Arrowhead Pharma (NASDAQ:ARWR) dosed the first patient in its Phase 2 clinical study of ARO-AAT for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated liver disease.
AATD is a rare genetic disorder where the AAT protein is misfolded and accumulates inside liver cells, leading to fibrosis, cirrhosis and increased risk of liver cancer. Currently, the only cure for AAT deficiency is a liver transplant.
ARO-ATT is a subcutaneously administered RNA interference therapeutic designed to decrease production of mutant AAT proteins.
The study will assess changes in a novel histological activity scale in response to ARO-AAT in some 12 participants who can elect to continue treatment in an open-label extension (OLE). Including the OLE, changes in the novel histological grading scale will be assessed after six,12,18 and 24 months of treatment.
“[This] is an important study within our ARO-AAT development program, which provides us with a key opportunity to assess patient response to treatment at various timepoints,”
Dr. Javier San Martin, Arrowhead’s CMO, said in a statement.
“This may prove to be helpful for future regulatory and commercial planning as the ARO-AAT development program progresses,” he added.
