This Day On The Street
Continue to site
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

Amicus Therapeutics Presents Additional 6-Month Results From Phase 3 Fabry Monotherapy Study At LDN World Symposium

Study 011: Stage 1 (Baseline to Month 6) Updated Data Highlights at LDN WORLD Symposium:

  • Post-Hoc Subgroup Analysis of Primary Endpoint (modified intent-to-treat (mITT), n=60): in a post-hoc sub-group analysis, patients with a higher baseline disease burden (0.3 inclusions or more per interstitial capillary, n=25) were compared to those with a lower baseline disease burden (0.3 or fewer inclusions per interstitial capillary, n=35). In the 25 patients (14 males and 11 females) with higher baseline disease burden, 7/11 (64%) on migalastat HCl and 2/14 (14%) on placebo were classified as responders. Among the 35 patients (8 males and 27 females) with lower baseline disease burden, 6/19 (32%) on migalastat HCl and 7/16 (44%) on placebo were classified as responders.
  • Urine GL-3: The observed median reduction in urine GL-3 from baseline was 17% for placebo and 12% for migalastat. However due to unexpected variability in the pre-treatment urine GL-3 data (>1.6 fold difference between values at screening and baseline), any potential treatment effects on urine GL-3 cannot be determined.
  • Renal Function from Baseline to Month 6: Renal function remained stable and changes from baseline were similar in both treatment groups during stage 1. Themean (SD) increase in estimated glomerular filtration rate (eGFR) was 2.7 (15.1) mL/min/1.73m 2 in the migalastat HCl group compared to a mean decrease of 2.4 (10.8) mL/min/1.73m 2 in the placebo group. No clinically meaningful changes in proteinuria were observed, and iohexol GFR data are currently being analyzed.
  • Safety: During the first 6 months, no drug-related serious adverse events have been observed. No subjects discontinued migalastat HCl therapy due to a treatment emergent adverse event and the majority of adverse events in both treatment groups were mild in nature.

John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated, "The 6-month results presented today at the WORLD Symposium support our commitment to the ongoing development of migalastat HCl monotherapy. We look forward to reporting the 12-month results from this study to add to the entirety of the data that the FDA has indicated would support a potential U.S. conditional approval."

2 of 5

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

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
Stocks Under $10

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

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

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
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free
AAPL $93.74 0.00%
FB $117.58 0.00%
GOOG $693.01 0.00%
TSLA $240.76 0.00%
YHOO $36.60 0.00%


Chart of I:DJI
DOW 17,773.64 -57.12 -0.32%
S&P 500 2,065.30 -10.51 -0.51%
NASDAQ 4,775.3580 -29.9330 -0.62%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs