Vertex Pharmaceuticals Highlights Cystic Fibrosis Program Targeting The CFTR Protein Responsible For Cystic Fibrosis

VX-770


Based on data presented to date, Vertex intends to work with global regulatory authorities on the design of a registration program for VX-770 which, if agreed upon, could begin in the first half of 2009.


In April and on October 23rd at the 22nd Annual North American Cystic Fibrosis Conference in Orlando, researchers presented interim data from Part 1 (14-day) and Part 2 (28-day), respectively, of a Phase 2a trial of VX-770, an investigational Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiator compound for the treatment of CF. In the Phase 2a study, researchers reported the following:


-- VX-770 was well tolerated when dosed at 25mg, 75mg and 150mg twice daily for 14 days and at 150mg and 250mg when dosed twice daily for 28-days.


-- No patients discontinued treatment and no serious adverse events were reported due to VX-770.


-- With measures of lung function (FEV1) and measures of CFTR activity (nasal potential difference (NPD) and sweat chloride levels) a dose response was observed.


-- Significant improvements in lung function, as measured by an increase from baseline in FEV1.


-- Significant improvements in the function of the CFTR protein, as measured by changes from baseline in biomarkers of CFTR activity - sweat chloride levels and NPD.


VX-809


Vertex recently completed dosing in two Phase 1 trials of VX-809, an investigational oral CFTR corrector compound for the treatment of CF, in healthy volunteers. The first trial is a single and multiple dose study while the second is a single dose study examining the pharmacokinetics (PK) and safety of a solid dosage form. Based on these results, Vertex has initiated a single dose PK and safety trial of VX-809 in patients with CF. The Company plans to initiate a Phase 2a study in patients with CF in the first half of 2009.


Advancements in Pipeline of Other Novel Drug Candidates


VX-509


Vertex is conducting a Phase 1 clinical trial of VX-509, a novel Janus kinase 3 (JAK3) inhibitor. It is anticipated that VX-509 will be investigated for the treatment of multiple immune-mediated inflammatory diseases.


Aurora Kinase Inhibitors


Vertex's collaborator Merck is conducting a Phase 1 clinical trial of the Aurora kinase inhibitor MK-5108 (VX-689) in patients with advanced and/or refractory solid tumors. In addition, in the third quarter Merck selected additional Aurora kinase inhibitors for development and Vertex recognized a milestone of $6.0 million.



About Vertex


Vertex Pharmaceuticals Incorporated is a global biotechnology company committed to the discovery and development of breakthrough small molecule drugs for serious diseases. The Company's strategy is to commercialize its products both independently and in collaboration with major pharmaceutical companies. Vertex's product pipeline is focused on viral diseases, inflammation, autoimmune diseases, cancer, pain and cystic fibrosis. Vertex co-discovered the HIV protease inhibitor, Lexiva, with GlaxoSmithKline.















Lexiva is a registered trademark of the GlaxoSmithKline group of companies.


PEGASYS is a registered trademark of Hoffman-La Roche Ltd.


Special Note Regarding Forward-looking Statements


This press release contains forward-looking statements, including statements regarding Vertex's expectation that (i) it is positioned to make a major impact on the treatment of HCV and CF, with the potential to conduct registration programs in parallel in 2009, (ii) it is well-positioned financially to support investment in its HCV and CF opportunities, (iii) it will have SVR data from the ADVANCE study in the first half of 2010, (iv) it will enroll approximately 500 patients in a Phase 3 clinical trial to evaluate 24-week and 48-week telaprevir-based regimens by the end of 2008 and SVR data from this study will be available in the first half of 2010, (v) the REALIZE study will complete enrollment in the first quarter of 2009, (vi) interim data from Phase 2 clinical study evaluating telaprevir dosing regimens support continued evaluation of twice-daily dosing of telaprevir, (vii) data from Phase 1b clinical trial of VX-500 in HCV patients will be available in the first quarter of 2009, (viii) it will reach agreement with regulatory authorities on a registration program for VX-770 and initiate the study in the first half of 2009, (ix) it will initiate a Phase 2a clinical trial of VX-809 in patients with CF in the first half of 2009, (x) VX-509 will be investigated for the treatment of multiple immune-mediated inflammatory diseases, (xi) the 2008 guidance (including its estimates of stock-based compensation expense) will be in the ranges referenced in the Company's previous guidance. While the Company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the outcomes for each of its planned clinical trials and studies, and in particular its planned clinical trials of telaprevir and VX-770, may not be favorable, that regulatory authorities may require supplemental clinical trials in order to support registration of telaprevir in any particular indication, that the Company will not be able to secure agreement from regulatory authorities on a registration program for VX-770, that there may be varying interpretations of data produced in one or more of our clinical trials, that enrollment may be more difficult or slower than we currently anticipate or that planned clinical trials may not start when planned due to regulatory issues, site startup delays, availability of clinical trial material or other reasons, that regulatory authorities will require more extensive data for a telaprevir or VX-770 NDA filing than currently expected, that one or more of the Company's assumptions underlying its revenue expectations -- including clinical and scientific progress that could lead to milestone payments under existing collaboration agreements or payments under new collaborations -- or its expense expectations -- including estimates of the variables that go into determining stock-based compensation expenses -- will not be realized, that unexpected costs associated with one or more of the Company's programs will necessitate a change in the Company's financial projections, that future competitive or other market factors may adversely affect the commercial potential for the Company's product candidates in HCV or cystic fibrosis , and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the Company's website at clinicaltrials. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.


Source

Michael Partridge

Vertex Pharmaceuticals Incorporated


View drug information on Lexiva; Pegasys.