Arrowhead and Takeda Report P-II Study (SEQUOIA) of Fazirsiran for Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
Shots:
- The P-II study evaluates fazirsiran vs PBO in 42 patients which showed a dose-dependent mean reduction in serum mutant alpha-1 antitrypsin protein (Z-AAT) concentration at (25/100/200mg) dose were (74%/89%/94%) @48wk. who had baseline fibrosis vs no change
- Reduction in total liver Z-AAT vs 26% increase, 94% median reduction at the postbaseline liver biopsy visit, 68% in histologic globule burden vs no change in PAS-D globule burden, worsening (7% vs 22%), improvement in portal inflammation & fibrosis (42% vs no change) & (50% vs 38%)
- The therapy was well tolerated with no treatment discontinuation due to TEAEs with no dose interruptions, or premature study withdrawals & the results were consistent with the P-II study. The therapy is being advanced in the planned P-III study
Ref: Takeda | Image: Takeda
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