Daplusiran/​Tomligisiran

- Conference Call and Webcast Today, November 29, 2023, at 4:30 p.m. ET PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc., (NASDAQ: ARWR) today announced financial results for its fiscal year ended September 30, 2023. The company is hosting a conference call today, November 29, 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 Presented new Phase 2 clinical data from the ongoing SHASTA-2 and MUIR studies of plozasiran (ARO-APOC3) and the ARCHES-2 study of zodasiran (ARO-ANG3) at the American Heart Association (AHA) Scientific Sessions. Key updates included: Plozasiran achieved mean max reductions of up to 90% in APOC3, 87% in triglycerides, and 63% in remnant cholesterol in patients with severe hypertriglyceridemia. In patients with mixed dyslipidemia, plozasiran achieved a mean max reduction in APOC3 of up to 89%, leading to robust and durable improvements in multiple atherogenic lipoproteins Zodasiran treatment resulted in substantial reductions of ANGPTL3, triglycerides, and LDL-cholesterol in patients with mixed dyslipidemia Plozasiran and zodasiran continue to show favorable safety pro Treatment emergent adverse events reported to date reflect the comorbidities and underlying conditions of the study populations Hosted a webinar featuring experts in the treatment and management of lipid and lipoprotein disorders to discuss plozasiran Daniel Gaudet, MD, PhD, Professor of Medicine at Université de Montréal, discussed plozasiran in the context of the current treatment landscape for severe hypertriglyceridemia Børge Nordestgaard, MD, Professor & Chief Physician, Copenhagen University Hospital, University of Copenhagen, Denmark, discussed the emergent role of remnant cholesterol in cardiovascular disease Steven Nissen, MD, Chief Academic Officer for the Heart and Vascular Institute at the Cleveland Clinic, discussed why the decrease in atherogenic lipoproteins observed with plozasiran has the potential to prevent cardiovascular outcomes Completed chronic GLP toxicology for pulmonary candidates ARO-RAGE, designed to reduce expression of the receptor for advanced glycation end products as a potential treatment for inflammatory pulmonary diseases, and ARO-MMP7, designed to reduce expression of matrix metalloproteinase 7 as a potential treatment for idiopathic pulmonary fibrosis Results were highly encouraging and suggest sufficient safety margins to proceed to Phase 2 studies Presented data on the company’s pipeline of pulmonary targeted RNAi therapeutic candidates, including clinical results for ARO-RAGE, at the European Respiratory Society (ERS) International Congress 2023. Key results included: Late-breaking clinical data for ARO-RAGE demonstrated deep and durable gene silencing in healthy volunteers and in patients with asthma Promising preclinical data across multiple pulmonary programs suggested broad potential applications for Arrowhead’s TRiM™ platform Filed for regulatory clearance to initiate a Phase 1/2a study of Arrowhead’s second RNAi therapeutic candidate targeting skeletal muscle, ARO-DM1, for patients with type 1 myotonic dystrophy Announced that GSK plc reached an agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, to transfer exclusive worldwide rights to further develop and commercialize JNJ-3989. JNJ-3989 (formerly ARO-HBV) was initially in-licensed by Janssen from Arrowhead in 2018 Selected Fiscal 2023 Year End Financial Results ARROWHEAD PHARMACEUTICALS, INC. CONSOLIDATED CONDENSED FINANCIAL INFORMATION (in thousands, except per share amounts) OPERATING SUMMARY Year Ended September 30, 2023 2022 Revenue $ 240,735 $ 243,231 Operating Expenses: Research and development 353,188 297,307 General and administrative expenses 92,549 124,431 Total Operating Expenses 445,737 421,738 Operating loss (205,002 ) (178,507 ) Interest income 15,299 5,033 Interest expense (18,326 ) - Other, net 1,538 765 Loss before income tax expense and noncontrolling interest (206,491 ) (172,709 ) Income tax expense 2,784 3,785 Net loss including noncontrolling interest (209,275 ) (176,494 ) Net loss attributable to noncontrolling interest, net of tax (4,000 ) (431 ) Net loss attributable to Arrowhead Pharmaceuticals, Inc. $ (205,275 ) $ (176,063 ) Net loss per share attributable to Arrowhead Pharmaceuticals, Inc. - Diluted $ (1.92 ) $ (1.67 ) Weighted-average shares used in calculating - Diluted 106,750 105,426 FINANCIAL POSITION SUMMARY September 30, 2023 2022 Cash, cash equivalents and restricted cash $ 110,891 $ 108,005 Investments 292,735 374,263 Total cash resources (cash and investments) 403,626 482,268 Other assets 361,926 209,671 Total Assets $ 765,552 $ 691,939 Current deferred revenue $ 866 $ 74,099 Long-term deferred revenue - 55,950 Other liabilities 477,524 143,551 Total Liabilities $ 478,390 $ 273,600 Total Arrowhead Pharmaceuticals, Inc. Stockholders' Equity $ 271,343 $ 398,520 Noncontrolling Interest 15,819 19,819 Total Noncontrolling Interest and Stockholders' Equity $ 287,162 $ 418,339 Total Liabilities, Noncontrolling Interest and Stockholders' Equity $ 765,552 $ 691,939 Shares Outstanding 107,312 105,960 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. 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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.

GSK and Arrowhead Pharmaceuticals have announced that they have reached an agreement with Janssen Pharmaceuticals to receive exclusive rights to its hepatitis B therapy, JNJ-3989. The Johnson & Johnson company’s investigational hepatitis B virus (HBV)-targeted small interfering ribonucleic acid (siRNA) therapeutic was initially licensed by Janssen from Arrowhead in 2018. Exclusive rights to the liver-targeted siRNA antiviral therapy will accelerate the expansion of GSK’s own hepatitis B treatment, bepirovirsen, which is currently in late-stage development, the company said. Under the terms of the deal, GSK will pay upfront and potential milestone-based payments totalling $1bn to Janssen and Arrowhead to acquire exclusive rights and obligations under the current license agreement between both companies. Janssen will continue to be responsible for the ongoing clinical trials of JNJ-3989 at its own expense and GSK will be responsible for future development and commercialisation activities. Additionally, tiered royalties on net dates pursuant to the original agreement will go to Arrowhead. GSK intends to evaluate JNJ-3989 in combination with its investigational antisense oligonucleotide, bepirovirsen, for the treatment of adult non-cirrhotic patients with chronic hepatitis B (CHB) on nucleos(t)ide analogue (NA) therapy. JNJ-3839 has the potential to move into a phase 2 sequential regimen trial with GSK’s bepirovirsen in 2024. Hepatitis B is a viral infection of the liver, caused by HBV, that can result in both acute and chronic liver disease. Estimated to affect 300 million people worldwide, CHB is a long-lasting infection and occurs when the body’s immune system is unable to fight off the virus and it persists in the blood and liver. Tony Wood, chief scientific officer at GSK, said: "[The company is] excited to build on promising results already demonstrated with bepirovirsen to investigate a novel sequential regimen with JNJ-3989. “We believe this approach could redefine the treatment paradigm for CHB by helping even more patients achieve functional cure." Arrowhead’s president and chief executive officer, Chris Anzalone, commented: “We… look forward to seeing this programme progress towards the goal of potentially helping the millions of patients with CHB worldwide without adequate treatment options.”