Benzinga | Apr 28, 2021 11:47AM EDT

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

* Arrowhead Pharmaceuticals Inc (NASDAQ: 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 (NYSE: 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.