ROCKVILLE, Md., July 24, 2019 /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 an expansion of its pipeline to include
novel treatments for hereditary angioedema (HAE) and
neurodegenerative diseases, including tauopathies, using NAV
Vectors to deliver therapeutic antibodies.
REGENXBIO's new gene therapy product candidate for the treatment
of HAE utilizes NAV Vectors that are designed to deliver a gene
encoding a therapeutic antibody that targets and binds to plasma
kallikrein, a key protein left unregulated in patients with
HAE.
In addition, REGENXBIO and Neurimmune AG announced earlier today
an exclusive license, development and commercialization agreement
to discover and develop novel AAV gene therapies using NAV Vectors
to deliver human antibodies against targets implicated in chronic
neurodegenerative diseases, including tauopathies.
"Using NAV Vectors to deliver therapeutic antibodies has
enormous potential for patients who lack treatments or who are
currently underserved by existing therapies, and provides a
significant opportunity to expand our pipeline through the
application of our AAV-mediated antibody delivery capabilities and
expertise to a number of validated and new targets in multiple
therapeutic areas and tissues," said Kenneth T. Mills, President and Chief Executive
Officer of REGENXBIO. "We are thrilled to announce our research
program for the treatment of HAE, a chronic, life-threatening
disease characterized by recurring severe swelling, most commonly
in the face, airway, intestines and limbs, for which a one-time
AAV-mediated antibody delivery approach may provide significant
benefit for patients. In addition, we are excited to partner with
Neurimmune and we believe the combination of Neurimmune's
human-derived antibodies with REGENXBIO's AAV expertise creates a
unique opportunity to develop significant therapies for chronic
neurodegenerative diseases."
"REGENXBIO is at the forefront of research and development of
AAV-mediated antibody gene therapies," said Olivier Danos, Ph.D., Chief Scientific Officer
of REGENXBIO. "Our pipeline expansion through our research for the
treatment of HAE and our previously announced partnership with
Neurimmune builds on promising results with our clinical-stage
RGX-314 program, where we are using our NAV Vectors to deliver a
therapeutic antibody for the treatment of wet AMD."
NAV Vectors for AAV-Mediated Antibody Delivery
REGENXBIO has established a new treatment modality to treat
serious and chronic diseases based on foundational technology and
intellectual property, as well as scientific, clinical and
manufacturing expertise for the development of one-time treatments
in multiple disease areas based on AAV-mediated antibody delivery
using NAV Vectors.
These AAV-mediated antibody delivery treatments share similar
gene therapy delivery technology and manufacturing processes, and
may potentially share similar pharmacology profiles, including,
safety and tolerability, and medicinal properties. Product
candidates in this modality will be designed to modify some of the
patients' own cells and may result in the sustained production of
therapeutic antibodies in vivo.
REGENXBIO's lead product candidate, RGX-314, consists of the NAV
AAV8 vector encoding a gene for an antibody fragment that binds
vascular endothelial growth factor (VEGF). RGX-314 is being
developed as a novel, one-time subretinal treatment for wet
age-related macular degeneration (wet AMD) and diabetic
retinopathy.
Existing standard of care for many diseases involves frequent
administration of therapeutic antibodies. A single administration
AAV gene therapy approach using NAV Vectors may provide improved
treatment options for patients with these diseases by reducing
their treatment burden or enabling treatments in tissues where it
is difficult to deliver sufficient amounts of therapeutic
antibodies via traditional delivery methods, such as in the central
nervous system.
REGENXBIO Program for the Treatment of Hereditary
Angioedema
HAE is a chronic and severe disease that results from
C1-inhibitor (C1-INH) deficiency. HAE is characterized by recurring
severe swelling (angioedema), most commonly in the face, airway,
intestines and limbs. Antibodies to plasma kallikrein, a key
protein left unregulated in patients with HAE, have been shown to
reduce the swelling and pain associated with HAE. These antibodies,
however, require frequent administration to reduce the occurrence
of angioedema events.
REGENXBIO's HAE program is focused on developing a novel,
one-time treatment utilizing a NAV Vector to deliver a gene
encoding for a therapeutic antibody that targets and binds to
plasma kallikrein. Following a single intravenous administration,
REGENXBIO's HAE product candidate is designed to allow liver cells
to produce therapeutic antibodies that are secreted into the blood.
In preclinical animal models, REGENXBIO has used NAV Vectors to
express therapeutic antibodies that target and bind to plasma
kallikrein. REGENXBIO expects to provide an update in early 2020 on
the HAE preclinical studies, as well as plans for entering clinical
trials.
REGENXBIO and Neurimmune Collaboration
As previously announced, REGENXBIO and Neurimmune will jointly
develop and commercialize novel therapies using AAV vectors to
deliver human antibodies with an initial focus on diseases
associated with the accumulation and deposition of the
microtubule-associated protein tau (tauopathies). The companies
have initiated their first exclusive collaboration program for the
treatment of tauopathies and will provide updates on program
progress as the collaboration advances.
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.
REGENXBIO 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,
research and development activities, preclinical studies 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, 2018, 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:
Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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