Amicus Therapeutics Introduces Proprietary "CHART" Platform Technology
New Platform Designation Reflects Development Strategy and Emphasis on
Next-Generation Enzyme Replacement Therapies for Lysosomal Storage Diseases
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
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.
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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