Arrowhead Pharmaceuticals Doses First Patient in AROAAT2002 Open Label Phase 2 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
AROAAT2002 (NCT03946449) is a pilot open-label, multi-dose, Phase 2 study to assess changes in a novel histological activity scale in response to ARO-AAT in patients with AATD associated liver disease. Approximately 12 participants will be enrolled in two sequential cohorts. All eligible participants will require a pre-dose biopsy and an end of study biopsy. Treated participants will also be offered the opportunity to continue treatment in an open-label extension (OLE). Including the OLE, changes in the novel histological grading scale will be assessed after 6 months, 12 months, 18 months, and 24 months of treatment with ARO-AAT.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA