Macro Tsimmis

intelligently hedged investment

Vertex (VRTX) update #18

Posted by intelledgement on Mon, 20 Oct 08

Our development-stage biotech company, Vertex (VRTX), made news today and for once it had nothing to do with telaprevir. The company released results from a small phase 2a study of their cystic fibrosis candidate drug, VX-770, and according to the press release, Vertex management deem these good enough to warrant a move into phase 3 trials in the USA and Europe, pending regulatory concurrence.

There are an estimated 30,000 people in the USA afflicted with cystic fibrosis (CF), a fatal genetic disease caused by a genetic mutation that impedes breathing. Currently available therapies treat CF by managing the symptoms of the disease…but not all that effectively, as the median life expectancy for people with CF is 37 years. In contrast, VX-770 is designed to increase the functionality of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein on cell walls, which in people with CF, fails to optimally balance salt and water—which results in the fluid buildup in the lungs that impedes breathing. Thus, if it works, it could virtually cure the disease…so long, presumably, as the patient keeps taking the drug, at least. 

To date, VX-770 has only been tested on people with a specific type of CFTR mutation known as G551D that is present in approximately 4% of the CF patient population in the USA. Vertex do expect to evaluate VX-770 in patients with other mutations that result in malfunctioning CFTR on the cell surface, but it is unclear if such an expansion would be part of the projected phase 3 trials or require a new set of phase 2 trials first.


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