- Late-breaker presentation to feature data
from VANTAGE study evaluating volixibat in primary biliary
cholangitis
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20241114053418/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben ir@mirumphama.com
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