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). In the phase III (STRIVE) trial, which compared VX-770 with placebo, all the primary and secondary endpoints were met. Following the trial results, the Vertex Pharma’s share price shot up 15%

The study, which enrolled 161 patients aged 12 years and older, dosed either VX-770 (150 mg) once daily or placebo twice daily. The trial data demonstrated that the candidate helped in improving the lung function (forced expiratory volume in one second, or FEV1) through week 24, which was then sustained through week 48, as compared with those who received placebo.

The data from the trial also showed that VX-770 met the primary endpoint of achieving a mean absolute improvement in lung function (10.6%) from baseline through week 24, when compared to placebo. Mean absolute improvement in lung function among those treated with the candidate through week 48 was 10.5%.

VX-770 also met the secondary endpoint in the study through week 48. 67% of the patients on the drug did not experience a pulmonary exacerbation (periods of worsening in signs and symptoms of the disease requiring treatment with antibiotics), compared with 41% of the patients who received placebo.

On average, patients who were given VX-770 gained nearly seven pounds through 48 weeks. Moreover, there was a reduction in the amount of salt in the sweat (sweat chloride) and fewer respiratory symptoms among people treated with VX-770.

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

Vertex Pharma also reported data from the DISCOVER study, which was conducted to provide additional safety data to support the new drug application (NDA) submission for VX-770. The trial did not demonstrate a statistically significant improvement in treating CF patients with the candidate when compared with placebo.

Results of the ENVISION trial, which is being conducted in children aged 6-11 year, are expected to be released in mid-2011. Vertex Pharma plans to make the US and European regulatory submission for VX-770 in the second half of 2011.

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 – Analyst Report). 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 seven 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.