ROCKVILLE, Md. and ZURICH, 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 and Neurimmune
AG, a leading clinical-stage Swiss biotech company translating
human immune memory into antibody therapeutics, today announced 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.
"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. "We are thrilled to initiate our partnership with
Neurimmune, which builds on promising results to date with our
RGX-314 clinical-stage program, where we are using our NAV Vectors
to deliver a therapeutic antibody for the treatment of wet
AMD."
"Neurimmune's Reverse Translational Medicine technology decodes
the genetic information of immune cells obtained from healthy aged
people to identify next generation antibody therapeutics," said
Jan Grimm, Ph.D., Chief Scientific
Officer of Neurimmune. "We are excited to partner with REGENXBIO
and combine our human antibody technology with REGENXBIO's AAV
expertise to develop vectorized human antibodies as a novel
treatment modality. We believe the potential to deliver sustained
levels of therapeutic human antibodies to the brain with AAV
vectors may have a significant impact on chronic neurodegenerative
conditions."
Under the exclusive license, development and commercialization
agreement, REGENXBIO and Neurimmune will jointly develop and
commercialize novel therapies using AAV vectors to deliver human
antibodies. Initially, the companies will focus on diseases
associated with the accumulation and deposition of the
microtubule-associated protein tau (tauopathies). REGENXBIO and
Neurimmune will be jointly responsible for the design and
development of vectorized antibody therapies and will share
associated development costs equally. Following an initial research
phase, on a target-by-target basis, each party will have the option
to continue as a co-development and co-commercialization partner in
the collaboration or to elect to receive a phase-based worldwide
royalty in lieu of continued development investment. 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.
Tau is an abundant protein with a primary function to stabilize
microtubules, a structural component of healthy neurons. Abnormal
forms of tau can misfold and accumulate in neurofibrillary tangles
that are hallmarks of several neurodegenerative diseases, including
Alzheimer's disease, corticobasal degeneration, frontotemporal
dementia, Pick's disease and progressive supranuclear palsy,
referred to collectively as tauopathies. Delivery of human
antibodies using AAV vectors has the potential to provide sustained
brain exposure of antibodies for the clearance of abnormal tau via
a one-time CNS administration.
REGENXBIO NAV Vectors for AAV-Mediated Antibody
Delivery
REGENXBIO has established foundational technology,
intellectual property, 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 to treat serious and chronic diseases.
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 making treatments possible in tissues
where it is difficult to deliver sufficient amounts of therapeutic
antibodies via traditional delivery methods, such as the central
nervous system.
Neurimmune's Reverse Translational Medicine
Platform
Reverse Translational Medicine (RTM™) is
Neurimmune's proprietary technology platform. It is based on the
scientific understanding and high-throughput analyses of human
immune responses to disease-related proteins in selected
populations including elderly with the capability to stay healthy
during the aging process. The technology translates the genetic
information obtained from human white blood cells into selective
high affinity antibody candidates. Neurimmune's human monoclonal
antibody therapeutics combine the full range of advantages provided
by human affinity maturation and tolerance selection. These
processes were optimized through human evolution over millions of
years for unsurpassed efficacy and safety.
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 (NAV Vectors). 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.
About Neurimmune AG
Neurimmune is a clinical-stage Swiss biotech company translating
human immune memory into therapeutics. Neurimmune's pipeline
comprises high-potential drug candidates at both clinical and
advanced preclinical development stages. Rights in antibodies
BIIB054 for Parkinson's disease and BIIB076 for Alzheimer's disease
were acquired by Biogen. Neurimmune has partnered with Biogen, TVM
and Eli Lilly's Chorus unit and Ono Pharmaceutical to discover and
develop human monoclonal antibodies for neurodegenerative diseases.
Neurimmune's pipeline includes human antibody programs for
neurodegenerative diseases, cardiomyopathy, type-2 diabetes as well
as a small molecule program for cognitive dysfunction.
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:
For REGENXBIO
Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
For Neurimmune
Business Development:
Fabian Buller, +41 44 755 46 46
fabian.buller@neurimmune.com
Media:
Helen O'Gorman, 212-850-5629
helen.ogorman@fticonsulting.com
View original content to download
multimedia:http://www.prnewswire.com/news-releases/regenxbio-and-neurimmune-announce-collaboration-to-develop-vectorized-antibodies-for-the-treatment-of-neurodegenerative-diseases-300889914.html
SOURCE REGENXBIO Inc.