Navigation Links
Amicus Therapeutics Presents Data from Clinical Ex Vivo Response Study and Phase 1 Studies of AT2220
Date:3/13/2008

Data Suggest Majority of Pompe Patients May Be Amenable to Chaperone

Therapy

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 an ex vivo response study and three Phase 1 studies of AT2220 (1-deoxynojirimycin HCl), Amicus' compound in development for the treatment of Pompe disease, at the American College of Medical Genetics (ACMG) Annual Meeting from March 12-16 in Phoenix, AZ. The results of the ex vivo response study along with the previously reported results of the Phase 1 studies support moving into Phase 2 trials in the first half of 2008.

Ex Vivo Response Study data

Interim data will be presented from an ex vivo response study designed to test the effect of AT2220 on various Pompe mutations. Blood and skin samples were collected from 30 Pompe patients (26 adults, 3 juveniles and 1 infant) with a variety of different mutations in acid alpha-glucosidase (GAA), the target enzyme in Pompe disease. Cells derived from these samples were then tested to determine whether treatment with AT2220 caused an increase in the level of GAA. Of the 26 patients with available data, 24 had cells that showed a dose responsive increase in GAA levels, including 22 patients who had at least 1 copy of the common splice site mutation IVS1-13T>G. It has been reported that more than 80% of Caucasian adult Pompe patients have at least 1 copy of this common splicing mutation.

Phase 1 AT2220 data

A total of 72 healthy volunteers were treated in three double-blind, placebo-controlled, dose escalation Phase 1 studies designed to evaluate the safety, tolerability and pharmacokinetics of AT2220. Across all three studies, AT2220 was shown to be generally safe and well tolerated at all doses. The
'/>"/>

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

Page: 1 2 3 4

Related medicine technology :

1. Amicus Therapeutics to Present at the 26th Annual JPMorgan Healthcare Conference
2. Amicus Therapeutics Announces Positive Results From Two Phase 1 Clinical Studies of AT2220 for Pompe Disease
3. Amicus Therapeutics Announces Plans to Present Phase 1 Data for AT2220 for Pompe Disease
4. Amicus Therapeutics Announces Second Quarter 2007 Financial Results
5. Ception Therapeutics Initiates Phase II/III Clinical Trial for Its Lead Product Reslizumab in Eosinophilic Esophagitis (EE) in Children
6. Cell Therapeutics, Inc. (CTI) Submits European Marketing Authorization Application for XYOTAX(TM)
7. InteKrin Therapeutics Announces Initiation of Phase 2b Study in Type 2 Diabetes with INT131
8. Oncology Drug Development Update - Molecular Profiling Redefines the Nature of Malignancy and Increases the Adoption of Targeted Therapeutics
9. TorreyPines Therapeutics Announces Strategic Plan with Focus on Clinical Development Activities to Maximize Value of Versatile Lead Compounds
10. Horizon Therapeutics Completes Enrollment in Phase 3 Trials for Lead Product Candidate HZT-501
11. Multiple Dose Clinical Trial of TorreyPines Therapeutics Tezampanel Demonstrates Compound is Safe and Well-Tolerated
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/10/2014)... Calif. , July 10, 2014  Breg, ... and services, has launched the FreeRunner™ knee brace ... who suffer from patella mal-tracking return to active ... differently than other braces, providing support when patients ... normally when they don,t. Patellofemoral issues ...
(Date:7/10/2014)... July 10, 2014  BC Technical, the largest nationwide, ... America , Inc., a leading provider of Siemens Nuclear ... long-term agreement naming BC Technical as the authorized provider ... MiE and BC Technical announced today that the ... BC Technical as the service and systems provider for ...
(Date:7/10/2014)... , July 10, 2014   LabStyle ... Management Solution, today announced the appointment of Professor ... member of its Board of Directors. ... and business experience to LabStyle as an entrepreneur, ... legal and corporate governance expert. He currently serves ...
Breaking Medicine Technology:Breg Introduces Knee Brace to Help Runners with Patellofemoral Pain Syndrome 2BC Technical Partners With MiE America To Be Authorized Provider Of Systems & Service 2LabStyle Innovations Welcomes Richard B. Stone to its Board of Directors 2LabStyle Innovations Welcomes Richard B. Stone to its Board of Directors 3LabStyle Innovations Welcomes Richard B. Stone to its Board of Directors 4
... Mass., Feb. 17, 2011 Echo Therapeutics, Inc. (OTC ... the Symphony™ tCGM System as a non-invasive, wireless, transdermal ... System for transdermal drug delivery, announced that development of ... schedule.  The company said that the system will be ...
... Technologies,™ Inc. today announced it has enrolled the first patient ... irolimus and a bioabsorbable p OL ymer for the treatment ... in the native coronary arteries) clinical trial.  Stefan Verheye, M.D., ... in the study. This clinical investigation is being conducted to ...
Cached Medicine Technology:Echo Therapeutics Reports Symphony™ tCGM System Ahead of Schedule 2Echo Therapeutics Reports Symphony™ tCGM System Ahead of Schedule 3Micell Technologies Enrolls First Patient in CE Mark DESSOLVE II Study of MiStent™ Drug-Eluting Coronary Stent (DES) 2Micell Technologies Enrolls First Patient in CE Mark DESSOLVE II Study of MiStent™ Drug-Eluting Coronary Stent (DES) 3Micell Technologies Enrolls First Patient in CE Mark DESSOLVE II Study of MiStent™ Drug-Eluting Coronary Stent (DES) 4
(Date:7/11/2014)... According to new report "C4ISR Market (2014 - ... a CAGR of 2.28% to reach $93.04 billion ... and 76 figures spread through 185 pages and ... , Early buyers will receive 10% customization on ... provides market analysis of the global C4ISR Market ...
(Date:7/11/2014)... the number of deer in an area can lower ... infections among people, new research indicates. "Our study ... human interactions with deer, infected nymphal ticks and human ... White-tailed deer are the primary host for adult blacklegged ... study included nearly all the permanent residents of a ...
(Date:7/11/2014)... Microbiology testing is a highly dynamic and ... is expected to pose the biggest challenge to suppliers ... growth to the ongoing spread of AIDS – still ... major factor leading to the upsurge of opportunistic infections, ... broader availability of immunosuppressants, among others. , Market ...
(Date:7/11/2014)... July 11, 2014 Datalogic brings a ... improving sanitation hospital wide. With their new Gryphon ... of disease, like the devastating MRSA, which claimed 5,500 ... GD4400-HC 2D and the Gryphon GM4400-HC 2D ... and can even read barcodes off of troublesome LCD ...
(Date:7/11/2014)... FRIDAY, July 11, 2014 (HealthDay News) -- Children ... greater risk for complications and potentially even death ... a new study. However, the Johns Hopkins ... "miniscule." The researchers analyzed data on nearly ... United States underwent over a 22-year period. ...
Breaking Medicine News(10 mins):Health News:C4ISR Market Estimated to Reach $93.04 Billion by 2019 - New Report by MarketsandMarkets 2Health News:C4ISR Market Estimated to Reach $93.04 Billion by 2019 - New Report by MarketsandMarkets 3Health News:C4ISR Market Estimated to Reach $93.04 Billion by 2019 - New Report by MarketsandMarkets 4Health News:World Virology and Bacteriology Testing Sector Discussed in Comprehensive VPG Research Report Published at MarketPublishers.com 2Health News:World Virology and Bacteriology Testing Sector Discussed in Comprehensive VPG Research Report Published at MarketPublishers.com 3Health News:Stop Spreading Disease with the New Disinfectant-Ready Bar Code Scanner 2Health News:Stop Spreading Disease with the New Disinfectant-Ready Bar Code Scanner 3Health News:Emergency Surgeries on Weekends Riskier for Kids: Study 2
... PITTSBURGH, April 29 Mylan Inc. (NYSE: ... Michael J. Monroe as senior vice,president - ... of the company,s senior,leadership team. In this ... and employee communications; corporate,branding/marketing; corporate media relations; ...
... also contributed to early dropout rate, study says , , ... problem drug users, and those not currently injecting drugs, ... a British study finds. , University of Kent and ... drug users in treatment programs and found that 25 ...
... Society finds cancer survivors who follow health behavior recommendations ... getting adequate exercisehave higher health-related quality of life (HRQoL) ... The study, which appears in the May issue of ... have low rates of smoking, but few are meeting ...
... factor most commonly used to predict whether an extremely ... that can help predict a preemies outcome have been ... Network, of which Yale is a member. , ... and whether the mother was given antenatal steroid mediation ...
... New York, NY -- A JDRF collaboration between Johns ... for the treatment of diabetic eye disease has performed ... using laser surgery. , These findings, representing the ... by The Johns Hopkins University, were presented Monday at ...
... Layoffs and Loss of Healthcare Services, PRINCETON, N.J., ... drives and longer waits for care.,Those are among the ... state,s $300 million in proposed healthcare cuts are approved., ... healthcare leaders from across,the state met with Gov. Jon ...
Cached Medicine News:Health News:Mylan Appoints Michael J. Monroe Senior Vice President for Global Public Affairs 2Health News:Youngest Drug Users Most Likely to Leave Treatment Programs 2Health News:Cancer survivors who practice healthy habits have higher quality of life 2Health News:Factors affecting survival, disability of extremely premature infants identified 2Health News:Promising early evidence of the superior benefits of drug therapy for diabetic eye disease 2Health News:Hospital CEOs Meet with Governor on Budget Cuts 2
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... ARUP Laboratories is a national reference ... laboratory research and development. ARUP offers an ... unique medical tests in clinical and anatomic ... ARUP Laboratories' clients include more than half ...
Medicine Products: