- ABBV-RGX-314 continues to be well tolerated in 50 patients
from dose levels 1 and 2 (Cohorts 1-3) with no drug-related serious
adverse events
- Dose level 2 prevented disease progression and reduced
vision-threatening events in non-proliferative diabetic retinopathy
patients at 1 Year
- 70.8% of patients achieved Diabetic Retinopathy Severity
Scale improvement vs. 25.0 % in control
- 0% of patients worsened ≥2 steps vs. 37.5 % in
control
- ABBV-RGX-314 reduced vision-threatening events by 89%
compared to control
- Conference call Monday, November
6, 8:30 a.m. ET
ROCKVILLE, Md., Nov. 3, 2023
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
additional positive data from the ongoing Phase II
ALTITUDE® trial of ABBV-RGX-314 for the
treatment of diabetic retinopathy (DR) without center-involved
diabetic macular edema (CI-DME) using in-office suprachoroidal
delivery. The data will be presented at the American Academy of
Ophthalmology meeting in San Francisco,
CA by Mark Barakat, M.D.,
Retinal Consultants of Arizona.
ABBV-RGX-314 is being investigated as a potential one-time gene
therapy for the treatment of wet age-related macular degeneration,
DR, and other chronic retinal conditions.
"We are pleased that ABBV-RGX-314 at dose level 2 continues to
be well tolerated and demonstrate clinically meaningful
improvements for patients with non-proliferative DR," said
Steve Pakola, M.D., Chief Medical
Officer of REGENXBIO. "A one-time, in-office injection of
ABBV-RGX-314 gene therapy has the potential to stabilize and
improve DR severity score and reduce the long-term risk of
vision-threatening events."
"DR is a leading cause of vision loss in working-age adults and
is a global public health concern, and I am encouraged by the
one-year positive results observed in the ALTITUDE trial of
ABBV-RGX-314," said Dr. Barakat. "There is an unmet need for
sustainable treatment options that prevent the progression of early
diabetic retinopathy to proliferative diabetic retinopathy and
development of vision threatening complications over the long term.
I look forward to seeing additional data from the ALTITUDE trial of
a one-time, in-office treatment of ABBV-RGX-314."
Data and Safety Summary
ALTITUDE is a multi-center, open-label, randomized, controlled,
dose-escalation trial evaluating the efficacy, safety and
tolerability of suprachoroidal delivery of ABBV-RGX-314 using the
SCS Microinjector® in patients with moderately severe or
severe non-proliferative diabetic retinopathy (NPDR) or mild
proliferative diabetic retinopathy (PDR). Patients in Cohort 1
received ABBV-RGX-314 at a dose level of 2.5x1011
genomic copies per eye (GC/eye) (dose level 1). Patients in Cohorts
2 and 3 received ABBV-RGX-314 at an increased dose level of
5x1011 GC/eye (dose level 2). Patients in Cohorts 1-3
did not receive prophylactic corticosteroid therapy before or after
administration of ABBV-RGX-314.
As of September 25, 2023,
ABBV-RGX-314 was reported to be well tolerated at dose levels 1 and
2. Seven serious adverse events were reported, none of which were
considered drug related. For patients in dose levels 1 and 2
(n=50), common ocular treatment-emergent adverse events in the
study eye through one year included conjunctival hemorrhage and
conjunctival hyperemia. Three patients had mild intraocular
inflammation (IOI), which resolved on topical corticosteroids. Six
patients had mild to moderate episcleritis and have resolved on
topical corticosteroids.
No cases of chorioretinitis, vasculitis, occlusion, or hypotony
were reported. Best Corrected Visual Acuity remained stable through
one year.
At one year, dose level 2 in NPDR patients prevented disease
progression as measured by the Early Treatment Diabetic Retinopathy
Study-Diabetic Retinopathy Severity Scale (DRSS). Importantly, dose
level 2 reduced the risk of developing vision-threatening events by
89% in these patients.
NPDR patients treated with ABBV-RGX-314 at dose levels 1 and 2
demonstrated clinically meaningful improvements in disease severity
and reduction of vision-threatening events. In dose level 2,
patients with baseline NPDR:
- 100% demonstrated stable to improved disease severity
- 70.8% achieved ≥1 step improvement vs. 25.0% in
control
- 0% worsened ≥2 steps vs. 37.5% in control
- 4.2% developed vision-threatening events vs. 37.5% in
control
In connection with this announcement, REGENXBIO will host a
conference call to discuss the Phase II ALTITUDE trial data with
Dr. Barakat 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. Pakola on Monday, November 6 at 8:30
a.m. ET. Listeners can register for the webcast via this
link. Analysts wishing to participate in the question and answer
session should use this link. A replay of the webcast will be
available via the company's investor website approximately two
hours after the call's conclusion. Those who plan on participating
are advised to join 15 minutes prior to the start time.
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 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 10 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. Chronic,
frequent treatment with anti-VEGF agents has been shown to improve
DR severity and reduce risk of progression to vision threatening
complications by > 70%. However, the majority of DR patients
without vision threatening complications are not treated with
anti-VEGF due to the unsustainable treatment burden of frequent
injections in the eye.
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 and AAV9. 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, 2022, 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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