- Data demonstrate significant benefit across
broadest range of genetic PFIC types ever studied
- Significant improvements observed across
multiple parameters including pruritus, serum bile acids, growth
and bilirubin
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that
data from the Phase 3 MARCH-PFIC study evaluating LIVMARLI®
(maralixibat) oral solution in patients with PFIC was published in
The Lancet Gastroenterology and Hepatology.
The MARCH-PFIC study was the largest, randomized, double-blind,
placebo-controlled study in patients with PFIC, confirming the
efficacy and safety of LIVMARLI, an IBAT inhibitor, across a broad
genetic range of PFIC types. The main efficacy analyses were
performed in two groups which included the BSEP deficiency (BSEP or
PFIC2) cohort and the All-PFIC cohort which included patients with
BSEP (PFIC2), FIC1 (PFIC1), MDR3 (PFIC3), TJP2 (PFIC4), or MYO5B
deficiencies.
The study met both the primary efficacy endpoint (mean change in
pruritus severity score between baseline and the last 12 weeks of
treatment [weeks 15-26]) and the key secondary endpoint (mean
change in total serum bile acid concentration between baseline and
the average of weeks 18, 22, and 26) with statistically significant
and clinically meaningful improvements observed by Week 2 and
sustained throughout the 26-week study in LIVMARLI-treated
participants in both the BSEP and All-PFIC cohorts. Improvements in
bilirubin concentrations, growth, and sleep disturbances with
LIVMARLI versus placebo were also observed. There were no new
safety signals for LIVMARLI throughout the treatment periods with
the most common adverse event being diarrhea, which was mostly mild
and transient.
The Phase 3 MARCH study presents the largest and most
comprehensive dataset demonstrating the therapeutic benefit of an
IBAT inhibitor across a broad range of PFIC types, including some
which have not been previously studied. In addition to the clinical
benefits observed, LIVMARLI significantly improved serum bile acids
and bilirubin which are both known to be predictive of
transplant-free survival. LIVMARLI represents a non-surgical,
pharmacological option to improve outcomes for patients with
PFIC.
“These data advance our understanding of LIVMARLI’s potential to
provide meaningful improvements in pruritus and a number of
parameters impacting patients with various PFIC types. The
clinically meaningful reductions in serum bile acid and bilirubin
levels also signify the potential for improvements in native liver
survival in PFIC, a step forward in considering the long-term care
and health of patients with PFIC,” said Dr. Alexander Miethke,
Cincinnati Children’s Hospital and lead author on the publication.
“LIVMARLI presents a new non-surgical treatment option in PFIC with
strong efficacy data and safety demonstrated across a range of PFIC
types, which is important for patients with PFIC types previously
unstudied.”
“We are pleased to see these data published and recognized by
The Lancet as they demonstrate meaningful improvements in many
clinical signs and symptoms seen in PFIC patients,” said Pam Vig,
PhD, chief scientific officer and head of research at Mirum.
“Patients with PFIC suffer from cholestasis which can lead to
debilitating cholestatic pruritus, as well as poor growth and
progressive liver disease. These data demonstrate the significant
impact LIVMARLI can have in this severe cholestatic setting.”
To read the full article, please visit the publication on The
Lancet Gastroenterology and Hepatology’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 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
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, the potential for LIVMARLI to
provide meaningful resolution to pruritis in PFIC and the clinical
significance of reduction of serum bile acids and total bilirubin
on native liver survival. 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. 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. 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/20240507339100/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben investors@mirumphama.com
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