News Details

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that data from its rare liver disease portfolio will be presented at the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting® 2026, taking place November 5-9, 2026, in Denver, Colorado.

Key Highlights

Mirum presentations at The Liver Meeting 2026 will include:

• Brelovitug (HDV): Late-breaking Phase 3 AZURE-1 results highlighting the efficacy and safety of brelovitug monotherapy across a broad chronic hepatitis delta virus (HDV) patient population, alongside longer-term data demonstrating deepening virologic and biochemical responses with continued treatment, including in patients with cirrhosis.
• Volixibat (PSC): Additional Phase 2b VISTAS study analyses reinforcing efficacy and safety profiles of volixibat across pruritus measures and baseline severity, as well as the impact of treatment on fatigue and sleep. A separate subgroup analysis evaluates pruritus improvement in patients with inflammatory bowel disease (IBD), without worsening IBD activity.
• LIVMARLI® (maralixibat) and Rare Cholestatic Liver Diseases: New clinical and real-world evidence examining maralixibat treatment outcomes in Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Separate research examines the underrecognized burden of pruritus in biliary atresia.

“The data we're presenting at The Liver Meeting reflect the depth of our commitment to rare liver diseases and our focus on generating evidence that addresses important needs for patients,” said Chris Peetz, Chief Executive Officer at Mirum. “From longer-term treatment responses with brelovitug to new insights into managing cholestatic pruritus, these presentations reinforce the progress we're making while highlighting the significant unmet needs that remain.”

Congress Presentations

Mirum’s presentations at The Liver Meeting 2026 reflect continued advancement across approved and investigational programs.

Brelovitug

Volixibat

LIVMARLI® (maralixibat) and Rare Cholestatic Liver Diseases

Full abstracts from The Liver Meeting 2026 are available on the AASLD Congress website. Following the congress, detailed analyses will be posted on the Publications & Presentations section of Mirum’s website.

About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets

LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com.

LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for the treatment of ALGS and PFIC. In October 2026, Mirum announced that the Phase 3 EXPAND study of maralixibat in additional rare cholestatic liver diseases, including biliary atresia, met its primary endpoint of improvement in cholestatic pruritus and all five prespecified secondary endpoints with statistical significance.

To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.

IMPORTANT SAFETY INFORMATION

Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.

LIVMARLI can cause side effects, including:

Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with ALGS and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.

US Prescribing Information
EU SmPC
Canadian Product Monograph

About Volixibat

Volixibat is an investigational oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study).

In 2026, Mirum shared that the Phase 2b VISTAS study of volixibat in PSC met its primary endpoint, with statistically significant and clinically meaningful reductions in pruritus observed in patients treated with volixibat. Volixibat’s safety profile in the study was generally consistent with the known effects of IBAT inhibition. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of cholestatic pruritus due to PSC.

In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study of volixibat in PBC. No new safety signals were observed in the study. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of cholestatic pruritus due to PBC.

About Brelovitug

Brelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy Designation for the treatment of chronic HDV and Orphan Drug Designation for the treatment of active hepatitis delta virus infection. Brelovitug also has PRIME and Orphan designations from the European Medicines Agency.

Brelovitug is currently being evaluated in the global Phase 3 AZURE clinical program. In September 2026, Mirum announced that the Phase 3 AZURE-1 study of brelovitug demonstrated strong antiviral activity in HDV and achieved the primary combined endpoint of virologic response and alanine aminotransferase (ALT) normalization at Week 24 in both dose arms. Longer-term, 48-week data from the Phase 2b portion of AZURE-1 demonstrated deepening viral suppression and increased rates of ALT normalization with continued treatment. Favorable safety and tolerability profiles were observed across these studies.

Mirum owns worldwide rights to brelovitug.

About Mirum Pharmaceuticals

Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), ATEBRIOZ™ (zilurgisertib) for fibrodysplasia ossificans progressiva (FOP), CHOLBAM® (cholic acid) for bile-acid synthesis disorders and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX).

Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).

Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the Company’s planned participation at a scientific congress and the potential presentations available at such meeting, Mirum’s continued or advancing leadership in rare liver disease, the potential benefit of Mirum products and product candidates in real world settings versus scientific presentations of data at the AASLD The Liver Meeting 2026 and the continued potential for Mirum to generate evidence that can make a difference for patients. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “expected,” “will,” “can,” “could,” “would,” “guidance,” “potential,” “continue” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties with the development of investigational medicines generally, including the failure of future studies to generate the same or similar data as prior studies, risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Quarterly Report for the quarter ended June 30, 2026, filed with the Securities and Exchange Commission on August 5, 2026, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. The Liver Meeting and AASLD are registered trademarks of American Association for the Study of Liver Diseases.