- Company has initiated Phase I/II
AFFINITY DUCHENNE™ trial of
RGX-202
- Company also enrolling newly active
observational screening study, AFFINITY BEYOND, evaluating AAV8
antibody prevalence in boys with Duchenne
-
Commercial-scale cGMP material from the REGENXBIO Manufacturing
Innovation Center to be used in the clinical
trial
- RGX-202 is a potential one-time AAV
Therapeutic for the treatment of Duchenne and includes an optimized
transgene for a novel microdystrophin and REGENXBIO's proprietary
NAV® AAV8 vector
ROCKVILLE, Md., Jan. 23,
2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced that the Phase I/II AFFINITY
DUCHENNE™ trial of RGX-202 for the treatment of Duchenne
muscular dystrophy (Duchenne) is now active and recruiting
patients. RGX-202 is designed to deliver a transgene for a novel
microdystrophin protein that includes the functional
elements of the C-Terminal (CT) domain found in naturally
occurring dystrophin. RGX-202 uses REGENXBIO's proprietary
NAV® AAV8 vector.
AFFINITY DUCHENNE is a multicenter, open-label dose evaluation
and dose expansion clinical trial to evaluate the safety,
tolerability and clinical efficacy of a one-time intravenous (IV)
dose of RGX-202 in patients with Duchenne.
Additionally, REGENXBIO is recruiting patients in the AFFINITY
BEYOND™ trial, an observational screening study. The
primary objective is to evaluate the prevalence of AAV8 antibodies
in patients with Duchenne up to 12 years of age. Information
collected in this study may be used to identify potential
participants for the AFFINITY DUCHENNE trial and potential future
trials of RGX-202.
"I am pleased that we are now able to initiate the trial for
RGX-202 and also begin enrollment activities in our AAV8 antibody
screening study," said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO.
"The RGX-202 program is a key piece of our '5x'25' strategy to have
five AAV Therapeutics either on the market or in late-stage
development by 2025. We look forward to continuing to work closely
with the Duchenne community as we advance a highly differentiated
product candidate developed with the potential to improve muscle
strength and motor function in boys with Duchenne."
"Duchenne muscular dystrophy is a devastating disease and there
are still unmet therapeutic needs," said Aravindhan Veerapandiyan,
M.D., a principal investigator in the study and Director of the
Comprehensive Neuromuscular Program, PPMD Certified Duchenne Care
Center, and Co-Director of the Muscular Dystrophy Association Care
Center at Arkansas Children's Hospital. "Gene therapies, like
RGX-202, have the potential to impact the progressive nature of
Duchenne."
REGENXBIO has manufactured additional clinical supply of RGX-202
in its in-house Manufacturing Innovation Center using the
NAVXpress™ process platform. Located
in REGENXBIO's 132,000 square foot headquarters
in Rockville, MD, the Manufacturing Innovation Center is
designed to meet global clinical and commercial regulatory
standards, and 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.
Additional information can be found on clinicaltrials.gov for
AFFINITY DUCHENNE and AFFINITY BEYOND.
AFFINITY
DUCHENNE™ Trial Design
In the dose evaluation phase of the trial, six ambulatory,
pediatric patients (ages 4 to 11 years old) with Duchenne are
expected to enroll in two cohorts with doses of 1x1014
genome copies (GC)/kg body weight (n=3) and 2x1014 GC/kg
body weight (n=3). After an independent safety data review for each
cohort, a dose expansion phase of the trial may allow for up to six
additional patients to be enrolled at each dose level (for a total
of up to nine patients in each dose cohort).
The trial design also consists of thorough safety measures
informed by the Duchenne community and engagement with key opinion
leaders, including a comprehensive, short-term, prophylactic
immunosuppression regimen to proactively mitigate potential
complement-mediated immunologic responses, and inclusion criteria
based on dystrophin gene mutation status, including DMD gene
mutations in exons 18 and above. Trial endpoints include safety,
immunogenicity assessments, pharmacodynamic and pharmacokinetic
measures of RGX-202, including microdystrophin protein levels in
muscle, and strength and functional assessments, including the
North Star Ambulatory Assessment (NSAA) and timed function tests.
Initial trial sites are located in the U.S., with additional sites
in Canada and Europe expected to follow.
AFFINITY BEYOND™
Observational Study
AFFINITY BEYOND is an observational screening study. The primary
objective is to evaluate the prevalence of anti-adeno-associated
serotype 8 (AAV8) antibodies in participants with Duchenne muscular
dystrophy. AAV gene therapies are delivered via viral vectors that
are not known to cause disease in humans. For AAV gene therapies
delivered systemically, it's important to screen patients for
antibodies to the vector. This observational study is intended to
help inform future clinical research in Duchenne.
About RGX-202
RGX-202 is designed to deliver a transgene for a novel
microdystrophin that includes the functional elements of the
C-Terminal (CT) domain found in naturally occurring dystrophin.
Presence of the CT domain has been shown in preclinical studies to
recruit several key proteins to the muscle cell membrane, leading
to improved muscle resistance to contraction-induced muscle damage
in dystrophic mice. Additional design features, including codon
optimization and reduction of CpG content, may potentially improve
gene expression, increase translational efficiency and reduce
immunogenicity. RGX-202 is designed to support the delivery and
targeted expression of genes throughout skeletal and heart muscle
using the NAV AAV8 vector, a vector used in numerous clinical
trials, and a well-characterized muscle-specific promoter
(Spc5-12).
About Duchenne Muscular
Dystrophy
Duchenne muscular dystrophy (Duchenne) is a rare genetic
disorder, caused by mutations in the gene responsible for making
dystrophin, a protein of central importance for muscle cell
structure and function. Duchenne primarily affects males with
approximately 1 in 3,500 to 1 in 5,000 males affected worldwide.
The absence of functional dystrophin protein in individuals with
Duchenne results in cell damage during muscle contraction, leading
to cell death, inflammation, and fibrosis in muscle tissues.
Initial symptoms of Duchenne include muscle weakness that is often
noticeable at an early age, with diagnosis typically occurring by 5
years of age. Over time, individuals with Duchenne experience
progressive muscle weakness and eventually lose the ability to
walk. Respiratory and heart muscles are also affected, leading to
difficulty breathing and the need for ventilator assistance, along
with the development of cardiomyopathy. There is presently no cure
for Duchenne.
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 "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, 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.
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey, ICR
Westwicke
339-970-2843
chris.brinzey@westwicke.com
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