Amicus Therapeutics Enters Collaboration With Biogen Idec for Parkinson's Disease



      Biogen Idec to Fund 100% of Discovery, Development, and
                      Commercialization Costs

CRANBURY, N.J., Sept. 10, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics
(Nasdaq:FOLD), a biopharmaceutical company at the forefront of
therapies for rare and orphan diseases, has entered a collaboration
with Biogen Idec (Nasdaq:BIIB) to discover, develop and commercialize
novel small molecules for the treatment of Parkinson's disease. The
collaboration will build upon preclinical studies at Amicus and
independent published research that suggest increasing activity of the
lysosomal enzyme glucocerobrosidase (GCase) in the brain may correct
alpha-synuclein pathology and other deficits associated with
Parkinson's disease.

"Our collaboration with Amicus complements our current strategy to
identify and develop novel therapies to address Parkinson's disease,"
said Tim Harris, Senior Vice President of Translational Medicine at
Biogen Idec. "Amicus has been a pioneer in the discovery of novel small
molecules that increase GCase activity in the brain, and we look
forward to working together to discover potential treatments for
Parkinson's disease."

Under terms of the multi-year agreement, Amicus and Biogen Idec will
collaborate in the discovery of a new class of small molecules that
target the GCase enzyme, for further development and commercialization
by Biogen Idec. Biogen Idec will be responsible for funding all
discovery, development, and commercialization activities. In addition
Amicus will be reimbursed for all full-time employees working on the
project. Amicus is also eligible to receive development and regulatory
milestones, as well as modest royalties on global net sales.

"This partnership combines Biogen Idec's leadership in
neurodegenerative diseases with our internal expertise in discovering
small molecules that enhance the activity of lysosomal enzymes," said
John F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics. "We believe that this collaboration is an important step
forward in validating the potential to address neurodegenerative
diseases by increasing relevant enzyme activity in the brain."

Link Between GCase and Alpha-Synuclein in Parkinson's Disease

Inherited genetic mutations in the GBA1 gene, which encodes the GCase
enzyme, have been identified as the most widespread genetic risk factor
for Parkinson's disease and Dementia with Lewy bodies. The accumulation
of alpha-synuclein in Lewy bodies in the brain is a hallmark of
Parkinson's disease. Independent research published over the past
decade has demonstrated a link between GCase deficiency and
alpha-synuclein accumulation, and suggests that improving the lysosomal
targeting of GCase and increasing enzyme activity may be a beneficial
therapeutic approach for Parkinson's disease and other
synucleinopathies.

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 novel, first-in-class treatments for a broad range of human
genetic diseases, with a focus on delivering new benefits to
individuals with lysosomal storage diseases. Amicus' lead programs
include the small molecule pharmacological chaperones migalastat HCl as
a monotherapy and in combination with enzyme replacement therapy (ERT)
for Fabry disease; and AT2220 (duvoglustat HCl) in combination with ERT
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 preclinical and clinical development of Amicus' candidate
drug products. Words such as, but not limited to, "look forward to,"
"believe," "expect," "anticipate," "estimate," "intend," "potential,"
"plan," "targets," "likely," "may," "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 results of preclinical studies, 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.
With respect to statements regarding projections of the Company's cash
position, actual results may differ based on market factors and the
Company's ability to execute its operational and budget plans. 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, 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.

FOLD--G


CONTACT: Investors/Media:
         Sara Pellegrino
         [email protected]
         (609) 662-5044

         Media:
         Dan Budwick
         [email protected]
         (973) 271-6085

Suggested Articles

Karen DeSalvo's appointment is the latest in a string of hires that deepen the tech giant’s investment in and focus on healthcare.

Spring Bank’s hepatitis B drug, given in tandem with Gilead’s Vemlidy, tamped down on chronic disease in 23% of patients in a phase 2 study.

Gilead names Dickinson to CFO role; MedImmune CSO Herbst nabs CSO job at Pyxis; Checkmate picks up Wooldridge for CMO.