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 announced the
Investigational New Drug application (IND) is active for the
planned multi-center, open-label, multiple-cohort, dose-escalation
Phase I clinical trial of RGX-314 for the treatment of wet
age-related macular degeneration (wet AMD).
“The goal of the RGX-314 program is to develop a single-dose
treatment for wet AMD that prevents future disease recurrence while
reducing or eliminating the need for regular injections that are
the current standard of care in wet AMD,” said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. “We are on
track to meet our next program objectives for RGX-314, beginning
with trial enrollment by mid-2017 and an interim trial update by
the end of the year, and we look forward to working with leading
U.S. researchers and retina surgeons on this novel clinical
program.”
RGX-314 is being developed under a multi-institutional
collaboration with world-renowned gene therapy and ophthalmology
experts James M. Wilson, M.D., Ph.D., Jean Bennett, M.D., Ph.D. and
Albert Maguire, M.D. from the University of Pennsylvania’s Gene
Therapy Program and Center for Advanced Retinal and Ocular
Therapeutics (Penn), respectively, and Peter Campochiaro, M.D. at
the Johns Hopkins Wilmer Eye Institute (Johns Hopkins).
“In animal studies, treatment with RGX-314 gene therapy led to
rapid and sustained anti-VEGF protein detected in the eyes of
treated animals. Preclinical studies have shown anti-VEGF mRNA and
protein distributed widely throughout the retina. This high protein
expression observed using RGX-314’s NAV AAV8 vector may make this
approach suitable for an ocular therapeutic in wet AMD,” said Dr.
Maguire.
Six leading retinal surgery centers across the United States,
including Penn and Johns Hopkins, are expected to participate in
the Phase I trial of RGX-314.
About the Phase I Clinical Trial of RGX-314
RGX‑314 will be evaluated in a Phase I, multi-center,
open-label, multiple-cohort, dose‑escalation study in adult
subjects with wet AMD in the United States. The study is expected
to 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 is designed to 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 of RGX-314 administered by subretinal
delivery. Following completion of the primary study period, it is
expected that subjects will enter the follow-up period and will
continue to be assessed until week 106 to assess long term
safety and durability of effect.
About Wet AMD
Wet AMD is characterized by loss of vision due to excess blood
vessel formation between two layers of cells in the retina, which
results in fluid leakage that can result in physical changes in the
structure of the retina and changes in vision. Wet AMD is a leading
cause of total and partial vision loss in the United States, Europe
and Japan and there may be over two million individuals 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 halt or
significantly impede the loss of vision in the majority of patients
with wet AMD. All of 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 vision loss with reduced frequency of
treatment.
About RGX-314
RGX-314 is being developed as a novel, one-time subretinal
treatment for wet AMD that includes the NAV AAV8 vector encoding a
gene for a monoclonal antibody fragment. The expressed protein is
designed to neutralize VEGF activity, modifying the pathway for
formation of new leaky blood vessels and retinal fluid
accumulation. 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 subretinal dose of RGX-314.
About REGENXBIO Inc.
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.
Note Regarding Penn
Penn has licensed certain Penn-owned AAV technologies to
REGENXBIO, including rights related to RGX-314. Dr. Wilson is an
advisor to REGENXBIO, and is a founder of, holds equity in, and
receives grants from REGENXBIO.
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 and objectives for its RGX-314
program, including REGENXBIO’s Phase I clinical trial of RGX-314.
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, including for RGX-314, 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 to conduct
clinical trials and to commercialize REGENXBIO’s product
candidates, the labeling for any approved products; the scope,
progress, expansion, and costs of developing and commercializing
REGENXBIO’s product candidates; competitive products; 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; 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.
CONTACT:
Investors
Heather Savelle, 646-395-3734
heather@argotpartners.com
Media
Laura Bagby, 312-448-8098
lbagby@6degreespr.com
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