Lumena Launches Pediatric ALGS Trial

Lumena Pharmaceuticals is testing LUM001 in children with Alagille syndrome (ALGS). The first patient has been dosed in the IMAGO Phase II study being conducted in the UK, and enrollment of pediatric patients is expected to begin later this year in the ITCH Phase II study in the U.S. The IMAGO study is a 13 week placebo-controlled study to evaluate the safety and efficacy of LUM001 in children with ALGS. The UK-based study will enroll children, two years of age and older, with ALGS.

ALGS is a rare form of cholestatic liver disease that results in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage that can cause liver failure. LUM001 is being evaluated for its ability to lower serum bile acids and alleviate severe itching, which is generally the most debilitating symptom afflicting patients with the disease. By reducing serum bile acids with a once-daily oral solution, LUM001 may offer a novel therapeutic approach to prevent bile acid mediated liver damage and alleviate severe itching.

Lumena also received Orphan Drug Designation by the FDA’s Office of Orphan Product Development for LUM001. The Orphan Designation coversfour rare cholestatic liver diseases including Alagille syndrome (ALGS); progressive familial intrahepatic cholestasis (PFIC); primary biliary cirrhosis (PBC); and primary sclerosing cholangitis (PSC).