Open report — full analysis, no account required.
Sign up to generate reports and read filings that aren't on the open list.
Get notified when MIRM files again. Create a free account and we'll email you the moment its next filing is analyzed.
Get filing alertsMirum's volixibat meets Phase 2b endpoint in PSC, plans H2 2026 NDA submission
Filed May 4, 2026 · Period ending May 4, 2026 · ~1 min read
Key Changes
-
high
Volixibat achieved statistically significant 1.64-point improvement over placebo (p<0.0001) in cholestatic pruritus among 111 PSC patients with moderate-to-severe itch, supporting potential first-in-class treatment claim.
Item 8.01 — Other Events verify on EDGAR → -
high
Pre-NDA meeting scheduled with FDA for summer 2026, with New Drug Application submission planned for second half of 2026, representing near-term regulatory milestone.
Item 8.01 — Other Events verify on EDGAR → -
high
Safety profile showed 40.3% diarrhea rate versus 8.6% placebo, with 9.1% premature discontinuation versus 2.5% placebo; gastrointestinal events and liver enzyme elevations consistent with IBAT inhibition mechanism.
Item 8.01 — Other Events verify on EDGAR → -
medium
Topline data from VANTAGE Phase 2b study evaluating volixibat in primary biliary cholangitis (PBC) now expected in Q1 2027, representing second potential indication.
Item 8.01 — Other Events verify on EDGAR →
Summary
Mirum announced positive Phase 2b results for volixibat in primary sclerosing cholangitis (PSC), a rare liver disease with no approved treatments for cholestatic pruritus. The 158-patient VISTAS trial met its primary endpoint, demonstrating a statistically significant 1.64-point improvement over placebo in itch severity among patients with moderate-to-severe symptoms.
The company positions volixibat as a potential first-in-class therapy for this indication. The safety profile warrants attention: 40% of volixibat patients experienced diarrhea versus 9% on placebo, and the treatment group had a 9% discontinuation rate versus 3% for placebo.
Liver enzyme elevations were also more common, though Mirum characterizes these as consistent with the drug's mechanism of action as an IBAT inhibitor. The regulatory path is now defined, with a pre-NDA meeting scheduled for summer 2026 and NDA submission planned for the second half of 2026. Investors should monitor the FDA's feedback on the Phase 2b data package and whether additional studies will be required for approval. A parallel Phase 2b trial in primary biliary cholangitis is expected to read out in Q1 2027, potentially expanding volixibat's addressable market.
Section-by-Section Diff
Event · Item 8.01 — Other Events
Mirum announced positive Phase 2b trial results for volixibat in PSC, meeting primary endpoint with planned NDA submission in H2 2026.
Added in current filing · verify on EDGAR → · paraphrased
Volixibat's safety profile was generally consistent with the known effects of IBAT inhibition, characterized primarily by gastrointestinal adverse events and elevations in liver laboratory parameters, including alanine aminotransferase ("ALT") and bilirubin. ... Participants with any treatment emergent adverse event ("TEAE"), n (%) Volixibat 20 mg BID (n=77) 72 (93.5) Placebo (n=81) 68 (84.0) ... TEAE that led to premature discontinuation from study Volixibat 7 (9.1) Placebo 2 (2.5) ... Diarrhea Volixibat 31 (40.3) Placebo 7 (8.6)
The safety profile showed 93.5% of volixibat patients experienced treatment-emergent adverse events versus 84.0% on placebo. Gastrointestinal events were most common, with diarrhea occurring in 40.3% of volixibat patients versus 8.6% on placebo. Premature discontinuation occurred in 9.1% of volixibat patients versus 2.5% on placebo, with liver enzyme elevations also more frequent in the treatment group.
Thanks — your feedback helps us improve report quality.
Figures/quotes linked to EDGAR · Narrative written by AI · Jun 23, 2026 · How we verify