The strong Phase 3 data across all endpoints de-risks the BA regulatory submission and meaningfully expands LIVMARLI's commercial opportunity into a new indication. Monitor for sNDA acceptance and potential accelerated review in H1 2027.
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Executive Summary
Mirum Pharmaceuticals announced positive topline results from the Phase 3 EXPAND study of LIVMARLI (maralixibat) in ultra-rare cholestatic liver diseases, including biliary atresia. The study met its primary endpoint and all five prespecified secondary endpoints with high statistical significance. The company plans to submit a supplemental New Drug Application in the first half of 2027.
Key Facts
- Phase 3 EXPAND study met primary endpoint (pruritus improvement) with p=0.0004
- All five prespecified secondary endpoints achieved statistical significance
- Maralixibat showed 70.1% reduction in serum bile acids vs 3.5% for placebo (p<0.0001)
- In biliary atresia with native liver subgroup: 96.4% of days with pruritus improvement vs 52.2% placebo (p=0.0045)
- No new safety signals observed; safety profile consistent with prior experience
- sNDA submission planned for first half of 2027
- Addressable BA patient population estimated at ~2,000 in US and Europe
Financial Impact
Expands LIVMARLI's addressable market by ~2,000 BA patients in US/Europe; existing ALGS market opportunity estimated at >$500M; PFIC adds ~1,000 pediatric and >4,000 adult patients
Risk Factors
- Regulatory submission still ~6-9 months away; FDA may request additional data
- Small study size (n=52) may raise questions about generalizability
- Competition from other pruritus treatments in cholestatic liver disease
Investment Themes
Documents Analyzed
This report is based on 4 SEC documents filed with EDGAR.
| Document | Accession Number |
|---|---|
| 8-K Filing (Primary) | 0001759425-26-000051 |
| Document: 0001759425-26-000051-index-headers.html | 0001759425-26-000051 |
| Document: 0001759425-26-000051-index.html | 0001759425-26-000051 |
| Document: 0001759425-26-000051.txt | 0001759425-26-000051 |
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
Oct 26, 2026 today | 8-K | — | awaiting T+60 | — | — |
Oct 13, 2026 13d ago | 8-K | $79.62 | awaiting T+60 | — | $80.39 (+0.97%) |
Sep 28, 2026 28d ago | 8-K | $87.96 | awaiting T+60 | — | $80.39 (−8.61%) |
Aug 19, 2026 9w ago | Insider Cluster | $97.18 | awaiting T+60 | — | $80.39 (−17.28%) |
Aug 12, 2026 10w ago | Insider Cluster | $98.77 | awaiting T+60 | — | $80.39 (−18.61%) |
Aug 11, 2026 10w ago | Insider Cluster | $98.97 | awaiting T+60 | — | $80.39 (+18.77%) |
Aug 10, 2026 11w ago | 144 | $98.23 | awaiting T+60 | — | $80.39 (−18.16%) |
Aug 5, 2026 11w ago | 8-K | $94.12 | awaiting T+60 | — | $80.39 (−14.58%) |
Jul 8, 2026 15w ago | Insider Cluster | $127.70 $82.93 | ▼ −35.06% | ▼ −37.44% | $80.39 (−37.05%) |
Jun 11, 2026 19w ago | Institutional Cluster | $99.54 $100.90 | ▲ +1.37% | ▼ −3.03% | $80.39 (−19.24%) |
US Market Status
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