Full Press Release Details
REGENXBIO Provides Year-End 2017 Corporate Update
- Initiated third cohort dosing of RGX-314 Phase I clinical trial for wet AMD -
- Initiated second cohort dosing of RGX-501 Phase I/II clinical trial for HoFH -
- Anticipate completing dosing and presenting topline data from RGX-314 and RGX-501 trials in late 2018 -
- Anticipate initiating dosing in clinical trials for MPS I and MPS
II in first half 2018 -
- Ended 2017 with greater than $175 million in cash, cash equivalents and marketable securities -
ROCKVILLE, Md., January 4, 2018 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 2017 corporate update.
In 2017, REGENXBIO achieved our key development goals for our lead product
candidate programs and expanded our research and development organization and capabilities. We expect to build upon this success in 2018 as we work toward advancing a robust clinical pipeline of product candidates that hold the potential to improve
treatment in many diseases, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. This coming year, we anticipate substantial clinical data reporting from programs using our NAV Technology Platform, including topline
data from 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 Audentes, Shire and Ultragenyx, In addition, we expect several new INDs for NAV Technology Licensee programs to be filed in 2018, as well as progress updates from AveXis on the
continued enrollment of their pivotal trial for SMA Type 1, which uses the NAV AAV9 vector.
Lead Product Candidate Clinical Trial Updates
Other Operational Highlights
In 2017, REGENXBIO invested in internal capabilities, expanded and enhanced its research and development organization including the addition of Olivier
Danos, Ph.D., as Chief Scientific Officer and initiated the build-out of a
state-of-the-art research and development facility, which is expected to be completed in the first half of 2018.
Anticipated 2018 Milestones
REGENXBIO expects to meet
the following anticipated milestones related to the clinical development of internal lead product candidate programs:
In addition, REGENXBIO expects to
announce the designation of a new pipeline lead program candidate in the second half of 2018.
NAV Technology Licensee Program Highlights
As of December 31, 2017, REGENXBIO s NAV Technology Platform was being applied in more than 20 partnered product candidates developed by 10 NAV
Technology Platform Licensees (NAV Technology Licensees). There were a number of NAV Technology Licensee advancements announced in 2017, which included:
Clinical Development of NAV Technology Platform
Expansion of NAV Technology Platform
As of December 31, 2017, REGENXBIO had more than $175 million in cash, cash equivalents and marketable securities. REGENXBIO s cash burn in 2017
was approximately $64 million, which is below the previously updated cash burn guidance range of between $75 million and $80 million, excluding the effect of REGENXBIO s underwritten public offering of common stock in March 2017,
which resulted in aggregate net proceeds to REGENXBIO of approximately $81.5 million after deducting underwriting discounts and commissions and offering expenses. REGENXBIO expects full-year 2018 cash burn to be between $85 million and
$95 million, which will support the continued development of its lead product candidate programs.
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.
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 of REGENXBIO s clinical trials; the timing and success of preclinical studies and clinical trials 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, 2016 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.