This Day On The Street
Continue to site right-arrow
ADVERTISEMENT
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
Cramer's Action Alerts PLUS - See his portfolio and get alerts BEFORE every trade. Learn more NOW!

Biotech Stock Mailbag: Amarin, Sarepta

You can be encouraged and excited about a drug (and a stock) while also being worried about unsettled risks that may blow up the entire endeavor. That pretty much sums up the current state of Sarepta at this point.

The paramount concern is the small trial size -- four DMD patients dosed at the highest 50 mg eteplirsen dose for 36 weeks compared against four "control" patients dosed with a placebo for 24 weeks followed by 12 weeks of eteplirsen. With so few patients, variability of response can muddy interpretation of results.

For this reason, the next look at the data after 48 weeks of is critically important. Some benefit observed in the six-minute walk test favoring eteplirsen over control at 36 weeks should be maintained at 48 weeks. It's okay if eteplirsen patients lose some additional walking ability, as long as the rate of disease progression observed is slower than that measured in the control group of patients.

Let's talk about the control patients for a second because they're just as important. Remember, these patients crossed over to treatment with eteplirsen after 24 weeks, so some benefit from the drug should be measurable by week 48. That means the steep rate of disease progression observed in the control patients through 36 weeks should start to ease up at 48 weeks.

This may sound counter-intuitive, but a narrowing of the walking distance benefit favoring etiplirsen patients over control patients at 48 weeks could be positive because it suggests that control patients are starting to benefit from eteplirsen as well.

A sustained benefit from a small number of patients isn’t going to be enough to assuage all doubt so Sarepta also needs to show that eteplirsen-treated patients have measurable increases in dystrophin levels --- the missing or malfunctioning protein that causes DMD. Any data that correlates increased dystrophin production to improvements in walking ability will go a long way towards demonstrating the efficacy of eteplirsen.

I mentioned in my story this week the possibility that Sarepta could seek accelerated approval of eteplirsen based on positive 48-week data. For this option to be viable, Sarepta needs the dystrophin production data to support the walk benefit. Even if all the data work in Sarepta's favor, the odds that FDA would approve eteplirsen based on this small study, even under new and more lenient regulations for orphan drugs, are small. In other words, don’t count on accelerated approval. Consider it upside. It's not a negative if FDA asks Sarepta to run another study before approval as long as the 48-week data look good.

3 of 4

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

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
Stocks Under $10

David Peltier uncovers low dollar stocks with serious upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Try it NOW
Only $9.95
Try it NOW
14-Days Free
Try it NOW

Check Out Our Best Services for Investors

Dividend Stock Advisor

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
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Options Profits

Our options trading pros provide over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • Actionable options commentary and news
  • Real-time trading community
Try it NOW
Try it NOW
Try it NOW
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!
SYM TRADE IT LAST %CHG
ARNA $4.51 0.00%
BCRX $10.20 0.00%
HZNP $20.53 0.00%
SRPT $13.90 0.00%
AAPL $128.46 0.00%

Markets

DOW 18,132.70 -81.72 -0.45%
S&P 500 2,104.50 -6.24 -0.30%
NASDAQ 4,963.5270 -24.3630 -0.49%

Partners Compare Online Brokers

Free Reports

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