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!

Hepatology Publishes 12-Week Phase I/II Extension Study Data Of Sebelipase Alfa In Adults With Late Onset LAL Deficiency

Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced publication of the 12-week Phase I/II extension study data of sebelipase alfa in adults with lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Hepatology.

“Children and adults with late onset LAL Deficiency can suffer from serious liver complications and accelerated atherosclerosis,” said Manisha Balwani, MD, MS, Assistant Professor of Genetics and Genomic Sciences and Assistant Professor of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, and principal investigator in the study. “Based on its mechanism of action, sebelipase alfa addresses the root cause of LAL Deficiency, and the Phase I/II extension study in adults with LAL Deficiency illustrates that sebelipase alfa improves patients’ abnormalities associated with the disease.”

About the Phase I/II extension study of sebelipase alfa in adults with late onset LAL Deficiency

Nine adults with LAL Deficiency were enrolled in the Phase I/II trial. After completing the initial portion of the Phase I/II trial, patients were allowed to continue treatment with sebelipase alfa as part of a long-term, open-label extension study.

In the extension study, patients received four once-weekly infusions of sebelipase alfa (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg) and then transitioned to every other week infusions of sebelipase alfa (1.0 mg/kg or 3.0 mg/kg). Eight of nine patients enrolled in the extension study. Data published in Hepatology were derived from the seven patients who completed the first 12 weeks of dosing in the extension study.

For these seven patients, sebelipase alfa produced mean percent decreases for ALT and AST from the initial baseline to week 12 of the extension study of 52% and 36%, respectively (p<0.05 for both). In addition, sebelipase alfa resulted in mean percent decreases from the initial baseline to week 12 of the extension study for LDL-C of 27% (p=0.078), total cholesterol of 22% (p=0.047), triglycerides of 28% (p=0.016), as well as mean increases in HDL of 15% (p=0.016).

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 17,083.80 -2.83 -0.02%
S&P 500 1,987.98 +0.97 0.05%
NASDAQ 4,472.1080 -1.5890 -0.04%

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