Enrollment in Global Phase 3 YOSEMITE Study of Zodasiran for the Treatment of Homozygous Familial Hypercholesterolemia

Arrowhead Pharmaceuticals completed enrollment in the global Phase 3 YOSEMITE study of zodasiran for homozygous familial hypercholesterolemia (HoFH). The study enrolled 70 patients, exceeding the initial target of 60 due to high physician and patient interest. The company anticipates study completion in mid-2027, with the primary endpoint measuring percent change in LDL-C at month 12. Pending successful results, Arrowhead intends to seek regulatory approvals in multiple geographies. Zodasiran is an investigational RNAi therapeutic targeting ANGPTL3 and has received Orphan Drug Designation from the FDA.

•3mo ago

Arrowhead Pharmaceuticals Reports Topline Results from Phase 3 SHASTA-3 and SHASTA-4 Studies of Plozasiran in Patients with Severe Hypertriglyceridemia

Arrowhead Pharmaceuticals reported positive topline results from the Phase 3 SHASTA-3 and SHASTA-4 studies of plozasiran in severe hypertriglyceridemia, achieving median triglyceride reductions of 79% and 81% respectively versus placebo. The studies met all primary and secondary endpoints, showing a statistically significant reduction in acute pancreatitis events, including a 100% reduction in high-risk patients, with no new safety signals observed. Management plans to submit a supplemental New Drug Application (sNDA) to the FDA before the end of 2026 to expand the drug's indication for severe hypertriglyceridemia.

•3mo ago

Arrowhead Pharmaceuticals Receives Marketing Authorization in the European Union for REDEMPLO® (plozasiran) to Reduce Triglycerides in Adults with Familial Chylomicronemia Syndrome (FCS)

The European Commission granted marketing authorization for REDEMPLO (plozasiran) to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). REDEMPLO is the first and only authorized treatment for adults with FCS that does not require genetic confirmation of the condition. The authorization is supported by data from the Phase 3 PALISADE study, where the 25 mg dose demonstrated an 80% reduction in triglycerides and an 83% reduction in the incidence of acute pancreatitis compared to placebo. Arrowhead is now engaging with national authorities across the EU to launch the product, which has already secured approval in the U.S., Canada, China, and Australia.

•4mo ago

Arrowhead Pharmaceuticals Presents New Clinical Data on RNAi-based Obesity and MASH Candidate ARO-INHBE at EASL 2026

Arrowhead presented interim Phase 1/2a data for ARO-INHBE at EASL 2026, showing a 44% placebo-adjusted reduction in liver fat content in obese patients with baseline liver fat >8%. The RNAi therapy also demonstrated dose-dependent Activin E reductions of up to 85.3% and enhanced visceral fat reduction when combined with low-dose tirzepatide. The drug was well-tolerated with no discontinuations due to adverse events, supporting the company's plan to engage regulators on Phase 2 designs for MASH and obesity.

•5mo ago

Arrowhead Pharmaceuticals Presents New Positive Clinical Cardiometabolic Data at the 94th European Atherosclerosis Society (EAS) Congress

Arrowhead presented positive clinical data for plozasiran at the 94th EAS Congress, showing that patients with moderate-to-severe renal or moderate hepatic impairment can take the standard 25 mg dose without adjustment. The data confirmed similar pharmacokinetic and pharmacodynamic responses in these impaired populations compared to controls, with no new safety signals identified. A separate case report suggested that preconception exposure to plozasiran may sustain triglyceride lowering throughout pregnancy, highlighting the drug's prolonged pharmacodynamic effects.

•5mo ago

Arrowhead Pharmaceuticals Reports Fiscal 2026 Second Quarter Results

Arrowhead reported Q2 revenue of $73.7 million, down from the prior year, but significantly strengthened its balance sheet with total cash resources of approximately $1.78 billion following recent financings. Deferred revenue increased substantially to $157.2 million, indicating strong future revenue recognition potential despite the current period's reported figures. Commercially, REDEMPLO prescriptions grew 40% over the last four weeks to exceed 400 total, and the U.S. wholesale acquisition cost was raised to a premium $45,000 per patient. The drug also secured regulatory approvals in Australia and China and received a positive CHMP opinion in Europe, expanding its global footprint. The company announced a licensing deal with Madrigal for ARO-PNPLA3 worth up to $1 billion and presented encouraging obesity data, showing ARO-INHBE combined with tirzepatide achieved 9.4% weight loss at 16 weeks compared to 4.8% for the control.

•5mo ago

Arrowhead Pharmaceuticals Licenses Clinical MASH Program Targeting PNPLA3 to Madrigal Pharmaceuticals

Arrowhead granted Madrigal an exclusive global license for ARO-PNPLA3, receiving $25 million upfront and eligibility for up to $975 million in milestones plus tiered royalties. Phase 1 data published in The New England Journal of Medicine showed a 46% reduction in liver fat for homozygous patients with the PNPLA3 I148M mutation, with no clinically meaningful adverse events. The transaction targets a genetic variant highly prevalent in Hispanic MASH patients and aligns with Arrowhead's strategy to partner assets outside its core cardiometabolic focus.

•5mo ago

Arrowhead Pharmaceuticals Receives TGA Approval of REDEMPLO® (plozasiran) in Australia, Expanding Global Access for Patients with Familial Chylomicronemia Syndrome (FCS)

Australia's TGA approved REDEMPLO (plozasiran) as the first and only treatment for Familial Chylomicronemia Syndrome (FCS) in the country. The approval expands global access to the siRNA therapy, which is already approved in the US, Canada, and China, and has a positive opinion from the EMA. The Phase 3 PALISADE study supported the approval, showing REDEMPLO reduced triglycerides by a median of 80% from baseline.

•6mo ago