Mirum Pharmaceuticals' Volixibat Meets Primary Endpoint in Phase 2b PSC Study, Plans H2 2026 NDA Submission

summarizeSummary

Mirum Pharmaceuticals announced positive Phase 2b results for volixibat in primary sclerosing cholangitis (PSC), demonstrating significant pruritus improvement and setting a path for an H2 2026 NDA submission.

check_boxKey Events

• Volixibat Achieves Primary Endpoint in PSC Study

  The VISTAS Phase 2b study for volixibat in primary sclerosing cholangitis (PSC) met its primary endpoint, showing a robust 2.72 point improvement in pruritus and a placebo-adjusted difference of 1.64 points (p<0.0001).

• Clear Regulatory Path Ahead

  A pre-New Drug Application (NDA) meeting for volixibat in PSC is scheduled with the FDA in summer 2026, with a planned NDA submission in the second half of 2026.

• Safety Profile Consistent with IBAT Inhibition

  Volixibat's safety profile was generally consistent with known IBAT inhibition effects, primarily characterized by gastrointestinal adverse events and elevations in liver laboratory parameters.

• PBC Data Expected Q1 2027

  Topline data from the VANTAGE Phase 2b study of volixibat in primary biliary cholangitis (PBC) is now expected in the first quarter of 2027.

auto_awesomeAnalysis

Mirum Pharmaceuticals reported highly positive Phase 2b results for volixibat in primary sclerosing cholangitis (PSC), a significant clinical milestone. The drug demonstrated a statistically significant improvement in pruritus, a key symptom, with a robust placebo-adjusted difference. This success, coupled with a scheduled pre-NDA meeting and a planned NDA submission in the second half of 2026, indicates a clear and accelerated path towards potential market approval. While topline data for the PBC study was pushed to Q1 2027, the strong PSC results for volixibat are a major positive catalyst, building on the company's recent positive clinical news for brelovitug.