- FDA will consider an accelerated approval pathway for
RGX-121; BLA filing expected in 2024
- Pivotal program is active and enrolling patients
- RGX-121 is a potential first-in-class, one-time gene therapy
for the treatment of MPS II
- Internal cGMP manufacturing process expected to support
BLA
ROCKVILLE, Md., Aug. 3, 2022
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
its intention to file a Biologics License Application (BLA) in 2024
using the FDA's accelerated approval pathway for RGX-121 for the
treatment of Mucopolysaccharidosis Type II (MPS II), also known as
Hunter Syndrome. The Company also announced that a pivotal program
for RGX-121 is active and enrolling patients. RGX-121 is an
investigational, one-time AAV Therapeutic using the NAV AAV9 vector
to deliver the gene that encodes the iduronate-2-sulfatase (I2S)
enzyme.
"We are pleased to share that our recent discussions with the
FDA support our plans to submit a BLA for RGX-121 in 2024 using the
accelerated approval pathway, which was created to allow for
expedited development of drugs that treat serious conditions and
provide a meaningful advantage over available therapies based on a
surrogate endpoint," said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO.
"We believe RGX-121 for the treatment of Hunter Syndrome has
demonstrated emerging positive impact on neurodevelopmental
function, and we intend to advance this program as quickly as
possible with the aim of providing a much-needed new treatment
option for the MPS II community."
"The accelerated approval pathway gives patients access to
potentially life-changing therapeutics sooner, which is why it is a
critical tool for the over 7,000 rare diseases, most of which lack
FDA-approved treatments," said Joseph
Muenzer, MD, PhD, Bryson Distinguished Professor in
Pediatrics Genetics, and Director, MPS Research and Treatment
Program, University of North Carolina at
Chapel Hill. "Boys with MPS II do not have time to wait. I
am encouraged by FDA's commitment to advance therapies for rare
diseases and their willingness to consider this important pathway
for RGX-121."
The ongoing Phase I/II trial of RGX-121 in children up to five
years old has been expanded into a pivotal Phase I/II/III trial,
named CAMPSIITE™. A BLA filing in 2024 is expected to be supported
by endpoints in the pivotal program, including changes from
baseline of glycosaminoglycans (GAGs) in the cerebrospinal fluid
(CSF) at four months. GAGs in the CSF have the potential to be
considered a surrogate biomarker that is reasonably likely to
predict clinical benefit in MPS II disease under the accelerated
approval pathway, as buildup of GAGs in the CSF of MPS II patients
correlates with clinical manifestations, including
neurodevelopmental deficits.
As presented in February 2022,
preliminary results from REGENXBIO's Phase I/II trial of RGX-121
indicated that RGX-121 continued to be well-tolerated with
dose-dependent reductions of GAGs in the CSF, with patients in
Cohort 3 approaching normal levels of certain GAGs in the CSF.
Measures of neurodevelopmental function from patients in Cohorts 1
and 2 demonstrated continued developmental skill acquisition up to
2 years after RGX-121 administration.
"MPS II families are in need of a treatment option that can
address the neurodevelopmental decline associated with this disease
that available enzyme replacement therapies cannot," said
Terri Klein, President and CEO of
the National MPS Society. "We are encouraged by today's
announcement and the hope that a one-time gene therapy offers this
community."
Pivotal Program (CAMPSIITE Trial)
CAMPSIITE is a multicenter, open-label trial enrolling boys with
MPS II, aged 4 months up to five years of age. CAMPSIITE is now
expected to enroll up to 10 MPS II patients to support the BLA
filing using the accelerated approval pathway, with the potential
to enroll additional patients. These patients will receive a dose
of 2.9x1011 GC/g of brain mass of RGX-121, which is the
same dose being evaluated in Cohort 3 of the Phase I/II trial. The
pivotal program is using commercial-scale cGMP material from
REGENXBIO's proprietary, high-yielding suspension-based
manufacturing process, named NAVXPress™. In addition to measuring
GAGs in the CSF, the trial will continue to collect
neurodevelopmental data and caregiver-reported outcomes.
CAMPSIITE is a global trial, which is expected to include sites
in the United States, Brazil and Canada. REGENXBIO has begun dosing patients in
the pivotal program.
"MPS II patients face irreversible neurodevelopmental decline
that significantly impacts a child's daily life and life
expectancy," said Paul Harmatz, MD,
professor, UCSF Department of Pediatrics, and principal
investigator of the CAMPSIITE study. "Current therapies are
inadequate, so we are excited to be a part of this study and eager
to see the potential impact on patients."
REGENXBIO plans to share new RGX-121 data at the Society for the
Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium on
August 31, 2022.
About RGX-121
RGX-121 is designed to use the NAV AAV9 vector to deliver the human
iduronate-2-sulfatase gene (IDS) which encodes the
iduronate-2-sulfatase (I2S) enzyme to the central nervous system
(CNS). Delivery of the IDS gene within cells in the CNS could
provide a permanent source of secreted I2S beyond the blood-brain
barrier, allowing for long-term cross correction of cells
throughout the CNS and consequent reduction of GAGs. RGX-121 has
received orphan drug product, rare pediatric disease and Fast Track
designations from the U.S. Food and Drug Administration.
RGX-121 is being evaluated in two on-going studies, CAMPSIITE, a
Phase I/II/III multicenter, open-label clinical trial of patients
up to five years old with MPS II and a Phase I/II multicenter,
open-label clinical trial in patients over the age of five years
old with MPS II.
About Mucopolysaccharidosis Type II (MPS II)
MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease
caused by a deficiency in the lysosomal enzyme
iduronate-2-sulfatase (I2S) leading to an accumulation of
glycosaminoglycans (GAGs) in tissues which ultimately results in
cell, tissue, and organ dysfunction, including in the central
nervous system (CNS). MPS II is estimated to occur in 1 in 100,000
to 170,000 births. In severe forms of the disease, early
developmental milestones may be met, but developmental delay is
readily apparent by 18 to 24 months. Specific treatment to address
the neurological manifestations of MPS II remains a significant
unmet medical need. Key biomarkers of I2S enzymatic activity in MPS
II patients have been shown to correlate with neurodevelopmental
manifestations of the disorder.
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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