Edison Investment Research - Arrowhead Pharmaceuticals Inc. (ARWR) Update: New drug candidate revealed

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Edison Investment Research

Update: New drug candidate revealed

Arrowhead announced at its recent analyst day its new RNAi-based drug candidate, ARC-AAT, which is developed for alpha-1 antitrypsin deficiency (AATD). ARC-AAT consists of Arrowheadâ€™s unique DPC delivery vehicle and an RNAi molecule based on a new RNA chemistry, and is able to achieve over 95% knockdown of the target protein in animal models. Arrowhead plans to file an IND of ARC-AAT in Q414, right after the release of its top-line results of the Phase IIa trial of ARC-520 in patients with chronic HBV infection in Q314. Our valuation is $1,075m, equivalent to $20.7/per basic share (or $17.3/per diluted share).

03/07/2014 5 EN

Underlying

Arrowhead Pharmaceuticals Inc.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a portfolio of RNA chemistries and several modes of delivery, the company's therapies trigger the RNA interference (RNAi) mechanism to induce knockdown of target genes. The company is focused on developing drugs for diseases with a genetic basis, typically characterized by the overproduction of one or more proteins that are involved with disease. The company's pipeline includes, among others, ARO-AAT, a candidate for the treatment of liver disease, and ARO-APOC3, which is designed to reduce production of Apolipoprotein C-III, a component of triglyceride rich lipoproteins.

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US04280A1007 NASDAQ Stock Market United States

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Edison Investment Research

Edison is an investment research and advisory company, with offices in North America, Europe, the Middle East and AsiaPac. The heart of Edison is our world renowned equity research platform and deep multi-sector expertise. At Edison Investment Research, our research is widely read by international investors, advisors and stakeholders. Edison Advisors leverages our core research platform to provide differentiated services including investor relations and strategic consulting.

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