SAN DIEGO, Feb. 11, 2019 (GLOBE NEWSWIRE) -- Arcturus Therapeutics Ltd. (NASDAQ: ARCT), a leading RNA medicines
company focused on the discovery, development and commercialization of therapeutics towards rare diseases, today announced that it
will reassume 100% global rights for its flagship asset, clinical development candidate ARCT-810, a messenger RNA (mRNA) drug to
treat OTC Deficiency. ARCT-810 was previously subject to a 50/50 collaboration between Arcturus and CureVac AG. CureVac elected not
to continue its obligations for the preclinical development of ARCT-810 under and pursuant to the terms of the collaboration.
The preclinical development program for ARCT-810, including Investigational New Drug Application (IND) enabling studies, remains
on track. Arcturus is planning to file an IND for ARCT-810 with the U.S. Food and Drug Administration (FDA) in the fourth quarter
of 2019.
CureVac remains committed to developing additional assets within the Arcturus collaboration utilizing CureVac mRNA and Arcturus
LUNAR® delivery technology. CureVac and Arcturus will announce when the next program has been selected.
“We have had a very productive collaboration with CureVac on ARCT-810 and are pleased that we have secured all of the
rights to this clinical development candidate. We are enthusiastic about the value of ARCT-810 and we believe it has the potential
to be a transformational treatment for patients suffering from OTC deficiency,” said Joseph Payne, President & CEO of
Arcturus. “Importantly, Arcturus has the resources and expertise to advance this program into the clinic. We are pursuing an
aggressive development timeline with an IND filing planned for this year. We look forward to our continued collaboration with
CureVac to advance therapies for patients in need of potential new treatment options.”
Arcturus plans to present further data and updates on the progress of ARCT-810 at the “TIDES: Oligonucleotide & Peptide
Therapeutics” conference, to take place May 20th to 23rd, 2019 in San Diego, CA.
About ARCT-810
ARCT-810, Arcturus’ first development candidate, represents a novel approach to treat ornithine transcarbamylase deficiency
(OTCD). ARCT-810 is based on mRNA and also utilizes Arcturus’ propriety lipid library and employs the Company's
LUNAR® delivery platform to safely and effectively deliver OTC mRNA to hepatocytes. ARCT-810 is an mRNA
replacement therapy designed to enable OTC-deficient patients to naturally produce healthy functional OTC enzyme in their own liver
cells. Arcturus is currently on track to submit an Investigational New Drug Application (IND) to the FDA in the fourth
quarter of 2019. ARCT-810 is advancing toward the clinic on the strength of preclinical proof-of-concept data, demonstrating that
LUNAR technology can deliver mRNA to liver cells and results in expression of functional OTC protein in animal models.
Replacing the deficient OTC protein restores the urea cycle pathway, resulting in reduced plasma ammonia and urinary orotate
concentrations.
About Ornithine Transcarbamylase Deficiency (OTCD)
OTC deficiency is caused by mutations in the OTC gene which leads to a non-functional or deficient OTC enzyme. OTCD is the most
common urea cycle disorder. Urea cycle disorders are a group of inherited metabolic disorders that make it difficult for
afflicted patients to remove toxic waste products, as proteins are digested. OTC deficiency is a life-threatening genetic
disease. OTC is a critical enzyme in the urea cycle, which takes place in liver cells, and 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. This can cause severe symptoms including vomiting, headaches, coma and death. 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 boys. 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 patients is a
low-protein diet and ammonia scavengers to try and prevent patients from accumulating ammonia. These treatments do not address the
underlying cause of disease.
About Arcturus Therapeutics Ltd.
Founded in 2013 and headquartered in San Diego, California, Arcturus Therapeutics Ltd. (NASDAQ: ARCT) is an RNA
medicines company with enabling technologies – LUNAR® lipid-mediated delivery and UNA Oligomer chemistry. Arcturus’
diverse pipeline of RNA therapeutics includes programs pursuing rare diseases, Hepatitis B, non-alcoholic steatohepatitis (NASH),
cystic fibrosis, and vaccines. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid
medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA and gene editing therapeutics.
Arcturus owns LUNAR lipid-mediated delivery and Unlocked Nucleomonomer Agent (UNA) technology including UNA Oligomers, which are
covered by its extensive patent portfolio (150 patents and patent applications, issued in the
U.S., Europe, Japan, China and other countries). Arcturus’ proprietary UNA technology can be used to target
individual genes in the human genome, as well as viral genes, and other species for therapeutic purposes. Arcturus’ commitment to
the development of novel RNA therapeutics has led to partnerships with Janssen Pharmaceuticals, Inc., part of the Janssen
Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company
Limited, Synthetic Genomics Inc., CureVac AG and the Cystic Fibrosis Foundation. For more
information, visit www.Arcturusrx.com, the content of which is not incorporated herein by reference.
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 regarding strategy, future operations, collaborations, future financial position, prospects, plans
and objectives of management, including statements relating to the status of the preclinical development program for ARCT-810, the
date that an IND may be filed with the FDA, the potential for ARCT-810, are forward-looking statements. Arcturus may not actually
achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements and
you should not place undue reliance on such forward-looking statements. Actual results and performance could differ materially from
those projected in any forward-looking statements as a result of many factors, including without limitation, an inability to
develop and market product candidates. Such statements are based on management’s current expectations and involve risks and
uncertainties, including those discussed under the heading “Risk Factors” in Arcturus’ Annual Report on Form 20-F for the fiscal
year ended December 31, 2017, filed with the SEC on May 14, 2018 and in subsequent filings with, or submissions to, the SEC. 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.
Contact
Neda Safarzadeh
Arcturus Therapeutics
(858) 900-2682
IR@ArcturusRx.com
Arcturus Investor Contact
Michael Wood
LifeSci Advisors LLC
(646) 597-6983
mwood@lifesciadvisors.com