REGENXBIO Announces Completion of Dosing of First Cohort in Phase I Clinical Trial of RGX-314 Gene Therapy for Wet AMD
September 20 2017 - 7:00AM
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 announced that the first cohort of six patients has
been dosed in a Phase I clinical trial evaluating RGX-314 for
patients with wet age-related macular degeneration (wet AMD).
“Dosing of the first six patients in the Phase I clinical trial
was completed at three of the multi-center trial’s study sites,”
said Stephen Yoo, M.D., Chief Medical Officer of REGENXBIO. “All
six study sites are now active to support enrollment in the next
dose cohort that is projected to start after review by the Data
Safety Monitoring Board this month. Our goal with this trial is to
confirm prior observations in preclinical animal models that
treatment with RGX-314 can deliver rapid and sustained therapeutic
effects to provide long-lasting treatment for patients with wet
AMD.”
Six leading retinal surgery centers across the United States are
participating in the Phase I trial of RGX-314. This multi-center,
open-label, multiple-cohort, dose-escalation clinical trial is
designed to assess the safety and tolerability of RGX-314 as a
one-time therapy for patients with previously treated wet AMD. For
further details on the trial, enrollment criteria and eligibility,
please contact patientadvocacy@regenxbio.com or visit
https://clinicaltrials.gov/ct2/show/NCT03066258.
REGENXBIO plans to share an interim trial update 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 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). 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 subretinal
delivery. Primary endpoints include safety and tolerability and
secondary endpoints include ocular examinations and imaging
(including BCVA and SD‑OCT) and the need for additional anti-VEGF
therapy. Following completion of the primary study period, subjects
will enter a follow-up period 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 subretinal 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 subretinal 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 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 research,
development and regulatory plans in connection with its NAV
Technology Platform and gene therapy treatments. 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.
CONTACTS:
Investors Heather Savelle, 617-340-6072
heather@argotpartners.com
Media Adam Pawluk, 202-591-4063
apawluk@jpa.com
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