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!

Remember Vertex? Important Cystic Fibrosis Data On Tap Next Week

Stocks in this article: VRTX

CAMBRIDGE, Mass. ( TheStreet) -- Vertex Pharmaceuticals (VRTX) has slipped from the radar screen of biotech investors, many of whom are still recovering from the American Society of Clinical Oncology annual meeting. But Vertex is presenting additional data on its combination therapy for cystic fibrosis at the European Cystic Fibrosis Society (ECFS) conference on June 13. Investors should take notice.

The first look at positive results from the combination of VX-661 and Kalydeco released by Vertex in April spiked shares from $52 to $85, as it indicated the Vertex drugs could effectively address a much larger share of the cystic fibrosis patient population. Kalydeco is currently only approved for the 4 percent of cystic fibrosis patients carrying the G551D mutation in the CFTR gene. Clearly, expanding the size of the addressable market would be quite meaningful to Vertex.

Cystic fibrosis is caused by dysfunction in the CFTR gene, which severely limits the ability of the CFTR protein to express in the epithelial cells of the lungs, intestines, and exocrine glands. Malfunctioning CFTR proteins inhibits the ability of chloride ions to cross those membranes, which ultimately creates a thick mucus that impedes the proper functioning of the organ and leads to numerous opportunistic infections. In general, a drug (or combinations) needs to address three issues to be effective as a cystic fibrosis therapy: Production of a functional CFTR protein, trafficking the protein to the proper place on the cell membrane, and gating it through the cell membrane.

Kalydeco is effective in the G551D population because these patients have functional CFTR proteins that are properly trafficked but have issues with gating. Kalydeco works as a "potentiator" by essentially opening the gates and letting chloride ions pass through. Kalydeco is not effective in the other 96 percent of cystic fibrosis patients because they have problems with either the production of the protein and/or trafficking it to the proper spot. This is where combination therapy comes into play. A "corrector" drug could handle protein function and trafficking while Kalydeco takes care of gating.

Vertex has three "correctors" in development (VX-809, VX-661, and VX-983) which attempt to address issues in production and trafficking. The ultimate goal is to develop a successful corrector/Kalydeco combination that gets enough functional protein across the cell membrane to produce a benefit for cystic fibrosis patients similar to what's been demonstrated by Kalydeco monotherapy in the G551D mutation population. Vertex's early combination data improved lung function only about half as well as Kalydeco. Still, the improvement in lung function and decrease in sweat chloride production were statistically significant, providing a clear proof of concept. It looks more like a matter of when, not if, Vertex will be able to develop an effective combination treatment for the much larger CF patient populations.

1 of 2

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 18,030.21 +6.04 0.03%
S&P 500 2,081.88 -0.29 -0.01%
NASDAQ 4,773.4720 +8.0480 0.17%

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