ROCKVILLE, Md., Oct. 22, 2018 /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, today announced that
Ultragenyx Pharmaceutical Inc. has exercised an option for an
exclusive worldwide license to REGENXBIO's NAV Vectors, including
NAV AAV9, for the treatment of CDKL5 Deficiency Disorder (CDD).
Under the terms of the license, REGENXBIO will receive an
upfront payment, ongoing fees, milestone payments and royalties on
net sales of products incorporating NAV Vectors. The option
exercised by Ultragenyx is the final remaining disease indication
option under the 2015 license agreement between REGENXBIO and
Dimension Therapeutics, Inc., a wholly owned subsidiary of
Ultragenyx. CDD is a severe and debilitating neurological disorder
that shares many features of Rett Syndrome, though the two
disorders are now considered distinct from each other.
"We are looking forward to initiating this program in CDD, a
disease with increasing awareness and diagnosis," said Emil D.
Kakkis, M.D., Ph.D., Chief Executive Officer and President of
Ultragenyx. "AAV9 has been shown to be effective for gene therapy
delivery to the central nervous system, and we believe it is
well-suited for this neurological indication."
Dr. Kakkis, will present the Keynote Address on Tuesday, October 23 at the 2018 CDKL5 Forum
hosted by the Loulou Foundation.
"This license for CDD further validates the NAV Technology
Platform for use in development of treatments for disorders of the
central nervous system," said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO.
"We are pleased to expand our partnership with Ultragenyx with this
new license, and to continue to enable their commitment to develop
gene therapy treatments for serious rare and ultra-rare genetic
diseases."
About CDD
CDD is an X-linked genetic disorder that
results in seizures that typically begin in the first few months of
life and severe intellectual and gross motor impairment. The CDKL5
gene provides instructions for making a protein that is essential
for normal brain development, with mutations causing a deficiency
in the protein level. CDD is caused by a deficiency in an enzyme
that may only be needed in small quantities for the brain to
function normally. In the past, CDD was commonly diagnosed as an
atypical Rett Syndrome until genetic diagnostics changed this view
and improved accurate diagnosis.
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 Pharmaceutical Inc.
Ultragenyx is a
biopharmaceutical company committed to bringing to 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 no approved therapies.
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.
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
cash flow. 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, 2017 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.
CONTACT:
Investors
Natalie
Wildenradt, 646-681-8192
natalie@argotpartners.com
Media
Adam Pawluk,
202-591-4063
apawluk@jpa.com
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