volixibat (SAR 548304) / Sanofi, Takeda, Mirum Pharmaceuticals, Ligand - LARVOL DELTA

Efficacy and Safety of Volixibat, an IBAT Inhibitor, in Patients With Primary Sclerosing Cholangitis and Moderate-to-Severe Pruritus: Results of the VISTAS Trial (ACG 2026) - "Abstract text will be released on Sunday, October 11, 2026, at 12:00 pm CT (1:00 pm ET) when the ACG 2026 embargo lifts."

Clinical • Dermatology • Hepatology • Primary Sclerosing Cholangitis • Pruritus

VISTAS: A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) (clinicaltrials.gov) - P2 | N=182 | Active, not recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | Trial completion date: Apr 2027 ➔ Mar 2028 | Trial primary completion date: Jun 2026 ➔ Feb 2026

Trial completion date • Trial primary completion date • Dermatology • Pruritus

VANTAGE: A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis (clinicaltrials.gov) - P2 | N=333 | Active, not recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Jul 2027 ➔ Dec 2028 | Trial primary completion date: Apr 2027 ➔ Jan 2027

Enrollment closed • Trial completion date • Trial primary completion date • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

FDA Grants Volixibat Breakthrough Therapy, Orphan Designations for PSC (HCPLive) - "The designations rest on topline results from the phase 2b VISTAS trial...'We intend to hold further discussions with the FDA before a potential NDA submission in the first half of 2027 based on VISTAS.'....That timeline is later than the second-half 2026 submission Mirum had previously outlined."

Breakthrough therapy • FDA filing • Immunology • Inflammation

Regulatory and Pipeline: Advancing Toward Multiple Milestones (Businesswire) - "Completed enrollment in the volixibat VANTAGE Phase 2b study in cholestatic pruritus due to primary biliary cholangitis (PBC); topline results expected in Q1 2027."

Enrollment closed • P2b data • Primary Biliary Cholangitis • Pruritus

Ligand Completes Acquisition of XOMA Royalty, Creating a Portfolio of More than 200 Biopharmaceutical Royalty Assets (The Manila Times) - Acquisition adds seven commercial products including VABYSMO (faricimab-svoa), OJEMDA (tovorafenib), and MIPLYFFA (arimoclomol), andmore than 100 development and commercial-stage assets, creating long-term opportunities to drive growth...Additionally, it includes 14 late-stage development programs, featuring Takeda’s mezagitamab and certain assets from Takeda’s externalized asset portfolio, such as osavampator, volixibat, and OHB-607, along with more than 100 assets in various stages of development....Ligand will provide an updated 5-year outlook during the Company’s Investor Day on December 8, 2026."

M&A • Bronchopulmonary Dysplasia • Diabetic Macular Edema • Diabetic Retinopathy • IgA Nephropathy • Immune Thrombocytopenic Purpura • Immunology • Low Grade Glioma • Lysosomal Storage Diseases • Metabolic Dysfunction-Associated Steatohepatitis • Primary Biliary Cholangitis • Wet Age-related Macular Degeneration

Efficacy and Safety of Volixibat, an IBAT Inhibitor, in Patients With Primary Sclerosing Cholangitis and Moderate-to-Severe Pruritus: Results of the VISTAS Trial (EASL 2026) - "VISTAS is the largest interventional study investigating the efficacy and safety of an IBAT inhibitor in PSC. The results will represent the first controlled evidence of a potential therapy addressing the symptom burden in PSC patients."

Clinical • Late-breaking abstract • CNS Disorders • Crohn's disease • Dermatology • Gastrointestinal Disorder • Hepatology • Immunology • Inflammation • Inflammatory Bowel Disease • Pruritus • Sleep Disorder • Ulcerative Colitis

Mirum Pharmaceuticals to Present Data Showcasing Leadership in Rare Liver Diseases at the EASL International Liver Congress 2026 (Yahoo Finance) - "Late-breaking VISTAS results demonstrate rapid, sustained, and clinically meaningful reductions in pruritus with volixibat in primary sclerosing cholangitis (PSC)."

P2 data • Real-world • Immunology • Inflammation

Mirum Pharmaceuticals Announces Primary Endpoint Met in VISTAS Study of Volixibat in Patients with Primary Sclerosing Cholangitis (Businesswire) - "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....Mirum 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....The full results from the VISTAS Phase 2b study will be presented in a late-breaking oral presentation at the...EASL International Liver Congress on May 30 at 2:15 p.m. CEST."

FDA event • FDA filing • P2b data • Immunology

Mirum also now expects topline data from its VANTAGE Phase 2b study of volixibat in primary biliary cholangitis (PBC) in the first quarter of 2027 (Businesswire)

P2b data • Immunology • Primary Biliary Cholangitis

VOLIXIBAT FOR THE TREATMENT OF CHOLESTATIC PRURITUS IN PRIMARY BILIARY CHOLANGITIS: AN ADAPTIVE, RANDOMIZED, PLACEBO-CONTROLLED PHASE 2B TRIAL (VANTAGE): 28-WEEK INTERIM RESULTS (AEEH 2026) - No abstract available

Clinical • P2b data • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

Volixibat Leads to Improvements in Fatigue and Sleep for Adults With PBC: Data From VANTAGE. (PubMed, Gastroenterol Hepatol (N Y)) - No abstract available

Journal • Fatigue

Efficacy, Safety and Tolerability of Volixibat, an IBAT Inhibitor, in Patients With Intrahepatic Cholestasis of Pregnancy. (PubMed, Liver Int) - P2 | "Volixibat demonstrated improvements in pruritus and sBA in patients with ICP. Trial Registration: NCT04718961."

Journal • Cholestasis • Dermatology • Hepatology • Pruritus

EXPLORING THE ROLE OF ILEAL BILE ACID TRANSPORTER INHIBITORS, LIKE VOLIXIBAT, IN THE REGULATION OF IL-31 AND SERUM BILE ACIDS IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: PRELIMINARY RESULTS: FROM THE VANTAGE TRIAL (AASLD 2025) - "Reductions in pruritus after VLX treatment were associated with decreases in both IL-31 and sBA. These findings support the connection between sBA and IL-31 as potential mediators of pruritus in PBC."

Clinical • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

VOLIXIBAT LEADS TO IMPROVEMENTS IN FATIGUE AND SLEEP FOR ADULTS WITH PRIMARY BILIARY CHOLANGITIS: DATA FROM VANTAGE (AASLD 2025) - "VANTAGE Part 1 results support the hypothesis that efficacious pruritus treatment with VLX may result in improvements in sleep and fatigue in PBC. Improvements in fatigue and sleep will be further evaluated in the ongoing Part 2 period of the VANTAGE study."

Clinical • Cholestasis • CNS Disorders • Dermatology • Fatigue • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus • Sleep Disorder

The Role of the Lower Airway Microbiome in Predicting Treatment Response to Immunotherapy in Patients With Non-Small Cell Lung Cancer (IASLC-WCLC 2025) - "Phylum-001 was significantlyincreased in non-responders (p = 0.004) while Phylum-002 was enriched inresponders (p = 0.003)...Larger multicentre studies are needed to furtherexplore its potential as a novel biomarker and therapeutic target. ."

Clinical • IO biomarker • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

VISTAS: A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) (clinicaltrials.gov) - P2 | N=182 | Active, not recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2025 ➔ Apr 2027 | Trial primary completion date: Sep 2025 ➔ Jun 2026

Enrollment closed • Trial completion date • Trial primary completion date • Dermatology • Pruritus

Intrahepatic Cholestasis of Pregnancy: Diagnosis, Management, and Future Directions-A Review of the Literature. (PubMed, Diagnostics (Basel)) - "Ursodeoxycholic acid (UDCA) remains the primary pharmacologic treatment of intrahepatic cholestasis of pregnancy; however, its effect on perinatal outcomes is debated. Investigational therapies-including Volixibat, FXR agonists, 4-phenylbutyrate, and NorUDCA-are under exploration...Future research should prioritize identifying predictive biomarkers, refining treatment algorithms, and assessing long-term outcomes for both mothers and offspring. Special attention should also be given to the investigation of novel therapeutic targets."

Journal • Review • Cholestasis • Dermatology • Hepatology • Pruritus

VANTAGE: A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis (clinicaltrials.gov) - P2 | N=260 | Recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | N=180 ➔ 260 | Trial completion date: Dec 2025 ➔ Jul 2027 | Trial primary completion date: Dec 2025 ➔ Apr 2027

Enrollment change • Trial completion date • Trial primary completion date • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

VOLIXIBAT FOR THE TREATMENT OF CHOLESTATIC PRURITUS IN PRIMARY BILIARY CHOLANGITIS: AN ADAPTIVE, RANDOMIZED, PLACEBO-CONTROLLED PHASE 2B TRIAL (VANTAGE): INTERIM RESULTS (DDW 2025) - No abstract available

Clinical • P2b data • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

4425: Cholestatic Liver Disease (DDW 2025) - "Description: This session will cover a range of topics, including the risks associated with GLP-1 receptor agonist therapy, the treatment of cholestatic pruritus with Volixibat, and the global burden of chronic liver disease. Attendees will also learn about the role of the ADAM9 gene in cholangiocarcinoma, the impact of obesity on acute liver failure outcomes, and the potential benefits of lipid-lowering therapy in primary sclerosing cholangitis.Learning Objectives:• Describe the association between GLP-1 receptor agonist therapy and the risk of gallbladder and biliary tract disease in individuals with overweight or obesity.• Discuss the role of the ADAM9 gene in mediating prognosis and the immune microenvironment in cholangiocarcinoma."

Biliary Cancer • Cholangiocarcinoma • Cholestasis • Dermatology • Genetic Disorders • Hepatology • Liver Failure • Obesity • Oncology • Pruritus • Solid Tumor • ADAM9

Volixibat Data from Mirum’s VANTAGE PBC Study Showcased at EASL (Businesswire) - P2 | N=180 | VANTAGE (NCT05050136) | Sponsor: Mirum Pharmaceuticals, Inc. | "Data from the interim analysis of VANTAGE were highlighted with new analyses through week 28. Patients were randomized to receive volixibat 20mg BID, 80mg BID, or placebo BID. The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale, from baseline to Week 17-Week 28. Volixibat treatment led to rapid (as early as week 1) and sustained reductions in ItchRO with the volixibat combined dose group achieving a statistically significant 3.78-point reduction from Baseline (p<0.0001) and a placebo-adjusted response of 2.51 (p=0.0004). Each volixibat dose achieved statistically significant and similar responses (20mg BID: -2.4 [P=0.0039]; 80mg BID: -2.6 [P=0.0010]). The analysis also provided data across secondary endpoints showing reductions in serum bile acids (sBA) and quality of life (QoL) measures."

P2 data • Primary Biliary Cholangitis

Abstract #OS-059 (Oral): Volixibat for the treatment of cholestatic pruritus in primary biliary cholangitis: an adaptive, randomized, placebo-controlled phase 2B trial (VANTAGE): 28-week interim analysis (Businesswire) - P2 | N=180 | VANTAGE (NCT05050136) | Sponsor: Mirum Pharmaceuticals, Inc. | "New 28-week data were reported from the Phase 2 VANTAGE study evaluating volixibat in patients with cholestatic pruritus related to primary biliary cholangitis (PBC). Thirty-one patients with moderate-to-severe pruritus were randomized (20mg VLX=10, 80mg VLX=10, placebo=11). Data showed that volixibat led to early and significant reductions in PBC-associated cholestatic pruritus and were maintained throughout the duration of the study. Further, 70% of volixibat-treated patients had a ≥50% reduction in serum bile acid levels from baseline. Additionally, patients treated with volixibat experienced improvements in fatigue and sleep. No new safety signals were observed through 28 weeks of treatment. The most common treatment-emergent adverse event was diarrhea which was mild to moderate in severity and transient in nature."

P2 data • Primary Biliary Cholangitis • Pruritus

Volixibat for the treatment of cholestatic pruritus in primary biliary cholangitis: an adaptive, randomized, placebo-controlled phase 2b trial (VANTAGE): 28-week interim results (EASL 2025) - "Volixibat led to early and significant reductions in PBC-associated cholestatic pruritus and fatigue, with no new safety signals observed through 28 weeks of treatment. Given similar results between VLX doses, the 20 mg BID dose was selected for Part 2 of VANTAGE, constituting a new promising therapy to address important symptoms in PBC.."

Clinical • P2b data • Dermatology • Fatigue • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

PILOT STUDY OF VOLIXIBAT CO-ADMINISTERED WITH OCA FOR PRIMARY BILIARY CHOLANGITIS (PBC) TREATMENT: THE VLX-602 TRIAL (AASLD 2024) - "IBAT inhibitors have shown efficacy to treat cholestatic pruritus, including in PBC, but concurrent use of an IBAT inhibitor and obeticholic acid (OCA) is not well studied. These safety pilot study data were consistent with the known safety profile of IBAT inhibitors. The addition of VLX to OCA treatment in PBC led to improvement in itch in some patients, with reversal of the effect upon VLX cessation. The results of this study justify additional investigation of the safety and efficacy of VLX for the treatment of PBC, with or without a background regimen of OCA."

Clinical • Cholestasis • Dermatology • Fatigue • Hepatology • Immunology • Infectious Disease • Primary Biliary Cholangitis • Pruritus • Respiratory Diseases