- Third quarter 2023 total revenue of $47.7
million - Completed acquisition of CHENODAL® and CHOLBAM® -
Announced positive Phase 3 RESTORE study results evaluating
CHENODAL (chenodiol) in patients with CTX; NDA filing expected in
H1 2024 - Conference call to provide business updates today,
November 2 at 1:30 p.m. PT/4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported
financial results for third quarter 2023 and provided a business
update.
“We made significant progress in the third quarter with record
LIVMARLI revenues in both the United States and Internationally,
the closing of the acquisition of CHENODAL and CHOLBAM and the
positive top line results from the unblinding of the RESTORE trial
of CHENODAL in CTX,” said Chris Peetz, president and chief
executive officer at Mirum. “The company is well positioned to
maintain this momentum with continued strong revenue growth and we
look forward to multiple exciting upcoming clinical and regulatory
catalysts.”
LIVMARLI®: Continued growth in Alagille
syndrome worldwide net sales and progressive familial intrahepatic
cholestasis (PFIC) near-term label expansion opportunity
- Third quarter 2023 global net sales of $38.7 million grew 106%
as compared to third quarter 2022.
- International commercial expansion ongoing with continued
strong demand.
- Presented 15 oral and poster presentations on clinical data and
real-world evidence validating LIVMARLI in patients with Alagille
syndrome and PFIC at the North American Society for Pediatric,
Gastroenterology, Hepatology and Nutrition (NASPGHAN) Annual
Meeting.
- Supplemental New Drug Application (sNDA) for LIVMARLI in PFIC
assigned Prescription Drug User Fee Act (PDUFA) date of March 13,
2024.
Completed acquisition of CHENODAL and CHOLBAM: establishes
Mirum’s leading growth franchise in pediatric hepatology
- Acquired two therapies approved in the U.S.: CHOLBAM (cholic
acid) for the treatment of bile acid synthesis disorders (BASD) and
Zellweger spectrum disorders (ZSD), and CHENODAL, an approved
medical necessity medicine for the treatment of cerebrotendinous
xanthomatosis (CTX).
- Announced positive Phase 3 data from the landmark RESTORE study
evaluating CHENODAL in patients with CTX. The Company expects to
submit a New Drug Application (NDA) to the U.S. Food and Drug
Administration (FDA) in the first half of 2024.
- Third quarter 2023 net sales of $9 million recorded since
acquisition close August 31st, 2023.
Pipeline: Three upcoming late-stage data
catalysts
- Phase 2b EMBARK study of LIVMARLI for pediatric patients with
biliary atresia topline data expected by year end.
- Phase 2b interim analyses for volixibat in PSC (VANTAGE study)
and in PBC (VISTAS study) expected in the first half of 2024.
Corporate and financial: Strong balance sheet and experienced
management team driving growth
- Total net revenue for the quarter ended September 30, 2023, was
$47.7 million compared to $18.8 million for the quarter ended
September 30, 2022. Net revenue for the nine months ended September
30, 2023, was $116.8 million compared to $49.2 million for the nine
months ended September 30, 2022.
- Total operating expenses were $72.9 million for the quarter
ended September 30, 2023, compared to $51.7 million for the quarter
ended September 30, 2022. Total operating expenses were $193.4
million for the nine months ended September 30, 2023 compared to
$146.2 million for the nine months ended September 30, 2022.
- As of September 30, 2023, Mirum had cash, cash equivalents, and
short-term investments of $306.0 million and convertible debt of
$316.3 million.
Business Update Conference Call
Mirum will host a conference call today, November 2, 2023, 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: 979561
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. LIVMARLI is also approved by the European
Commission for the treatment of cholestatic pruritus in patients
with ALGS two months and older. It is the only approved medication
to treat cholestatic pruritus associated with Alagille syndrome.
For more information for U.S. residents, please visit
LIVMARLI.com.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including 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.
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. 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. 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. We believe the prevalent patient
population in the United States with CTX exceeds 1,000.
About Mirum Pharmaceuticals
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 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 three investigational
treatments for debilitating liver diseases. The LIVMARLI
development program includes the Phase 2b EMBARK study for biliary
atresia. Mirum’s second investigational IBAT inhibitor is
volixibat, which 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.
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
LIVMARLI, CHENODAL and CHOLBAM including growth in year over year
net product sales, planned LIVMARLI launches in additional
international markets, label expansion into PFIC, CHENODAL
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” 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 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.
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,
2023
2022
2023
2022
Revenue: Product sales, net
$
47,725
$
18,780
$
109,320
$
47,156
License revenue
-
-
7,500
2,000
Total revenue
47,725
18,780
116,820
49,156
Operating expenses: Cost of sales (1)
10,228
2,932
22,019
7,880
Research and development
26,117
26,217
71,674
75,737
Selling, general and administrative
36,528
22,513
99,696
62,598
Total operating expenses (2)
72,873
51,662
193,389
146,215
Loss from operations
(25,148
)
(32,882
)
(76,569
)
(97,059
)
Other income (expense): Interest income
4,061
1,352
9,960
1,714
Interest expense
(3,574
)
(3,971
)
(11,542
)
(11,620
)
Change in fair value of derivative liability
-
-
-
232
Loss on extinguishment of RIPA
-
-
(49,076
)
-
Other income (expense), net
1,322
(192
)
237
953
Net loss before for income taxes
(23,339
)
(35,693
)
(126,990
)
(105,780
)
(Benefit) provision for income taxes
249
13
766
(6,546
)
Net loss
$
(23,588
)
$
(35,706
)
$
(127,756
)
$
(99,234
)
Net loss per share, basic and diluted
$
(0.57
)
$
(1.02
)
$
(3.28
)
$
(3.03
)
Weighted-average shares of common stock outstanding, basic
41,098,920
34,927,790
38,973,060
32,809,365
Weighted-average shares of common stock outstanding, diluted
41,098,920
34,927,790
38,973,060
32,825,314
(1) Amounts include intangible
amortization expense as follows:
Intangible amortization
$
2,582
$
1,040
$
5,099
$
1,815
(2) Amounts include stock-based
compensation expense as follows:
Research and development
$
2,626
$
2,517
$
8,013
$
7,519
Selling, general and administrative
5,759
4,391
17,290
12,592
Total stock-based compensation
$
8,385
$
6,908
$
25,303
$
20,111
Mirum Pharmaceuticals, Inc. Condensed Consolidated
Balance Sheet Data September 30, 2023 December
31, 2022 (unaudited)
Assets Current assets: Cash and
cash equivalents
$
303,059
$
28,003
Short-term investments
2,971
123,716
Accounts receivable
47,834
23,994
Inventory
22,250
5,565
Prepaid expenses and other current assets
12,465
8,947
Total current assets
388,579
190,225
Restricted cash and cash equivalents
-
100,000
Intangible assets, net
258,338
58,954
Other noncurrent assets
3,725
3,727
Total assets
$
650,642
$
352,906
Liabilities and Stockholders’ Equity Current liabilities:
Accounts payable
$
7,369
$
8,690
Accrued expenses
66,213
54,018
Other current liabilities
1,082
2,021
Total current liabilities
74,664
64,729
Revenue interest liability, net
-
140,351
Convertible notes, net
306,022
-
Other noncurrent liabilities
875
5,789
Total liabilities
381,561
210,869
Commitments and contingencies Stockholders' equity: Preferred stock
-
-
Common stock
5
4
Additional paid-in capital
791,114
535,074
Accumulated deficit
(520,580
)
(392,824
)
Accumulated other comprehensive loss
(1,458
)
(217
)
Total stockholders' equity
269,081
142,037
Total liabilities and stockholders' equity
$
650,642
$
352,906
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231102727938/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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