This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

Amicus Therapeutics Achieves Target Enrollment In Second Phase 3 Fabry Monotherapy Study

56 Patients Now Randomized to Switch from Enzyme Replacement Therapy (ERT) to Migalastat HCl or to Remain on ERT Final Enrollment Expected Ahead of Year-End Target

CRANBURY, N.J., Oct. 22, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases, today announced that it has now achieved target enrollment and has 56 patients in its second Phase 3 study ( Study 012) of migalastat HCl monotherapy for Fabry disease. Screening is now closed at participating sites, and final enrollment is anticipated by year-end 2012. Amicus, in collaboration with GlaxoSmithKline (GSK), is developing the investigational pharmacological chaperone Migalastat HCl for the treatment of Fabry disease.

Study 012 (The ATTRACT, or FAB-AT1001-012 Study) Highlights:

  • First clinical study to compare oral migalastat HCl to standard-of-care ERTs (Fabrazyme ® and Replagal ®)
  • Enrolled males and females with Fabry disease, who had genetic mutations amenable to migalastat HCl as a monotherapy, and were on ERT for a minimum of 12 months
  • Primary outcome measure is renal function assessed by Glomerular Filtration Rate (GFR) at 18 months

John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated, "We are very pleased to have met the enrollment objectives for this important Phase 3 Fabry monotherapy study ahead of the year-end target. The willingness of these Fabry patients to switch from an approved ERT to migalastat HCl to participate in this study highlights the unmet medical needs that persist in this community. We look forward to evaluating the effects of migalastat HCl, as well as currently used ERTs, on renal function over the course of 18 months of treatment in this study."

Amicus and GSK are co-developing all formulations of migalastat HCl under a global Fabry collaboration. Migalastat HCl monotherapy is in Phase 3 development ( Study 011 and Study 012) for Fabry patients with genetic mutations that are amenable to this chaperone monotherapy, as determined by a cell-based assay. Study 011 is a placebo-controlled study intended primarily to support U.S. registration, and Study 012 compares migalastat HCl to ERT to primarily support global registration. Migalastat HCl co-administered with ERT is in Phase 2 ( Study 013) and migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary ERT (JR-051, recombinant human alpha-Gal A enzyme) is in preclinical development. Amicus has commercial rights to all Fabry products in the United States and GSK has commercial rights to all of these products in the rest of world.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company is developing orally administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 (duvoglustat HCl) co-administered with ERT in Phase 2 for Pompe disease.

1 of 3

Select the service that is right for you!

COMPARE ALL SERVICES
Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!
DOW 17,001.22 -38.27 -0.22%
S&P 500 1,988.40 -3.97 -0.20%
NASDAQ 4,538.5510 +6.4470 0.14%

Brokerage Partners

Rates from Bankrate.com

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs