Arcturus Therapeutics Announces Clinical Trial Application for ARCT-032 Received Approval to Proceed into First-in-Human Studies to Treat Cystic Fibrosis
01 February 2023 - 12:30AM
Business Wire
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 that the
Clinical Trial Application (CTA) for ARCT-032, an inhaled
investigational mRNA medicine to treat cystic fibrosis (CF),
received approval to proceed into a Phase 1 First-in-Human study in
New Zealand.
“Arcturus continues to progress ARCT-032, a potential mRNA
medicine for people with cystic fibrosis regardless of their
underlying mutation type, through the approval of a CTA to proceed
into First-in-Human studies,” said Joseph Payne, President and CEO
of Arcturus Therapeutics. “We believe the advantages driven by our
proprietary LUNAR® delivery technology, advanced mRNA purification
processes, and manufacturing know-how may allow ARCT-032 to restore
healthy CFTR protein in the lungs of people with CF, including
those that currently do not have effective treatment options.
Preclinical data shared at the recent North American Cystic
Fibrosis Conference demonstrated robust expression and functional
restoration of CFTR in human bronchial epithelial cells from CF
donors, providing additional support for the advancement of
ARCT-032 into clinical development.”
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide
distribution. Mutations in the Cystic Fibrosis Transmembrane (CFTR)
gene result in a reduction or absence of CFTR quantity and/or
function in the airways, causing insufficient chloride transport to
maintain airway surface homeostasis. CF mucus is more difficult to
clear, thus clogging the airways and leading to infection,
inflammation, respiratory failure, or other life-threatening
complications. Currently approved CFTR modulator therapies are
designed to increase function of the CFTR channel to help reduce
symptoms yet are ineffective in many people with CF as a result of
their underlying mutations.
About ARCT-032
ARCT-032 will utilize Arcturus' LUNAR® lipid-mediated
aerosolized platform to deliver CFTR messenger RNA to the lungs.
Expression of a functional copy of the CFTR mRNA in the lungs of
people with CF has the potential to restore CFTR activity and
mitigate the downstream effects that cause progressive lung
disease. The ARCT-032 program is supported by preclinical data in
rodents, ferrets and primates, as well as demonstrating restoration
of expression and function in human bronchial epithelial cells.
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 virus. 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 expectations for or likelihood of success of any
collaborations, the likelihood of success (including safety and
efficacy) of ARCT-032, the planned initiation, design or completion
of clinical trials (including the planned ARCT-032 Phase 1 study),
the likelihood that preclinical data will be predictive of future
clinical results, the likelihood that ARCT-032 will provide broad,
or any, clinical benefit (including the ability of ARCT-032 to
restore healthy CFTR protein), the ability to enroll, and timing
for enrollment of, subjects in clinical trials, the timing and
nature of any study results, the likelihood that a patent will
issue from any patent application, 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.
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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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