Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease at Liver Meeting® 2017
Data Presented in Late Breaking Poster
Company Expects to File Clinical Trial Application in First Quarter 2018 to Begin First-in-Human Studies of ARO-AAT
The poster presentation, titled, "Subcutaneous delivery of a RNA interference (RNAi) therapeutic candidate for alpha-1 antitrypsin deficiency (AATD)-related liver disease produces deep and prolonged knockdown of plasma AAT," highlighted the results of studies in which ARO-AAT produced deep and prolonged knockdown of alpha-1 antitrypsin (AAT) to levels that appear to be near full suppression of the liver production. The efficacy of ARO-AAT was evaluated in the transgenic PiZ mouse model and in nonhuman primates (NHPs) by measuring the reduction in plasma or serum levels of AAT. Inhibition of synthesis and subsequent reduction of the mutant Z-AAT accumulation in the livers of patients with alpha-1 liver disease may lead to the prevention, and potential reversal, of liver injury.
Preliminary safety of ARO-AAT was also assessed in rats and NHPs at dose levels up to and including 300 mg/kg, which is approximately 100 times the expected human clinical dose. In the rat study, clinical laboratory values were indistinguishable between groups receiving ARO-AAT and control groups. In addition, there were no histopathology findings deemed to be related to ARO-AAT. In the NHP study, there were no abnormal clinical observations, body weight changes, clinical chemistries, nor organ weight findings noted.
Subcutaneous delivery of a RNA interference (RNAi) therapeutic candidate for alpha-1 antitrypsin deficiency (AATD)-related liver disease produces deep and prolonged knockdown of plasma AAT
- Publication Number: LB-21
- Session: Late-Breaking Poster Session
Session Date and Time:
October 23, 2017, from 8:00 AM to 5:30 PM
Christine I. Wooddell, et al.
A copy of the poster may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
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