Arrowhead Presents Updated Data from Phase 2 SEQUOIA Study of Investigational RN… – Press Release

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– Data Presented During Oral Session at EASL 2023 Demonstrate Fazirsiran’s Promising Impact on Key Markers of Liver Disease

– AATD-LD is a Rare Genetic Disease Affecting Children and Adults with no Approved Treatments

– Takeda and Arrowhead are Advancing Fazirsiran and Actively Enrolling in 160 patient Phase 3 REDWOOD Study

Arrowhead Pharmaceuticals Inc. ARWR today presented updated results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD). The SEQUOIA Phase 2 data are consistent with the promising results from an open-label Phase 2 trial of fazirsiran (AROAAT2002) that were published in The New England Journal of Medicine. Takeda TAK and Arrowhead are further investigating fazirsiran in the ongoing pivotal Phase 3 REDWOOD clinical study which is actively recruiting a total of 160 patients.

The updated Phase 2 clinical data were presented at the European Association for the Study of the Liver (EASL) Congress 2023 in an oral presentation titled, “Fazirsiran reduces liver Z-alpha-1 antitrypsin synthesis, decreases globule burden and improves histological measures of liver disease in adults with alpha-1 antitrypsin deficiency: a randomized placebo-controlled phase 2 study,” which may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

“The clinical results from the Phase 2 SEQUOIA study of fazirsiran were clear and compelling. Fazirsiran treatment demonstrated a substantial effect on several key markers of liver disease,” said Javier San Martin, M.D., chief medical officer at Arrowhead. “Together with our partners at Takeda, we are now conducting the Phase 3 REDWOOD study to further investigate fazirsiran’s potential in patients with AATD-LD and F2 to F4 liver fibrosis.”

Key findings from the SEQUOIA study include the following:

  • Fazirsiran reduced serum Z-AAT concentration in a…

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