Amicus Therapeutics Announces Positive Interim Results From Phase 2Clinical Trial of Plicera for Gaucher Disease
CRANBURY, N.J., January 07, 2008 /PRNewswire-FirstCall/ -- Amicus Therapeutics Inc. , a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today positive interim results from a Phase 2 clinical trial of Plicera(TM) (isofagamine tartrate) for Gaucher disease.
Summary of Interim Study Results
The primary objective of this study is to evaluate 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 deficient in Gaucher patients) levels in white blood cells.
Thirty individuals with Gaucher disease currently receiving enzyme replacement therapy were enrolled in this study and enrollment is complete. The protocol required that patients temporarily stop enzyme replacement therapy with Cerezyme(R) (imiglucerase) to receive Plicera for 4 weeks.
Interim data are available for 16 women and 4 men between the ages of 18 and 63. The patients had 10 different mutations and on average 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 cohort dosed least frequently.
"We are very encouraged by these early results in this first study of Plicera in patients with Gaucher Disease who have switched off of Cerezyme(R) to Plicera," said John F. Crowley, President and CEO of Amicus Therapeutics. "We look forward to presenting the final data at ACMG and to moving this program forward as rapidly as possible for people living with Gaucher disease."
Amicus expects that the complete results will be presented at the 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 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 for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease. The Company has completed Phase 1 clinical trials of AT2220 for the treatment of Pompe disease.
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 for the treatment of Fabry disease, the plans for the Phase 3 clinical trial for Amigal, the Phase II clinical trials for Plicera(TM) for the treatment of Gaucher disease, and the effect of the completion of the Phase I clinical trials for AT2220 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 able to obtain, maintain and successfully enforce adequate patent and other intellectual property protection of its product candidates; 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.
Posted: January 2008