-Interim results from Phase II AAVIATE
and ALTITUDE trials of dose level 3 cohorts with short-course
prophylactic steroid eye drops resulted in zero cases of
intraocular inflammation
-Initial safety results from Cohort 1
of the AFFINITY DUCHENNE study support well-tolerated profile to
date; initial efficacy data to be presented at World Muscle Society
meeting in October
-A new program in Duchenne with preclinical
data using innovative science to produce near full-length wild-type
dystrophin; expects to file IND to the FDA in the first half of
2025
-Detailed updates will be presented at its
Virtual Investor Day, today at 8:30 a.m.
ET
ROCKVILLE, Md., July 11,
2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) will announce today new interim data from the Phase II
AAVIATE® and ALTITUDE® trials of ABBV-RGX-314
suprachoroidal delivery in wet AMD and diabetic retinopathy (DR).
The company will also provide a comprehensive Duchenne program
overview including initial safety data for RGX-202 and a new exon
53 program.
REGENXBIO will host a virtual Investor Day today at 8:30 a.m. ET to discuss these updates. The
webcast and slides of the presentation can be accessed in the
Investors section of REGENXBIO's website at www.regenxbio.com.
"We continue to perform at a high level as we execute on our
mission of improving lives through the curative potential of gene
therapy," said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. "Today, we are
pleased to share an update on REGENXBIO's pipeline of AAV-based
gene therapies with a focus on our retinal and Duchenne programs.
The safety and tolerability data presented today for the high-dose
cohorts of our in-office wet AMD and DR programs is encouraging and
further validate the potential of the suprachoroidal space for AAV
delivery to treat chronic retinal diseases."
Mr. Mills continued, "I am also thrilled to share an update on
our Duchenne program, from our neuromuscular franchise, which
includes progress on RGX-202 and our new exon-skipping program that
we have initiated. We continue to develop highly differentiated,
transformational treatments with an unwavering commitment to
advance therapies designed to prevent ambulation loss and improve
the quality of life in boys worldwide, regardless of age, who
suffer from Duchenne."
"I believe we have a clear and definable path to achieve our '5
by '25' vision," said Mr. Mills. "We are advancing a strong
pipeline of AAV-based therapeutic candidates in diseases
representing large current market opportunities and that address
significant unmet needs for millions of patients."
Key Investor Day Highlights
Retinal Program Update
ABBV-RGX-314 is an investigational one-time AAV
therapeutic being developed in collaboration with AbbVie for the
treatment of wet AMD, DR and other chronic retinal conditions.
ABBV-RGX-314 uses the NAV® AAV8 vector to deliver a gene
encoding therapeutic antibody fragment to inhibit vascular
endothelial growth factor (VEGF).
Interim data to be presented today from the Phase II AAVIATE
& ALTITUDE trials demonstrated that ABBV-RGX-314 suprachoroidal
delivery administered to patients in cohorts at dose level 3
(1.0x1012 genome copies per eye) with short-course
(seven-week) prophylactic topical steroid eye drops (N=39) resulted
in zero cases of intraocular inflammation. Time of
post-administration follow up ranged from six weeks to six
months.
- ABBV-RGX-314 Suprachoroidal Delivery for the Treatment of
Wet AMD
-
- Patients in Cohort 6 (dose level 3, N=20) in the Phase II
AAVIATE trial all received short-course prophylactic ocular
steroids following ABBV-RGX-314 administration, to evaluate the
ability to prevent or reduce the occurrence of mild to moderate
intraocular inflammation seen in previous cohorts.
- As of June 12, 2023, ABBV-RGX-314
was reported to be well tolerated in 20 patients from Cohort 6,
with no drug-related serious adverse events. Time of
post-administration follow up ranged from six weeks to six
months.
- Short-course of ocular steroid prophylaxis meaningfully reduced
the occurrence of mild to moderate intraocular inflammation
seen in previous cohorts. In all 10 patients who received the
short-course (seven-week) prophylactic topical steroid eye drops
there were zero cases of intraocular inflammation.
- REGENXBIO expects to report additional interim data from the
Phase II AAVIATE trial, including full 6-month results from Cohorts
5 and 6, at the Hawaiian Eye and Retina meeting being held
January 13-19, 2024.
- ABBV-RGX-314 Suprachoroidal Delivery for the Treatment of
DR
-
- Patients in Cohorts 4 and 5 (dose level 3, N=29) in the
Phase II ALTITUDE trial were stratified by Early Treatment Diabetic
Retinopathy Study-Diabetic Retinopathy Severity Scale (DRSS)
levels, and all received short-course (seven-week) prophylactic
topical steroid eye drops following ABBV-RGX-314
administration.
- As of June 12, 2023, ABBV-RGX-314
was reported to be well tolerated in 29 patients from Cohorts 4 and
5, with no drug-related serious adverse events. Time of
post-administration follow up ranged from 12 weeks to six
months.
- There were zero cases of intraocular inflammation.
- REGENXBIO expects to report additional interim data from the
Phase II ALTITUDE trial at the American Academy of Ophthalmology
meeting being held November 3-6,
2023.
Duchenne Program Update
RGX-202 is an investigational one-time AAV
therapeutic for Duchenne Muscular Dystrophy (Duchenne), using the
NAV AAV8 vector to deliver a transgene for a novel microdystrophin
that includes the functional elements of the C-Terminal (CT) domain
as well as a muscle-specific promoter to support a targeted therapy
for improved resistance to muscle damage associated with
Duchenne.
REGENXBIO will present interim safety data from Cohort 1 of the
ongoing Phase I/II AFFINITY DUCHENNE™ trial, which continues to
recruit patients (aged 4 to 11 years) and is using commercial-scale
cGMP material from the REGENXBIO Manufacturing Innovation
Center.
- As of July 6, 2023, RGX-202 was
reported to be well-tolerated in two patients dosed to date, aged 4
and 10 years, with no drug-related serious adverse events. Time of
post-administration follow up was 45 days and more than three
months.
- REGENXBIO will share additional interim data of the AFFINITY
DUCHENNE trial, including longer-term safety and microdystrophin
protein expression levels in muscle at three months, at the World
Muscle Society Congress being held October
3-7, 2023.
New Exon Skipping Program: REGENXBIO will also
announce today the development of a potential one-time gene therapy
for Duchenne, which is based on a novel exon-skipping construct.
REGENXBIO scientists have demonstrated that AAV-mediated exon
skipping by the expression of antisense ribonucleic acid (AS RNA)
sequences has the potential to restore high and sustained levels of
near full-length dystrophin in Duchenne patients with relevant
mutations in the DMD gene.
- REGENXBIO's new candidate is derived and characterized from the
company's NAV Technology Platform, designed to use a
muscle-specific capsid and deliver three AS RNAs targeting exon 53
of the DMD gene.
- Proof of concept data from preclinical studies in the
hDMDdel52/mdx mouse model of Duchenne demonstrated over 90% exon 53
skipping efficiency, broad and robust (up to 50% of expected
wild-type dystrophin levels were restored) expression of near
full-length dystrophin in skeletal and heart muscle, and
improvements in muscle histopathology, with durability up to three
months, which is the longest timepoint measured to date.
- REGENXBIO estimates that nearly 50% of Duchenne patients have
mutations of the DMD gene that are amenable to exon skipping
and can potentially be addressed with its AAV-mediated exon
skipping platform.
- The company is initiating Investigational New Drug (IND)
application-enabling studies and expects to submit an IND to the
FDA in the first half of 2025.
Virtual Investor Day Webcast Information
The virtual
event will take place on Tuesday, July 11,
2023, at 8:30 a.m. Eastern
Time. The live webcast and presentation slides can be
accessed in the Investors section of REGENXBIO's website at
www.regenxbio.com. An archived replay of the webcast will be
available in the Investors section of the website for approximately
30 days following the presentation.
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 its internal pipeline and licensed programs into
pivotal-stage or commercial products by 2025.
Forward-Looking Statement
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.
Contact:
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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