- Late-breaker poster presentation accepted
featuring LIVMARLI® data showing long-term maintenance of
response in patients with PFIC
- Four poster presentations will highlight
LIVMARLI PFIC data and volixibat proof-of-concept trial data in
intrahepatic cholestasis of pregnancy
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that
data from its LIVMARLI and volixibat programs will be presented at
The Liver Meeting®, the American Association for the Study of Liver
Diseases (AASLD) annual meeting, taking place November 10-14, 2023,
in Boston, Massachusetts.
Presentations during AASLD
Late-breaker Poster Presentation
November 13, 2023 from 1:00-2:00 p.m. as part of the late-breaker
poster presentation session Poster Number 5048-C: Long-term
maintenance of response and improved liver health with maralixibat
in patients with progressive familial intrahepatic cholestasis
(PFIC): 2-year data from the MARCH-ON study
Poster Presentations Presented
November 13, 2023 from 1:00-2:00 p.m. Poster 4602-C:
Improvements in pruritus with maralixibat are associated with
improved quality of life for patients with progressive familial
intrahepatic cholestasis: Data from the MARCH-PFIC trial
Poster 4604-C: Maralixibat leads to significant
improvement in cholestatic pruritus for children with progressive
familial intrahepatic cholestasis due to TJP2 or MYO5B deficiency:
Data from the MARCH-PFIC trial
Poster 4605-C: Maralixibat leads to significant
improvements in cholestatic pruritus for children with progressive
familial intrahepatic cholestasis without a genetic diagnosis: Data
from the MARCH-PFIC trial
Poster 4547-C: Efficacy, safety and tolerability of
volixibat in patients with intrahepatic cholestasis of pregnancy: A
case series of 4 patients
Full data will be available within the Publications and
Presentations section on Mirum’s website on November 13.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered,
once-daily, ileal bile acid transporter (IBAT) inhibitor and the
only approved medication 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.
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 the U.S. in
cholestatic pruritus in PFIC patients three months of age and
older, and in Europe, in PFIC for patients two months of age and
older.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including biliary
atresia. LIVMARLI has received Breakthrough Therapy designation for
ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and
biliary atresia. To learn more about ongoing clinical trials with
LIVMARLI, please visit Mirum’s clinical trials section on the
company’s website.
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause 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
gastrointestinal bleeding and bone fractures.
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
cholestatic pruritus in patients with Alagille syndrome in the U.S.
(three months and older), in Europe (two months and older), and in
Canada. Mirum has also submitted LIVMARLI for approval in the U.S.
in cholestatic pruritus in PFIC patients three months of age and
older and in Europe in PFIC for patients two months of age and
older. Cholbam is FDA-approved for the treatment of bile acid
synthesis disorders due to single enzyme defects 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 three investigational
treatments for debilitating liver diseases. The LIVMARLI
development program includes the Phase 2b EMBARK study for biliary
atresia. Mirum’s second investigational IBAT inhibitor is
volixibat, which 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.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter.
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,
including the therapeutic potential and/or commercial viability of
our product candidates and technologies in various liver disease
indications. 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/20231030353736/en/
Media: Erin Murphy 510-508-6521 media@mirumpharma.com
Investors: Andrew McKibben ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
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