ROCKVILLE, Md., Sept. 9, 2020 /PRNewswire/ -- 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 patient has been dosed in
the AAVIATE trial, a Phase II trial to evaluate the suprachoroidal
delivery of RGX-314 using the SCS Microinjector for the
treatment of wet age-related macular degeneration (wet AMD). The
Company expects to report interim data from the first cohort of
this trial by the end of 2020.
"The initiation of this trial is an important milestone for
REGENXBIO, as it is the first clinical trial to evaluate the
delivery of any gene therapy, including RGX-314, to the
suprachoroidal space. RGX-314 has shown evidence of potentially
meaningful treatment effect in patients with wet AMD when delivered
subretinally in the ongoing Phase I/IIa trial, and we are excited
about the potential of the targeted, in-office suprachoroidal
approach, which may provide additional RGX-314 delivery options for
patients. We look forward to providing additional updates from this
trial later this year," said Steve
Pakola, M.D., Chief Medical Officer of REGENXBIO.
AAVIATE is a multi-center, open-label, randomized,
active-controlled, dose-escalation trial that will evaluate the
efficacy, safety and tolerability of suprachoroidal delivery of
RGX-314 using the SCS Microinjector, a targeted, in-office route of
administration. The trial is expected to enroll approximately 40
patients with severe wet AMD across two cohorts. Patients in each
cohort will be randomized to receive RGX-314 versus monthly 0.5 mg
ranibizumab intravitreal injection at a 3:1 ratio, and two dose
levels of RGX-314 will be evaluated: 2.5x1011 GC/eye and
5x1011 GC/eye. Patients will not receive prophylactic
immune suppressive corticosteroid therapy before or after
administration of RGX-314.
The primary endpoint of the trial is mean change in vision in
patients dosed with RGX-314, as measured by best corrected visual
acuity (BCVA), at Week 40 from baseline, compared to patients
receiving monthly injections of ranibizumab. Other endpoints
include mean change in central retinal thickness (CRT) and number
of anti-VEGF intravitreal injections received following
administration of RGX-314.
About RGX-314
RGX-314 is being developed as a potential one-time treatment for
wet AMD, diabetic retinopathy, and other chronic retinal
conditions. RGX-314 consists of the NAV AAV8 vector, which encodes
an antibody fragment designed to inhibit vascular endothelial
growth factor (VEGF). RGX-314 is believed to inhibit the VEGF
pathway by which new, leaky blood vessels grow and contribute to
the accumulation of fluid in the retina.
REGENXBIO is advancing two separate routes of administration of
RGX-314 to the eye, through a standardized subretinal delivery
procedure as well as delivery to the suprachoroidal space.
REGENXBIO has licensed certain exclusive rights to the SCS
Microinjector® from Clearside Biomedical, Inc. to
deliver gene therapy treatments to the suprachoroidal space of the
eye.
About Wet AMD
Wet AMD is characterized by loss of vision due to new, leaky
blood vessel formation in the retina. Wet AMD is a significant
cause of vision loss in the United
States, Europe and
Japan, with up to 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
prevent progression of vision loss in the majority of patients.
These therapies, however, require life-long intraocular injections,
typically repeated every four to 12 weeks in frequency, to maintain
efficacy. Due to the burden of treatment, patients often
experience a decline in vision with reduced frequency of treatment
over time.
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 and
clinical trials. 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, the impact of the COVID-19 pandemic or similar public
health crises on REGENXBIO's business, 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, 2019, 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.
SCS Microinjector® is a registered trademark of
Clearside Biomedical, Inc. All other trademarks referenced herein
are registered trademarks of REGENXBIO.
Contacts:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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