Monday, October 20, 2008

Vertex lands positive results from cystic fibrosis Phase 2a trials

By Mass High Tech Staff

Biotech firm Vertex Pharmaceuticals Inc. has reported encouraging results from the 28-day, second part of a Phase 2a clinical trial of the company’s oral drug VX-770 to treat cystic fibrosis patients who have a particular protein mutation, the G551D CFTR mutation.

In the trial, the company said patients experienced no significant side effects nor abandoned the trial due to the 150 mg or 250 mg doses twice a day. Instead, the trial showed improvements in lung functionality and operations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. The protein CFTR exists in healthy people for the purpose of recognizing a lung-damaging bacteria and removing it. In patients with cystic fibrosis, this protein is either missing or faulty, causing them to develop chronic lung infection and almost always leading to early death before the age of 35 years, ScienceDaily reports. Improvements in lung and CFTR functions were marked by changes in both the sweat chloride and the “nasal potential difference” for those patients receiving the VX-770, compared to no changes for those patients receiving the placebo.

According to Cambridge-based Vertex (Nasdaq: VRTX) officials, the results “support the hypothesis that improving chloride ion transport in CF patients may correlate with improvements in lung function.”

Researchers at Harvard Medical School first reported the linkage of the CFTR protein to cystic fibrosis in September.

With 1,132 employees as of March 2008, Vertex is a global biotechnology company focused on the development of small molecule drugs for serious diseases. The company reported a 2007 net loss of $391.3 million on revenue of $199 million.