\nOverview of Arrowhead\u2019s CNS Pipeline<\/p>\n<\/td>\n
\nVince Anzalone, CFA, Arrowhead<\/p>\n<\/td>\n<\/tr>\n
\nSilencing MAPT in Alzheimer\u2019s Disease and other Tauopathies\u200b<\/p>\n<\/td>\n
\nAdam Boxer, M.D., Ph.D., UCSF<\/p>\n<\/td>\n<\/tr>\n
\nTRiM\u2122 CNS Delivery Platform & Overview of ARO-MAPT\u200b<\/p>\n<\/td>\n
\nJames Hamilton, M.D., MBA, Arrowhead<\/p>\n<\/td>\n<\/tr>\n
\nInterim Results from ARO-MAPT Phase 1\/2a Study\u200b<\/p>\n<\/td>\n
\nJames Hamilton, M.D., MBA<\/p>\n<\/td>\n<\/tr>\n
\nKey Takeaways<\/p>\n<\/td>\n
\nVince Anzalone, CFA<\/p>\n<\/td>\n<\/tr>\n
\nQ&A<\/p>\n<\/td>\n
\nPanel<\/p>\n<\/td>\n<\/tr>\n<\/table>\n
About ARO-MAPT<\/b><\/p>\n
\nARO-MAPT is designed to silence CNS expression of the microtubule associated protein tau (MAPT) gene, which encodes the tau protein. Aggregation of the toxic tau protein is believed to be a key driver in multiple tauopathies, including Alzheimer\u2019s disease. By preventing or potentially reversing tau protein accumulation, ARO-MAPT has potential to prevent or slow disease progression.<\/p>\n
About the AROMAPT-SC-1001 Phase 1\/2 Study<\/b><\/p>\n
\nAROMAPT-SC-1001 (NCT07221344<\/a>) is a Phase 1\/2a placebo-controlled dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-MAPT in up to 64 normal healthy volunteers (NHV) and up to 48 subjects with early Alzheimer\u2019s disease (AD), defined as mild cognitive impairment due to Alzheimer\u2019s disease and mild Alzheimer\u2019s disease dementia. In Part 1a of the study, NHVs will receive between one and three weekly doses of ARO-MAPT or placebo by subcutaneous injection. In Parts 1b and 2a, NHVs and subjects with early AD will receive between two and three weekly doses followed by one to two additional doses, for a total of up to 5 subcutaneous doses of ARO-MAPT or placebo.<\/p>\n About Arrowhead Pharmaceuticals<\/b><\/p>\n \nArrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM\u2122) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.<\/p>\n \nFor more information, please visit www.arrowheadpharma.com<\/a>, or follow us on X (formerly Twitter) at @ArrowheadPharma<\/a>, LinkedIn<\/a>, Facebook<\/a>, and Instagram<\/a>. To be added to the Company’s email list and receive news directly, please visit http:\/\/ir.arrowheadpharma.com\/email-alerts<\/a>.<\/p>\n Safe Harbor Statement under the Private Securities Litigation Reform Act:<\/b><\/p>\n 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 \u201cmay,\u201d \u201cwill,\u201d \u201cexpect,\u201d \u201cbelieve,\u201d \u201canticipate,\u201d \u201chope,\u201d \u201cintend,\u201d \u201cplan,\u201d \u201cproject,\u201d \u201ccould,\u201d \u201cestimate,\u201d \u201ccontinue,\u201d \u201ctarget,\u201d \u201cforecast\u201d or \u201ccontinue\u201d 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, products or product candidate or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates; and our beliefs and expectations around the potential uses and value of the TRiM\u2122 platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products, demand for our products, 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, 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.<\/i><\/p>\n Source: <\/b>Arrowhead Pharmaceuticals, Inc.<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20260929257404\/en\/<\/a><\/span><\/p>\n \nArrowhead Pharmaceuticals, Inc. Paul Graves Investors: Media: KEYWORDS:<\/b> United States North America California<\/p>\n INDUSTRY KEYWORDS:<\/b> Health Neurology Genetics Clinical Trials Pharmaceutical Biotechnology<\/p>\n<\/p>\n
\n
Vince Anzalone, CFA
\n
+1 626-304-3400
\n
ir@arrowheadpharma.com
\n<\/a><\/p>\n
\n
+1 626-304-3400
\n
media@arrowheadpharma.com
\n<\/a><\/p>\n
\n<\/b>
LifeSci Advisors, LLC
\n
Brian Ritchie
\n
+1 212-915-2578
\n
britchie@lifesciadvisors.com
\n<\/a><\/p>\n
\n<\/b>
LifeSci Communications, LLC
\n
Kendy Guarinoni, Ph.D.
\n
+1 724-910-9389
\n
kguarinoni@lifescicomms.com<\/a><\/p>\n
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