Mirum Pharmaceuticals announced that its VISTAS Phase 2b study of volixibat met the primary endpoint in patients with primary sclerosing cholangitis (PSC). The study reported a 2.72‑point reduction from baseline in cholestatic pruritus and a 1.64‑point placebo‑adjusted improvement, with a p‑value of less than 0.0001. PSC is a rare, progressive liver disease with no approved medical therapies, making this result a significant advance for patients and the company’s pipeline.
"These results mark an important milestone for the PSC community, where there are currently no approved therapies," said Joanne Quan, M.D., Chief Medical Officer at Mirum. "In a disease that has been historically difficult to study and treat, VISTAS delivered a clear and compelling efficacy signal. The observed statistically significant and clinically meaningful reductions in pruritus demonstrate the potential for volixibat to address one of PSC's most burdensome and defining symptoms." "We believe this is an outstanding treatment effect," Quan added during a May 4 investor call. "The magnitude of improvement is consistent with what we have observed in other cholestatic indications and reflects a meaningful benefit for patients." CEO Chris Peetz called the readout "a defining moment" for the company and for PSC patients, noting that the results represent "the first successful demonstration of a therapy addressing a core symptom of PSC in a controlled study."
The VISTAS success positions volixibat as a strong candidate for FDA breakthrough designation and supports Mirum’s strategy to expand its rare‑disease portfolio beyond its flagship product Livmarli. The company will present the full data at the EASL International Liver Congress on May 30 and held an investor call on May 4 to discuss the results and next steps. The milestone could accelerate regulatory discussions and pave the way for a future NDA submission, potentially opening a new therapeutic area for Mirum.
Mirum’s broader pipeline includes the approved product Livmarli and other candidates such as Cholbam and Ctexli. In January 2026 the company acquired Bluejay Therapeutics for $268.6 million, adding brelovitug to its portfolio. For 2026, Mirum has guided global net product sales to approximately $630 million to $650 million, reflecting confidence in its expanding product mix.
The VISTAS readout represents the first successful demonstration of a therapy addressing a core symptom of PSC in a controlled study, marking a pivotal moment for Mirum and for patients with this rare disease.
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