- Data showcases sustained clinical benefit
following seven years of LIVMARLI treatment in patients with
Alagille syndrome
- Long-term extension data from LIVMARLI PFIC
studies highlight improvement in key liver markers and growth
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that
data from new analyses showing long-term treatment with LIVMARLI
were presented during the European Association for the Study of the
Liver (EASL) Annual Congress, June 5-8, 2024, in Milan, Italy.
Presentations from studies evaluating LIVMARLI® (maralixibat) oral
solution in patients with Alagille syndrome (ALGS) and Progressive
Familial Intrahepatic Cholestasis (PFIC) included:
THU-094: Clinical Benefits with Maralixibat for Patients with
Alagille Syndrome are Durable Through 7 Years of Treatment: Data
from the MERGE Study Presented by Doug Mogul, MD, PhD – Mirum
Pharmaceuticals, Inc., Foster City, CA, USA An analysis of ALGS
studies evaluating LIVMARLI for up to seven years of treatment were
presented to demonstrate the durability of response to LIVMARLI
across multiple clinical and laboratory outcomes. Data demonstrated
that nearly all patients who remained on LIVMARLI for seven years
experienced benefit including clinically meaningful reductions in
pruritus and serum bile acids, along with improvements in
height.
View the full presentation.
THU-129: Maralixibat Improves Growth in Patients with
Progressive Familial Intrahepatic Cholestasis: Data from the
MARCH/MARCH-ON Trials Presented by Professor Richard J.
Thompson, MD, PhD – King's College, London, UK This analysis
reported on the long-term impact of LIVMARLI on improvements in
growth across a variety of PFIC types in 60 patients from the
All-PFIC cohort of the MARCH/MARCH-ON clinical trials. Patients
treated with LIVMARLI from the All-PFIC cohort had a statistically
significant improvement in height and weight z-scores which
persisted through 82 weeks of treatment.
View the full presentation.
THU-158: Improvements in Serum Bile Acid Levels are
Associated with Improvements in Key Markers of Liver Health After
Maralixibat Treatment in Children with Progressive Familial
Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials
Presented by Lorenzo D’Antiga, MD - Papa Giovanni XXIII Hospital,
Bergamo, Italy This analysis aimed to assess correlations between
serum bile acid (sBA) improvements and key liver parameters,
including bilirubin. These data showed that reductions in sBA after
treatment with LIVMARLI are correlated with reductions in
bilirubin, a key marker of liver disease. These results indicate
the potential for LIVMARLI to have disease modifying effects and
improvements in liver health for patients with PFIC.
View the full presentation.
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.
LIVMARLI has received positive opinion by CHMP in Europe for the
treatment of PFIC in patients three months of age and older. A
decision by the European Commission is expected by the third
quarter of 2024.
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. LIVMARLI has received positive opinion by
CHMP in Europe for the treatment of PFIC in patients three months
of age and older. A decision by the European Commission is expected
by the third quarter of 2024. 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
regarding patient growth data while on LIVMARLI, the reliability of
long-term data, and 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/20240606079628/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben investors@mirumphama.com
Mirum Pharmaceuticals (NASDAQ:MIRM)
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