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

Rhea-AI Summary

Arrowhead Pharmaceuticals (ARWR) announced it will present topline data from its SEQUOIA Phase 2 study of fazirsiran on January 9, 2023, at 8:30 a.m. ET. This investigational RNA interference therapy aims to treat alpha-1 antitrypsin deficiency, a rare genetic liver disease. The Phase 3 study will be co-developed with Takeda. AAT deficiency affects 1 in 3,000-5,000 individuals in the U.S. Arrowhead and Takeda's collaboration includes a profit-sharing structure and potential milestone payments exceeding $1 billion.

Positive

• Topline data from SEQUOIA Phase 2 study to be presented, indicating progress in clinical development.
• Collaboration with Takeda enhances development capabilities and market potential for fazirsiran.
• Potential first-in-class RNAi therapy addressing significant unmet needs in AAT deficiency treatment.
• Milestone payments up to $740 million available, enhancing financial outlook.

Negative

• None.

- Announcement on January 9, 2023, with Webcast at 8:30 a.m. ET

PASADENA, Calif.--(BUSINESS WIRE)-- 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.

To register for the webcast, visit the Events and Presentations page under the Investors section of www.arrowheadpharma.com.

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.

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. 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,” or “continue” 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 or product candidates, including anticipated regulatory submissions and clinical program results, prospects, or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, 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, our ability to successfully develop and commercialize drug candidates, 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20221221005061/en/

Arrowhead Pharmaceuticals, Inc.

Vince Anzalone, CFA

626-304-3400

ir@arrowheadpharma.com

Investors:

LifeSci Advisors, LLC

Brian Ritchie

212-915-2578

britchie@lifesciadvisors.com

www.lifesciadvisors.com

Media:

LifeSci Communications, LLC

Josephine Belluardo, Ph.D.

646-751-4361

jo@lifescicomms.com

www.lifescicommunications.com

Source: Arrowhead Pharmaceuticals, Inc.

FAQ

What is the purpose of the SEQUOIA Phase 2 study for Arrowhead Pharmaceuticals?

The SEQUOIA Phase 2 study evaluates the efficacy of fazirsiran as a treatment for alpha-1 antitrypsin deficiency.

When will Arrowhead Pharmaceuticals present the topline data from the SEQUOIA study?

Arrowhead Pharmaceuticals will present topline data on January 9, 2023, at 8:30 a.m. ET.

What is fazirsiran and what condition does it aim to treat?

Fazirsiran is an investigational RNA interference therapy designed to reduce mutant alpha-1 antitrypsin protein levels, targeting alpha-1 antitrypsin deficiency.

What collaboration exists between Arrowhead Pharmaceuticals and Takeda?

Arrowhead and Takeda have a collaboration agreement to co-develop fazirsiran, sharing profits and responsibilities in commercialization.

What financial opportunities are associated with the Arrowhead and Takeda collaboration?

Arrowhead Pharmaceuticals could receive up to $740 million in milestone payments along with tiered royalties on net sales.