This Day On The Street
Continue to site right-arrow
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!

Sarepta May Have Found Treatment Breakthrough for Muscular Dystrophy

Stocks in this article: SRPT VRTX

BOTHELL, Wash. ( TheStreet) -- Sarepta Therapeutics (SRPT) unveiled the first data demonstrating that an experimental "genetic fix" can significantly improve the walking performance of patients with muscular dystrophy.

Results from the small study are still preliminary but the Sarepta drug, known as eteplirsen, is a potential medical breakthrough because it may be the first to treat the underlying cause of Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is no effective treatment.

Last April, Sarepta presented data showing that eteplirsen increases levels of a key protein involved in muscle function but Tuesday's results were the first to show a more tangible, or real-world, benefit measured by an improvement in the distance a patient can walk over six minutes.

Sarepta shares more than doubled in value to $8.58 after the new eteplirsen data were released. The stock's rapid rise can be attributed not only to the promising eteplirsen data but also because Sarepta may be able to seek FDA approval without having to conduct another clinical trial under new regulations designed to speed the development and approval of drugs for rare diseases.

Sarepta recently changed its corporate name from AVI Biopharma.

In the study, DMD patients treated weekly with 50 mg of eteplirsen and followed for 36 weeks demonstrated a decline of 8.7 meters in a six-minute walk test compared to baseline. Patients treated with a placebo for 24 weeks followed by eteplirsen for 12 weeks -- the study's control arm -- took the same walking test but showed a decline of 78 meters from baseline.

The treatment benefit favoring eteplirsen as measured by walking distance was 69.4 meters over 36 weeks and was statistically significant, Sarepta said.

"The magnitude of this clinical benefit is an unprecedented treatment effect in DMD. This result represents a major advance in the pursuit of a disease modifying treatment for this severe, progressive and life-threatening disease," said Dr. Jerry Mendell, Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb study, in a statement.

DMD is a rare disease and as such, Sarepta enrolled a very small number of patients in this initial study. Just four patients were treated with the 50 mg dose of eteplirsen compared against four patients treated with a placebo and delayed eteplirsen.

1 of 3

Select the service that is right for you!

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 17,756.83 -21.32 -0.12%
S&P 500 2,063.73 +2.50 0.12%
NASDAQ 4,752.76 +4.3640 0.09%

Brokerage Partners

Rates from

  • 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