- Mirum has acquired Travere’s rights and assets
related to Cholbam® and Chenodal®
- Travere received $210 million at closing and
is eligible for up to $235 million in potential sales-based
milestone payments
- Expands Mirum’s leadership in rare liver
disease with two additional commercial products and a near-term
Phase 3 label expansion opportunity
- Advances Travere’s strategy to deliver new
treatment standards from its pipeline of innovative medicines for
rare diseases and strengthens financial foundation
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) and Travere
Therapeutics, Inc. (NASDAQ: TVTX) today announced the completion of
Mirum’s previously announced acquisition of Travere’s bile acid
product portfolio that includes Cholbam® (cholic acid) and
Chenodal® (chenodiol), two medications addressing rare diseases in
high-need settings (the “Asset Purchase”). In connection with the
closing of the Asset Purchase, Travere received an upfront payment
of $210 million from Mirum, and remains eligible to receive up to
$235 million in potential sales-based milestone payments.
Mirum has acquired Travere’s rights to Cholbam, indicated 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 of liver disease, and Chenodal,
indicated for the treatment of radiolucent stones in the
gallbladder, which is also under Phase 3 clinical evaluation for
cerebrotendinous xanthomatosis (CTX). The closing of the Asset
Purchase was subject to the satisfaction of customary closing
conditions, including the expiration of the waiting period under
the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976. In
connection with the closing, Travere will provide certain
transitional services to Mirum.
To support the acquisition, Mirum secured $210 million in
funding from a syndicate of existing investors led by Frazier Life
Sciences and other existing stockholders and new investors that
include Avidity Partners, BVF Partners, Longitude Capital,
Sofinnova Investments, Inc., and an undisclosed leading healthcare
investment fund to finance the upfront payment for the
acquisition.
About Cholbam® (cholic acid)
The FDA approved Cholbam® (cholic acid) capsules in March 2015,
the first FDA-approved treatment for pediatric and adult patients
with bile acid synthesis disorders due to single enzyme defects,
and for adjunctive treatment of patients with peroxisome biogenesis
disorder-Zellweger spectrum disorder. The effectiveness of Cholbam®
has been demonstrated in clinical trials for bile acid synthesis
disorders and the adjunctive treatment of peroxisomal disorders. An
estimated 200 to 300 patients are current candidates for
therapy.
CHOLBAM® (cholic acid) Indication
Cholbam is a bile acid indicated for
- Treatment of bile acid synthesis disorders due to single enzyme
defects.
- Adjunctive treatment of peroxisomal disorders, including
Zellweger spectrum disorders, in patients who exhibit
manifestations of liver disease, steatorrhea, or complications from
decreased fat-soluble vitamin absorption.
LIMITATIONS OF USE
The safety and effectiveness of CHOLBAM on extrahepatic
manifestations of bile acid synthesis disorders due to single
enzyme defects or peroxisomal disorders, including Zellweger
spectrum disorders, have not been established.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS – Exacerbation of liver
impairment
- Monitor liver function and discontinue CHOLBAM in patients who
develop worsening of liver function while on treatment.
- Concurrent elevations of serum gamma glutamyltransferase (GGT)
and alanine aminotransferase (ALT) may indicate CHOLBAM
overdose.
- Discontinue treatment with CHOLBAM at any time if there are
clinical or laboratory indicators of worsening liver function or
cholestasis.
ADVERSE REACTIONS
- The most common adverse reactions (≥1%) are diarrhea, reflux
esophagitis, malaise, jaundice, skin lesion, nausea, abdominal
pain, intestinal polyp, urinary tract infection, and peripheral
neuropathy.
Please see full Prescribing Information for
additional Important Safety Information.
About Chenodal® (chenodiol)
Chenodal® is a synthetic oral form of chenodeoxycholic acid
(CDCA), a naturally occurring primary bile acid synthesized from
cholesterol in the liver. The FDA approved Chenodal for the
treatment of people with radiolucent stones in the gallbladder. In
2010, Chenodal was granted orphan drug designation for the
treatment of cerebrotendinous xanthomatosis "CTX), a rare autosomal
recessive lipid storage disease.
While Chenodal® is not currently approved for CTX, it received a
medical necessity determination in the U.S. by the FDA and has been
used as the standard of care for more than three decades. Efforts
are being made to obtain FDA approval of Chenodal for the treatment
of CTX and a Phase 3 clinical trial for this indication was
initiated in January 2020. The prevalence of CTX is estimated in
the literature to be as high as 1 in 70,000 in the overall
population.
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 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.
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases affecting children and
adults. LIVMARLI, an oral ileal bile acid transporter (IBAT)
inhibitor, is currently being evaluated in clinical trials for
pediatric liver diseases and includes the EMBARK Phase 2b clinical
trial for patients with biliary atresia. In addition, Mirum has an
expanded access program open across multiple countries for eligible
patients with ALGS and PFIC.
Mirum’s second investigational treatment, volixibat, an oral
IBAT inhibitor, is being evaluated in two potentially
registrational studies including the VISTAS Phase 2b clinical trial
for adults with primary sclerosing cholangitis and the VANTAGE
Phase 2b clinical trial for adults with primary biliary
cholangitis.
About Travere Therapeutics
At Travere Therapeutics, we are in rare for life. We are a
biopharmaceutical company that comes together every day to help
patients, families and caregivers of all backgrounds as they
navigate life with a rare disease. On this path, we know the need
for treatment options is urgent – that is why our global team works
with the rare disease community to identify, develop and deliver
life-changing therapies. In pursuit of this mission, we
continuously seek to understand the diverse perspectives of rare
patients and to courageously forge new paths to make a difference
in their lives and provide hope – today and tomorrow. For more
information, visit travere.com.
Mirum Pharmaceuticals, Inc. Forward-Looking
Statements
This press release contains “forward-looking statements" within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. Such forward-looking statements, including those regarding
the anticipated benefits of the Chenodal and Cholbam acquisition,
as well as expected commercial viability of such products and
potential additional indications being studied involve risks and
uncertainties. The Company’s experience and results may differ
materially from the experience and results anticipated in such
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: litigation relating to the
transactions (the “Asset Purchase”) discussed; risks that the Asset
Purchase disrupts the current plans or operations of the Company;
the ability of the Company to retain and hire key personnel;
competitive responses to the Asset Purchase; unexpected costs,
charges or expenses resulting from the Asset Purchase; potential
adverse reactions or changes to relationships with customers,
suppliers, distributors and other business partners resulting from
the announcement or completion of the Asset Purchase; the Company’s
ability to achieve the synergies expected from the Asset Purchase,
as well as delays, challenges and expenses associated with
integrating the businesses; the impact of overall industry and
general economic conditions, including inflation, interest rates
and related monetary policy by governments in response to
inflation; geopolitical events, and regulatory, economic and other
risks associated therewith; and continued uncertainty resulting
from broader macroeconomic conditions. 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.
Travere Therapeutics, Inc. Forward-Looking Statements
This press release contains “forward-looking statements” as that
term is defined in the Private Securities Litigation Reform Act of
1995. Without limiting the foregoing, these statements are often
identified by the words “look forward to”, “will,” “may”, “might”,
“believes”, “anticipates”, “plans”, “expects”, “intends,”
“potential” or similar expressions. In addition, expressions of
strategies, intentions or plans are also forward-looking
statements. Such forward-looking statements include, but are not
limited to, references to: the potential sales-based milestone
payments under the purchase agreement; estimated patient
populations; and expectations regarding Travere’s products,
pipeline, and strategy to deliver new treatment standards. Such
forward-looking statements are based on current expectations and
involve inherent risks and uncertainties, including factors that
could delay, divert or change any of them, and could cause actual
outcomes and results to differ materially from current
expectations. No forward-looking statement can be guaranteed.
Travere faces the risk that disruptions from the Asset Purchase and
the transition of the business to the acquiror will harm Travere’s
business, including current plans and operations; potential adverse
reactions or changes to business relationships resulting from the
announcement or completion of the Asset Purchase; and the risk that
Travere will not receive some or all of the potential sales-based
milestone payments under the purchase agreement. Travere also faces
the risk that its cash runway will not extend as far as anticipated
and that it will be unable to raise additional funding that may be
required to complete development of any or all of its product
candidates, including as a result of macroeconomic conditions; the
risk that the results from the Phase 3 DUPLEX Study of sparsentan
in FSGS will not serve as a basis for a regulatory submission for
approval of sparsentan for FSGS; the risk that the Phase 3 PROTECT
Study of sparsentan in IgAN will not demonstrate that sparsentan is
safe or effective or serve as the basis for further approval of
sparsentan; risks relating to Travere’s dependence on contractors
for clinical drug supply and commercial manufacturing;
uncertainties relating to patent protection and exclusivity periods
and intellectual property rights of third parties; risks associated
with regulatory interactions; risks and uncertainties relating to
competitive products, including current and potential future
generic competition with certain of Travere’s products, and
technological changes that may limit demand for Travere’s products.
Travere also faces additional risks associated with global and
macroeconomic conditions, including health epidemics and pandemics,
including risks related to potential disruptions to clinical
trials, commercialization activity, supply chain, and manufacturing
operations. You are cautioned not to place undue reliance on these
forward-looking statements as there are important factors that
could cause actual results to differ materially from those in
forward-looking statements, many of which are beyond our control.
Travere undertakes no obligation to publicly update any
forward-looking statement, whether as a result of new information,
future events, or otherwise. Investors are referred to the full
discussion of risks and uncertainties, including under the heading
“Risk Factors”, as included in Travere’s most recent Form 10-K,
Form 10-Q and other filings with the Securities and Exchange
Commission.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230905204912/en/
Mirum Pharmaceuticals,
Inc.
Media: Erin Murphy 510-508-6521 media@mirumpharma.com
Investors: Andrew McKibben ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
Travere Therapeutics
Media: Nivi Nehra Vice President, Corporate Communications
888-969-7879 mediarelations@travere.com
Investors: Naomi Eichenbaum Vice President, Investor Relations
888-969-7879 ir@travere.com
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