Businessweek

Vertex Pharmaceuticals Incorporated engages in the discovery, development, manufacture, and commercialization of small molecule drugs for the treatment of serious diseases. Products INCIVEK: INCIVEK (telaprevir) is an orally-administered hepatitis C virus (HCV) protease inhibitor for adults with genotype 1 HCV infection that is prescribed in combination with pegylated-interferon (peg-IFN), and ribavirin (RBV). INCIVEK is approved by the United States Food and Drug Administration (FDA) and Health Canada. In 2011, the company’s collaborators, Janssen Pharmaceutica, N.V. (Janssen) and Mitsubishi Tanabe Pharma Corporation (Mitsubishi Tanabe), obtained marketing approval for telaprevir from the European Commission and the Japanese Ministry of Health, Labor and Welfare, respectively. Janssen markets telaprevir under the brand name INCIVO in Europe. Mitsubishi Tanabe markets telaprevir under the brand name TELAVIC in Japan. KALYDECO: KALYDECO (ivacaftor) is an orally-administered cystic fibrosis (CF) transmembrane conductance regulator (CFTR) potentiator that is approved in the United States, Canada, and the European Union for the treatment of patients six years of age and older with CF who have at least one copy of the G551D mutation in the CFTR gene. Drug Candidates CF Ivacaftor (monotherapy): Ivacaftor monotherapy is approved (as KALYDECO) as a treatment for patients six years of age and older with CF who have the G551D mutation on at least one allele. The company is also developing a pediatric formulation of ivacaftor that could be used to treat children two to five years of age. It is conducting three Phase 3 label-expansion clinical trials and a Phase 2 clinical trial of ivacaftor monotherapy. VX-809 in Combination with Ivacaftor: In February 2013, the company initiated an international pivotal Phase 3 clinical program to evaluate combinations of VX-809 and ivacaftor in patients with CF who are homozygous (a copy on both genes) with the F508del mutation in their CFTR gene. The company plans to conduct two 24-week Phase 3 clinical trials designed to support approval of the combination of VX-809 and ivacaftor for patients with CF 12 years of age and older. VX-661: The company is also conducting a Phase 2 clinical trial of VX-661, a second CFTR corrector compound. In this clinical trial, it is evaluating VX-661 as both a monotherapy and in combination with ivacaftor in patients with CF who are homozygous for the F508del mutation. HCV Infection VX-135, an HCV nucleotide analogue, is designed to inhibit the replication of HCV by inhibiting the HCV NS5B polymerase enzyme through mechanisms of action distinct from non-nucleoside HCV polymerase inhibitors, such as VX-222. In July 2012, the company has completed a Phase 1 clinical trial that evaluated the safety and tolerability of single ascending doses of ALS-2200 (formulated as VX-135) in healthy volunteers and the safety, tolerability and effects on viral kinetics of multiple ascending doses of ALS-2200 in treatment-naïve patients with genotype 1 HCV infection. The company is conducting Phase 2 clinical trials to evaluate VX-135 in combination with RBV. VX-222, a non-nucleoside HCV polymerase inhibitor, is designed to inhibit the replication of HCV by inhibiting the HCV NS5B polymerase. VX-222 has been evaluated in a Phase 2 clinical trial in combination with telaprevir and RBV in treatment-naïve patients with genotype 1 HCV infection. Autoimmune Diseases (Rheumatoid Arthritis) VX-509: 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 autoimmune disease pathology. Influenza VX-787: 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 aims to treat influenza A through a mechanism that is different from neuraminidase inhibitors. The company has received final data from a Phase 2 clinical trial of VX-787 that enrolled healthy volunteers who were infected with live influenza virus. Collaborations Alios BioPharma, Inc. (Alios): In 2011, the company entered into a license and collaboration agreement with Alios, a privately-held biotechnology company. Pursuant to the agreement, it is collaborating on the research, development, and commercialization of VX-135 (ALS-2200), an HCV nucleotide analogue discovered by Alios. Significant Events In April 2013, the company has entered into an agreement with Bristol-Myers Squibb Company to conduct Phase 2 studies of once-daily all-oral treatment regimens containing the company’s nucleotide analogue HCV polymerase inhibitor VX-135 and Bristol-Myers Squibb's NS5A replication complex inhibitor daclatasvir for the treatment of hepatitis C. Government Regulation In the United States, the FDA regulates drugs under the Federal Food, Drug and Cosmetic Act and implementing regulations. It is also subject to regulation in the United States under the Occupational Safety and Health Act, the Environmental Protection Act, the Toxic Substances Control Act, the Resource Conservation and Recovery Act, and other federal, state, local and foreign statutes and regulations. History Vertex Pharmaceuticals Incorporated was founded in 1989. The company was incorporated in Massachusetts in 1989.