We develop solutions to complex diseases

Clinical development

Urea cycle disorders (UCDs) and hepatic encephalopathy (HE) are 2 distinct diseases that share a common problem: the body’s inability to effectively remove ammonia, a potent neurotoxin, through normal metabolic processes. Our development efforts continue in the area of UCDs, and we are planning to initiate phase III clinical trials in HE at the end of 2014.

— A UCD is a rare, inherited genetic defect caused by an enzyme deficiency in the urea cycle. In its most severe form, a UCD is fatal if left untreated. Learn more. »

— HE is a devastating and costly disease that may occur in patients with cirrhosis or acute liver failure. Our development program for HE will contribute vital new clinical insights into this devastating disease.

What's in the pipeline.