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Mirum Pharmaceuticals to Showcase Data from its LIVMARLI and Volixibat Clinical Programs at AASLD’s The Liver Meeting

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Mirum Pharmaceuticals (NASDAQ: MIRM) announced its participation at AASLD's The Liver Meeting, scheduled for November 15-19, 2024, in San Diego. The company will present multiple clinical studies including the VANTAGE trial studying volixibat for primary biliary cholangitis, MARCH-ON trial data on Progressive Familial Intrahepatic Cholestasis, and research on LIVMARLI (maralixibat). Presentations will cover various aspects including pruritus improvements, caregiver burden, real-world usage in biliary atresia, and bile acid correlations. The event will also feature a fireside discussion with a physician and an ALGS patient sharing experience with LIVMARLI.

Mirum Pharmaceuticals (NASDAQ: MIRM) ha annunciato la sua partecipazione all'AASLD's The Liver Meeting, in programma dal 15 al 19 novembre 2024 a San Diego. L'azienda presenterà numerosi studi clinici tra cui il trial VANTAGE che studia il volixibat per la colangite biliare primaria, i dati del trial MARCH-ON sulla Colestasi Intraepatica Familiare Progressiva e ricerche su LIVMARLI (maralixibat). Le presentazioni tratteranno diversi aspetti, compresi i miglioramenti del prurito, il carico sui caregiver, l'uso reale nella atresia biliare e le correlazioni con gli acidi biliari. L'evento includerà anche una discussione informale con un medico e un paziente affetto da ALGS che condivideranno la loro esperienza con LIVMARLI.

Mirum Pharmaceuticals (NASDAQ: MIRM) anunció su participación en The Liver Meeting de la AASLD, programado del 15 al 19 de noviembre de 2024 en San Diego. La compañía presentará varios estudios clínicos, incluyendo el ensayo VANTAGE que estudia el volixibat para la colangitis biliar primaria, datos del ensayo MARCH-ON sobre la Colestasis Intrahepática Familiar Progresiva y investigaciones sobre LIVMARLI (maralixibat). Las presentaciones cubrirán diversos aspectos, incluyendo mejoras en el prurito, la carga sobre los cuidadores, el uso en el mundo real en la atresia biliar y correlaciones con los ácidos biliares. El evento también contará con una discusión junto a la chimenea con un médico y un paciente de ALGS que compartirán su experiencia con LIVMARLI.

Mirum Pharmaceuticals (NASDAQ: MIRM)은 2024년 11월 15일부터 19일까지 샌디에이고에서 열리는 AASLD의 The Liver Meeting에 참석한다고 발표했습니다. 이 회사는 기본 담즙성 담관염을 위한 volixibat를 연구하는 VANTAGE 시험, 진행성 가족성 간내 담즙 정체에 대한 MARCH-ON 시험 데이터, LIVMARLI(maralixibat)에 대한 연구를 포함한 다수의 임상 연구를 발표할 예정입니다. 발표 내용은 가려움증 개선, 간병인 부담, 담즙 색전증에서의 실제 사용 및 담즙산 상관관계를 포함한 다양한 측면을 다룰 것입니다. 또한, 이 행사에서는 LIVMARLI에 대한 경험을 공유하는 의사와 ALGS 환자가 함께하는 간담회도 진행됩니다.

Mirum Pharmaceuticals (NASDAQ: MIRM) a annoncé sa participation à The Liver Meeting de l'AASLD, qui se tiendra du 15 au 19 novembre 2024 à San Diego. L'entreprise présentera plusieurs études cliniques, y compris l'essai VANTAGE étudiant le volixibat pour la cholangite biliaire primitive, les données de l'essai MARCH-ON sur la cholestase intra-hépatique familiale progressive et des recherches sur LIVMARLI (maralixibat). Les présentations couvriront divers aspects, y compris les améliorations des démangeaisons, le fardeau des aidants, l'utilisation réelle dans l'atrésie biliaire et les corrélations avec les acides biliaires. L'événement comprendra également une discussion autour du feu avec un médecin et un patient ALGS partageant leur expérience avec LIVMARLI.

Mirum Pharmaceuticals (NASDAQ: MIRM) gab die Teilnahme an der The Liver Meeting der AASLD bekannt, die vom 15. bis 19. November 2024 in San Diego stattfinden wird. Das Unternehmen wird mehrere klinische Studien präsentieren, darunter die VANTAGE-Studie, die volixibat bei primärer biliärer Cholangitis untersucht, die Daten der MARCH-ON-Studie zur progressiven familiären intrahepatischen Cholestase sowie Forschungen zu LIVMARLI (maralixibat). Die Präsentationen werden verschiedene Aspekte abdecken, darunter Verbesserungen beim Pruritus, die Belastung der Angehörigen, die tatsächliche Nutzung bei biliärer Atresie und die Zusammenhänge mit Gallensäuren. Die Veranstaltung wird auch eine informelle Diskussion mit einem Arzt und einem ALGS-Patienten beinhalten, die ihre Erfahrungen mit LIVMARLI teilen.

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- Late-breaker presentation to feature data from VANTAGE study evaluating volixibat in primary biliary cholangitis

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will present data at the American Association for the Study of Liver Disease’s (AASLD) The Liver Meeting®, taking place November 15-19, 2024, in San Diego, California.

Enclosed below are the titles that have been accepted for presentation during the meeting. The abstracts are available via the AASLD website. Full analyses will be available following their presentation within the Publications & Presentation section on Mirum’s website.

Presentations

Abstract #5038: Volixibat for Cholestatic Pruritus in Primary Biliary Cholangitis: An Adaptive, Randomized, Placebo-controlled Phase 2b Trial (VANTAGE): Interim Results
**Late-breaker poster presentation**
Monday, November 18 from 1:00-2:00pm, Poster Hall C
Presented by Dr. Kris Kowdley, Washington State University, Seattle, Washington, USA

Abstract #184: Improvements in Pruritus are Associated with Improvements in Growth in Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH-ON trial
**Oral presentation**
Sunday, November 17 from 12:15-12:30pm during the ‘Advances in Pediatric Liver Disease’ session, Ballroom 6C
Presented by Dr. Alexander Miethke, Cincinnati Children’s Hospital, Cincinnati, Ohio, USA

Abstract #4347: Pilot Study of Volixibat Co-administered with OCA for Primary Biliary Cholangitis (PBC) Treatment: The VLX-602 Trial
Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C
Presented by Dr. Kris Kowdley, Washington State University, Seattle, Washington, USA

Abstract #4400: Impact of Maralixibat on Caregiver Burden for Patients with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis
Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C
Presented by Dr. Natasha Dilwali, The Johns Hopkins School of Medicine, Baltimore, Maryland, USA

Abstract #4419: Real-world Use of Maralixibat in Biliary Atresia: A Case Series
Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C
Presented by Dr. Natasha Dilwali, The Johns Hopkins School of Medicine, Baltimore, Maryland, USA

Abstract #4432: The Relationship Between Serum Bile Acids and Event-Free Survival Following the Use of Maralixibat for Progressive Familial Intrahepatic Cholestasis: Data from MARCH/MARCH-ON
Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C
Presented by Professor Richard Thompson, King’s College London, United Kingdom

Abstract #4436: Bile Acid Subspecies are Correlated with Pruritus and Bilirubin Improvement in PFIC Patients Treated with Maralixibat: Data from MARCH and MARCH-ON
Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C
Presented by Professor Henkjan Verkade, University of Groningen, Groningen, Netherlands

Alagille Syndrome Patient & Physician Fireside Discussion
During the meeting, Mirum will host a session spotlighting a pediatric hepatologist and an adult patient living with ALGS. Dr. Ajay Jain, St. Louis University, will discuss clinical insights and patient, Emma, will share her experience with LIVMARLI.
Sunday, November 17 from 1:30-2:00 p.m. in Exhibit Hall TLM Theater #2

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution 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 ALGS and PFIC. 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 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.

US Prescribing Information
EU SmPC
Canadian Product Monograph

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.

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 U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver 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. Lastly, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S.

To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).

Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s planned participation at a scientific conference, including data presentation title and synopsis for both commercial and clinical programs, which may include discussion of the Company’s clinical and research data relating to the therapeutic potential and/or commercial viability of LIVMARLI or other Company product candidates in various liver disease indications and in patient populations that are investigational only. Words such as “will,” “could,” “would,” “potential” 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 and the other risks described in Mirum’s filings with the Securities and Exchange Commission. 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. A further description of risks and uncertainties can be found in Mirum’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors,” as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov.

Media Contact:

Erin Murphy

media@mirumpharma.com

Investor Contact:

Andrew McKibben

ir@mirumphama.com

Source: Mirum Pharmaceuticals, Inc.

FAQ

When will Mirum Pharmaceuticals (MIRM) present at The Liver Meeting 2024?

Mirum Pharmaceuticals will present at The Liver Meeting from November 15-19, 2024, in San Diego, California.

What are the key clinical trials MIRM will present at AASLD 2024?

MIRM will present data from the VANTAGE study on volixibat, MARCH-ON trial on Progressive Familial Intrahepatic Cholestasis, and VLX-602 trial on volixibat co-administered with OCA for Primary Biliary Cholangitis.

What will be discussed in MIRM's late-breaker presentation at AASLD 2024?

The late-breaker presentation will feature interim results from the VANTAGE study, evaluating volixibat for cholestatic pruritus in primary biliary cholangitis.

Mirum Pharmaceuticals, Inc.

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