- RGX-314 produced by the NAVXpress platform process has been
well-tolerated and demonstrated a similar clinical profile to the
initial adherent cell culture process
- NAVXpress platform process is incorporated in the two
ongoing pivotal trials and is expected to be used for future
commercialization of RGX-314; the two pivotal trials are expected
to support BLA submission in 2024
- Company to host live webcast with wet AMD Key Opinion
Leaders to discuss new interim Phase II bridging study data, today,
Saturday, February 11, at
11:30 a.m. ET
ROCKVILLE, Md., Feb. 11,
2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced results from a Phase II bridging study
evaluating the pharmacodynamics, safety and efficacy of RGX-314, a
potential one-time therapy delivered subretinally using cGMP
material produced by the company's NAVXpress™ bioreactor
platform process. The data is being presented at the Angiogenesis,
Exudation, and Degeneration 2023 Conference by Charles C. Wykoff, M.D., PhD., Director of
Research at Retina Consultants of Texas; Chairman of Research, Retina
Consultants of America; and Deputy Chair of Ophthalmology for the
Blanton Eye Institute, Houston Methodist Hospital.
"The interim results observed in the Phase II bridging study
show a similar clinical profile between our manufacturing
processes. We believe our approach, focused on early product
quality and process control, allows us to efficiently transition
from clinical trials to commercial readiness," said Curran Simpson, Chief Operating Officer
of REGENXBIO. "This update provides validation of our plans
for the NAVXpress platform process to support the production of
RGX-314 in anticipation of future commercialization."
"There is a significant need for treatment options that can
reduce the burden of frequent injections for wet AMD patients while
maintaining optimal function and anatomic outcomes," said Dr.
Wykoff. "The clinical profile of RGX-314 manufactured using the
commercial-scale process is encouraging, as is the potential of a
one-time therapy for the treatment of wet AMD."
Interim Data Summary from the Phase II Bridging Study of
RGX-314 using Subretinal Delivery
The Phase II bridging
study is designed to evaluate RGX-314 using subretinal delivery
across two dose levels (6.4x1010 GC/eye and
1.3x1011 GC/eye) in 60 patients with wet AMD. At each
dose level, patients are assigned into two cohorts, with half of
the patients at each dose level receiving RGX-314 produced by the
NAVXpress platform process, and the other half receiving RGX-314
produced by the adherent cell culture manufacturing process that
was used in the Phase I/IIa trial of RGX-314 for the treatment of
wet AMD. The primary endpoint of the study is RGX-314 target
protein concentration in the eye at Month 6. Secondary endpoints
include safety and tolerability, change from baseline in Best
Corrected Visual Acuity (BCVA), change in central retinal thickness
(CRT) and need for supplemental anti-VEGF injections.
As of November 14, 2022, RGX-314
was well tolerated across 46 patients dosed in cohorts at both dose
levels. Five SAEs were reported, none of which were considered
related to RGX-314. In the high dose cohorts, all common treatment
emergent adverse events (TEAEs) through 6 months in the study eye
were mild or moderate and included post-operative conjunctival
hemorrhage, post-operative inflammation and retinal pigmentary
changes.
The two high dose cohorts have fully enrolled and completed six
month visit assessments. Data presented at Angiogenesis highlighted
the results from these cohorts (n=30). In these cohorts, target
protein concentrations in the eye were similar between the
manufacturing processes. Patients in the two high dose cohorts also
demonstrated stable to improved BCVA and CRT, and meaningful
reductions in anti-VEGF burden, with a majority of subjects
injection-free.
Pivotal Trials for the Treatment of RGX-314 for wet AMD using
Subretinal Delivery
To support future commercialization of
RGX-314, the cGMP material produced by the Company's NAVXpress
platform process has been incorporated in the ongoing pivotal
trials, ATMOSPHERE® and ASCENT™, for the
treatment of wet AMD using RGX-314 delivered subretinally. These
pivotal trials are multi-center, randomized, active-controlled
trials to evaluate the efficacy and safety of a
single-administration of RGX-314 using subretinal delivery versus
standard of care in patients with wet AMD. The two pivotal trials
are designed to evaluate the same dose levels being used in the
Phase II bridging study.
"The RGX-314 program is central to our '5x'25' strategy to have
five AAV Therapeutics either on the market or in late-stage
development by 2025. Our NAVXpress platform process is producing
cGMP material at the REGENXBIO Manufacturing Innovation Center
and has supported the advancement of several of our on-going
clinical programs," said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO.
"We are one of only a few gene therapy companies with a cGMP
facility capable of producing at scales of up to 2,000 liters,
which we believe represents a key differentiator for REGENXBIO that
we expect to use across our ongoing clinical trials to support
accelerating the development of our AAV Therapeutics, including
RGX-314."
A live discussion of the Phase II bridging study data with Dr.
Wykoff and Dr. Peter Kaiser, Chaney
Family Endowed Chair in Ophthalmology Research and
Professor of Ophthalmology, Cleveland Clinic Lerner College of
Medicine and Cole Eye Institute, and Dr. Steve Pakola, Chief Medical Officer of REGENXBIO
will stream on the "Investors" events page of the REGENXBIO website
at 11:30 a.m. ET. The presentation is
available on the "Presentations and Publications" section of the
REGENXBIO website at www.regenxbio.com.
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, it
is difficult for patients to adhere to frequent injections, which
can lead to a decline in vision over time.
About NAVXpress™
NAVXpress is REGENXBIO's
proprietary, high-yielding manufacturing platform process for NAV
vector production that can be applied across multiple AAV
Therapeutics. The suspension-based manufacturing platform process
has demonstrated robust scalability from bench-scale to 2,000 liter
batches with consistent yield and product purity demonstrated via
comparability studies.
The NAVXpress platform is used at the REGENXBIO Manufacturing
Innovation Center, a state-of-the-art facility built to meet global
clinical and commercial regulatory standards. It includes two
independent bulk drug substance production suites, a final drug
product suite and integrated quality control labs. REGENXBIO is one
of only a few gene therapy companies worldwide with a cGMP facility
capable of production at scales up to 2,000 liters.
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 "5 x '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, 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
View original content to download
multimedia:https://www.prnewswire.com/news-releases/regenxbio-presents-interim-data-from-phase-ii-bridging-study-evaluating-the-clinical-performance-of-rgx-314-using-the-navxpress-manufacturing-platform-process-301744570.html
SOURCE REGENXBIO Inc.