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
$1 buys you full access to ALL of TheStreet's Subscription Services! Learn More

Vertex Motors Down Cystic Fibrosis Highway

Stocks in this article: VRTX

CAMBRIDGE, Mass. ( TheStreet) -- Back in June, I used the simple roadmap (above) to explain how Vertex Pharmaceuticals (VRTX) will one day come to dominate cystic fibrosis therapy and generate annual sales of $10 billion with little to no competition.

On Monday, Vertex announced positive results from the first of several follow-on phase III studies aimed at expanding treatment with Kalydeco as a stand-alone therapy. This new use for Kalydeco encompasses about 400 cystic fibrosis patients with a non-G551D gating mutation. If you're keeping track using the roadmap, these patients are included in the smaller blue box.

Four hundred new cystic fibrosis patients translates into approximately $100 million in additional revenue for Vertex, according to Baird analyst Brian Skorney, so it's a relatively small step in the overall expansion of Vertex's cystic fibrosis business.

Vertex intends to file for Kalydeco label expansion for the non-G551D gating mutation in the second half of year in the U.S. and Europe. In the U.S., FDA has already granted Kalydeco breakthrough therapy designation, so the review and approval should happen quickly.

Next up for Vertex is a phase III study of Kalydeco monotherapy in cystic fibrosis patients with an R117H mutation. Results are expected in the second half of this year and if positive, will lead to another regulatory expansion filing in early 2014.

The R117H mutation is found in about 3 percent of cystic fibrosis patients worldwide, according to Vertex.

Another study of Kalydeco in cystic fibrosis patients with "residual function" mutations is ongoing with results expected in the first half of next year.

All of these Kalydeco monotherapy expansion studies combined represent about 5,000 new cystic fibrosis patients in that smaller blue box on the roadmap.

After that, the next important destination is the combination studies of Kalydeco and VX-809. Two large phase III studies, dubbed TRAFFIC and TRANSPORT, in patients with the homozygous 508del mutation will have results in the first half of next year. There are about 30,000 cystic fibrosis patients in the larger blue box, so it's a very important milestone for Vertex.

Vertex reported $99 million in Kalydeco sales in the second quarter, well above Street consensus. The company also raised guidance on Kalydeco sales for the remainder of the year.

--Written by Adam Feuerstein in Boston.



>To contact the writer of this article, click here: Adam Feuerstein.

>To follow the writer on Twitter, go to http://twitter.com/adamfeuerstein.

>To submit a news tip, send an email to: tips@thestreet.com.

Follow TheStreet on Twitter and become a fan on Facebook.

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!

COMPARE ALL SERVICES
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 17,810.06 +91.06 0.51%
S&P 500 2,063.50 +10.75 0.52%
NASDAQ 4,712.97 +11.1030 0.24%

Brokerage Partners

Rates from Bankrate.com

  • 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