CRANBURY, N.J., March 4, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), today introduced Chaperone-Advanced Replacement Therapy (CHART™) as the brand name for its technology platform that combines unique pharmacological chaperones with enzyme replacement therapy (ERT) for lysosomal storage diseases.
John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "We are pleased to introduce CHART as the brand name and designation for our chaperone-ERT combination platform. CHART reflects the breadth of the opportunities at Amicus to develop chaperones co-administered with marketed ERTs, or co-formulated with proprietary enzymes as next-generation ERTs. It is a platform that has already demonstrated proof of concept in the clinic and in multiple pre-clinical studies. The direct co-formulation of our proprietary enzymes with specific small molecule chaperone stabilizers may improve treatment outcomes, reduce immunological responses and potentially enable more convenient delivery routes with these next-generation ERTs. Our strategic vision is to build out this CHART platform to deliver new benefits for patients and create multiple sources of value for our shareholders. This platform will indeed chart a course to a very bright future for Amicus."
Chaperone-Advanced Replacement Therapy (CHART)
Amicus is leveraging the CHART platform to improve currently marketed ERTs through co-administration of a pharmacological chaperone prior to ERT infusion, and to develop next-generation ERTs that consist of proprietary lysosomal enzyme therapies co-formulated with pharmacological chaperones.
ERTs are the standard of care for many lysosomal storage diseases. A recombinant or gene-activated human enzyme is manufactured for intravenous infusion into the circulation for transport to the lysosome. Once in lysosomes, ERT can perform the function of a person's own missing or deficient enzyme. However, these therapeutic enzymes may unfold and lose activity at any stage in the process, from the infusion bag to the bloodstream to the eventual uptake into cells and tissue. This instability, unfolding, and loss of enzyme activity may impact treatment outcomes with ERT.
Amicus is investigating whether the CHART platform can improve ERT outcomes and deliver several new benefits for patients with lysosomal storage diseases (LSDs). In a chaperone-advanced replacement therapy, a unique pharmacological chaperone binds to the infused therapeutic enzyme, stabilizing the enzyme in its properly folded and active form. This proposed CHART mechanism may allow for enhanced tissue uptake, greater lysosomal activity, more reduction of storage material, and lower immunogenicity compared to ERT alone. Improvements in the physical stability of the enzymes may also enable more convenient delivery routes for next-generation ERTs.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage diseases include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.
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 Quarterly Report on Form 10-Q for the quarter ended June 30, 2012. 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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