• Arrowhead Pharma Shares Soar on Early Liver Data for RNAi Drug

    11 days ago - By Xconomy

    An Arrowhead Pharmaceuticals drug for a rare liver disease now has early Phase 2 results and the data so far look good. Not only did the therapy reduce levels of a mutant protein associated with the disorder, but patients also showed improvements in several biological measures of liver injury. The results are for just four patients from a small 16-patient study testing the drug, ARO-AAT, as a treatment for liver disease associated with alpha-1 antitrypsin deficiency. But the early results are an encouraging sign for Arrowhead and its approach to treating inherited diseases by stopping...
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