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's Eteplirsen Walk-A-Thon Now Stretches For 84 Weeks

CAMBRIDGE, Mass. (TheStreet) -- A handful of pre-teen boys suffering with Duchenne muscular dystrophy have now been treated with Sarepta Therapeutics' (SRPT) eteplirsen for 84 weeks. The boys are still walking, defying the debilitating and progressive nature of a disease that would be forcing them into wheelchairs without treatment.

As with previous updates from Sarepta's eteplirsen study, no significant safety problems were reported. No serious adverse events, no hospitalizations or treatment discontinuations.

Well, there was one disruption in the study but it was actually more evidence of eteplirsen's profound benefit for DMD patients. Here's how Sarepta describes it:

One boy in the placebo/delayed-treatment cohort was not able to perform the 6MWT at the Week 84 clinic visit due to a physical injury unrelated to treatment, and therefore had no 6MWT data captured at the Week 84 time point. The boy has recovered from the injury, continues to be ambulatory and is expected to be evaluated on the 6MWT at future clinic visits.

Sarepta doesn't say, but the injured boy suffered a broken leg, according to people familiar with his case. The leg healed and the boy is walking again normally. Boys with DMD who break a leg aren't supposed to recover and walk again normally, but in this case, he is.

After 84 weeks, DMD patients treated with eteplirsen are walking, on average, 9.2 meters less over six minutes than they were at the start of the study. When the same walk test was performed at 74 weeks, the decrease in walking distance was 2.2 meters, on average.

Another small group of DMD patients started the study on placebo and switched over to eteplirsen after 24 weeks. Initially, the walking ability of these boys declined while on placebo, but it's since stabilized following treatment with eteplirsen.

"We now have demonstrated stability of walking for over a year and a half in the original eteplirsen treatment cohort in boys who are now 11 years old on average, an age when many DMD boys have lost the ability to walk," said Sarepta CEO Chris Garabedian, in a statement. "In addition, the placebo/delayed-treatment cohort, which has now received eteplirsen for over a year, has demonstrated a stabilization in walking ability for 48 weeks compared with the precipitous decline observed earlier in the study before dystrophin was confirmed in these patients. Overall, we believe the data across all treatment cohorts are remarkably consistent and continue to support eteplirsen as a potential treatment option in DMD."

Sarepta continues to work work with the FDA to reach agreement on an accelerated approval filing strategy for eteplirsen.

Today's positive update from the eteplirsen study makes Sarepta's case even stronger.

Sarepta shares are up 5 percent to $40.10 in early Wednesday trading.

-- Reported by Adam Feuerstein in Boston.

Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

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 17,098.45 +18.88 0.11%
S&P 500 2,003.37 +6.63 0.33%
NASDAQ 4,580.2710 +22.5760 0.50%

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