In patients struggling with liver damage and impaired liver function, it is important that drug treatments avoid causing additional harm to the liver. LUM001 was specifically designed to have limited systemic exposure and targets the Apical Sodium-dependent Bile Acid Transporter (ASBT), which is localized on the luminal surface of the lower portion of the small intestines. The ASBT is responsible for recycling bile acids back to the liver. Inhibiting the ASBT may mimic the effects of successful surgical approaches that reduce bile accumulation in the liver, thereby improving liver function and symptoms of disease.

Extensive toxicology studies have demonstrated that LUM001 carries no increased liver toxicities, and clinical studies of LUM001 show that it reduces serum bile acid levels. In addition to improving liver function, reducing serum bile acid levels may reduce disease symptoms, such as extreme itching, in patients with cholestatic liver disease. LUM001 was originally developed as a cholesterol lowering drug. The compound advanced into Phase II clinical development and more than 1,400 relatively healthy study subjects received the drug. After more than 150 patient-years of exposure, LUM001 was shown to be generally safe and well tolerated.

Lumena is leveraging more than 10 years of experience in preclinical and clinical development with LUM001 to initiate Phase II clinical studies in pediatric and adult patients with several different types of cholestatic liver disease. Lumena is currently enrolling adult patients with primary biliary cirrhosis (PBC), a chronic disease that causes the bile ducts in the liver to become inflamed and damaged, in a Phase II clinical study of LUM001. The company is also currently enrolling pediatric patients in a Phase II clinical study of LUM001 in children with Alagille syndrome (ALGS), a rare congenital disorder that is also present with cholestatic liver disease. Find out more about current trials here. Lumena plans to initiate a Phase II clinical study of LUM001 in Progressive Familial Intrahepatic Cholestasis (PFIC), and a proof of concept trial in patients with primary sclerosing cholangitis (PSC).