Mirum Pharmaceuticals Spotlights Advancements in Rare Liver Disease With New Data From LIVMARLI® and Volixibat Programs at AASLD’s The Liver Meeting® 2025
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biotechnology company focused on developing and commercializing therapies for rare and severe liver disorders, announced a broad set of new presentations from its clinical programs during the upcoming American Association for the Study of Liver Diseases (AASLD) The Liver Meeting®. The annual scientific conference, taking place November 7–11, 2025, in Washington, D.C., will feature multiple presentations dedicated to Mirum’s portfolio, including the latest findings on LIVMARLI® (maralixibat) and volixibat.
These presentations underscore Mirum’s continued commitment to addressing significant gaps in care for individuals living with pediatric and adult cholestatic liver diseases—complex conditions often characterized by persistent pruritus, progressive liver damage, and limited therapeutic options. The new data draw from a mix of clinical trial analyses, real-world experience, and genetic testing initiatives, expanding the clinical evidence base supporting two of Mirum’s leading therapeutic candidates.
Building on a Strong Clinical Foundation
Mirum’s presence at The Liver Meeting® is anchored by advancing research in primary biliary cholangitis (PBC), Alagille syndrome (ALGS), and progressive familial intrahepatic cholestasis (PFIC), as well as conditions such as primary sclerosing cholangitis (PSC) and other cholestatic disorders. The central focus centers on volixibat, an investigational ileal bile acid transporter (IBAT) inhibitor, and LIVMARLI®, a first-in-class IBAT inhibitor already approved in multiple regions for pediatric cholestatic liver diseases.
The selection of data presentations highlights the company’s strategy: combine rigorously conducted clinical trials with real-world evidence to form a broad and dynamic picture of the clinical utility of its therapies across diverse patient groups.
Key Oral Presentation: VANTAGE Trial Insights in PBC
A major highlight during the conference will be an oral presentation providing deeper analysis from the Phase 2b VANTAGE trial evaluating volixibat in adults with primary biliary cholangitis, a chronic autoimmune liver disease characterized by biliary inflammation, cholestasis, and progressive fibrosis. Many PBC patients suffer from debilitating fatigue and sleep disturbance, two symptoms often poorly controlled with standard care.
Oral Presentation Details
Abstract #0215: Volixibat Leads to Improvements in Fatigue and Sleep for Adults with Primary Biliary Cholangitis: Data from VANTAGE Trial
Date/Time: Monday, November 10, 2025, 3:00–3:15 pm
Session: Novel Therapies and Monitoring in Cholestatic Liver Disease
Location: Room 145, Level 1, Convention Center
Presenter: Dr. Kris Kowdley, Liver Institute Northwest, Seattle, Washington, USA
This presentation is expected to provide key insights into volixibat’s therapeutic potential beyond improving biochemical markers. By specifically assessing fatigue and sleep quality—both major contributors to PBC-related morbidity—the analysis offers a nuanced perspective on how volixibat may help improve quality of life for individuals with PBC.
Poster Presentations: Real-World Experience, Genetic Insights & Immunologic Markers
Alongside new VANTAGE trial analyses, Mirum researchers and collaborators will deliver several poster presentations on maralixibat and volixibat. These posters expand the understanding of disease burden, real-world therapeutic impact, and biological markers relevant to cholestatic illnesses.
Real-World Insights in PSC
Abstract #4411: Maralixibat, an Ileal Bile Acid Transporter Inhibitor, Reduces Cholestatic Pruritus in PSC: Real-World Experience
Date/Time: Monday, November 10, 2025, 1:00–2:00 pm
Location: Posters & Exhibit Hall, Level 2, Convention Center
Presenter: Dr. Adrielly Martins, University of Miami, Florida, USA
This study offers real-world evidence on maralixibat’s ability to alleviate pruritus among patients with PSC, a disease with sparse treatment options and an often devastating progression. Real-world data provide important context for treatment decisions, particularly for symptoms that significantly impair daily functioning.
Genetic Testing in Cholestasis
Abstract #4412: Clinical Utility of Comprehensive Genetic Testing in Adult Cholestasis: Insights from 856 Panel Tests
Date/Time: Monday, November 10, 2025, 1:00–2:00 pm
Location: Posters & Exhibit Hall, Level 2, Convention Center
Presenter: Dr. Tiziano Pramparo, Mirum Pharmaceuticals, Inc.
This presentation highlights the growing role of genetic testing in evaluating adults with unexplained cholestasis. By analyzing more than 850 panel tests, the study aims to identify diagnostic trends and key genetic contributors to disease, guiding more precise therapies and treatment pathways.
Mechanistic Insights From the VANTAGE Study
Abstract #4415: 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
Date/Time: Monday, November 10, 2025, 1:00–2:00 pm
Location: Posters & Exhibit Hall, Level 2, Convention Center
Presenter: Dr. Alan Bonder, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA
This research looks at volixibat’s potential mechanistic relationship with IL-31, a cytokine believed to play an important role in cholestatic pruritus. The preliminary results could deepen scientific understanding of this biological pathway, providing insight into how volixibat may impact pruritus symptomology in PBC.
Assessing Pruritus Burden in PSC
Abstract #4511: Physician Reported Assessment of Pruritus Amongst Patients with PSC: Results from a Real-World Survey in France, Germany, Italy and the United States
Date/Time: Monday, November 10, 2025, 1:00–2:00 pm
Location: Posters & Exhibit Hall, Level 2, Convention Center
Presenter: Dr. Marlyn Mayo, UT Southwestern Medical Center, Dallas, Texas, USA
This multi-regional survey details physician perspectives on the burden of pruritus among PSC patients. The findings offer a window into symptomatic management patterns and the unmet clinical needs across major global health markets.
Caregiver Burden in ALGS & PFIC
Abstract #4532: Impact of Maralixibat on Caregiver Burden for Patients with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis: Baseline and 6-month Follow-up
Date/Time: Monday, November 10, 2025, 1:00–2:00 pm
Location: Posters & Exhibit Hall, Level 2, Convention Center
Presenter: Dr. Natasha Dilwali, Columbia University Irving Medical Center, New York, New York, USA
This study assesses caregiver burden over time and evaluates whether maralixibat may contribute to lessening this burden. Conditions such as ALGS and PFIC often place significant emotional, logistical, and financial strain on caregivers, making caregiver-focused outcomes an important measure of therapeutic impact.
Fireside Discussion: Focus on PFIC Diagnosis and Treatment
Mirum will also convene an educational fireside session dedicated to improving awareness and clinical understanding of PFIC. The session will emphasize diagnostic pathways, with an emphasis on the role of genetic testing, and explore practical considerations for adult patients living with a disease historically associated with childhood onset.
Session Details
Topic: PFIC Diagnosis & Management
Date/Time: Sunday, November 9, 2025, 1:30–2:00 pm
Location: Exhibit Hall TLM Theater
Speaker: Dr. Robert Gish, Loma Linda University
The event will incorporate a discussion of LIVMARLI’s clinical data in PFIC, providing physicians with a clinical framework for integrating recent advances in patient care.
Expanding Treatment Potential for Rare Liver Diseases
Mirum’s activities at The Liver Meeting demonstrate its steady progress in bringing new therapies to patients who face limited options. In particular, the company continues to explore the therapeutic potential of IBAT inhibition—a mechanism aimed at reducing the accumulation of bile acids that contribute to liver injury and pruritus.
LIVMARLI is the first approved IBAT inhibitor and represents a significant step forward for patients with ALGS and PFIC. Volixibat—still in clinical development—may expand treatment utility to adult conditions such as PBC, a disease in which significant symptom burden persists despite available standard treatments.
About LIVMARLI® (maralixibat)
LIVMARLI is an orally administered IBAT inhibitor approved by the U.S. Food and Drug Administration (FDA) and European regulators for multiple pediatric cholestatic liver diseases. It is:
- U.S.-approved for cholestatic pruritus associated with ALGS (three months of age and older)
- European-approved for ALGS in patients two months of age and older
- U.S.-approved for cholestatic pruritus in PFIC (12 months of age and older)
- European-approved for PFIC (three months of age and older)
The therapy has orphan designation for both ALGS and PFIC. Ongoing clinical studies, including the Phase 3 EXPAND study, are evaluating LIVMARLI in additional cholestatic settings.
Summary Safety Information
LIVMARLI is contraindicated in PFIC type 2 patients with severe bile salt export pump defects. Treatment may lead to liver injury and gastrointestinal side effects, as well as fat-soluble vitamin deficiency. Regular clinical and laboratory monitoring is recommended to ensure patient safety. Patients and caregivers are encouraged to consult with prescribing physicians if concerning symptoms arise.
More detailed prescribing information is available through U.S., European, and Canadian regulatory documents.
Continuing Scientific Momentum
The comprehensive slate of oral and poster presentations planned for The Liver Meeting® reflects Mirum’s continued dedication to addressing the multifaceted challenges of cholestatic liver diseases. Through advancing clinical programs, expanding real-world evidence, and building genetic understanding, the company aims to expand therapeutic possibilities and improve quality of life for patients and families affected by rare liver disorders.
Conference abstracts are available on the AASLD website, and following the meeting, additional analyses will be posted to the Publications and Presentations section of Mirum’s corporate website.
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