ROCKVILLE, Md., Dec. 1, 2020 /PRNewswire/ -- REGENXBIO Inc.
(Nasdaq: RGNX), a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy based on its proprietary NAV® Technology
Platform, today announced that the first patient has been dosed in
the Phase I/II trial of RGX-111 for the treatment of
Mucopolysaccharidosis Type I (MPS I). RGX-111 is an investigational
one-time gene therapy designed to deliver the α-l-iduronidase
(IDUA) gene directly to the central nervous system (CNS) using the
NAV AAV9 vector.
MPS I is a rare, autosomal recessive genetic disease caused by
deficiency of IDUA, an enzyme required for the breakdown of the
polysaccharides in lysosomes. These polysaccharides, called
glycosaminoglycans (GAGs), accumulate in tissues of MPS I patients,
resulting in characteristic storage lesions and diverse clinical
signs and symptoms including in the central nervous system (CNS),
which can include excessive accumulation of fluid in the brain,
spinal cord compression, and cognitive impairment.
"We are leaders in the development of one-time treatments using
our NAV Technology Platform and proprietary delivery procedure
which is designed to administer our gene therapy candidates
directly to the central nervous system (CNS). RGX-111 is our second
product candidate for the treatment of a rare, neurodegenerative
disease to be dosed in patients, following RGX-121, which is in
clinical development for MPS II," said Steve Pakola, M.D., Chief Medical Officer of
REGENXBIO. "We believe one-time treatment with RGX-111 can provide
sustainable IDUA enzyme for patients, potentially preventing the
progression of disease. The dosing of the first patient in the
clinical trial for RGX-111 marks an important milestone in our
neurodegenerative disease program and our commitment to the MPS
community."
The Phase I/II trial is a multi-center, open-label, dose
escalation trial that will evaluate the safety, tolerability and
pharmacodynamics of RGX-111 delivered to patients with MPS I via
injection directly into the cerebrospinal fluid (CSF). Up to five
patients will be enrolled at two dose levels: 1.0x1010
GC/g of brain mass and 5.0x1010 GC/g of brain mass. The
primary endpoint of the study is safety and tolerability of
RGX-111. Other endpoints include the effect of RGX-111 on
biomarkers of IDUA enzyme activity in the CSF, serum and urine,
neurocognitive development and other outcome measures.
About RGX-111
RGX-111 is a product candidate for the treatment of
Mucopolysaccharidosis Type I (MPS I), also known as Hurler
syndrome. RGX-111 is designed to use the AAV9 vector to deliver the
α-l-iduronidase (IDUA) gene. Delivery of the enzyme that is
deficient within 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. This strategy could also provide rapid IDUA
delivery to the brain, potentially preventing 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
deficiency of IDUA, an enzyme required for the breakdown of the
polysaccharides in lysosomes. These polysaccharides, called
glycosaminoglycans (GAGs), accumulate in tissues of MPS I patients,
resulting in characteristic storage lesions and diverse clinical
signs and symptoms including in the central nervous system (CNS),
which 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.
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 in
multiple therapeutic areas.
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," "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, 2019, 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. 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:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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