New ARCT-154 clinical booster data demonstrate
promising durability; 44- and 39-fold increases in neutralizing
antibody response against Omicron BA.1 and BA.2 at Day 91
New cold chain data for Arcturus’ lyophilized
vaccine platform shows favorable stability, shipping, and storage
advantages
European Commission grants Orphan Medicinal
Product Designation to ARCT-810, a novel mRNA candidate for
ornithine transcarbamylase (OTC) deficiency
Investor conference call at 4:30 p.m. EDT
today
Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”,
“Arcturus Therapeutics”, Nasdaq: ARCT), a global, late-stage
clinical 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 second quarter ended June 30, 2022, and provided
corporate updates.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20220809006007/en/
Figure 1: Exploratory pseudovirus
microneutralization (MNT) assay results (left: BA.1, right: BA.2),
showing GMFR levels of neutralizing antibody responses over Day 1
(baseline levels prior to boosting with ARCT-154) calculated with
virus neutralization concentrations (with 95% confidence intervals)
obtained for participants (for BA.1 and BA.2: n = 12/12, Day 91).
(Graphic: Business Wire)
“Arcturus continues to demonstrate increasing value of our next
generation mRNA vaccine and therapeutic platforms,” said Joseph
Payne, President and CEO of Arcturus Therapeutics. “Additional data
from our Phase 1/2 booster study demonstrates that ARCT-154
provides sustained neutralizing antibodies against Omicron variants
BA.1/BA.2 three months after booster vaccination. We are further
pleased to share new data demonstrating the stability of our
lyophilized vaccines under a broad range of transportation and
storage conditions. Our work on the LUNAR-OTC program has been
acknowledged with Orphan Medicinal Product Designation issued by
the European Commission, which is promising news for the continued
development of ARCT-810 for OTC deficient patients.”
Recent Corporate Highlights
- Arcturus shared additional data from the ARCT-154 arm of its
ongoing Phase 1/2 study in the U.S., Singapore, and South Africa
evaluating a single 5-mcg booster dose of ARCT-154 given at least
five months following two primary doses of Comirnaty®. In
exploratory microneutralization titer (MNT) assays, ARCT-154
demonstrates a robust neutralizing antibody response against the
Omicron BA.1 and BA.2 variants up to 91 days after administration.
Against BA.1 the Geometric Mean Fold Rise (GMFR) was 44-fold at Day
91 compared to 54-fold at Day 29. Against BA.2 the GMFR was 39-fold
at Day 91 compared to 47-fold at Day 29 (Figure 1). Six-month data
for Omicron variants, including BA.5, is being collected and will
be shared this quarter.
- New stability data supports the advantages of using a
lyophilized vaccine powder to mitigate the cold chain challenges
associated with frozen liquid mRNA vaccines. Our lyophilized COVID
vaccine powder demonstrated room temperature stability (25°C RT;
60% RH) for 4 days, refrigerator stability (2-8°C) for 6 months,
and a predicted long-term stability (-25°C to -15°C) for 18 months.
Importantly, the lyophilized powder was not impacted by multiple
temperature cycling stress (-20°C to 2-8°C or RT) enabling global
shipping logistics and supply of a stable usable solid COVID
vaccine product at 2-8°C.
- ARCT-810 Orphan Medicinal Product Designation: Arcturus was
notified by the European Commission that ARCT-810 has been
designated as an Orphan Medicinal Product for the treatment of OTC
deficiency. The designation provides significant incentives to
promote the development of the drug including protocol assistance,
access to the centralized authorization procedure, fee reductions,
and research grants, as well as 10 years of market
exclusivity.
- ARCT-810, the Company’s mRNA therapeutic candidate for OTC
deficiency will be evaluated in a randomized, double-blind,
placebo-controlled, nested single and multiple ascending dose Phase
2 study in 24 adolescents and adults with OTC deficiency.
Participating sites have identified several dozen patients in
pre-screening, with the goal of obtaining interim proof-of-concept
data by year end.
- Due to additional non-clinical data requirements, ARCT-032 (the
Company’s inhaled mRNA therapeutic candidate for cystic fibrosis)
is now expected to file for a Clinical Trial Application in Q4
2022.
- The Vinbiocare manufacturing facility in Hanoi, Vietnam, is
anticipated to achieve commercial scale production capability in Q4
2022.
- The Vietnam Ministry of Health (MOH) is undergoing a
significant reorganization resulting in a delay of the ARCT-154
Emergency Use Authorization (EUA) for the primary series. During
the reorganization, the MOH communicated that the Clinical section
review is complete and adequate. The MOH provided comments to the
Chemistry, Manufacturing, and Controls (CMC) section, requesting
three manufacturing runs to support full Market Authorization.
ARCT-154 is now expected to receive EUA in Q4 2022, with full
Marketing Authorization anticipated in early 2023 if booster
studies meet noninferior immunogenicity and safety objectives.
- Arcturus expects a registrational booster study for ARCT-154 to
begin in Q4 2022. Based on recent health authority guidance, the
Company is considering an updated design consisting of two trials
to support global registration of ARCT-154 as a booster.
Financial Results for Second Quarter Ended June 30,
2022
Revenues in conjunction with strategic alliances and
collaborations: Arcturus’ primary sources of revenues were from
consulting and related technology transfer fees, reservation fees,
license fees and collaborative payments received from research and
development arrangements with pharmaceutical and biotechnology
partners. For the three months ended June 30, 2022, the Company
reported revenue of $27.1 million compared with $2.0 million for
the three months ended June 30, 2021, and $5.2 million for the
three months ended March 31, 2022. The increase in revenue during
the three months ended June 30, 2022, as compared to the three
months ended March 31, 2022, and June 30, 2021, was primarily due
to revenue of $12.5 million related to the one-time recognition of
reservation fees from the Israel MOH and an increase in revenue
from Vinbiocare related to shipments of drug substance to validate
their manufacturing facility.
Operating expenses: Total operating expenses for the
three months ended June 30, 2022, were $49.2 million compared with
$55.7 million for the three months ended June 30, 2021, and $55.7
million for the three months ended March 31, 2022. The decline in
operating expenses during the three months ended June 30, 2022, as
compared to the three months ended March 31, 2022 was due primarily
to a decrease in clinical trial expenses related to ARCT-154.
Research and development expenses: Research and
development expenses for the three months ended June 30, 2022, were
$38.2 million compared with $45.7 million for the three months
ended June 30, 2021, and $44.9 million for the three months ended
March 31, 2022. The decline in operating expenses during the three
months ended June 30, 2022, as compared to the three months ended
March 31, 2022, and June 30, 2021, was due primarily to a decrease
in clinical trial expenses.
Net Loss: For the three months ended June 30, 2022,
Arcturus reported a net loss of approximately $21.6 million, or
($0.82) per basic and diluted share, compared with a net loss of
$54.6 million, or ($2.07) per basic and diluted share in the three
months ended June 30, 2021, and a net loss of $51.2 million, or
($1.94) per basic and diluted share in the three months ended March
31, 2022.
Cash Position: The Company’s cash balance totaled $283.5
million as of June 30, 2022, compared to a cash balance of $370.5
million at December 31, 2021. Based on the current pipeline, the
Company’s cash position is expected to be sufficient to support
operations into late 2023.
Earnings Call: Tuesday, August 9, 2022 @ 4:30 pm EDT
Domestic: 1-800-263-0877 International: 1-323-794-2094 Conference
ID: 8017918 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’ 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 III, and hepatitis B virusArcturus’ 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 protected by 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 including, amongst
others, Janssen Pharmaceuticals, Inc., part of the Janssen
Pharmaceutical Companies of Johnson & Johnson, Ultragenyx
Pharmaceutical, Inc., and the Cystic Fibrosis Foundation. Please
connect with us on Twitter and LinkedIn. For more information visit
www.ArcturusRx.com.
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 expectations for or likelihood of success of any
collaborations, the promise of the company’s platform technologies
for multiple types of nucleic acid medicines, the likelihood of
success (including safety and efficacy) of the Company’s pipeline
(including ARCT-154, ARCT-810, ARCT-032 and a STARR mRNA candidate
for influenza), the likelihood that any independent verification by
Arcturus, or any regulatory body’s assessment of data will be
consistent with the information shared by Vinbiocare from the
ARCT-154 study in Vietnam, the likelihood that the clinical results
of ARCT-154 studies (including the efficacy, booster durability or
other antibody responses), or any other non-clinical or clinical
data, will be predictive of future clinical results or efficacy,
predictive of results against existing or future COVID variants, or
sufficient for any regulatory approval, completion and final data
readouts of, our Phase 1/2 booster study of ARCT-154, our ability
to enroll and timing of enrollment of subjects in clinical trials,
including the planned Phase 2 study for ARCT-810, or any other
clinical trials, the timing of interim data from the ARCT-810 study
or any other data, the likelihood or timing of an EUA or regulatory
approval in Vietnam for ARCT-154 or for any regulatory approval,
the likelihood, and timing for, a filing to proceed with a clinical
study of ARCT-032, the completion (including timing) of
Vinbiocare’s manufacturing facility (including actual commercial
production capabilities, if any), the likelihood that a patent will
issue from any patent application, its financial projections,
current cash position and expected cash burn 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 U.S. Securities and Exchange Commission (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
(in thousands, except par value
information)
June 30, 2022
December 31, 2021
(unaudited)
Assets
Current assets:
Cash and cash equivalents
$
283,491
$
370,492
Accounts receivable
2,247
3,367
Prepaid expenses and other current
assets
5,767
5,102
Total current assets
291,505
378,961
Property and equipment, net
8,951
5,643
Operating lease right-of-use asset,
net
34,480
5,618
Equity-method investment
—
515
Non-current restricted cash
2,078
2,077
Total assets
$
337,014
$
392,814
Liabilities and stockholders’
equity
Current liabilities:
Accounts payable
$
5,762
$
10,058
Accrued liabilities
33,614
23,523
Current portion of long-term debt
27,018
22,474
Deferred revenue
26,349
43,482
Total current liabilities
92,743
99,537
Deferred revenue, net of current
portion
5,590
19,931
Long-term debt, net of current portion
35,761
40,633
Operating lease liability, net of current
portion
32,203
4,502
Total liabilities
$
166,297
$
164,603
Stockholders’ equity
Common stock: $0.001 par value; 60,000
shares authorized; 26,434 issued and outstanding at June 30, 2022
and 26,372 issued and outstanding at December 31, 2021
26
26
Additional paid-in capital
590,913
575,675
Accumulated deficit
(420,222
)
(347,490
)
Total stockholders’ equity
170,717
228,211
Total liabilities and stockholders’
equity
$
337,014
$
392,814
ARCTURUS THERAPEUTICS HOLDINGS
INC. AND ITS SUBSIDIARIES
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands except per share
data)
Three Months Ended
June 30,
March 31,
2022
2021
2022
Revenue
$
27,093
$
2,001
$
5,244
Operating expenses:
Research and development, net
38,189
45,679
44,893
General and administrative
10,993
10,042
10,730
Total operating expenses
49,182
55,721
55,623
Loss from operations
(22,089
)
(53,720
)
(50,379
)
Loss from equity-method investment
(131
)
(328
)
(384
)
Gain (loss) from foreign currency
1,217
(13
)
158
Finance expense, net
(560
)
(520
)
(564
)
Net loss
$
(21,563
)
$
(54,581
)
$
(51,169
)
Net loss per share, basic and diluted
$
(0.82
)
$
(2.07
)
$
(1.94
)
Weighted-average shares outstanding, basic
and diluted
26,425
26,323
26,376
Comprehensive loss:
Net loss
$
(21,563
)
$
(54,581
)
$
(51,169
)
Comprehensive loss
$
(21,563
)
$
(54,581
)
$
(51,169
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220809006007/en/
IR and Media Contacts Arcturus Therapeutics
IR@arcturusrx.com
Kendall Investor Relations Carlo Tanzi, Ph.D. (617) 914-0008
ctanzi@kendallir.com
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