Press release
Arrowhead Pharmaceuticals to Host Key Opinion Leader Webinar on ARO-ENaC for Treatment of Cystic Fibrosis
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that on July 28, 2020, at 12:00 p.m. EDT it will host a key
About this update from Arrowhead Pharmaceuticals, Inc.
[{"type":"text","content":"\n \n\n PASADENA, Calif.--(BUSINESS WIRE)--\nArrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that on July 28, 2020, at 12:00 p.m. EDT it will host a key opinion leader webinar on ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF).\n\n\nThe webinar will feature a presentation by Marcus Mall, M.D., Professor and Director of the Department of Pediatric Pulmonology and Immunology at The Charité University Medical Center Berlin, who will discuss the current treatment landscape and unmet medical need in treating patients with CF. Arrowhead management will also provide a review of the ARO-ENaC program, which utilizes Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform and is the company’s first inhaled RNAi candidate to target pulmonary epithelium. The webinar may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.\n\n\nCF is a rare disease caused by genetic mutations in the CFTR gene that lead to progressive deterioration in lung function due to poor clearance of mucus and associated recurrent infections. ARO-ENaC is designed to reduce activity of the epithelial sodium channel alpha subunit (αENaC) in the airways of the lung. In patients with CF, CFTR dysfunction causes increased ENaC activity which contributes to airway dehydration and reduced mucociliary transport. This predisposes patients to persistent lung infections, structural damage, and progressive loss of pulmonary function. ENaC has been extensively explored as a potential therapeutic target for CF, but the development of inhaled small molecule ENaC inhibitors has been limited by on-target renal toxicity and short duration of action in the lung.\n\n\nArrowhead is initiating AROENaC1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, and pharmacokinetic effects of ARO-ENaC in normal healthy volunteers and to evaluate the safety and tolerability and explore efficacy in patients with CF. Exploratory objectives in patients with CF include assessing the effects of ARO-ENaC on changes in lung clearance index (LCI) and evaluating changes in forced expiratory volume (FEV1).\n\n\nMarcus Mall, M.D., is Professor and Director of the Department of Pediatric Pulmonology and Immunology at The...