Pfizer in cystic fibrosis alliance
pharmafile | November 21, 2012 | News story | Research and Development, Sales and Marketing | CFFT, CFTR, FDA, Pfizer
Pfizer and Cystic Fibrosis Foundation Therapeutics (CFFT) have signed a six-year pre-clinical deal to find new drugs to treat the rare, life-threatening genetic disease cystic fibrosis (CF).
CFFT, the drug discovery arm of the Cystic Fibrosis Foundation charity, will invest up to $58 million in a bid to treat people with Delta F508 – a mutation which almost 90% of CF sufferers have.
It is characterised by a defective protein called CFTR, which does not fold correctly so is unable to reach the cell surface. This means it cannot help maintain the proper amount of salt and fluids flowing into airways, leading to a build-up of thick, sticky mucus in the body’s tubes and passageways.
These blockages can in turn lead to serious lung infections and damage to the digestive system and other organs.
The idea of the collaboration between Pfizer and CFFT is to bring into a clinical environment one or more candidates that help restore this defective protein’s normal function.
The two organisations already have strong ties, with Pfizer buying biotech company FoldRx Pharmaceuticals in 2010, in an acquisition which included FoldRx’s pre-existing CF research programme with CFFT.
Robert Beall, president of the CF Foundation, said it was important to develop therapies that treat the ‘root cause’ of the disease.
Meanwhile Jose-Carlos Gutierrez-Ramos, senior vice president of Pfizer BioTherapeutics R&D, praised industry/patient tie-ups as “increasingly critical in expediting the translation of science into new treatments”.
There are relatively few treatments for CF on the market: Vertex’s drug Kalydeco received FDA approval in January to treat a small sub-set of CF patients – those who have the G551D mutation in the CFTR gene.
Vertex is currently conducting trials with Kalydeco and its own pipeline drug VX-809 in CF, although there was a problem over VX-809 data.
However, Kalydeco itself is groundbreaking, since no other medicines are available to treat the cause of CF, rather than just the symptoms.
Adam Hill
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