- 2024 net product sales of approximately $336
million exceeds upper end of guidance range; preliminary and
unaudited estimate
- 2025 expected global net product sales of
$420 million to $435 million
- VISTAS study of volixibat in primary
sclerosing cholangitis expected to complete enrollment in second
half 2025; topline data expected 2026
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its
preliminary and unaudited estimates for full-year 2024 net product
sales, year-end cash balance, corporate updates, and full-year 2025
outlook.
“2024 marked a significant year for Mirum as we accelerated our
commercial business and achieved significant development
milestones,” said Chris Peetz, chief executive officer of Mirum.
“We continued our leadership in cholestatic disease with a label
expansion for LIVMARLI and positive interim analyses for volixibat
in PSC and PBC. Additionally, we advanced our rare genetic
neurology efforts with the NDA submission for chenodiol and the
in-licensing of MRM-3379 for Fragile X syndrome. With proven
commercial execution in ultra-rare disease and a compelling
pipeline in larger indications, we believe we are well-positioned
for sustained growth in the years ahead.”
Future Expectations and Milestones
- 2025 Guidance: expect continued revenue growth with global net
product sales of approximately $420 million to $435 million and
positive cash flow from operations
- Volixibat VISTAS study in primary sclerosing cholangitis (PSC)
expected to complete enrollment in second half 2025; topline data
expected in 2026
- Volixibat VANTAGE study in primary biliary cholangitis (PBC)
expected to complete enrollment in 2026
- LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic
conditions expected to complete enrollment in 2026
- FDA Prescription Drug User Fee Act (PDUFA) date for chenodiol
in cerebrotendinous xanthomatosis (CTX) is March 28, 2025
- Expect to initiate Phase 2 study for MRM-3379 in Fragile X
Syndrome (FXS) in 2025
2024 Highlights
Commercial: Continued leadership in rare disease with a
franchise in hepatology and genetic neurology
- 2024 estimated LIVMARLI net product sales of approximately $213
million and CHOLBAM and CHENODAL net product sales of approximately
$123 million
- Total estimated net product sales of approximately $99 million
in Q4 2024 including approximately $64 million in LIVMARLI net
sales and approximately $35 million in net sales from CHOLBAM and
CHENODAL
- Expanded global footprint; 30 countries with commercial access,
including successful reimbursement negotiation and launch in the
four major European markets
Regulatory and Pipeline: Expanding Mirum’s leadership across
multiple rare diseases and larger orphan settings
- Positive interim results for volixibat in VISTAS PSC and
VANTAGE PBC studies
- Volixibat granted breakthrough therapy designation for
treatment of cholestatic pruritus in PBC by the U.S. Food and Drug
Administration (FDA)
- Volixibat granted orphan drug designation for the treatment of
cholestatic pruritus in PBC by the FDA
- LIVMARLI approved by the FDA for cholestatic pruritus in
progressive familial intrahepatic cholestasis (PFIC) patients 12
months and older
- LIVMARLI approved in Europe for treatment of PFIC in patients
three months and older
- Initiated the LIVMARLI EXPAND Phase 3 study for pruritus in
rare cholestatic conditions
- New Drug Application (NDA) submitted for chenodiol in CTX
- In-licensed global rights to PDE4D inhibitor MRM-3379 for
FXS
Corporate and Financial: Sustained financial strength
- Achieved positive cash flow from operations in Q3 2024
- Cash, cash equivalents and investments of approximately $287
million as of December 31, 2024 compared to $286.3 million as of
December 31, 2023
- Announced the appointment of Joanne Quan, MD as Chief Medical
Officer
The foregoing amounts relating to 2024 financial data are
unaudited and preliminary and are subject to completion of
financial closing procedures. Additional information and disclosure
would be required for a more complete understanding of the
company’s financial position and results of operations as of
December 31, 2024.
Mirum will present at the 43rd annual J.P. Morgan Healthcare
Conference in San Francisco on Wednesday, January 15, 2025, at 9:45
a.m. PT. The presentation and question and answer session will be
webcast live and can be accessed by visiting the Investors and
Media section of Mirum’s corporate website. The replay of the
webcast will be available for 30 days.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered,
ileal bile acid transporter (IBAT) inhibitor approved by the U.S.
Food and Drug Administration for two pediatric cholestatic liver
diseases. It is approved for the treatment of cholestatic pruritus
in patients with Alagille syndrome (ALGS) in the U.S. three months
of age and older and in Europe for patients two months of age and
older. It is also approved in the U.S. for the treatment of
cholestatic pruritus in patients with progressive familial
intrahepatic cholestasis (PFIC) 12 months of age and older and in
Europe for the treatment of PFIC in patients three months of age
and older. For more information for U.S. residents, please visit
LIVMARLI.com.
LIVMARLI has received Breakthrough Therapy designation for ALGS
and PFIC type 2 and orphan designation for ALGS and PFIC. LIVMARLI
is currently being evaluated in the Phase 3 EXPAND study in
additional settings of cholestatic pruritus. 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 Volixibat
Volixibat is an oral, minimally absorbed agent designed to
selectively inhibit the ileal bile acid transporter (IBAT).
Volixibat may offer a novel approach in the treatment of adult
cholestatic diseases by blocking the recycling of bile acids,
through inhibition of IBAT, thereby reducing bile acids
systemically and in the liver. Volixibat is currently being
evaluated in Phase 2b studies for primary sclerosing cholangitis
(VISTAS study), and primary biliary cholangitis (VANTAGE study). In
June, Mirum announced positive interim results from the Phase 2b
VANTAGE study showing statistically significant improvement in
pruritus as well as meaningful reductions in serum bile acids and
improvements in fatigue for patients treated with volixibat. No new
safety signals were observed, and the most common adverse event was
diarrhea with all cases mild to moderate.
About CHOLBAM® (cholic acid) capsules
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 Cerebrotendinous Xanthomatosis
Cerebrotendinous xanthomatosis (CTX) is a rare, progressive and
underdiagnosed disorder of cholesterol metabolism affecting many
parts of the body. In people with CTX, the body is unable to break
down cholesterol properly causing toxins (e.g., cholestanol and
bile alcohols) to build up throughout the body over time. The
disorder is inherited in an autosomal recessive genetic manner.
Signs and symptoms of CTX include neonatal cholestasis (jaundice or
bile flow interruption), chronic diarrhea, the development of
bilateral cataracts before the age of 18, development of tendon
xanthomas (fatty deposits in the tendons) during teenage years or
later, and neurologic deterioration. The types, combinations and
severity of symptoms can be different from person to person making
diagnosis challenging and often delayed.
About chenodiol tablets
Chenodiol tablets is another name for chenodeoxycholic acid
(CDCA). CDCA is a naturally occurring bile acid that was originally
approved for the treatment of people with radiolucent stones in the
gallbladder. More recently, the US Food and Drug Administration
(FDA) granted chenodiol orphan drug designation for
cerebrotendinous xanthomatosis (CTX). CTX is a rare progressive
disorder that can affect the brain, spinal cord, tendons, eyes and
arteries. Chenodiol is not yet indicated for the treatment of CTX
but has received a medical necessity determination in the U.S. by
the FDA.
About Mirum Pharmaceuticals
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 12
months of age and older; in Europe, it is approved for patients
with PFIC three months of age and older. Mirum is also initiating
the Phase 3 EXPAND study, a label expansion opportunity for
LIVMARLI in additional settings of cholestatic pruritus. 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 investigational treatments
for several rare diseases. Volixibat, an IBAT inhibitor, is being
evaluated in two potentially registrational studies including the
Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and
Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat
has been granted Breakthrough Therapy Designation for the treatment
of cholestatic pruritus in patients with PBC. Chenodiol, has been
evaluated in a Phase 3 clinical study, RESTORE, to treat patients
with CTX, with positive topline results reported in 2023. Mirum has
submitted a new drug application with the FDA for the approval of
chenodiol to treat CTX in the U.S. Lastly, Mirum is planning for a
Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the
treatment of Fragile X syndrome, a rare genetic neurocognitive
disorder.
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, commercial results for our approved
products, including continued growth in year over year net product
sales, being on track to achieve our revised financial guidance,
our expected financial results as of December 31, 2024, including
our net product sales and cash, cash equivalents and investments,
delivering life changing medicines for patients suffering from rare
diseases, the results, enrollment, conduct and progress of Mirum’s
ongoing and planned studies for its product candidates, including
newly in-licensed product candidates, the timing and results of
interim analyses of our ongoing studies and the regulatory approval
path for its product candidates globally, including the anticipated
PDUFA date for chenodiol for CTX and additional international
launches expected in 2025. 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 “expected,” “will,” “could,” “would,” “guidance,”
“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 geopolitical and macroeconomic events, and the other
risks described in Mirum’s Quarterly Report on Form 10-Q for the
quarter ended September 30, 2024 and subsequent 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/20250113693031/en/
Investor Contacts: Andrew McKibben ir@mirumpharma.com
Media Contact: Erin Murphy media@mirumpharma.com
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