Navigation Links
Amicus Therapeutics Announces FDA Agreement to Commence Phase 2 Study of AT2220 Co-administered with Enzyme Replacement Therapy for Pompe Disease
Date:3/8/2011

CRANBURY, N.J., March 8, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND).  AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease.  Based on data provided by Amicus, the FDA agreed with the Company's proposal to resume clinical development of AT2220, starting with a new Phase 2 study designed to evaluate the use of AT2220 when co-administered with enzyme replacement therapy (ERT) in subjects with Pompe disease.  The Company expects to commence this study in the first half of 2011 and to report preliminary results in the second half of 2011.

Co-administration of Pharmacological Chaperones with ERTAmicus previously reported promising preclinical data demonstrating that the co-administration of a pharmacological chaperone with ERT has the potential to address key limitations of ERT. The addition of a pharmacological chaperone has been shown to prevent the loss of activity of ERT in the circulation, increase tissue uptake, and increase substrate reduction in multiple disease-relevant tissues. Preclinical proof of concept has been established for Fabry disease and Pompe disease.

About AT2220Data from Phase 1 studies in 72 healthy volunteers demonstrated that AT2220 was generally safe and well tolerated at all doses evaluated with no drug-related serious adverse events. Based on these data and encouraging safety data from preclinical studies, Amicus initiated a Phase 2 clinical trial of AT2220 as a monotherapy treatment in adults with Pompe disease.  The protocol involved initial treatment with a high dose of AT2220.  Two patients enrolled in the trial experienced adverse events categorized as serious and probably related to treatment with AT2220, and as a result the IND was placed on clinical hold.

Amicus completed a thorough investigation of the events, including the completion of additional preclinical and Phase 1 studies.  As a result the Company decided to continue development of AT2220 co-administered with ERT but not as a monotherapy.  

About Pompe Disease Pompe disease affects an estimated 5,000-10,000 individuals world-wide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression. The early onset form of the disease is the most severe, progresses most rapidly, and is characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually lead to death from cardio-respiratory failure between 1 and 2 years of age. The late onset form of the disease begins between childhood and adulthood and has a slower rate of progression that is characterized by musculoskeletal and pulmonary symptoms that usually lead to progressive muscle weakness and respiratory insufficiency. A high majority of patients have the late onset form of the disease. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in AT2220 in the United States.

About Amicus Therapeutics Amicus Therapeutics is a biopharmaceutical company focused on developing treatments for rare diseases.  The Company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of diseases including lysosomal storage disorders and diseases of neurodegeneration.  Amicus' lead program, Amigal (migalastat HCl) is in Phase 3 development for the treatment of Fabry disease and is partnered with GSK Rare 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 Amicus' candidate drug products and the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "targets," "likely," "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. 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, 2010. 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. CONTACT:Amicus Therapeutics(609) 662-2000ir@amicustherapeutics.com   FOLD-G


'/>"/>

SOURCE Amicus Therapeutics
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Amicus Therapeutics Initiates Phase 2 Clinical Trial Evaluating Amigal™ Co-administered with Enzyme Replacement Therapy
2. Amicus Therapeutics to Present at the 29th Annual J.P. Morgan Healthcare Conference on Tuesday, January 11, 2011
3. Amicus Therapeutics Awarded $500,000 Grant from The Michael J. Fox Foundation for Development of Next-Generation Pharmacological Chaperone to Treat Parkinsons Disease
4. Amicus Therapeutics Announces Third Quarter 2010 Financial Results
5. GSK and Amicus Therapeutics Enter Exclusive Worldwide Agreement to Develop and Commercialize Amigal™ for Fabry Disease
6. Amicus Therapeutics to Host Conference Call and Webcast to Discuss Exclusive Worldwide Agreement with GSK to Develop and Commercialize Amigal™ for Fabry Disease
7. Amicus Therapeutics to Host Its Second Quarter 2010 Financial Results Conference Call and Webcast on August 5, 2010
8. Amicus CEO Represents BIO on Rare Diseases at U.S. Senate Committee on Health, Education, Labor and Pensions Bipartisan Hearing
9. Alzheimers Drug Discovery Foundation Funds Amicus Therapeutics To Advance Pharmacological Chaperone Technology
10. Fenwal Receives FDA Clearance for Amicus(R) Separator With Wireless Connectivity
11. Amicus Therapeutics to Host its First Quarter 2010 Financial Results Conference Call and Webcast on May 6, 2010
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/25/2016)... , Deutschland und GERMANTOWN, Maryland ... Zusammenarbeit mit Therawis bedient ... bei Brustkrebs   QIAGEN N.V. (NASDAQ: ... gab heute bekannt, eine Lizenz- und Entwicklungsvereinbarung mit ... Assays für die Onkologie eingegangen zu sein. Ein ...
(Date:5/25/2016)... , May 25, 2016 According ... Share, Development, Growth and Demand Forecast to 2022 - ... Pump and Others)" published by P&S Market Research, the ... million in 2015, and it is expected to grow ... type, the insulin pump segment is expected to witness ...
(Date:5/25/2016)... , May 25, 2016 MedDay, a ... announces that an oral presentation entitled "High doses of biotin ... trial" will be given by Professor Ayman Tourbah , ... Congress of the European Academy of Neurology (EAN) in ... related disorders 3" will take place on Sunday, 29 May ...
Breaking Medicine Technology:
(Date:5/26/2016)... ... May 26, 2016 , ... ... for the Foundation Fighting Blindness, Long Island Chapter on June 4, 2016, 1:30-3:30 ... , Dr. Maisel, founder of Retina Group of New York , is ...
(Date:5/26/2016)... , ... May 26, 2016 , ... In an effort to provide hair restoration information ... both Snapchat users and those who do not use the app. Dr. Mohebi, the founder ... page, Dr. Mohebi Live . , Dr. Mohebi says, “The positive response to ...
(Date:5/26/2016)... ... 26, 2016 , ... Leadership of Life Science Logistics (LSL), ... has earned its ISO 13485 certification, indicating the company’s quality control system for ... policies associated with ISO quality standard 13485. , BSI Group America, Inc., ...
(Date:5/26/2016)... ... 2016 , ... The Lung Institute has partnered with the Gulfcoast ... June 6 at their clinic in downtown Tampa. The class is complimentary for the ... has created a free downloadable 4 Week Smoking Cessation Guide for those ...
(Date:5/26/2016)... (PRWEB) , ... May 26, 2016 , ... ... offering campers a multitude of activities from daily practices, arts & crafts, discussions, ... Camp Directors Amber East-D’Anna and Christy Evans have combined backgrounds in kids’ yoga, ...
Breaking Medicine News(10 mins):