- 85 subjects dosed across five cohorts in
AAVIATE® trial
evaluating RGX-314 for the treatment of wet AMD using
suprachoroidal delivery
- Company expects to report additional suprachoroidal data
later this year
ROCKVILLE, Md., July 8, 2022
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it
has completed enrollment in Cohort 5 of the Phase II
AAVIATE® trial of RGX-314 for the treatment of wet
age-related macular degeneration (wet AMD) using in-office
suprachoroidal delivery.
"We are encouraged by the excellent progress we have made
advancing RGX-314 suprachoroidal delivery in both our AAVIATE and
ALTITUDE™ trials for the treatment of wet AMD and diabetic
retinopathy," said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. "RGX-314, has
the potential through delivery to the suprachoroidal space to offer
the millions of patients facing vision loss from these retinal
diseases with a one-time, in-office treatment option. We plan to
present additional suprachoroidal data this year."
Results from Cohort 1 of the Phase II ALTITUDE trial of RGX-314
for the treatment of diabetic retinopathy were presented at the
Angiogenesis, Exudation, and Degeneration 2022 conference and will
be encored at the upcoming American Society of Retina Specialists
Annual Meeting.
AAVIATE Clinical Trial
The multi-center, open-label, randomized, active-controlled,
dose-escalation Phase II AAVIATE trial is evaluating the efficacy,
safety and tolerability of suprachoroidal delivery of RGX-314 in
patients with wet AMD using the Clearside SCS Microinjector®.
Twenty patients in Cohort 1 were randomized to receive RGX-314 at a
dose level of 2.5x1011 genomic copies per eye (GC/eye)
through one injection versus monthly 0.5 mg ranibizumab
intravitreal injection at a 3:1 ratio. Twenty patients in Cohort 2
were randomized to receive RGX-314 at a dose level of
5x1011 GC/eye through two injections versus monthly 0.5
mg ranibizumab intravitreal injection at a 3:1 ratio. Cohort 3 is
evaluating RGX-314 at the same dose level as Cohort 2 in 20
patients who are neutralizing antibody (NAb) positive. Cohort 4 is
evaluating RGX-314 in 15 patients at a dose level of
1x1012 GC/eye and Cohort 5 is evaluating the same dose
level of RGX-314 in 20 patients who are NAb positive. Patients in
these cohorts did not receive prophylactic immune suppressive
corticosteroids before or after administration of RGX-314.
About RGX-314
RGX-314, being developed in collaboration with AbbVie, is being
investigated 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 research in 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 repeated intraocular
injections, 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,
including late-stage and commercial programs, in multiple
therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to
progress five AAV Therapeutics from our internal pipeline and
licensed programs into pivotal-stage or commercial products by
2025.
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," "assume," "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, 2021, 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. Except as required by law,
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 trademark of Clearside Biomedical, Inc.
All other trademarks referenced herein are registered trademarks of
REGENXBIO.
Contacts:
Dana
Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.brinzey@westwicke.com
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SOURCE REGENXBIO Inc.