Takeda (TKPHF.PK, TAK) and Arrowhead Pharmaceuticals Inc. (ARWR) Monday announced positive results from the Phase 2 Sequoia clinical study of investigational fazirsiran for the treatment of liver disease associated with alpha-1 antitrypsin deficiency.
The companies said the Sequoia study has shown that patients with baseline fibrosis who received 25 mg, 100 mg, or 200 mg of fazirsiran demonstrated a mean reduction in serum mutant alpha-1 antitrypsin protein concentration.
The study also showed that PAS-D globule burden was reduced from a baseline mean of 5.9 to a post-baseline mean of 2.3 at the postbaseline liver biopsy visit. Improvement in portal inflammation was observed in 42 percent and 50 percent of patients achieved an improvement in fibrosis of at least one point by METAVIR stage.
Researchers at the University of Florida said therapies that block protein production may allow the liver to clear the toxic protein and potentially heal.
Further, Takeda plans to initiate TAK-999-3001 Phase 3 study to evaluate the efficacy and safety of fazirsiran in the treatment of alpha-1 antitrypsin deficiency-associated liver disease with METAVIR stage F2 to F4 fibrosis.
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