Arrowhead Gets FDA Clearance to Begin Phase 2/3 Study for Liver Disease Med

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has received clearance from the U.S. Food and Drug Administration to proceed with an adaptive Phase 2/3 trial with the potential to serve as a pivotal registrational study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).

Arrowhead intends to initiate the adaptive design, Phase 2/3 study of ARO-AAT in patients with AATD associated liver disease at various sites in the U.S. in the second quarter of 2019, followed by various international sites in Europe, pending regulatory submission and review. The proposed primary objectives are to evaluate safety and pharmacodynamic dose response, and to evaluate efficacy, defined as an improvement in a histologic grading scale of AATD associated liver disease, and no worsening of liver fibrosis based on Ishak score on end of study biopsy. The company plans to provide additional study details following its initiation.

https://www.biospace.com/article/releases/arrowhead-pharmaceuticals-receives-fda-clearance-to-begin-phase-2-3-study-of-aro-aat-for-treatment-of-alpha-1-liver-disease/