Macro Tsimmis

intelligently hedged investment

Vertex (VRTX) update #9

Posted by intelledgement on Thu, 27 Mar 08

Hey, shares of Vertex (VRTX) were up today, and it had nothing to do with telaprevir or hepatitis C. The company announced preliminary results of a small Phase 2a trial of VX-770, an investigational CF potentiator. “The interim analysis showed that dosing of VX-770…as an oral agent for 14 days resulted in improved lung function and in improved function of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein as measured by changes in sweat chloride levels and changes in nasal potential difference (NPD),” according to the press release.

These are interim results based on only 20 patients, all of whom carry the G551D mutation in the gene that causes CF. This patient population amounts to only about 1200 people in the USA, although Vertex plan to expand testing to cover more diverse CF patients in future (there are an estimated 30,000 people with CF in the USA overall). Be that as it may, the results are pretty spectacular, with lung function improving 10% in patients who received the highest dosage. There is no approved CF treatment that actually improves lung functionality, in effect rolling back the progression of the disease.

VRTX closed at $19.23, up 8% on the day.

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