Enanta Announces Results of INTREPID Study of EDP-305 for the Treatment of Primary Biliary Cholangitis - Seite 3

“Based on these data, it’s clear that EDP-305 demonstrated evidence of target engagement with robust reductions in markers of liver injury,” said Kris Kowdley, M.D., FACP, FACG, AGAF, FAASLD, Director, Liver Institute Northwest and Clinical Professor, Elson S. Floyd College of Medicine, Washington State University, the Principal Investigator for the study. “These data suggest that a dose between 1.0 mg and 2.5 mg could hit an optimal level of efficacy and tolerability, which could bode well for future studies in NASH. I look forward to seeing future data on EDP-305.”

About Primary Biliary Cholangitis

Primary biliary cholangitis (PBC) is a chronic disease of the liver that slowly destroys the medium-sized bile ducts within the liver. Bile is a digestive liquid that is made in the liver. It travels through the bile ducts to the small intestine, where it helps digest fats and fatty vitamins.

In patients with PBC, the bile ducts are destroyed by inflammation. This causes bile to remain in the liver, where its increased levels gradually damage liver cells and cause cirrhosis or scarring of the liver. As cirrhosis progresses and the amount of scar tissue in the liver increases, the liver loses its ability to function, leading to potential liver failure, liver transplantation or hepatocellular carcinoma. PBC affects mostly women, but more men are now being diagnosed. The disorder usually becomes apparent during middle age, initially affecting most individuals between the ages of 45 to 65 years. However, the disorder has been diagnosed in females as young as 22 years of age and in females in their early 90s. It has been estimated that PBC is one of the most common autoimmune diseases, affecting nearly 1 in 1000 women over the age of 40.¹

About EDP-305, a Farnesoid X Receptor Agonist

EDP-305 is a potent Farnesoid X receptor (FXR) agonist and Enanta’s lead product candidate being developed primarily for the treatment of NASH. FXR is a nuclear receptor and a main regulator of bile acid levels in the liver and small intestine. It responds to bile acids by regulating gene transcription of key enzymes and transporters, many of which play important roles in lipid metabolism, insulin resistance, inflammation, and fibrosis. EDP-305 represents a class of FXR agonists that has been designed to take advantage of increased binding interactions with the receptor. This non-bile acid class contains steroid and non-steroid components and does not contain the carboxylic acid group normally present in other classes of FXR agonists and natural bile acids that can lead to the formation of taurine and glycine conjugates.