Amicus Therapeutics Announces Presentations and Posters at 14th Annual WORLDSymposium™ 2018

CRANBURY, N.J., Jan. 22, 2018 (GLOBE NEWSWIRE) -- 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

About Amicus Therapeutics
Amicus 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 Statements
This 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.


Amicus Therapeutics
Sara Pellegrino, IRC
Senior Director, Investor Relations
(609) 662-5044

Pure Communications
Jennifer Paganelli
(347) 658-8290