REGENXBIO to Present on RGX-314 Gene Therapy Program for Wet AMD at 35th Annual Scientific Meeting of the American Society of...
August 11 2017 - 6:14PM
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, will present at the 35th Annual Scientific Meeting of the
American Society of Retina Specialists (ASRS) in Boston, MA.
“RGX-314 gene therapy is designed to be a one-time wet AMD
treatment administered subretinally which may result in a sustained
local production of an anti-VEGF antibody fragment and potentially
alleviate the burden of frequent anti-VEGF administration,” said
Stephen Yoo, M.D., Chief Medical Officer of REGENXBIO. “We are
presenting details about the ongoing Phase I study design and
subretinal delivery of RGX-314.”
Details of the presentation follow below:
Title: RGX-314 Gene Therapy Subretinal
Delivery for the Treatment of Neovascular Age-Related Macular
Degeneration Presenter: Jeffrey S. Heier,
M.D., Co-president and Medical Director and Director of Retina
Research at Ophthalmic Consultants of BostonSession
date/time: Saturday, August 12, 2017, 8:50 a.m. EDT
Session title: AMD 1 Symposium
Room: Hynes Convention Center, Boston, MA
Six leading retinal surgery centers across the United States are
participating in the Phase I trial of RGX-314. For further details
on the trial, enrollment criteria and eligibility, please contact
patientadvocacy@regenxbio.com or visit
https://clinicaltrials.gov/ct2/show/NCT03066258.
Patient recruitment and enrollment is continuing and REGENXBIO
expects to provide an interim update from the RGX-314 clinical
trial by the end of 2017.
About the Phase I Clinical Trial of RGX-314
RGX‑314 is currently being evaluated in a Phase I,
multi-center, open-label, multiple-cohort, dose‑escalation study in
adult subjects with wet age-related macular degeneration (wet AMD)
in the United States. The study will include approximately eighteen
previously treated wet AMD subjects that are responsive to
anti-vascular endothelial growth factor (anti-VEGF) therapy and are
50 years of age or older. The study will evaluate three doses of
RGX-314 (3 × 10^9 genome copies (GC)/eye,
1 × 10^10 GC/eye, and 6 × 10^10 GC/eye).
Primary endpoints include adverse events, certain laboratory
measures (including immunological parameters) and ocular
examinations and imaging (including BCVA and SD‑OCT). The primary
purpose of the clinical study is to evaluate the safety and
tolerability of RGX-314 at 24 weeks after a single dose
administered by sub-retinal delivery. Following completion of the
primary study period, subjects will enter a follow-up evaluation
and will continue to be assessed until week 106 for long term
safety and durability of effect.
About RGX-314
RGX-314 is being developed as a one-time sub-retinal treatment
for wet AMD. It includes the NAV AAV8 vector encoding an antibody
fragment which inhibits VEGF, modifying the pathway for formation
of new leaky blood vessels which lead to retinal fluid accumulation
and vision loss. In preclinical animal models with conditions
similar to macular degeneration, significant and dose-dependent
reduction of blood vessel growth and prevention of disease
progression was observed after a single sub-retinal dose of
RGX-314.
About Wet AMD
Wet AMD is characterized by loss of vision due to new leaky
blood vessel formation in the retina. This results in fluid leakage
that can manifest in physical changes in the structure of the
retina and loss of vision. Wet AMD is a significant cause of vision
loss in the United States, Europe and Japan.
There may be more than 2 million people living with wet AMD in
these geographies alone.
Current anti-VEGF therapies have significantly changed the
landscape for treatment of wet AMD, becoming the standard of care
due to their ability to improve vision and retinal fluid in the
majority of patients. These therapies, however, require repetitive
and inconvenient intraocular injections, typically ranging from
every four to eight weeks in frequency, to maintain efficacy.
Patients often experience a decline in the initial vision gain from
therapy with reduced frequency of treatment over time.
About REGENXBIO
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 Licensees are applying the NAV
Technology Platform in the development of a broad pipeline of
product 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 research,
development and regulatory plans in connection with its NAV
Technology Platform and gene therapy treatments. 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 and its development partners; 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 REGENXBIO’s product
candidates and technology; REGENXBIO’s growth strategies;
REGENXBIO’s competition; 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;
REGENXBIO’s expenses and revenue; regulatory developments
in the United States and foreign countries; the sufficiency of
REGENXBIO’s cash resources and needs for additional financing; and
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, 2016 and Quarterly Report on Form 10-Q
for the quarter ended June 30, 2017, which have been filed with the
Securities and Exchange Commission (SEC) and are available on the
SEC’s website at www.sec.gov. 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.
CONTACT:
Investors
Heather Savelle, 617-340-6072
heather@argotpartners.com
Media
Adam Pawluk, 202-591-4063
apawluk@jpa.com
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