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Mirum reports Phase 3 EXPAND study met primary endpoint for LIVMARLI in ultra-rare cholestatic liver diseases
MIRMMirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals announced that the Phase 3 EXPAND study of LIVMARLI (maralixibat) for treating cholestatic pruritus in patients aged six months or older with ultra-rare cholestatic liver diseases, including biliary atresia, met its primary endpoint and achieved statistical significance across all five prespecified secondary endpoints. The trial enrolled 52 patients randomized to maralixibat or placebo during a 20-week double-blind period. Maralixibat demonstrated a statistically significant improvement in pruritus compared with placebo, showing a mean 1.81-point reduction from baseline versus 0.83 points for placebo, and a 70.1% reduction in serum bile acids versus 3.5% for placebo. The company plans to submit a supplemental New Drug Application in the United States in the first half of 2027 based on these results. Mirum Pharmaceuticals operates as a biopharmaceutical company focused on developing treatments for rare liver diseases.