Mirum Pharmaceuticals Announces Positive Phase 3 LIVMARLI Data for Cholestatic Liver Diseases

Mirum Pharmaceuticals announced positive topline results from its Phase 3 EXPAND study for LIVMARLI (maralixibat) in treating cholestatic pruritus (itchiness) in patients with ultra-rare cholestatic liver diseases. The study successfully met its primary endpoint and all five secondary endpoints, demonstrating a statistically significant improvement in symptoms.

Key Details

• Primary Endpoint Met: The study showed a statistically significant improvement in pruritus, with maralixibat achieving a placebo-adjusted mean improvement of 0.98 points on the ItchRO(Obs) severity score (p=0.0004).
• Secondary Endpoint Success: Treatment with maralixibat resulted in a 70.1% reduction from baseline in serum bile acids (sBA), compared to 3.5% for placebo (p<0.0001).
• Regulatory Submission Planned: Based on these positive results, the company plans to submit a supplemental New Drug Application (sNDA) in the United States in the first half of 2027.