ARCT-154 Phase 3 COVID-19 booster trial
achieved primary endpoint demonstrating strong immune response and
favorable safety profile
Meiji Seika Pharma submitted ARCT-154 New Drug
Application in Japan
New ARCT-154 booster clinical data demonstrate
one-year durability across a panel of variants
Received FDA Fast Track Designation and Rare
Pediatric Disease Designation for ARCT-810 for OTC deficiency
Received regulatory approval of ARCT-032 to
proceed into a Phase 1b clinical study in CF patients
Investor conference call at 4:30 p.m. ET
today
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 second quarter and six
months ended June 30, 2023 and provided corporate updates.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20230807532754/en/
Geometric mean fold rise (GMFR) of
neutralizing antibodies against SARS-CoV-2 variants (versus
pre-booster levels) after ARCT-154 booster vaccination measured by
validated pseudoviral microneutralization assays (N=12). Error bars
represent the 95% CI. GMFR values are included in white.
Participants who received any COVID-19 vaccines or had
laboratory-confirmed SARS-CoV-2 infection during the follow-up
period were excluded from immunogenicity analysis at sampling times
after the event. (Graphic: Arcturus Therapeutics)
“Arcturus has continued to achieve significant operational
progress alongside our exclusive global vaccines partner CSL
Seqirus, highlighted by the NDA submission of ARCT-154, for the
COVID-19 primary series vaccine and booster, in Japan by CSL
Seqirus’ partner Meiji Seika Pharma,” said Joseph Payne, President
& CEO of Arcturus Therapeutics. “As demonstrated by the
recently published ARCT-154 Phase 3 head-to-head booster data
versus Comirnaty®, we believe that our next generation STARR® mRNA
platform is meaningfully differentiated. In addition to ARCT-154,
we have also made significant progress with other clinical and
pre-clinical mRNA therapeutic programs, and we look forward to
sharing clinical data from our ARCT-810 and ARCT-032 programs later
this year.”
Recent Corporate Highlights
- The primary endpoint was achieved in the ARCT-154 Phase 3
booster vaccine study, demonstrating non-inferiority of immune
response against SARS-CoV-2 ancestral strain compared to Comirnaty.
Superiority of ARCT-154 in neutralizing antibody response against
SARS-CoV-2 Omicron BA.4/5 variant was also demonstrated as a key
secondary endpoint.
- The study compared immune responses between ARCT-154 and
Comirnaty booster doses (original strain) in Japanese adults (N =
828 and randomized 1:1 to ARCT-154 and Comirnaty) that were
previously immunized with two doses of mRNA COVID-19 vaccine then a
third booster dose of Comirnaty at least 3 months prior to
enrollment.
- ARCT-154, a self-amplifying mRNA vaccine, was administered at 5
mcg, a significantly lower dose relative to Comirnaty (30
mcg).
- Initial study results have been published in MedRxiv. The data
suggests increased immunogenicity associated with ARCT-154 at Day
29 with 1.43-fold higher geometric mean titers of neutralizing
antibodies against the vaccine strain versus Comirnaty.
- At the time of interim data cut, ARCT-154 was considered safe
and tolerable with no safety concerns identified.
- Meiji Seika Pharma and the Japanese government provided funding
for the ARCT-154 Phase 3 booster study.
- Meiji Seika Pharma is responsible for obtaining regulatory
approval, distribution, sales and marketing of ARCT-154 in
Japan.
- Meiji Seika Pharma announced a collaboration with ARCALIS Co.,
Ltd. to establish integrated cGMP mRNA vaccine manufacturing
capabilities, from drug substance to drug product, in Japan.
ARCALIS recently announced that construction has been completed for
their state-of-the-art manufacturing facility in Japan.
- A Phase 1/2 clinical trial demonstrated one-year durability of
immune response following ARCT-154 booster vaccine administration
using validated microneutralization (MNT) assays. The geometric
mean fold rise (GMFR) in neutralizing antibodies remained greater
than 10-fold above baseline for one year across a panel of variants
(ref: Figure), including Omicron BA.1.
- The LUNAR-FLU program continues to progress with funding and
operational support from CSL Seqirus. LUNAR-FLU utilizes Arcturus’
validated next generation STARR® mRNA platform.
- In June, the Company announced that the U.S. Food and Drug
Administration (FDA) had granted Fast Track Designation to
ARCT-810, the Company’s mRNA therapeutic candidate for ornithine
transcarbamylase (OTC) deficiency. The Company has also recently
received Rare Pediatric Disease Designation from the FDA for
ARCT-810, which is designed to recognize serious or
life-threatening manifestations primarily affecting patients under
18 years of age. Due to such designation, if ARCT-810 achieves
approval for a pediatric indication in the original rare pediatric
disease product application, Arcturus will receive a voucher for
priority review of a subsequent marketing application for a
different product.
- ARCT-810 Phase 1b single ascending dose study in the U.S. has
completed enrollment and dosing of all cohorts (N = 16
patients).
- 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. The Company expects to share
interim data on biological activity from a subset of patients in
the coming months.
- ARCT-032, the Company’s inhaled mRNA therapeutic for cystic
fibrosis, has completed dosing in a Phase 1 study in New Zealand,
including 32 subjects across four (4) ascending single-dose
cohorts. The Company received regulatory approval of a protocol
amendment to allow the transition to a Phase 1b clinical study of
ARCT-032 in up to 8 adult cystic fibrosis patients.
Financial Results for the Three and Six Months Ended June 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 June 30, 2023, we reported revenue of $10.5
million compared with $27.1 million for the three months ended June
30, 2022. Revenue decreased by $16.6 million during the three
months ended June 30, 2023 as compared to the prior year period.
The decrease was primarily attributable to a decrease in revenue of
$12.7 million related to the termination of the agreement with
Vinbiocare and a decrease in revenue of $12.5 million related to
the agreement with the Israeli Ministry of Health. The decrease was
primarily offset by an increase in revenue of $8.6 million related
to the collaboration agreement with CSL Seqirus and the grant
agreement with BARDA which were both executed in the second half of
2022. Revenue increased by $58.5 million during the six months
ended June 30, 2023 as compared to the six months ended June 30,
2022. The increase was attributable to an increase in revenue of
$87.6 million primarily related to the collaboration agreement with
CSL Seqirus and the grant agreement with BARDA which were both
executed in the second half of 2022.
Operating expenses:
Total operating expenses for the three months ended June 30,
2023 were $65.9 million compared with $49.2 million for the three
months ended June 30, 2022. Total operating expenses for the six
months ended June 30, 2023 were $131.4 million compared with $104.8
million for the six months ended June 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 $52.7 million for the three months ended June 30,
2023, compared with $38.2 million in the comparable period last
year, primarily reflecting increased clinical research and
manufacturing costs of $11.4 million and an increase of $2.9
million in personnel related costs. Research and development
expenses were $104.4 million for the six months ended June 30,
2023, compared with $83.1 million in the comparable period last
year, primarily reflecting increased manufacturing costs of $11.1
million, an increase of $5.9 million in personnel related costs, an
increase in consulting expenses of $1.8 million, an increase of
$1.3 million in facilities expense and a decrease of contra
research and development expenses recognized of $2.7 million. The
increase was primarily offset by a decrease of clinical-related
expenses of $1.2 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
$13.2 million and $27.0 million for the three and six months ended
June 30, 2023, respectively, compared with $11.0 million and $21.7
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 June 30, 2023, Arcturus reported a
net loss of approximately $52.6 million, or ($1.98) per diluted
share, compared with a net loss of $21.6 million, or ($0.82) per
diluted share in the three months ended June 30, 2022. For the six
months ended June 30, 2023, Arcturus reported a net loss of
approximately $1.8 million, or ($0.07) per diluted share, compared
with a net loss of $72.7 million, or ($2.75) per diluted share in
the six months ended June 30, 2022.
Cash Position and Balance Sheet:
Cash, cash equivalents and restricted cash were $380.6 million
as of June 30, 2023 and $394.0 million on December 31, 2022. We
have collected approximately $300.0 million in upfront payments and
milestones from CSL Seqirus as of June 30, 2023. Additionally, we
received $23.6 million under the manufacturing and supply of
ARCT-154 from CSL Seqirus during the quarter ended June 30, 2023.
We expect to continue to receive future milestone payments from CSL
Seqirus that will support the ongoing development of the covid and
flu programs. The cash runway remains extended through the
beginning of 2026 based on the current pipeline and programs.
Earnings Call: Monday, August 7, 2023 @ 4:30 pm ET
- Domestic: 1-877-407-0784
- International: 1-201-689-8560
- Conference ID: 13738872
- 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 (samRNA) and (iii) mRNA drug substance along with drug
product manufacturing expertise. 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 mRNA, 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
potential of the Company’s platform technology to be meaningfully
differentiated from other technologies, the anticipated timing and
sharing of clinical data including from the Company’s ARCT-810 and
ARCT-032 programs, 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 anticipated
conduct of the ARCT-032 study in New Zealand, the likelihood that
preclinical or clinical data will be predictive of future clinical
results, the likelihood that the interim study results of the
ARCT-154 Phase 3 booster vaccine study will be predictive of, or
consistent with, the complete study results, the likelihood that a
patent will issue from any patent application, 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
June 30, 2023
December 31,
2022
(in thousands, except par value
information)
(unaudited)
Assets
Current assets:
Cash and cash equivalents
$
323,471
$
391,883
Restricted cash
55,000
—
Accounts receivable
2,799
2,764
Prepaid expenses and other current
assets
3,974
8,686
Total current assets
385,244
403,333
Property and equipment, net
12,722
12,415
Operating lease right-of-use asset,
net
30,553
32,545
Non-current restricted cash
2,127
2,094
Total assets
$
430,646
$
450,387
Liabilities and stockholders’
equity
Current liabilities:
Accounts payable
$
13,619
$
7,449
Accrued liabilities
28,763
30,232
Current portion of long-term debt
—
60,655
Deferred revenue
47,963
28,648
Total current liabilities
90,345
126,984
Deferred revenue, net of current
portion
25,725
20,071
Operating lease liability, net of current
portion
28,111
30,216
Other non-current liabilities
1,290
2,804
Total liabilities
145,471
180,075
Stockholders’ equity
Common stock, $0.001 par value; 60,000
shares authorized; issued and outstanding shares were 26,574 at
June 30, 2023 and 26,555 at December 31, 2022
27
27
Additional paid-in capital
625,085
608,426
Accumulated deficit
(339,937
)
(338,141
)
Total stockholders’ equity
285,175
270,312
Total liabilities and stockholders’
equity
$
430,646
$
450,387
ARCTURUS THERAPEUTICS HOLDINGS
INC. AND ITS SUBSIDIARIES
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
Three Months Ended
Six Months Ended
June 30,
June 30,
(in thousands, except per share data)
2023
2022
2023
2022
Revenue:
Collaboration revenue
$
9,565
$
27,093
$
89,294
$
32,337
Grant revenue
954
—
1,510
—
Total revenue
10,519
27,093
90,804
32,337
Operating expenses:
Research and development, net
52,668
38,189
104,436
83,082
General and administrative
13,225
10,993
26,987
21,723
Total operating expenses
65,893
49,182
131,423
104,805
Loss from operations
(55,374
)
(22,089
)
(40,619
)
(72,468
)
Loss from equity-method investment
—
(131
)
—
(515
)
Gain (loss) from foreign currency
149
1,217
(179
)
1,375
Gain on debt extinguishment
—
—
33,953
—
Finance income (expense), net
3,252
(560
)
5,729
(1,124
)
Net loss before income taxes
(51,973
)
(21,563
)
(1,116
)
(72,732
)
Provision for income taxes
577
-
680
Net loss
$
(52,550
)
$
(21,563
)
$
(1,796
)
$
(72,732
)
Net loss per share, basic and diluted
$
(1.98
)
$
(0.82
)
$
(0.07
)
$
(2.75
)
Weighted-average shares outstanding, basic
and diluted
26,563
26,425
26,557
26,401
Comprehensive loss:
Net loss
$
(52,550
)
$
(21,563
)
$
(1,796
)
$
(72,732
)
Comprehensive loss
$
(52,550
)
$
(21,563
)
$
(1,796
)
$
(72,732
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230807532754/en/
IR and Media Contacts Arcturus Therapeutics Neda
Safarzadeh VP, Head of IR/PR/Marketing (858) 900-2682
IR@ArcturusRx.com
Kendall Investor Relations Carlo Tanzi, Ph.D. (617) 914-0008
ctanzi@kendallir.com
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