Follow-Up Data on Vertex's Kalydeco

Vertex Pharmaceuticals Inc. ( VRTX - Snapshot Report ) recently announced new data from a long-term follow-up study, PERSIST, of Kalydeco in patients suffering from cystic fibrosis (CF).

The PERSIST study is being conducted in patients who had participated in one of the pivotal studies (STRIVE or ENVISION) with Kalydeco and who continued being treated with Kalydeco. The data from the follow-up study showed that improvement in lung function, respiratory symptoms and weight gain in patients treated with Kalydeco for 48 weeks in STRIVE or ENVISION was durable for up to 96 weeks of treatment.

Patients with CF aged 6 years and above who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene were enrolled in the ongoing PERSIST extension study. Those enrolled in this study met certain eligibility criteria after completing treatment in the phase III STRIVE or ENVISION studies.

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 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 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.