Mirum Pharmaceuticals to Unveil Phase 3 Data for LIVMARLI in Rare…

Event summary

Mirum Pharmaceuticals will host an investor call on October 12, 2026, to share topline results from the Phase 3 EXPAND study of LIVMARLI® (maralixibat) for ultra-rare cholestatic liver diseases.
The study evaluates LIVMARLI® for cholestatic pruritus in patients aged 6 months or older, including those with biliary atresia.
Mirum Pharmaceuticals focuses on rare liver and genetic diseases, with a portfolio including LIVMARLI®, ATEBRIOZ™, CHOLBAM®, and CTEXLI®.
The company's pipeline includes volixibat for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug for chronic hepatitis delta virus (HDV), and MRM-3379 for Fragile X syndrome (FXS).

The big picture

Mirum Pharmaceuticals is positioning itself as a leader in rare disease therapeutics, with a focus on liver and genetic conditions. The upcoming Phase 3 data for LIVMARLI® could significantly enhance its market position, particularly in the treatment of ultra-rare cholestatic liver diseases. The company's strategic focus on high-impact medicines and disciplined execution aims to drive growth in a competitive biopharmaceutical landscape.

What we're watching

Clinical Efficacy: How the topline results from the Phase 3 EXPAND study will impact the regulatory pathway and commercial potential of LIVMARLI® in ultra-rare cholestatic liver diseases.
Market Expansion: Whether Mirum Pharmaceuticals can leverage positive data to expand the use of LIVMARLI® beyond its current indications in Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
Pipeline Progress: The pace at which Mirum Pharmaceuticals can advance its clinical-stage pipeline, including volixibat for PSC and PBC, brelovitug for HDV, and MRM-3379 for FXS.