The commercial arm of the Cystic Fibrosis Foundation in the US has agreed to provide Pfizer with $58m in funding to help advance the drugmaker’s CF drug discovery programme.
The aim is to develop new treatments for the most common genetic mutation in CF – known as delta F508 – advancing one or two candidates into clinical trials by the end of the six-year project.
Pfizer has increasingly opted to forge collaborations with public groups, such as patient organisations, to help it advance therapeutic programmes for rare disease, and the deal with Cystic Fibrosis Foundation Therapeutics (CFFT) ties in with that strategy.
This type of public-private partnership is “increasingly critical in expediting the translation of science into new treatments,” commented Jose-Carlos Gutierrez-Ramos, senior VP of Pfizer BioTherapeutics R&D.
In people with the delta F508 mutation, a defective protein called CFTR does not fold correctly and is unable to reach the cell surface, where it is needed to help maintain the proper flow of salt and fluids into the airways.
As a result, thick secretions form in the airways, leading to serious lung infections and lung damage, said Pfizer. Nearly 90 per cent of people with CF have at least one copy of the delta F508 mutation, and the project with CFFT will focus on identifying therapies that help restore the normal function of CFTR.
The pharma company’s interest in CF came with its acquisition of FoldRx Pharmaceuticals in 2010. FoldRx had a five-year, $22m collaboration with CFFT aimed at using its yeast-based, high throughput screening platform to identify new CF drugs.
The lead drug in FoldRx’ portfolio – tafamidis for the rare genetic condition transthyretin familial amyloid polyneuropathy – was filed for approval in the US and Europe last year, although the submission was turned down by the FDA on the grounds that it needed more data to complete its review.