Amicus Therapeutics (Nasdaq: FOLD) and the Perelman School of
Medicine at the University of Pennsylvania (Penn) today announced a
major expansion to their collaboration with rights to pursue
collaborative research and development of novel gene therapies for
lysosomal disorders (LDs) and 12 additional rare diseases. The
collaboration has been expanded from three to six programs for rare
genetic diseases and now includes: Pompe disease, Fabry disease,
CDKL5 deficiency disorder (CDD), Niemann-Pick Type C (NPC),
Mucopolysaccharidosis Type IIIB (MPS IIIB), as well as a next
generation program in Mucopolysaccharidosis Type IIIA (MPS IIIA),
both also known as part of Sanfillipo Syndrome. In addition to
these three new programs, a discovery research agreement provides
Amicus with exclusive disease-specific access to rights to
collaborate with Penn’s Gene Therapy Program (GTP) to develop
potentially disruptive new gene therapy platform technologies and
programs for the majority of lysosomal disorders and 12 additional
rare diseases.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus stated, “The extension of our collaboration with
Penn is a bold step forward in our commitment to create potential
cures that may alleviate an enormous amount of suffering for
countless numbers of people in the world living with rare diseases,
many of them children. Together with Penn we are now able to focus
on additional lysosomal disorders, as well as several more
prevalent rare diseases for which we can apply our understanding of
underlying disease biology in rare metabolic disease, Amicus’
protein-engineering and development expertise and the world
renowned capabilities of Dr. Jim Wilson’s laboratory to develop
novel gene therapy candidates. With a globally approved precision
medicine product for Fabry, a late-stage biologic product with
breakthrough therapy designation for Pompe, and now the industry’s
largest rare disease gene therapy pipeline, Amicus is
well-positioned to become a leading global biotechnology company at
the forefront of human genomic medicine.”
Building off the initial success of the ongoing
Amicus-Penn collaboration, including compelling initial preclinical
proof-of-concept data in Pompe disease, this expanded relationship
will continue to combine Amicus’ protein engineering and
glycobiology expertise with Penn’s gene transfer technologies to
develop novel gene therapies designed for optimal cellular uptake,
targeting, dosing, safety and manufacturability.
“This agreement is a significant step forward in
creating a world class industry-academia gene therapy partnership
in rare diseases,” said James M. Wilson, MD PhD, Professor of
Medicine and Pediatrics at the Perelman School of Medicine. “We
have already seen highly encouraging preclinical results and
proof-of-concept in Pompe disease through our existing
collaboration and are excited by what we can further achieve
together. We are looking forward to expanding the relationship
further for additional preclinical programs and committing to the
research required to further advance the technology platforms at
Penn. We have seen the first results of our combined capabilities
and platforms and I believe that we can further expand and
accelerate our efforts to rapidly develop gene therapies for many
more patients with unmet needs.”
“Penn Medicine has put Philadelphia on the map
as the global epicenter of gene therapy research and development,
and under the leadership and vision of Jim Wilson, our expanded
agreement with Amicus is an exciting milestone for a field which is
in the midst of transformative breakthroughs,” said J. Larry
Jameson, MD, PhD, Executive Vice President of the University of
Pennsylvania for the Health System and Dean of the Perelman School
of Medicine. “We are thrilled to be part of this collaboration,
which will help to bolster our city’s growing reputation as a
magnet for talent and an engine for gene therapy innovation.”
Extended Research, Collaboration and
License Agreement for Six Rare Metabolic DiseasesPenn’s
vector technology is designed to improve targeting, tropism,
safety, immunogenicity, and gene delivery, while Amicus’ protein
engineering capabilities may optimize protein expression,
secretion, targeting and uptake of the target protein. The
agreement between Amicus and Penn is a Research, Collaboration and
License Agreement, with funding provided to Penn to advance the
preclinical research programs in the Wilson Lab and to license
certain technologies invented under the funded Research
Collaboration. The initial collaboration program focused on
developing innovative new gene therapies for Pompe disease, Fabry
disease, CDD and one additional undisclosed rare metabolic
disorder. With the extension of the agreement, NPC and MPS IIIB, as
well as a next-generation program for MPS IIIA, have now been added
as three additional collaboration
programs.
New Next Generation Research Program: R&D Engine for
LDs and Additional Rare DiseasesUnder a new five-year
next-generation research agreement, Penn will conduct discovery
research to develop potentially disruptive new gene therapy
technologies. Amicus will continue to advance its own research and
technology platforms to combine with Penn’s technologies, which can
be used in the collaborative research programs for the disease
indications. Terms of the agreement include a $10 million annual
investment from Amicus, each year for five years, into GTP’s
discovery research program with the ability to extend. Amicus will
receive exclusive disease-specific rights to collaborate with GTP
to research and develop products for many lysosomal disorders. The
Amicus rights for additional collaborative research programs also
include additional rare diseases, including Rett Syndrome, Angelman
Syndrome, Myotonic Dystrophy and select other muscular dystrophies.
For many of these indications, there is potential to apply
protein-engineering and targeting motifs to enable cross-correction
with next-generation gene therapy technology.
Conference Call and Webcast on May 29,
2019 at 8:30 a.m. ETAmicus Therapeutics will host a
conference call and audio webcast today, May 29, 2019 at 8:30 a.m.
ET to discuss the expanded collaboration. Interested participants
and investors may access the conference call by dialing
877-303-5859 (U.S./Canada) or 678-224-7784 (international),
conference ID: 1695255.
A live audio webcast and accompanying slide deck
can also be accessed via the Investors section of the Amicus
Therapeutics corporate website at http://ir.amicusrx.com/, and will
be archived for 30 days. Web participants are encouraged to
register on the website 15 minutes prior to the start of the call.
A replay of the call will be available for seven days beginning at
11:30 a.m. ET on May 29, 2019. Access numbers for this replay are
855-859-2056 (U.S./Canada) and 404-537-3406 (international);
conference ID: 1695255.
About Amicus TherapeuticsAmicus
Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. With extraordinary patient focus, Amicus
Therapeutics is committed to advancing and expanding a robust
pipeline of cutting-edge, first- or best-in-class medicines for
rare metabolic diseases. For more information please visit the
company’s website at www.amicusrx.com.
About the Perelman School of Medicine at
the University of PennsylvaniaPenn Medicine is one of
the world’s leading academic medical centers, dedicated to the
related missions of medical education, biomedical research, and
excellence in patient care. Penn Medicine consists of
the Raymond and Ruth Perelman School of Medicine at the
University of Pennsylvania (founded in 1765 as the nation’s
first medical school) and the University of Pennsylvania
Health System, which together form a $7.8 billion enterprise. The
Perelman School of Medicine has been ranked among the top medical
schools in the United States for more than 20 years, according
to U.S. News & World Report's survey of research-oriented
medical schools. The School is consistently among the nation's top
recipients of funding from the National Institutes of Health, with
$425 million awarded in the 2018 fiscal year. The University of
Pennsylvania Health System’s patient care facilities include: the
Hospital of the University of Pennsylvania and Penn Presbyterian
Medical Center—which are recognized as one of the nation’s top
“Honor Roll” hospitals by U.S. News & World Report—Chester
County Hospital; Lancaster General Health; Penn Medicine Princeton
Health; and Pennsylvania Hospital, the nation’s first hospital,
founded in 1751. Additional facilities and enterprises include Good
Shepherd Penn Partners, Penn Home Care and Hospice Services,
Lancaster Behavioral Health Hospital, and Princeton House
Behavioral Health, among others. Penn Medicine is powered by a
talented and dedicated workforce of more than 40,000 people. The
organization also has alliances with top community health systems
across both Southeastern Pennsylvania and Southern New Jersey,
creating more options for patients no matter where they live. Penn
Medicine is committed to improving lives and health through a
variety of community-based programs and activities. In fiscal year
2018, Penn Medicine provided more than $525 million to benefit our
community.
Forward-Looking StatementThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to the collaboration with the University of Pennsylvania,
and the development of potential gene therapy product candidates.
The inclusion of forward-looking statements should not be regarded
as a representation by us that any of our plans will be achieved.
Any or all of the forward-looking statements in this press release
may turn out to be wrong and can be affected by inaccurate
assumptions we might make or by known or unknown risks and
uncertainties. For example, the benefits of this collaboration may
never be realized, the potential that results of clinical or
preclinical studies indicate that the product candidates are unsafe
or ineffective; the potential that it may be difficult to enroll
patients in our clinical trials; the potential that regulatory
authorities, including the FDA, EMA, and PMDA, may not grant or may
delay approval for our product candidates; the potential that
preclinical and clinical studies could be delayed because we
identify serious side effects or other safety issues; the potential
that we may not be able to manufacture or supply sufficient
clinical or commercial products; the potential that we will need
additional funding to complete all of our studies and manufacturing
and the potential that certain individuals may not continue to
support the development of product candidates. In addition, all
forward-looking statements are subject to other risks detailed in
our Annual Report on Form 10-K for the year ended December 31, 2018
as well as our Quarterly Report on Form 10-Q for the quarter ended
March 31, 2019. You are cautioned not to place undue reliance on
these forward-looking statements, which speak only as of the date
hereof. All forward-looking statements are qualified in their
entirety by this cautionary statement, and we undertake no
obligation to revise or update this presentation to reflect events
or circumstances after the date hereof.
Penn’s Financial Disclaimer -
As an inventor of technology licensed or optioned to Amicus
pursuant to this Gene Therapy Collaboration, Dr. Wilson and Penn
may receive additional financial benefits under the license in the
future.
CONTACTS:
Investors/Media:Amicus TherapeuticsSara
Pellegrino, IRCVice President, Investor Relations & Corporate
Communicationsspellegrino@amicusrx.com (609) 662-5044
Media:Amicus TherapeuticsMarco WinklerDirector,
Corporate Communicationsmwinkler@amicusrx.com(609) 662-2798
Penn Medicine:Karen KreegerSenior Science
Communications Manager
Karen.kreeger@pennmedicine.upenn.edu (215) 459-0544
FOLD–G
Amicus Therapeutics (NASDAQ:FOLD)
Historical Stock Chart
From Feb 2024 to Mar 2024
Amicus Therapeutics (NASDAQ:FOLD)
Historical Stock Chart
From Mar 2023 to Mar 2024