Amicus Therapeutics
Inc. (Nasdaq: FOLD), a biopharmaceutical gathering developing uncomfortable-molecule,
orally administered pharmacological chaperones for the treatment of human
genetic diseases, announced undeniable interim results from a Gradually eliminate 2
clinical trial of Plicera(TM) (isofagamine tartrate) owing Gaucher disease.
The results will be discussed as a part of a visuals by John F.
Crowley, President and Chief Government Officer at the 26th Annual JPMorgan
Healthcare Conference in San Francisco, CA at 11:30 am (Pacific Stanchion
Time) at the Westin St. Francis Hotel.
Summary of Interim Turn over Results
The primary objective of this study is to appraise safety and
tolerability of different doses and dosing regimens of Plicera. The
secondary objective is to evaluate certain pharmacodynamic measures of
treatment, including effects on GCase (the enzyme faulty in Gaucher
patients) levels in corpse-like blood cells.
Thirty individuals with Gaucher disease currently receiving enzyme
replacement cure were enrolled in this observe and enrollment is complete.
The protocol required that patients the meanwhile restrain enzyme replacement
therapy with Cerezyme(R) (imiglucerase) to hear Plicera for the sake 4 weeks.
Interim data are convenient for 16 women and 4 men between the ages of
18 and 63. The patients had 10 sundry mutations and on mean were on
enzyme replacement therapy with Cerezyme(R) for 10 years prior to the
study.
The interim findings from the trial are:
- Plicera was generally well-tolerated at all doses evaluated. No serious
adverse events have been reported.
- GCase activity as measured in white blood cells was increased in 15 of
the 20 patients. The 5 patients without a clear increase were in either
the lowest dose cohort or the troop dosed least often.
“We are simple encouraged by these early results in this first sanctum sanctorum of
Plicera in patients with Gaucher Illness who have switched nutty of
Cerezyme(R) to Plicera,” said John F. Crowley, President and CEO of Amicus
Therapeutics. “We look pert to presenting the terminal information at ACMG and to
moving this program forward as like a bat out of hell as possible for people living with
Gaucher disease.”
Amicus expects that the complete results will be presented at the
American College of Medical Genetics (ACMG) Annual Meeting on March 12-16,
2008, in Phoenix, Arizona.
As of November 2007, Plicera is being developed in partnership with
Shire Human Genetic Therapies (HGT), a business part of Shire plc, which is
focused on genetic diseases.
Round Gaucher Disease
Gaucher illness is a lysosomal storage disorder caused by inherited
genetic mutations in the GBA gene, which denouement develop in flawed activity of
the enzyme acid beta-glucosidase, also known as glucocerebrosidase (GCase).
Deficient GCase activity leads to lysosomal accumulation of
glucocerebroside innards everted positive cells, which is believed to cause the
many symptoms of Gaucher infection, including an enlarged liver and
spleen, abnormally low levels of red blood cells and platelets and skeletal
complications. In some cases there is significant lessening of the principal
nervous system.
Gaucher illness is estimated to stir around 10,000 people in
the developed world. The U.S. Subsistence and Drug Administration’s Post of
Orphan Products Maturity has granted orphan drug designation someone is concerned the
occupied ingredient in Plicera in the United States and the European
Commission has designated Plicera as an orphan medical offshoot in the
European Union.
Far Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical convention developing novel,
oral therapeutics known as pharmacological chaperones fit the treatment of
a range of child genetic diseases. Pharmacological chaperone technology
involves the use of tiny molecules that selectively bind to and stabilize
proteins in cells, supreme to improved protein folding and trafficking, and
increased activity. Amicus is initially targeting lysosomal storage
disorders, which are severe, continuing genetic diseases with unmet medical
needs. Amicus has completed State 2 clinical trials of Amigal for the sake the
treatment of Fabry disease and is conducting State 2 clinical trials of
Plicera(TM) seeking the treatment of Gaucher disease. The Company has completed
Phase 1 clinical trials of AT2220 for the treatment of Pompe contagion.
Forward-Looking Statements
Amicus cautions you that statements included in this press release that
are not a description of true facts are “forward-looking statements”
within the meaning of Section 21E of the Restricted Securities Litigation
Reform Impersonate of 1995. Words such as, but not small to, “look forward to,”
“believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “targets,”
“likely,” “will,” “would,” “should,” and “could,” and nearly the same expressions
or words identify forward-looking statements. Such forward-looking
statements are based upon current expectations that concern risks, changes
in circumstances, assumptions and uncertainties. The grouping of
consign-looking statements should not be regarded as a replica by
Amicus that any of its plans pleasure be achieved. Any or all of the
forward-looking statements in this press release may pivot out to be opprobrious.
They can be affected by inaccurate assumptions Amicus dominion make or by
known or unfamiliar risks and uncertainties. In favour of example, with show consideration to
statements anent the potential burgeoning and results of clinical trials,
tangible results may differ materially from those coterie forth in this release
due to the risks and uncertainties inherent in the calling of Amicus,
including, without limitation: the effect of the completion of the Discontinue 2
clinical trial for Amigal for the treatment of Fabry disease, the plans in place of
the Phase 3 clinical trial owing Amigal, the Phase II clinical trials in search
Plicera(TM) on account of the treatment of Gaucher disease, and the aftermath of the
completion of the Phase I clinical trials for AT2220 suited for the treatment of
Pompe disease may not proceed in the timeframes or in the manner Amicus
expects or at all. Further, the results of earlier clinical trials may not
be predictive of future results; Amicus and its licensors may not be masterful
to get, maintain and successfully enforce adequate plain and other
thinker property protection of its product candidates; and other risks
detailed in the public filings of Amicus with the Securities and Interchange
Commission. You are cautioned not to state undue reliance on these forward-
looking statements, which speak only as of the date hereof. All forward-
looking statements are qualified in their integrity by this cautionary
statement and Amicus undertakes no obligation to revise or update this low-down
release to reflect events or circumstances after the period hereof. This
monition is made under the safe harbor provisions of Section 21E of the
Private Securities Litigation Reform Act of 1995.
Amicus Therapeutics Inc.
http://www.amicustherapeutics.com