This Day On The Street
Continue to site
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

Sarepta Is Still A Buy: Recent Concerns Much Ado About Nothing

Patients in the "delayed treatment" cohort -- this group received placebo through 24 weeks, followed by 38 weeks of eteplirsen -- show a similar pattern once on drug. Despite some variability in absolute 6MWT scores at 36 weeks (-71 meters from baseline), 48 weeks (-61 meters), and 62 weeks (-78 meters), the data again fall within a 5% margin that suggests at least disease stability.

Sarepta's press release and the accompanying data slides, both of which were posted to the company's website on Friday, show multiple analyses on the 62-week data that can be confusing at first. Rather than focus on each analysis -- based on maximum (highest baseline value; the primary endpoint), mean, minimum (lowest baseline), and "Day 1" (tests were administered on consecutive days) 6MWT scores -- investors should note the data offer solid concurrence for eteplirsen's placebo-subtracted treatment effect. (Patients had two 6MWT scores taken at baseline, but only one score at 62 weeks. That explains the differences in adjusted mean 6MWT change from baseline for the various analyses.)

The consistent placebo-subtracted benefit should reassure eteplirsen worriers, especially since FDA reviewers routinely cut data several ways to look for red flags.

The final concern about the 62-week data has to do with eteplirsen's safety data. The morpholino-based chemistry used to design eteplirsen had shown no adverse effects thus far. Sarepta on Friday noted one eteplirsen-treated patient had a transient finding of proteinuria (protein in the urine) that did not recur, didn't cause any complications nor require discontinuation of therapy. I don't think this single adverse events is a big deal, but it's worth keeping an eye on. It's worth noting toxicity concerns have long surrounded competitor GlaxoSmithKline's (GSK - Get Report) drisapersen although I don't believe safety is a big problem for that drug either.

The complexities of human biology can make biotech investing a treacherous endeavor, and Sarepta isn't home free yet. Nonetheless, eteplirsen is clearly a meaningfully active drug and the unmet medical need in DMD is plainly established. I'm not sure of eteplirsen's exact path to market, but the drug will most likely get there and Sarepta's still modest $645 million market capitalization doesn't assume much.

For the patient investor, that's an exciting combination.

Sadgeghi has no position in Sarepta.

Nathan Sadeghi-Nejad has 15 years experience as a professional health-care investor, most recently as a sector head for Highside Capital. He has worked on the sell side (with independent research boutiques Sturza's Medical Research and Avalon Research) and the buyside (at Kilkenny Capital prior to Highside). Sadeghi-Nejad is a graduate of Columbia University and lives in New York. You can follow him on Twitter @natesadeghi.
2 of 2

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
Only $9.95
14-Days Free
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
GSK $46.59 0.00%
SRPT $13.42 0.00%
AAPL $130.28 0.00%
FB $81.53 0.00%
GOOG $565.06 0.00%

Markets

DOW 18,080.14 +21.45 0.12%
S&P 500 2,117.69 +4.76 0.23%
NASDAQ 5,092.0850 +36.0220 0.71%

Partners Compare Online Brokers

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs