Profile: Vertex Pharmaceuticals Inc (VRTX.O)

Vertex Pharmaceuticals Incorporated (Vertex), incorporated in 1989, is in the business of discovering, developing, manufacturing and commercializing small molecule drugs for the treatment of serious diseases. The Company’s two products are INCIVEK (telaprevir), which is approved for the treatment of patients with genotype 1 hepatitis C virus (HCV), infection, and KALYDECO (ivacaftor), which is approved in the United States for the treatment of patients six years of age and older with cystic fibrosis (CF), who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), gene. The Company has ongoing clinical programs involving drug candidates intended for the treatment of HCV infection, CF, rheumatoid arthritis, influenza and epilepsy. Its HCV clinical programs are focused on developing all-oral, interferon-free combinations of HCV drugs and drug candidates that further improve treatment options available to patients with HCV infection. INCIVEK (telaprevir) achieved initial commercial acceptance following its approval in May 2011. It started marketing KALYDECO in the United States in January 2012. In June 2011, it entered into a license and collaboration agreement with Alios BioPharma, Inc.

HCV INFECTION

Telaprevir is an orally-administered HCV protease inhibitor that is indicated for the treatment of treatment-naive and treatment-failure adults with genotype 1 HCV infection. The Company markets telaprevir in the United States and Canada under the brand name INCIVEK, Janssen markets telaprevir under the brand name INCIVO in the United Kingdom, Germany, France, Sweden, Austria, Finland, Denmark, Switzerland and Norway, and Mitsubishi Tanabe markets telaprevir under the brand name TELAVIC in Japan. Patients who are prescribed a telaprevir-based treatment regimen receive telaprevir, peg-IFN and RBV for 12 weeks. Peg-IFN is a medicine that is administered weekly by injection. Telaprevir is indicated for three-times-daily dosing and is being evaluated in a fully-enrolled Phase 3b clinical trial designed to support a supplemental New Drug Application (NDA), for twice-daily dosing, and comparable applications in the European Union.

The Company is conducting Phase 3b clinical trials to evaluate telaprevir-based combination regimens as treatments for genotype 1 HCV infection in patients who also have HIV infection and in patients who experience recurrent genotype 1 HCV infection following a liver transplant. In addition, it is evaluating a 12-week telaprevir-based combination regimen in a Phase 3 clinical trial in patients with genotype 1 HCV who have a specific variant in the patient's IL-28B gene, which is referred to as the CC variant. Its HCV drug candidates, VX-222, ALS-2200 and ALS-2158, are designed to inhibit the replication of HCV by inhibiting the HCV NS5b polymerase enzyme. The Company is evaluating VX-222 in combination with telaprevir and RBV and plan to evaluate multiple combination regimens that incorporate its other HCV drug candidates.

Non-nucleoside HCV polymerase inhibitors, such as its investigational drug candidate VX-222, bind to the NS5b polymerase enzyme, changing its shape and inhibiting its enzymatic activity. HCV nucleotide analogues, such as its investigational drug candidates ALS-2200 and ALS-2158, also act on the HCV NS5b polymerase enzyme. VX-222 is being evaluated in a Phase 2 clinical trial referred to as ZENITH. ZENITH is designed to evaluate combination treatment regimens of telaprevir, VX-222 and RBV, with and without peg-IFN. The Company has completed dosing in two, all-oral three-drug treatment arms of ZENITH in which treatment-naive patients with genotype 1a HCV infection and genotype 1b HCV infection received VX-222 in combination with telaprevir and RBV. In ZENITH, it also evaluated two dose levels of VX-222 in combination with telaprevir, RBV and peg-IFN in two treatment arms that enrolled a total of 59 patients with genotype 1 HCV infection. In December 2011, its collaborator, Alios, and the Company initiated Phase 1 clinical trials to evaluate the safety and tolerability of single ascending doses of each of ALS-2200 and ALS-2158 taken alone in healthy volunteers, and of multiple ascending doses of each of ALS-2200 and ALS-2158 taken alone in patients with genotype 1 HCV.

CYSTIC FIBROSIS

The Company holds worldwide development and commercialization rights to KALYDECO, VX-809 and VX-661. KALYDECO (ivacaftor) is an orally-administered CFTR potentiator approved in January 2012 in the United States for the treatment of patients six years of age and older with CF who has the G551D mutation on at least one allele. The Company is seeking approval from the European Commission to market ivacaftor for the treatment of patients with CF six years of age and older with the G551D mutation in the CFTR gene and with certain other mutations in the CFTR gene that result in gating defects. CFTR gene include the G551D mutation.

The Company is investigating treatment regimens combining KALYDECO with its investigational correctors VX-809 and VX-661. VX-809 and VX-661 are oral CFTR corrector compounds. The Company is evaluating VX-809 in combination with ivacaftor in patients with CF who have the F508del mutation in the CFTR gene. During the year ended December 31, 2011, it obtained interim data from Part 1 of a Phase 2 clinical trial designed to evaluate multiple combination regimens of ivacaftor and VX-809, which enrolled 62 patients with CF with the F508del mutation on both alleles. Part 1 of the clinical trial evaluated a 200 mg dose of VX-809, or placebo, alone for 14 days and then in combination with two doses of ivacaftor, or placebo, for 7 days. In Part 2, the Company isevaluating VX-809 alone for 28 days followed by VX-809 in combination with KALYDECO for 28 days compared to placebo. It initiated a Phase 2 clinical trial of VX-661 in 2012.

IMMUNE-MEDIATED INFLAMMATORY DISEASES

VX-509 is an investigational oral drug candidate intended to inhibit Janus kinase 3 (JAK3), which is involved in the modulation of a type of white blood cell, referred to as a lymphocyte, that is central to auto-immune disease pathology. In 2011, the Company completed a Phase 2a clinical trial that evaluated VX-509 in patients with rheumatoid arthritis.

EPILEPSY

VX-765 is an interleukin-1 converting enzyme (ICE), inhibitor. VX-765 is designed to inhibit an enzyme that controls the generation of two cytokines, IL-1b and IL-18. In 2011, we completed a Phase 2a clinical trial of VX-765 that randomized approximately 60 patients with treatment-resistant epilepsy. This clinical trial was designed to evaluate the safety, tolerability and clinical activity of VX-765. The Company has initiated a Phase 2b clinical trial of VX-765 to evaluate longer dosing of VX-765 in patients with treatment-resistant epilepsy.

INFLUENZA

VX-787 is an investigational drug candidate intended for the treatment of influenza A, which is typically the predominate strain of influenza and includes H1 (pandemic) and H5 (avian) influenza strains. VX-787 focuses to treat influenza A in a way that is distinct from neuraminidase inhibitors. The Company has begun Phase 1 clinical development of VX-787.

The Company competes with Abbott Laboratories, Achillion Pharmaceuticals, Inc., Boehringer Ingelheim, Bristol-Myers Squibb Company, Gilead Sciences, Inc., Hoffman-La Roche, Idenix Pharmaceuticals, Inc. and Janssen.