Vertex extends cystic fibrosis research collaboration

Under the extended agreement, Cystic Fibrosis Foundation Therapeutics (CFFT) will provide an additional $22 million to Vertex for continued research funding through early 2008. The first payment is to be received in the first quarter of 2006.

These funds will support the development of Vertex’s “corrector” compounds that act to restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, the defective cell membrane protein responsible for the progression of cystic fibrosis (CF).

Vertex has identified selective ion channel modulators for the treatment of CF. With the support of CFFT, Vertex’s CF research has focused on two possible alternative approaches to CF treatment, known as “potentiator” and “corrector” approaches. Each approach might address a different molecular defect in the CFTR protein that is responsible for CF.

Defects in the CFTR protein affect the transport of chloride and other ions across cells, and lead to the accumulation of thick, sticky mucus in the lungs. This mucus fosters chronic infection and inflammation and ultimately results in irreversible lung damage. Potentiator compounds may work by increasing the probability that the CFTR channel is open, which could result in an increase in chloride transport across the cell surface.

“Vertex’s cystic fibrosis research program has yielded two innovative approaches for the treatment of this disease,” said Dr Joshua Boger, chairman, president and CEO of Vertex. “This research extension of the CFFT collaboration underscores our commitment to developing new treatments for this major unmet medical need and supports Vertex’s continued investment focus in this key therapeutic area.”