Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced
results from a preliminary analysis of data from a 28-day Phase 2a
clinical trial of VX-809 in patients with cystic fibrosis (CF) who are
homozygous for the F508del mutation. VX-809, an oral investigational
Cystic Fibrosis Transmembrane Conductance Regulator protein (CFTR)
corrector, was well-tolerated across all four dose groups studied. In
the trial, VX-809 showed a statistically significant decline in sweat
chloride at both the 100 mg and 200 mg once-daily doses, suggesting that
the activity of the CFTR protein was increased in patients during
dosing. Additionally, VX-809 demonstrated a dose response in change in
sweat chloride across the four dose groups. On the basis of these
results, Vertex plans to initiate a combination trial of VX-809 and
VX-770, an investigational CFTR potentiator, in the second half of 2010.
VX-809 and VX-770 were developed with support from Cystic Fibrosis
Foundation Therapeutics, Inc., the nonprofit affiliate of the Cystic
Fibrosis Foundation.
“While the median predicted age of survival for patients with CF has
increased to more than 37 years of age, there are no approved therapies
that directly target the underlying defect of this disease”