Vertex Pharmaceuticals Inc.
(
VRTX
) recently announced final data from a phase II study of VX-809 and
Kalydeco. The phase II study evaluated adults suffering from cystic
fibrosis (CF) and two copies (homozygous) of the most common
mutation in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene, F508del.
The study evaluated patients with increasing doses of VX-809
alone for the first 28 days. The next 28 days evaluated the effect
of the combination of VX-809 and Kalydeco. The data revealed that
patients receiving the combination of VX-809 and Kalydeco showed a
significant improvement in lung function compared to placebo from
day 28 to 56.
The positive data from the phase II study will encourage
Vertex's plans to begin a pivotal study evaluating the combination
of VX-809 (600mg) and Kalydeco (250mg) in homozygous subjects in
early 2013.
Very recently, Vertex had announced positive data on Kalydeco
from a long-term follow-up study, PERSIST, that was conducted in
patients suffering from CF. Vertex plans to initiate three
additional pivotal studies this year to evaluate the safety and
efficacy of Kalydeco.
A life threatening genetic disease, CF as estimated by Vertex,
afflicts approximately 70,000 people worldwide.
We remind investors that Kalydeco was approved in the US, in
February 2012, for treating CF patients 6 years and above. Vertex
recorded sales of $18.4 million for Kalydeco in the first quarter
of 2012.
Vertex 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
opinion recently.
Our Take
We note that Kalydeco had a strong first quarter and, with the
probable EU launch, the drug can be a major growth driver in the
future. We expect investor focus to remain on Kalydeco's EU
approval and its performance in the US.
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.
Zacks Investment
Research