Vertex Drug Does It Again

Vertex Pharmaceuticals Inc. (VRTX - Snapshot Report) recently reported positive data from a late stage trial on VX-770, being evaluated for the treatment of cystic fibrosis (CF). All the primary and secondary endpoints were met in the phase III (ENVISION) trial, which compared VX-770 with placebo.

The study, which enrolled 52 patients aged 6 to 11 years, dosed either VX-770 (150 mg) or placebo twice daily. The trial data demonstrated that the difference in mean absolute improvement from baseline in lung function through 24 weeks in children treated with VX-770 was 12.5%, while the difference in mean relative improvement from baseline was 17.4%, as compared to placebo.

VX-770 also met the secondary endpoints of improvements in other measures of disease, including weight gain and a reduction in sweat chloride, through week 24.

We note that ENVISION is one of the three studies being conducted with VX-770. The other two include the phase II DISCOVER trial and the phase III STRIVE trial.

Last month, Vertex Pharma reported data from the STRIVE and DISCOVER trials. While the STRIVE study met all the primary and secondary endpoints, the DISCOVER trial did not demonstrate a statistically significant improvement in treating CF patients with the candidate when compared to placebo.

We note that the company is on track to apply for approval of VX-770 in the US and EU in the second half of 2011. The regulatory submissions will be based on data from all the aforementioned three studies.

Once approved, Vertex Pharma could face competition for VX-770 from ataluren, which is being developed by PTC Therapeutics Inc. in collaboration with Genzyme Corp. (GENZ). Ataluren is currently in a late-stage trial.

Vertex Pharma also has a phase IIa trial in progress evaluating VX-770 in combination with VX-809 in people with two copies of the F508del mutation. The first part of the study will evaluate VX-809 (200 mg), or placebo, dosed alone for 14 days and the next part will evaluate VX-809 in combination with VX-770 (150 mg or 250 mg), or placebo, for the next 7 days. Vertex Pharma plans to report data from the first part of the trial in the first half of 2011.

We note that VX-770 has fast track status in the US and orphan drug designation in the US and Europe.

Our Take

We currently have a Neutral recommendation on Vertex Pharma, which is supported by a Zacks #3 Rank (short-term Hold rating). The positive data and the eventual approval of VX-770 should help in the long-term growth of the company. Meanwhile, another key candidate in Vertex Pharma’s pipeline, telaprevir, is currently under regulatory review for the treatment of hepatitis C virus. The US Food and Drug Administration (FDA) has set May 23, 2011, as the target date for telaprevir.

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