Mirum reports LIVMARLI Phase 3 trial success

• Mirum Pharmaceuticals (NASDAQ:MIRM) reported that LIVMARLI (maralixibat) met the primary endpoint in the Phase 3 EXPAND study evaluating treatment for cholestatic pruritus in rare liver diseases.
• The trial showed a statistically significant improvement in itching severity and reduced serum bile acids compared with placebo among pediatric patients.
• Mirum Pharmaceuticals said it plans to submit a supplemental New Drug Application in the United States during the first half of 2027 based on EXPAND results.

Mirum Pharmaceuticals (NASDAQ:MIRM) announced topline results from the Phase 3 EXPAND study evaluating LIVMARLI (maralixibat) in patients aged 6 months and older with ultra-rare cholestatic liver diseases, including biliary atresia.

The study enrolled 52 patients, including 46 pediatric and six adult patients, who were randomized during a 20-week double-blind period to receive maralixibat 285 µg/kg twice daily or placebo.

“These results are especially important because patients with ultra-rare cholestatic liver diseases continue to face a significant burden from pruritus,” said Rob Myers, M.D., chief medical officer at Mirum Pharmaceuticals.

Maralixibat produced a mean 1.81-point improvement from baseline in average worst-daily ItchRO(Obs) severity score during Weeks 13–20 compared with a 0.83-point improvement for placebo, resulting in a placebo-adjusted difference of 0.98 points (p=0.0004).

The company reported a 70.1% reduction from baseline in serum bile acids with maralixibat compared with a 3.5% reduction for placebo, while safety findings remained consistent with previous clinical experience.

Mirum Pharmaceuticals develops therapies for rare diseases, including liver disorders, and plans to use the EXPAND data to support potential regulatory expansion of LIVMARLI.