Arcturus Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ARCT-032, for the Treatment of Cystic Fibrosis
November 27 2023 - 8:30AM
Business Wire
First cystic fibrosis patient in Phase 1b study
successfully completed two administrations of ARCT-032
On track to share interim Phase 1b data in H1
2024
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 the U.S. Food and Drug Administration (FDA) has granted
Orphan Drug Designation for the Company’s product candidate
ARCT-032 to treat cystic fibrosis (CF).
The FDA’s Office of Orphan Products Development grants orphan
status to drugs being developed to treat, prevent, or diagnose a
rare disease or condition affecting fewer than 200,000 people in
the United States. The designation provides significant incentives
to promote the development of the drug including the potential for
market exclusivity for seven years upon FDA approval, eligibility
for tax credits for qualified clinical trials, waiver of
Prescription Drug User Fee Act Application fee, and eligibility to
receive regulatory guidance from the FDA in the design of an
overall drug development plan.
“Orphan Drug Designation is a very important regulatory
milestone in our development plan for ARCT-032,” said Joseph Payne,
President, and Chief Executive Officer of Arcturus Therapeutics.
"We are executing diligently to accelerate ARCT-032 as a potential
new treatment option for people with cystic fibrosis.”
The first CF patient in our Phase 1b study successfully
completed two administrations of ARCT-032. We remain on track to
share interim Phase 1b data in H1 2024.
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.
About ARCT-032
ARCT-032 utilizes 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 CFTR
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 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 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 ability to
enroll, and timing for enrollment of, subjects in clinical trials,
the likelihood of sharing and timing for sharing interim Phase 1b
data, 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
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