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!

Isis Earns $10 Million Milestone Payment From Biogen Idec For Advancement Of ISIS-DMPK Rx To Treat Myotonic Dystrophy





CARLSBAD, Calif., Oct. 16, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $10 million milestone payment from Biogen Idec related to the selection and advancement of ISIS-DMPK Rx to treat myotonic dystrophy type I (DM1). 

(Logo:  http://photos.prnewswire.com/prnh/20130807/LA60006LOGO)

"We are very pleased with the successes we are having in our alliance with Biogen Idec.  The progress we have made is evidenced in our spinal muscular atrophy and myotonic dystrophy programs.  In less than two years, we discovered and advanced ISIS-DMPK Rx into development, and we plan to begin human clinical studies next year," said B. Lynne Parshall, chief operating officer at Isis.  "This summer we and Biogen Idec built upon our successful relationship to create a broad strategic alliance that combines our antisense technology with Biogen Idec's knowledge of neurodegenerative diseases and global reach, with the goal of bringing new drugs to the market to treat patients with neurological disorders."

DM1 is a rare genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and disabling muscle spasms.  DM1, the most common form of muscular dystrophy in adults, is estimated to affect approximately 150,000 patients in the United States, Europe and Japan.  DM1 is caused by a genetic defect in the dystrophia myotonica-protein kinase (DMPK) gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long toxic RNA, which accumulates in the cell and prevents the production of proteins needed for normal cellular function.  The severity and age of onset of DM1 correlates with the number of triplet repeats, which increases from one generation to the next.  There are no disease-modifying therapies for patients with DM1 and current treatments are intended to manage symptoms and minimize disability.  ISIS-DMPK Rx is designed to correct the underlying genetic defect that causes DM1.

"Myotonic dystrophy represents an ideal opportunity for antisense as the disease-causing gene produces a toxic RNA that is not easily targeted with traditional therapeutic approaches," said C. Frank Bennett, Ph.D., senior vice president of research at Isis.  "In our preclinical studies, we and our collaborators, Drs. Charles Thornton and Thurman Wheeler at the University of Rochester, have been able to target the toxic RNA with antisense, remove the toxic RNA and restore normal cell function.  We look forward to working with Biogen Idec to move this program into human clinical trials."    

In June 2012, Isis entered into an alliance with Biogen Idec to discover and develop an antisense drug targeting DMPK for the treatment of DM1.  Under the terms of the agreement, Isis received an upfront payment of $12 million and is eligible to receive up to $59 million in milestone payments associated with the clinical development of ISIS-DMPK Rx, including this $10 million milestone payment.  Biogen Idec has the option to license ISIS-DMPK Rx from Isis up through completion of the Phase 2 study.  Isis could receive up to another $200 million in a license fee and regulatory milestone payments plus double-digit royalties on sales of ISIS-DMPK­ Rx.  Isis is responsible for global development of ISIS-DMPK Rx through completion of Phase 2 clinical studies, with Biogen Idec providing advice on the clinical study design and regulatory strategy.  If Biogen Idec exercises its option, it will assume global development, regulatory and commercialization responsibilities.

ABOUT ISIS PHARMACEUTICALS, INC.Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners.  Isis' broad pipeline consists of 30 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer.  Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO™, in the United States for the treatment of patients with HoFH.  Isis' patents provide strong and extensive protection for its drugs and technology.  Additional information about Isis is available at www.isispharm.com.

ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENTThis press release includes forward-looking statements regarding Isis' alliance with Biogen Idec and the discovery, development, activity, therapeutic potential, safety and commercialization of ISIS-DMPK Rx for the treatment of DM1.  Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement.  Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs.  Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements.  Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis.  As a result, you are cautioned not to rely on these forward-looking statements.  These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2012, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc.  KYNAMRO™ is a trademark of Genzyme Corporation.

1 of 2

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 17,119.12 +12.42 0.07%
S&P 500 2,000.07 +0.05 0.00%
NASDAQ 4,571.7960 +1.1590 0.03%

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