Arcturus Therapeutics Receives Orphan Medicinal Product Designation from the European Commission (EC), for ARCT-032, for the Treatment of Cystic Fibrosis
22 Febbraio 2024 - 2:30PM
Business Wire
Access to protocol assistance, centralized
authorization process, fee reductions and 10 years of market
exclusivity
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 medicines to treat unmet medical needs within liver and
respiratory rare diseases, today announced that the European
Commission (EC), based on a positive opinion issued by the European
Medicines Agency (EMA), has granted orphan medicinal product
designation for the Company’s product candidate ARCT-032 to treat
Cystic Fibrosis (CF). In November 2023, the U.S. Food and Drug
Administration (FDA) granted orphan drug designation to ARCT-032
for CF.
Orphan medicinal product designation by the EC is available to
novel therapeutics that prevent or treat life-threatening or
chronically debilitating conditions that affect fewer than five in
10,000 persons in the EU. 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.
“We are pleased to receive orphan medicinal product designation
from the EC for ARCT-032, as it represents a significant milestone
for our CF program,” said Joseph Payne, President & CEO of
Arcturus Therapeutics. “We will continue to advance ARCT-032 as a
potential new treatment option for people with CF regardless of
their genetic mutations.”
After successful completion of the single-ascending-dose portion
of the study in healthy adults, the first participant with CF in
the Phase 1b study part enrolled and completed two administrations
of ARCT-032. Arcturus remains 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. Arcturus developed the first
self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world
to be approved. Arcturus 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 (over 400 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 the continued clinical
development of ARCT-032, the likelihood of sharing and timing for
sharing interim Phase 1b data, likelihood of success (including
safety and efficacy) of ARCT-032, the Company’s strategy and future
operations, 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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