Expected cash runway extended to the end of
2026
$35 million milestone achieved under CSL
collaboration
ARCT-154 remains on track for Japan-NDA
approval in December
Enrollment target reached in Phase 3 bivalent
COVID vaccine comparison trial
Enrollment initiated in ARCT-032 Phase 1b
study; dosing first cystic fibrosis patient this month
ARCT-032 received Rare Pediatric Disease
Designation for cystic fibrosis from the FDA
Investor conference call at 4:30 p.m. ET
November 14, 2023
Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”,
Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines
company focused on the development of infectious disease vaccines
and opportunities within liver and respiratory rare diseases, today
announced its financial results for the third quarter ended Sept.
30, 2023, and provided corporate updates.
“We had considerable progress this quarter expanding our next
generation STARR® vaccine platform,” said Joseph Payne, President
& CEO of Arcturus Therapeutics. “Our monovalent ARCT-154 COVID
vaccine remains on track for approval in December and we reached
our target enrollment for the bivalent COVID vaccine Phase 3 study,
with PMDA-approval anticipated Q3 2024.”
Mr. Payne continued: “We have also advanced our mRNA therapeutic
pipeline with the scheduled dosing of our first participant in our
Phase 1b study with ARCT-032, an inhaled mRNA therapeutic candidate
for individuals with cystic fibrosis. This study will advance our
understanding of the safety and tolerability of ARCT-032 in
patients. It advances our effort to provide benefit to the CF
population with the largest unmet need, including those who are not
candidates for any of the currently approved CFTR modulators.”
“We are happy to announce our expected cash runway was extended
to the end of 2026,” announced Andrew Sassine, Chief Financial
Officer. “A combination of lower expenses, additional development
milestones and accelerated timelines for manufacturing technology
transfer to CSL have contributed to the extended runway.
Additionally, substantial funding was obtained by ARCALIS, our
joint venture mRNA manufacturing partner, from the Japanese
Government with up to $165 million committed to date. We expect
this facility to become a leading manufacturer of mRNA-based
vaccines and therapeutics, with the ability to manufacture vaccines
within 100 days of an emerging viral strain.”
Recent Corporate Highlights
- Updated preliminary Phase 3 COVID-19 booster data were
presented at the 11th International mRNA Health Conference in
Berlin. In comparison to an FDA-approved monovalent mRNA vaccine,
monovalent ARCT-154 showed multi-fold improvement in durability and
multi-fold superior titers of neutralizing antibodies against
Omicron BA.4/5 at 6 months post-boost. The Phase 3 booster
durability data were consistent with the Phase 1/2 booster clinical
trial durability data collected previously which were presented at
the 9th ESWI Influenza Conference in Valencia.
- ARCALIS Inc., the Company’s manufacturing joint venture in
Japan to support the production of mRNA vaccines and therapeutics,
continues to make operational progress while also obtaining
financial support from the Japanese government. ARCALIS has
completed the construction of a state-of-the-art mRNA drug
substance manufacturing facility and based on additional funds from
the Japanese government announced in August 2023, construction of
an associated DNA template manufacturing facility is underway. In
total to date, up to $165 million has been awarded to ARCALIS by
the Japanese government to build mRNA Drug Substance and mRNA-based
Drug Product manufacturing capabilities, and to construct a DNA
template manufacturing facility.
- The LUNAR-FLU (ARCT-2138) program continues to progress with
funding and operational support from CSL. LUNAR-FLU utilizes
Arcturus’ validated next-generation STARR® mRNA platform.
Initiation of a Phase 1 clinical trial (N = 132) is expected to
begin soon.
- Arcturus achieved a milestone for $35 million and anticipates
receipt from CSL in November 2023. The milestone payment will be
used to fund development activities for the LUNAR-COV19 vaccine
program under its collaboration with CSL.
- The anticipated global commercial launch schedule for the
Company’s validated, next generation STARR® mRNA COVID vaccine
platform has recently been presented by CSL with anticipated
marketing authorization approvals expected to occur between
2024-2026 in key markets such as Japan, EU, United Kingdom, and the
U.S.
- The initial enrollment target of 850 participants has been
reached in a Phase 3 bivalent COVID vaccine trial designed to
compare immunogenicity to bivalent Comirnaty®. The enrollment
process will be completed in November 2023.
- The Company remains committed to the development of ARCT-810,
an mRNA therapeutic candidate for ornithine transcarbamylase (OTC)
deficiency.
- ARCT-810 Phase 1b single ascending dose study in the U.S. has
completed enrollment and dosing of all cohorts (N = 16 patients).
Arcturus expects the final database lock to occur in the fourth
quarter of 2023.
- ARCT-810 Phase 2 study in UK and Europe will enroll up to 24
adolescents and adults with OTC deficiency. The ongoing study is
evaluating two dose levels and includes up to six (6) bi-weekly
administrations for each participant.
- Updated guidance of interim Phase 2 data in H1 2024 and taking
various actions to address the continued challenging enrollment
rate in Europe, by adding study sites and patient services.
- The Company is advancing ARCT-032, an inhaled mRNA therapeutic
candidate for cystic fibrosis formulated with Arcturus’ LUNAR®
delivery technology.
- Completed dosing in a Phase 1 study in New Zealand of 32
healthy subjects across four (4) ascending single-dose
cohorts.
- Phase 1b enrollment initiated October 2023, with dosing of
first participant scheduled this month. The study is designed to
enroll up to 8 adults with cystic fibrosis, with each participant
receiving two administrations of ARCT-032.
- In September 2023, the CF Foundation agreed to increase its
financial commitment to $25 million to advance ARCT-032.
- In October 2023, ARCT-032 received Rare Pediatric Disease
Designation from the FDA. As such, if ARCT-032 achieves FDA
approval for a pediatric indication, Arcturus is eligible to
receive a priority review voucher of a subsequent marketing
application for a different product.
- New proof of activity in vivo (G551D CF Ferret model) data
presented at the North American Cystic Fibrosis Conference (NACFC)
in November. The ferrets in the study require continuous treatment
with the CFTR modulator Kalydeco® to prevent disease progression. A
single administration of ARCT-032 showed successful transfection of
airway epithelial cells and restoration of mucociliary clearance
above the level maintained with Kalydeco.
Financial Results for the Three and Nine Months Ended
September 30, 2023
Revenues in conjunction with strategic alliances and
collaborations:
Arcturus’ primary sources of revenues were from license fees,
consulting and related technology transfer fees, reservation fees
and collaborative payments received from research and development
arrangements with pharmaceutical and biotechnology partners. For
the three months ended September 30, 2023, we reported revenue of
$45.1 million compared with $13.4 million for the three months
September 30, 2022. Revenue increased by $31.7 million during the
three months ended September 30, 2023, as compared to the prior
year period. The increase was primarily attributable to revenue
recognized from the collaboration agreement with CSL Seqirus and
grant revenue recognized from the agreement with BARDA. Revenue
increased by $90.3 million during the nine months ended September
30, 2023, as compared to the nine months ended September 30, 2022.
The increase was attributable to an increase in revenue of $133.0
million primarily related to the collaboration agreement with CSL
Seqirus. This increase was primarily offset by less revenues in
2023 from other COVID program customers.
Operating expenses:
Total operating expenses for the three months ended September
30, 2023, were $64.5 million compared with $50.2 million for the
three months ended September 30, 2022. Total operating expenses for
the nine months ended September 30, 2023, were $195.9 million
compared with $155.0 million for the nine months ended September
30, 2022.
Research and development expenses:
Our research and development expenses consist primarily of
external manufacturing costs, in-vivo research studies and clinical
trials performed by contract research organizations, clinical and
regulatory consultants, personnel related expenses, facility
related expenses and laboratory supplies related to conducting
research and development activities. Research and development
expenses were $51.1 million for the three months ended September
30, 2023, compared with $37.7 million in the comparable period last
year, primarily reflecting increased clinical research and
manufacturing costs of $11.6 million and an increase of $2.0
million in personnel related expenses. Research and development
expenses were $155.5 million for the nine months ended September
30, 2023, compared with $120.8 million in the comparable period
last year, primarily reflecting increased manufacturing costs of
$27.8 million.
General and Administrative Expenses:
General and administrative expenses primarily consist of
salaries and related benefits for our executive, administrative,
legal and accounting functions and professional service fees for
legal and accounting services as well as other general and
administrative expenses. General and administrative expenses were
$13.4 million and $40.4 million for the three and nine months ended
September 30, 2023, respectively, compared with $12.5 million and
$34.2 million in the comparable periods last year. The increases
resulted primarily from personnel expenses due to increased
headcount and salaries, increased travel, and consulting expenses
as well as increased rent expense associated with the new
facility.
Net Loss:
For the three months ended September 30, 2023, Arcturus reported
a net loss of approximately $16.2 million, or ($0.61) per diluted
share, compared with a net loss of $35.3 million, or ($1.33) per
diluted share in the three months ended September 30, 2022. For the
nine months ended September 30, 2023, Arcturus reported a net loss
of approximately $18.0 million, or ($0.68) per diluted share,
compared with a net loss of $108.0 million, or ($4.09) per diluted
share in the nine months ended September 30, 2022.
Cash Position and Balance Sheet:
Cash, cash equivalents and restricted cash were $369.1 million
as of September 30, 2023, and $394.0 million on December 31, 2022.
We have achieved a total of approximately $365.0 million in upfront
payments and milestones from CSL Seqirus as of September 30, 2023.
We expect to continue to receive future milestone payments from CSL
Seqirus that will support the ongoing development of the covid, flu
and three other respiratory vaccine programs. The expected cash
runway extends to the end of 2026 based on the current pipeline and
programs.
Earnings Call: Tuesday, November 14, 2023 @ 4:30 pm
ET
- Domestic: 1-877-407-0784
- International: 1-201-689-8560
- Conference ID: 13740896
- Webcast: Link
About Arcturus Therapeutics
Founded in 2013 and based in San Diego, California, Arcturus
Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage
clinical mRNA medicines and vaccines company with enabling
technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA
Technology (sa-mRNA) and (iii) mRNA drug substance along with drug
product manufacturing expertise. The Company has an ongoing global
collaboration for innovative mRNA vaccines with CSL Seqirus, and a
joint venture in Japan, ARCALIS, focused on the manufacture of mRNA
vaccines and therapeutics. Arcturus’ pipeline includes RNA
therapeutic candidates to potentially treat ornithine
transcarbamylase deficiency and cystic fibrosis, along with its
partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and
influenza. Arcturus’ versatile RNA therapeutics platforms can be
applied toward multiple types of nucleic acid medicines including
messenger RNA, small interfering RNA, circular RNA, antisense RNA,
self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’
technologies are covered by its extensive patent portfolio (patents
and patent applications issued in the U.S., Europe, Japan, China,
and other countries). 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,
the likelihood of success of the Company’s pipeline (including
ARCT-032 and ARCT-810) and partnered programs (including the
COVID-19 and flu programs partnered with CSL Seqirus), the
likelihood that ARCALIS will receive all, or any portion of, the
funding under awards from the Japanese government, the continued
progress of ARCALIS and expectations for ARCALIS’s facility, the
anticipated conduct, including continued enrollment, of the
ARCT-032 study, the results of the ARCT-032 study, the likelihood
of the CF program to provide benefit to the CF population, the
continued progress of the LUNAR-FLU program, the likelihood and
timing of regulatory approvals of any products including ARCT-154
in Japan or anywhere else, the likelihood that preclinical or
clinical data will be predictive of future clinical results, the
anticipated receipt of $35 million from CSL for a milestone,
likelihood of a commercial launch for the COVID vaccine platform
and schedule therefor, the timing for completion of the Phase 3
bivalent COVID vaccine trial, the Company’s continued commitment to
ARCT-810, the timing of the final database lock for the ARCT-810
Phase 1b study, the completion of enrollment of, and timing for
interim data from, the ARCT-810 Phase 2 study, the Company’s
continued advancement of ARCT-032, the enrollment of the Phase 1b
clinical study of ARCT-032, the likelihood that ARCT-032 will be
approved or provide Arcturus with a priority review voucher, the
likelihood of achieving future milestones under the CSL Seqirus
collaboration, the anticipated expenses of the Company and the
expected timelines for a manufacturing technology transfer to CSL,
the likelihood or timing of collection of accounts receivables
including expected payments from CSL Seqirus, its current cash
position and expected cash burn and runway, and the impact of
general business and economic conditions. 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. These statements are
only current predictions or expectations, and are subject to known
and unknown risks, uncertainties, and other factors that may cause
our or our industry’s actual results, levels of activity,
performance or achievements to be materially different from those
anticipated by the forward-looking statements, including those
discussed under the heading "Risk Factors" in Arcturus’ most recent
Annual Report on Form 10-K, and in subsequent filings with, or
submissions to, the SEC, which are available on the SEC’s website
at www.sec.gov. 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.
Trademark Acknowledgements
The Arcturus logo and other trademarks of Arcturus appearing in
this announcement, including LUNAR® and STARR®, are the property of
Arcturus. All other trademarks, services marks, and trade names in
this announcement are the property of their respective owners.
ARCTURUS THERAPEUTICS HOLDINGS
INC. AND ITS SUBSIDIARIES
CONDENSED CONSOLIDATED BALANCE
SHEETS
September 30, 2023
December 31, 2022
(in thousands, except par value
information)
(unaudited)
Assets
Current assets:
Cash and cash equivalents
$
311,918
$
391,883
Restricted cash
35,000
—
Accounts receivable
38,220
2,764
Prepaid expenses and other current
assets
8,130
8,686
Total current assets
393,268
403,333
Property and equipment, net
12,715
12,415
Operating lease right-of-use asset,
net
29,534
32,545
Non-current restricted cash
22,133
2,094
Total assets
$
457,650
$
450,387
Liabilities and stockholders’
equity
Current liabilities:
Accounts payable
$
18,362
$
7,449
Accrued liabilities
28,553
30,232
Current portion of long-term debt
—
60,655
Deferred revenue
40,768
28,648
Total current liabilities
87,683
126,984
Deferred revenue, net of current
portion
41,911
20,071
Long-term debt
20,000
—
Operating lease liability, net of current
portion
27,018
30,216
Other non-current liabilities
976
2,804
Total liabilities
177,588
180,075
Stockholders’ equity
Common stock, $0.001 par value; 60,000
shares authorized; issued and outstanding shares were 26,723 at
September 30, 2023 and 26,555 at December 31, 2022
27
27
Additional paid-in capital
636,194
608,426
Accumulated deficit
(356,159
)
(338,141
)
Total stockholders’ equity
280,062
270,312
Total liabilities and stockholders’
equity
$
457,650
$
450,387
ARCTURUS THERAPEUTICS HOLDINGS
INC. AND ITS SUBSIDIARIES
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
Three Months Ended
Nine Months Ended
September 30,
September 30,
(in thousands, except per share data)
2023
2022
2023
2022
Revenue:
Collaboration revenue
$
43,376
$
13,369
$
132,670
$
45,706
Grant revenue
1,764
—
3,274
—
Total revenue
45,140
13,369
135,944
45,706
Operating expenses:
Research and development, net
51,077
37,688
155,513
120,770
General and administrative
13,377
12,488
40,364
34,211
Total operating expenses
64,454
50,176
195,877
154,981
Loss from operations
(19,314
)
(36,807
)
(59,933
)
(109,275
)
Loss from equity-method investment
—
—
—
(515
)
Gain (loss) from foreign currency
4
1,862
(175
)
3,237
Gain on debt extinguishment
—
—
33,953
—
Finance income (expense), net
3,981
(321
)
9,710
(1,445
)
Net loss before income taxes
(15,329
)
(35,266
)
(16,445
)
(107,998
)
Provision for income taxes
893
—
1,573
—
Net loss
$
(16,222
)
$
(35,266
)
$
(18,018
)
$
(107,998
)
Net loss per share, basic and diluted
$
(0.61
)
$
(1.33
)
$
(0.68
)
$
(4.09
)
Weighted-average shares outstanding, basic
and diluted
26,574
26,467
26,559
26,423
Comprehensive loss:
Net loss
$
(16,222
)
$
(35,266
)
$
(18,018
)
$
(107,998
)
Comprehensive loss
$
(16,222
)
$
(35,266
)
$
(18,018
)
$
(107,998
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231114821639/en/
IR and Media Contacts Arcturus Therapeutics Neda
Safarzadeh VP, Head of IR/PR/Marketing (858) 900-2682
IR@ArcturusRx.com
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