Vertex Pharmaceuticals Inc. will receive an additional $22 million in research funding through 2008 for its work in collaboration with Cystic Fibrosis Foundation Therapeutics Inc. to find effective therapies for the genetic disease.

The research program began in 2000 between CFFT, a nonprofit affiliate of the Cystic Fibrosis Foundation based in Bethesda, Md., and Aurora Biosciences Corp., of San Diego, a company later acquired by Vertex. CFFT and Vertex extended the agreement in May 2004 with another $21 million in funding. (See BioWorld Today, May 25, 2004.)

The new contracted payments will support Vertex's research into corrector compounds, which may work by increasing the number of cystic fibrosis transmembrane conductance regulator (CFTR) channels on the cell surface. CFTR is the defective cell membrane protein responsible for the progression of cystic fibrosis. It affects the transport of chloride and other ions across cells, and leads to accumulation of thick mucus in the lungs.

Cambridge, Mass.-based Vertex also has worked with CFFT on a different approach, the use of potentiator compounds for cystic fibrosis. Potentiator compounds may work by increasing the probability that the CFTR channel is open, thereby increasing chloride transport across the cell surface.

"There's great potential in both approaches," said Zach Barber, a media relations specialist with Vertex, who added that "this is the second extension of the collaboration," which to date has "resulted in payments of more than $40 million" to Vertex.

Cystic fibrosis is a genetic disease that affects about 30,000 people in the U.S. It is characterized by a defect in the CFTR gene that causes the body to produce the abnormal mucus that leads to chronic lung infections and impairs digestion. In the mid-1950s, few children with the disease lived to attend elementary school, but today, the median predicted age of survival for those with cystic fibrosis is the mid-30s.

People with cystic fibrosis inherit two defective genes - one from each parent. More than 10 million Americans are unknowing carriers of the defective cystic fibrosis gene. Treatment of the disease involves clearing mucus from the lungs on a daily basis through chest physical therapy - a vigorous clapping on the back and chest. Other treatments include TOBI (tobramycin solution for inhalation, Chiron Corp.), an antibiotic for lung infections; Pulmozyme (dornase alfa, recombinant, Genentech Inc.), a mucus-thinning drug; and azithromycin, an antibiotic.

About 90 percent of people with cystic fibrosis take pancreatic enzyme supplements to help with food digestion, according to data from the Cystic Fibrosis Foundation.

"There is currently no disease-modifying treatment available to patients," Barber told BioWorld Today. "A treatment that addresses the underlying defect in CF could transform" the disease's treatment landscape.

With the $22 million in additional funding, Vertex hopes to bring a corrector compound by the first quarter of 2008 to the level of development already reached by its potentiator compound, which is expected to begin a clinical trial later in the year.

Clinical trials will determine whether potentiator and corrector compounds would be used together or separately in treating cystic fibrosis.

Vertex identified the compounds through its expertise in ion channels, including high-content cell assays and medicinal chemistry. It retains all rights to develop and commercialize the compounds, but would pay CFFT royalties on net sales.

It's too early to say whether Vertex would partner the compounds for clinical development and commercialization, Barber said.

The market is small - one for which a company of Vertex's size easily could build up its own sales force, although the company has not disclosed any such plans.

Vertex has two approved compounds, Agenerase and Lexiva/Telzir, for HIV infection and AIDS, as well as several candidates in clinical development including VX-385 for HIV; merimepodib for hepatitis C virus (HCV); VX-950 for HCV; VX-702 for rheumatoid arthritis; VX-765 for psoriasis; pralnacasan for inflammatory disease; and VX-680 and VX-944 for oncology.

In December, the company signed a potential $405 million deal with London-based GlaxoSmithKline plc for worldwide development and commercialization of a preclinical pain product, VX-409. (See BioWorld Today, Dec. 14, 2005.)

Vertex's stock (NASDAQ:VRTX) lost 25 cents Thursday to close at $33.67.