Arcturus Therapeutics Receives U.S. FDA Fast Track Designation for ARCT-810, mRNA Therapeutic Candidate for Ornithine Transcarbamylase Deficiency
June 01 2023 - 7:30AM
Business Wire
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 that the U.S. Food and Drug Administration (FDA) has
granted Fast Track Designation to ARCT-810, the Company’s mRNA
therapeutic candidate for ornithine transcarbamylase (OTC)
deficiency.
Fast Track Designation is designed to facilitate development and
expedite review of new therapeutics intended to treat serious or
life-threatening conditions that demonstrate the potential to
address important unmet medical needs. Programs granted Fast Track
Designation may receive important benefits including more frequent
interactions with FDA review teams and the ability to obtain
rolling review of a Biologics License Application (BLA). In
addition, BLA applications may receive a priority review, with the
FDA aiming to complete its review within six months, instead of the
standard ten months review timeline.
“ARCT-810 has the potential to be an important new medicine for
individuals living with OTC deficiency and we are very pleased to
have obtained Fast Track Designation for this program. This FDA
designation provides several meaningful benefits that we expect
will accelerate development and, hopefully, support the approval of
this investigational therapy,” said Juergen Froehlich, MD, MBA,
FCP, Chief Medical Officer, of Arcturus Therapeutics. “We look
forward to more frequent interactions with the FDA and to
acceleration of ARCT 810 as a potential new treatment option that
addresses the root cause of OTC deficiency.”
About Ornithine Transcarbamylase (OTC) Deficiency
OTC deficiency is a serious urea cycle disorder with a
prevalence of approximately 10,000 people worldwide. A lack of OTC,
a critical urea cycle enzyme in liver cells, results in high blood
ammonia levels that can cause diminished cognitive ability,
seizures, coma and death. The current standard of care for
OTC-deficient patients is a low-protein diet, nutritional
supplements and ammonia scavengers to help prevent the accumulation
of ammonia. These treatments do not address the underlying cause of
OTC deficiency, for which there are no FDA approved medicines.
About ARCT-810
ARCT-810 is a development candidate that represents a novel
approach to treat ornithine transcarbamylase deficiency. ARCT-810
utilizes Arcturus’ mRNA design construct and proprietary
manufacturing process. ARCT-810 was developed using Arcturus’
extensive and proprietary lipid library and employs its LUNAR®
platform to deliver OTC mRNA to hepatocytes. ARCT-810 is an
investigational mRNA medicine designed to enable OTC-deficient
patients to naturally produce functional OTC enzyme in their own
liver cells. Enabling patients to produce their own OTC enzyme has
the potential to restore urea cycle activity, reduce the risk of
metabolic decompensation, and improve quality of life.
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 (samRNA) and (iii) mRNA drug substance along with drug
product manufacturing expertise. 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 likelihood of success of the Company’s pipeline (including
ARCT-810) and partnered programs, the potential for the Company’s
platform to result in novel vaccines or therapeutics, the benefits
that the Company might receive from Fast Track Designation for
ARCT-810, continued or accelerated development of ARCT-810,
likelihood of approval of ARCT-810 or its potential for therapeutic
benefit including restoration of urea cycle activity, 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
Kendall Investor Relations Carlo Tanzi, Ph.D. (617) 914-0008
ctanzi@kendallir.com
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