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Mirum Pharmaceuticals Meets Primary Endpoint in Phase 3 Trial of LIVMARLI for Rare Liver Diseases

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MIRM$86.01 5.73%

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) announced positive topline results from its Phase 3 EXPAND study evaluating LIVMARLI (maralixibat) in patients aged six months and older with ultra-rare cholestatic liver diseases, including biliary atresia. The 52-patient trial met its primary endpoint, showing a statistically significant reduction in severe itching compared to placebo, while also achieving all five prespecified secondary endpoints.

The trial demonstrated a mean 1.81-point improvement in worst-daily itch severity scores for patients treated with maralixibat, compared to 0.83 points for placebo. Additionally, treated patients experienced a 70.1% reduction in serum bile acids compared to a 3.5% reduction in the placebo group. Safety results were consistent with previous clinical experience with no new safety issues identified.

Following these results, the company plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration in the first half of 2027. Investors will watch for regulatory submission updates as Mirum seeks to expand approval for LIVMARLI beyond its existing pediatric indications.

Key points

  • The Phase 3 EXPAND trial of LIVMARLI met its primary endpoint and all five prespecified secondary endpoints in ultra-rare cholestatic liver diseases.
  • Pediatric patients taking maralixibat showed a 1.81-point improvement in itch severity scores versus 0.83 points for placebo.
  • Serum bile acids dropped 70.1% in the maralixibat group compared to 3.5% for patients receiving placebo.
  • Mirum Pharmaceuticals plans to file a supplemental New Drug Application in the United States in the first half of 2027.

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Questions and answers

What were the Phase 3 results for Mirum Pharmaceuticals' LIVMARLI study?

The Phase 3 EXPAND study met its primary endpoint with significant itch reduction in pediatric patients and achieved all five secondary endpoints, alongside a 70.1% drop in serum bile acids compared to 3.5% for placebo.

When does Mirum Pharmaceuticals plan to file for FDA approval for LIVMARLI's new indication?

Mirum plans to submit a supplemental New Drug Application in the United States in the first half of 2027 based on the Phase 3 EXPAND trial results.

What conditions was LIVMARLI tested for in the Phase 3 EXPAND trial?

The trial evaluated LIVMARLI in patients aged six months and older suffering from ultra-rare cholestatic liver diseases, including biliary atresia.