Amicus Therapeutics Announces Presentations and Posters at 13th Annual WORLDSymposium™ 2017
January 24 2017 - 7:30AM
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
at the forefront of therapies for rare and orphan diseases, today
announced that 3 oral presentations and 9 posters highlighting its
development programs for Lysosomal Storage Disorders will be
included at the 13th Annual WORLDSymposium™ 2017, to be held
February 13-17, 2017 in San Diego, CA.
Oral Platform Presentations:
Pompe Disease:
- Stabilized next-generation recombinant human acid
alpha-glucosidase ATB200 clears accumulated glycogen and reverses
cellular dysfunction to increase functional muscle strength in a
mouse model of Pompe disease – Hung Do, PhD, Amicus Therapeutics,
Inc. (Wednesday, February 15 at 2:15 p.m. PT)
Fabry Disease:
- Efficacy and safety of migalastat, an oral pharmacologic
chaperone for Fabry disease: results from two randomized phase 3
studies, FACETS and ATTRACT – Ulla Feldt-Rasmussen, MD, PhD,
Department of Medical Endocrinology, Rigshospitalet Copenhagen
University Hospital, Copenhagen, Denmark (Thursday, February 16 at
4 p.m. PT)
- Efficacy of migalastat in a cohort of male patients with the
classic Fabry phenotype in the FACETS phase 3 study – Dominique P.
Germain, MD, PhD, Division of Medical Genetics at the University of
Versailles and Assistance Publique - Hôpitaux de Paris (Thursday,
February 16 at 3:30 p.m. PT)
Poster Session: Tuesday, February 14, 4:30-6:30pm
PT
Fabry Disease:
- Response of patients with Fabry disease with the amenable GLA
mutation p.N215S to treatment with migalastat – Derralynn Hughes,
MD, Department of Academic Haematology, Royal Free Hospital, NHS
Foundation Trust, University College London, London, UK (Poster
#150)
- Effects of treatment with migalastat on the combined endpoint
of kidney globotriaosylcermide accumulation and diarrhea in
patients with Fabry disease: results from the phase 3 FACETS
study – Dominique P. Germain, MD, PhD, Division of
Medical Genetics at the University of Versailles and Assistance
Publique - Hôpitaux de Paris (Poster #103)
- Migalastat exposures in Japanese healthy volunteers and
non-Japanese subjects provide evidence that they are similar to
Japanese patients with Fabry disease - Franklin Johnson, MS, Amicus
Therapeutics, Inc. (Poster #159)
- Efficacy and safety of migalastat, an oral pharmacologic
chaperone for Fabry disease: results from two randomized phase 3
studies, FACETS and ATTRACT – Ulla Feldt-Rasmussen, MD, PhD,
Department of Medical Endocrinology, Rigshospitalet Copenhagen
University Hospital, Copenhagen, Denmark (Poster #84)
- Efficacy of migalastat in a cohort of male patients with the
classic Fabry phenotype in the FACETS phase 3 study – Dominique P.
Germain, MD, PhD, Division of Medical Genetics at the University of
Versailles and Assistance Publique - Hôpitaux de Paris (Poster
#102)
Poster Session: Wednesday, February 15, 4:30-6:30 p.m.
PT
Fabry Disease:
- Migalastat improves diarrhea in patients with Fabry disease:
results from the FACETS double-blind, placebo-controlled phase 3
study – Raphael Schiffmann, MD, Institute of Metabolic Disease,
Baylor Research Institute, Dallas, TX (Poster #299)
Pompe Disease:
- First-in-human preliminary pharmacokinetic and safety data on a
novel recombinant acid-α-glucosidase, ATB200, co-administered with
the pharmacological chaperone, AT2221, in ERT-experienced Pompe
patients – Franklin Johnson, MS, Amicus Therapeutics, Inc.
(Poster #LB-26)
- A novel recombinant human acid alpha-glucosidase, ATB200, leads
to greater substrate reduction and improvement in Pompe
disease-relevant markers compared to alglucosidase alfa in Gaa KO
mice – Yi Lun, MS, Amicus Therapeutics, Inc. (Poster
#207)
- Stabilized next-generation recombinant human acid
alpha-glucosidase ATB200 clears accumulated glycogen and reverses
cellular dysfunction to increase functional muscle strength in a
mouse model of Pompe disease – Hung Do, PhD, Amicus Therapeutics,
Inc. (Poster #74)
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 Therapeutics Amicus Therapeutics
(Nasdaq: FOLD) is a biotechnology company at the forefront of
therapies for rare and orphan diseases. The Company has a robust
pipeline of advanced therapies for a broad range of human genetic
diseases. Amicus’ lead programs in development include the small
molecule pharmacological chaperone migalastat as a monotherapy for
Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as
novel enzyme replacement therapy (ERT) and biologic products for
Fabry disease, Pompe disease, and other rare and devastating
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 timing and
reporting of results from preclinical studies and clinical trials,
the prospects and timing of the potential regulatory approval of
our product candidates, commercialization plans, financing plans,
and the projected cash position for the Company. In particular,
this press release relates to the preliminary data from a global
Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221. The
inclusion of forward-looking statements arising from this
preliminary data and study 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 the goals, progress,
timing, and outcomes of discussions with regulatory authorities,
and in particular the potential goals, progress, timing, and
results of preclinical studies and 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 we
may not be successful in commercializing Galafold in Europe or our
other product candidates if and when approved; 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. The
preliminary data and Phase 1/2 study discussed herein is inherently
preliminary and early in the study, derived from a limited patient
set, and later trial results with this patient set or others may
not be consistent with these preliminary results. With respect to
statements regarding projections of the Company's cash position,
actual results may differ based on market factors and the Company's
ability to execute its operational and budget plans. 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,
2015 and Quarterly Report on Form 10-Q for the quarter ended
September 30, 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.
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
MWW PR
Sid Dinsay
sdinsay@mww.com
(646) 381-9017
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