Arrowhead Pharmaceuticals, Inc.

Thank you, Jonathan.

Good afternoon, everyone. Thank you for joining us today to discuss Arrowhead's results for its fiscal 2021. The first quarter ended December 31, 2020. With us today from management are President and CEO, Dr. Christopher Anzalone, who will provide an overview of the quarter, Dr. James Hamilton, our Senior Vice President of Discovery and Translational Medicine, who has graciously agreed to sub in for Dr. Javier San Martin while he is out ill today. and Ken Muskowski, our Chief Financial Officer, who will give a review of the financials. In addition, James Hazard, our Chief Commercial Officer, will be available during the Q&A portion of today's call. Before we begin, I would like to remind you that comments made during today's call contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements, other than statements of historical fact, and including without limitation those with respect to Arrowhead's goals, plans and strategies are forward-looking statements. These include statements regarding our expectations around the development, safety and efficacy of our drug candidates, projected cash runway and expected future development and commercialization activities. These statements represent management's current expectations and are subject to numerous risks and uncertainties that could cause actual results to differ materially Thanks, Vince.

Good afternoon, everyone, and thank you for joining us today. The last quarter was an important period for us and our shareholders because we demonstrated clear advances in multiple development programs that we believe represent key value drivers for the respective product candidates and our proprietary trim platform. We see this as positioning us well to build substantial value throughout 2021. We seek to create value in two primary ways. First, we push RNAi and our platform Thank you very much. As we look at 2021, we expect substantial progress on both fronts, with several opportunities in the first half of the calendar year alone. For instance, by the middle of the year, we could have clinical proof of concept for our ability to bring RNAi outside the liver, opening a whole new range of addressable diseases without adequate treatments. We also expect to begin three large Phase IIb studies and a Phase 3 study during this time frame, and we may also have a better idea about how we in Takeda may be able to streamline the Arrow AAT clinical program to accelerate that drug candidate to market. Let's begin by looking at our earlier stage programs. The power of Arrowhead's TRMM platform is our ability to rapidly discover and develop multiple new drugs targeting diverse disease areas in a way that has been predictable, reproducible, and with an attractive safety profile. Each new program builds upon prior programs, which could make them progressively lower risk. We've seen this in our liver-targeted programs and are applying this concept to our extra-hepatic initiatives. We hope to do in many other tissues what we have done in the liver. Given our clinical experience in hepatocyte-targeted programs, specifically in HPV, AAT, APOC3, ANG3, and LPAA, We generally expect future hepatocyte-directed candidates, such as AeroHSD and others, to also be highly potent and well-tolerated. Creating this type of expectation in a new cell type could represent a substantial value creation event. Now imagine leveraging that not only in a single new cell type, but with many new cell types. As we have said in the past, we expect to be able to target a new cell type every 18 to 24 months. If we can pull this off, we would expect it to provide a huge amount of leverage in value creation. This is exciting and it's one reason I believe that the progress we've made over the recent time periods could be just a prelude to something bigger. We think we are now on the cusp of having clinical data to assess the ability of TRMM to knock down target genes in the lung and in solid tumors and are working toward bringing our first muscle targeted program into the clinic late this summer.