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Vertex Pharmaceuticals Inc. (VRTX - Analyst Report) 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.

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