- RMAT recognizes that the preliminary clinical evidence from
RGX-121, a potential one-time AAV Therapeutic, indicates the
potential to address unmet medical needs for MPS II
- RMAT designation is for gene therapies
intended to treat or cure serious condition in order to expedite
the drug development and review processes
- CAMPSIITE™ trial is enrolling MPS II patients as part of a
pivotal program that incorporates material from the NAVXpress™
platform process manufactured at the REGENXBIO Manufacturing
Innovation Center and continues to support plan to file Biologics
License Application in 2024 using the accelerated approval
pathway
ROCKVILLE, Md., May 23, 2023
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
that the U.S. Food and Drug Administration (FDA) has granted
Regenerative Medicine Advanced Therapy (RMAT) designation for
RGX-121, an investigational one-time AAV Therapeutic for the
treatment of Mucopolysaccharidosis Type II (MPS II), also known as
Hunter syndrome. RMAT designation is designed to expedite the drug
development and review processes for promising new treatments,
including gene therapies, and recognizes that the preliminary
clinical evidence from RGX-121 indicates its potential to address
unmet medical needs for MPS II. RGX-121 is currently being studied
in the CAMPSIITE™ trial that is enrolling MPS II patients as part
of a pivotal program that incorporates material from the NAVXpress™
platform process manufactured at the REGENXBIO Manufacturing
Innovation Center and continues to support plans to file Biologics
License Application (BLA) in 2024 using the accelerated approval
pathway.
"We are pleased that the FDA has granted RMAT designation for
RGX-121 for its potential to address the neurological
manifestations of MPS II and prevent or stabilize cognitive
decline, which remains a significant unmet need for this
community," said Steve Pakola, M.D.,
Chief Medical Officer of REGENXBIO. "This is an important
regulatory milestone, and we remain on track to complete enrollment
of CAMPSIITE in the first half of 2023 to support a BLA filing in
2024 using the accelerated approval pathway."
Established under the 21st Century Cures Act, a drug is eligible
for RMAT designation if it is intended to treat, modify, reverse or
cure a serious or life-threatening disease or condition, and
preliminary clinical evidence indicates that the drug or therapy
has the potential to address unmet medical needs for such disease
or condition.
RMAT designation includes the benefits of early FDA interactions
to discuss surrogate or intermediate endpoints, potential ways to
support accelerated approval and satisfy post-approval
requirements, potential priority review of the BLA and other
opportunities to expedite development and review. In addition,
sponsors of products that have been granted RMAT designation and
which received accelerated approval may be able to fulfill the
post-approval requirements from clinical evidence obtained from
sources other than the traditional confirmatory clinical
trials.
"Severe Hunter syndrome is a progressively debilitating disorder
that affects a child's physical and mental development and leads to
a shortened lifespan, and I applaud the FDA for granting RMAT
designation to RGX-121," said Joseph
Muenzer, M.D., Ph.D., Director, Muenzer MPS
Research and Treatment Center, Bryson Distinguished
Professor in Pediatrics Genetics, University
of North Carolina at Chapel Hill. "I am encouraged by the
FDA's recognition of the potential of a gene therapy like RGX-121
to improve the quality of life for boys with severe Hunter syndrome
and the urgent need for new treatment options."
"We know that families are waiting for new treatment options for
this serious disease and that every day matters," said Terri Klein, President and Chief Executive
Officer, National MPS Society. "The expedited development of new
treatment options like RGX-121 are critical to families facing this
devastating diagnosis."
RGX-121 Program Highlights
The Phase I/II/III CAMPSIITE™ trial of RGX-121 for MPS II patients
aged 4 months up to 5 years is ongoing and in February 2023, REGENXBIO announced additional
interim data from the Phase I/II part of the trial, demonstrating
that RGX-121 continued to be well-tolerated across 15 patients.
Patients receiving the pivotal program dose level continued to
demonstrate the largest reductions in CSF GAGs, including Heparin
Sulfate (HS) and HS D2S6, which approached normal levels at 48
weeks. CSF GAGs 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. In addition,
improvements in neurodevelopmental and daily activity skill
acquisition were observed up to three years after RGX-121
administration.
A Phase I/II trial of RGX-121 for the treatment of pediatric
patients with MPS II over the age of five years old is also
ongoing.
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. 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.
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), including heparan sulfate (HS) 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 include its substrate
heparan sulfate (HS) D2S6, 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 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.
Contacts:
Dana Cormack
Corporate
Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey, ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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