- RGX-111 is an investigational AAV Therapeutic for the
treatment of severe MPS I that is part of REGENXBIO's
clinical-stage pipeline of neurodegenerative disease
programs.
- Expanded Cohort 2 enrollment is complete; eight patients
have received RGX-111 in the trial
- Company intends to manufacture commercial-scale cGMP
material to support the continued development of
RGX-111
- Company expects additional interim updates from the RGX-111
trial in the first half of 2023
ROCKVILLE, Md., Dec. 8, 2022
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it
has completed dosing in the expanded Cohort 2 of the Phase I/II
trial of RGX-111 for the treatment of severe Mucopolysaccharidosis
Type I (MPS I). The trial has now completed dosing, with eight
patients enrolled across two dose cohorts. As previously reported,
RGX-111 has also been administered to a patient with MPS I through
a single-patient IND.
RGX-111 is an investigational one-time gene therapy designed to
deliver the gene that encodes the α-l-iduronidase (IDUA) enzyme
using the NAV AAV9 vector. RGX-111 is administered directly to the
CNS. The primary endpoint of the trial is to evaluate the safety of
RGX-111. Secondary and exploratory endpoints include biomarkers of
IDUA enzyme activity in the cerebrospinal fluid (CSF), serum and
urine, neurodevelopmental assessments, and caregiver reported
outcomes. Patients were treated across two dose cohorts:
1.0x1010 genome copies per gram (GC/g) of brain
mass (n=2) and 5.0x1010 GC/g of brain mass (n=6).
In the single-patient IND for RGX-111, a severe MPS I patient was
dosed with 1x1010 GC/g of brain mass.
"We have made great progress this year advancing our pipeline of
neurodegenerative disease programs, and completing the dosing in
this MPS I trial is another important clinical milestone as we
continue to develop potential one-time gene therapies for
patients," said Steve Pakola, M.D.,
Chief Medical Officer of REGENXBIO. "Earlier, at this year's
WORLDSymposium meeting, we presented data on biomarker and
neurodevelopmental assessments that indicated an encouraging CNS
profile in patients dosed with RGX-111. We intend to advance
the program for RGX-111 with the aim of providing a
much-needed new treatment option for the MPS I community as quickly
as possible."
The Phase I/II trial of RGX-111 is a global trial, which
includes sites in the United
States and Brazil. Many of these trial sites are also
participating in REGENXBIO's on-going CAMPSIITE™ trial,
the pivotal program for RGX-121 for the treatment of
Mucopolysaccharidosis Type II (MPS II).
"Our '5x'25' strategy is to have five gene therapies either on
the market or in late-stage development by 2025. Earlier this year,
we announced that the pivotal program for RGX-121 is active and
enrolling patients, making this our second internal program to
enter late-stage development. RGX-111 is our next
most-advanced clinical candidate in our neurodegenerative disease
pipeline and over 30 children with severe MPS I and MPS II have
been dosed in our trials using the NAV AAV9 vector," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "I would like to express my
deep gratitude to our employees and clinical partners, as well as
patients and their families in the MPS community, for their ongoing
commitment and support in the development of both RGX-111 and
RGX-121."
REGENXBIO plans to continue having early and frequent
communication with regulatory agencies about pathways to expedite
development of its neurodegenerative disease pipeline. In the first
half of 2023, REGENXBIO expects to use its Manufacturing
Innovation Center to produce RGX-111 commercial-scale cGMP material
from its proprietary, high-yielding suspension-based
manufacturing process, named NAVXPress™. The pivotal
program for RGX-121 is using the NAVXPress manufacturing
process.
Additional interim data from the Phase I/II trial of RGX-111 is
also expected to be reported in the first half of 2023.
About RGX-111
RGX-111 is designed to use the AAV9 vector to deliver the
α-l-iduronidase (IDUA) gene to the central nervous system (CNS).
Delivery of the IDUA gene within the cells in the central nervous
system (CNS) could provide a permanent source of secreted IDUA
beyond the blood-brain barrier, allowing for long-term
cross-correction of cells throughout the CNS. By providing rapid
IDUA delivery to the brain, RGX-111 could potentially help prevent
the progression of cognitive deficits that otherwise occurs in MPS
I patients. RGX-111 has received orphan drug product, rare
pediatric disease and Fast Track designations from the U.S. Food
and Drug Administration.
About Mucopolysaccharidosis Type I
(MPS I)
MPS I is a rare autosomal recessive genetic disease caused by a
deficiency in the lysosomal enzyme alpha-L-iduronidase (IDUA),
leading to an accumulation of glycosaminoglycans (GAGs) including
heparan sulfate (HS) in tissues which ultimately results in cell,
tissue, and organ dysfunction, including in the central nervous
system (CNS). This can include excessive accumulation of fluid in
the brain, spinal cord compression, and cognitive impairment. MPS I
is estimated to occur in 1 in 100,000 births. Current disease
modifying therapies for MPS I include hematopoietic stem cell
transplant (HSCT) and enzyme replacement therapy with a recombinant
form of human IDUA administered intravenously. However, intravenous
enzyme therapy does not treat the CNS manifestations of MPS I, and
HSCT can be associated with clinically significant morbidity and
mortality. Key biomarkers of IDUA enzymatic activity in MPS I
patients include its substrate heparan sulfate (HS), which has been
shown to correlate with neurocognitive 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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