Cystic fibrosis is a genetic disease affecting approximately 30,000 people in the United States. A defect in the CFTR gene causes the body to produce abnormally thick, sticky mucus that leads to chronic, life-threatening lung infections and impairs digestion.
VX-770 is the first of a new class of oral agents that specifically target a key mechanism underlying CF. The drug candidate advanced into preclinical development based on a successful five-year research collaboration with CFFT that incorporated capabilities and proprietary research from Vertex's San Diego research site.
VX-770 may act to restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, the defective cell membrane protein responsible for the progression of 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 of patients with CF.
This mucus fosters chronic infection and inflammation, and results in irreversible lung damage. Potentiator compounds such as VX-770 are designed to increase the probability that the CFTR channel is open, which could result in an increase in chloride transport across the cell surface in some patients.
In laboratory experiments, using cells from patients with CF where CFTR proteins are present on the cell surface, VX-770 has restored the function of defective CFTR channels.
Under the terms of the agreement, CFFT will pay Vertex approximately $13.3 million (€11 million) in development support through the fourth quarter of 2007.
Vertex plans to initiate clinical development of VX-770 in the second quarter of 2006 and to progress to clinical studies in patients with CF in the second half of the year. Vertex retains worldwide rights to develop and commercialise VX-770.
"VX-770 represents a novel approach to addressing the progression of cystic fibrosis and is the first clinical drug candidate to emerge from our innovative CF research efforts," said Joshua Boger, Chairman, President and Chief Executive Officer of >Vertex.
"We appreciate CFFT's continued support for the development of VX-770, and we look forward to initiating the first clinical study of this novel compound in patients with CF later in 2006."
Additional terms will see CFFT and Vertex share certain costs associated with clinical development of VX-770.
CFFT will provide to Vertex approximately $13.3 million to support clinical development of VX-770 through the fourth quarter of 2007.
Vertex retains worldwide rights to develop and commercialise VX-770. Upon commercialisation, Vertex would pay CFFT certain royalties and sales milestones based on specific net sales thresholds.
Vertex initiated its CF research program in May 2000 as part of a collaboration with CFFT, and expanded the agreement in May 2004.
In addition to the new collaboration announced today, in January 2006, Vertex and CFFT entered into an expanded research collaboration to discover novel compounds known as correctors, which may work by increasing the number of CFTR channels on the cell surface.
"We are excited to continue to work with Vertex on this exciting small molecule drug candidate. Our support should accelerate the clinical development of VX-770, a compound that holds great potential for changing the course of this disease," said Robert Beall, President and Chief Executive Officer of the >Cystic Fibrosis Foundation (CFFT).
"The collaboration announced today offers hope that together we may one day soon provide a new treatment option to patients with CF. Our common goal to accelerate the clinical development of VX-770 represents yet another example of the productive collaborative history we share with Vertex in CF research."