Arrowhead, Takeda Reach $1 Billion Deal for Rare Liver Disease Treatment

Arrowhead Pharmaceuticals and Japanese drugmaker Takeda have struck a $1 billion licensing deal to develop a new treatment for alpha-1 antitrypsin-associated liver disease (AATLD).

The investigational drug, ARO-AAT, is meant to reduce mutations of the alpha-1 antitrypsin protein, the leading cause of AALTD progression.

Alpha-1 antitrypsin-associated deficiency, a rare genetic disorder, afflicts approximately one in 3,000 to 5,000 in the U.S. and one in 2,500 in Europe. With no cure, liver transplants remain the standard of care.

Early data from a phase 2 trial of ARO-AAT in four individuals indicated that it may arrest liver harm without causing major adverse events in the lungs.