Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare
liver disease, today announced that it has entered into a
definitive agreement to sell its Rare Pediatric Disease Priority
Review Voucher (“PRV”) for $110 million.
The PRV was granted by the U.S. Food and Drug Administration in
September 2021 with the approval of LIVMARLI™ (maralixibat) oral
solution for the treatment of cholestatic pruritus in patients with
Alagille syndrome one year of age and older.
Pursuant to the agreement, Mirum will receive a payment of $110
million upon the closing of the transaction, which is subject to
customary closing conditions and is expected to occur following the
expiration of the applicable U.S. antitrust clearance
requirements.
Jefferies LLC acted as the exclusive financial advisor to Mirum
for this transaction.
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 one year of
age and older and is the only FDA-approved medication to treat
cholestatic pruritus associated with Alagille syndrome. For more
information, please visit LIVMARLI.com.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including
progressive familial intrahepatic cholestasis and 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.
About Alagille syndrome
Alagille syndrome (ALGS) is a rare genetic disorder in which
bile ducts are abnormally narrow, malformed and reduced in number,
which leads to bile accumulation in the liver and ultimately
progressive liver disease. The estimated incidence of ALGS is one
in every 30,000 people.1 In patients with ALGS, multiple organ
systems may be affected by the mutation, including the liver,
heart, kidneys and central nervous system.2 The accumulation of
bile acids prevents the liver from working properly to eliminate
waste from the bloodstream and, according to recent reports, 60% to
75% of patients with ALGS have a liver transplant before reaching
adulthood.3 Signs and symptoms arising from liver damage in ALGS
may include jaundice (yellowing of the skin), xanthomas
(disfiguring cholesterol deposits under the skin), and pruritus
(itch)2. The pruritus experienced by patients with ALGS is among
the most severe in any chronic liver disease and is present in most
affected children by the third year of life.4
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause serious 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 bone
fractures and gastrointestinal bleeding.
Prescribing information
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company
dedicated to transforming the treatment of rare liver diseases.
Mirum’s approved medication is LIVMARLI™ (maralixibat) oral
solution which is approved in the U.S. for the treatment of
cholestatic pruritus in patients with Alagille syndrome one year of
age and older.
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases affecting children and
adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter
(IBAT) inhibitor, is currently being evaluated in clinical trials
for pediatric liver diseases and includes the MARCH Phase 3 study
for progressive familial intrahepatic cholestasis (PFIC) and the
EMBARK Phase 2b study for patients with biliary atresia. In
addition, Mirum has an expanded access program open in Canada,
Australia, the UK and several countries in Europe for eligible
patients with Alagille syndrome.
Mirum has submitted a Marketing Authorization Application to the
European Medicines Agency for maralixibat for the treatment of
cholestatic liver disease in patients with Alagille syndrome.
Mirum’s second investigational treatment, volixibat, also an
oral IBAT inhibitor, is being evaluated in two registrational
studies including the OHANA Phase 2b study for pregnant women with
intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study
for adults with primary sclerosing cholangitis. Mirum is planning
to launch a Phase 2b study in primary biliary cholangitis later
this year.
To augment its pipeline in cholestatic liver disease, Mirum has
acquired the exclusive option to develop and commercialize gene
therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2,
respectively, from Vivet Therapeutics SAS, following preclinical
evaluation and investigational new drug-enabling studies.
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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 closing of the PRV sale and
Mirum’s receipt of the proceeds from the proposed sale. 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,” “could,”
“would,” “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, 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.
References
1Danks, et al. Archives of Disease in Childhood 1977 2Johns
Hopkins Medicine.
hopkinsmedicine.org/health/conditions-and-diseases/Alagille-syndrome
3Vandriel, et al. GALA EASL 2020; Kamath, et al. Hepatology
Communications 2020 4Elisofon, et al. Journal of Pediatric
Gastroenterology and Nutrition 2010
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version on businesswire.com: https://www.businesswire.com/news/home/20211117005636/en/
Mirum Contacts: Media: Erin Murphy media@mirumpharma.com
Investors: Ian Clements, Ph.D.
ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com 212.600.1902
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