Navigation Links
Amicus Therapeutics Presents Positive Data from Phase 2 Clinical Trial of Plicera(TM) for Gaucher Disease

Data Support Commencement of Longer-term Phase 2 Switching Study

CRANBURY, N.J., March 13 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today that the Company will present positive results from a Phase 2 clinical study of Plicera(TM) (isofagomine tartrate) for Gaucher disease at the American College of Medical Genetics (ACMG) Annual Meeting from March 12-16 in Phoenix, AZ. Results from the fully enrolled Phase 2 trial support the previously reported interim findings that Plicera was generally safe and well tolerated at all doses and increased target enzyme activity levels in a majority of patients.

Phase 2 Plicera data presented at ACMG

The primary objective of this study was to evaluate safety and tolerability of different doses and dosing regimens of Plicera. The secondary objective was to evaluate certain pharmacodynamic measures of treatment, including effects on GCase (the enzyme deficient in individuals with Gaucher disease) levels in white blood cells.

Thirty patients with Gaucher disease (8 men and 22 women between the ages of 18 and 63) were enrolled, and there were 12 unique alleles represented including the most common N370S and L444P mutations. Patients were on enzyme replacement therapy (ERT) with imiglucerase for an average of 9 years prior to entering the trial, and they temporarily discontinued ERT to receive Plicera for the 4 week duration of the study.
The key findings from the trial were as follows:

-- Plicera was generally well-tolerated at all doses evaluated, and no

serious adverse events were reported.

-- GCase activity as measured in white blood cells was increased in 20

of the 26 patients with evaluable GCase data, and 5 of the 6 patients

without a clear increase were either in the lowest dose cohort or the

cohort dosed least frequently.

-- As expected in this short term study, the levels of relevant markers

of Gaucher disease including platelet counts, hemoglobin levels,

glucocerebroside (substrate) levels, chitotriosidase activity and

pulmonary activation-related chemokine (PARC) levels were maintained.

"These data give us great confidence in moving our Gaucher program forward," said John F. Crowley, President and CEO of Amicus Therapeutics. "In addition to a 6-month Phase 2 study in individuals naive to ERT, which is currently underway, we plan to initiate a longer-term study in individuals switching from enzyme replacement therapy to Plicera in the second half of this year."

As of November 2007, Plicera is being developed in partnership with Shire Human Genetic Therapies (HGT), a business unit of Shire plc, which is focused on genetic diseases.

About Gaucher Disease

Gaucher disease is a lysosomal storage disorder caused by inherited genetic mutations in the GBA gene, which result in deficient activity of the enzyme acid beta-glucosidase, also known as glucocerebrosidase (GCase). Deficient GCase activity leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease, including an enlarged liver and spleen, abnormally low levels of red blood cells and platelets and skeletal complications. In some cases there is significant impairment of the central nervous system.

Gaucher disease is estimated to affect approximately 10,000 people in the developed world. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in Plicera in the United States and the European Commission has designated Plicera as an orphan medicinal product in the European Union.

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(TM) for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease. The Company recently completed Phase I clinical trials of AT2220 for the treatment of Pompe disease.

Forward-Looking Statements

Amicus cautions you that statements included in this press release that are not a description of historical facts are "forward-looking statements" within the meaning of Section 21E 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 potential progress 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 effect of the completion of the Phase 2 clinical trial for Amigal(TM) for the treatment of Fabry disease, the plans for the Phase 3 clinical trial for Amigal(TM), the Phase 2 clinical trials for Plicera(TM) for the treatment of Gaucher disease and the effect of the completion of the Phase 1 clinical trials for AT2220 for the treatment of Pompe disease. Further, the results of earlier clinical trials may not be predictive of future results; and other risks detailed in the public filings of Amicus 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.


SOURCE Amicus Therapeutics
Copyright©2008 PR Newswire.
All rights reserved

Related biology technology :

1. Amicus Therapeutics to Present at the Merrill Lynch Global Pharmaceutical, Biotech & Medtech 2007 Conference
2. Amicus Therapeutics Announces Third Quarter 2007 Financial Results
3. Meredith Martin Addy Named Chair of AIPLAs Amicus Committee
4. Amicus Therapeutics to Present at the Lazard Capital Markets Fourth Annual Healthcare Conference
5. Amicus Therapeutics to Host R&D Day
6. Amicus Therapeutics Announces Positive Results From Phase 2 Clinical Trials of Amigal(TM) for Fabry Disease
7. Amicus Therapeutics Announces Fourth Quarter and Full Year 2007 Financial Results & Program Advancements
8. Amicus Therapeutics to Present at the Merrill Lynch Global Pharmaceutical, Biotechnology & Medical Device Conference
9. Amicus Therapeutics to Present at the BIO CEO & Investor Conference 2008
10. Vicus Therapeutics to Present at the 234th American Chemical Society National Meeting in Boston, MA
11. DJO Incorporateds Pending Merger With ReAble Therapeutics Clears U.S. Antitrust Review
Post Your Comments:
(Date:11/26/2015)... CHESHAM , England , November 26, ... Lightpoint Medical, an innovative medical device company specializing in ... Euro grant from the European Commission as part of the ... enabling the company to carry out a large-scale clinical trial ... -->      (Logo: , ...
(Date:11/25/2015)... Nov. 25, 2015  PharmAthene, Inc. (NYSE MKT: PIP) ... a stockholder rights plan (Rights Plan) in an effort ... carryforwards (NOLs) under Section 382 of the Internal Revenue ... PharmAthene,s use of its NOLs could be substantially ... defined in Section 382 of the Code. In general, ...
(Date:11/25/2015)... -- Studies reveal the differences in species ... the way for more effective treatment for one of the ... --> --> Gum disease ... cats, yet relatively little was understood about the bacteria associated ... conducted by researchers from the WALTHAM Centre for Pet Nutrition ...
(Date:11/25/2015)... 2015  Neurocrine Biosciences, Inc. (Nasdaq: NBIX ) ... CEO of Neurocrine Biosciences, will be presenting at the ... New York . .   ... 5 minutes prior to the presentation to download or ... will be available on the website approximately one hour ...
Breaking Biology Technology:
(Date:11/16/2015)... Calif. , Nov 16, 2015  Synaptics ... of human interface solutions, today announced expansion of ... TouchView ™ touch controller and display driver ... revolution of smartphones. These new TDDI products add ... TD4100 (HD resolution), TD4302 (WQHD resolution), and TD4322 ...
(Date:11/10/2015)... , Nov. 10, 2015  In this ... the basis of product, type, application, disease ... in this report are consumables, services, software. ... are safety biomarkers, efficacy biomarkers, and validation ... report are diagnostics development, drug discovery and ...
(Date:11/2/2015)... Nov. 2, 2015  SRI International has been awarded ... preclinical development services to the National Cancer Institute (NCI) ... provide scientific expertise, modern testing and support facilities, and ... pharmacology and toxicology studies to evaluate potential cancer prevention ... The PREVENT Cancer Drug Development Program is an NCI-supported ...
Breaking Biology News(10 mins):