- Net product sales for LIVMARLI® (maralixibat)
oral solution totaled $17.5 million in the second quarter of 2022
and $28.4 million year-to-date
- Conference call to provide business updates
today, August 4 at 1:30 p.m. PT/4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported
financial results for the second quarter ended June 30, 2022 and
provided a business update.
“It is inspiring to see LIVMARLI’s impact on the burden of
disease in Alagille syndrome and we continue to see growing demand
with $17.5 million in net product sales in the second quarter,”
said Chris Peetz, president and chief executive officer of Mirum.
“We expect a strong finish to 2022 with topline MARCH PFIC Phase 3
data and anticipated EMA approval in Alagille syndrome by the end
of the year.”
Key Operational Highlights
- Acquired Satiogen Pharmaceuticals, previously a licensing
partner and now a wholly-owned subsidiary, reducing Mirum’s royalty
and milestone obligations.
- Presented key data at recent medical conferences, including:
- A six-year natural history comparison showing a 70% improvement
(p-value <0.0001) in event-free and transplant-free survival
with LIVMARLI and an analysis of predictors of six-year event-free
survival.
- New analysis on four-year growth improvement and real-world
experience with LIVMARLI in patients with Alagille syndrome.
- Data from the largest multinational survey highlighting the
impact of pruritus on quality of life for patients with primary
sclerosing cholangitis.
- Appointed Saira Ramasastry, managing partner of Life Sciences
Advisory, LLC, as a member of the Company’s board of directors and
audit committee.
Upcoming Anticipated Milestones
- LIVMARLI (maralixibat)
- Potential approval of MAA for the treatment of cholestasis in
patients with Alagille syndrome in the fourth quarter of 2022.
- Topline data evaluating more than 90 patients in the MARCH PFIC
Phase 3 clinical trial in the fourth quarter of 2022.
- Topline data in the EMBARK Phase 2b clinical trial for biliary
atresia in the second half of 2023.
- Volixibat
- Interim analysis for the VISTAS Phase 2b clinical trial for
primary sclerosing cholangitis in mid-2023.
- Interim analysis for the OHANA Phase 2b clinical trial for
intrahepatic cholestasis of pregnancy in the first half of
2023.
- Interim analysis for the VANTAGE Phase 2b clinical trial for
primary biliary cholangitis in the second half of 2023.
Financial Results
- Net product sales revenue of LIVMARLI for the quarter ended
June 30, 2022 was $17.5 million, compared to none for the second
quarter of 2021.
- Total operating expenses for the quarter ended June 30, 2022
were $48.9 million, compared to $48.4 million for the second
quarter of 2021.
- Research and development expenses for the second quarter ended
June 30, 2022 were $25.4 million, compared to $35.0 million for the
comparable prior-year period. The decrease was primarily due to a
decrease in license fees and collaboration funding for the
terminated Vivet collaboration agreement and a development
milestone for the EMBARK Phase 2b clinical trial for biliary
atresia, offset by increased clinical trial expenses and employee
headcount.
- Selling, general and administrative (SG&A) expenses for the
second quarter of 2022 were $21.0 million, compared to $13.4
million for the comparable prior-year period. SG&A investment
increased in the second quarter of 2022 versus the second quarter
of 2021, primarily due to personnel and consulting expenses as the
company increased in size to support commercial activities for
LIVMARLI, marketing and patient support expenses associated with
commercial activities for LIVMARLI, and an increase in
administrative costs.
- For the quarter ended June 30, 2022, Mirum reported a net loss
of $26.9 million, or $0.84 net loss per share, compared with a net
loss of $43.9 million, or $1.45 net loss per share, for the same
period in 2021.
- As of June 30, 2022, Mirum had cash, cash equivalents,
restricted cash equivalents and investments of $225.0 million.
Business Update Conference Call
Mirum will host a conference call today, August 4, 2022 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 (888) 510-2553
International:
1 (646) 960-0473
Passcode:
9354200
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) 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 (PFIC) 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 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 Phase 2b clinical trial),
intrahepatic cholestasis of pregnancy (OHANA Phase 2b clinical
trial), and primary biliary cholangitis (VANTAGE Phase 2b clinical
trial).
About Mirum Pharmaceuticals
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. 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 clinical
trial for progressive familial intrahepatic cholestasis (PFIC) and
the EMBARK Phase 2b clinical trial for patients with biliary
atresia. In addition, Mirum has an expanded access program open
across multiple countries for eligible patients with ALGS and
PFIC.
Mirum has submitted a Marketing Authorization Application to the
European Medicines Agency for LIVMARLI for the treatment of
cholestasis in patients with Alagille syndrome.
Mirum’s second investigational treatment, volixibat, an oral
IBAT inhibitor, is being evaluated in three potentially
registrational studies including the VISTAS Phase 2b clinical trial
for adults with primary sclerosing cholangitis, the OHANA Phase 2b
clinical trial for pregnant women with intrahepatic cholestasis of
pregnancy, and the VANTAGE Phase 2b clinical trial for adults with
primary biliary cholangitis.
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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, meeting the disclosed milestones,
anticipated commercial approvals, success of and treatment demand
for LIVMARLI, timely receipt of data that support continued
investment in the ongoing and planned clinical trials for Mirum’s
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 “continued,” “later,” “will,” “anticipate,” “expect,”
“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 the COVID-19 pandemic and macroeconomic events, such as
the ongoing instability in financial markets or the military
conflict between Ukraine and Russia and related sanctions, and the
other risks described more fully 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.
Mirum Pharmaceuticals, Inc. Condensed Consolidated
Statement of Operations Data (in thousands, except share and
per share amounts) (Unaudited)
Three Months Ended
Six Months Ended
June 30,
June 30,
2022
2021
2022
2021
Revenue: Product sales, net
$
17,484
$
-
$
28,376
$
-
License revenue
-
11,000
2,000
11,000
Total revenue
17,484
11,000
30,376
11,000
Operating expenses: Cost of sales
2,524
-
4,948
-
Research and development
25,432
35,048
49,520
73,182
Selling, general and administrative
20,969
13,353
40,085
22,832
Total operating expenses (1)
48,925
48,401
94,553
96,014
Loss from operations
(31,441
)
(37,401
)
(64,177
)
(85,014
)
Other income (expense): Interest income
293
80
362
229
Interest expense
(3,875
)
(4,776
)
(7,649
)
(8,157
)
Change in fair value of derivative liability
232
(1,272
)
232
(938
)
Other income (expense), net
1,299
(514
)
1,145
(530
)
Net loss before for income taxes
(33,492
)
(43,883
)
(70,087
)
(94,410
)
(Benefit) provision for income taxes
(6,570
)
11
(6,559
)
16
Net loss
$
(26,922
)
$
(43,894
)
$
(63,528
)
$
(94,426
)
Net loss per share, basic and diluted
$
(0.84
)
$
(1.45
)
$
(2.00
)
$
(3.13
)
Weighted-average shares of common stock outstanding, basic
32,164,174
30,274,749
31,732,596
30,190,352
Weighted-average shares of common stock outstanding, diluted
32,179,171
30,274,749
31,740,136
30,190,352
(1
)
Amounts include stock-based compensation expense as follows:
Research and development
$
2,416
$
2,015
$
5,001
$
4,758
Selling, general and administrative
4,225
2,808
8,201
5,350
Total stock-based compensation
$
6,641
$
4,823
$
13,202
$
10,108
Mirum Pharmaceuticals, Inc. Selected Condensed
Consolidated Balance Sheet Data (in thousands)
June 30,
December 31,
2022
2021
(Unaudited) Cash, cash equivalents, restricted cash
equivalents and investments
$
224,953
$
261,524
Working capital
102,071
123,996
Total assets
297,233
294,651
Accumulated deficit
(320,687
)
(257,159
)
Total stockholders' equity
110,687
120,212
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version on businesswire.com: https://www.businesswire.com/news/home/20220804005911/en/
Investor Contact: Sam Martin Argot Partners
ir@mirumpharma.com
Media Contact: Erin Murphy media@mirumpharma.com
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