Arrowhead's ARO-AAT Treatment Associated With Improved Fibrosis In Patients With Alpha-1 Liver Disease

Arrowhead Pharmaceuticals Inc ARWR has announced positive interim 48-week liver biopsy results from the Phase 2 AROAAT2002 study evaluating ARO-AAT for rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
AATD is the lack of a protein made by the liver released into the bloodstream. AATD is a genetic disease that can affect the liver or lung. The alpha-1 protein is designed to protect tissues in the body from being attacked by its own enzymes.
The company co-develops this second-generation investigational RNA interference (RNAi) therapeutic with Takeda Pharmaceutical Co Ltd TAK.
The results demonstrated that ARO-AAT treatment led to a consistent and substantial reduction in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer; a consistent decrease in histological globule burden; improvements in fibrosis; and improvements in other relevant biomarkers of liver health.
Four of the five patients achieved a 1 or greater stage improvement in the Metavir fibrosis stage, with no worsening of fibrosis in the fifth patient.
All five patients demonstrated reductions in histological globule assessment scores.
Total intra-hepatic Z-AAT decreased by 77-97%.
After 24 weeks of treatment with ARO-AAT in cohort 1 (n=4), two of the four patients achieved a 1 or greater stage improvement in the Metavir fibrosis stage, with no worsening of fibrosis in the other two patients.
Arrowhead intends to present additional interim AROAAT2002 data at an upcoming medical congress.
Price Action: ARWR shares are up 1.88% at $75.92 during market trading on the last check Wednesday.

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