# Mirum Pharmaceuticals Meets Primary Endpoint in Phase 3 EXPAND Trial for Maralixibat

- URL: https://stocks.freetheai.org/news/mirum-pharmaceuticals-meets-primary-endpoint-in-phase-3-expand-trial-for-aec763
- Published: 2026-10-13T12:13:01Z
- Category: company_news; sentiment: bullish; impact: high
- Symbols: [NASDAQ:MIRM](https://stocks.freetheai.org/symbols/NASDAQ-MIRM)
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Mirum Pharmaceuticals, Inc. (MIRM) announced that its Phase 3 EXPAND study evaluating maralixibat (LIVMARLI) met its primary endpoint and key secondary endpoints in patients with cholestatic pruritus. In the 52-patient trial, maralixibat demonstrated a statistically significant improvement in itch severity scores compared to placebo, alongside a substantial reduction in serum bile acid levels.

The positive clinical data strengthens the therapeutic profile of maralixibat across cholestatic liver conditions. Beyond meeting the primary endpoint, the therapy achieved statistically significant outcomes across all five prespecified secondary measures, including key endpoints within the subgroup of patients with biliary atresia retaining their native liver. The safety findings remained consistent with earlier clinical experience, presenting no new safety concerns.

Looking ahead, Mirum Pharmaceuticals plans to submit a supplemental New Drug Application to U.S. regulators in the first half of 2027. Investors and healthcare stakeholders will be watching the regulatory submission process as the company seeks to expand the approved uses for the medication.

## Key points

- The Phase 3 EXPAND study met its primary endpoint, with maralixibat achieving a 1.81 itch score improvement versus 0.83 for placebo.
- Maralixibat reduced serum bile acids by 70.1% compared to 3.5% in the placebo group.
- All five prespecified secondary endpoints reached statistical significance, including key measures in the biliary atresia subgroup.
- Safety results were consistent with prior clinical trials, showing no new safety signals.
- Mirum Pharmaceuticals plans to submit a U.S. supplemental New Drug Application in the first half of 2027.

## Questions and answers

### What were the results of the Mirum Pharmaceuticals Phase 3 EXPAND trial?

The Phase 3 EXPAND trial met its primary endpoint, showing maralixibat significantly reduced itch severity and lowered serum bile acids by 70.1% compared to placebo, with no new safety signals observed.

### When does Mirum Pharmaceuticals plan to file for U.S. regulatory approval for maralixibat?

Mirum Pharmaceuticals plans to submit a supplemental New Drug Application for maralixibat to U.S. regulators in the first half of 2027.

### Did maralixibat meet secondary endpoints in the EXPAND study?

Yes, all five prespecified secondary endpoints achieved statistical significance, including three endpoints evaluated in the biliary atresia native liver subgroup.