Arrowhead Pharmaceuticals (NASDAQ:ARWR) jumps 10% on positive interim 24-week liver biopsy results in four subjects from AROAAT2002, an open-label Phase 2 study of ARO-AAT, the second generation investigational RNA interference (RNAi) therapeutic being developed as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). The study assesses ~16 patients.
Serum and total intra-hepatic mutant AAT protein (Z-AAT) decreased in all four patients by up to 93% and 95%, respectively.
Three of four patients demonstrated reductions in intra-hepatic Z-AAT polymer, with a maximum reduction of 97%.
Maximum reductions of 66% in circulating alanine aminotransferase (ALT) and 58% in gamma-glutamyl transferase (GGT) observed in all four patients.
All patients demonstrated improved transient elastography FibroScan values, with three of four patients exhibiting greater than 20% reductions.
“These data are very encouraging and suggest that ARO-AAT may rapidly ameliorate liver injury. It is particularly reassuring to see the decrease in liver enzymes, which suggests that elevations are related to proteotoxic stress that could be addressed with ARO-AAT therapy rather than reflecting co-morbidities,” said Professor Pavel Strnad, M.D., University Hospital Aachen, Germany, and an investigator on the trial.
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