This Day On The Street
Continue to site
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

Vertex Motors Down Cystic Fibrosis Highway

CAMBRIDGE, Mass. ( TheStreet) -- Back in June, I used the simple roadmap (above) to explain how Vertex Pharmaceuticals (VRTX - Get Report) 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

>To submit a news tip, send an email to:

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.

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
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
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free
VRTX $82.92 2.80%
AAPL $92.76 -0.92%
FB $117.57 -0.42%
GOOG $697.20 0.22%
TSLA $213.00 -4.30%


Chart of I:DJI
DOW 17,644.21 -7.05 -0.04%
S&P 500 2,053.26 +2.14 0.10%
NASDAQ 4,729.1960 +3.5570 0.08%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs