ROCKVILLE, Md., Oct. 27, 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, today announced that it will receive a milestone payment
of $80.0 million from Novartis AG
based upon the achievement of $1.0
billion in cumulative net sales of Zolgensma. REGENXBIO
expects to recognize this revenue in the third quarter of 2020.
"Zolgensma is a transformative, one-time gene therapy for the
treatment of pediatric patients with SMA, a debilitating and
potentially deadly disease, and the first approved gene therapy
based on REGENXBIO's NAV Technology. We believe that this
achievement provides further validation of the broad potential of
NAV Technology to improve the lives of patients in need," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "We are pleased to have
contributed to a therapy that has impacted the lives of over 700
patients with SMA and their families around the world."
Zolgensma uses REGENXBIO's proprietary NAV AAV9 vector and was
designed and developed by AveXis, Inc., now known as Novartis Gene
Therapies. In May 2019, Zolgensma was
granted U.S. Food and Drug Administration (FDA) approval for the
treatment of spinal muscular atrophy (SMA) in pediatric patients
who are less than two years of age, and has since been approved in
Europe, Japan and other countries. REGENXBIO continues
to receive tiered royalties on sales of Zolgensma. Since FDA
approval in 2019, REGENXBIO has earned more than $140.0 million in revenues from royalties and
commercial milestone payments related to Zolgensma through the
third quarter of 2020.
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 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, the impact of the
COVID-19 pandemic or similar public health crises on REGENXBIO's
business, 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.
Zolgensma® is a registered trademark of Novartis Gene Therapies.
All other trademarks referenced herein are registered trademarks of
REGENXBIO.
Contacts:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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SOURCE REGENXBIO Inc.