ROCKVILLE, Md. and NOVATO, Calif., March
31, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:RGNX),
a leading clinical-stage biotechnology company seeking to improve
lives through the curative potential of gene therapy based on its
proprietary NAV Technology Platform, and Ultragenyx Pharmaceutical
Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the
development and commercialization of novel products for serious
rare and ultra-rare diseases, today announced a new exclusive,
worldwide license agreement, extending the companies' existing gene
therapy partnership.
Under the terms of the agreement, REGENXBIO has granted
Ultragenyx an exclusive, worldwide license, with rights to
sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the
development and commercialization of gene therapy treatments for a
rare metabolic disorder. In return for these rights, REGENXBIO will
receive an upfront payment of $7
million, ongoing fees, milestone payments, and royalties on net sales of products
incorporating the licensed intellectual property.
"We are pleased to expand our long-standing relationship with
Ultragenyx through this new license agreement, which will enable
Ultragenyx to apply our proprietary NAV Vectors to the development
of a new gene therapy for rare diseases," said Kenneth T. Mills, President and Chief Executive
Officer of REGENXBIO. "Today's announcement provides further
validation of the breadth and depth of our intellectual property
portfolio, and we remain committed to working closely with leading
companies to enable the development of important gene therapy
programs."
"This latest license agreement reinforces Ultragenyx's
commitment to developing gene therapies for multiple rare diseases
and adds a new indication to our relationship with REGENXBIO," said
Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of
Ultragenyx. "
Ultragenyx is a longstanding licensee partner of REGENXBIO. In
addition to today's agreement, Ultragenyx has in-licensed NAV
Vectors for the development of gene therapies to treat Ornithine
Transcarbamylase (OTC) Deficiency, Glycogen Storage Disease Type Ia
(GSDIa), CDKL5 Deficiency, Hemophilia A, and Wilson Disease.
About REGENXBIO
Inc.
REGENXBIO is a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy. REGENXBIO's NAV Technology Platform, a proprietary
adeno-associated virus (AAV) gene delivery platform, consists of
exclusive rights to more than 100 novel AAV vectors, including
AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV
Technology Platform Licensees are applying the NAV Technology
Platform in the development of a broad pipeline of candidates in
multiple therapeutic areas.
About Ultragenyx
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.
REGENXBIO Forward-Looking Statements
This press release includes "forward-looking statements," within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. These statements express a belief, expectation or
intention and are generally accompanied by words that convey
projected future events or outcomes such as "believe," "may,"
"will," "estimate," "continue," "anticipate," "design," "intend,"
"expect," "could," "plan," "potential," "predict," "seek,"
"should," "would" or by variations of such words or by similar
expressions. The forward-looking statements include statements
relating to, among other things, REGENXBIO's future operations and
clinical trials. REGENXBIO has based these forward-looking
statements on its current expectations and assumptions and analyses
made by REGENXBIO in light of its experience and its perception of
historical trends, current conditions and expected future
developments, as well as other factors REGENXBIO believes are
appropriate under the circumstances. However, whether actual
results and developments will conform with REGENXBIO's expectations
and predictions is subject to a number of risks and uncertainties,
including the timing of enrollment, commencement and completion and
the success of clinical trials conducted by REGENXBIO, its
licensees and its partners, the timing of commencement and
completion and the success of preclinical studies conducted by
REGENXBIO and its development partners, the timely development and
launch of new products, the ability to obtain and maintain
regulatory approval of product candidates, the ability to obtain
and maintain intellectual property protection for product
candidates and technology, trends and challenges in the business
and markets in which REGENXBIO operates, the size and growth of
potential markets for product candidates and the ability to serve
those markets, the rate and degree of acceptance of product
candidates, and other factors, many of which are beyond the control
of REGENXBIO. Refer to the "Risk Factors" and "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" sections of REGENXBIO's Annual Report on Form 10-K for
the year ended December 31, 2019, and
comparable "risk factors" sections of REGENXBIO's Quarterly Reports
on Form 10-Q and other filings, which have been filed with the U.S.
Securities and Exchange Commission (SEC) and are available on the
SEC's website at www.sec.gov. All of the forward-looking statements
made in this press release are expressly qualified by the
cautionary statements contained or referred to herein. The actual
results or developments anticipated may not be realized or, even if
substantially realized, they may not have the expected consequences
to or effects on REGENXBIO or its businesses or operations. Such
statements are not guarantees of future performance and actual
results or developments may differ materially from those projected
in the forward-looking statements. Readers are cautioned not to
rely too heavily on the forward-looking statements contained in
this press release. These forward-looking statements speak only as
of the date of this press release. REGENXBIO does not undertake any
obligation, and specifically declines any obligation, to update or
revise any forward-looking statements, whether as a result of
new information, future events or otherwise.
Ultragenyx Forward-Looking Statements
Except 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 third parties, 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 Annual Report filed on Form 10-K with the Securities
and Exchange Commission on February 14,
2020, and its subsequent periodic reports filed with the
Securities and Exchange Commission.
REGENXBIO Contacts:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
Ultragenyx Contacts:
Investors & Media
Danielle Keatley
415-475-6876
dkeatley@ultragenyx.com
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