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Want Diversified Upside in Biotechnology? Check out LABUArrowhead Pharmaceuticals NASDAQ: ARWR presented 12-month results from its Phase 3 SHASTA-3 and SHASTA-4 trials evaluating plozasiran in adults with severe hypertriglyceridemia, reporting substantial reductions in triglycerides and acute pancreatitis events versus placebo. The company said the data were presented at the European Society of Cardiology Congress in Munich and have been accepted for publication in a major medical journal.

Plozasiran, marketed as REDEMPLO for familial chylomicronemia syndrome, or FCS, is approved to reduce triglycerides in FCS patients in the U.S., European Union, Canada, Australia and China. The severe hypertriglyceridemia indication remains investigational and has not been reviewed or approved by regulators, Arrowhead said.

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Phase 3 results Gerald Watts, Winthrop Professor at the University of Western Australia, presented the findings from the two pivotal trials. SHASTA-3 and SHASTA-4 enrolled adults with severe hypertriglyceridemia, defined as triglyceride levels above 500 milligrams per deciliter. Participants received 25 milligrams of plozasiran or placebo through four subcutaneous injections over 12 months, alongside background dietary and conventional lipid-lowering treatment.

Watts said plozasiran produced triglyceride reductions of about 80% from baseline at three months, with reductions sustained through 12 months. More than 90% of plozasiran-treated patients reached triglyceride levels below 500 milligrams per deciliter, a threshold associated with elevated acute pancreatitis risk. More than half of treated patients achieved normal fasting triglyceride levels below 150 milligrams per deciliter.

Secondary measures also showed reductions in remnant cholesterol, non-HDL cholesterol and apolipoprotein C3, or ApoC3, according to the presentation. Plozasiran is designed to reduce hepatic production of ApoC3, a regulator of triglyceride metabolism.

In a pooled analysis of the two studies, plozasiran reduced cumulative acute pancreatitis events by roughly 80% relative to placebo, Watts said. The presentation cited a 44.1% absolute risk reduction and a number needed to treat of 24 over one year for the overall trial population.

In exploratory analyses of higher-risk groups, the company reported more than a 90% relative reduction in acute pancreatitis among patients with a prior history of the condition. In a smaller subgroup with triglycerides above 880 milligrams per deciliter and prior acute pancreatitis, Arrowhead reported no acute pancreatitis events among plozasiran-treated patients. Watts cautioned that the subgroup involved small numbers and was exploratory.

Safety observations Watts said patient retention exceeded 90% and adherence to the four-dose regimen was nearly 100%. Treatment discontinuations due to adverse events were low and similar between treatment groups, according to the presentation.

The company reported no anaphylaxis, systemic hypersensitivity or meaningful changes in platelet counts, liver enzymes or liver fat fraction relative to placebo. Investigators observed a small increase in measures related to glycemic control in some analyses, though Watts described the change in glycated hemoglobin, or HbA1c, as small and not clinically meaningful at the end of treatment.

Børge Nordestgaard, professor and chief physician at Copenhagen University Hospital, said the glycemic-control observation appeared consistent with the broader ApoC3 inhibitor class. He described the safety profile presented for plozasiran as promising while noting that longer-term follow-up will be needed.

Market focus and regulatory plans Jennifer Hellawell, Arrowhead’s head of clinical development in cardiometabolic disease, said severe hypertriglyceridemia affects approximately 1% of the population, or more than 3 million people in the U.S. The company defines a high-risk segment of about 1 million U.S. patients as those with triglycerides at or above 880 milligrams per deciliter, or those above 500 milligrams per deciliter with a prior history of acute pancreatitis.

Andy Davis, Arrowhead’s head of cardiometabolic commercial operations, said the company intends to initially focus a potential severe hypertriglyceridemia launch on those high-risk patients. Arrowhead has identified more than 20,000 healthcare professionals across lipidology, endocrinology, preventive cardiology, internal medicine and primary care who treat these patients, Davis said.

Vince Anzalone, Arrowhead’s senior vice president of finance and investor relations, said the company remains on schedule to file a supplemental new drug application seeking approval for plozasiran in severe hypertriglyceridemia before year-end. Arrowhead recently purchased a priority review voucher, which Anzalone said could accelerate the potential U.S. review timeline. Subject to a positive regulatory review, the company is targeting a potential launch in 2027.

Arrowhead estimates the U.S. severe hypertriglyceridemia opportunity could represent $3 billion to $4 billion in annual sales over the long term. The company also highlighted other cardiometabolic programs, including zodasiran for homozygous familial hypercholesterolemia and ARO-DIMER-PA, an investigational dual-targeting therapy for mixed hyperlipidemia.

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.

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PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the European Commission (EC) has formally granted marketing authorization for REDEMPLO® (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). REDEMPLO is the first and only siRNA medicine authorized by the EC for adults with FCS, diagnosed either by the presence of clinical criteria or genetic testing.

Importantly, the ability to diagnose and treat without requiring a genetic test could enable earlier treatment, which is particularly relevant in rare diseases such as FCS.

Share“FCS drives an elevated risk of recurrent and potentially fatal episodes of acute pancreatitis. Results from the PALISADE study demonstrate that plozasiran can achieve significant and sustained reductions in triglycerides for these patients,” said Professor Børge Nordestgaard, Department of Clinical Medicine, University of Copenhagen and President, European Atherosclerosis Society. “Importantly, the ability to diagnose and treat without requiring a genetic test could enable earlier treatment, which is particularly relevant in rare diseases such as FCS.”

Announcing its recommendation for the approval of REDEMPLO, the European Medicines Agency noted, "Although other authorised medicines can help people with FCS confirmed by genetic testing, REDEMPLO does not require genetic confirmation of the condition, thus providing a treatment option for more adults with FCS and addressing the unmet medical need in these patients."

“Today's approval marks a pivotal moment for people living with familial chylomicronemia syndrome. As a patient myself and having spoken with countless others living with FCS through leading our organization, I know firsthand how devastating the burden of FCS is on every dimension of daily life. The constant uncertainty, the worry, the fear of acute pancreatitis, the chronic pain and fatigue are challenges the FCS community faces every single day, on top of the long and often frustrating journey to receiving a diagnosis,” added Rosa Pérez Jiménez, President of Familial Chylomicronemia Association (Asociación de Quilomicronemia Familiar) Spain. “This new therapeutic option gives renewed hope to patients who have waited far too long to be seen, understood, and treated.”

Harnessing Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform, REDEMPLO is designed to suppress production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by inhibiting their breakdown and clearance.

“We are pleased to have received EC approval for REDEMPLO as a new treatment option for people living with genetically or clinically confirmed FCS. With this approval secured, we are engaging with relevant national authorities and healthcare communities across the European Union to bring REDEMPLO to people living with FCS as quickly and efficiently as possible,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead Pharmaceuticals. “This ongoing cadence of regulatory approvals around the world reflects the strength of our clinical data and the real progress being made across our diverse pipeline of siRNA-based therapies that leverage our proprietary TRiM™ platform.”

EC regulatory approval was supported by clinical data from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial in 75 adults with clinically diagnosed or genetically confirmed FCS.1,2 The PALISADE study met its primary endpoint and all multiplicity-controlled key secondary endpoints. In PALISADE, 25 mg REDEMPLO reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo. Additionally, the combined doses of 25 mg and 50 mg plozasiran significantly reduced the incidence of acute pancreatitis (odds ratio, 0.169; p=0.0292). The odds of acute pancreatitis were 83% lower in the pooled plozasiran groups compared with the placebo group. The most common adverse reactions were hyperglycaemia (12.8%), headache (6.8%), nausea (4.7%), and injection site reaction (4.7%).1,2

About Familial Chylomicronemia Syndrome (FCS)

Familial chylomicronemia syndrome is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 10 mmol/L (880 mg/dL). Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options to adequately treat FCS.

About REDEMPLO® (plozasiran)

REDEMPLO (plozasiran) is currently approved by the U.S. Food and Drug Administration, Health Canada, China’s National Medical Products Administration, the Australian Therapeutic Goods Administration, and by the European Commission as an adjunct to diet to reduce triglycerides for adults with FCS. REDEMPLO is the first and only siRNA treatment approved in these countries to be studied in both clinically diagnosed and genetically confirmed patients living with FCS.

REDEMPLO is designed to suppress the production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting APOC3 with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is self-administered via subcutaneous injection once every three months.

REDEMPLO has been granted Orphan Medicinal Product Designation by the EMA for the treatment of patients with FCS, and Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by the U.S. FDA for the treatment of patients with FCS. In December 2025, plozasiran was also granted Breakthrough Therapy designation by the U.S. FDA in severe hypertriglyceridemia.

Plozasiran is also being investigated in the SHASTA-3 (NCT06347003), SHASTA-4 (NCT06347016), and SHASTA-5 (NCT06880770) Phase 3 studies in adults with severe hypertriglyceridemia and the MUIR-3 (NCT06347133) Phase 3 study in adults with hypertriglyceridemia.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.

For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company’s email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline, products or product candidates or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts of REDEMPLO (plozasiran) on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates if approved; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products if approved, demand for our products, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

References

Watts GF, Rosenson RS, Hegele RA, Goldberg IJ, Gallo A, Mertens A, Baass A, Zhou R, Muhsin M, Hellawell J, et al. Plozasiran for managing persistent chylomicronemia and pancreatitis risk. N Engl J Med. 2024;392:127–137. https://doi.org/10.1056/nejmoa2409368 PMID: 39225259.Watts GF, Hegele RA, Rosenson RS et al. Temporal Effects of Plozasiran on Lipids and Lipoproteins in Persistent Chylomicronemia. Circulation. 2025:151(10); 733-736; https://doi.org/10.1161/CIRCULATIONAHA.124.072860 PMID:39549263.Source: Arrowhead Pharmaceuticals, Inc.

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