San Diego-based Lumena Pharmaceuticals on Friday announced the dosing of the first patient in the INDIGO Phase 2 clinical trial of its lead drug candidate, LUM001, in children with progressive familial intrahepatic cholestasis (PFIC).
Additionally, the CAMEO Phase 2 clinical trial of LUM001 in adults with primary sclerosing cholangitis (PSC) is open for enrollment.
LUM001 is being developed as a therapy for cholestatic liver diseases, including PFIC, PSC, Alagille syndrome and primary biliary cirrhosis.
These diseases result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage that may ultimately result in liver failure.
Severe itching is generally the most debilitating symptom afflicting children and adults with these diseases. LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles bile acids from the intestine to the liver.
Blocking bile acid transport in the intestine with ASBT inhibitors, such as LUM001, has the potential to lower bile acid levels in the body, slow disease progression, improve liver function and enhance the quality of life for patients suffering from cholestatic liver diseases.