Study 010 Preliminary Results – 3 Cohorts of AT2220 Co-Administered with ERT (n=16)
Study 010 is investigating single ascending oral doses of AT2220 (50 mg, 100 mg, 250 mg, and 600 mg) co-administered with Myozyme or Lumizyme in patients with Pompe disease. Each patient receives one infusion of ERT alone, and then a single dose of AT2220 just prior to the next ERT infusion. Highlights from the first three dose cohorts of AT2220 were as follows:
Safety: To date, single doses of AT2220 co-administered with ERT have been generally well-tolerated, with no drug-related adverse events reported. In addition, AT2220 was cleared from muscle to negligible levels by Day 7 in all three cohorts. Per the study protocol, an independent data safety monitoring board reviewed safety data from the first 3 cohorts and determined that the study should continue into the fourth and highest dose group (Cohort 4). Amicus has fully enrolled Cohort 4 and continues to anticipate results during the fourth quarter 2012.
rhGAA Enzyme Activity in Plasma: 24-hour plasma PK was measured at each infusion. Dose-related increases in rhGAA activity in plasma samples in 16 out of 16 patients following co-administration suggest an increase in properly folded, active rhGAA enzyme available for uptake into tissue.
|rhGAA Enzyme Activity in Plasma Area Under the Curve (AUC)|
|ERT + AT2220 vs. ERT Alone|
|Cohort||AT222 Dose with ERT||Mean Fold-Increase vs. ERT Alone (Range)|
|1 (n=4)||50 mg||1.5 (1.2 to 1.6)|
|2 (n=6)||100 mg||1.7 (1.5 to 1.9)|
|3 (n=6)||250 mg||2.0 (1.6 to 2.6)|
|rhGAA Enzyme Activity in Muscle|
|ERT + AT2220 vs. ERT Alone|
|Study 010 Cohorts 1-3 (n=16)||Fold-Increase vs. ERT alone|
|Cohort 1: ERT + AT2220 50 mg (n=4)|
|Muscle Biopsy Day 7 (n=4)||0.7, 0.9, 1.0, 1.4|
|Cohort 2: ERT + AT2220 100 mg (n=6)|
|Muscle Biopsy Day 3 (n=3)||0.8, 1.4, 1.6|
|Muscle Biopsy Day 7 (n=3)||1.0, 1.1, 1.6|
|Cohort 3: ERT + AT2220 250 mg (n=6)|
|Muscle Biopsy Day 3 (n=3)||0.8, 1.0, 1.4|
|Muscle Biopsy Day 7 (n=3)*||1.0, 1.9|
|*1 ERT sample insufficient for determining fold-increase with ERT+AT2220|
- Myozyme alone elicited a T-cell response in 36% of samples, which decreased to 14% in the presence of AT2220.
- Lumizyme alone elicited a T-cell response in 28% of samples, which decreased to 10% in the presence of AT2220.
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