Amicus Therapeutics Initiates Phase 2 Clinical Trial Evaluating Amigal™ Co-administered With Enzyme Replacement Therapy
CRANBURY, N.J., Feb. 9, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced the dosing of the first subject in a Phase 2 clinical trial designed to evaluate the co-administration of its investigational drug Amigal™ (migalastat hydrochloride) with enzyme replacement therapy (ERT) for Fabry disease. This open-label Phase 2 study is investigating drug-drug interactions between Amigal and the ERTs Fabrazyme® and Replagal®. Preclinical studies have demonstrated that coadministration of Amigal results in a prolonged circulating half-life of ERT, increased enzyme activity in cells, and greater substrate reduction in target tissues compared to that seen with ERT alone. Amigal is being developed in collaboration with GlaxoSmithKline (GSK), as part of an alliance between GSK Rare Diseases and Amicus.
John F. Crowley, Chairman and CEO of Amicus, stated "The commencement of this clinical study is an important milestone for Amicus and for pharmacological chaperone technology. Based on encouraging preclinical results we believe the co-administration of Amigal with ERT has the potential to provide significant benefit for people with Fabry disease. The advancement of this approach is a part of our vision to provide two new treatment options for Fabry patients in the future—Amigal as a monotherapy or Amigal co-administered with ERT. Finally, we also believe this study will provide us with valuable proof of concept for applying this approach to other lysosomal storage diseases and more broadly to other indications treated with therapeutic proteins."
Phase 2 Study Design
The Phase 2 trial will evaluate the safety and pharmacokinetics of Amigal and ERT when co-administered in 18 male patients with Fabry disease, ages 18-65, who have been receiving ERT for at least one month before study entry. Patients enrolled in this study need not have a genetic mutation responsive to Amigal as a monotherapy. Each patient will receive ERT alone and then ERT after administration of a single oral dose of Amigal. There will be two cohorts of nine patients treated with one of two Amigal dose levels.
Select the service that is right for you!COMPARE ALL SERVICES
Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.
- $2.5+ million portfolio
- Large-cap and dividend focus
- Intraday trade alerts from Cramer
- Weekly roundups
Access the tool that DOMINATES the Russell 2000 and the S&P 500.
- Buy, hold, or sell recommendations for over 4,300 stocks
- Unlimited research reports on your favorite stocks
- A custom stock screener
- Upgrade/downgrade alerts
Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.
- Diversified model portfolio of dividend stocks
- Alerts when market news affect the portfolio
- Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
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.
- Real Money + Doug Kass Plus 15 more Wall Street Pros
- Intraday commentary & news
- Ultra-actionable trading ideas
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.
- 100+ monthly options trading ideas
- Actionable options commentary & news
- Real-time trading community
- Options TV