Arrowhead Pharmaceuticals Doses First Patient in SEQUOIA Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
SEQUOIA (NCT03945292) is a placebo-controlled, adaptive design Phase 2/3 study to evaluate the safety, efficacy, and tolerability of ARO-AAT administered subcutaneously to patients with AATD associated liver disease. SEQUOIA is designed to enroll 120 patients who will receive at least 9 doses, or approximately two years of treatment, with ARO-AAT or placebo. Doses will be administered on day 1, 29, and approximately every 12 weeks thereafter. The four-arm placebo-controlled Part A component of the study will feed seamlessly into a two-arm placebo-controlled Part B component. The primary objective for Part A is to select a single dose level for use in Part B of the study based on a combined evaluation of safety and pharmacodynamic dose response in each Part A cohort using change from baseline in soluble liver mutant AAT (Z-AAT), insoluble liver Z-AAT, and serum AAT levels as pharmacodynamic metrics. The primary objective for Part B is to evaluate efficacy, as assessed by the proportion of ARO-AAT treated patients relative to placebo achieving a 2-point improvement in a histologic grading scale of AATD associated liver disease, and no worsening of liver fibrosis on end of study biopsy.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA