Travere Therapeutics, Inc. (NASDAQ: TVTX) and Mirum
Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that they have
entered into a definitive agreement for the sale of Travere’s bile
acid product portfolio that includes Cholbam® (cholic acid) and
Chenodal® (chenodiol), two medications addressing rare diseases in
high-need settings.
Under the terms of the definitive agreement, Mirum will purchase
Travere’s bile acid product portfolio for up to $445 million,
consisting of $210 million upfront and $235 million in potential
sales-based milestone payments. Mirum will acquire 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).
“This agreement is an important step forward in Travere's
strategy to deliver our pipeline of innovative medicines to
patients living with rare diseases,” said Eric Dube Ph.D.,
president and chief executive officer of Travere Therapeutics. “The
sale of the bile acid portfolio will enable us to further focus our
efforts on the ongoing, and successful launch of FILSPARI™ for IgA
nephropathy, pursuing a potential regulatory path forward for
sparsentan in FSGS, and the development of pegtibatinase for the
treatment of classical homocystinuria, all of which we believe have
the potential to be future treatment standards in their respective
indications. This divestment will also strengthen our financial
foundation by meaningfully extending our cash runway and allow us
to maximize our growth potential. We look forward to working with
Mirum to ensure a seamless transition and continuing the commitment
to delivering these important medicines to patients in the rare
liver disease community.”
“The addition of the bile acid replacement therapies from
Travere will strengthen our pipeline and offer an opportunity to
leverage our unique expertise in the development and
commercialization of treatments in rare and underserved liver
diseases,” said Chris Peetz, president and chief executive officer
at Mirum. “This synergistic acquisition of the bile acid portfolio
along with the opportunity to sponsor the genetic testing program
will help to reinforce our leadership position in pediatric
hepatology. We look forward to building on the meaningful work
initiated by the talented Travere team and delivering on our
commitment to advancing research and bringing treatments to rare
liver disease patients in need.”
Transaction DetailsPer the terms of the
agreement, Mirum will acquire Travere’s rights to the bile acid
product portfolio consisting of Cholbam® and Chenodal®. Travere
will receive an upfront payment of $210 million and be eligible for
up to $235 million in sales-based milestone payments based on
annual net sales thresholds tiered from $125 to $500 million.
Travere has also agreed to provide certain transitional services.
The transaction is expected to close in the third quarter of 2023,
subject to regulatory clearance and customary closing
conditions.
For Travere, Lazard is acting as financial advisor and Cooley is
acting as legal advisor. For Mirum, Evercore is advising on the
acquisition and Gibson, Dunn & Crutcher is acting as legal
advisor.
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) IndicationCholbam 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 USEThe 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.
DRUG INTERACTIONS
- Inhibitors of Bile Acid Transporters: Avoid concomitant use of
inhibitors of the bile salt efflux pump (BSEP) such as
cyclosporine. Concomitant medications that inhibit canalicular
membrane bile acid transporters such as the BSEP may exacerbate
accumulation of conjugated bile salts in the liver and result in
clinical symptoms. If concomitant use is deemed necessary,
monitoring of serum transaminases and bilirubin is
recommended.
- Bile Acid Binding Resins: Bile acid binding resins such as
cholestyramine, colestipol, or colesevelam adsorb and reduce bile
acid absorption and may reduce the efficacy of CHOLBAM. Take
CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an
interval as possible) after a bile acid binding resin.
- Aluminum-based Antacids: Aluminum-based antacids have been
shown to adsorb bile acids in vitro and can reduce the
bioavailability of CHOLBAM. Take CHOLBAM at least 1 hour before or
4 to 6 hours (or at as great an interval as possible) after an
aluminum-based antacid.
PREGNANCY No studies in pregnant women or
animal reproduction studies have been conducted with CHOLBAM. Women
who become pregnant during CHOLBAM treatment are encouraged to call
1-844-202-6262.
LACTATION Endogenous cholic acid is present in
human milk. Clinical lactation studies have not been conducted to
assess the presence of CHOLBAM in human milk, the effects of
CHOLBAM on the breastfed infant, or the effects of CHOLBAM on milk
production. The developmental and health benefits of breastfeeding
should be considered along with the mother’s clinical need for
CHOLBAM and any potential adverse effects on the breastfed infant
from CHOLBAM or from the underlying maternal condition.
GERIATRIC USE It is not known if elderly
patients respond differently from younger patients.
HEPATIC IMPAIRMENT
- Discontinue treatment with CHOLBAM if liver function does not
improve within 3 months of the start of treatment.
- Discontinue treatment with CHOLBAM at any time if there are
clinical or laboratory indicators of worsening liver function or
cholestasis. Continue to monitor laboratory parameters of liver
function and consider restarting at a lower dose when the
parameters return to baseline.
OVERDOSAGE Concurrent elevations of serum GGT
and serum ALT may indicate CHOLBAM overdose. In the event of
overdose, the patient should be monitored and treated
symptomatically. Continue to monitor laboratory parameters of liver
function and consider restarting at a lower dose when the
parameters return to baseline.
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 labeled for
CTX, it received a medical necessity determination in the US by the
FDA and has been used as the standard of care for more than three
decades. Travere is working to obtain FDA approval of Chenodal for
the treatment of CTX and initiated a Phase 3 clinical trial for
this indication 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 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.
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, and
in Europe for the same indication in patients two months of age and
older.
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.
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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: expectations regarding the ability to
obtain regulatory and other approvals and satisfy other closing
conditions for the contemplated transaction, successfully close the
contemplated transaction and successfully transition the business
to the acquiror; the expected timing of the contemplated
transaction and related matters; expectations regarding future
sales of Chenodal and Cholbam and the outcome of continuing
studies, potential future approvals, and sales of such products,
and the related impact on the potential sales-based milestone
payments under the purchase agreement; expectations regarding
Travere’s other products and products in development, including
continued progress with the FILSPARI launch and a potential
regulatory path forward for sparsentan in FSGS; the timing and
achievement of additional development and regulatory milestones;
the advancement of pipeline products; expectations regarding and
results of Travere’s ongoing and future trials, studies and
analyses related to its various products including but not limited
to Chenodal, Cholbam, sparsentan and pegtibatinase; Travere’s cash
runway; and future growth prospects. 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 the
contemplated transaction will not close on the planned timeline or
at all due to a delay or failure to obtain regulatory approval or
for any other reason; the possible occurrence of any event, change
or other circumstance or condition that could give rise to the
termination of the purchase agreement for the proposed transaction;
the incurrence of significant transaction costs whether or not the
proposed transaction is consummated; the potential for litigation
relating to the proposed transaction; and the risk that disruptions
from the proposed transaction 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 proposed transaction; 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.
Contacts
Travere Therapeutics
Media:Nivi NehraVice President, Corporate
Communications888-969-7879mediarelations@travere.com
Investors: Naomi EichenbaumVice President, Investor
Relations888-969-7879ir@travere.com
Mirum Pharmaceuticals, Inc.
Media:Erin Murphy510-508-6521media@mirumpharma.com
Investors:Andrew McKibben ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
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