ARO-DUX4

PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced its plan to host a 2024 Summer Series of R&D webinars that highlight multiple clinical stage RNA interference (RNAi) based medicines that utilize the company’s proprietary Targeted RNAi Molecule (TRiMTM) platform. Each webinar in the Summer Series will focus on a specific therapeutic area and will feature presentations by Arrowhead team members about the company’s clinical candidates and external key opinion leaders, who will discuss the respective disease areas and treatment landscapes. 2024 Summer Series Schedule: Muscular – ARO-DUX4 and ARO-DM1 Date: May 23, 2024 Time: 2:00 pm ET Cardiometabolic – Plozasiran and zodasiran Date: June 25, 2024 Time: TBD Pulmonary – ARO-RAGE, ARO-MUC5AC, ARO-MMP7 Date: July 16, 2024 Time: TBD Obesity/Metabolic – ARO-INHBE and one additional new program to be disclosed Date: August 15, 2024 Time: TBD Central Nervous System – Platform and pipeline updates Date: September 25, 2024 Time: TBD Christopher Anzalone, Ph.D., President and CEO at Arrowhead, said: “Arrowhead has made significant progress this year with our pipeline. We now have 14 investigational RNAi-based medicines in the clinic that leverage Arrowhead’s proprietary TRiMTM platform, four of which are in late-stage development. We are excited to launch the 2024 Summer Series of R&D webinars, which will provide an overview of the respective treatment landscapes, the biological rationale and preclinical data supporting each target, and our clinical development strategy for each pipeline program to be highlighted.” Each event will be approximately 90 minutes in duration with live webcasts and replays available on the Events and Presentations page under the Investors section of the Arrowhead website. Registration links and additional details regarding times, agendas, and speakers will be provided on the Arrowhead website and social media channels prior to each event. 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 X (formerly Twitter) at @ArrowheadPharma or on LinkedIn. 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.

Conference Call and Webcast Today, August 7, 2023 at 4:30 p.m. ET PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc., (NASDAQ: ARWR) today announced financial results for its fiscal third quarter ended June 30, 2023. The company is hosting a conference call today, Aug. 7, 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 Hosted a Research & Development Day 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. Key updates included: New Phase 1 clinical data on ARO-RAGE showing a continued dose response in healthy volunteers with a single inhaled dose of 184 mg achieving mean knockdown of 90% and a maximum of 95% in bronchoalveolar lavage fluid (BALF) Serum sRAGE reductions in asthma patients consistent with effects seen in healthy volunteers at the 44 mg dose, the only dose level with results currently available in asthma patient cohorts TRiMTM platform for intrathecal administration for central nervous system (CNS) delivery demonstrates 90-95% dose-dependent mRNA knockdown in disease-relevant spinal cord and cortex brain regions in non-human primates TRiMTM platform for adipose tissue delivery achieved up to 98% knockdown and maintained greater than 85% knockdown over 31 weeks TRiMTM dimer platform for delivery to hepatocytes achieved equivalent or better knockdown of two separate target genes with longer duration than monomer mixture Completed enrollment of the global PALISADE Phase 3 clinical trial evaluating ARO-APOC3 for the treatment of familial chylomicronemia syndrome. The company anticipates that the primary portion of the study will be complete in the second quarter of 2024 with a data readout shortly thereafter Presented interim data from the ongoing Phase 2 GATEWAY clinical study of ARO-ANG3 in patients with homozygous familial hypercholesterolemia at the 91st European Atherosclerosis Society Congress showing that ARO-ANG3 achieved 44-48% mean reductions in LDL-C on top of continued standard of care Presented updated results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency at the European Association for the Study of the Liver (EASL) Congress 2023 Filed an application to initiate a Phase 1 clinical trial of ARO-SOD1, an RNAi-based investigational medicine 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 is the first therapeutic candidate designed for delivery to the CNS to enter clinical studies that leverages the TRiMTM platform Filed an application to initiate a Phase 1/2 clinical trial of ARO-DUX4, the company’s investigational RNAi therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). ARO-DUX4 is the first clinical candidate utilizing the TRiMTM platform to target disease associated genes in skeletal muscle 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) 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 Selected Fiscal 2023 Third Quarter Financial Results Three Months Ended June 30, OPERATING SUMMARY 2023 2022 (unaudited) Revenue $ 15,825 $ 32,412 Operating Expenses: Research and development 94,757 72,180 General and administrative expenses 23,771 33,141 Total Operating Expenses 118,528 105,321 Operating loss (102,703) (72,909) Interest income 4,172 1,240 Interest expense (5,158) - Other, net 306 (377) Loss before income tax expense and noncontrolling interest (103,383) (72,046) Income tax expense 742 - Net loss before noncontrolling interest (104,125) (72,046) Net loss attributable to noncontrolling interest, net of tax (1,179) - Net loss attributable to Arrowhead Pharmaceuticals, Inc. $ (102,946) $ (72,046) Net loss per share attributable to Arrowhead Pharmaceuticals, Inc. - Diluted $ (0.96) $ (0.68) Weighted-average shares used in calculating - Diluted 107,004 105,753 FINANCIAL POSITION SUMMARY June 30, 2023 September 30, 2022 (unaudited) Cash, cash equivalents and restricted cash $ 105,334 $ 108,005 Short-term investments 346,369 268,391 Long-term investments 42,758 105,872 Total cash resources (cash and investments) 494,461 482,268 Other assets 301,395 209,671 Total Assets $ 795,856 $ 691,939 Current deferred revenue $ 16,905 $ 74,099 Long-term deferred revenue - 55,950 Other liabilities 396,966 143,551 Total Liabilities $ 413,871 $ 273,600 Total Arrowhead Pharmaceuticals, Inc. Stockholders' Equity $ 364,830 $ 398,520 Noncontrolling Interest 17,155 19,819 Total Noncontrolling Interest and Stockholders' Equity 381,985 418,339 Total Liabilities, Noncontrolling Interest and Stockholders' Equity $ 795,856 $ 691,939 Shares Outstanding 107,102 105,960 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.