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 CEO: Next Eteplirsen FDA Meeting in October

Stocks in this article: SRPT RNA GSK

CAMBRIDGE, Mass. ( TheStreet) -- Sarepta Therapeutics (SRPT) CEO Chris Garabedian spoke to investors earlier this afternoon at the Morgan Stanley healthcare conference. He provided some incremental updates on the eteplirsen program in Duchenne muscular dystrophy.

Here are some of the highlights of Garabedian's investor chat:

-- Sarepta plans to take another set of muscle biopsy samples from the DMD patients in the phase II trial to provide FDA with additional data on dystrophin production. Biotech trader Dan Rosenblum has the right take on what this means:

-- On a related note, Garabedian dissed DMD rivals Prosensa (RNA) and GlaxoSmithKline (GSK) by pointing out the "low bar" used by the companies to assess dystrophin production in the "DEMAND-2" study of drisapersen. Three different tests were used, but only one had to be positive.

In August, Glaxo posted data showing dystrophin increases from baseline in 72% of DMD boys treated with 6 mg of continuous drispersen and 59% of DMD boys treatedwith 6 mg of intermittent drisapersen.

We'll see a more detailed look at the drisapersen dystrophin data from the DEMAND-2 study in October at the World Muscle Society meeting.

-- Sarepta has scheduled a meeting with FDA in October to discuss the requirements for the CMC (chemistry, manufacturing and controls) section of the planned eteplirsen approval filing. A hoped-for CMC meeting in September wasn't possible, Garabedian said.

Sarepta's guidance for an eteplirsen FDA filing in the first half of 2014 assumes a "reasonable amount of flexibility" from FDA regarding CMC requirements, said Garabedian. This includes stuff stability testing data and comparability of eteplirsen made at small scale versus larger scale.

-- On Sarepta's R&D efforts to develop additional exon-skipping drugs, animal testing for an exon 45 skipping drug is underway and similar studies for an exon 53 skipper will be started later in the third quarter. The company continues to guide towards filing INDs for two new drugs next year.

-- Sarepta is still blocked in Europe by Prosensa's drisapersen patent but Garabedian said the appeal process continues. Sarepta plans to meet with European regulators at the end of the year to discuss the feasability of an approval filing, even if it means the risk of being sued for patent infringement.

"When you have a good drug for a devastating disease that is good for patients, we ill do everything we can to make that drug available," Garabedian said.

-- 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!

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 17,918.16 +113.36 0.64%
S&P 500 2,073.65 +3.00 0.14%
NASDAQ 4,773.1120 +7.7320 0.16%

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