{"id":957497,"date":"2026-05-04T08:05:43","date_gmt":"2026-05-04T12:05:43","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/mirum-pharmaceuticals-announces-primary-endpoint-met-in-vistas-study-of-volixibat-in-patients-with-primary-sclerosing-cholangitis\/"},"modified":"2026-05-04T08:05:43","modified_gmt":"2026-05-04T12:05:43","slug":"mirum-pharmaceuticals-announces-primary-endpoint-met-in-vistas-study-of-volixibat-in-patients-with-primary-sclerosing-cholangitis","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/mirum-pharmaceuticals-announces-primary-endpoint-met-in-vistas-study-of-volixibat-in-patients-with-primary-sclerosing-cholangitis\/","title":{"rendered":"Mirum Pharmaceuticals Announces Primary Endpoint Met in VISTAS Study of Volixibat in Patients with Primary Sclerosing Cholangitis"},"content":{"rendered":"

<\/p>\n

Mirum Pharmaceuticals Announces Primary Endpoint Met in VISTAS Study of Volixibat in Patients with Primary Sclerosing Cholangitis<\/b><\/p>\n

\n–\u00a0Statistically significant and clinically meaningful 2.72 point reduction from baseline and 1.64 point placebo-adjusted (p=<0.0001) reduction in primary endpoint of cholestatic pruritus<\/i><\/p>\n

– Pre-NDA meeting with the U.S. Food and Drug Administration (FDA) scheduled for summer 2026<\/i><\/p>\n

– Full results to be presented in late-breaking oral presentation at the EASL International Liver Congress 2026 <\/i><\/p>\n

\n–\u00a0Mirum to host conference call to discuss Mirum\u2019s recent clinical readouts, today, May 4 at 8:30 am ET<\/i><\/p>\n

FOSTER CITY, Calif.–(BUSINESS WIRE<\/a>)–
\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced the primary endpoint was met in the VISTAS Phase 2b study evaluating volixibat, an investigational oral ileal bile acid transporter (IBAT) inhibitor, in patients with primary sclerosing cholangitis (PSC).<\/p>\n

\nThe VISTAS Phase 2b study included 158 patients with PSC who were assigned to either a primary analysis cohort (moderate to severe itch; n=111) or a secondary analysis cohort (mild itch; n=47) based on itch severity at baseline, as measured by the Adult Itch Reported Outcome (ItchRO) scale. Patients in both cohorts were treated with volixibat 20 mg twice daily (BID) or placebo. Per the study protocol, the endpoint was evaluated in the primary analysis cohort.<\/p>\n

\nThe results of the VISTAS Phase 2b study support the potential for volixibat to become the first treatment for cholestatic pruritus in patients with PSC. In the primary analysis cohort, treatment with volixibat demonstrated a robust 2.72 point improvement in pruritus and a placebo-adjusted difference of 1.64 points in the primary endpoint (p<0.0001), as measured by the Adult ItchRO scale, reflecting change from baseline to the average of the last 12 weeks of treatment. Statistically significant improvements in pruritus were observed within two weeks of treatment and were also observed in the secondary cohort of patients with mild itch at baseline. Key efficacy data from the VISTAS Phase 2b study are presented below in the Change from Baseline (Primary Analysis Cohort) <\/b>table.<\/p>\n

\nVolixibat\u2019s 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. Key safety data from the VISTAS Phase 2b study are presented below in the Safety Summary (Primary and Secondary Cohorts)<\/b> table.<\/p>\n

\nMirum has a pre-New Drug Application (NDA) meeting for volixibat in PSC scheduled with the U.S. FDA in summer 2026, with a planned NDA submission in the second half of 2026.<\/p>\n

\n\u201cThese results mark an important milestone for the PSC community, where there are currently no approved therapies,\u201d said Joanne Quan, M.D., Chief Medical Officer at Mirum. \u201cIn 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\u2019s most burdensome and defining symptoms.\u201d<\/p>\n

\n\u201cCholestatic pruritus is one of the most common symptoms in PSC, which remains a challenging disease to manage,\u201d said Kris Kowdley, M.D., Director, Liver Institute Northwest, Professor, Elson S. Floyd College of Medicine at Washington State University and an investigator for the VISTAS study. \u201cIn clinical practice, we have very limited options to effectively address this symptom. Results like these are encouraging, as they suggest the potential for a targeted approach that could represent a meaningful advance for PSC patients.\u201d<\/p>\n

\n\u201cAfter decades of commitment from patients, families and researchers participating in PSC studies, these results represent real hope for what could become the first approved treatment,\u201d said Ricky Safer, Founder and CEO of PSC Partners Seeking a Cure. \u201cFor many patients, the itch associated with PSC is constant and deeply disruptive \u2013 affecting sleep, daily activities and overall quality of life. Seeing meaningful progress in reducing this burden is incredibly encouraging for a community that has long had limited options.\u201d<\/p>\n

\nThe full results from the VISTAS Phase 2b study will be presented in a late-breaking oral presentation at the European Association for the Study of the Liver (EASL) International Liver Congress on May 30 at 2:15 p.m. CEST.<\/p>\n

\nMirum also now expects topline data from its VANTAGE Phase 2b study of volixibat in primary biliary cholangitis (PBC) in the first quarter of 2027.<\/p>\n

Change from Baseline (Primary Analysis Cohort)<\/b><\/p>\n\n\n\n\n\n\n\n
\n

\n\u00a0<\/p>\n<\/td>\n

\n

Volixibat<\/b><\/p>\n

20 mg BID<\/b><\/p>\n

(n=54)<\/b><\/p>\n<\/td>\n

\n

Placebo<\/b><\/p>\n

(n=57)<\/b><\/p>\n<\/td>\n

\n

Difference<\/b><\/p>\n

(VLX –<\/b><\/p>\n

PBO)<\/b><\/p>\n<\/td>\n

\n

p-value<\/b><\/p>\n<\/td>\n<\/tr>\n

\n

\nLS Mean change in Adult ItchRO (SE)*<\/p>\n<\/td>\n

\n

\n-2.72 (0.240)<\/p>\n<\/td>\n

\n

\n-1.08 (0.241)<\/p>\n<\/td>\n

\n

\n-1.64<\/p>\n<\/td>\n

\n

\n<0.0001<\/p>\n<\/td>\n<\/tr>\n

\n

\n\u22652 point reduction in Adult ItchRO*<\/p>\n<\/td>\n

\n

\n55.6%<\/p>\n<\/td>\n

\n

\n26.3%<\/p>\n<\/td>\n

\n

\n29.3%<\/p>\n<\/td>\n

\n

\n0.0019<\/p>\n<\/td>\n<\/tr>\n

\n

\nLS Mean changes in sBA (SE)<\/p>\n<\/td>\n

\n

\n-33.7 (12.14)<\/p>\n<\/td>\n

\n

\n2.1 (11.87)<\/p>\n<\/td>\n

\n

\n-35.8<\/p>\n<\/td>\n

\n

\n0.0324<\/p>\n<\/td>\n<\/tr>\n

<\/tr>\n
\n

\n*Adult ItchRO is an 11-point scale (0 = no itch; 10 = worst itch imaginable). LSMean = model-adjusted mean from a mixed model for repeated measures (MMRM). Values represent change from baseline to the average of the last 12 weeks of treatment (weekly averaged worst daily itch score).<\/p>\n<\/td>\n<\/tr>\n<\/table>\n

Safety Summary (Primary and Secondary Cohorts)<\/b><\/p>\n\n\n\n\n\n\n\n\n
\n

\n\u00a0<\/p>\n<\/td>\n

\n

Volixibat<\/b><\/p>\n

20 mg BID<\/b><\/p>\n

(n=77)<\/b><\/p>\n<\/td>\n

\n

Placebo<\/b><\/p>\n

(n=81)<\/b><\/p>\n<\/td>\n<\/tr>\n

\n

\nParticipants with any treatment emergent adverse event (TEAE), n (%)<\/p>\n<\/td>\n

\n

\n72 (93.5)<\/p>\n<\/td>\n

\n

\n68 (84.0)<\/p>\n<\/td>\n<\/tr>\n

\n

\nGrade 3 or higher TEAEs<\/p>\n<\/td>\n

\n

\n10 (13.0)<\/p>\n<\/td>\n

\n

\n9 (11.1)<\/p>\n<\/td>\n<\/tr>\n

\n

\nSerious TEAEs<\/p>\n<\/td>\n

\n

\n8 (10.4)<\/p>\n<\/td>\n

\n

\n5 (6.2)<\/p>\n<\/td>\n<\/tr>\n

\n

\nTEAE that led to death<\/p>\n<\/td>\n

\n

\n0<\/p>\n<\/td>\n

\n

\n1 (1.2)<\/p>\n<\/td>\n<\/tr>\n

\n

\nTEAE that led to premature discontinuation from study<\/p>\n<\/td>\n

\n

\n7 (9.1)<\/p>\n<\/td>\n

\n

\n2 (2.5)<\/p>\n<\/td>\n<\/tr>\n

\n

\nStudy discontinuation due to diarrhea<\/p>\n<\/td>\n

\n

\n3 (3.9)<\/p>\n<\/td>\n

\n

\n1 (1.2)<\/p>\n<\/td>\n<\/tr>\n<\/table>\n\n\n\n\n
\n

\nAdditional safety observations:<\/p>\n<\/td>\n<\/tr>\n

\u00a0<\/td>\n<\/tr>\n
\n