Navigation Links
Amicus Therapeutics Presents Data From Preclinical and Phase 1,Studies of Amigal for Fabry Disease

Studies in Mice Show Reduction of Enzyme Substrate

CRANBURY, N.J., March 19, 2007 /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 results from studies of Amigal(TM) (migalastat hydrochloride, AT1001), Amicus' compound in development for the treatment of Fabry disease, at the of Medical Genetics (ACMG) Annual Meeting from March 21-25 in Nashville, TN. This presentation will include the first data from the Company demonstrating the reduction of globotriaosylceramide (GL-3), the lipid substrate that accumulates in Fabry disease, after oral administration of a pharmacological chaperone. These data were not available at the time of abstract submission but will be included in the presentation at the meeting.

Amigal is designed to selectively bind to and stabilize alpha- galactosidase A (alpha-GAL), the enzyme deficient in Fabry disease. This deficiency leads to lysosomal accumulation of GL-3, which is believed to cause the various symptoms of Fabry disease. Amigal facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down GL-3.

At the ACMG meeting, Amicus scientists will present data from several studies that examined the in vitro and in vivo effects of Amigal in cell lines, mice and healthy volunteers. Among the key findings:

    - In vitro exposure to Amigal increased the level of alpha-GAL in cells

      derived from healthy volunteers and from Fabry patients.

    - Oral administration of Amigal resulted in a dose-dependent increase in

      alpha-GAL levels in various tissues of normal mice and Fabry mice

      genetically modified to produce alpha-GAL with a human missense


    - Oral administration of Amigal to healthy vo
lunteers in a Phase 1

      clinical study resulted in a dose-dependent increase of alpha-GAL levels

      in white blood cells.

    - Oral administration of Amigal significantly decreased the level of GL-3

      in the skin and heart of Fabry mice and showed a trend towards reduction

      in the kidney.

    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 (alpha-GAL). Deficient alpha-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. 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 underdiagnosed. 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 is currently conducting Phase 2 clinical trials for its two lead compounds, Amigal(TM) for Fabry disease, and AT2101 for Gaucher disease. The company is currently conducting Phase 1 trials with AT2220 for the treatment of Pompe disease.

CONTACT: Corporate: Matthew Patterson, Amicus Therapeutics,+1-609-662-2000, or Media: Dan Budwick, BMC Communications Group,+1-212-477-9007 ext. 14

Terms and conditions of use apply
Copyright © 2007 PR Newswire Association LLC. All rights reserved.
A United Business Media Company


Related medicine technology :

1. Amicus Therapeutics Presents Positive Results From Phase 1 Clinical Studies of Plicera for Gaucher Disease
2. Amicus Therapeutics Presents Preclinical Data from Studies of Plicera for Gaucher Disease
3. Cell Therapeutics, Inc. Management to Discuss Todays Announcement of Interim Pixantrone Study Results
4. Halozyme Therapeutics Enhanze Technology Large Protein Molecule Therapeutic Clinical Trial Results Presented at the 34th Annual Meeting of the Controlled Release Society
5. CV Therapeutics Identifies Potential Anti-Diabetic Mechanism of Action for Ranexa
6. Fat Kills Cancer: Turning Stem Cells from Fat Tissue into Personalized, Cancer-Targeted Therapeutics
7. A 4-Week Therapy with Transition Therapeutics E1-I.N.T. Leads to Sustained Reductions in Blood Glucose Levels for 6 Months Post-treatment in Type 2 Diabetes Patients
8. Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury; PrimeCell Therapeutics Provided Pre-Clinical Study
9. Opexa Therapeutics Reports Positive Top-line Data in Phase I/II Extension Trial with Tovaxin for Multiple Sclerosis
10. Orexigen Therapeutics to Present Data on Contrave Impact on Visceral Fat and Insulin Resistance at American Diabetes Association 67th Scientific Sessions in Chicago June 23
11. Allos Therapeutics Reports Results for Phase 3 Enrich Study of Efaproxyn in Women With Brain Metastases Originating From Breast Cancer
Post Your Comments:
(Date:11/26/2015)... ) has announced ... High Viscosity Drugs" report to their offering. ... addition of the "Self Administration of High ... --> Research and Markets ( ) ... Administration of High Viscosity Drugs" report to ...
(Date:11/25/2015)... SYDNEY , Nov. 26, 2015  The total global ... nearly 7% over 2015-2016. Latin America ... Asia , (excluding Japan ), is ... continues to face increased healthcare expenditure. In 2013-2014, ... expenditure declined from 43.5% in 2008-2009 to 41.2% in 2013-2014. ...
(Date:11/25/2015)... , Nov. 25, 2015 On Tuesday, ... federal bellwether trial against Wright Medical Technology, Inc. ... their Conserve metal-on-metal hip implant device, awarded $11 ... a two week trial and three days of ... hip device was defectively designed and unreasonably dangerous, ...
Breaking Medicine Technology:
(Date:11/27/2015)... VVA (PRWEB) , ... November 27, 2015 , ... ... motto of progress through sharing, the 2016 Building Better Radiology Marketing ... The conference will begin on Sunday, March 6, 2016, at Caesars Palace in ...
(Date:11/27/2015)... ... November 27, 2015 , ... Keeping in mind ... mental health and wellness consultation, has collaborated with a leading web-based marketplace for ... gap experienced by parents and bring advice from parenting experts within their reach. ...
(Date:11/27/2015)... ... , ... Avid collector, Andrew Hawley from Vintage Rock Posters, announces his search ... one of Joplin's most famous and beautiful concert posters. The concert was held on ... Arbor. The According to Hawley, "It is hard to believe that Joplin's stardom was ...
(Date:11/26/2015)... , ... November 26, 2015 , ... ... real-time eReferral system for diagnostic imaging in the Waterloo region. Using the Ocean ... Nuclear Medicine tests directly from their electronic medical record (EMR) without the need ...
(Date:11/26/2015)... ... 2015 , ... PRMA Plastic Surgery is updating their record books yet again ... free flap breast reconstruction surgery! , “What an accomplishment for the PRMA team, says ... and it’s an honor to have served all of these women.” , PRMA is ...
Breaking Medicine News(10 mins):