- 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
- Commercial-scale cGMP material from the REGENXBIO
Manufacturing Innovation Center to be used in the clinical
trial
- AFFINITY DUCHENNE™ Phase I/II trial of RGX-202 is active and
recruiting patients; anticipated to report initial trial data in
the second half of 2023
ROCKVILLE, Md., April 11,
2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced that the U.S. Food and Drug Administration
(FDA) has granted Fast Track designation for RGX-202, a potential
one-time gene therapy for the treatment of Duchenne muscular
dystrophy (Duchenne).
Fast Track designation aims to facilitate the development and
expedite the review of new therapeutics that are intended to treat
serious or life-threatening conditions and that demonstrate the
potential to address unmet medical needs. Therapies granted this
designation are given the opportunity for more frequent
interactions with the FDA and may qualify for priority review. The
FDA has granted RGX-202 Orphan Drug Designation and Rare Pediatric
Disease Designation.
"Fast Track designation, along with our capabilities to conduct
our clinical trials using commercial-scale cGMP material, will
further support the efficient development of RGX-202 from clinic to
commercial readiness," said Kenneth T.
Mills, President, and Chief Executive Officer of REGENXBIO.
"RGX-202 is a key part of our '5x25' strategy, and we look forward
to continuing to work closely with the FDA and the
Duchenne community as we advance a highly differentiated
product candidate developed with the potential to make a meaningful
difference for patients. We look forward to reporting initial data
from our clinical trial of RGX-202 in the second half of this
year."
"We are pleased that the FDA has granted Fast Track designation
for RGX-202," said Debra Miller,
Founder and CEO of CureDuchenne. "Accelerating the development of
medicines for Duchenne, especially potential one-time gene
therapies like RGX-202, is critical for this community."
RGX-202 Clinical Program
In January,
REGENXBIO announced that the Phase I/II AFFINITY
DUCHENNE™ trial of RGX-202 for the treatment of
Duchenne is now active and recruiting patients.
AFFINITY DUCHENNE is a multicenter,
open-label dose evaluation and dose expansion clinical study to
evaluate the safety, tolerability and clinical efficacy of a
one-time intravenous (IV) dose of RGX-202 in patients with
Duchenne. 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).
Additionally, REGENXBIO is recruiting patients in the
AFFINITY BEYOND™ trial, an observational screening study 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.
REGENXBIO Gene Therapy Manufacturing
REGENXBIO has
manufactured additional clinical supply of RGX-202 in its in-house
Manufacturing Innovation Center using the NAVXpress™ platform
process. Located in REGENXBIO's 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.
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). RGX-202 has been granted Fast Track, Orphan Drug and
Rare Pediatric Disease designations by the FDA.
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 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.
Contacts:
Dana
Cormack
Corporate
Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey, ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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