Arrowhead Pharmaceuticals Presents Preclinical Data on ARO-ENaC at the North American Cystic Fibrosi | ARWR Message Board Posts

Arrowhead Pharmaceuticals, Inc.

  ARWR website

ARWR   /  Message Board  /  Read Message

 

 






Keyword
Subject
Between
and
Rec'd By
Authored By
Minimum Recs
  
Previous Message  Next Message   Post Message   Post a Reply return to message boardtop of board
Msg  10073 of 10097  at  10/31/2019 12:20:15 PM  by

Patc


Arrowhead Pharmaceuticals Presents Preclinical Data on ARO-ENaC at the North American Cystic Fibrosis Conference

Arrowhead Pharmaceuticals Presents Preclinical Data on ARO-ENaC at the North American Cystic Fibrosis Conference
Business Wire Business Wire•October 31, 2019
PASADENA, Calif.--(BUSINESS WIRE)--

Arrowhead Pharmaceuticals Inc. (ARWR) today presented preclinical data at the 2019 North American Cystic Fibrosis Conference (NACFC) on ARO-ENaC, an inhaled RNAi therapeutic being developed as a potential treatment for cystic fibrosis (CF), which is a rare disease caused by genetic mutations that lead to mucus buildup in the lungs and pancreas. Arrowhead is currently conducting IND/CTA-enabling studies to support regulatory filings in the first half of 2020 for first-in-human studies.

Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead, said: “We view the epithelial sodium channel, ENaC, as an exciting target in the treatment of CF and one that is well suited to an RNAi-based intervention. Based on its mechanism of action, ARO-ENaC has the potential to provide clinical benefit to all patients with CF, regardless of genotype, and may prove useful in combination with existing or new CFTR-targeted therapies. The data presented today show that ARO-ENaC can accelerate mucociliary clearance in normal sheep and also preserve airway physiology in a sheep disease model of impaired mucociliary clearance. In addition, animal models indicate that ARO-ENaC can reduce ENaC activity in the lung but avoid impact in the kidney, which has historically been a key safety liability for prior investigational small molecule ENaC inhibitors.”

ARO-ENaC is designed to reduce production of the epithelial sodium channel alpha subunit (αENaC) in the airways of the lung. In patients with CF, increased ENaC activity contributes to airway dehydration and reduced mucociliary transport. In CF lung disease, patients can have difficulty breathing and experience frequent and persistent lung infections. Various human genetic studies have validated ENaC 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.

Key points presented at NACFC 2019 include the following:

Arrowhead's pulmonary epithelial TRiM™-enabled delivery platform increased potency of RNAi trigger-mediated silencing of αENaC mRNA, and durably reduced whole lung gene expression in rodents
In normal sheep, inhaled ARO-ENaC produced dose-dependent acceleration of mucociliary clearance, which is a lung physiology endpoint linked to pulmonary function in patients with CF
Inhaled ARO-ENaC preserves lung clearance in a sheep disease model of mucostasis
ARO-ENaC may offer a new renal-sparing, genotype-agnostic therapy for all patients with CF, and exhibiting the potential for an extended duration of action that should minimize treatment burden
Arrowhead is also expanding its pulmonary delivery platform to address additional lung disease targets, particularly those that are inaccessible to traditional small molecule or antibody approaches
A copy of the poster presentation can be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.


     e-mail to a friend      printer-friendly     add to library      
| More
Recs: 3     Views: 217
Previous Message  Next Message   Post Message   Post a Reply return to message boardtop of board






About Us  •  Contact Us  •  Follow Us on Twitter  •  Members Directory  •  Help Center  •  Advertise
Not a member yet? What are you waiting for? Create Account
Want to contribute? Support InvestorVillage by donating
© 2003-2019 Investorvillage.com. All rights reserved. User Agreement
   
Financial Market Data provided by
.


Loading...