Vertex Pharmaceuticals Inc.
) recently corrected previously reported interim data (responder
analysis) from a phase II study of VX-809 and Kalydeco. Vertex also
provided some additional data from the study.
The interim data from the phase II study showed improvements in
lung function in adults with cystic fibrosis (CF) who had two
copies of the F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. The interim data was on 37
patients who were treated with VX-809 and Kalydeco and 11 patients
with one or two copies of the F508del mutation receiving
Earlier this month, Vertex had provided interim data from the
study revealing that approximately 46% patients in the VX-809 plus
Kalydeco arm experienced at least 5% improvement in lung function
from baseline, while 30% of patients in receiving the combination
therapy experienced an improvement of at least 10% from baseline to
However, Vertex in its recent announcement, reported that
approximately 35% (13/37) patients in the VX-809 plus Kalydeco arm
experienced at least 5% absolute improvement in lung function from
baseline and approximately 19% patients experienced an absolute
improvement of at least 10% from baseline to day 56. The company
also mentioned that the previously reported results were relative
and not absolute.
Vertex also provided additional data from the interim analysis
on patients with two copies of the F508del mutation. Patients
treated with VX-809 and Kalydeco showed at least 8.5% absolute
improvement in lung function, in comparison to placebo.
Moreover, people treated with placebo experienced an absolute
decline of about 4.6% in lung function, whereas patients treated
with the combination showed approximately 4% absolute improvement
in lung function from the baseline to day 56.
Vertex intends to start a pivotal study of VX-809 and Kalydeco
in patients with two copies of the F508del mutation, once final
results are out and based on discussions with regulatory agencies.
The company expects complete data to be available in mid-2012.
We remind investors that Kalydeco was approved in the US, in
February 2012, for treating CF patients above 6 years of age.
Vertex Pharma is set to gain approval of the drug for the same
indication in the European Union (EU), with the European Committee
for Medicinal Products for Human Use having given a positive
The incorrect interim data from the phase II study of VX-809 and
Kalydeco was disappointing. This led to a 10.9% fall in the
company's stock price.
We currently have a Neutral recommendation on Vertex Pharma. The
stock carries a Zacks #3 Rank (Hold rating) in the short run
VERTEX PHARM (VRTX): Free Stock Analysis Report
To read this article on Zacks.com click here.