ROCKVILLE, Md., July 31, 2019 /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 it entered into a license agreement with Pfizer
Inc.
Under the terms of the agreement, REGENXBIO has granted Pfizer a
non-exclusive worldwide license, with rights to sublicense, to
REGENXBIO's NAV AAV9 vector for the development and
commercialization of gene therapies for the treatment of
Friedreich's ataxia, the most common hereditary ataxia. In return
for these rights, REGENXBIO will receive an upfront payment, and
has the potential to receive ongoing fees, development and
commercial milestone payments, and royalties on net sales of
products incorporating the licensed intellectual property.
"This license agreement further validates the strength of our
intellectual property portfolio and the potential of NAV AAV9 for
the treatment of systemic and CNS manifestations of movement
disorders," said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. "We are pleased
to establish our relationship with Pfizer as they advance this
program to develop a potential gene therapy treatment for
Friedreich's ataxia."
"We are excited to partner with REGENXBIO on the use of the NAV
AAV9 vector for the treatment of Friedreich's ataxia, a condition
with significant unmet medical need," said Seng Cheng, Senior Vice President and Chief
Scientific Officer of Pfizer's Rare Disease Research Unit. "We
believe the AAV9 vector has the potential to have a profound impact
on patients with severe and debilitating diseases where treatment
options are limited today."
"Friedreich's ataxia is a debilitating, life-shortening,
degenerative neuro-muscular disorder that affects about one in
50,000 people in the United
States, and there are currently no treatments available,"
said Ronald J. Bartek, President,
Director and Co-Founder of the Friedreich's Ataxia Research
Alliance. "We applaud these efforts to develop new potential NAV
Technology-based gene therapies for our under-served patient
community."
About Friedreich's Ataxia
Friedreich's ataxia (FA) is the most common hereditary
ataxia. FA patients have a genetic mutation in the FXN gene,
which limits the production of the protein frataxin, causing a
variety of debilitating symptoms and complications including loss
of coordination and balance, muscle weakness, impaired vision,
hearing and speech, scoliosis, diabetes, and
cardiomyopathy.
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.
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, 2018 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:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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