New Hope for PSC: Volixibat Trial Meets Primary Goal

Mirum Pharmaceuticals has announced positive results from its VISTAS Phase IIb clinical study of volixibat, an investigational treatment for primary sclerosing cholangitis (PSC). The global trial successfully met its primary goal in reducing pruritus, a significant symptom for PSC patients.

Volixibat demonstrated a statistically significant and clinically meaningful reduction in itch severity as measured by the ItchRO scale. Improvements were noted as early as two weeks into the treatment. The therapy's safety profile aligns with known effects of ileal bile acid transporter inhibition, with gastrointestinal issues and elevated liver parameters being the main adverse events.

The company is preparing for a pre-new drug application meeting with the US Food and Drug Administration in mid-2026, with plans to submit the NDA later in the same year. This development offers a potential new therapeutic option for PSC, a rare and serious liver condition with no currently approved treatments.

Full trial results are scheduled for presentation at the European Association for the Study of the Liver International Liver Congress on May 30, 2026. Mirum is also investigating volixibat for primary biliary cholangitis (PBC) and expects top-line data from its VANTAGE Phase IIb study in early 2027.