Full Press Release Details
REGENXBIO Provides Year-End 2015 Corporate Update
January 7, 2016 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 2015 corporate update.
We enter 2016 with great momentum and a strong balance sheet that will
enable us to continue to advance our clinical and corporate objectives, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. As of December 31, 2015, our lead product candidate programs remain on track and are
expected to enter clinical phase, and we ve expanded the breadth and value of our NAV Technology Platform with the sublicensing of new programs. We ve also grown our team in support of REGENXBIO s mission of developing and
commercializing gene therapy products that transform the lives of patients suffering from severe diseases.
Year-End 2015 Highlights
Recent highlights related to REGENXBIO s internal lead product candidate programs and the sublicensing of its NAV Technology Platform include:
Anticipated 2016 Milestones
Anticipated milestones related to the clinical development of REGENXBIO s internal lead product candidate programs include:
2016 Financial Guidance
As of December 31, 2015,
REGENXBIO had more than $210 million in cash, cash equivalents and marketable securities. In 2016, REGENXBIO currently expects its cash burn to be between $60 million and $70 million, which will support the development of its lead product candidate
programs and the anticipated growth of its internal pipeline.
Management Team Appointments
As of December 31, 2015, REGENXBIO had 37 full-time employees, including three recent management team appointments dedicated to the execution of
REGENXBIO s lead programs: Mari Maurer, Vice President of Clinical Operations; Lynne Fahey McGrath, M.P.H., Ph.D., Vice President of Regulatory Affairs; and Jay Vora, Ph.D., Vice President of Program Management.
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. As of December 31, 2015, REGENXBIO s NAV Technology Platform is being applied in the
development of 28 product candidates for a variety of diseases, including five internally developed candidates and 23 partnered candidates developed by REGENXBIO s licensees.
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 expected cash burn for 2016, REGENXBIO s research, development and
regulatory plans for RGX-111, RGX-314, RGX-501 and other gene therapies. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of
them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the ability to obtain and maintain
regulatory approval of 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 our product candidates; REGENXBIO s ability to establish and maintain development
partnerships; REGENXBIO s expectations regarding federal, state and foreign regulatory requirements; 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 prospectus dated September 16, 2015, filed pursuant to Rule 424(b)(4) under the
Securities Act of 1933, as amended, with the Securities and Exchange Commission (SEC) on September 17, 2015 (the Prospectus) and Quarterly Report on Form 10-Q for the quarter ended September 30, 2015, each available on
the SEC s website at www.sec.gov. Additional factors may be described in those sections of REGENXBIO s Annual Report on Form 10-K for the year ended December 31, 2015, to be filed with the SEC in
the first quarter of 2016. In addition to the risks described above and in the Prospectus, Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other 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
Elizabeth Broder, 646-378-2945
Laura Bagby, 312-448-8098