Vertex Pharmaceutcals was one of a number of pharma and biotech companies to attend the PiperJaffray healthcare conference in London last week, which gave international companies the opportunity to provide pipeline updates.
Vertex, a biotechnology company that focuses on infectious diseases, inflammation, cancer and pain management provided details of its small molecule treatments within these areas, as well as a progress report on the collaborations established with Big Pharma as it strives to bring its treatments to market.
Central to its product pipeline is the progress already achieved on a potential first-in-class treatment for cystic fibrosis, a genetic disease marked by the accumulation of thick sticky mucus in the lungs and the development of life-threatening lung infections.
Vertex's oral potentiator compound, VX-770 has recently entered phase I clinical study, which has been achieved in conjunction with the Cystic Fibrosis Foundation (CFFT).
Results of previous trials suggested that VX-770 could 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.
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.
As well as the Phase I progress, the drug has also been granted fast track designation by the FDA on the grounds that it has shown promise in treating the life threatening illness and filling an, as yet, unmet need.
Vertex's product pipeline for cancer was also of interest, with details of two drug candidates. One nearing phase I completion and the other commencing phase II..
John Alam, Executive Vice President and Chief Medical Officer told an audience of journalists and investors that the latest preclinical results of VX-944, an oral inosine monophosphate dehydrogenase (IMPDH) inhibitor suggested that VX-944 has potent anti-tumour activity.
"Phase I clinical studies of VX-944 in healthy volunteers demonstrated that VX-944 is orally bioavailable and well-tolerated," he added.
In February 2005, Vertex announced that it entered into a licensing agreement for VX-944 with Avalon Pharmaceuticals.
Recent reports in medical literature and presentations at scientific conferences provide a strong clinical rationale for the development of IMPDH inhibitors generally, and VX-944 specifically, in hematologic malignancies.