Full Press Release Details
REGENXBIO Provides Year-End 2016 Corporate Update
ROCKVILLE, Md., January 6, 2017 REGENXBIO Inc. (Nasdaq: RGNX), a leading biotechnology company focused on the development,
commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy based on its proprietary NAV Technology Platform, today provided a year-end 2016 corporate update.
In 2016, REGENXBIO saw substantial progress in meeting the development goals for our lead product candidate programs and building our core
organization; we expect this to continue in 2017 as we work toward generating a robust clinical pipeline of product candidates that hold the potential to improve treatment in many diseases. This year we anticipate clinical data reporting from
programs using our NAV Technology, including our trials of RGX-314 for the treatment of wet age-related macular degeneration, RGX-501 for the treatment of homozygous familial hypercholesterolemia, and trials from our licensees AveXis, Dimension and
Audentes, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. In addition, we expect several new INDs for NAV Technology programs to be filed in 2017, further expanding the clinical pipeline of NAV-based gene
therapies that have the potential to benefit patients in need.
Year-End 2016 Highlights
Recent highlights related to REGENXBIO s internal lead product candidate programs include:
Anticipated 2017 Milestones
REGENXBIO expects to meet
the following anticipated milestones related to the clinical development of internal lead product candidate programs:
NAV Technology Licensee Program Highlights
December 31, 2016, REGENXBIO s NAV Technology Platform was being applied in more than 20 partnered product candidates developed by nine NAV Technology Platform Licensees (NAV Licensees). There were a number of NAV Licensee advancements
announced in 2016, which included:
Clinical Development of NAV Technology Platform
Expansion of NAV Technology Platform
2017 Financial Guidance
As of December 31, 2016, REGENXBIO had more than $155 million in cash, cash equivalents and marketable securities, completing 2016 within the previously
updated cash burn guidance range of between $55 million and $60 million. REGENXBIO expects full-year 2017 cash burn to be between $75 million and $85 million, which will support the continued development of its lead product candidate programs.
REGENXBIO is a leading
biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. REGENXBIO s NAV Technology Platform, a
proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO s mission is to transform the lives of patients suffering from severe diseases with
significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts
and third-party NAV Technology Platform Licensees. REGENXBIO and its 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 contains
forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, REGENXBIO s NAV Technology Platform, REGENXBIO s collaborations with its NAV Technology
Licensees, REGENXBIO s financial guidance, REGENXBIO s research, development and regulatory plans for RGX-111, RGX-121, RGX-314, RGX-501 and other gene therapies and the research, development and regulatory plans of REGENXBIO s NAV
Technology Licensees. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking
statements. All of REGENXBIO s development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Meaningful factors
which could cause actual results to differ include, but are not limited to, the timing of enrollment, commencement and completion of REGENXBIO s clinical trials; the timing and success of preclinical studies and clinical trials conducted by
REGENXBIO, its development partners and its NAV Technology Licensees; the ability to obtain and maintain regulatory approval to conduct clinical trials and to commercialize REGENXBIO s product candidates, and the labeling for any approved
products; the scope, progress, expansion, and costs of developing and commercializing REGENXBIO s product candidates; REGENXBIO s ability to obtain and maintain intellectual property protection for REGENXBIO s product candidates and
technology; trends and challenges in REGENXBIO s business and the markets in which REGENXBIO operates; REGENXBIO s ability to attract or retain key personnel; the size and growth of the potential markets for REGENXBIO s product
candidates and the ability to serve those markets; the rate and degree of market acceptance of any of REGENXBIO s product candidates; REGENXBIO s ability to establish and maintain development partnerships, including those with NAV
Technology Licensees; REGENXBIO s expenses and revenue, the sufficiency of REGENXBIO s cash resources and needs for additional financing, regulatory developments in the United States and foreign countries, as well as other factors
discussed in 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, 2015
and Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which are available on the SEC s website at www.sec.gov. Additional factors may be set forth in those sections of REGENXBIO s Annual Report on Form 10-K for
the year ended December 31, 2016, to be filed in the first quarter of 2017. In addition to the risks described above and in REGENXBIO s filings with the SEC, other unknown or unpredictable factors also could affect REGENXBIO s
results. There can be no assurance that the actual results or developments anticipated by REGENXBIO will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, REGENXBIO. Therefore, no
assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.
All forward-looking statements
contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. REGENXBIO cautions investors not to rely too heavily on the forward-looking statements REGENXBIO makes or that are made on its
behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). REGENXBIO undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such
forward-looking statements, whether as a result of new information, future events or otherwise.
Heather Savelle, 646-395-3734
Laura Bagby, 312-448-8098