Drug intelligence / Profile preview

LUM001 + ursodeoxycholic acid

Development stage
Phase 2
Lead developer
Mirum Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

This is a **combination therapy** consisting of maralixibat (LUM001), a potent and selective inhibitor of the ileal bile acid transporter (IBAT; also known as apical sodium-dependent bile acid transporter or ASBT), and ursodeoxycholic acid (UDCA), a hydrophilic bile acid commonly used as first-line therapy for cholestatic liver diseases. Maralixibat decreases reabsorption of bile acids in the terminal ileum, thus lowering systemic bile acid levels, while UDCA helps protect cholangiocytes and improves bile flow. This combination is being studied to treat cholestatic pruritus and other symptoms in diseases such as **primary biliary cholangitis (PBC)** and **Alagille syndrome (ALGS)** in patients who often remain symptomatic despite existing UDCA monotherapy[1][7]. LUM001 is orally administered and is under investigation in multiple clinical studies, including trials testing its efficacy in patients already taking UDCA[7].

Other names
maralixibat + ursodeoxycholic acid
02

Targets

AKR1C2 (Aldo-keto reductase family 1 member C2)SLC10A2 (Solute carrier family 10 member 2)BLVRA (Biliverdin reductase A)FXR (Farnesoid x-activated receptor)SLC23A2 (Sodium-dependent vitamin C transporter 2)

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