Funding Supports Ongoing Evaluation of AT2220 on ERT-Related Immunogenicity

CRANBURY, N.J., Feb. 7, 2012 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced the receipt of a two-year, $186,000 grant from the Muscular Dystrophy Association (MDA). The grant will be used to evaluate the effect of the pharmacological chaperone AT2220 (duvoglustat HCl) on immunogenicity related to the ERT alglucosidase alfa, the only approved treatment for Pompe disease.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics said, “Immunogenicity is one of several significant potential limitations within the current class of ERTs for lysosomal storage disorders, particularly for patients with Pompe disease. The MDA grant validates our early preclinical work and supports our ongoing evaluation of AT2220 on ERT-related immunogenicity, as well as the broader potential for our chaperone-ERT platform to improve patient outcomes with ERTs.”

Formation of antibodies in Pompe patients undergoing ERT therapy adversely affects the efficacy of alglucosidase alfa, causing some patients to withdraw from treatment. Preclinical results to date suggest that AT2220 when co-administered with ERT may mitigate immunogenicity induced by the ERT for Pompe disease. By stabilizing the structure of alglucosidase alfa, AT2220 may decrease the antigenicity of alglucosidase alfa since unfolded proteins are generally more antigenic than properly folded proteins.

As part of the MDA grant, Amicus will investigate the ability of AT2220 to mitigate ERT-specific immunogenicity from blood samples obtained in the ongoing open-label Phase 2 drug-drug interaction study (Study 010) of AT2220 co-administered with ERT in individuals with Pompe disease and from normal donors.

“The primary focus of our research program is the development of strategies for diagnosis and treatment of neuromuscular diseases,” said MDA Interim President and Medical Director Valerie Cwik, M.D. “We are pleased to support the work Amicus is doing to potentially improve the current FDA-approved ERT treatment for Pompe disease, which sometimes leads to an unwanted immune response.”

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The MDA’s latest round of grants, which became effective February 1, 2012, allocates more than $12 million to support 38 research projects investigating the causes of, and potential treatments for, a number of forms of neuromuscular disease. The new grants were approved by MDA’s Board of Directors based on recommendations from the Association’s Scientific and Medical Advisory Committees. For more information, go to

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan 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 migalastat HCl is in Phase 3 for the treatment of Fabry disease.

About AT2220 for Pompe Disease

AT2220 is an investigational, orally-administered pharmacological chaperone owned exclusively by Amicus. The Company is currently investigating AT2220 (duvoglustat HCl) co-administered with ERT in a Phase 2 study (Study 010) in individuals with Pompe disease. In acid alpha-glucosidase (GAA) knock-out mouse models of Pompe disease, AT2220 co-administered with ERT increased ERT activity in plasma and uptake into key tissues, which corresponded with greater reductions in muscle glycogen, compared to ERT alone. Collectively these preclinical data highlight the potential for AT2220 to improve ERT in patients with Pompe disease.

Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in GAA activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily muscle). Pompe disease affects an estimated 5,000 to 10,000 individuals worldwide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to clinical development of Amicus’ candidate drug products and the timing and reporting of results from 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 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.


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Source: Amicus Therapeutics, Inc.

Tagged: chaperone, grants