ARCT-810 Phase 2 study completed enrollment at
0.3 mg/kg dose level in Europe with data expected later this
year
ARCT-810 to expand Phase 2 clinical program in
the U.S. to enroll patients with more severe disease
ARCT-032 IND for Phase 2 multiple ascending
dose study to be submitted in the next 60 days
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 is providing mid-year updates for ARCT-810, an
investigational mRNA therapeutic to treat ornithine
transcarbamylase (OTC) deficiency, and ARCT-032, an investigational
inhaled mRNA therapeutic to treat cystic fibrosis (CF).
“CFTR replacement using mRNA therapeutics is an area of
significant medical interest, as it may benefit null patients and
potentially many other CF patients,” said Dr. Juergen Froehlich,
Chief Medical Officer of Arcturus Therapeutics. “The safety profile
of ARCT-032 observed in Phase 1 and after two administrations in
Phase 1b for patients dosed to date, including the trend of lung
function improvement in the Class I participant, is encouraging.
These data are supportive of expanding our clinical program to the
U.S. and other regions with a Phase 2 multiple ascending dose
protocol, and I look forward to a successful conduct and outcome of
this planned study. We are also excited to have started patient
screening for the expansion of our ARCT-810 clinical program in the
U.S. to more severely affected OTC deficiency patients.”
ARCT-810 (OTC Deficiency)
Update
The double blind ARCT-810 Phase 2 study in the EU and UK has
completed enrollment of eight (8) subjects, including adolescents
and adults, at the 0.3 mg/kg dose level. The participants in this
group are randomized 3:1 and receive 6 doses of ARCT-810 or placebo
administered every 14 days. Treatment and follow-up are ongoing
with the safety and complete set of biomarker data expected later
this year.
ARCT-810 is expanding the Company’s clinical program in the U.S.
by enrolling patients with more severe disease. Patient screening
has been initiated and the Company expects the Phase 2 clinical
program to be completed in the United States. More details
pertaining to the U.S. trial expansion and interim data will be
provided later this year.
ARCT-032 (CF) Update
The Company plans to submit an IND application in the next 60
days for an ARCT-032 Phase 2 multiple ascending dose study designed
to identify a safe and effective dose in Class I (null) and other
CF participants who do not benefit from CFTR modulators. This study
is supported by safety and tolerability data collected in healthy
volunteers (N = 32) and the ongoing two-administration Phase 1b
study. No serious adverse events (SAEs) have been observed in any
clinical trial participants to date. No febrile reactions have been
observed within the target dose range of the planned Phase 2 study.
The Phase 1b study is ongoing with the seventh and final
participant scheduled to be dosed soon. Of the seven total CF
participants in Phase 1b, six are receiving CFTR modulator
treatment while one subject has Class I mutations and therefore
does not benefit from modulator therapy. The Class I CF subject had
low lung function at baseline (ppFEV1 below 50%) and showed an
improvement of 4% in ppFEV1 on Day 8, after receiving two
well-tolerated administrations, with no febrile reactions.
About Ornithine Transcarbamylase Deficiency
Ornithine transcarbamylase (OTC) deficiency is the most common
urea cycle disorder. Urea cycle disorders are a group of inherited
metabolic disorders that make it difficult for affected patients to
remove toxic waste products as proteins are digested. OTC
deficiency is caused by mutations in the OTC gene which leads to a
non-functional or deficient OTC enzyme. OTC is a critical enzyme in
the urea cycle, which takes place in liver cells, and together with
the other enzymes in the urea cycle converts ammonia to urea. This
conversion does not occur properly in patients with OTC deficiency
and ammonia accumulates in their blood, acting as a neurotoxin and
liver toxin. A lack of the OTC enzyme in liver cells can results in
high blood ammonia levels and cause seizures, coma, and death in
untreated patients. OTC deficiency is an inherited disease that can
cause developmental problems, seizures and death in newborn babies.
It is an X-linked disorder, so is more common in males. Patients
with less severe symptoms may present later in life, as adults.
There is currently no cure for OTC deficiency, apart from liver
transplant. However, this treatment comes with significant risk of
complications such as organ rejection, and transplant recipients
must take immunosuppressant drugs for the rest of their lives.
Current standard of care for OTC deficiency patients is a
well-controlled, but challenging to maintain, low-protein diet and
treatment with ammonia scavengers to try and prevent patients from
accumulating ammonia. These treatments do not address the
underlying cause of disease. In Europe and the U.S., approximately
10,000 people have OTC deficiency.
About ARCT-810
ARCT-810 is an intravenously administered investigational mRNA
therapeutic designed to express normal functional OTC enzyme in the
liver of individuals with OTC deficiency. ARCT-810 has received
Orphan Medicinal Product Designation from the European Medicines
Agency (EMA), including an approved pediatric investigation plan
(PIP), and Orphan Drug Designation along with Rare Pediatric
Disease Designation from the U.S. Food and Drug Administration
(FDA) for the treatment of ornithine transcarbamylase deficiency.
OTC is a key enzyme in the urea cycle which converts toxic ammonia
into urea. Elevated ammonia can lead to metabolic crises with
progressive and irreversible neurocognitive damage. A safe and
effective mRNA therapeutic may restore normal functional OTC enzyme
in the liver which could eliminate the risk of future metabolic
crises. ARCT-810 is based on Arcturus’ mRNA design construct and
proprietary manufacturing process. ARCT-810 also utilizes Arcturus’
extensive and propriety lipid library and employs the Company's
LUNAR® delivery platform to deliver OTC mRNA to hepatocytes.
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide
prevalence. 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. Standard of care
for many CF individuals include CFTR modulators. Nearly 40,000
people in the U.S. and more than 105,000 people worldwide are
living with CF. Approximately 15% of individuals with CF do not
benefit from CFTR modulator medicines due to dysfunctional or
absent CFTR protein and/or drug intolerance.
About ARCT-032
ARCT-032 is an inhaled investigational mRNA therapeutic designed
to express normal functional CFTR in the lungs of individuals with
CF. ARCT-032 has received Orphan Medicinal Product Designation from
the European Medicines Agency (EMA) and Orphan Drug Designation
along with Rare Pediatric Disease 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. Lung disease is the
leading cause of morbidity and mortality in people with CF.
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 likelihood of success (including safety and efficacy) of
ARCT-810 or ARCT-032, the likelihood that the trend of lung
function changes in the Class I participant for ARCT-032 will
continue or provide meaningful efficacy, the expected timing for
the safety and biomarker data for ARCT-810, the planned expansion
of the ARCT-810 clinical program and the Company’s ability and
decision to enroll and conduct such expansion, the continued
clinical development of ARCT-810, the potential for CFTR
replacement to benefit null patients or other CF patients, the
continued clinical development of ARCT-032 including the likelihood
of and timing for submission of an IND for and plans to conduct a
Phase 2 study, the likelihood that clinical results received to
date (including data in the ARCT-032 Phase 1b study) will be
predictive of future clinical results or sufficient to further
advance the CF program, the likelihood of and timing for expanding
the CF clinical program 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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