CRANBURY, N.J., Jan. 24, 2017 (GLOBE NEWSWIRE) — 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 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 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.
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CONTACT: 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
