JNJ-75220795

Conference Call and Webcast Today, May 2, 2023 at 4:30 p.m. ET PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced financial results for its fiscal second quarter ended March 31, 2023. The company is hosting a conference call today, May 2, 2023, at 4:30 p.m. ET to discuss the results. Webcast and Conference Call and Details Investors may access a live audio webcast on the Company's website at . A replay of the webcast will be available approximately two hours after the conclusion of the call. For analysts that wish to participate in the conference call, please register at . Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call. Selected Recent Events Reported interim results from an ongoing Phase 1/2 clinical study of ARO-RAGE, an investigational RNAi therapeutic for treatment for inflammatory pulmonary diseases, such as asthma. These data represent the first clinical demonstration of the potential utility of Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform optimized for delivery to the lungs. Key results from Part 1 of the study in normal healthy volunteers included the following: Mean maximum reduction in soluble RAGE (sRAGE) as measured in serum after two doses of 92 mg on Day 1 and Day 29 was 80% with a maximum reduction of 90% Duration of pharmacologic effect persisted for at least 6 weeks Mean reduction in sRAGE as measured in bronchoalveolar lavage fluid (BALF) at Day 31 after a single dose of 92 mg was 75% with a maximum reduction of 92% Mean maximum reduction of 56% with a maximum reduction of 68% in serum sRAGE was also observed after a single dose of 92 mg Safety and tolerability Overall, no patterns of adverse changes in any clinical safety parameters No reported serious or severe adverse events No dropouts related to drug or related to adverse events These results include 4 of 5 escalating dose levels. Data are not yet available for single or multiple dose cohorts at 184 mg, the highest dose being tested Announced a planned R&D Day on June 1, 2023, which will include presentations on cardiometabolic, pulmonary, and newly announced central nervous system (CNS) pipeline programs The company expanded its TRiMTM platform to include an optimized intrathecal administration for CNS delivery with good distribution throughout the brain and in all relevant brain cell types. The first development candidate to utilize this new delivery platform, ARO-SOD1, is on track for a clinical trial application filing in the third quarter of 2023 to begin clinical studies In preclinical studies, ARO-SOD1 achieved 95% spinal cord tissue mRNA knockdown after a single intrathecal dose in human SOD1 transgenic rats and maintained greater than 80% spinal cord tissue mRNA knockdown three months after a single intrathecal dose in non-human primates Earned a $40 million milestone payment from Takeda (TSE:4502/NYSE:TAK) after the first patient was dosed in the Phase 3 REDWOOD clinical study of fazirsiran (TAK-999/ARO-AAT) for the treatment of alpha-1 antitrypsin deficiency associated liver disease (AATD-LD) Received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ARO-APOC3 for reducing triglycerides in adult patients with familial chylomicronemia syndrome Earned a $30 million milestone payment from GSK (LSE/NYSE: GSK) following the start of GSK’s Phase 2b trial of GSK4532990, formerly called ARO-HSD, an investigational RNAi therapeutic for the treatment of patients with non-alcoholic steatohepatitis (NASH) Initiated dosing in a Phase 1/2a single ascending dose and multiple ascending dose clinical study of ARO-MMP7, Arrowhead’s third pulmonary targeted investigational RNAi therapeutic being developed as a potential treatment for idiopathic pulmonary fibrosis Reported interim results from an ongoing Phase 1/2 clinical study of ARO-C3, an investigational RNAi therapeutic for treatment of various complement mediated diseases, demonstrating mean reductions of 88% in C3 and 91% in AH50, a marker of alternative complement pathway hemolytic activity Regained rights to ARO-PNPLA3, formerly called JNJ-75220795, which was part of a 2018 research collaboration and option agreement between Arrowhead and Janssen Pharmaceuticals, Inc. ARO-PNPLA3 is an investigational RNAi therapeutic developed using Arrowhead’s proprietary TRiMTM platform and designed to reduce liver expression of patatin-like phospholipase domain containing 3 (PNPLA3) as a potential treatment for patients with NASH Announced topline results with Takeda from the Phase 2 SEQUOIA clinical study of investigational fazirsiran for the treatment of AATD-LD and provided an outline of a Phase 3 study that was co-developed by Takeda and Arrowhead and will be conducted by Takeda. Key results from SEQUOIA included the following: Fibrosis regression observed in 50% of patients receiving fazirsiran Median reductions of 94% of Z-AAT accumulation in the liver and mean reduction of 68% in histologic globule burden Treatment emergent adverse events were generally well balanced between fazirsiran and placebo groups Results consistent with AROAAT-2002 open-label study previously published in The New England Journal of Medicine Selected Fiscal 2023 Second Quarter Financial Results Three Months Ended March 31, OPERATING SUMMARY 2023 2022 (unaudited) Revenue $ 146,267 $ 151,805 Operating Expenses: Research and development 74,881 75,985 General and administrative expenses 23,221 34,267 Total Operating Expenses 98,102 110,252 Operating income 48,165 41,553 Interest income 4,560 1,054 Interest expense (5,067 ) - Other, net 8 1,759 Income before income tax expense and noncontrolling interest 47,676 44,366 Income tax expense - - Net income before noncontrolling interest 47,676 44,366 Net loss attributable to noncontrolling interest, net of tax (999 ) - Net income attributable to Arrowhead Pharmaceuticals, Inc. $ 48,675 $ 44,366 Net income per share attributable to Arrowhead Pharmaceuticals, Inc. - Diluted $ 0.45 $ 0.41 Weighted-average shares used in calculating - Diluted 108,143 107,929 FINANCIAL POSITION SUMMARY March 31, 2023 September 30, 2022 (unaudited) Cash, cash equivalents and restricted cash $ 134,959 $ 108,005 Short-term investments 346,046 268,391 Long-term investments 78,834 105,872 Total cash resources (cash and investments) 559,839 482,268 Other assets 331,469 209,671 Total Assets $ 891,308 $ 691,939 Current deferred revenue $ 29,839 $ 74,099 Long-term deferred revenue 1,075 55,950 Other liabilities 395,288 143,551 Total Liabilities $ 426,202 $ 273,600 Total Arrowhead Pharmaceuticals, Inc. Stockholders' Equity $ 446,772 $ 398,520 Noncontrolling Interest 18,334 19,819 Total Noncontrolling Interest and Stockholders' Equity 465,106 418,339 Total Liabilities, Noncontrolling Interest and Stockholders' Equity $ 891,308 $ 691,939 Shares Outstanding 106,869 105,960 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 , or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit . 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,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology 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 forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. 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: Contacts Arrowhead Pharmaceuticals, Inc. Vince Anzalone, CFA 626-304-3400 ir@arrowheadpharma.com Investors: LifeSci Advisors, LLC Brian Ritchie 212-915-2578 britchie@lifesciadvisors.com Media: LifeSci Communications, LLC Jason Braco, Ph.D. 646-751-4361 jbraco@lifescicomms.com Source: Arrowhead Pharmaceuticals, Inc. View this news release online at:

- Event to include presentations on cardiometabolic, pulmonary, and newly announced central nervous system pipeline programs PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it will host a Research & Development (R&D) Day on June 1, 2023, beginning at 9 a.m. ET in New York City to discuss progress towards the company’s “20 in 25” goal to grow its pipeline of RNAi therapeutics that leverage the proprietary Targeted RNAi Molecule (TRiMTM) platform to a total of 20 clinical stage or marketed products in the year 2025. Arrowhead also announced that company has expanded its TRiMTM platform to include an optimized intrathecal administration for central nervous system (CNS) delivery with good distribution throughout the brain and in all relevant brain cell types. The first development candidate to utilize this new delivery platform, ARO-SOD1, is on track for a clinical trial application (CTA) filing in the third quarter of 2023 to begin clinical studies. ARO-SOD1 is designed to reduce expression of superoxide dismutase 1 (SOD1) in the CNS as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations. ARO-SOD1 was highly active against its target with a long duration of effect in multiple preclinical models suggesting that it may be administered quarterly or less frequently. In preclinical studies, ARO-SOD1 achieved 95% spinal cord tissue mRNA knockdown after a single intrathecal dose in human SOD1 transgenic rats and maintained greater than 80% spinal cord tissue mRNA knockdown three months after a single intrathecal dose in non-human primates. The R&D Day will feature presentations by three key opinion leaders: Ira Goldberg, M.D. (NYU Langone Medical Center), who will discuss the unmet medical need in treating patients with hypertriglyceridemia and mixed dyslipidemia in atherosclerotic cardiovascular disease (ASCVD); Matthias Salathe, M.D. (University of Kansas Medical Center), who will discuss the muco-obstructive and inflammatory pulmonary disease landscape; and Michael Benatar, M.D., Ph.D. (University of Miami Miller School of Medicine), who will discuss ALS caused by SOD1 mutations. Drs. Goldberg, Salathe, and Benatar will be available to answer questions following their presentations. The R&D Day will also feature presentations by the Arrowhead team who will provide updates on multiple programs, including: Cardiometabolic programs: ARO-APOC3 for the treatment of patients with familial chylomicronemia syndrome, severe hypertriglyceridemia, and mixed dyslipidemia in ASCVD. Currently in a Phase 3 study and multiple Phase 2b studies. ARO-ANG3 for the treatment of patients with familial hypercholesterolemia. Currently in multiple Phase 2b studies. Pulmonary programs: ARO-RAGE for the treatment of patients with asthma. Currently in a Phase 1/2 study. ARO-MUC5AC for the treatment of patients with muco-obstructive pulmonary diseases. Currently in a Phase 1/2 study. Central nervous system: Development of the TRiMTM platform for CNS delivery. ARO-SOD1 for treatment of patients with ALS caused by SOD1 mutations. A CTA filing is planned for the third quarter of 2023. Additional liver targeted programs: ARO-C3 for treatment of patients with various complement mediated diseases. Currently in a Phase 1/2 study. ARO-PNPLA3 for treatment of patients with non-alcoholic steatohepatitis. Currently in a Phase 1/2 study. This event is intended for institutional investors, sell-side research analysts, and business development professionals only. Please RSVP in advance if you plan to attend, as space is limited. To reserve a seat, please click here to register. A copy of the presentation materials and webcast links may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website. 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 , or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit . 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,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology 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 forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. 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.