Mirum Pharmaceuticals, Inc. MIRM
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) is a GI/hepatology drug developer. Its lead program, LIVMARLI (maralixibat), is in Phase 3 for Cholestatic pruritus in Alagille syndrome and PFIC. Its next catalyst on record is Volixibat: PBC topline data (Q1 2027).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: Volixibat — PBC topline data (Q1 2027). 6 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| LIVMARLI (maralixibat) | Alagille syndrome, PFIC (approved); additional rare cholestatic conditions (EXPAND Phase 3) | small molecule | Approved |
| CTEXLI (chenodiol) | Cerebrotendinous xanthomatosis | small molecule | Approved |
| Cholbam (cholic acid) | Bile acid synthesis disorders | small molecule | Approved |
| Brelovitug (BJT-778) | Chronic hepatitis D | antibody | Phase 3 |
| Zilurgisertib | Fibrodysplasia ossificans progressiva | small molecule | Phase 3 |
| Volixibat | Primary sclerosing cholangitis; primary biliary cholangitis | small molecule | Phase 2 |
| MRM-3379 | Fragile X syndrome | small molecule | Phase 2 |
Recent results and decisions
- Oct 12, 2026met Phase 3 EXPAND met primary endpoint: ItchRO(Obs) improved 1.81 vs 0.83 points on placebo (difference 0.98, p=0.0004), plus all five secondary endpoints.
The study met its primary endpoint and achieved statistical significance across all five prespecified secondary endpoints, including the three endpoints in patients with BA and a native liver.
Filing - Sep 28, 2026met Phase 3 AZURE-1 met primary endpoint at Week 24: 56% (300 mg QW) and 45% (900 mg Q4W) vs 0% delayed treatment achieved virologic response plus ALT normalization, p<0.0001.
At Week 24, treatment with brelovitug met the primary endpoint, with 56% of patients in the 300 mg QW arm and 45% in the 900 mg Q4W arm achieving the combined endpoint of virologic response (≥2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA [<LLOQ, TND]) and ALT normalization, comp
Filing - Sep 28, 2026positive 48-week Phase 2b AZURE-1 data showed deepening viral suppression, with combined endpoint at 55% in both arms (vs 45% and 35% at Week 24).
Between Week 24 and Week 48, viral suppression deepened and rates of ALT normalization increased.
Filing
2 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 GI/hepatology companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.