ROCKVILLE, Md., Aug. 25, 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 the clearance of the Investigational New
Drug (IND) application by the U.S. Food and Drug Administration
(FDA) to evaluate the suprachoroidal delivery of RGX-314 in
patients with diabetic retinopathy (DR). The IND is active, and
REGENXBIO plans to begin dosing patients in a Phase II trial,
ALTITUDE, in the second half of 2020.
"Diabetic retinopathy is the leading cause of blindness in
patients 24 to 75 years of age and affects approximately 8 million
people in the United States alone.
Without treatment, a large proportion of patients with DR develop
proliferative disease and vision threatening complications,
including center-involved diabetic macular edema. We believe
one-time treatment with RGX-314 can provide sustainable, long-term
anti-VEGF delivery to the eye, potentially reducing the severity of
DR and preventing vision threatening complications," said
Steve Pakola, M.D., Chief Medical
Officer of REGENXBIO. "We are pleased to advance this program, and
look forward to initiating our second clinical trial utilizing
suprachoroidal delivery, building upon clinical and preclinical
data from the RGX-314 program."
ALTITUDE is a multi-center, open label, randomized, controlled
dose-escalation study that will evaluate the efficacy, safety and
tolerability of suprachoroidal delivery of RGX-314. The trial is
expected to enroll 40 patients with DR across two cohorts. Patients
will be randomized to receive RGX-314 versus observational control
at a 3:1 ratio, and two dose levels of RGX-314 will be evaluated:
2.5x1011 GC/eye and 5.0x1011 GC/eye. Patients
will not receive prophylactic immune suppressive corticosteroid
therapy before or after administration of RGX-314.
The primary endpoint of the study is the proportion of patients
that improve in DR severity based on the Early Treatment Diabetic
Retinopathy Study-Diabetic Retinopathy Severity Scale (ETDRS-DRSS)
at 48 weeks. Other endpoints include safety and development of
DR-related ocular complications.
The Company expects to report interim data from this trial in
2021.
About RGX-314
RGX-314 is being developed as a
potential one-time treatment for wet AMD, diabetic retinopathy, and
other additional chronic retinal conditions treated with anti-VEGF.
RGX-314 consists of the NAV AAV8 vector encoding an antibody
fragment which is designed to inhibit VEGF, modifying the pathway
for formation of new leaky blood vessels which lead to retinal
fluid accumulation and vision loss.
About Diabetic Retinopathy
Diabetic retinopathy (DR)
is the leading cause of vision loss in adults between 24 and 75
years of age worldwide. DR affects approximately 8 million people
in the United States alone. The
spectrum of DR severity ranges from non-proliferative diabetic
retinopathy (NPDR) to proliferative diabetic retinopathy (PDR) and
as DR progresses, a large proportion of patients develop vision
threatening complications, including diabetic macular edema (DME)
and neovascularization that can lead to blindness. Current
treatment options for patients with DR include "watchful waiting",
anti-VEGF treatment, retinal laser or surgical treatment. Anti-VEGF
treatments have been shown to reduce the severity of DR and prevent
vision threatening complications by at least 75% in patients with
moderately severe to severe NPDR.
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.
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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