Vertex Pharmaceuticals, Inc., a biotechnology company, engages in the discovery, development, and commercialization of small molecule drugs for the treatment of serious diseases. The company's lead drug candidate is telaprevir, an oral hepatitis C protease inhibitor, and a new class of antiviral treatments in development targeting HCV infection, a serious and life-threatening disease. The company is conducting three major Phase 2b clinical trials of telaprevir.
In June 2006, the company entered into a collaboration agreement with Janssen Pharmaceutica, N.V., or Janssen, a Johnson & Johnson company, relating to telaprevir. Under its agreement with Janssen, the company retained exclusive commercial rights to telaprevir in North America and would lead the clinical development program. Janssen would be responsible for the commercialization of telaprevir, including the manufacture of its own commercial supply of telaprevir for the Janssen territories, which include the territories outside of North America and the Far East.
In addition to telaprevir, the company focuses on: VX-702, a p38 MAP kinase inhibitor, which the company is investigating for the treatment of rheumatoid arthritis; VX-770, a cystic fibrosis transmembrane regulator, or CFTR, potentiator, which the company is investigating for the treatment of CF; and VX-883, a Vertex-discovered dual-mechanism investigational antibiotic, which is in preclinical development for the treatment of patients with bacterial infection.
The company's pipeline also includes various drug candidates that are being developed by its collaborators. The most advanced of these drug candidates is MK-0457 (VX-680), an Aurora kinase inhibitor that is being developed by Merck & Co., Inc. for the treatment of cancer.
Products and Clinical Development Programs
VX-702: The company is conducting a 12-week, 120-patient Phase 2a clinical trial in patients with RA to evaluate the safety, tolerability and anti-inflammatory effects of VX-702 on a background of methotrexate. The company holds worldwide development and commercialization rights to VX-702, except for Japan and certain Far East countries, where it is collaborating with Kissei Pharmaceutical Co., Ltd. In 2006, Kissei completed a Phase 1 clinical trial of VX-702 in Japan in RA.
VX-770: VX-770 is a small molecule drug candidate designed to potentiate the gating activity of the CFTR protein, a chloride ion transporter on the cell surface that is functionally defective in patients with cystic fibrosis. VX-770 was discovered by the company in its ongoing research collaboration with Cystic Fibrosis Foundation Therapeutics Incorporated, or CFFT, and with the support and participation of the Cystic Fibrosis Foundation. The company holds worldwide development and commercialization rights to VX-770. In 2006, the company completed a Phase 1 clinical trial of VX-770 in 63 individuals, including healthy volunteers and patients with CF.
VX-883 (gyrase inhibition for the treatment of bacterial infection): VX-883 is a Vertex-discovered dual-mechanism investigational antibiotic in preclinical development that targets both DNA gyrase and topoisomerase IV. DNA gyrase and topoisomerase IV are enzymes that are essential to bacteria during the replication process. The company holds worldwide development and commercial rights to VX-883.
Collaborator-Led Programs
Lexiva/Telzir: The company's HIV PI, fosamprenavir calcium, fosamprenavir calcium, is marketed under the name Lexiva in the United States and under the name Telzir in the European Union. Lexiva/Telzir was co-discovered by the company and GlaxoSmithKline and was developed by GlaxoSmithKline pursuant to the company's collaboration with them. GlaxoSmithKline has worldwide marketing rights for Lexiva/Telzir, and the company has the right to conduct certain promotional and educational activities for Lexiva/Telzir in the United States and the European Union. The company also has the right to supply bulk drug substance to GlaxoSmithKline.
Lexiva/Telzir is a prodrug of amprenavir, which also was discovered and developed under the company's collaboration with GlaxoSmithKline and marketed under the name Agenerase. Lexiva/Telzir has replaced Agenerase in worldwide markets. Lexiva/Telzir is approved for sale in approximately 45 countries worldwide, including the United States, France, Germany, Spain, Italy, the United Kingdom and Canada.
MK-0457 (VX-680) and MK-6592 (VX-667): Aurora kinase inhibition for the treatment of cancer (Merck & Co., Inc.)
The company is collaborating with Merck in the area of Aurora kinase inhibitors, including MK-0457 (VX-680), MK-6592 (VX-667) and additional potential follow-on compounds. MK-0457 (VX-680) is a potent inhibitor of Aurora kinases and of flt-3 kinase, a receptor tyrosine kinase that is known to be inappropriately activated in different types of leukemia. As part of the collaboration, the company conducted a joint research program with Merck to characterize MK-0457 (VX-680) activity across a range of cancer types and to identify additional drug candidates targeting the Aurora kinases. Merck holds worldwide development and commercialization rights to MK-0457 (VX-680), MK-6592 (VX-667) and certain additional compounds identified during the research program.
MK-0457 (VX-680)
In December 2006, Merck initiated a pivotal Phase 2 clinical trial of MK-0457 (VX-680) in patients with treatment-resistant chronic myelogenous leukemia, or CML, and Philadelphia chromosome-positive acute lymphocytic leukemia, or Ph+ ALL, containing the T315I BCR-ABL mutation based on encouraging results from a Phase 1 clinical trial of MK-0457 (VX-680).
Merck also is conducting a Phase 2a clinical trial of MK-0457 (VX-680) in patients with lung cancer, a Phase 1 clinical trial of MK-0457 (VX-680) in patients with recurrent or non-responsive solid tumors and a Phase 1 clinical trial of MK-0457 (VX-680) in patients with solid tumor and/or colorectal cancer.
MK-6592 (VX-667)
In 2005, Merck selected MK-6592 (VX-667), a second Aurora kinase inhibitor, for preclinical development. In December 2006, Merck initiated a Phase 1 clinical trial of MK-6592 (VX-667) in oncology patients with advanced solid tumors.
AVN-944 (VX-944): IMPDH inhibition for the treatment of cancer (Avalon Pharmaceuticals, Inc.)
The company's collaborator Avalon Pharmaceuticals is developing AVN-944 (VX-944), an IMPDH inhibitor, for the treatment of advanced hematological malignancies, such as leukemia, lymphoma or myeloma. Inosine 5-monophosphate dehydrogenase, or IMPDH, is an enzyme thought to be critical for the synthesis of quanosine triphosphate, a molecule required for DNA synthesis and cellular signalling. AVN-944 (VX-944) also significantly prolonged survival in a model of aggressive mouse leukemia. In a single-dose, dose-escalation Phase 1 clinical trial of AVN-944 (VX-944) in healthy volunteers, data indicated that AVN-944 (VX-944) was orally bioavailable. Avalon holds worldwide development and commercialization rights to AVN-944 (VX-944).
VX-409: Selective sodium channel modulation for the treatment of pain (GlaxoSmithKline plc)
GlaxoSmithKline is engaged in the development of VX-409, an oral, subtype-selective sodium channel modulator, and certain additional back-up compounds as potential drug candidates for the treatment of pain. GlaxoSmithKline holds worldwide development and commercialization rights to VX-409 and certain backup compounds.
Other Programs
VX-692 (gyrase inhibition for the treatment of bacterial infection)
VX-692 is a Vertex-discovered investigational antibiotic, which targets both DNA gyrase and topoisomerase IV and is active, in vitro, against Gram-positive and Gram-negative bacterial pathogens. The company is evaluating VX-692 in preclinical development. It holds worldwide development and commercial rights to VX-692.
VX-166 (caspase inhibition for the treatment of liver disease and sepsis)
VX-166 is a Vertex-discovered inhibitor of multiple caspases. The company holds worldwide rights to VX-166.
VX-765 (ICE inhibition for the treatment of inflammatory diseases)
The company discovered and has completed certain development activities with respect to two interleukin-1 converting enzyme, or ICE, inhibitors for the treatment of inflammatory diseases, VX-765 and pralnacasan. ICE is an enzyme that controls the release of active IL-1 (one of two forms of IL-1) and IL-18, which have been correlated with disease states in a number of acute and chronic inflammatory diseases.
The company's first generation ICE inhibitor, pralnacasan, was developed in collaboration with Sanofi-Aventis (then Aventis). Phase 2 clinical trials of pralnacasan conducted by Aventis suggested that treatment with pralnacasan produced positive anti-inflammatory effects in patients with RA and led to dose-dependent suppression of the production of IL-1.
Merimepodib (VX-497) (IMPDH inhibition for the treatment of autoimmune diseases)
The company is completing the data analysis phase of the company's Phase 2b clinical trial of merimepodib, an oral, small molecule inhibitor of inosine 5-monophosphate dehydrogenase, or IMPDH, for the treatment of HCV infection. In the HCV field, the company is focusing its efforts on the development of direct antivirals such as telaprevir.
Collaboration
Janssen Pharmaceutica, N.V.: In June 2006, the company entered into a license, development, manufacturing, and commercialization agreement with Janssen. Under the collaboration agreement, the company would collaborate with Janssen to develop and commercialize telaprevir.
Mitsubishi Pharma Corporation: In 2004, the company entered into a license, development and commercialization agreement with Mitsubishi for the development and commercialization of telaprevir, in Japan and certain other Far East countries. Under the terms of the agreement, Mitsubishi has the right to develop and commercialize telaprevir in its territory. Mitsubishi is designing a Phase 2 clinical program of telaprevir in the Far East.
Kissei Pharmaceutical Co., Ltd.: The company is working with Kissei to develop and commercialize VX-702, which was discovered during the company's p38 MAP kinase research collaboration. Kissei has exclusive rights to develop and commercialize VX-702 in Japan and certain Far East countries, and co-exclusive rights in China, Taiwan and South Korea. The company retains exclusive marketing rights outside the Far East.
Avalon Pharmaceuticals, Inc.: In 2005, the company entered into a license agreement with Avalon for the development and commercialization of the IMPDH inhibitor AVN-944 (VX-944) for the treatment of cancer. Under the agreement, Avalon has the exclusive worldwide right and responsibility to develop and commercialize AVN-944 (VX-944) for the treatment of cancer.
Competition
The company’s competitors include Schering-Plough Corporation, InterMune, Inc. in collaboration with Roche, and Gilead Sciences in collaboration with Achillion Pharmaceuticals. In addition, the company competes with Idenix Pharmaceuticals, Inc. in collaboration with Novartis, ViroPharma Incorporated in collaboration with Wyeth, and Roche. In addition to Vertex, other companies with open or planned Phase 2 p38 MAP programs in RA include GlaxoSmithKline, Pfizer, and Roche. The company also competes with PTC Therapeutics, Inc. and Altus Pharmaceuticals, Inc. In the field of HIV protease inhibition, the company competes with Abbott Laboratories, Bristol-Myers Squibb, Gilead Sciences, Inc., Johnson & Johnson and Pfizer Inc.
History
Vertex Pharmaceuticals, Inc. was founded in 1989.
