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

Three-Year Data From Phase 2 Trial Of Genzyme Gaucher Disease Oral Compound Suggest Sustained Or Further Improvement Across All Endpoints

Genzyme Corporation (NASDAQ: GENZ) today announced three-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including bone disease, at the three-year timepoint. The results were presented for the first time this week at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada.

Genzyme previously reported that the eliglustat tartrate phase 2 trial had met its primary endpoint at one year, and that data demonstrated continued improvement through two years. The primary composite endpoint was a clinically meaningful response in at least two of three endpoints: improvements in spleen size, hemoglobin and platelet levels. The study has continued with 19 patients through three years. The extension phase of this trial is still ongoing.

Eliglustat tartrate continued to show robust clinical response through three years:
  • Spleen volume decreased from baseline by a mean of 61 percent and liver volume decreased from baseline by 29 percent.
  • Hemoglobin level increased from baseline by a mean of 2.6 grams per deciliter.
  • Platelet count increased from baseline by a mean of 91 percent.

The study also analyzed the clinical response of patients in the phase 2 trial with respect to achieving therapeutic goals. Due to the heterogeneity of Gaucher disease, therapeutic goals were previously developed by experts involved in the treatment of Gaucher patients to assess their response to enzyme replacement therapy (ERT). Most patients dosed with eliglustat tartrate met established therapeutic goals for hemoglobin, platelets, spleen volume and liver volume, demonstrating progressive and clinically meaningful responses in multiple organ systems. At three years, 100 percent of patients met at least 3 of the 4 therapeutic goals developed for hematologic and organ volume parameters.

1 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 $93.64 -0.11%
FB $118.57 0.84%
GOOG $698.21 0.75%
TSLA $241.80 0.43%
YHOO $36.53 -0.19%


Chart of I:DJI
DOW 17,891.16 +117.52 0.66%
S&P 500 2,081.43 +16.13 0.78%
NASDAQ 4,817.5940 +42.2360 0.88%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs