Lumena Begins PBC Trial

Lumena Pharmaceuticals has begun dosing the first patient in the Phase II trial of its drug candidate LUM001 in patients with primary biliary cirrhosis (PBC). LUM001 is being developed as a possible therapy for a number of cholestatic liver diseases, including PBC, Alagille syndrome, progressive familial intrahepatic cholestasis and primary sclerosing cholangitis, that result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage and, ultimately, liver failure. In the Phase II study, LUM001 is being evaluated for its ability to alleviate the symptom of severe itching associated with PBC.

LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles intestinal bile acids back into the circulation. By reducing serum bile acids with a once-daily oral tablet, LUM001 may offer a novel therapeutic approach for alleviating the severe itching and progressive liver damage associated with many cholestatic liver diseases.

The Phase II study is a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of LUM001 in combination with ursodeoxycholic acid (UDCA) versus UDCA plus placebo in adults with PBC. This international, multi-center study will enroll 60 adult patients experiencing moderate to severe pruritus (itching). The primary endpoint of the study is change from baseline in pruritus compared to placebo. Secondary endpoints include changes from baseline in liver enzymes and other biochemical markers of cholestatic liver disease compared to placebo.

LUM001 has received Orphan Drug Designation from the FDA for four rare cholestatic liver diseases: primary biliary cirrhosis, progressive familial intrahepatic cholestasis, Alagille syndrome, and primary sclerosing cholangitis. The company has also initiated a global clinical program to study LUM001 in children with Alagille syndrome and will initiate Phase II studies in patients with progressive familial intrahepatic cholestasis, and primary sclerosing cholangitis in late 2013.