CRANBURY, N.J., Feb. 10, 2016 -- Amicus Therapeutics (Nasdaq:FOLD), a biotechnology company at the forefront of therapies for rare and orphan diseases, today announced that 3 oral presentations and 6 posters highlighting its development programs for Lysosomal Storage Disorders will be included at the 12th Annual WORLDSymposium™ 2016, to be held February 29-March 4, 2016 in San Diego, CA.
Oral Platform Presentations:
Fabry Disease:
- Podocyte Globotriaosylceramide (GL-3) Content in Male Adult Patients with Fabry Disease Reduces Following 6-12 Months of Treatment with Migalastat – Behzad Najafian, M.D., University of Washington (Wednesday, March 2 at 10:15 a.m. PT)
- Comparison of Integrated White Blood Cell Alpha-Galactosidase A Activity Exposure Between Every-Other-Day Orally Administered Migalastat and Biweekly Infusions of Agalsidase Beta or Agalsidase Alfa – Franklin Johnson, Amicus Therapeutics, Inc. (Thursday, March 3 at 3 p.m. PT)
Pompe Disease:
- Co-Administration of the Pharmacological Chaperone AT2221 with a Proprietary Recombinant Human Acid Alpha-Glucosidase Leads to Greater Plasma Exposure and Substrate Reduction Compared to Alglucosidase Alfa – Richie Khanna, Amicus Therapeutics, Inc. (Tuesday, March 1 at 4 p.m. PT)
Poster Sessions: Tuesday, March 1, 4:30-6:30pm PT and Wednesday, March 2, 4:30-6:00 p.m. PT
Fabry Disease:
- The Validation of Pharmacogenetics in the Identification of Fabry Patients for Treatment with Migalastat – Elfrida Benjamin, Amicus Therapeutics, Inc. (Poster #38)
- Phenotype of Fabry Disease in Patients with Mutations Amenable to Migalastat – Derralynn Hughes, DPhil, University College London (Poster #137)
- Comparison of Integrated White Blood Cell Alpha-Galactosidase A Activity Exposure Between Every-Other-Day Orally Administered Migalastat and Biweekly Infusions of Agalsidase Beta or Agalsidase Alfa -- Franklin Johnson, Amicus Therapeutics, Inc. (Poster #149)
- Podocyte Globotriaosylceramide (GL-3) Content in Male Adult Patients with Fabry Disease Reduces Following 6-12 Months of Treatment with Migalastat – Behzad Najafian, M.D., University of Washington (Poster #216)
- Persistence of Positive Renal and Cardiac Effects of Migalastat in Fabry Patients with Amenable Mutations Following 30 Months of Treatment in the ATTRACT Study – Daniel Bichet, M.D., University of Montreal (Poster #LB-01)
Pompe Disease:
- Co-Administration of the Pharmacological Chaperone AT2221 with a Proprietary Recombinant Human Acid Alpha-Glucosidase Leads to Greater Plasma Exposure and Substrate Reduction Compared to Alglucosidase Alfa – Richie Khanna, Amicus Therapeutics, Inc. (Poster #160)
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) products for Fabry disease, Pompe disease, and other Lysosomal Storage Disorders.
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 Amicus' candidate drug products, the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products and the projected cash position for the Company. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "potential," "plan," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus 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 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 the business of Amicus, including, without limitation: the potential that results of clinical or pre-clinical 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 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 will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future 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, 2014 and Form 10-Q for the quarter ended June 30, 2015. 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 Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
FOLD–G
CONTACTS: Investors/Media: Amicus Therapeutics Sara Pellegrino Senior Director, Investor Relations [email protected] (609) 662-5044 Media: Pure Communications Dan Budwick [email protected] (973) 271-6085


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