LIVMARLI Clears All Phase 3 EXPAND Hurdles; Mirum Eyes sNDA in H1 2027
MIRM sits 36% above its 52-week low of $63.232.
Summary
LIVMARLI met its primary endpoint and all five secondary endpoints in the Phase 3 EXPAND study for ultra-rare cholestatic liver diseases, with an sNDA planned for H1 2027.
Key Events · Product Development and Regulatory · MIRM
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Phase 3 EXPAND Meets All Endpoints
LIVMARLI achieved a placebo-adjusted 0.98-point improvement in ItchRO(Obs) severity (p=0.0004) and a 69.0% treatment effect on serum bile acids (p<0.0001) in the primary pediatric population.
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Biliary Atresia Subgroup Shows Stronger Efficacy
In BA patients with native liver, LIVMARLI showed a 1.15-point placebo-adjusted itch improvement (p=0.0002) and a 73.1% treatment effect on serum bile acids (p<0.0001).
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sNDA Submission Planned for H1 2027
Mirum plans to submit a supplemental New Drug Application in the US in the first half of 2027 based on the EXPAND results, targeting label expansion into ultra-rare cholestatic liver diseases.
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Safety Profile Consistent with Prior Experience
No new safety signals observed. Diarrhea was the most common adverse event (64.0% maralixibat vs 11.1% placebo); Grade 3+ TEAEs were similar between arms (24.0% vs 22.2%).
Analysis · MIRM · Life Sciences
The Phase 3 EXPAND study delivered on its primary endpoint and all five prespecified secondary endpoints, with a placebo-adjusted 0.98-point improvement in itch severity (p=0.0004) and a 69% treatment effect on serum bile acids (p<0.0001). Results were even stronger in the biliary atresia subgroup, which showed a 1.15-point placebo-adjusted itch improvement. This validates LIVMARLI's expansion beyond its current ALGS and PFIC indications into ultra-rare cholestatic liver diseases, including biliary atresia — a population with no approved therapies. An sNDA submission is planned for the first half of 2027, positioning LIVMARLI for label expansion into a market the company estimates at over $500 million for ALGS alone, plus ~2,000 addressable BA patients. The safety profile was consistent with prior experience, with diarrhea as the most common adverse event (64% vs 11.1% placebo) but no new safety signals.
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At the time of this filing, MIRM was trading at $86.20 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $5.6B. The 52-week trading range was $63.23 to $130.00. This filing was assessed with positive market sentiment and an importance score of 8 out of 10.