Mirum Pharmaceuticals to Present Data at Upcoming Medical Congresses
- 28-week data from Phase 2b VANTAGE PBC study highlighting improvements in itch and fatigue will be presented at EASL
- Data from Mirum studies presented at DDW, EASL, and ESPGHAN congresses
“We are excited to see the continued evidence generated by Mirum medicines and product candidates across a number of settings,” said
EASL –
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
Immune-mediated and Cholestatic Diseases Session,
Presented by:
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.
Additional data will be shared following the formal presentation on
EASL Symposium
Adult Cholestatic Liver Disease: Focusing on Clinical Outcomes and the Future of IBAT Inhibitors
An archive of the symposium will be available following the event.
DDW
Oral Presentations
Presentation 784: Volixibat for the Treatment of Cholestatic Pruritus in Primary Biliary Cholangitis: An Adaptive, Randomized, Placebo-controlled Phase 2B Trial (VANTAGE): Interim Results
Cholestatic Liver Disease session, Room 6C
Presented by
Presentation 1086: Comprehensive Analysis of Cholestasis Genetic Panel Results From 2016–2022 in Children and Young Adults: Insights into Diagnostic Yield
Human and Experimental Cholestatic and Autoimmune Liver Diseases, Room 6F
Presented by
Poster Presentations
Poster Su1756:
Genetic Insights Into PFIC-associated Genes in Unexplained Chronic Cholestasis and Liver Disease: Frequency and Implications of Variant Combinations
Pediatric Hepatology Session, DDW Poster Hall, C-E
Presented by
Poster Mo1627: Improvements in Pruritus, Serum Bile Acids, and Total Bilirubin Following Treatment with Maralixibat in Patients with Primary Sclerosing Cholangitis
Human and Experimental Cholestatic and Autoimmune Liver Diseases, DDW Poster Hall C-E
Presented by
ESPGHAN –
Oral presentations
Abstract 1066: Improvements in pruritus are associated with growth in patients with progressive familial intrahepatic cholestasis: Data from the MARCH-ON trial
Nutrition and Hepatology Session, Hall 3G
Presented by Professor
Abstract 1089: The relationship between serum bile acids and event-free survival following the use of maralixibat for progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH/MARCH-ON trials
Hepatology Abstracts, Hall 3G
Presented by Professor
Abstract 1110: Improvements in pruritus after maralixibat treatment are associated with improved health-related quality of life for patients with cholestatic liver disease
Hepatology Abstracts, Hall 3G
Presented by Dr.
Abstract 1098: Bile acid subspecies are correlated with pruritus and bilirubin improvement in PFIC patients treated with maralixibat: Data from MARCH and MARCH-ON
Hepatology Abstracts, Hall 3G
Presented by Dr. Henkjan Verkade,
Poster presentations
Abstract 1102: Greater improvements in bilirubin were observed in pruritus responders after maralixibat treatment in patients with PFIC: data from the MARCH/MARCH-ON trials
General Hepatology 02 Session, e-
Presented by Professor
Abstract 1094: Impact of long-term maralixibat treatment on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in
General Hepatology 03 & Transplantation Session, e-
Presented by Dr.
Abstract 1107: Clinical benefits of maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: Data from the MERGE study
General Hepatology Session 02, e-
Presented by Dr.
ESPGHAN Symposium
Hall 3C
Growing Knowledge and Evidence: Long-term impact of IBAT inhibition
Full presentations will be available within the Publications and Presentations section on Mirum’s website.
About Volixibat
Volixibat is an 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 June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC.
About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets
LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the
LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.
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 Alagille syndrome 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 Alagille syndrome 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.
LIVMARLI is available in oral solution or tablet formulations and is taken by mouth 30 minutes before a meal. For Alagille syndrome, LIVMARLI is taken one time each day in the morning. For PFIC, LIVMARLI is taken two times each day. If you take the oral solution, use the oral dosing dispenser to measure your dose.
US Prescribing Information
EU SmPC
Canadian Product Monograph
About
LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the
CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.
Mirum's late-stage pipeline includes two investigational treatments for several rare diseases.
Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder.
To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (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 anticipated presentations of data from Mirum’s LIVMARLI and volixibat studies, including the VANTAGE study of volixibat in patients with pruritus due to PBC. 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 “will,” “could,” “can,” “would,” “potential,” “hope,” “opportunity,” 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 associated with Mirum’s business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum’s Annual Report on Form 10-K for the year ended
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