Full Press Release Details
Therapeutics and Cystic Fibrosis Foundation Extend Agreement to Advance ARCT-032, an Investigational Messenger RNA (mRNA) Therapeutic
to Treat Cystic Fibrosis
CF Foundation Increases Financial Commitment
SAN DIEGO--(BUSINESS WIRE)--Sept. 26, 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,
announced today that Arcturus and the Cystic Fibrosis Foundation (CF Foundation) have extended their ongoing agreement. The CF Foundation
agreed to increase its financial commitment to ~$25 million to advance ARCT-032, a novel messenger RNA (mRNA) therapeutic candidate formulated
with Arcturus' LUNAR delivery technology. This agreement extends a prior research program, initiated in 2017, which has resulted
in the advancement of ARCT-032 into ongoing clinical studies.
"We are thrilled to extend our productive
relationship with the Cystic Fibrosis Foundation, and we are grateful for their meaningful financial support," said Pad Chivukula,
Ph.D., Chief Scientific Officer of Arcturus. "ARCT-032 has the potential to express fully functional CFTR protein in the lung, thereby
addressing the root cause of cystic fibrosis. The resources and valuable expertise provided by the CF Foundation will support the continued
clinical development of ARCT-032, including the completion of a Phase 1b study that we plan to initiate imminently in adults living with
ARCT-032 is made up of mRNA that codes for CFTR,
encapsulated by lipid nanoparticles utilizing LUNAR delivery technology that has been optimized for inhaled lung delivery. ARCT-032 is
agnostic to the underlying mutations associated with the disease and has the potential to provide clinical benefits across a wide range
of those living with CF, including those not served by currently approved CFTR modulators. The program has completed dosing in a Phase
1 study in healthy volunteers.
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with
a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence
of CFTR protein 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 some people with CF because of their underlying mutations.
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 (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. The Company has ongoing collaborations
with CSL Seqirus and Meiji Seika Pharma, and a joint venture with ARCALIS. 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 expectations for or likelihood of success of any collaborations, the continued
clinical development of ARCT-032, 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 1b 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
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