- LIVMARLI (maralixibat) late-breaker oral
presentation on a six-year event-free survival analysis in Alagille
syndrome compared to a natural history cohort; selected for Best of
the Liver Meeting
- Second late-breaker presentation to highlight
predictors of event-free survival in patients treated with
maralixibat.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that
new data will be presented at the American Association for the
Study of Liver Disease annual congress, The Liver Meeting®,
November 12-15, 2021.
Highlights of the meeting will take place during two
late-breaker sessions where new analyses from LIVMARLI™
(maralixibat) oral solution clinical studies will be presented. The
late-breaker oral presentation will provide real world evidence
analytics on a six-year event-free survival analysis in patients
with Alagille syndrome (ALGS) treated with maralixibat compared
with a natural history control cohort from the Global Alagille
Alliance Study (GALA) database. The second late-breaker
presentation is a poster which will highlight an analysis of
predictors of long-term event-free survival and transplant-free
survival in patients with ALGS enrolled in three long-term
maralixibat clinical trials.
All abstracts are now available on the AASLD website and in the
October supplement of HEPATOLOGY.
LATE-BREAKER PRESENTATIONS Oral session Abstract LO: Application of real-world
evidence analytics: A 6-year event-free survival analysis in
Alagille syndrome of the GALA clinical research database and
maralixibat treated patients Bettina E. Hansen, PhD and Binita
M. Kamath, MBBChir, on behalf of the GALA study group; presented by
Bettina Hansen, Senior Biostatistician, Toronto Centre for Liver
Disease, Scientist, Toronto General Hospital Research Institute
& Associate Professor, Institute of Health Policy, Management
and Evaluation, University of Toronto Monday, November 15, 2021 –
12:30 p.m. ET This abstract has been selected as a part of the Best
of the Liver Meeting series.
Poster session Abstract LP16:
Predictors of 6-Year event-free survival in patients with Alagille
syndrome treated with maralixibat, an IBAT inhibitor Ronald J.
Sokol, MD, FAASLD, Professor of Pediatrics and Vice Chair of
Clinical and Translational Research in the Department of Pediatrics
at the University of Colorado School of Medicine (SOM) and
Children’s Hospital Colorado
POSTER PRESENTATIONS
Abstract 1955: Healthcare
resource utilization in patients with Alagille syndrome Noelle
Ebel, MD, Clinical Assistant Professor, Director of the Alagille
Syndrome Program, and Associate Program Director for the Transplant
Hepatology Fellowship at Stanford University
Abstract 1946: Cost of pediatric
liver transplant among commercial and Medicaid insured patients
Tamir Miloh, MD, Professor of Pediatrics and Medical Director of
Pediatric Transplant Hepatology, Miami Transplant Institute
About LIVMARLI™ (maralixibat) oral solution
LIVMARLI™ (maralixibat) oral solution is an orally administered,
once-daily, ileal bile acid transporter (IBAT) inhibitor approved
by the U.S. Food and Drug Administration for the treatment of
cholestatic pruritus in patients with Alagille syndrome one year of
age and older and is the only FDA-approved medication to treat
cholestatic pruritus associated with Alagille syndrome. For more
information, please visit LIVMARLI.com.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including
progressive familial intrahepatic cholestasis and biliary atresia,
of which both have received Breakthrough Therapy designation and
Orphan Drug designation. To learn more about ongoing clinical
trials with LIVMARLI, please visit Mirum’s clinical trials section
on the company’s website.
About Alagille syndrome
Alagille syndrome (ALGS) is a rare genetic disorder in which
bile ducts are abnormally narrow, malformed and reduced in number,
which leads to bile accumulation in the liver and ultimately
progressive liver disease. The estimated incidence of ALGS is one
in every 30,000 people.1 In patients with ALGS, multiple organ
systems may be affected by the mutation, including the liver,
heart, kidneys and central nervous system.2 The accumulation of
bile acids prevents the liver from working properly to eliminate
waste from the bloodstream and, according to recent reports, 60% to
75% of patients with ALGS have a liver transplant before reaching
adulthood.3 Signs and symptoms arising from liver damage in ALGS
may include jaundice (yellowing of the skin), xanthomas
(disfiguring cholesterol deposits under the skin), and pruritus
(itch)2. The pruritus experienced by patients with ALGS is among
the most severe in any chronic liver disease and is present in most
affected children by the third year of life.4
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause serious side effects, including:
Changes in liver tests. Changes in certain liver tests
are common in patients with Alagille syndrome and can worsen during
treatment with LIVMARLI. These changes may be a sign of liver
injury and can be serious. Your healthcare provider should do blood
tests 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) or loss of
appetite.
Stomach and intestinal (gastrointestinal) problems.
LIVMARLI can cause stomach and intestinal problems, including
diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with
Alagille syndrome but may worsen during treatment. Your healthcare
provider should do blood tests before starting and during
treatment.
Other common side effects reported during treatment were bone
fractures and gastrointestinal bleeding.
Prescribing information
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company
dedicated to transforming the treatment of rare liver diseases.
Mirum’s approved medication is LIVMARLI™ (maralixibat) oral
solution which is approved in the U.S. for the treatment of
cholestatic pruritus in patients with Alagille syndrome one year of
age and older.
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases affecting children and
adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter
(IBAT) inhibitor, is currently being evaluated in clinical trials
for pediatric liver diseases and includes the MARCH Phase 3 study
for progressive familial intrahepatic cholestasis (PFIC) and the
EMBARK Phase 2b study for patients with biliary atresia. In
addition, Mirum has an expanded access program open in Canada,
Australia, the UK and several countries in Europe for eligible
patients with Alagille syndrome.
Mirum has submitted a Marketing Authorization Application to the
European Medicines Agency for maralixibat for the treatment of
cholestatic liver disease in patients with Alagille syndrome.
Mirum’s second investigational treatment, volixibat, also an
oral IBAT inhibitor, is being evaluated in two registrational
studies including the OHANA Phase 2b study for pregnant women with
intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study
for adults with primary sclerosing cholangitis. Mirum is planning
to launch a Phase 2b study in primary biliary cholangitis later
this year.
To augment its pipeline in cholestatic liver disease, Mirum has
acquired the exclusive option to develop and commercialize gene
therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2,
respectively, from Vivet Therapeutics SAS, following preclinical
evaluation and investigational new drug-enabling studies.
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Forward-Looking Statements
This press release includes forward-looking statements
pertaining to the Company’s planned participation at a scientific
conference, which may include discussion of the Company’s business
and operations, including the discovery, development and
commercialization of our product candidates and technologies, and
the therapeutic potential thereof, the success of our
collaborations with partners and any potential future
collaborations. Such forward-looking statements are subject to
risks and uncertainties that could cause actual results to differ
materially from those expressed or implied in such statements.
Applicable risks and uncertainties include those relating to our
preclinical research and clinical programs and other risks
identified under the heading “Risk Factors” included in our most
recent Form 10-Q and Form 10-K filings and in other future filings
with the SEC. The forward-looking statements contained in this
press release reflect Mirum's current views with respect to future
events, and Mirum does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
The Liver Meeting® is a registered trademark of the American
Association for the Study of Liver Diseases.
1Danks, et al. Archives of Disease in Childhood 1977 2Johns
Hopkins Medicine.
hopkinsmedicine.org/health/conditions-and-diseases/Alagille-syndrome
3Vandriel, et al. GALA EASL 2020; Kamath, et al. Hepatology
Communications 2020 4Elisofon, et al. Journal of Pediatric
Gastroenterology and Nutrition 2010
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Mirum Contacts:
Media: Erin Murphy media@mirumpharma.com
Investors: Ian Clements, Ph.D. ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
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