ARCT-021 single-shot COVID-19 STARR™ mRNA
vaccine to be advanced to Phase 3 clinical development – on track
to initiate Phase 3 study in Q2
ARCT-021 single shot immunogenicity profile
compares favorably with new data generated from recipients of a
single dose of an approved conventional mRNA vaccine
ARCT-810 mRNA therapeutic for ornithine
transcarbamylase deficiency to be advanced to Phase 2 clinical
development – on track to file CTA for Phase 2 multiple dose study
in Q2
ARCT-032 mRNA therapeutic for cystic fibrosis:
completed successful pre-IND interaction with FDA – on track to
file CTA in Q4
Investor conference call at 4:30 p.m. ET
today
Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”,
Nasdaq: ARCT), a leading clinical-stage messenger RNA medicines
company focused on the development of infectious disease vaccines
and significant opportunities within liver and respiratory rare
diseases, today announced its financial results for the quarter and
full year ended December 31, 2020 and provided a corporate
update.
“Based on highly promising clinical data from our Phase 1/2
study and emerging mRNA vaccine immunological data, we are
advancing ARCT-021 for further development in Phase 3. We are
presently preparing to move forward a 5 µg single dose regimen, to
be confirmed based on pending Phase 2 data. Our self-amplifying
mRNA-based investigational vaccine may provide a differentiated
clinical profile and characteristics that support widespread
distribution across the globe. Our expectation is that successful
protection from COVID-19 will require repeated vaccination of
billions of individuals for years to come and that ARCT-021 will be
re-dosable. We believe that a re-dosable, more easily distributable
single shot mRNA vaccine would be a valuable option for many
countries,” said Joseph Payne, President and CEO of Arcturus.
“In addition to advancing our vaccine franchise, we have made
continued progress advancing our pipeline of promising liver and
lung mRNA therapeutic candidates. After successfully beginning
enrollment in the U.S. for our Phase 1b study for ARCT-810, a
therapeutic candidate for Ornithine Transcarbamylase (OTC)
Deficiency, we have now also received approval from Health Canada
to enroll subjects. We look forward to obtaining clinical data this
year,” concluded Mr. Payne.
Recent Corporate
Highlights
ARCT-021, Vaccine Candidate for SARS-CoV-2
1) Ongoing Phase 2 clinical study and planning for Phase 3
development:
- More than 500 participants dosed across USA and Singapore
- Phase 3 study initiation remains on track for Q2
- Targeting application for Emergency Use Authorization in one or
more jurisdictions in H2 2021
2) Encouraging new supportive data from Duke-NUS Medical
School submitted for publication:
- Dr. Eng Eong Ooi, Professor of Emerging Infectious Diseases at
Duke-NUS Medical School, and colleagues, examined the adaptive
immune responses of an approved conventional mRNA vaccine after a
single administration
- Data suggest that binding antibodies and cellular immunity are
associated with COVID-19 protection at early timepoints after the
first injection
- ARCT-021 single shot immunogenicity profile in Phase 1/2 study
compares favorably, providing additional support for the potential
efficacy of the ARCT-021 vaccine
Day
BNT162b2 (30 µg); N=20
ARCT-021 (5µg); N=22
IgG (≥ 4-fold rises)
10
80%
--
14
--
81%*
PRNT (% Detectable)
10
10%
--
28
--
59%
ELISpot (SFUs) Median
change from baseline
10
28
--
15
--
211
21
13
--
*Increases to 100% by Day 36 IgG = Immunoglobulin G binding
antibodies to full length spike protein PRNT = plaque reduction
neutralization test SFUs = Spot Forming Units per million
peripheral blood mononuclear cells
3) Completed Phase 1/2 clinical study and supporting
preclinical studies:
- Phase 1/2 study is complete and final data are under analysis;
plan to submit results for publication in Q2
- ARCT-021 demonstrated robust protection with single dose in a
primate challenge model and in a human ACE2 receptor transgenic
mouse challenge model
- ARCT-021 demonstrated robust protection with a single dose in
mice depleted of B cells, whereas depletion of T lymphocytes
yielded no protection following virus challenge, emphasizing the
importance of ARCT-021 induced cellular immunity
4) Manufacturing:
- Recently received $46.6M from Singapore EDB to support ARCT-021
stockpiling
- Manufacturing of lyophilized ARCT-021 to support Phase 3 and
initial commercial supply well on track
- Stability studies for lyophilized ARCT-021 at -20°C, 2-8°C and
room temperature storage conditions ongoing
5) Agreements:
- Strategic, government, and country supply agreement discussions
continue to progress
ARCT-810, Therapeutic Candidate for Ornithine
Transcarbamylase (OTC) Deficiency
- Received approval from Health Canada to enroll subjects into
Phase 1b study
- CTA filing for Phase 2 multiple dose study on track for Q2
ARCT-032, Therapeutic Candidate for Cystic Fibrosis
- Completed successful pre-IND interaction with FDA
- CTA filing on track for Q4
Acquisition of Exclusive License to mRNA Manufacturing
Technology from Alexion Pharmaceuticals
- The technology supports Arcturus’ highly efficient processes to
manufacture high purity mRNA vaccine and therapeutic candidates at
kilogram scale
- Extends the substantial intellectual property portfolio held by
Arcturus
Financial results for the quarter and full year ended
December 31, 2020
Revenues in conjunction with strategic alliances and
collaborations: Arcturus’ primary source of revenues is from
license fees and collaboration payments received from research and
development arrangements with our pharmaceutical and biotechnology
partners.
On a quarterly basis, total revenue for the three months ended
December 31, 2020 was $2.2 million and was relatively flat when
compared to the $2.3 million of the quarter ended September 30,
2020.
On a yearly basis, reported revenues of $9.5 million during the
year ended December 31, 2020, decreased from $20.8 million in the
year ended December 31, 2019. The decline in collaboration revenues
primarily relates to three factors: a $5.6 million decrease in
reimbursements from CureVac associated with the OTC collaboration
that ended in the third quarter of 2019, a decrease in one-time
license revenue of $3.3 million from Synthetic Genomics that
occurred in 2019, and lower activity with other collaboration
partners.
Operating expenses: On a quarterly basis, total operating
expenses for the three months ended December 31, 2020 were $33.3
million compared with $23.3 million for quarter ended September 30,
2020, and $13.8 million in same period of 2019. Approximately $8
million of the sequential increase in operating expenses during the
quarter ended December 31, 2020 was due to the ramp in the Covid-19
program related expenses, which included additional personnel,
manufacturing and clinical trial expenses. The current quarter
operating expenses were partially offset by $2.7 million in funds
awarded under the Singapore vaccine grants and by the Cystic
Fibrosis Foundation.
On a yearly basis, total operating expenses were $81.1 million
for the year-ended December 31, 2020 compared with $46.3 million
for the year ended December 31, 2019. The current year operating
expenses were partially offset by $15.2 million of funds earned
under the Singapore vaccine grants and funds awarded by the Cystic
Fibrosis Foundation. The increase in net expenditures for the year
ended December 31, 2020 as compared to the prior year was due
primarily to the increased activity in clinical and manufacturing
expenditures related to the Company’s Covid-19 and OTC programs as
well as increased personnel costs and other facility costs related
to the organizational growth of the Company.
Net loss: For the three months ended December 31, 2020
Arcturus reported a net loss of approximately $31.1 million, or
($1.25) per basic and diluted share, compared with a net loss in
the three months ended September 30, 2020 of $21.0 million, or
($0.92) per basic and diluted share, and three months ended
December 31, 2019 of $11.0 million, or ($0.76) per basic and
diluted share.
For the year ended December 31, 2020, net loss was approximately
$72.1 million, or ($3.55) per basic and diluted share, compared
with a net loss for the year ended 2019 of $26.0 million, or
($2.15) per basic and diluted share.
Cash and Cash Equivalents: The Company’s cash balance was
$463.0 million as of December 31, 2020, compared to cash and cash
equivalents of $301.1 million on September 30, 2020. The increase
in cash and cash equivalents compared to the prior year is
primarily due to the receipt of approximately $162 million in net
proceeds from our December 2020 public offering. Subsequent to the
end of the quarter, in January 2021 the Company received $46.6
million in funds under a manufacturing loan from Singapore EDB for
our Covid-19 vaccine program. Based on our current pipeline, the
Company’s cash position is expected to be sufficient to support
operations for more than two years.
Monday, March 1 @ 4:30 p.m. ET
Domestic:
877-407-0784
International:
201-689-8560
Conference ID:
13716298
Webcast:
http://public.viavid.com/index.php?id=143486
About Arcturus Therapeutics
Founded in 2013 and based in San Diego, California, Arcturus
Therapeutics Holdings Inc. (Nasdaq: ARCT) is a clinical-stage mRNA
medicines and vaccines company with enabling technologies: (i)
LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology and
(iii) mRNA drug substance along with drug product manufacturing
expertise. Arcturus’ diverse pipeline of RNA therapeutic and
vaccine candidates includes mRNA vaccine programs for SARS-CoV-2
(COVID-19) and Influenza, and other programs to potentially treat
Ornithine Transcarbamylase (OTC) Deficiency, and Cystic Fibrosis
along with partnered programs including Glycogen Storage Disease
Type 3, Hepatitis B Virus, and non-alcoholic steatohepatitis
(NASH). Arcturus’ versatile RNA therapeutics platforms can be
applied toward multiple types of nucleic acid medicines including
messenger RNA, small interfering RNA, replicon RNA, antisense RNA,
microRNA, DNA, and gene editing therapeutics. Arcturus’
technologies are covered by its extensive patent portfolio (209
patents and patent applications, issued in the U.S., Europe, Japan,
China and other countries). Arcturus’ commitment to the development
of novel RNA therapeutics has led to collaborations with Janssen
Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies
of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda
Pharmaceutical Company Limited, CureVac AG, Synthetic Genomics
Inc., Duke-NUS Medical School, and the Cystic Fibrosis Foundation.
For more information visit www.ArcturusRx.com. In addition, please
connect with us on Twitter and LinkedIn.
Forward Looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties for purposes of the
safe harbor provided by the Private Securities Litigation Reform
Act of 1995. Any statements, other than statements of historical
fact included in this press release, are forward-looking
statements, including those regarding strategy, future operations,
collaborations, the likelihood of success, and the efficacy or
safety, of the Company’s pipeline, including ARCT-021, ARCT-810 or
ARCT-032, the planned initiation, design or completion of clinical
trials, the likelihood that the Company will obtain clearance from
regulatory authorities to proceed with planned clinical trials, the
likelihood that preclinical or clinical data will be predictive of
future clinical results, the likelihood that clinical data will be
sufficient for regulatory approval or completed in time to submit
an application for regulatory approval within a particular
timeframe, the ability to enroll subjects in clinical trials, the
Company’s efforts to develop a vaccine against COVID-19 and
therapeutic potential thereof based on the Company’s mRNA
therapeutics, the ability of the Company to scale up manufacturing
of vaccine doses or to manufacture and scale up manufacturing of
any other product or substance, the likelihood that a patent will
issue from any patent application, the results of advancements in
the Company’s manufacturing methods and technologies, including
purification and lyophilization, its current cash position and
expected cash burn and the impact of general business and economic
conditions. Actual results and performance could differ materially
from those projected in any forward-looking statements as a result
of many factors including, without limitation, the ability to
enroll subjects in clinical trials as a result of the COVID-19
pandemic, the impact of commercialization of third-party COVID-19
vaccines on the design, and ability to conduct, clinical trials,
the availability of manufacturing capacity and raw materials,
unexpected clinical results, and general market conditions that may
prevent such achievements or performance. Arcturus may not actually
achieve the plans, carry out the intentions or meet the
expectations or projections disclosed in any forward-looking
statements such as the foregoing and you should not place undue
reliance on such forward-looking statements. Such statements are
based on management’s current expectations and involve risks and
uncertainties, including those discussed under the heading "Risk
Factors" in Arcturus’ Annual Report on Form 10-K for the fiscal
year ended December 31, 2020, and in subsequent filings with, or
submissions to, the SEC. Except as otherwise required by law,
Arcturus disclaims any intention or obligation to update or revise
any forward-looking statements, which speak only as of the date
they were made, whether as a result of new information, future
events or circumstances or otherwise.
ARCTURUS THERAPEUTICS HOLDINGS
INC. AND ITS SUBSIDIARIES
CONSOLIDATED BALANCE
SHEETS
(in thousands, except par
value information)
As of December 31,
2020
2019
Assets
Current assets:
Cash and cash equivalents
$
462,895
$
71,353
Accounts receivable
2,125
2,179
Prepaid expenses and other current
assets
2,769
758
Total current assets
467,789
74,290
Property and equipment, net
3,378
2,349
Operating lease right-of-use asset,
net
5,182
5,134
Equity-method investment
—
263
Non-current restricted cash
107
107
Total assets
$
476,456
$
82,143
Liabilities and stockholders’
equity
Current liabilities:
Accounts payable
$
10,774
$
5,793
Accrued liabilities
20,639
7,134
Deferred revenue
18,108
8,397
Total current liabilities
49,521
21,324
Deferred revenue, net of current
portion
12,512
15,182
Long-term debt, net of current portion
13,845
14,995
Operating lease liability, net of current
portion
4,025
4,850
Total liabilities
79,903
56,351
Stockholders’ equity:
Common stock: $0.001 par value; 60,000
shares authorized and 26,192 shares issued and outstanding at
December 31, 2020; 30,000 shares authorized and 15,138 shares
issued and outstanding at December 31, 2019
26
15
Additional paid-in capital
540,343
97,445
Accumulated deficit
(143,816
)
(71,668
)
Total stockholders’ equity
396,553
25,792
Total liabilities and stockholders’
equity
$
476,456
$
82,143
ARCTURUS THERAPEUTICS HOLDINGS
INC. AND ITS SUBSIDIARIES
CONSOLIDATED STATEMENTS OF
OPERATIONS AND COMPREHENSIVE LOSS
(in thousands, except per
share data)
Year Ended December
31,
2020
2019
2018
Collaboration revenue
$
9,539
$
20,789
$
15,753
Operating expenses:
Research and development, net
57,846
33,640
16,982
General and administrative
23,217
12,662
20,582
Total operating expenses
81,063
46,302
37,564
Loss from operations
(71,524
)
(25,513
)
(21,811
)
Loss from equity-method investment
(263
)
(32
)
(302
)
Finance (expense) income, net
(361
)
(446
)
328
Net loss
(72,148
)
(25,991
)
(21,785
)
Net loss per share, basic and diluted
$
(3.55
)
$
(2.15
)
$
(2.16
)
Weighted-average shares outstanding, basic
and diluted
20,305
12,069
10,069
Comprehensive loss:
Net loss
$
(72,148
)
$
(25,991
)
$
(21,785
)
Unrealized gain on short-term
investments
—
—
3
Comprehensive loss
$
(72,148
)
$
(25,991
)
$
(21,782
)
Fourth Quarter 2019
(unaudited)
First Quarter 2020
(unaudited)
Second Quarter 2020
(unaudited)
Third Quarter 2020
(unaudited)
Fourth Quarter 2020
(unaudited)
Collaboration revenue
$
2,968
$
2,646
$
2,322
$
2,333
$
2,238
Research and development expenses, net
11,994
7,917
7,944
17,699
24,286
General and administrative expenses
1,791
4,191
4,420
5,572
9,034
Loss from operations
(10,817
)
(9,462
)
(10,042
)
(20,938
)
(31,082
)
Net loss
(10,989
)
(9,777
)
(10,263
)
(21,004
)
(31,104
)
Net loss per share, basic and diluted
$
(0.76
)
$
(0.67
)
$
(0.55
)
$
(0.92
)
$
(1.25
)
Weighted average shares outstanding, basic
and diluted
14,505
14,521
18,794
22,938
24,886
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210301005926/en/
IR and Media Contacts Arcturus Therapeutics Neda
Safarzadeh (858) 900-2682 IR@ArcturusRx.com
Kendall Investor Relations Carlo Tanzi, Ph.D. (617) 914-0008
ctanzi@kendallir.com
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