Arrowhead Pharmaceuticals to Present Topline Data from the Phase 2 SEQUOIA Study Evaluating Fazirsiran and Describe Design for Planned Phase 3 Study
PASADENA, Calif.--(BUSINESS WIRE)--Dec 21, 2022--
Arrowhead 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.
About Alpha-1 Antitrypsin-Associated Deficiency
Alpha-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.
Individuals 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.
About Takeda and Arrowhead Collaboration and License Agreement
In October 2020, Arrowhead and Takeda announced a collaboration and licensing agreement to develop fazirsiran. Under the terms of the agreement, Arrowhead and Takeda will co-develop fazirsiran, which, if approved, will be co-commercialized in the U.S. under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global commercialization strategy and receive an exclusive license to commercialize fazirsiran with Arrowhead eligible to receive tiered royalties of 20-25% on net sales. Arrowhead received an upfront payment of $300 million and is eligible to receive potential development, regulatory and commercial milestones up to $740 million.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
View source version on businesswire.com:https://www.businesswire.com/news/home/20221221005061/en/
CONTACT: Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
LifeSci Advisors, LLC
LifeSci Communications, LLC
Josephine Belluardo, Ph.D.
KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA
INDUSTRY KEYWORD: INFECTIOUS DISEASES FDA GENETICS CLINICAL TRIALS CARDIOLOGY BIOTECHNOLOGY GENERAL HEALTH PHARMACEUTICAL HEALTH ONCOLOGY
SOURCE: Arrowhead Pharmaceuticals, Inc.
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PUB: 12/21/2022 07:30 AM/DISC: 12/21/2022 07:31 AM