Vertex Pharmaceuticals Inc. (VRTX) recently reported final data from a late-stage trial (STRIVE) on VX-770, which is being studied for the treatment of cystic fibrosis (CF). The data were presented at the European Cystic Fibrosis Society (ECFS) conference.
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 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%.
Additionally, VX-770 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 of signs and symptoms of the disease, requiring treatment with antibiotics), compared with 41% of the patients who received placebo. Moreover, patients treated with VX-770 had 55% less chance of experiencing a pulmonary exacerbation compared to those treated with the placebo.
On an average, patients on VX-770 gained nearly seven pounds through 48 weeks and experienced a reduction in the amount of salt in the sweat (sweat chloride), along with fewer respiratory symptoms.
We note that STRIVE is one of three studies being conducted by Vertex Pharma with VX-770. The other two include the phase II DISCOVER study and the phase III ENVISION study. All three studies have been concluded, and based on their results, the company plans to submit global regulatory applications for approval.
At the ECFS conference, Vertex Pharma also reported data from the phase III ENVISION study. The company stated that the data obtained after 48 weeks of treatment with VX-770 were consistent with the 24-week data announced in March.
The ENVISION study enrolled 52 patients aged 6–11 years, who were dosed with either VX-770 (150 mg) or placebo twice daily. 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 improvement in other measures of disease, including weight gain and a reduction in sweat chloride.
Vertex Pharma plans to file the new drug application (NDA) with the US Food and Drug Administration (FDA) and the marketing authorization application (MAA) with the European Medicines Agency (EMA) in the second half of 2011, seeking approval to market VX-770 in the US and EU as a treatment for CF.
Vertex Pharma is planning an expanded access program for VX-770 in order to meet the immediate critical needs of patients with at least one copy of the G551D mutation. With the expanded access program, these patients might benefit from the candidate prior to potential approval in the US. The company anticipates opening the program at the US trial sites by July, following permission from the FDA.
Vertex Pharma is also working with regulatory bodies outside the US for implementing additional expanded access programs in ex-US countries, with the target of opening the programs for eligible patients in the second half of 2011.
Our View
We currently have a Neutral recommendation on Vertex Pharma, which carries a Zacks #3 Rank (short-term Hold rating). We are pleased with the data on VX-770.
We note that other companies like PTC Therapeutics, Inc. are also developing treatments for CF. PTC Therapeutics’ late-stage candidate ataluren is being developed in collaboration with Genzyme, which is now a part of Sanofi-Aventis (SNY).
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