other material
confidence high
sentiment positive
materiality 0.75
Mirum LIVMARLI Phase 3 EXPAND meets primary endpoint; itch score -0.98 vs placebo, sNDA set for H1 2027
Mirum Pharmaceuticals, Inc.
- Phase 3 EXPAND enrolled 52 patients (46 pediatric, 6 adult); ItchRO(Obs) improved 1.81 points vs 0.83 for placebo, a 0.98-point difference (p=0.0004).
- All five prespecified secondary endpoints met, including three in biliary atresia with native liver: itch -2.16 vs -1.01 (p=0.0002) and sBA -75.5% vs -9.0% (p<0.0001).
- Serum bile acids fell 70.1% from baseline vs 3.5% for placebo in the overall population, a 69.0% treatment effect (p<0.0001).
- Safety consistent with prior maralixibat experience; diarrhea in 64.0% of treated vs 11.1% placebo patients, no deaths and no new safety signals.
- Mirum plans a U.S. supplemental New Drug Application in H1 2027; sees ~2,000 addressable BA patients in U.S./Europe.