Navigation Links
Amicus Therapeutics Announces Positive Results From Phase 2 Clinical Trials of Amigal(TM) for Fabry Disease

CRANBURY, N.J., Dec. 19 /PRNewswire-FirstCall/ -- Amicus Therapeutics Inc. (Nasdaq: FOLD), a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today positive results from its recently completed Phase 2 clinical trials of Amigal(TM) (migalastat hydrochloride) for Fabry disease. As of November 2007, Amigal is being developed in partnership with Shire Human Genetic Therapies (HGT), a business unit of Shire plc, which is focused on genetic diseases. The results will be discussed as a part of an "R&D Day" meeting being hosted by the company today from 4:30 to 7:00 PM at the Four Seasons Hotel in New York City. A live audio web cast of the presentation will be available to all interested parties through the Company's website at Interested parties should connect at least 15 minutes prior to the presentation to ensure adequate time for any software download that may be required to join the webcast. An archive of the webcast will be available at the same address until approximately January 2, 2008.

"The completion of these trials is a major milestone for Amicus and these clinical results represent an important proof of concept for the pharmacological chaperone platform technology," said John F. Crowley, President and CEO of Amicus Therapeutics. "We look forward to advancing our program in Fabry as well as Gaucher, Pompe, and other important therapeutic targets utilizing this new approach to the treatment of a wide range of human genetic diseases."

Summary of Study Results:

The primary objective of the Phase 2 trials was to evaluate the safety and tolerability of treatment with Amigal. The secondary objective was to evaluate certain pharmacodynamic measures of treatment, including effects on a-GAL (the target enzyme deficient in Fabry patients) and levels of GL-3 (the substrate that builds up in the cells of patients) in cells and tissues affected by the disease. An additional objective was the preliminary assessment of cardiac and renal function.

The four open-label, multi-national Phase 2 trials of Amigal enrolled 18 men and 9 women with Fabry disease between the ages of 17 and 65. The four studies examined various dose levels and frequencies of Amigal administration and had 12 or 24 week primary treatment arms with an optional treatment extension.

Twenty-six patients completed the primary treatment arms and all entered the optional treatment extension. The 26 patients had 21 different missense genetic mutations that cause Fabry disease. The mutations represented the full spectrum of Fabry patients, including those with both early-onset and late- onset forms of the disease. Twenty-three patients are currently being treated with Amigal under the treatment extension, including 8 who have been treated for more than a year and 4 who have been treated for almost 2 years.

"The positive results of these first trials of a pharmacological chaperone in Fabry disease are impressive," said Raphael Schiffmann, M.D., Lead Investigator at the Metabolic Neurology Branch of the National Institute of Neurological Disorders and Stroke (NINDS), a part of the National Institutes of Health, and a principal investigator in one of the Amigal clinical trials. "I believe this technology has the potential to be an important new treatment option for many Fabry disease patients."

The key findings in the Phase 2 studies were:

* Amigal was generally safe and well-tolerated at all doses evaluated. No

drug-related serious adverse events were reported during the primary

treatment arm, and none have been reported during the treatment


* Twenty-four out of 26 patients demonstrated an increase in a-GAL as

measured in white blood cells, kidney, and skin.

* a-GAL increases were seen in patients with both low levels of residual

enzyme activity (<3%) at baseline as well as patients presenting with

higher baseline levels (greater than or equal to 3%).

* Kidney GL-3 levels as measured in urine or biopsies were decreased in

patients who demonstrated greater increases in levels of a-GAL.

* Renal and cardiac function results were encouraging, including those

seen in patients treated for nearly two years.

* Patient responses were consistent with the results of in vitro testing

of Fabry mutations, thus improving the ability to select likely

responders for future studies.

"These data demonstrate that Amigal has a meaningful effect on a range of genetic mutations in Fabry disease and thus has the potential to treat a significant portion of the Fabry patient population," said William Wilcox, M.D., Ph.D., Director of the Metabolic Disorders Clinic at Cedars-Sinai Medical Center and a principal investigator in one of the Amigal clinical trials.

Amicus expects that the results will be presented again at the American College of Medical Genetics (ACMG) Annual Meeting on March 12-16, 2008, in Phoenix, Arizona.

Based on the results of these Phase 2 trials, Amicus and Shire plan to meet with US and European regulatory authorities to discuss the design of a Phase 3 clinical trial for Amigal.

About Fabry Disease

Fabry disease is a lysosomal storage disorder caused by inherited genetic mutations in the GLA gene, which result in deficient activity of the enzyme alpha-galactosidase A (a-GAL). Deficient a-GAL activity leads to lysosomal accumulation of globotriaosylceramide (GL-3), which is believed to cause the various symptoms of Fabry disease, including pain, kidney failure and increased risk of heart attack and stroke. Amigal is designed to selectively bind to and stabilize a-GAL, which facilitates proper trafficking of the enzyme to the lysosomes, where it is needed to break down GL-3. In a previous Phase 1 study, Amigal was shown to be safe and well tolerated and to increase GLA levels in white blood cells in healthy human volunteers. Additionally, pre-clinical studies showed that treatment of Fabry transgenic mice with Amigal can increase GLA activity and significantly reduce GL-3 accumulation in heart, kidney, skin and plasma.

Fabry disease is estimated to affect approximately 5,000 to 10,000 people in the developed world, but recent evidence suggests that the disease may be significantly under diagnosed. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan designation for Amigal in the United States, and the European Commission has designated Amigal 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.

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 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.


SOURCE Amicus Therapeutics Inc.
Copyright©2007 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. Vicus Therapeutics to Present at the 234th American Chemical Society National Meeting in Boston, MA
7. DJO Incorporateds Pending Merger With ReAble Therapeutics Clears U.S. Antitrust Review
8. PeriCor Therapeutics Announces Closing of Licensing Agreement for Acadesine with Schering-Plough Corporation
9. Systems Medicine (SM), a Subsidiary of Cell Therapeutics, Inc. (CTI), Announces Execution of CRADA with NCI/NIH to Develop Potential Cancer Treatments Targeting the Insulin Growth Factor (IGF) Pathway
10. Amsterdam Molecular Therapeutics to Release H1 2007 Results on August 29, 2007
11. Nektar Therapeutics Appoints Tim Harkness as Chief Financial Officer
Post Your Comments:
(Date:10/12/2017)... ... October 12, 2017 , ... ... launched Rosalindâ„¢, the first-ever genomics analysis platform specifically designed for life science ... in honor of pioneering researcher Rosalind Franklin, who made a major contribution ...
(Date:10/11/2017)... ... 11, 2017 , ... The CRISPR-Cas9 system has ... and avoiding the use of exogenous expression plasmids. The simplicity of programming this ... gain-of-function studies. , This complement to loss-of-function studies, such as with RNAi ...
(Date:10/11/2017)... Alto, CA, USA (PRWEB) , ... October 11, 2017 , ... ... set to take place on 7th and 8th June 2018 in San Francisco, CA. ... policy influencers as well as several distinguished CEOs, board directors and government officials from ...
(Date:10/11/2017)...  VMS BioMarketing, a leading provider of patient support solutions, ... Educator (CNE) network, which will launch this week. The VMS ... care professionals to enhance the patient care experience by delivering ... health care professionals to help women who have been diagnosed ... ...
Breaking Biology Technology:
(Date:6/30/2017)... 30, 2017 Today, American Trucking Associations ... of face and eye tracking software, became the ... program. "Artificial intelligence and advanced ... monitor a driver,s attentiveness levels while on the ... to detect fatigue and prevent potential accidents, which ...
(Date:5/23/2017)... , May 23, 2017  Hunova, the first robotic gym for the ... been officially launched in Genoa, Italy . The first ... and the USA . The technology was developed ... market by the IIT spin-off Movendo Technology thanks to a 10 million ... News Release, please click: ...
(Date:5/6/2017)... 5, 2017 RAM Group , ... new breakthrough in biometric authentication based on a ... properties to perform biometric authentication. These new sensors are ... created by Ram Group and its partners. This sensor ... supply chains and security. Ram Group is a ...
Breaking Biology News(10 mins):