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>>箭头制药主机关键意见领袖研讨会上ARO-ENaC的囊性纤维化治疗

Abstract:
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) is hosting a key opinion leader webinar today at 12:00 PM EDT on ARO-ENaC, the company�s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF).

Arrowhead Pharmaceuticals Hosts Key Opinion Leader Webinar on ARO-ENaC for Treatment of Cystic Fibrosis

帕萨迪纳。CA |发表于2020年7月28日

The webinar features a presentation by Marcus Mall, M.D., professor and director of the Department of Pediatric Pulmonology and Immunology at The Charit� University Medical Center Berlin, who will discuss the current treatment landscape and unmet medical need in treating patients with CF. Arrowhead management is providing a review of the ARO-ENaC program, which utilizes Arrowhead�s proprietary Targeted RNAi Molecule (TRiM�) platform and is the company�s first inhaled RNAi candidate to target pulmonary epithelium. The live webinar and replay may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

在研讨会讨论的要点包括以下内容:

Marcus Mall, M.D., professor and director of the Department of Pediatric Pulmonology and Immunology at The Charit� University Medical Center Berlin

上皮钠通道(ENaC的)起着CF肺疾病的病理生理学中起重要作用,并且代表有前途的替代目标,以提高气道表面的水化和粘液的病人关,独立于它们的CFTR基因型的
A substantial number of CF patients (without F508del allele) cannot be treated with current CFTR modulators and could benefit from ENaC inhibition
Partial rescue of CFTR with current CFTR modulators does not prevent progression of CF lung disease, resulting in an unmet medical need for further improvement of targeted CF therapy
ENaC inhibition has potential to act synergistically with CFTR modulators by improving the driving force for chloride/fluid secretion mediated by mutant CFTR rescued by CFTR modulators
ENaC inhibition has potential as a novel therapeutic approach to improve mucus clearance and provide clinical benefits to patients with other muco-obstructive lung diseases, including COPD
Erik Bush, Ph.D., vice president, biology, Arrowhead Pharmaceuticals

cy钠是一种有效的治疗目标stic fibrosis and muco-obstructive lung disease
ARO-的ENaC克服小分子抑制剂的关键限制
ARO-ENaC的吸入选择性沉默的ENaC表达在肺,倍增几周的粘液纤毛清除的给药后,用没有全身活性的证据保留间隙在mucostasis的羊疾病模型(例如电解质失衡)
Promising results observed in various preclinical toxicology studies
Javier San Martin, M.D., chief medical officer, Arrowhead Pharmaceuticals

AROENaC1001,在多达24名正常健康志愿者(NHVS)和高达30名CF患者1/2期研究,预计将开始投药,在2020年八月
潜在1/2期读出在2021上半可包括在NHVS和患者的安全性,和肺功能的在通过用力呼气容积(FEV1)测量患者的评估和肺清除指数(LCI)
There is a significant and growing population of patients without adequate response to current CF treatment
Arrowhead is exploring potential accelerated regulatory pathways for patients with the highest unmet need, including Class I patients and other patient subsets with insufficient response to standard of care
CF是一种罕见的疾病CFTR基因造成的遗传突变导致肺功能进行性恶化,由于粘液的清除差,感染的复发。ARO-ENaC的旨在减少在肺的气道上皮钠通道α亚基的活性。在CF患者CFTR功能障碍的原因增加ENaC的活动,这有助于呼吸道脱水和减少纤毛运输。这容易使人患者持续性肺部感染,结构损伤和肺的功能逐渐丧失。的ENaC已广泛探索,作为CF的潜在治疗靶标,但吸入的小分子的ENaC抑制剂的开发已经通过基于目标的肾毒性和肺中的作用持续时间短的限制。

Marcus Mall, M.D., is professor and director of the Department of Pediatric Pulmonology and Immunology at The Charit� University Medical Center Berlin. Before this appointment, he was head of the Division of Pediatric Pulmonology & Allergy and the Cystic Fibrosis Center at the Department of Pediatrics, University Hospital Heidelberg, Germany, and from 2011 to 2018, director of the Department of Translational Pulmonology at the Heidelberg University Medical School, Heidelberg and member of the Board of Directors of the German Center for Lung Research (DZL). He is an active member of several professional societies, including the European Cystic Fibrosis Society (ECFS), the European Respiratory Society (ERS) and the American Thoracic Society (ATS). He serves on journal editorial boards and on the Board of the ECFS. Dr. Mall qualified in Medicine at the University of Freiburg, Freiburg, Germany, and received his clinical training at the Universities of Freiburg and Heidelberg, and his postdoctoral training at the University of North Carolina at Chapel Hill, NC, USA, where he was appointed assistant professor of Medicine. In 2005, he received a grant from the European Commission to establish a Marie Curie Excellence Team for CF research at the University of Heidelberg, and in 2009 he was awarded the prestigious Heisenberg Professorship by the German Research Foundation. He is board certified in Pediatrics, Pediatric Pulmonology, Allergology and Infectious Diseases. Dr. Mall's research is focused on the molecular and cellular pathogenesis of CF and other chronic airway diseases, and the development of novel diagnostic approaches and therapeutic strategies. His research has been funded by the German Research Foundation (DFG), the German Ministry for Education and Research (BMBF), the European Commission, and others, and he has received several research awards. During his postdoctoral research, Dr. Mall developed a mouse with airway-specific overexpression of the epithelial sodium channel (βENaC), the first animal model with CF-like lung disease. His current research focus is on interdisciplinary translational research projects, integrating basic research with cohort studies and early phase clinical trials, to improve our understanding of CF lung disease and the translation of research results into the clinic.

####

About Arrowhead Pharmaceuticals, Inc.
箭头药品开发,通过沉默导致他们的基因治疗顽固性疾病的药物。使用RNA化学和递送的有效模式的广泛组合,箭头疗法触发RNA干扰机制来诱导靶基因的快速,深,且耐用击倒。RNA干扰或RNAi,是一种存在于活的抑制特定基因的表达,从而影响生产特定蛋白质的细胞机制。Arrowhead�s RNAi-based therapeutics leverage this natural pathway of gene silencing.

欲了解更多信息,请访问www.arrowheadpharma.com,或跟随我们的Twitter @ArrowheadPharma。要添加到该公司的电子邮件列表,并直接接收新闻,请访问:http://ir.arrowheadpharma.com/email-alerts.

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. 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 various factors and uncertainties, including the safety and efficacy of our product candidates, 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, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

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联系方式:
箭头制药公司
Vince Anzalone, CFA
626-304-3400


投资者和媒体:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578

www.lifesciadvisors.com

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