Partnership leverages Takeda’s leadership in
rare disease, gastroenterology, and hepatology to advance
maralixibat in a major market
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Takeda
Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) announced that
the companies have entered into an exclusive licensing agreement
for the development and commercialization of maralixibat in Japan
for Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia (BA). Maralixibat, an
investigational, orally administered medication, is being evaluated
globally in ALGS, PFIC, and BA.
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Under the terms of the agreement, Takeda will be responsible for
regulatory approval and commercialization of maralixibat in Japan.
Takeda will also be responsible for development, including
conducting clinical studies in cholestatic indications.
“Takeda is a leading global biopharmaceutical company with
extensive experience in development and commercialization of novel
therapies to treat rare diseases as well as gastroenterology and
hepatology, making them an ideal partner as we look to accelerate
the delivery of maralixibat to children living with rare liver
diseases in Japan,” said Chris Peetz, president and chief executive
officer of Mirum. “As we approach potential commercialization in
the United States and complete the recent filing for Alagille
syndrome in Europe, our goal is to partner with top companies
outside of North America and Europe to ensure global reach for
patients with these terrible diseases. We are excited for Takeda to
engage in the development of maralixibat and collaborate in our
effort to advance this potentially life-changing therapy.”
“There is a significant unmet medical need for a treatment to
help patients with cholestatic diseases such as ALGS and PFIC in
Japan and developing novel treatment for those patients suffering
from rare liver diseases is a top priority for Takeda’s global
R&D strategy,” said Dr. Naoyoshi Hirota, general manager of
Takeda development center Japan. “This agreement reinforces
Takeda’s commitment to developing highly differentiated medicines
to improve the health and quality of life of patients.”
Mirum has submitted a new drug application (NDA) to the U.S.
Food and Drug Administration (FDA) for maralixibat for the
treatment of cholestatic pruritus in patients with Alagille
syndrome. The NDA is currently under priority review with a PDUFA,
or FDA decision date, of September 29, 2021. Mirum also recently
submitted a Marketing Authorization Application to the European
Medicines Agency for maralixibat for the treatment of cholestatic
liver disease in patients with ALGS.
About Maralixibat
Maralixibat is a novel, minimally absorbed, orally administered
investigational drug being evaluated in several rare cholestatic
liver diseases. Maralixibat inhibits the apical sodium dependent
bile acid transporter (ASBT), resulting in more bile acids being
excreted in the feces, leading to lower levels of bile acids
systemically, thereby potentially reducing bile acid mediated
effects. More than 1,600 individuals have received maralixibat,
including more than 120 children who have received maralixibat as
an investigational treatment for Alagille syndrome (ALGS) and
progressive familial intrahepatic cholestasis (PFIC). In the ICONIC
Phase 2b ALGS clinical trial, patients taking maralixibat had
significant reductions in bile acids and pruritus compared to
placebo. In a Phase 2 PFIC study, a genetically defined subset of
BSEP deficient (PFIC2), patients responded to maralixibat with an
increase in transplant-free survival. The U.S. Food and Drug
Administration has granted maralixibat Breakthrough Therapy
designation for the treatment of pruritus associated with ALGS in
patients one year of age and older and for PFIC2. Maralixibat was
shown to have a tolerable safety profile in the studies. The most
frequent treatment-related adverse events were diarrhea and
abdominal pain. Maralixibat has been studied extensively and its
safety database represents the largest database for an ASBT
inhibitor.
Until maralixibat is approved and available for prescribing, the
medication is available to patients with ALGS through Mirum’s
expanded access program. For more information, please visit
ALGSEAP.com. For further information about maralixibat’s ongoing
studies in pediatric liver disease, please visit the study
websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study
for biliary atresia.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company focused on the development and
commercialization of a late-stage pipeline of novel therapies for
debilitating liver diseases. Mirum’s lead product candidate,
maralixibat, is an investigational oral drug in development for
Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA
for maralixibat for the treatment of cholestatic pruritus in
patients with ALGS in the U.S. The NDA has been accepted for
priority review by the FDA with a PDUFA action date of September
29, 2021. Additionally, Mirum has submitted a marketing
authorization application for maralixibat to the European Medicines
Agency for the treatment of cholestatic liver disease for patients
with ALGS. Mirum is also developing volixibat, also an oral
ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic
cholestasis of pregnancy, and primary biliary cholangitis. For more
information, visit MirumPharma.com.
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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About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a
global, values-based, R&D-driven biopharmaceutical leader
headquartered in Japan, committed to discover and deliver
life-transforming treatments, guided by our commitment to patients,
our people and the planet. Takeda focuses its R&D efforts on
four therapeutic areas: Oncology, Rare Genetics and Hematology,
Neuroscience, and Gastroenterology (GI). We also make targeted
R&D investments in Plasma-Derived Therapies and Vaccines. We
are focusing on developing highly innovative medicines that
contribute to making a difference in people’s lives by advancing
the frontier of new treatment options and leveraging our enhanced
collaborative R&D engine and capabilities to create a robust,
modality-diverse pipeline. Our employees are committed to improving
quality of life for patients and to working with our partners in
health care in approximately 80 countries and regions.
For more information, visit https://www.takeda.com.
Disclaimer
The drug information contained herein is intended to disclose
corporate information. Nothing contained in this document should be
considered a solicitation, promotion, or indication for any
prescription drug, including those currently under development.
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 development and
commercialization by Takeda of maralixibat in Japan for various
indications, Mirum and Takeda’s receipt of revenue in connection
with the license agreement with Takeda, as well as the regulatory
approval pathway for maralixibat. 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.
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COMPANY CONTACTS: Mirum
Media: Erin Murphy media@mirumpharma.com
Takeda Media: Tatsuhiro Kanoo
Tatsuhiro.kanoo@takeda.com
Mirum Investors: Ian Clements, Ph.D.
ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
Takeda Investors: Christopher O’Reilly
takeda.ir.contact@takeda.com
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