Volixibat for cholestatic pruritus in PBC

On February 25^th, Mirum hosted its Q4/FY 2025 earnings call. Guidance largely mirrored the company's JPM 2026 presentation, with continued emphasis on PSC as volixibat’s (iBATi) primary indication. [Webcast, Press Release, February Corporate Presentation, Form 10-K] 

Key Takeaways: 

• Topline readout for the Phase 2 VANTAGE study of volixibat in PBC remains expected in H1 2027  

• Mirum remains on track for an NDA filing for volixibat in PSC in H2 2026 

Volixibat in PBC: 

• Timelines for Phase 2 VANTAGE (N=260; PCD: Apr 2027) trial remained unchanged 

o   Enrollment is expected to complete in H2 2026 

o   Topline data remains expected in H1 2027 

Volixibat in PSC: 

• Timelines for Phase 2 VISTAS (N=182; PCD: Jun 2026) trial remained unchanged 

o   Mirum reiterated the trial is fully enrolled 

o   Topline data remains expected in Q2 2026, with NDA submission anticipated for H2 2026 

• There remain two plausible US approval paths for volixibat in PBC-associated pruritis, depending on whether Mirum can file based on Phase 2b VANTAGE data alone or needs confirmatory Phase 3 data: 

o   Phase 2b filing from VANTAGE trial: Approval around H1 2028 

o   Phase 3 required: Approval likely shifts to H1 2032

• Mirum continues to lead with PSC, highlighting VISTAS trial topline data in Q2 2026 and potential NDA filing in H2 2026. The company appears to be positioning volixibat’s primary indication as PSC with PBC as an add-on, highlighting the asset would be the first approved medicine specifically for PSC-related pruritis