Vertex Pharmaceuticals (VRTX - Get Report) swapped around the primary and secondary endpoints of the two phase III clinical trials dubbed "Traffic" and "Transport" which are investigating combination therapy in cystic fibrosis patients.
The primary endpoint of both cystic fibrosis studies is now the absolute change in lung function, which before Thursday was a key secondary endpoint. Relative change in lung function was the primary endpoint but is now a key secondary endpoint.
The decision to swap out endpoints was made as "part of ongoing discussions with the FDA and was not based on anything going on in the study or anything observed in the study," said Vertex spokesman Zach Barber.
Barber added: "We consider this more procedural because we were always planning to measure and report both absolute and relative change endpoints. Nothing changes in terms of the powering of the study."
The "Traffic" and "Transport" studies investigate the combination of Kalydeco and VX-809 in cystic fibrosis patients with the F508del homozygous mutation. Results from these two studies are expected in mid-2014 and have the potential to deliver $6 billion or so in new sales to the company. To call these studies important for the future of Vertex's cystic fibrosis franchise is an understatement, and naturally, investors are paying very close attention to them.
Absolute change in lung function was the primary endpoint of the phase III studies which Vertex used to get Kalydeco approved for cystic fibrosis patients with the G551D mutation. With that said, Vertex executives in the past have talked about a 5% relative improvement in lung function as an important efficacy threshold for the Kalydeco-VX-809 combination therapy.
The "Traffic" and "Transport" studies are designed with 90% power to detect a 3% absolute and 5% relative improvement in lung function, measured by forced expiratory volume in 1 second (FEV1).
10/14/15 - 11:47 AM EDT
05/19/15 - 04:42 PM EDT
05/19/15 - 12:48 PM EDT
05/18/15 - 08:06 AM EDT
05/05/15 - 02:43 PM EDT
05/04/16 - 17:09 PM EDT
05/04/16 - 16:49 PM EDT
05/04/16 - 16:24 PM EDT
05/04/16 - 14:38 PM EDT
05/03/16 - 17:03 PM EDT
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.
Trifecta Stocks analyzes over 4,000 equities weekly to find the elite 1% of stocks that pass rigorous quantitative, fundamental and technical tests.
Access the tool that DOMINATES the Russell 2000 and the S&P 500.
Chris Versace, using sophisticated stock screening and fundamental research, identifies potentially explosive small and mid-cap stocks.
Master swing trader Alan Farley uses his sophisticated software screens to review thousands of stocks each day for you, to find just the handful that meet his demanding criteria.