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
TheStreet Open House

Biotech Stock Mailbag: Sarepta, Chelsea, IPO Fever

Stocks in this article: SRPT RNA CHTP OMED EPZM

BOSTON ( TheStreet) -- The Biotech Stock Mailbag is open for business.

I haven't written about Sarepta Therapeutics (SRPT - Get Report) lately mainly because there hasn't been much change to the story. I'm as bullish as I've ever been about the efficacy and safety of eteplirsen and the FDA agreeing to an early approval filing. I still believe the FDA's affirmative decision to allow Sarepta to file based on data from the current, small phase II study (when it happens) is tantamount to an approval. The eteplirsen data have strengthened over time. Parents and advocates working on behalf of the Duchenne muscular dystrophy kids have been very effective at lobbying FDA and Congress, pushing for early approval of eteplirsen.

FDA, in turn, has been incredibly responsive, even solicitous, to the DMD community. The agency's top officials -- Margaret Hamburg, Janet Woodcock and Bob Temple -- are all personally involved in the eteplirsen review. Yes, eteplirsen is already under review by FDA even without an "official" filing. They're not swapping summer grilling recipes at all these face-to-face meetings.

I checked in by phone recently with Jenn McNary, advocate and mother to two children, Max and Austin, with DMD. As most Sarepta followers know, McNary has been very involved in the fight for eteplirsen's early approval. Max is a patient in Sarepta's phase II study and has benefitted tremendously from eteplirsen. Austin, wheelchair bound, has not been able to get eteplirsen, unfortunately.

"There is no way the FDA is giving us this level of feedback and access just to shut us down," said McNary.

Is McNary worried at all about FDA telling Sarepta that another, larger study is required before seeking eteplirsen's approval?

"It's not going to happen," she said, without hesitation.

Sarepta bulls are accused by the bears of being overly emotional and not looking objectively at the paucity (and problems) with the eteplirsen data. There's some truth to the charge. Emotion and anecdotal evidence have factored into my bullishness, I readily admit. But I've met Max and Austin personally and it's very hard to be an eteplirsen skeptic when you watch Max glide across a room with almost no discernible limp to fetch a plate of food for his brother Austin, confined to a wheelchair.

1 of 4

Check Out Our Best Services for Investors

Action Alerts PLUS

Jim Cramer and Stephanie Link 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

Jim Cramer's protégé, 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
Try it NOW
Try it NOW
Try it NOW

Check Out Our Best Services for Investors

Dividend Stock Advisor

Jim Cramer's protégé, 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!

Markets

DOW 17,191.37 -195.84 -1.13%
S&P 500 2,002.16 -27.39 -1.35%
NASDAQ 4,637.9940 -43.5030 -0.93%

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