- Second quarter global net product sales of $77.8 million, on
track to achieve full-year guidance of $310 to $320 million
- Positive interim results for volixibat in VISTAS PSC and
VANTAGE PBC studies
- LIVMARLI for treatment of PFIC granted marketing authorization
in Europe
- Initiating LIVMARLI EXPAND study for pruritus in rare
cholestatic conditions
- Conference call to provide business updates today, August 7 at
1:30 p.m. PT/4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported
financial results for second quarter 2024 and provided a business
update.
“The second quarter was marked by significant achievements
across both our commercial medicines and development pipeline as we
continued to execute on our strategic priorities,” said Chris
Peetz, chief executive officer of Mirum. “The positive interim
results of volixibat in the VISTAS PSC and VANTAGE PBC studies are
exciting steps towards potential new treatment options for patients
suffering from these rare liver diseases. We also achieved key
regulatory milestones expanding access to LIVMARLI for patients
with PFIC beginning with an approval in Europe for patients three
months and older and a label expansion in the US to include
patients 12 months and older, and we filed a new drug application
for chenodiol in CTX. Finally, we are exploring LIVMARLI’s
potential to treat cholestatic pruritus in other indications with
the initiation of the EXPAND study.”
Commercial: Strong growth across all three approved
medicines
- Second quarter 2024 global net product sales of $77.8 million
grew 139% compared to the second quarter 2023.
- LIVMARLI second quarter net sales were $47.2 million,
representing 45% growth compared to the second quarter 2023.
- Second quarter 2024 CHOLBAM and CHENODAL net sales were $30.5
million.
Regulatory and Pipeline: Achieved significant milestones and
expanding pipeline
- Positive Interim results for volixibat in the VISTAS PSC and
VANTAGE PBC studies.
- LIVMARLI approved in Europe for PFIC in patients three months
and older.
- LIVMARLI approved in the US for cholestatic pruritus in PFIC in
patients 12 months and older.
- Initiating the Phase 3 EXPAND study, a label expansion
opportunity for LIVMARLI in additional settings of cholestatic
pruritus in the second half of the year.
- New Drug Application (NDA) submitted for chenodiol in CTX.
Corporate and Financial: Strong balance sheet
- As of June 30, 2024, Mirum had cash, cash equivalents and
investments of $295.4 million compared to $286.3 million as of
December 31, 2023. Uses of cash in the second quarter 2024 included
a $10 million milestone payment due to Takeda for the FDA approval
of LIVMARLI for PFIC.
- Total operating expenses were $102.1 million for the quarter
ended June 30, 2024, compared to $61.8 million for the quarter
ended June 30, 2023. Total operating expense included $17.7 million
of non-cash stock-based compensation and depreciation and
amortization expense for the quarter ended June 30, 2024, compared
to $9.7 million for the quarter ended June 30, 2023.
Business Update Conference Call
Mirum will host a conference call today, August 7, 2024, at 1:30
p.m. PT/4:30 p.m. ET, to 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: 771307
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,
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. 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. 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 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, 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
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. 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 2 VISTAS study for primary sclerosing
cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary
cholangitis. Lastly, 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.
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, continued commercial success for our
approved products, including continued growth in year over year net
product sales, being on track to achieve our financial guidance,
delivering life changing medicines for patients suffering from rare
diseases, 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 “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
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. A further description of risks and uncertainties can be found
in Mirum’s Annual Report on Form 10-K for the fiscal year ended
December 31, 2023 and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors,” as well
as in its subsequent reports on Form 8-K, all of which are filed
with the U.S. Securities and Exchange Commission and available at
www.sec.gov.
Mirum Pharmaceuticals,
Inc.
Condensed Consolidated
Statement of Operations Data
(in thousands, except share
and per share amounts)
(Unaudited)
Three Months Ended June
30,
Six Months Ended June
30,
2024
2023
2024
2023
Revenue:
Product sales, net
$
77,760
$
32,497
$
146,677
$
61,595
License and other revenue
115
5,000
420
7,500
Total revenue
77,875
37,497
147,097
69,095
Operating expenses:
Cost of sales (1)
20,227
6,812
38,057
11,791
Research and development
32,672
22,009
64,894
45,557
Selling, general and administrative
49,208
32,949
94,846
63,168
Total operating expenses (2)
102,107
61,770
197,797
120,516
Loss from operations
(24,232
)
(24,273
)
(50,700
)
(51,421
)
Other income (expense):
Interest income
3,486
3,627
7,119
5,899
Interest expense
(3,569
)
(3,726
)
(7,146
)
(7,968
)
Loss from termination of revenue interest
purchase agreement
—
(49,076
)
—
(49,076
)
Other income (expense), net
312
(274
)
2,069
(1,085
)
Net loss before provision for income
taxes
(24,003
)
(73,722
)
(48,658
)
(103,651
)
Provision for income taxes
635
316
1,259
517
Net loss
(24,638
)
(74,038
)
(49,917
)
(104,168
)
Net loss per share, basic and diluted
$
(0.52
)
$
(1.94
)
$
(1.06
)
$
(2.75
)
Weighted-average shares of common stock
outstanding, basic and diluted
47,235,080
38,107,334
47,081,315
37,892,513
(1) Amounts include intangible
amortization expense as follows:
Intangible amortization
$
5,593
$
1,258
$
10,995
$
2,517
(2) Amounts include stock-based
compensation expense as follows:
Cost of sales
$
318
$
—
$
318
$
—
Research and development
3,546
2,672
7,407
5,387
Selling, general and administrative
7,971
5,685
15,560
11,531
Total stock-based compensation
$
11,835
$
8,357
$
23,285
$
16,918
Mirum Pharmaceuticals,
Inc.
Condensed Consolidated Balance
Sheet Data
(Unaudited)
June 30, 2024
December 31, 2023
Assets
Current assets:
Cash and cash equivalents
$
233,245
$
286,326
Short-term investments
45,126
—
Accounts receivable
60,430
67,968
Inventory
20,438
22,312
Prepaid expenses and other current
assets
8,590
10,935
Total current assets
367,829
387,541
Restricted cash
250
—
Long-term investments
17,075
—
Intangible assets, net
261,768
252,925
Other noncurrent assets
13,832
6,155
Total assets
$
660,754
$
646,621
Liabilities and Stockholders’
Equity
Current liabilities:
Accounts payable
$
9,842
$
7,416
Accrued expenses
101,394
78,544
Operating lease liabilities, current
1,072
1,104
Total current liabilities
112,308
87,064
Operating lease liabilities,
noncurrent
8,244
617
Convertible notes payable, net
307,242
306,421
Other liabilities
3,972
3,849
Total liabilities
431,766
397,951
Commitments and contingencies
Stockholders’ equity:
Preferred stock
—
—
Common stock
5
5
Additional paid-in capital
835,616
803,260
Accumulated deficit
(606,156
)
(556,239
)
Accumulated other comprehensive (loss)
income
(477
)
1,644
Total stockholders’ equity
228,988
248,670
Total liabilities and stockholders’
equity
$
660,754
$
646,621
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240807306996/en/
Investor Contact: Andrew McKibben ir@mirumpharma.com Media
Contact: Erin Murphy media@mirumpharma.com
Mirum Pharmaceuticals (NASDAQ:MIRM)
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