Arrowhead presents new data on treatment of Cystic Fibrosis

Arrowhead announced the presentation of new preclinical data on ARO-ENaC, formerly referred to as ARO-Lung1, for the treatment of cystic fibrosis, or CF, at the American Thoracic Society, or ATS 2018. ARO-ENaC is the first product candidate to leverage the Targeted RNAi Molecule, or TRiM, platform targeting the pulmonary epithelium to address diseases of the lung. ARO-ENaC is an inhaled RNA interference therapeutic targeting the epithelial sodium channel alpha subunit, or alphaENaC, for the treatment of CF, a rare disease caused by a genetic mutation that leads to mucus buildup in the lungs and pancreas. CF patients can have difficulty breathing and experience frequent and persistent lung infections. Increased ENaC activity contributes to drying mucus in the airway and a reduced ability of the lung to clear toxins and infectious agents. Inheritance of poorly functioning ENaC genes by CF patients leads to milder lung disease. Researchers have been interested in developing therapeutics that decrease ENaC activity in CF patients. However, the development of inhaled small molecule inhibitors has been limited by their short duration of action and unwanted effects resulting from ENaC inhibition in the kidney. ARO-ENaC is designed specifically to address those deficiencies by selectively reducing ENaC in the lung, while sparing the kidney, with a long duration of effect

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