- U.S. commercial launch underway following
U.S. FDA approval of LIVMARLI™ (maralixibat) oral solution
- Six-year analysis showing significant
improvement in event-free survival with LIVMARLI compared to
natural history cohort (p<0.0001) presented as late-breaking,
Best of The Liver Meeting presentation at the American Association
for the Study of Liver Diseases (AASLD) The Liver Meeting® 2021
- The Lancet published data from the pivotal
LIVMARLI ICONIC study demonstrating significant and durable
responses in pruritus and other clinically meaningful improvements
in patients with ALGS
- Conference call to provide business updates
and discuss data presented at AASLD today, November 15 at 1:30 p.m.
PT/4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today reported
financial results for the quarter ended September 30, 2021, and
provided a business update.
“The FDA approval and launch of LIVMARLI capped a
transformational third quarter for Mirum and the Alagille syndrome
patient community. The launch of LIVMARLI, the first and only
approved medication for the treatment of cholestatic pruritus in
patients one year of age and older with Alagille syndrome, is off
to a strong start,” said Chris Peetz, president and chief executive
officer at Mirum. “We believe the launch of LIVMARLI, coupled with
a strong balance sheet and an advancing late-stage clinical
pipeline, positions Mirum for sustained growth as we lead the way
in rare liver disease worldwide.”
Recent Key Operational Highlights
- Received U.S. Food and Drug Administration (FDA) approval for
and launched LIVMARLI for the treatment of cholestatic pruritus in
patients with Alagille syndrome (ALGS) one year of age and
older.
- Presented LIVMARLI six-year event-free survival late breaker
data at AASLD, showing significant improvement in event-free and
transplant-free survival (p<0.0001).
- Presented clinical prognostic markers that are predictive of
event-free and transplant-free survival in patients treated with
LIVMARLI, furthering the understanding of clinical management of
ALGS.
- Published four-year safety and efficacy data from the ICONIC
pivotal study in The Lancet, highlighting that LIVMARLI provides
durable and clinically meaningful improvements across multiple
disease parameters in patients with ALGS.
- Submitted a Marketing Authorization Application (MAA) for
LIVMARLI for the treatment of cholestatic liver disease in patients
with ALGS to the European Medicines Agency (EMA).
- Entered into an exclusive licensing agreement to develop and
commercialize LIVMARLI for rare pediatric liver diseases in
Japan.
- Pamela Vig, Ph.D., chief scientific officer at Mirum, promoted
to Head of R&D.
- Appointed William C. Fairey, a global commercial and corporate
strategy leader in rare disease, as an independent director to the
Board of Directors.
- Initiated screening in VANTAGE Phase 2b study of volixibat in
primary biliary cholangitis.
Financial Results
- Licensing revenue for the quarter ended September 30, 2021 was
$5.0 million, which was associated with Mirum’s license and
collaboration agreement with GC Pharma, compared to none for the
third quarter of 2020.
- Total operating expenses for the quarter ended September 30,
2021 were $47.8 million, compared to $21.7 million for the third
quarter of 2020.
- Research and development expenses for the third quarter ended
September 30, 2021 were $30.5 million, compared to $16.0 million
for the comparable prior-year period. The increase was primarily
due to Vivet Collaboration Agreement program development funding,
increases related to volixibat clinical trial expenses for primary
sclerosing cholangitis (PSC), primary biliary cholangitis (PBC) and
intrahepatic cholestasis of pregnancy (ICP) and related
manufacturing activities supporting clinical supply, increases in
personnel and other compensation-related expenses and increases for
outside consulting services, regulatory fees and other general
development expenses.
- General and administrative (G&A) expenses for the third
quarter of 2021 were $17.4 million, compared to $5.7 million for
the comparable prior-year period. G&A investment increase in
the third quarter of 2021 versus the third quarter of 2020 was
primarily due to increased personnel and operational costs
associated with the launch of LIVMARLI, as well as expenses related
to general legal and public relation activities.
- For the quarter ended September 30, 2021, Mirum reported a net
loss of $47.1 million, or $1.55 per share, compared with a net loss
of $21.5 million, or $0.86 per share for the same period in
2020.
- As of September 30, 2021, Mirum had cash, cash equivalents, and
short-term investments of $205.0 million.
Upcoming Anticipated Milestones
- Pipeline
- LIVMARLI (maralixibat)
- Phase 3 MARCH-PFIC topline data expected in the second quarter
of 2022.
- Potential EMA approval of LIVMARLI for cholestatic liver
disease in patients with ALGS in second half of 2022.
- Phase 2b EMBARK study for biliary atresia enrolling; topline
data expected in 2023.
- Volixibat:
- Interim analyses expected in 2022 for the Phase 2b OHANA study
for ICP and Phase 2b VISTAS study for PSC, two potentially
registrational studies.
Business Update Conference Call
Mirum will host a conference call today, November 15, 2021 at
1:30 p.m. PT/4:30 p.m. ET, to provide business updates and discuss
data presented today at AASLD. Join the call using the following
details:
Conference Call Details:
U.S. toll-free:
844-200-6205
International:
646-904-5544
Passcode:
588077
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 one year of
age and older and is the only FDA-approved medication to treat
cholestatic pruritus associated with Alagille syndrome. For more
information, please visit LIVMARLI.com.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including
progressive familial intrahepatic cholestasis and biliary atresia.
LIVMARLI has received Breakthrough Therapy designation for ALGS and
PFIC type 2 and orphan designation for ALGS, PFIC and biliary
atresia. 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 serious 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.
Prescribing information
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 IBAT 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), intrahepatic
cholestasis of pregnancy (OHANA study) and primary biliary
cholangitis (VANTAGE study).
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 one year of
age and older.
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases affecting children and
adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter
(IBAT) inhibitor, is currently being evaluated in clinical trials
for pediatric liver diseases and includes the MARCH Phase 3 study
for progressive familial intrahepatic cholestasis (PFIC) and the
EMBARK Phase 2b study for patients with biliary atresia. In
addition, Mirum has an expanded access program open in Canada,
Australia, the UK and several countries in Europe for eligible
patients with Alagille syndrome.
Mirum has submitted a Marketing Authorization Application to the
European Medicines Agency for maralixibat for the treatment of
cholestatic liver disease in patients with Alagille syndrome.
Mirum’s second investigational treatment, volixibat, also an
oral IBAT inhibitor, is being evaluated in three registrational
studies including the OHANA Phase 2b study for pregnant women with
intrahepatic cholestasis of pregnancy, the VISTAS Phase 2b study
for adults with primary sclerosing cholangitis, and the VANTAGE
Phase 2b study for primary biliary cholangitis.
To augment its pipeline in cholestatic liver disease, Mirum has
acquired the exclusive option to develop and commercialize gene
therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2,
respectively, from Vivet Therapeutics SAS, following preclinical
evaluation and investigational new drug-enabling studies.
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Forward-Looking Statements
This press release includes forward-looking statements
pertaining to the Company’s planned participation at a scientific
conference, which may include discussion of the Company’s revenue,
business, and operations, including the discovery, development and
commercialization of our product candidates and technologies, and
the therapeutic potential thereof, the continuation of our clinical
trials, and the success of our collaborations with partners and any
potential future collaborations. Such forward-looking statements
are subject to risks and uncertainties that could cause actual
results to differ materially from those expressed or implied in
such statements. Applicable risks and uncertainties include those
relating to our preclinical research and clinical programs and
other risks identified under the heading “Risk Factors” included in
our most recent Form 10-Q and Form 10-K filings and in other future
filings with the SEC. The forward-looking statements contained in
this press release reflect Mirum's current views with respect to
future events, and Mirum does not undertake and specifically
disclaims any obligation to update any forward-looking
statements.
Mirum Pharmaceuticals, Inc. Condensed Consolidated
Statement of Operations Data (in thousands, except share and
per share amounts) (Unaudited)
Three Months Ended
Nine Months Ended
September 30,
September 30,
2021
2020
2021
2020
License revenue
$
5,000
$
-
$
16,000
$
-
Operating expenses: Research and development
30,471
15,984
103,653
51,879
General and administrative
17,353
5,732
40,185
15,466
Total operating expenses (1)
47,824
21,716
143,838
67,345
Loss from operations
(42,824
)
(21,716
)
(127,838
)
(67,345
)
Other income (expense): Interest income
72
237
301
1,391
Interest expense
(5,667
)
-
(13,824
)
-
Change in fair value of derivative liability
1,355
-
417
-
Other expense, net
(35
)
(30
)
(565
)
(109
)
Net loss before provision for income taxes
(47,099
)
(21,509
)
(141,509
)
(66,063
)
Provision for (benefit from) income taxes
9
(3
)
25
4
Net loss
$
(47,108
)
$
(21,506
)
$
(141,534
)
$
(66,067
)
Net loss per share, basic and diluted
$
(1.55
)
$
(0.86
)
$
(4.68
)
$
(2.65
)
Weighted-average shares of common stock outstanding, basic and
diluted
30,367,727
25,132,916
30,250,127
24,965,178
(1) Amounts include stock-based
compensation expense as follows:
Research and development
$
3,035
$
1,361
$
7,792
$
3,662
General and administrative
4,380
2,067
9,731
5,313
Total stock-based compensation
$
7,415
$
3,428
$
17,523
$
8,975
Mirum Pharmaceuticals, Inc. Selected
Condensed Consolidated Balance Sheet Data (in thousands)
September 30,
December 31,
2021
2020
(Unaudited) Cash, cash equivalents and short-term
investments
$
205,031
$
231,820
Working capital
146,418
217,888
Total assets
235,167
240,864
Accumulated deficit
(314,705
)
(173,171
)
Total stockholders' equity
56,396
172,095
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211115006202/en/
Investor Contacts: Ian Clements, Ph.D. ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
Media Contact: Erin Murphy media@mirumpharma.com
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