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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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
