Mirum says Phase 3 EXPAND study of Livmarli met its primary endpoint
Mirum Pharmaceuticals announced topline results from the Phase 3 EXPAND study of Livmarli (maralixibat) in ultra-rare cholestatic liver diseases, saying the study met its primary endpoint. In the pediatric primary analysis, pruritus improved 1.81 points from baseline on maralixibat versus 0.83 points on placebo, and the company plans to submit a U.S. supplemental New Drug Application in the first half of 2027.
Key points
- Primary endpoint: 1.81-point improvement on maralixibat vs. 0.83 on placebo, a placebo-adjusted difference of 0.98 points (p=0.0004)
- Serum bile acids fell 70.1% on maralixibat vs. 3.5% on placebo, a 69.0% treatment effect (p<0.0001)
- No new safety signals; diarrhea reported in 64.0% of maralixibat patients vs. 11.1% on placebo
- Supplemental New Drug Application planned in the United States in the first half of 2027
The filing
Written by AI on Nov 9, 2026, 11:43 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on October 13, 2026.
Briefs are written by AI from the filing text and can contain mistakes — open the filing before acting on anything. Impact (1-5) and tone are the AI's reading of the filing for shareholders, not a price forecast. Not investment advice.