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!

Sanofi And Regeneron Announce Publication Of Positive Phase 2 Results For Lipid-Lowering PCSK9 Antibody In The Lancet

"Heterozygous familial hypercholesterolemia is a common, serious, and often undiagnosed cause of early heart disease. There remains a high degree of unmet need in these patients as a large percentage are unable to reach optimal LDL-C goals despite being on maximal lipid-lowering therapy," said Evan A. Stein, M.D., Ph.D., Director of the Metabolic and Atherosclerosis Research Center in Cincinnati, Ohio, and Principal Investigator of the study. "These data suggest that SAR236553/REGN727 may provide a new option, on top of existing therapies, to lower LDL-cholesterol and finally reach LDL-C goals for these difficult-to-treat patients."

Sanofi and Regeneron also announced today that based on discussions with the U.S. and European regulatory authorities, they intend to initiate a global Phase 3 program with SAR236553/REGN727 in June. This will be the first Phase 3 program of an investigational drug targeting PCSK9.

"These data, along with recently presented data in patients with hypercholesterolemia, further support our belief that blocking PCSK9 with our antibody has the potential to offer a novel mechanism for lowering LDL-cholesterol in a broad range of patients," said George D. Yancopoulos, M.D., Ph.D., Chief Scientific Officer of Regeneron and President of Regeneron Laboratories. Dr. Elias Zerhouni, President, Global Research & Development, Sanofi, added: "Our global Phase 3 program will include patients with high unmet medical need, such as patients with familial hypercholesterolemia or with elevated cardiovascular risk who cannot reach their LDL-cholesterol goals with current standard therapies. The program reflects our excitement and commitment to develop this potential therapeutic option for these patients."

About PCSK9

PCSK9 is known to be a determinant of circulating LDL-C levels, as it binds to LDL receptors resulting in their degradation so that fewer are available on liver cells to remove excess LDL-C from the blood. [ 4] Moreover, traditional LDL-lowering therapies such as statins actually stimulate the production of PCSK9, which limits their own ability to lower LDL-C. [ 5] Blocking the PCSK9 pathway is therefore a potentially novel mechanism for lowering LDL-C.

About SAR236553/REGN727 and the Phase 2 Heterozygous Familial Hypercholesterolemia trial

SAR236553/REGN727 is a fully human monoclonal antibody directed against PCSK9, administered via subcutaneous injection. By inhibiting PCSK9, a determinant of circulating LDL-C levels in the blood, SAR236553/REGN727 increases the number of free LDL receptors which can bind to circulating LDL-C and clear it from the bloodstream. SAR236553/REGN727 was created using Regeneron's VelocImmune ® technology.

Study 1003 was a randomized, double-blind, placebo-controlled, dose-finding study in patients with heFH. The primary objective of the trial was to assess the efficacy of various subcutaneous doses and dosing regimens of SAR236553/REGN727 on LDL-C in patients with heFH. Seventy-seven patients were randomized to either placebo or one of four active dose regimens of 150 mg at four-week intervals (Q4W), 200 mg Q4W, 300 mg Q4W, or 150 mg at two-week intervals (Q2W). At baseline, all patients had LDL-C greater than or equal to 100 mg/dL (2.59 mmol/L) and were on stable daily statin therapy (the type and dosage of statin was at the discretion of the investigator) with or without ezetimibe, for at least six weeks prior to screening. The majority of patients, 77%, were taking a high intensity dose of a statin, and 71% were also taking ezetimibe 10 mg at the time of the screening visit and throughout the trial. Despite this aggressive therapy, the mean baseline LDL-C for all study participants was approximately 155 mg/dL (4 mmol/L). The primary endpoint of the study was the change in LDL-C from baseline over the 12-week study period. Patients were followed for a total of 20 weeks for safety. [2]

About Heterozygous Familial Hypercholesterolemia

2 of 5

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 16,943.81 +28.74 0.17%
S&P 500 1,967.57 +2.89 0.15%
NASDAQ 4,415.49 +19.2860 0.44%

Brokerage Partners

Rates from Bankrate.com

  • Mortgage
  • Credit Cards
  • Auto
Advertising Partners

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