- $186-188 million in total revenue and
$178-180 million net product sales expected for 2023, preliminary
and unaudited
- LIVMARLI net products sales of $141-143
million expected for 2023, representing approximately 89%
year-over-year growth, preliminary and unaudited
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its
preliminary and unaudited estimates for full-year 2023 revenue and
net product sales, corporate updates, and full-year 2024
outlook.
“2023 was a transformative year for Mirum as we cemented our
position as a leader in rare disease and dramatically advanced our
operating and financial scale. We expanded our reach and impact
through strong global adoption of LIVMARLI and the addition of
CHOLBAM and CHENODAL,” said Chris Peetz, president and chief
executive officer. “In the year ahead, we are excited about the
growth prospects of all three commercial medicines, the potential
label expansion opportunities for LIVMARLI and CHENODAL and key
volixibat analyses in primary sclerosing cholangitis and primary
biliary cholangitis.”
2023 Highlights
- Established a leading pediatric hepatology franchise with three
commercial medicines
- 2023 estimated LIVMARLI net product sales of $141-143 million
representing approximately 89% growth over 2022 LIVMARLI net
product sales
- Total expected net product sales of $69-71 million in Q4 2023
including $41-43 million in LIVMARLI net sales and approximately
$28 million in net sales from CHOLBAM and CHENODAL
- Acquired two therapies commercially available in the U.S.:
CHOLBAM and CHENODAL
- Announced positive Phase 3 data from the RESTORE study
evaluating CHENODAL in cerebrotendinous xanthomatosis (CTX)
patients
- Expanded LIVMARLI’s US label in Alagille syndrome (ALGS) to
include infants 3 months of age and older
- Grew international business to 18 countries with reimbursed
access
- Company fully financed with strong balance sheet and financial
performance
- Announced the appointment of Eric Bjerkholt as Chief Financial
Officer
The foregoing amounts relating to 2023 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, 2023.
2024 Expectations and Milestones
- Expect continued revenue growth across all three commercial
medicines
- U.S. Food and Drug Administration (FDA) Prescription Drug User
Free Act (PDUFA) date for LIVMARLI in progressive familial
intrahepatic cholestasis (PFIC) is March 13, 2024.
- New Drug Application (NDA) submission for CHENODAL in CTX
planned in first half 2024
- Volixibat VISTAS study in primary sclerosing cholangitis
blinded interim analysis expected first half 2024
- Volixibat VANTAGE study for primary biliary cholangitis interim
analysis expected in first half 2024
Mirum will present at the 42nd annual J.P. Morgan Healthcare
Conference in San Francisco on Wednesday, January 10, 2024 at 10:30
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,
once-daily, ileal bile acid transporter (IBAT) inhibitor and the
only approved medication by the U.S. Food and Drug Administration
for the treatment of cholestatic pruritus in patients with Alagille
syndrome (ALGS) three months of age and older. LIVMARLI is also
approved by the European Commission for the treatment of
cholestatic pruritus in patients with ALGS two months and older.
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.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases. LIVMARLI has
received Breakthrough Therapy designation for ALGS and PFIC type 2
and orphan designation for ALGS and PFIC. To learn more about
ongoing clinical trials with LIVMARLI, please visit Mirum’s
clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause 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.
US Prescribing Information EU SmPC
About Volixibat
Volixibat is an oral, minimally absorbed agent designed to
selectively inhibit the apical sodium dependent bile acid
transporter (ASBT). Volixibat may offer a novel approach in the
treatment of adult cholestatic diseases by blocking the recycling
of bile acids, through inhibition of ASBT, thereby reducing bile
acids systemically and in the liver. Phase 1 and Phase 2 studies of
volixibat demonstrated on-target fecal bile acid excretion, a
pharmacodynamic marker of ASBT inhibition, in addition to decreases
in LDL cholesterol and increases in 7αC4 which are markers of bile
acid synthesis. Volixibat has been evaluated in more than 400
individuals across multiple clinical trials. The most common
adverse events reported were mild to moderate gastrointestinal
events observed in the volixibat groups.
Volixibat is currently being evaluated in Phase 2b studies for
primary sclerosing cholangitis (VISTAS study), and primary biliary
cholangitis (VANTAGE study).
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.
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) tablets
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.
About Mirum Pharmaceuticals, Inc.
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
cholestatic pruritus in patients with Alagille syndrome in the U.S.
(three months and older), in Europe (two months and older), and in
Canada. 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. CHOLBAM is FDA-approved for the treatment of bile acid
synthesis disorders due to single enzyme defects 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 two investigational
treatments for high-need rare diseases. Volixibat, also an IBAT
inhibitor, is being evaluated in two potentially registrational
studies including the Phase 2b VISTAS study for primary sclerosing
cholangitis and Phase 2b VANTAGE study for primary biliary
cholangitis. CHENODAL, has been evaluated in a Phase 3 clinical
study, RESTORE, to treat patients with CTX.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter.
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: continued commercial success and
growth prospects for LIVMARLI, CHENODAL and CHOLBAM, including
growth in LIVMARLI year-over-year net product sales, planned
LIVMARLI launches in additional international markets and label
expansion into PFIC, the timing and results of LIVMARLI’s PDUFA
date, CHENODAL’s potential full indication in CTX, becoming a
global leader in rare disease, the results, conduct and progress of
Mirum’s ongoing and planned studies for its product candidates and
the regulatory approval path for its product candidates globally.
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
“forward,” “planned,” “poised,” “positioned,” “potential”, “will,”
“prospects,” “opportunities” 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 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240108332335/en/
Investor Contacts: Andrew McKibben ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
Media Contact: Erin Murphy media@mirumpharma.com
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