Amicus Therapeutics Announces Presentations and Posters at 14th Annual WORLDSymposium™ 2018
January 22 2018 - 4:01PM
Amicus Therapeutics (Nasdaq:FOLD), today announced that two oral
presentations and seven posters highlighting its development
programs for Lysosomal Storage Disorders will be included at the
14th Annual WORLDSymposium™ 2018, to be held February 5-9, 2018 in
San Diego, CA.
Oral Platform Presentations:
Pompe Disease:
- Updated results from ATB200-02: a first-in-human, open-label,
phase 1/2 study of ATB200 co-administered with AT2221 in adults
with Pompe disease – Tahseen Mozaffar, MD, Director, Neuromuscular
Program, Neurology School of Medicine at UC Irvine (Thursday,
February 8 at 1:15 p.m. PT)
Fabry Disease:
- Ten years of migalastat treatment in a patient with Fabry
disease: a case report – Raphael Schiffmann, MD, Baylor Research
Institute (Thursday, February 8 at 1:45 p.m. PT)
Poster Session: Tuesday, February 6, 4:30-6:30pm
PT
Fabry Disease:
- Pregnancy outcome after exposure to
migalastat: a case study – Gere Sunder-Plassmann, MD, Department of
Medicine III, Division of Nephrology and Dialysis, Medical
University of Vienna, Vienna, Austria (Poster #129)
- Cardiac outcomes with long-term migalastat treatment in
patients with Fabry disease: results from phase 3 trials –
Dominique P. Germain, MD, PhD, Division of Medical Genetics at the
University of Versailles and Assistance Publique - Hôpitaux de
Paris (Poster #LB-18)
Pompe Disease:
- First-in-human preliminary pharmacokinetic data on a novel
recombinant acid α-glucosidase, ATB200, co-administered with the
pharmacological chaperone, AT2221, in patients with late-onset
Pompe disease – Franklin Johnson, MS, Amicus Therapeutics, Inc.
Cranbury, USA. (Poster #168)
Poster Session: Wednesday, February 7, 4:30-6:30pm
PT
Fabry Disease:
- Renal outcomes with up to 9 years of migalastat in patients
with Fabry disease: results from an open-label extension study –
Kathleen Nicholls, MD, Royal Melbourne Hospital (Poster
#270)
- A next-generation Fabry enzyme replacement therapy: a
proprietary human α-galactosidase A co-formulated with a
pharmacological chaperone, AT1001, shows greater substrate
reduction than standard of care in Fabry mice – Su Xu, PhD, Amicus
Therapeutics, Inc. Cranbury, USA (Poster #408)
Pompe Disease:
- The patient and clinician point of view: living with late-onset
Pompe disease – Nita Patel, RN, GCN, CCR, Amicus Therapeutics, Inc.
Cranbury, USA (Poster #298)
- Updated results from ATB200-02: a first-in-human, open-label,
phase 1/2 study of ATB200 co-administered with AT2221 in adults
with Pompe disease – Tahseen Mozaffar, MD, Director, Neuromuscular
Program, Neurology School of Medicine at UC Irvine (Poster
#LB-38)
The goal of the WORLDSymposia is to provide
an interdisciplinary forum to explore and discuss specific areas of
interest, research, and clinical applicability related to lysosomal
diseases. Each year, WORLDSymposia hosts a scientific meeting
presenting the latest information from basic science, translational
research, and clinical trials for lysosomal diseases. This
symposium is designed to help researchers and clinicians to better
manage and understand diagnostic options for patients with
lysosomal diseases, identify areas requiring additional basic and
clinical research, public policy and regulatory attention, and
identify the latest findings in the natural history of lysosomal
diseases. For more information please visit
www.worldsymposia.org.
About Amicus TherapeuticsAmicus
Therapeutics (Nasdaq:FOLD) is a global, patient-centric
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. The cornerstone of the Amicus portfolio is
migalastat, an oral precision medicine for people living with Fabry
disease who have amenable genetic mutations. Migalastat is
currently approved under the trade name Galafold™ in the European
Union, with additional approvals granted and pending in several
geographies. The future value driver of the Amicus pipeline is
ATB200/AT2221, a novel, late-stage, potential best-in-class
treatment paradigm for Pompe disease. The Company is committed to
advancing and expanding a robust pipeline of cutting-edge, first-
or best-in-class medicines for rare metabolic diseases.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to preclinical and clinical development of our 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, with respect to statements
regarding clinical trials, actual results may differ materially
from those set forth in this release due to the risks and
uncertainties inherent in our business, including, without
limitation: 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; and the
potential that we will need additional funding to complete all of
our studies. Further, the results of earlier preclinical studies
and/or clinical trials may not be predictive of future results for
any of our 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, 2016. 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 news release to reflect events or circumstances after
the date hereof.
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCSenior Director, Investor
Relationsspellegrino@amicusrx.com (609) 662-5044
Media:Pure
CommunicationsJennifer Paganelli jpaganelli@purecommunications.com
(347) 658-8290
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