- A single ABBV-RGX-314 gene therapy treatment has the
potential to become a new standard-of-care option among anti-VEGF
treatments by sustaining vision health long term and overcoming the
clinical challenges of managing wet AMD due to the treatment burden
of chronic anti-VEGF injections
- Patients who received therapeutic doses resulted in stable
or improved vision and retinal anatomy up to 2 years
- Additional long-term follow-up data has demonstrated durable
treatment effect, with stable or improved vision, up to 4
years
- Enrollment is on track in pivotal trials of ABBV-RGX-314 in
wet AMD that are expected to support global regulatory submissions
in late 2025 through the first half of 2026
ROCKVILLE, Md., March 28,
2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced the publication of results from the Phase I/IIa
trial evaluating the safety and tolerability of a single dose of
subretinal ABBV-RGX-314 for the treatment of wet age-related
macular degeneration (wet AMD). Two-year data were published
in The Lancet in a paper titled "Gene therapy
for neovascular age-related macular degeneration by subretinal
delivery of RGX-314: a phase 1/2a dose-escalation study." These
positive study results informed the ongoing pivotal trials of
ABBV-RGX-314, a potential one-time gene therapy, for the treatment
of wet AMD.
"We have started 2024 with strong, positive new data from the
ABBV-RGX-314 program, and we believe that there is
multi-billion-dollar potential for ABBV-RGX-314 to become a
first-in-class gene therapy for wet AMD and the standard of care to
treat and prevent progression of diabetic retinopathy," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "To have these Phase I/IIa
data published in The Lancet highlights the groundbreaking
work of our scientists and investigators, and further validates the
clinically transformative nature of ABBV-RGX-314 as a potential
one-time gene therapy for wet AMD that may help patients maintain
or improve their vision."
The findings highlighted in The Lancet
demonstrated that a single administration of ABBV-RGX-314 was
generally well tolerated. Stable or improved visual acuity and
retinal thickness was observed with few or no supplemental
anti-VEGF injections in most patients at two years. Patients who
received therapeutic doses demonstrated sustained levels of
ABBV-RGX-314 protein and stable or improved vision and retinal
anatomy with few, to no, supplemental anti-VEGF injections in most
participants up to two years. REGENXBIO has also reported
additional positive interim data from a long-term follow-up study
of ABBV-RGX-314 supporting that treatment continues to be
well-tolerated and demonstrates long-term, durable treatment effect
up to four years.
"The publication of the ABBV-RGX-314 Phase I/IIa trial results
in The Lancet reinforces the encouraging long-term clinical
data observed using subretinal delivery and underscores the
potential of ABBV-RGX-314 gene therapy to offer a new approach to
the clinical management of wet AMD," said Jeffrey S. Heier, M.D., Director of the
Vitreoretinal Service and Director of Retina Research at Ophthalmic
Consultants of Boston and primary
investigator for the trial. "Wet AMD is a chronic, life-long
disease and real-world evidence shows patients are losing
significant vision over time, and the burden of frequent anti-VEGF
injections needed to manage their wet AMD is a major reason why. A
single treatment of ABBV-RGX-314 that can potentially provide
long-lasting treatment outcomes and a strong safety profile would
offer a novel approach to treating this serious and blinding
disease."
ABBV-RGX-314 is currently being evaluated in patients with wet
AMD in two pivotal trials called ATMOSPHERE® and
ASCENT™. Enrollment is on track and these trials are expected to
support global regulatory submissions with the U.S. Food and
Drug Administration and the European Medicines
Agency in late 2025 through the first half of 2026.
About ABBV-RGX-314
ABBV-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. ABBV-RGX-314
consists of the NAV® AAV8 vector, which
encodes an antibody fragment designed to inhibit vascular
endothelial growth factor (VEGF). ABBV-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 ABBV-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 frequent, 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 REGENXBIO Inc.
REGENXBIO is a leading
clinical-stage biotechnology company seeking to improve lives
through the curative potential of gene therapy. Since its founding
in 2009, REGENXBIO has pioneered the development of AAV
Therapeutics, an innovative class of gene therapy medicines.
REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal
and rare diseases, including ABBV-RGX-314 for the treatment of wet
AMD and diabetic retinopathy, being developed in collaboration with
AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the
treatment of MPS II. Thousands of patients have been treated with
REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA
for children with spinal muscular atrophy. Designed to be one-time
treatments, AAV Therapeutics have the potential to change the way
healthcare is delivered for millions of people. For more
information, please visit www.regenxbio.com.
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, 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, 2023, 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.
Zolgensma® is a registered trademark of Novartis Gene
Therapies. 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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