Ultragenyx Pharmaceutical, Inc. (Nasdaq: RARE), a biopharmaceutical
company focused on the development of novel products for serious
rare and ultra-rare genetic diseases and Arcturus Therapeutics
Holdings Inc. (Nasdaq: ARCT), a leading messenger RNA medicines
company, today announced that they have expanded their
collaboration to discover and develop mRNA, DNA and siRNA
therapeutics for up to 12 rare disease targets.
“This expanded collaboration further solidifies our mRNA
platform by adding additional targets and expanding our ability to
potentially treat more diseases,” said Emil D. Kakkis, M.D., Ph.D.,
Chief Executive Officer and President of Ultragenyx. “We are
pleased with the progress of our ongoing collaboration. Our most
advanced mRNA program, UX053 for the treatment of Glycogen Storage
Disease Type III, is expected to move into the clinic next year,
and we look forward to further building upon the initial success of
this partnership.”
“The expansion of the collaboration with Ultragenyx underscores
the partnership’s early successes and ongoing commitment,” said
Joseph Payne, President and Chief Executive Officer of Arcturus.
“Our expertise in the discovery, early development and
manufacturing of RNA medicines aligns well with Ultragenyx’s proven
clinical development and commercial experience in rare diseases.”
He further added, “We look forward to utilizing Ultragenyx’s
seasoned experience and presence on our Board of Directors to
advance mRNA therapeutics as a new class of genetic medicines, and
build Arcturus into a leading, independent biopharmaceutical
company that delivers value to patients and shareholders.”
In connection with the amendment to the license agreement,
Ultragenyx made a $6 million cash upfront payment to Arcturus.
Ultragenyx is also purchasing 2,400,000 shares of Arcturus’ common
stock at a stated value of $10 per share. Ultragenyx has an option
to purchase an additional 600,000 shares of Arcturus’ common stock
at $16 per share. Ultragenyx will become Arcturus’ largest
shareholder with Karah Parschauer, J.D., General Counsel of
Ultragenyx, joining Arcturus’ Board of Directors and Emil D.
Kakkis, M.D., Ph.D., Chief Executive Officer and President of
Ultragenyx, joining as an Arcturus Board Observer.
Arcturus is entitled to preclinical, clinical, regulatory, and
sales milestone payments for each product developed under the
collaboration. Under the amended license agreement, certain
early-stage milestone payments are reduced and the total potential
milestone payments are increased due to the expanded number of
targets. Arcturus is also entitled to reimbursement of related
research expenses and royalties on commercial sales.
The original collaboration and license agreement between
Ultragenyx and Arcturus was signed in October 2015. The two
companies have been working together to develop mRNA therapeutic
candidates for certain rare disease targets. The first disclosed
indication under the collaboration is Glycogen Storage Disease Type
III, and an Investigational New Drug (IND) application for this
mRNA therapeutic program, UX053, is expected to be filed in
2020.
About Ultragenyx Pharmaceutical, Inc.Ultragenyx
is a biopharmaceutical company committed to bringing patients novel
products for the treatment of serious rare and ultra-rare genetic
diseases. The company has built a diverse portfolio of approved
therapies and product candidates aimed at addressing diseases with
high unmet medical need and clear biology for treatment, for which
there are typically no approved therapies treating the underlying
disease.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx’s strategy is predicated upon time and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the Company's
website at www.ultragenyx.com.
About Arcturus Therapeutics Holdings Inc.
Founded in 2013 and based in San Diego, California, Arcturus
Therapeutics Holdings Inc. (NASDAQ: ARCT) is an RNA medicines
company with enabling technologies – LUNAR® lipid-mediated delivery
and Unlocked Nucleomonomer Analog (UNA) chemistry – and mRNA drug
substance along with drug product manufacturing. Arcturus’ diverse
pipeline of RNA therapeutics includes programs to potentially treat
Ornithine Transcarbamylase (OTC) Deficiency, Cystic Fibrosis,
Glycogen Storage Disease Type 3, Hepatitis B, and non-alcoholic
steatohepatitis (NASH). Arcturus’ versatile RNA therapeutics
platforms can be applied toward multiple types of nucleic acid
medicines including small interfering RNA, messenger RNA, replicon
RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics.
Arcturus technologies are covered by its extensive patent portfolio
(154 patents and patent applications, issued in the U.S., Europe,
Japan, China and other countries). 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. and the Cystic Fibrosis Foundation. For more information,
visit www.Arcturusrx.com.
Ultragenyx Forward-Looking StatementsExcept for
the historical information contained herein, the matters set forth
in this press release, including statements related to Ultragenyx's
expectations regarding plans for its clinical programs and clinical
studies, future regulatory interactions, and the components and
timing of regulatory submissions are forward-looking statements
within the meaning of the "safe harbor" provisions of the Private
Securities Litigation Reform Act of 1995. Such forward-looking
statements involve substantial risks and uncertainties that could
cause our clinical development programs, collaboration with, and
investment, in Arcturus, future results, performance or
achievements to differ significantly from those expressed or
implied by the forward-looking statements. Such risks and
uncertainties include, among others, the uncertainties inherent in
the clinical drug development process, such as the regulatory
approval process, the timing of regulatory filings and approvals
(including whether such approvals can be obtained), and other
matters that could affect sufficiency of existing cash, cash
equivalents and short-term investments to fund operations and the
availability or commercial potential of our products and drug
candidates. Ultragenyx undertakes no obligation to update or revise
any forward looking statements. For a further description of the
risks and uncertainties that could cause actual results to differ
from those expressed in these forward-looking statements, as well
as risks relating to the business of Ultragenyx in general, see
Ultragenyx's Quarterly Report filed on Form 10-Q with the
Securities and Exchange Commission on May 7, 2019, and its
subsequent periodic reports filed with the Securities and Exchange
Commission.
Arcturus 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, the likelihood of success of the
Company’s technology or potential development of any products, the
status of the preclinical development program for any of the
clinical development programs of Arcturus, the status of
IND-enabling studies and early clinical development related to any
of the clinical development programs of Arcturus, the sufficiency
of any drug substances or drug products of the Company to meet the
Company’s current clinical goals or expectations, the date that an
IND may be filed with the FDA, the potential market or success for
the clinical development programs of Arcturus, current standards of
care, and the Company’s future cash and financial position 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 such as the
foregoing 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,
inability to generate positive verifiable data, unexpected clinical
results, unforeseen expenses and general market conditions that may
prevent such achievement or performance. 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 10-K for the fiscal
year ended December 31, 2018, filed with the SEC on March 18, 2019
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.
Investor Relations & Media Contacts
Arcturus TherapeuticsNeda
Safarzadeh858-900-2682IR@ArcturusRx.com
Ultragenyx Pharmaceutical, Inc.Danielle
Keatley415-475-6876IR@ultragenyx.com
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