- NDA for maralixibat for cholestatic pruritus
in Alagille syndrome under priority review; PDUFA date is September
29, 2021 - Commercial preparations complete in anticipation of U.S.
launch of maralixibat in ALGS - Conference call to provide business
update today, August 5 at 1:30 p.m. PT/4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today reported
financial results for the quarter ended June 30, 2021 and provided
a business update.
“We are pleased with the progress we have made in the last
quarter as we prepare for the potential FDA approval of maralixibat
and advance our late-stage clinical development pipeline,” said
Chris Peetz, president and chief executive officer at Mirum.
“Alagille syndrome is a terrible disease, and we know patients and
physicians are waiting for treatment options. We are launch ready
in the United States and have amplified our reach to patients
globally with our expanded access program and recent partnerships
to accelerate potential approvals in a number of markets.”
Recent Key Operational Highlights
- Entered into an exclusive licensing agreement with GC Pharma to
develop and commercialize maralixibat for rare liver diseases in
South Korea.
- Presented maralixibat transplant-free survival data for
progressive familial intrahepatic cholestasis type 2 (PFIC2) and
long-term safety analyses for Alagille syndrome (ALGS) at the 6th
World Congress of Pediatric Gastroenterology, Hepatology and
Nutrition (WCPGHAN) Annual Meeting 2021.
- Appointed Carol L. Brosgart, M.D. as an independent director to
the Board of Directors.
- Presented an integrated safety analysis of maralixibat in
patients with ALGS and unveiled a multi-national survey of patient
reported outcomes from pregnant women with intrahepatic cholestasis
of pregnancy (ICP) at the EASL International Liver Congress
2021.
Financial Results
- Licensing revenue for the quarter ended June 30, 2021 was $11.0
million, which was associated with our license and collaboration
agreement with CANbridge Pharmaceuticals, Inc., compared to none
for the second quarter of 2020.
- Total operating expenses for the quarter ended June 30, 2021
were $48.4 million, compared to $23.6 million for the second
quarter of 2020.
- Research and development expenses for the quarter ended June
30, 2021 were $35.0 million, compared to $18.6 million for the
comparable prior-year period. The increase was primarily driven by
an upfront payment and funding associated with the Vivet gene
therapy programs and costs associated with the initiation of
volixibat clinical studies.
- General and administrative expenses for the second quarter of
2021 were $13.4 million, compared to $5.0 million for the
comparable prior-year period. The increase was primarily due to
increased commercialization and headcount costs.
- For the quarter ended June 30, 2021, Mirum reported a net loss
of $43.9 million, or $1.45 per share, compared with a net loss of
$23.3 million, or $0.93 per share for the same period in 2020.
- As of June 30, 2021, Mirum had cash, cash equivalents, and
short-term investments of $238.8 million.
Upcoming Anticipated Milestones
- Commercial and Regulatory
- The FDA has issued a PDUFA date of September 29, 2021, and, if
approved, Mirum will launch maralixibat in the U.S. representing a
commercial opportunity estimated at more than $500 million.
- Marketing authorization application (MAA) for PFIC2 decision
expected in early 2022.
- Mirum plans to submit an MAA for ALGS in Europe based on
results from the ICONIC study and six-year event-free survival
analyses.
- Pipeline and New Data
- Maralixibat
- Topline data from the Phase 3 MARCH PFIC study is expected in
the second quarter of 2022.
- Enrollment in the Phase 2b EMBARK study of maralixibat in
biliary atresia is ongoing; data expected in 2023.
- Volixibat
- Enrollment ongoing in the potentially registrational Phase 2b
OHANA study for ICP; interim analysis expected in 2022.
- Enrollment ongoing in the Phase 2b VISTAS study for primary
sclerosing cholangitis (PSC); interim analysis expected in
2022.
- A Phase 2b study in primary biliary cholangitis (PBC) is
planned for the second half of 2021.
Business Update Conference Call
Mirum will host a conference call today, August 5, 2021 at 1:30
p.m. PT/4:30 p.m. ET, to provide a review of the second quarter
activities and to discuss business updates. Join the call using the
following details:
Conference Call Details:
U.S. toll-free:
844.200.6205
International:
646.904.5544
Passcode:
097025
You may also access the call via webcast by visiting the
Investors section on Mirum’s corporate website. A replay of this
webcast will be available for 30 days.
About Maralixibat
Maralixibat is a novel, minimally absorbed, orally administered
investigational drug being evaluated in several rare cholestatic
liver diseases. Maralixibat inhibits the apical sodium dependent
bile acid transporter (ASBT), resulting in more bile acids being
excreted in the feces, leading to lower levels of bile acids
systemically, thereby potentially reducing bile acid mediated liver
damage and related effects and complications. More than 1,600
individuals have received maralixibat, including more than 120
children who have received maralixibat as an investigational
treatment for Alagille syndrome (ALGS) and progressive familial
intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS
clinical trial, patients taking maralixibat had significant
reductions in bile acids and pruritus compared to placebo, as well
as reduction in xanthomas and accelerated growth long-term. In a
Phase 2 PFIC study, a genetically defined subset of BSEP deficient
(PFIC2), patients responded to maralixibat with an increase in
transplant-free survival. The U.S. Food and Drug Administration has
granted maralixibat Breakthrough Therapy designation for the
treatment of pruritus associated with ALGS in patients one year of
age and older and for PFIC2. Maralixibat was generally
well-tolerated throughout the studies. The most frequent
treatment-related adverse events were diarrhea and abdominal pain.
Maralixibat has been studied extensively and its safety database
represents the largest database for an ASBT inhibitor.
Until maralixibat is approved and available for prescribing, the
medication is available to patients with ALGS through Mirum’s
expanded access program. For more information, please visit
ALGSEAP.com. For further information about maralixibat’s ongoing
studies in pediatric liver disease, please visit the study
websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study
for biliary atresia.
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 intrahepatic
cholestasis of pregnancy (OHANA study). Mirum plans to initiate a
Phase 2b study for primary biliary cholangitis later this year.
About Mirum Pharmaceuticals
Mirum Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company focused on the development and
commercialization of a late-stage pipeline of novel therapies for
debilitating liver diseases. Mirum’s lead product candidate,
maralixibat, is an investigational oral drug in development for
Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA
for maralixibat in the treatment of cholestatic pruritus in
patients with ALGS. The NDA has been accepted for priority review
by the FDA with a PDUFA action date of September 29, 2021.
Additionally, Mirum’s marketing authorization application for the
treatment of pediatric patients with PFIC2 has been accepted for
review (validated) by the European Medicines Agency. Mirum is also
developing volixibat, also an oral ASBT-inhibitor, in primary
sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and
primary biliary cholangitis. For more information, visit
MirumPharma.com.
To augment its pipeline in cholestatic liver disease, Mirum has
acquired the exclusive option to develop and commercialize two gene
therapy programs, VTX-803 and VTX-802 for PFIC3 and PFIC2,
respectively, from Vivet Therapeutics, following preclinical
evaluation and IND-enabling studies.
Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.
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, the potential regulatory approval of
maralixibat as well as the benefits and expected market opportunity
for maralixibat. 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,” “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 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
Six Months Ended
June 30,
June 30,
2021
2020
2021
2020
License revenue
$
11,000
$
-
$
11,000
$
-
Operating expenses: Research and development
35,048
18,555
73,182
35,895
General and administrative
13,353
5,042
22,832
9,734
Total operating expenses (1)
48,401
23,597
96,014
45,629
Loss from operations
(37,401
)
(23,597
)
(85,014
)
(45,629
)
Interest income
80
405
229
1,154
Interest expense
(4,776
)
-
(8,157
)
Change in fair value of derivative liability
(1,272
)
-
(938
)
Other expense, net
(514
)
(56
)
(530
)
(79
)
Net loss before provision for income taxes
(43,883
)
(23,248
)
(94,410
)
(44,554
)
Provision for income taxes
11
3
16
7
Net loss
$
(43,894
)
$
(23,251
)
$
(94,426
)
$
(44,561
)
Net loss per share, basic and diluted
$
(1.45
)
$
(0.93
)
$
(3.13
)
$
(1.79
)
Weighted-average shares of common stock outstanding, basic and
diluted
30,274,749
25,056,123
30,190,352
24,880,387
(1) Amounts include stock-based
compensation expense as follows:
Research and development
$
2,015
$
1,260
$
4,758
$
2,301
General and administrative
2,808
1,714
5,350
3,246
Total stock-based compensation
$
4,823
$
2,974
$
10,108
$
5,547
Mirum Pharmaceuticals,
Inc.
Selected Condensed
Consolidated Balance Sheet Data
(in thousands)
June 30,
December 31,
2021
2020
(Unaudited)
Cash, cash equivalents and short-term investments
$
238,841
$
231,820
Working capital
199,338
217,888
Total assets
247,668
240,864
Accumulated deficit
(267,597
)
(173,171
)
Total stockholders' equity
95,737
172,095
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210805005953/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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