Vertex and CFFT extends CF collaboration

By Wai Lang Chu

- Last updated on GMT

Related tags Cystic fibrosis

Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics
have extended their research collaboration initiated in May 2000,
in which CFFT will provide an additional $22 million to further
develop Vertex's therapies that restore the function of a defective

The latest extension builds on the last extension to the deal, which occurred in May 2004, and continues the research and development between the companies into the cystic fibrosis transmembrane conductance regulator (CFTR) protein, the defective cell membrane protein responsible for the progression of cystic fibrosis (CF).

Under the extended agreement, CFFT will provide to Vertex approximately $22 million (€18.2 million) in additional contracted payments for corrector research through the first quarter of 2008, with the first payment to be received in the first quarter of 2006.

Vertex retains the right to develop and commercialise any compounds discovered under the agreement, and will pay CFFT royalties on net sales.

The hope is that by focusing on ion channels, including high-content cell assays and medicinal chemistry, identification of selective ion channel modulators for the treatment of CF will be possible.

Indeed, Vertex's CF research has focused on two possible alternative approaches to CF treatment, known as "potentiator" and "corrector" approaches.

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 work by increasing the probability that the CFTR channel is open, which could result in an increase in chloride transport across the cell surface.

Corrector compounds work by increasing the number of CFTR channels on the cell surface.

"The expansion of this collaboration with Vertex underscores our belief that the development of small molecule agents to address the underlying defect of cystic fibrosis has the greatest potential to change the treatment of the disease,"​ said Robert Beall, president and CEO of the CF Foundation and CFFT.

Cystic fibrosis is a genetic disease affecting approximately 30,000 people in the US. 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.

"Vertex's cystic fibrosis research program has yielded two innovative approaches for the treatment of this disease,"​ said 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,"​ he added.

A number of pharmaceutical and biotechnology companies are currently in the throes of Cystic Fibrosis, in which the emphasis is on treating the disease with minimum side effects.

Inspire Pharmaceuticals recently announced the initiation of an additional Phase 2 clinical trial to evaluate Inspire's drug candidate, INS37217 Respiratory (denufosol tetrasodium), as a potential treatment for cystic fibrosis (CF).

Likewise, Altus Pharmaceuticals presented results in October last year, from its Phase II clinical trial of ALTU-135 (TheraCLEC) in cystic fibrosis (CF) patients with malabsorption due to pancreatic insufficiency.

ALTU-135 is an orally-delivered, microbially-derived enzyme replacement therapy in development that utilises the Company's protein crystallisation and cross-linking technology for the treatment of malabsorption due to exocrine pancreatic insufficiency.

ALTU-135 consists of three enzymes, lipase, protease and amylase that are delivered in a consistent ratio and is designed to improve fat, protein, and carbohydrate absorption in pancreatic insufficient individuals.

Related topics Preclinical Research

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