Sarepta Hits Grand Slam With Muscular Dystrophy Drug Trial Result
CAMBRIDGE, Mass. (TheStreet) --Sarepta Therapeutics (SRPT) said 48 weeks of treatment with an experimental drug eteplirsen produced significant amounts of a critical protein needed for muscle function and helped boys with a form of muscular dystrophy improve their walking ability.
The results from the mid-stage study are not only positive but medically groundbreaking because never before has a drug been shown to reverse the progressive decline in muscle function that is a hallmark of Duchenne muscular dystrophy.
Some of the DMD patients treated with eteplirsen increased the distance they were able to walk in six minutes by 21 meters over the course of the study.
Sarepta intends to meet with the U.S. Food and Drug Administration about seeking early approval for eteplirsen, the company said.Shares of Sarepta more than doubled in value after Wednesday's announcement, up 115% to $32.50. The stock was already up more than 300% in value since July when positive, interim results from the eteplirsen study were announced. After 48 weeks of treatment, four DMD patients treated with a 50 mg dose of eteplirsen demonstrated a 21-meter increase in the six-minute walk test from baseline. By comparison, patients who received placebo for 24 weeks followed by 24 weeks of eteplirsen showed a decline of 68.4 meters from baseline in the six-minute walk test. The overall benefit of 89.4 meters was statistically significant. There was no statistically significant difference in walking distance between patients treated with 30 mg of eteplirsen and the placebo/delayed treatment patients. Along with the improved walking ability, eteplirsen also caused an increase in levels of dystrophin, a key protein that supports muscle function. Eteplirsen at both doses resulted in a statistically significant increase in dystrophin to 47% of normal. The placebo/delayed treatment group of patients saw a smaller increase of dystrophin to 38% of normal. Dystrophin is a protein that plays key role in muscle function and repair. The genetic inability to make dystrophin is what causes muscular dystrophy. Eteplirsen is designed to "skip over" the section of damaged gene in DMD patients and therefore restore the gene's ability to produce partially functioning dystrophin. -- Reported by Adam Feuerstein in Boston. Follow @AdamFeuerstein
Select the service that is right for you!COMPARE ALL SERVICES
Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.
- $2.5+ million portfolio
- Large-cap and dividend focus
- Intraday trade alerts from Cramer
- Weekly roundups
Access the tool that DOMINATES the Russell 2000 and the S&P 500.
- Buy, hold, or sell recommendations for over 4,300 stocks
- Unlimited research reports on your favorite stocks
- A custom stock screener
- Upgrade/downgrade alerts
Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.
- Diversified model portfolio of dividend stocks
- Alerts when market news affect the portfolio
- Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
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.
- Real Money + Doug Kass Plus 15 more Wall Street Pros
- Intraday commentary & news
- Ultra-actionable trading ideas
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.
- 100+ monthly options trading ideas
- Actionable options commentary & news
- Real-time trading community
- Options TV