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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We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.