Amicus Therapeutics
(Nasdaq: FOLD), a biopharmaceutical company developing small molecule,
orally-administered pharmacological chaperones for the treatment of human
genetic diseases, announced that it has successfully completed an End
of Phase 2 meeting for Amigal(TM) with the U.S. Food and Drug
Administration (FDA). The FDA indicated that the data from the completed
Phase 2 clinical studies of Amigal support the start of Phase 3 development
and agreed that Amigal meets the criteria to be considered for accelerated
approval. The Agency further indicated that it is not opposed to the use of
a surrogate primary endpoint, pending further discussion and final
agreement on the Phase 3 trial design.
Amicus, along with its partner Shire Human Genetic Therapies, Inc.
("Shire"), is engaged in ongoing discussions with the FDA and the European
Medicines Agency (EMEA) regarding plans for a global Phase 3 clinical
development program for Amigal. In line with previous guidance, Amicus
expects to complete these interactions in the second half of 2008, and
subject to the outcome of the discussions, the Company plans to initiate
Phase 3 development of Amigal in the first half of 2009.
"We are very pleased with the outcome of the End of Phase 2 meeting
with the FDA," said John F. Crowley, President and CEO of Amicus
Therapeutics. "We look forward to continuing our work with the FDA and EMEA
to design a global Phase 3 program for Amigal in Fabry disease."
The meeting followed the successful conduct of Phase 2 clinical studies
in patients with Fabry disease that showed that Amigal was generally safe
and well-tolerated at all doses evaluated. In a majority of patients,
treatment with Amigal resulted in increased activity of the enzyme
deficient in Fabry patients (alpha-GAL) and a reduction of kidney GL-3 as
measured in urine.
In parallel with the regulatory process, 23 of the original 26 patients
will continue to be treated with Amigal in a voluntary Phase 2 extension
study to monitor long term safety and efficacy and to evaluate additional
doses and dose regimens. Data from this extension study are expected to be
available by Q1 2009. In addition, Amicus expects to conduct clinical
pharmacology studies to support the Phase 3 program.
Amicus is developing Amigal as part of a strategic collaboration with
Shire Human Genetic Therapies, Inc., a wholly-owned subsidiary of Shire
Limited, to develop and commercialize Amicus' three lead pharmacological
chaperone compounds for lysosomal storage disorders. Under the agreement,
Shire received commercial rights outside of the United States. Amicus
retains all U.S. rights.
About Amigal
Amigal (migalastat hydrochloride) is an experimental, oral therapy for
the treatment of Fabry disease, a lysosomal storage disorder, which can
cause damage to specific areas of the body, including the kidneys, heart,
nervous system, and skin. Amigal, a pharmacological chaperone, acts by
selectively binding to the misfolded enzyme responsible for Fabry disease,
alpha-GAL. After binding to the enzyme, Amigal promotes the proper folding,
processing, and trafficking of the enzyme from the endoplasmic reticulum to
its final destination, the lysosome, the area of the cell where the enzyme
does its work. Once it reaches the lysosome, the pharmacological chaperone
is displaced, and the enzyme can perform its normal, biological function,
which is the breakdown of its natural substrate, GL-3.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing novel,
oral therapeutics known as pharmacological chaperones for the treatment of
a range of human genetic diseases. Pharmacological chaperone technology
involves the use of small molecules that selectively bind to and stabilize
proteins in cells, leading to improved protein folding and trafficking, and
increased activity. Amicus is initially targeting lysosomal storage
disorders, which are severe, chronic genetic diseases with unmet medical
needs. Amicus has completed Phase 2 clinical trials of Amigal for the
treatment of Fabry disease and is conducting Phase 2 clinical trials of
Plicera(TM) for the treatment of Gaucher disease and AT2220 for the
treatment of Pompe disease.
Forward-Looking Statements
This press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995. 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
progress, timing and outcomes of ongoing discussions with regulatory
authorities and the potential 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 inability to reach
final agreement with regulatory agencies on the use of a surrogate endpoint
and phase 3 trial design for Amigal, the potential that results of clinical
or pre-clinical studies indicate that the product candidates are unsafe or
ineffective; and, our dependence on third parties in the conduct of our
clinical studies; further, the results of earlier clinical trials may not
be predictive of future results; and other risks detailed in our annual
Report on Form 10-K for the year ended December 31, 2007, and our other
public filings with the Securities and Exchange Commission. 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.
Amicus Therapeutics
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