Mirum Pharmaceuticals Announces Data from EMBARK Phase 2b Study for Biliary Atresia
18 12월 2023 - 10:30PM
Business Wire
- Study did not meet primary or secondary
endpoints.
- LIVMARLI was generally well-tolerated, with
no new safety findings.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced
top-line results of the Phase 2 EMBARK study evaluating LIVMARLI®
(maralixibat) oral solution versus placebo given as an adjuvant
therapy to Kasai surgery in patients with biliary atresia. The
study did not meet its primary endpoint of mean change in bilirubin
from baseline to Week 26, or the key secondary endpoints. Baseline
characteristics were well-balanced between the groups. LIVMARLI was
generally well-tolerated, with no new safety findings.
“We are disappointed in the outcome of the study in this
post-surgery, high-need disease setting,” said Chris Peetz,
president and chief executive officer at Mirum. “We are grateful to
the patients, families, and clinicians who participated in the
study and advanced the science around this rare liver disease.”
About the EMBARK Study
The Phase 2b EMBARK study is a randomized, double-blind,
placebo-controlled clinical trial evaluating the safety and
efficacy of LIVMARLI® (maralixibat) oral solution in patients with
biliary atresia who have undergone a Kasai surgery. Patients
enrolled in the study were ≥21 days old and <90 days old at the
time of the Kasai surgery and had a clinical diagnosis of biliary
atresia; patients underwent a Kasai surgery within three weeks
prior to randomization in EMBARK. The primary endpoint was mean
change in total bilirubin through Week 26, followed by secondary
endpoints including mean change in total serum bile acids, and
proportion of patients who required liver transplant or had another
liver related event over the 26 weeks.
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
is 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 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.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Such forward-looking statements include statements
regarding, among other things Mirum’s clinical strategy, the
therapeutic potential of IBATs in biliary atresia and any potential
path forward for LIVMARLI in biliary atresia as well as the
progress of Mirum’s ongoing and planned studies for its product
candidates and the regulatory approval path for its product
candidates globally. 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 “forward,” “planned,” “poised,”, “positioned” “potential”,
“will” 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, the
impact of the COVID-19 pandemic, and the other risks described in
Mirum’s filings with the Securities and Exchange Commission. 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20231218379840/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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