- Six-month data from single-patient, investigator-initiated
trial showed that one-time administration of RGX-181 was well
tolerated, achieved sustained gene expression and demonstrated
clinically meaningful improvements across multiple measures
including reduced (86%) seizure frequency.
- Investigators observed encouraging neurodevelopmental skill
acquisition at 6 months
ROCKVILLE, Md., Aug. 30,
2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:
RGNX) today announced that initial interim data from a
first-in-human single-patient, investigator-initiated trial of
RGX-181 for the treatment of late-infantile neuronal ceroid
lipofuscinosis type 2 (CLN2) disease, a form of Batten disease,
were presented at the Society for the Study of Inborn Errors of
Metabolism (SSIEM) Annual Symposium in Jerusalem.
"CLN2 is a debilitating disease caused by mutations in the
CLN2 gene resulting in a deficiency of the TPP1 enzyme,
which is needed to break down specific peptides associated with
cellular waste. Symptoms include seizures; loss of motor, language,
and cognitive skills; vision loss and premature death; and existing
treatments do not stop or reverse most manifestations of the
disease," said Steve Pakola, M.D.,
Chief Medical Officer of REGENXBIO. "We are encouraged by the
initial results demonstrating that RGX-181 is well tolerated and
dramatically reduced the number of seizures in the patient enrolled
in this trial."
"As someone who treats patients with this devastating disease, I
see the limitations of the current standard of care," said
Carolina Fischinger de Souza, M.D.,
Ph.D., Hospital de Clínicas de Porto
Alegre, Brazil and investigator of this trial. "The
remarkable decrease in seizures, encouraging safety results and
reduction in ERT frequency highlight the potential of this gene
therapy to provide a meaningful treatment option to the CLN2
patient community."
Data Summary and Safety Data
Today, a physician investigator from the Hospital de
Clinicas in Porto Alegre, Brazil reported initial results
from a five-year-old child who received a one-time intracisternal
dose of RGX-181. Time of post-administration follow up was six
months.
As of June 30, 2023, RGX-181 was
well tolerated with no serious adverse events. Key efficacy
measures demonstrated sustained levels of TPP1 along with increased
intervals between enzyme replacement therapy (ERT) infusions and an
86% reduction in seizure frequency through six months, leading to
withdrawal of two anti-epileptic medications. Encouraging
improvements in fine motor and expressive language skills were also
observed.
"These results represent the third consecutive program in our
clinical-stage pipeline for rare neurodegenerative conditions that
have shown a potential one-time gene therapy is well tolerated and
results in physiologically relevant levels of gene expression and
clinically meaningful changes in young children. We have received
important regulatory designations for each of these three pipeline
programs," said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. "Our first BLA
filing for a rare neurodegenerative disease, Hunter syndrome, is
planned for 2024 using the Accelerated Approval pathway, and we
look forward to advancing our additional investigational AAV
therapeutics as quickly as possible."
About RGX-181
RGX-181 is being developed as a novel, one-time treatment for
CLN2 disease utilizing the NAV AAV9 vector to deliver the gene
encoding for TPP1, the enzyme deficient in children with CLN2
disease. Following administration of a single intracisternal
injection, RGX-181 treatment is designed to provide a durable
source of TPP1, potentially leading to long-term correction of
cells throughout the CNS. In an animal model for CLN2 disease,
treatment with RGX-181 has been shown to restore TPP1 activity to
levels greater than those in non-affected animals and to improve
neurobehavioral function and survival. The extent of CNS correction
observed suggests that RGX-181 has the potential to be an important
and suitable one-time therapeutic option for patients with CLN2
disease.
About CLN2 Disease
Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2)
disease, a form of Batten disease, is a rare, pediatric-onset,
autosomal recessive, neurodegenerative lysosomal storage disorder
caused by mutations in the TPP1 gene. Deficiency in TPP1 enzymatic
activity results in lysosomal accumulation of storage material and
degeneration of nerve cells, particularly in the brain and retina.
CLN2 disease is characterized by seizures, rapid deterioration of
language and motor functions, cognitive decline, rapid loss of
vision and blindness, and premature death by mid-childhood. Onset
of symptoms is generally between two to four years of age with
initial features of recurrent seizures (epilepsy), language delay
and difficulty coordinating movements (ataxia). There is currently
no cure for CLN2 disease. Current treatment options include CNS
enzyme replacement therapy, wherein recombinant TPP1 is
administered into the lateral ventricles via a permanently
implanted device on a biweekly basis, and palliative care. There
are currently no approved treatments to treat ocular manifestations
of CLN2 disease.
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, clinical trials, costs and cash flow. 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, 2022, 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:
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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