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 (NASDASQ: ARWR) and its approach to treating inherited diseases by stopping genes from producing… Read more »