Full Press Release Details
REGENXBIO Reports Continued Progress Across Programs in Year-End
2018 Corporate Update
ROCKVILLE, Md., Jan. 03, 2019 (GLOBE NEWSWIRE) 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 provided a year-end 2018 corporate
2018 was a pivotal year for the advancement and validation of the NAV Technology Platform across many diseases, as we achieved significant
clinical and regulatory progress for our proprietary product candidates, as well as across our licensee network. In 2018, we received over $200 million from our partners, and we believe our strong financial position will enable us to execute on
our corporate goals throughout 2019, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. This year, a key focus will be the acceleration and broadening of our ophthalmology franchise as we build on the promising
results observed to date in our ongoing Phase I clinical trial evaluating RGX-314 for wet age-related macular degeneration (wet AMD). Through the immediate expansion of
this trial into a Phase IIa clinical trial, we re committed to expediting the development of RGX-314 as a one-time therapy, not only in the hopes of providing a
potential long-lasting treatment solution for the wet AMD patient population, but also to potentially address substantial unmet needs in a broad range of other retinal conditions.
Mr. Mills continued: Another key upcoming milestone for the NAV Technology Platform is the potential approval of Novartis ZOLGENSMA by the
FDA for the treatment of SMA Type I, anticipated in May 2019. This approval would be the first for a NAV Technology-based therapy, and it would provide a post-launch commercial revenue stream to REGENXBIO. In combination with our innovative
technology platform, robust internal clinical pipeline and broad licensee network, we are positioned for a transformative 2019 as we accelerate our progress in realizing the curative potential of gene therapy.
Lead Product Candidate Updates
Anticipated 2019 Milestones
REGENXBIO expects to
meet the following anticipated milestones related to the clinical development of internal lead product candidates in 2019:
NAV Technology Licensee Program Highlights
December 31, 2018, REGENXBIO s NAV Technology Platform was being applied in more than 20 partnered product candidates in development by NAV Technology Licensees. Thirteen of these partnered product candidates are in active clinical
development, and one partnered product candidate has been
submitted for Biologics License Application (BLA) approval with the FDA. Many NAV Technology Licensee advancements and achievements were announced in 2018, including:
Acquisition of AveXis by Novartis
Advancement and Commercialization of NAV Technology Licensee Clinical Programs
Expansion of NAV Technology Licensees
As of December 31, 2018, REGENXBIO had more than $470 million in cash, cash equivalents and marketable securities. Based on its
current operating plan, and excluding any commercial revenue from ZOLGENSMA, REGENXBIO expects that its balance in cash, cash equivalents and marketable securities will be between $330 million and $350 million at the end of
2019, which will be used to support the continued development of its lead product candidate programs. Importantly, REGENXBIO anticipates adding commercial revenue from ZOLGENSMA to its existing base of partner revenue this year, pending FDA
REGENXBIO management will present a company overview detailing these goals and guidance at the 37th Annual J.P Morgan Healthcare Conference on Wednesday, January 9, 2019 at 4:30 p.m. PT. A live webcast of the presentation can be accessed in the Investors section of the REGENXBIO website at
www.regenxbio.com. An archived replay of the webcast will be available on the same website for approximately 30 days following the presentation.
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,
clinical trials, costs 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.
Natalie Wildenradt, 646-681-8192
Adam Pawluk, 202-591-4063