ROCKVILLE, Md., March 4,
2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced the closing of its previously
announced strategic partnership with Nippon Shinyaku.
Under the terms of the agreement, REGENXBIO and Nippon Shinyaku
will develop and commercialize RGX-121 (clemidsogene
lanparvovec) for Mucopolysaccharidosis II (MPS II), also known
as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I),
also known as Hurler syndrome in the
United States and Asia.
"RGX-121 and RGX-111 represent potentially transformative new
medicines for patient populations in great need of new options,"
said Curran M. Simpson, President
and CEO of REGENXBIO. "This partnership bolsters our ability to
make important progress on these programs, and we look forward to
serving the MPS community with Nippon Shinyaku."
RGX-121 is on track to be the first gene therapy for MPS II,
with potential FDA approval as early as late 2025.
About RGX-121
RGX-121 is a potential one-time AAV
therapeutic for the treatment of boys with MPS II. RGX-121
expressed protein is structurally identical to normal I2S. 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.
RGX-121 has received Orphan Drug Product, Rare Pediatric
Disease, Fast Track and Regenerative Medicine Advanced Therapy
designations from the U.S. Food and Drug
Administration and advanced therapy medicinal products (ATMP)
classification from the European Medicines Agency.
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). 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.
Positive interim data from a Phase I/II trial of RGX-111 were
reported in February 2023. RGX-111
has received Orphan Drug Product, Rare Pediatric Disease and Fast
Track designations from the U.S. Food and Drug
Administration.
ABOUT REGENXBIO Inc.
REGENXBIO is a leading
clinical-stage biotechnology company seeking to improve lives
through the curative potential of gene therapy. Since its founding
in 2009, REGENXBIO has pioneered the development of AAV
Therapeutics, an innovative class of gene therapy medicines.
REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal
and rare diseases, including ABBV-RGX-314 for the treatment of wet
AMD and diabetic retinopathy, being developed in collaboration with
AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the
treatment of MPS II. Thousands of patients have been treated with
REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA
for children with spinal muscular atrophy. Designed to be one-time
treatments, AAV Therapeutics have the potential to change the way
healthcare is delivered for millions of people. For more
information, please visit www.regenxbio.com.
ABOUT NIPPON SHINYAKU
Based on Nippon
Shinyaku's business philosophy, "Helping people lead healthier,
happier lives," we aim to be an organization trusted by the
community through creating unique medicines that will bring hope to
patients and families suffering from illness. Please visit our
website (www.nippon-shinyaku.co.jp/english/) for products or
detailed information.
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 collaboration with Nippon Shinyaku and
REGENXBIO's future operations, clinical trials, and regulatory
plans. 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
anticipated completion of REGENXBIO's proposed transaction with
Nippon Shinyaku, the outcome of REGENXBIO's proposed collaboration
with Nippon Shinyaku, whether the milestones contemplated by the
proposed transaction will be achieved, the timing of enrollment,
commencement and completion and the success of clinical trials
conducted by REGENXBIO, 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,
2023, 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:
George E. MacDougall
Investor Relations
IR@regenxbio.com
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