ARCT-032 is safe and well tolerated with no
serious adverse events (SAEs) in 36 study participants, including 4
adults with cystic fibrosis (CF)
Phase 1b interim data includes a CF participant
with Class 1 mutations and three participants with F508del
mutations being treated with Trikafta®
Early trend of improved lung function with an
average absolute response of +4.0% (ranging up to +9%) and relative
change of +5.8% FEV1 on Day 8, after two doses of ARCT-032
Clinical data is consistent with pre-clinical
CF ferret model data
Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”,
Nasdaq: ARCT), a global 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 presented Phase 1 results in healthy volunteers and
Phase 1b interim data in people with CF for ARCT-032, an inhaled
investigational mRNA therapeutic, at the 47th European Cystic
Fibrosis Conference in Glasgow, Scotland.
ARCT-032 administration was generally safe and well tolerated
with no serious or severe adverse events in healthy volunteers and
the first four dosed participants with CF. The Phase 1b trial
showed improvements in FEV1 (Forced Expiratory Volume in 1 second)
in the four adults with CF after two inhaled administrations. The
absolute change in percent predicted FEV1 averaged +4.0% on Day 8
(5 days after 2nd dose). The relative change in FEV1 averaged +5.8%
on Day 8. The observed increases in FEV1 are encouraging and
consistent with the previously reported data in the CF ferret model
that demonstrated markedly improved mucociliary clearance (MCC)
after a single dose of ARCT-032. Of the four participants in Phase
1b to date, one had 2 Class I mutations and the other three had
F508del mutations and were being treated with Trikafta®.
Phase 1b Interim ppFEV1* Data
Subject #
Age
Sex
Genotype
On Trikafta®
Baseline ppFEV1
Day 8 ppFEV1
1
24
F
F508
Yes
83%
85%
2
43
M
F508/G85E
Yes
72%
81%
3
27
F
F508
Yes
68%
69%
4
40
F
G542X (Class I)
No
45%
49%
*ppFEV1 = percent predicted Forced
Expiratory Volume in 1 second
No bronchospasm or febrile reactions were observed in the CF
participants. Dose-related, mild-to-moderate febrile reactions
(elevated temperature associated with headache, muscle aches, back
pain, or nausea) occurred in some healthy volunteers. Dose-related
transient declines in FEV1 observed in healthy volunteers were
mitigated by pretreatment with albuterol, a commonly used
bronchodilator. No serious adverse events or dose limiting
toxicities were observed at any dose level.
“We are pleased to present positive ARCT-032 Phase 1 results and
Phase 1b interim data showing that single doses at all dose levels
in healthy volunteers and two doses in the first four CF
participants were safe and well tolerated with no serious adverse
events. It is encouraging to see favorable lung function
improvements in all currently dosed CF participants in this early
study,” said Dr. Juergen Froehlich, Chief Medical Officer of
Arcturus Therapeutics. “We look forward to completing the Phase 1b
trial shortly and to evaluate the further potential for lung
function improvement of treatment with ARCT-032 in people with CF
in a larger, multiple-dose clinical study.”
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 has received Orphan Medicinal Product Designation from
the European Medicines Agency (EMA) and Orphan Drug Designation
from the U.S. Food and Drug Administration (FDA) to treat Cystic
Fibrosis. 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 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 (Kostaive®)
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 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
including the ability to complete and timing for completion of the
CF Phase 1b study, likelihood of success (including safety and
efficacy) of ARCT-032, the likelihood that the interim results will
be predictive of future clinical results, the likelihood of sharing
and timing for sharing interim and final Phase 1b data, the plans
to conduct a larger, multiple-dose clinical study 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.
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.
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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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