- Net product sales totaled $178.9 million, and
full year 2023 revenue totaled $186.4 million, delivering 142%
growth over 2022 total revenue
- 2024 expected global net product sales of
$310 million to $320 million
- LIVMARLI PFIC PDUFA date on track for March
13, 2024
- Volixibat VISTAS and VANTAGE interim analyses
expected in first half of 2024
- Conference call to provide business updates
today, February 28 at 1:30 p.m. PT/4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported
financial results for the fourth quarter and year-end 2023 and
provided a business update.
“2023 was a landmark year for Mirum as we significantly expanded
our commercial footprint, realizing $179 million in net product
sales, and achieved another year of strong global LIVMARLI growth.
We also successfully integrated CHOLBAM and CHENODAL into our
portfolio, which strengthened our position as a leader in rare
disease,” said Chris Peetz, chief executive officer at Mirum. “In
2024, we look forward to further expanding the reach of our
approved medicines, potential LIVMARLI and CHENODAL label
expansions and interim analyses for our volixibat adult
indications. Operationally and financially, we are well positioned
for this next phase of growth and remain committed to our mission
of delivering life changing medicines to patients in need.”
Commercial: Leading Pediatric Hepatology Franchise
- Net product sales totaled $178.9 million, and full year 2023
revenue totaled $186.4 million, representing 142% growth over 2022
total revenue.
- 2023 LIVMARLI net product sales totaled $141.8 million,
representing approximately 89% growth over 2022 net product
sales.
- International business grew to 18 countries with reimbursed
access to LIVMARLI.
- Expect continued revenue growth across all approved medicines
in 2024 with expected global net product sales of $310 million to
$320 million.
Regulatory & Pipeline: Multiple Upcoming 2024
Milestones
- 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 in first half 2024.
- Volixibat VANTAGE study in primary biliary cholangitis interim
analysis expected in first half 2024.
Corporate and Financial: Strong Balance Sheet, Financially
Sustaining Scale
- Total net product sales for the full year ended December 31,
2023, was $178.9 million compared to $75.1 million for the full
year ended December 31, 2022.
- Total operating expenses were $293.0 million for the full year
ended December 31, 2023, compared to $208.3 million for the full
year ended December 31, 2022.
- The increase in year-over-year operating expense was due to a
$32.2 million increase in cost of goods sold and a $56.8 million
increase in sales, general and administrative expense, partially
offset by a $4.2 million decrease in research and development
expense.
- As of December 31, 2023, Mirum had cash and cash equivalents of
$286.3 million.
- Announced the appointment of Joanne Quan, M.D. as Chief Medical
Officer.
The foregoing financial information is unaudited and subject to
change, and actual results may vary from the foregoing.
Business Update Conference Call
Mirum will host a conference call today, February 28, 2024, at
1:30 p.m. PT/4:30 p.m. ET, to discuss its financial results,
guidance, and provide business updates. Join the call using the
following details:
Conference Call Details:
U.S./Toll-Free: +1 833 470 1428 International: +1 404 975 4839
Passcode: 089049
You may also access the call via webcast by visiting the Events
& Presentations section on Mirum’s website. A replay of this
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 approved
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. for
the treatment of cholestatic pruritus in patients with PFIC three
months of age and older, and in Europe for the treatment of PFIC in
patients two months of age and older.
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
is 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 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. 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. 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) 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. A Phase 3
clinical trial for this indication has recently been completed and
based on the positive results, a New Drug Application (NDA)
submission for CHENODAL in CTX planned in first half 2024. We
believe the prevalent patient population in the United States with
CTX exceeds 1,000.
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
other regions globally. 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 three
months of age and older. 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 two investigational
treatments for debilitating liver diseases. Volixibat, 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. Lastly, CHENODAL, has been evaluated in a Phase 3
clinical study, RESTORE, to treat patients with CTX, with positive
topline results reported in 2023.
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 for our
approved products, including growth in year over year net product
sales, having sufficient cash and cash equivalent reserves, planned
expansion into additional international markets, LIVMARLI label
expansion into PFIC, the timing and results of LIVMARLI’s PDUFA,
CHENODAL potential full indication in CTX, delivering life changes
medicines and becoming a global leader in rare disease, the
results, conduct and progress of Mirum’s ongoing and planned
studies for its product candidates, the timing and results of
interim analyses of our ongoing studies 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 “expected,” “forecasts,” “forward,”
“planned,” “poised,”, “positioned” “potential”, “will” 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, 2023 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.
Mirum Pharmaceuticals, Inc. Condensed Consolidated
Statement of Operations Data (in thousands, except share and
per share amounts)
(Unaudited)
Three Months Ended Year ended December
31, December 31,
2023
2022
2023
2022
Revenue: Product sales, net
$
69,554
$
27,906
$
178,874
$
75,062
License revenue
-
-
7,500
2,000
Total revenue
69,554
27,906
186,374
77,062
Operating expenses: Cost of sales (1)
22,510
4,494
44,529
12,374
Research and development
30,935
31,105
102,609
106,842
Selling, general and administrative
46,184
26,468
145,880
89,066
Total operating expenses (2)
99,629
62,067
293,018
208,282
Loss from operations
(30,075
)
(34,161
)
(106,644
)
(131,220
)
Other income (expense): Interest income
3,775
2,143
13,735
3,857
Interest expense
(3,563
)
(4,359
)
(15,105
)
(15,979
)
Change in fair value of derivative liability
-
674
-
906
Loss from termination of revenue interest purchase agreement
-
-
(49,076
)
-
Other (expense) income, net
(3,061
)
(588
)
(2,824
)
365
Net loss before for income taxes
(32,924
)
(36,291
)
(159,914
)
(142,071
)
Provision for (benefit from) income taxes
225
140
991
(6,406
)
Net loss
$
(33,149
)
$
(36,431
)
$
(160,905
)
$
(135,665
)
Net loss per share: Basic
$
(3.94
)
$
(4.01
)
Diluted
$
(3.94
)
$
(4.02
)
Weighted-average shares of common stock outstanding: Basic
40,885,124
33,839,072
Diluted
40,885,124
33,982,493
(1) Amounts include intangible
amortization expense as follows:
Intangible amortization
$
5,305
$
1,078
$
10,404
$
2,893
(2) Amounts include stock-based
compensation expense as follows:
Research and development
$
2,879
$
2,531
$
10,892
$
10,050
Selling, general and administrative
6,841
4,365
24,131
16,957
Total stock-based compensation
$
9,720
$
6,896
$
35,023
$
27,007
Mirum Pharmaceuticals, Inc. Condensed Consolidated
Balance Sheet Data (Unaudited) December 31,
2023 December 31, 2022 Assets Current
assets: Cash and cash equivalents
$
286,326
$
28,003
Short-term investments
-
123,716
Accounts receivable
67,968
23,994
Inventory
19,590
5,565
Prepaid expenses and other current assets
10,935
8,947
Total current assets
384,819
190,225
Restricted cash and cash equivalents
-
100,000
Intangible assets, net
252,925
58,954
Other noncurrent assets
6,155
3,727
Total assets
$
643,899
$
352,906
Liabilities and Stockholders’ Equity Current liabilities:
Accounts payable
$
7,416
$
8,690
Accrued expenses
77,161
54,018
Other current liabilities
1,104
2,021
Total current liabilities
85,681
64,729
Revenue interest liability, net
-
140,351
Convertible notes, net
306,421
-
Other noncurrent liabilities
617
5,789
Total liabilities
392,719
210,869
Commitments and contingencies Stockholders' equity: Preferred stock
-
-
Common stock
5
4
Additional paid-in capital
803,260
535,074
Accumulated deficit
(553,729
)
(392,824
)
Accumulated other comprehensive income (loss)
1,644
(217
)
Total stockholders' equity
251,180
142,037
Total liabilities and stockholders' equity
$
643,899
$
352,906
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240228211669/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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