- Seven abstracts highlighting ALGS and PFIC
data, including three oral presentations
- Long-term extension data from Phase 3
MARCH-ON PFIC study presented at plenary session and selected among
the highest scoring abstracts
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data
presented during the 56th European Society for Paediatric,
Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual
Meeting which took place this week in Milan, Italy. Data from
LIVMARLI® (maralixibat) oral solution clinical studies and
real-world settings in Alagille syndrome (ALGS) and Progressive
Familial Intrahepatic Cholestasis (PFIC) were presented in oral and
poster presentations during the meeting.
“We continue to build upon the strong body of evidence
demonstrating LIVMARLI’s potential to provide long-term benefit to
PFIC patients across key quality of life and liver disease
parameters, as well as improvements in varied genetic types of
PFIC,” said Pam Vig, PhD, chief scientific officer and head of
research at Mirum. “Further, we are pleased to demonstrate data
that shows children treated with LIVMARLI are reducing or
discontinuing some antipruritic medications and nutritional
supplements.”
Abstract 587: Long-term
Maintenance of Response and Improved Liver Health with Maralixibat
in Patients with Progressive Familial Intrahepatic Cholestasis:
2-year Data From the MARCH-ON Study *Plenary Session:
Hepatology Highest Scoring Abstracts* Presented by Professor
Richard J. Thompson, MD, King’s College London, United Kingdom
Patients with PFIC showed significant and sustained improvements in
pruritus severity, serum bile acid (sBA) levels, total bilirubin
and growth following up to two years of LIVMARLI treatment. Similar
improvements in pruritus and sBA were seen in patients originally
randomized to placebo who received LIVMARLI in the open-label
study.
Abstract 594: Maralixibat Leads
to Significant Reductions in Bilirubin for Patients with
Progressive Familial Intrahepatic Cholestasis: Data from the MARCH
Trial Presented by Lorenzo D’Antiga, MD, Papa Giovanni XXIII
Hospital, Bergamo, Italy Patients treated with LIVMARLI experienced
significant decreases in both total and direct bilirubin compared
to placebo. 40% of the patients with abnormal bilirubin at baseline
treated with LIVMARLI achieved normalization versus none in the
placebo group. Further, these reductions in bilirubin were
consistent with reductions in sBAs.
Abstract 600: Maralixibat Impact
on Concomitant Medication Use for the Treatment of Cholestatic
Pruritus in Alagille Syndrome: Real-World Experience Presented
by Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster
City, California, USA Real-world evidence from 116 patients treated
with LIVMARLI for at least one year showed that more than one-third
of patients were able to discontinue ≥1 concomitant antipruritic
medication, and one in five patients discontinued ≥2 medications.
Reductions in concomitant medication usage were seen across all
medication types and suggest that LIVMARLI may reduce the
polypharmacy burden within the first year of treatment.
Other presentations featured during ESPGHAN include:
Abstract 592: Maralixibat Leads
to Significant Improvements in Cholestatic Pruritus for Children
with Progressive Familial Intrahepatic Cholestasis Without a
Genetic Diagnosis: Data from the MARCH Trial Presented by
Professor Richard J. Thompson, MD, King’s College, London, United
Kingdom
Abstract 599: Maralixibat Leads
to Improvements in Cholestatic Pruritus for Children with
Progressive Familial Intrahepatic Cholestasis Due to MDR3
Deficiency: Data From the MARCH/MARCH-ON Trials Presented by
Professor Richard J. Thompson, MD, King’s College, London, United
Kingdom
Abstract 595: Improvements in
Pruritus with Maralixibat are Associated with Improved Quality of
Life for Patients with Progressive Familial Intrahepatic
Cholestasis: Data From the MARCH Trial Presented by Douglas B.
Mogul, MD, PhD, Mirum Pharmaceuticals, Inc., Foster City,
California, USA
Abstract 597: Maralixibat Can
Improve Cholestatic Pruritus in Children with Progressive Familial
Intrahepatic Cholestasis Who Previously Underwent a Surgical
Biliary Diversion: Data From the MARCH/MARCH-ON Trials
Presented by Lorenzo D’Antiga, MD, Papa Giovanni XXIII Hospital,
Bergamo, Italy
To view the full presentations, please visit the Publications
section of Mirum’s website.
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 (ALGS)
three months of age and older and for progressive familial
intrahepatic cholestasis (PFIC) five years of age and older.
LIVMARLI is also the only approved IBAT inhibitor approved by
the European Commission for the treatment of cholestatic pruritus
in patients with ALGS two months and older, and by Health Canada
for the treatment of cholestatic pruritus in ALGS. For more
information for U.S. residents, please visit LIVMARLI.com.
Mirum has also submitted LIVMARLI for approval in Europe in PFIC
for patients two months of age and older.
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 five
years of age and older. Mirum has submitted for approval in Europe
for the treatment of PFIC in patients two months of age and older.
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 2b VISTAS study for primary sclerosing
cholangitis and Phase 2b VANTAGE study for primary biliary
cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3
clinical study, RESTORE, to treat patients with CTX, with positive
topline results reported in 2023.
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, which
may include discussion of the Company’s clinical and research data
relating to the therapeutic potential and/or commercial viability
of LIVMARLI in various liver disease indications and in patient
populations that are investigational only. 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,” “goal,” “potential” and similar
expressions are intended to identify forward-looking statements.
The accuracy of such statements is subject to a number of risks,
uncertainties and assumptions including, but are not limited to,
the following factors: the uncertainties inherent in research and
development; the uncertainties inherent in business and financial
planning, including, without limitation, risks related to Mirum’s
business and prospects, adverse developments in our focused
markets, or adverse developments in the U.S. or global regulatory
environment or economies generally; the continued impact of
COVID-19 on our business, operations and financial results; and
competitive developments. Other factors that might cause such a
difference include those discussed in the Company’s filings with
the SEC. 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240518739027/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben investors@mirumphama.com
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