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
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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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 future success of our scientific studies, our ability to successfully develop 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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