Arrowhead Pharmaceuticals to Present Topline Data from the Phase 2 SEQUOIA Study Evaluating Fazirsiran and Describe Design for Planned Phase 3 Study

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[{"type":"text","content":"\n- Announcement on January 9, 2023, with Webcast at 8:30 a.m. ET\n\n PASADENA, Calif.--(BUSINESS WIRE)--\nArrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that on January 9, 2023, at 8:30 a.m. ET, it will present topline data from the SEQUOIA Phase 2 study evaluating fazirsiran and provide an outline of a Phase 3 study that was co-developed with and will be run by Takeda. Fazirsiran is a potential first-in-class investigational RNA interference (RNAi) therapy designed to reduce production of the mutant alpha-1 antitrypsin protein (Z-AAT) as a potential treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency. Fazirsiran is being developed under a collaboration and licensing agreement between Arrowhead and Takeda.\n\nTo register for the webcast, visit the Events and Presentations page under the Investors section of www.arrowheadpharma.com.\n\nAbout Alpha-1 Antitrypsin-Associated Deficiency\n\nAlpha-1 Antitrypsin-Associated Deficiency (AATD) is a rare genetic disorder associated with liver disease in children and adults and pulmonary disease in adults. AATD is estimated to affect 1 per 3,000-5,000 people in the United States and 1 per 2,500 in Europe. The protein AAT is primarily synthesized and secreted by liver hepatocytes. Its function is to inhibit enzymes that can break down normal connective tissue. The most common disease variant, the Z mutant, has a single amino acid substitution that results in improper folding of the protein. The mutant protein cannot be effectively secreted and accumulates in globules inside the hepatocytes. This triggers continuous hepatocyte injury, leading to fibrosis, cirrhosis, and increased risk of hepatocellular carcinoma.\n\nIndividuals with the homozygous PiZZ genotype have severe deficiency of functional AAT that may lead to pulmonary disease and liver disease. Lung disease is frequently treated with AAT augmentation therapy. However, augmentation therapy does nothing to treat liver disease, and there is no specific therapy for hepatic manifestations. There is a significant unmet need as liver transplant, with its attendant morbidity and mortality, is currently the only available cure.\n\nAbout Takeda and Arrowhead Collaboration and License Agreement\n\nIn October 2020, Arrowhead and Takeda announced a collaboration and licensing agreement to de...

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