- Aligned with FDA on content of BLA and plans for
submission:
- Submission of a rolling BLA using the accelerated approval
pathway expected to start in Q3 2024
- Confirmatory trial expected to begin in H2 2025
- FDA confirmed RGX-121 commercial bulk drug is comparable to
clinical material
- Positive biomarker, neurocognitive and systemic data will be
part of BLA submission
ROCKVILLE, Md., June 18,
2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced it completed a successful Pre-Biologics
License Application (BLA) meeting for RGX-121 for the treatment
Mucopolysaccharidosis Type II (MPS II), where it finalized details
of its BLA with the U.S. Food and Drug Administration (FDA).
The FDA continues to be aligned with REGENXBIO's plan to use
cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a
key biomarker of brain disease activity, as a surrogate endpoint
reasonably likely to predict clinical benefit to support
accelerated approval of RGX-121. Additionally, REGENXBIO and the
FDA discussed manufacturing, non-clinical, device delivery system
and other critical elements of the BLA, including a confirmatory
study designed to verify and describe the predicted clinical
benefit.
REGENXBIO completed its database lock for the pivotal program
and expects to initiate submission of a rolling BLA in the third
quarter of 2024. REGENXBIO expects an FDA inspection of its
Manufacturing Innovation Center in the first half of 2025.
Commercial bulk drug and clinical trial material, both manufactured
using REGENXBIO's proprietary NAVXpress™ platform process, were
confirmed to be comparable.
A confirmatory study of RGX-121 is expected to initiate
enrollment in the second half of 2025, prior to potential FDA
approval. Based on an expected priority review, potential approval
of the planned BLA could result in receipt of a Rare Pediatric
Disease Priority Review Voucher in 2025.
"This positive engagement with the FDA marks an important
milestone for REGENXBIO and RGX-121 on the path towards potential
approval of the first gene therapy for Hunter syndrome," said
Curran Simpson, Chief Operating
Officer of REGENXBIO and President and CEO-elect. "Alignment with
the FDA on important elements of this BLA, like our proprietary
NAVXpress™ platform process and inspection of our commercial-ready
manufacturing facility, provide a solid foundation as we advance to
pivotal stage for our other rare program, RGX-202, and work to
diligently to expedite its development for the Duchenne
community."
"The MPS II community is in need of new treatment options that
have the potential to address the neurocognitive impacts of this
devastating disease," said Dr. Matthew
Ellinwood, Chief Scientific Officer of the National MPS
Society. "We are pleased with REGENXBIO's continued positive
discussions with and are encouraged by the totality of the
biomarker, neurocognitive and systemic data for RGX-121 and the
impact its potential approval may have for the families we
serve."
Topline results from the Phase I/II/III CAMPSIITE®
trial of RGX-121 presented earlier this year demonstrated that the
pivotal phase of the trial met its primary endpoint with
statistical significance. Pivotal results were consistent with data
from the dose-finding phase of CAMPSIITE, in which the majority
of patients were shown to be exceeding expectations in
neurodevelopmental function compared to natural history data up to
four years. Long-term follow-up of patients treated with RGX-121
also showed there was a high rate of patients for whom trial
investigators chose to discontinue standard-of-care intravenous
enzyme replacement therapy (ERT) or were allowed to remain
ERT-naïve. As of January 3,
2024, RGX-121 continues to be well tolerated in 25 patients
dosed across all phases of the CAMPSIITE trial. REGENXBIO expects
to share additional safety and efficacy data from the CAMPSIITE
trial in the second half of 2024.
About the CAMPSIITE® Trial
CAMPSIITE is a
Phase I/II/III multicenter, open-label trial for boys aged four
months up to five years with neuronopathic MPS II. The
primary endpoint of the trial is measurement of CSF GAGs. Accurate
and sensitive measurements of CSF GAGs, such as HS D2S6, have the
potential to be considered a surrogate endpoint 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.
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.
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. RGX-21
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 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 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. 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.
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, 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.
Zolgensma® is a registered trademark of Novartis Gene
Therapies. All other trademarks referenced herein are registered
trademarks of REGENXBIO.
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
View original content to download
multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-successful-pre-bla-meeting-with-fda-to-support-accelerated-approval-pathway-for-rgx-121-for-the-treatment-of-mps-ii-302175172.html
SOURCE REGENXBIO Inc.