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 Readies Late-Stage Cystic Fibrosis Drug Studies

( TheStreet) -- Vertex Pharmaceuticals (VRTX - Get Report) announced Tuesday night the design of two phase III studies for its combination therapy to treat the most common form of cystic fibrosis. The studies will each run for six months, so results could be ready as early as the end of 2013 or during first half of 2014.

If the studies are positive, Vertex intends to file for U.S. approval in 2014.

The combination therapy program is vital to Vertex's future growth because the form of cystic fibrosis targeted -- patients with a genetic mutation known as F508del -- make up about half of the 70,000 patients worldwide. Vertex's currently approved cystic fibrosis drug Kalydeco targets only 4% of patients with the G551D mutation. Kalydeco sales totaled $172 million in 2012.

The studies announced Tuesday will evaluate the two different doses of an experimental medicine VX-809 in combination with Kalydeco. Each study will enroll 500 cystic fibrosis patients randomized to either the VX-809/Kalydeco arms or a placebo for six months of treatment. The studies' primary endpoint will be the relative improvement in lung function of VX-809/Kalydeco compared to placebo.

Last fall, Vertex presented data from a phase II study demonstrating that a 600 mg dose of VX-809 and Kalydeco worked synergistically to improve lung function in cystic fibrosis patients with the F508del mutation compared to placebo. This same dose combination will be tested in the phase III study along with a higher 800 mg (actually, 400 mg given twice a day) dose of VX-809 plus Kalydeco.

Vertex also announced new data from this phase II study on Tuesday night showing similar lung function improvements between the 800 mg and 600 mg doses of VX-809. For this reason, the higher dose was included in the phase III studies.

Along with the two phase III studies in adult patients, Vertex will also conduct a six-month study of the combination therapy in pediatric patients ages 6 to 11. This study, along with the data from the adult studies, may be used to expand the combination therapy's approval into younger patients.

In January, FDA anointed Kalydeco and VX-809 with Breakthrough Therapy Designation as part of the agency's efforts to accelerate the development and approval of drugs for serious and life-threatening disease. Vertex did not say whether Breakthrough Designation played a specific role in the VX-809/Kalydeco phase III program but the relatively short six-month duration of the studies plus the ability to test the combination in children at the same time does accelerate the development of the combination therapy. If the data from the studies are positive, the drugs could be approved sooner than expected and for more patients.

Vertex is conducting a phase II study of another experimental cystic fibrosis drug known as VX-661, which could be more potent than VX-809. Results are expected before the end of the second quarter.

Ahead of Tuesday's announcement, Vertex shares closed down 1.5% to $44.18. The stock was up slightly in after-hours trading.

-- 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.

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 $85.20 1.40%
AAPL $93.16 -0.09%
FB $117.68 -0.11%
GOOG $703.22 0.26%
TSLA $211.54 0.00%


Chart of I:DJI
DOW 17,648.97 -11.74 -0.07%
S&P 500 2,048.63 -2.00 -0.10%
NASDAQ 4,709.3430 -7.7510 -0.16%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs