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

• REDEMPLO® (plozasiran) is listed in the Lauer-Taxe® database as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronaemia syndrome (FCS), diagnosed either based on clinical symptoms alone or confirmed with a genetic test
• People living with this rare disease have extremely high triglyceride levels and a substantially higher risk of acute pancreatitis and related long-term complications, often resulting in a reduced quality of life
• European marketing authorisation is based on positive results from the Phase 3 PALISADE study where plozasiran significantly reduced median fasting triglyceride levels at the 25 mg recommended dose from baseline to Month 10, compared to placebo (p <0.0001)

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that people in Germany who live with a rare metabolic condition called Familial Chylomicronaemia Syndrome (FCS) are the among first in the European Union to have a new treatment option to reduce dangerously high levels of triglycerides (TGs, a type of fat) in their bloodstream. Following its marketing authorization by the European Commission in June 2026, REDEMPLO® (plozasiran) is now listed in the Lauer-Taxe® database and thus able to be prescribed, dispensed and reimbursed in Germany. REDEMPLO is authorized for use alongside diet, by adults diagnosed with FCS based on clinical symptoms or a genetic test, unlike other treatments that can be used only after genetic confirmation. It is taken as a subcutaneous injection once every three months.

"People living with FCS face a life-long risk of recurrent and potentially fatal acute pancreatitis, and the possibility to treat using plozasiran based on a clinical diagnosis, without requiring a genetic test, could shorten a diagnostic journey that is often long and frustrating. This process is accelerated still further when physicians remain aware of the combination of symptoms that suggest FCS," said Professor Ioanna Gouni-Berthold, Head of the Lipid Clinic and Lipid Research Clinic, University of Cologne. "The availability of plozasiran in Germany provides physicians with an important new option to treat this debilitating disease."

"We are thrilled to bring REDEMPLO as a new treatment option in Germany for people living with genetically or clinically confirmed FCS," said Christopher Anzalone, Ph.D., President and CEO at Arrowhead Pharmaceuticals. "While we are working to make plozasiran available for people living with FCS in several European countries over the coming months, Germany is our first focus in the European Union. We are glad to be able to support eligible people in Germany with this new treatment option, as we continue the relevant processes in other European countries."

Regulatory approval for REDEMPLO was based on findings from the PALISADE study (NCT05089084), a Phase 3 placebo-controlled study to evaluate the efficacy and safety of plozasiran in 75 adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint of the study was the median percent change from baseline in fasting TGs versus placebo at Month 10. Additional information on study design can be found on clinicaltrials.gov (NCT05089084). The most common adverse reactions were hyperglycaemia (12.8%), headache (6.8%), nausea (4.7%), and injection site reaction (4.7%).

About FCS

Familial Chylomicronaemia Syndrome (FCS) 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 and other long-term complications.

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 portfolio of clinical and commercial RNAi therapeutics through its targeted RNAi molecule (TRiM™) platform. At Arrowhead, we rapidly advance potential RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we care for.

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.

Source: Arrowhead Pharmaceuticals, Inc.

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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
+1 626-304-3400
ir@arrowheadpharma.com

Paul Graves
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media@arrowheadpharma.com

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