- 97% reduction in treatment burden at nine months after
treatment with ABBV-RGX-314
- Data consistent with that from multiple previous studies
demonstrating favorable safety and efficacy profile
- Well tolerated with zero cases of intraocular inflammation
in a setting of no prophylactic steroids
- Data highlight the potential of ABBV-RGX-314 to treat both
eyes in wet AMD
ROCKVILLE, Md., Oct. 21,
2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced positive data from the Phase II fellow eye
sub-study evaluating the subretinal delivery of ABBV-RGX-314 in
patients with bilateral wet age-related macular degeneration (wet
AMD). The new data were presented at the American Academy of
Ophthalmology (AAO) meeting by Arshad
Khanani, M.D., M.A., FASRS, Director of Clinical Research
at Sierra Eye Associates, Reno, NV.
"The results presented at AAO from the Phase II sub-study, the
first to evaluate a gene therapy in fellow eyes for wet AMD,
demonstrate that ABBV-RGX-314 could be a treatment for patients
with bilateral disease and add to the already-robust data
demonstrating ABBV-RGX-314's potential to impact the treatment
paradigm for patients with wet AMD," said Curran Simpson, President and Chief Executive
Officer of REGENXBIO. "With more treated patients and the
longest-term data of any gene therapy program for wet AMD,
REGENXBIO, with our partner AbbVie, are well-positioned to bring
the first gene therapy to market with the hope of preserving
long-term vision for millions of patients globally with wet
AMD."
"The majority of our patients with wet AMD
eventually have bilateral disease and face a substantial
treatment burden with frequent lifelong injections in both
eyes. This leads to suboptimal real-world vision outcomes with
current standard of care," said Dr. Khanani. "The fellow eye
dosing data with ABBV-RGX-314 is a milestone for the field of
gene therapy for common retinal diseases, as this is the first
time we have performed bilateral treatment for wet AMD
patients. These results, combined with the durable
treatment effect up to four years shown in long-term
follow up, highlight the potential of ABBV-RGX-314
as a one-time effective treatment option for patients
with wet AMD."
Data and Safety Summary
The fellow eye sub-study was
designed to evaluate the safety and efficacy of a single dose
(1.3x1011 GC/eye) of ABBV-RGX-314 using subretinal
delivery in the fellow eye of previously treated patients. The
second eye was treated with ABBV-RGX-314 approximately one year or
more after administration of ABBV-RGX-314 in the first eye. This
dose is being evaluated in the ongoing pivotal trials of
ABBV-RGX-314 and is similar to one of the doses evaluated in the
Phase I/IIa trial, which demonstrated durable treatment effect up
to four years in a long-term follow up study.
The fellow eye sub-study data at nine months includes nine
patients who received ABBV-RGX-314 using subretinal delivery in the
Phase I/IIa or bridging studies and elected to receive treatment in
their second eye. Prior to ABBV-RGX-314 administration, these
patients had a high treatment burden in the fellow eye and had
received an average of nine anti-VEGF injections in the year prior
to entering the study, including anti-VEGF injections intended to
be longer-lasting treatments.
At nine months post-administration of ABBV-RGX-314, key findings
from the Phase II fellow eye sub-study include:
- 97% reduction in annualized anti-VEGF treatment burden
- 100% of patients required either zero or one supplemental
injection
- 78% of patients were completely injection-free
Additionally, patients demonstrated sustained best-corrected
visual acuity (BCVA) and central retinal thickness (CRT) at nine
months. ABBV-RGX-314 produced similar levels of aqueous protein in
both treated eyes.
As of September 11, 2024,
ABBV-RGX-314 was well tolerated in the treated fellow eye with no
drug related serious adverse events. No cases of intraocular
inflammation, chorioretinitis, vasculitis, occlusion or hypotony
were observed. No prophylactic steroids were used in this trial,
other than those typically used in vitrectomy surgery. Common
adverse events included mild retinal pigmentary changes occurring
in periphery and post-operative conjunctival hemorrhage, which all
resolved within days to weeks.
Data presented is available on the "Publications" section of the
REGENXBIO website at www.regenxbio.com.
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.
CONTACT:
Dana Cormack
Corporate Communications
DCORMACK@regenxbio.com
George E. MacDougall
Investor Relations
IR@regenxbio.com
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