Arrowhead Pharmaceuticals, Inc. have announced that they have completed patient enrollment for a Phase 1 clinical trial investigating ARO-AAT, an investigational therapy for the treatment of a genetic liver disease associated with alpha-1 antitrypsin deficiency. The full article can be found here, at Business Wire.
The experimental therapy is planned to be tested in a Phase I clinical study called AROAAT1001. It is a single and multiple ascending dose study designed to investigate how safe and tolerable the therapy is in healthy adults. There will be seven cohorts that receive the therapy at doses of 35mg, 100mg, 200mg, and 300mg. Some volunteers will be given a placebo, while others will take ARO-AAT.
ARO-AAT is a second generation RNA interference (RNAi) therapeutic. RNAi techniques are often used to turn off, or ‘silence’ certain genes. ARO-AAT is believed to function by reducing the production of the Z-AAT protein, which is involved in the liver disease experienced by some patients with alpha-1 antitrypsin deficiency. This may stop the liver disease from progressing, and even allow it to repair. ARO-AAT is subcutaneously administered, which means it is administered underneath the dermis and epidermis skin layers.
Alpha-1 antitrypsin deficiency (A1AD), the condition ARO-AAT is being developed to treat, is a genetic condition linked to lung and liver disease. The condition may cause early symptoms such as wheezing and shortness of breath after low levels of exercise. A1AD can also cause weight loss, fatigue, a raised heart rate after standing, and frequent respiratory infections. An estimated 10% of infants with A1AD also develop liver disease. A1AD affects approximately 1 in every 1,500 to 3,500 people.