Vertex Pharmaceuticals is a biotechnology company with activities spanning the length of the pharmaceutical product pipeline, from target identification through to clinical trials and marketing. Most of its activity has been in collaboration with much larger pharmaceutical firms, though some of its recent work has been done independently. Vertex was founded in 1989 by Joshua Boger, credited as being one of the main reasons for the company's early success due to his strong fund-raising and company cash flow management. Vertex was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. Vertex went about understanding a disease and then tried to develop a process to cure it. In 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer. Its capital investments include a headquarters in Cambridge, Massachusetts, and two research facilities, in San Diego, California, and Oxford, England. The company's beginnings were profiled by Barry Werth in the 1994 book "The Billion-Dollar Molecule".
In November 2010, Vertex Pharmaceuticals completed its first NDA under its own name for the drug Telaprevir, a novel oral treatment of Hepatitis C. Development and commercialization of Telaprevir is shared with Johnson and Johnson for European distribution and Mitsubishi for the far east. Telaprevir, a protease inhibitor, is the first in this class to reach NDA status, ahead of Merk's boceprevir.
Vertex Pharmaceuticals Incorporated (Vertex), incorporated in 1989, is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of diseases. The Company is engaged in phase-I clinical trials and/or nonclinical activities with respect to a range of additional drug candidates, including compounds intended for the treatment of hepatitis C virus (HCV) infection, cystic fibrosis (CF) and influenza. In November 2010, the Company submitted a new drug application (NDA), requesting approval to market telaprevir in the United States for the treatment of patients with chronic HCV infection.
Telaprevir (VX-950)
Telaprevir, the Company’s lead drug candidate, is an orally-administered hepatitis C protease inhibitor. Telaprevir works by inhibiting the NS3-4A serine protease, an enzyme necessary for HCV replication. It has collaboration agreements with Janssen Pharmaceutica, N.V. (Janssen), a Johnson & Johnson company, and Mitsubishi Tanabe Pharma Corporation (Mitsubishi Tanabe), relating to the development and commercialization of telaprevir. On November 23, 2010, the Food and Drug Administration (FDA) received its NDA for telaprevir. In January 2011, the telaprevir NDA was accepted for filing by the FDA. Its registration program for telaprevir include the REALIZE clinical trial, a phase-III clinical trial in patients infected with genotype-I HCV who failed to achieve an sustained viral response (SVR) with prior interferon-based treatment, and two phase-III clinical trials, ADVANCE and ILLUMINATE for the treatment of patients infected with genotype-I HCV. REALIZE was a pivotal three-arm double-blinded placebo-controlled clinical trial of telaprevir-based treatment regimens. REALIZE included patient groups, such as null responders, partial responders and relapsers.
ADVANCE was a pivotal three-arm double-blinded placebo-controlled clinical trial that enrolled patients with genotype-I HCV infection. ADVANCE had two telaprevir-based treatment arms, one in which patients received 12 weeks of telaprevir-based triple combination therapy and one in which patients received 8 weeks of telaprevir-based triple combination therapy. ILLUMINATE was a supplemental phase-III clinical trial that included evaluation of 24-week and 48-week total treatment durations for patients infected with genotype-I HCV who achieved an extended rapid viral response (eRVR) in response to a telaprevir-based treatment regimen. This clinical trial was a randomized, open-label trial that enrolled 540 patients. ILLUMINATE was designed to supplement SVR data obtained from ADVANCE by evaluating the benefits and risks, for patients achieving an eRVR, of extending total treatment duration from 24 to 48 weeks.
VX-222
HCV polymerase inhibitors, including the Company’s HCV polymerase inhibitor VX-222, are direct-acting antiviral agents that inhibit the replication of HCV, but through a mechanism distinct from HCV protease inhibitors, such as telaprevir. The Company is conducting a phase-IIa clinical trial in patients with genotype-I HCV designed to evaluate response-guided combination treatment regimens of telaprevir and VX-222. Dosing in this clinical trial began in August 2010. This trial included two treatment arms of patients receiving two-drug treatment regimens consisting of telaprevir and VX-222 and two treatment arms of patients receiving four-drug treatment regimens consisting of telaprevir, VX-222, pegylated-interferon (peg-IFN) and ribavirin (RBV). During the year ended December 31, 2010, it discontinued both of the two-drug treatment arms. The remaining two original treatment arms, with four-drug treatment regimens, are continuing without modification. In addition to the clinical trial evaluating VX-222 in combination with telaprevir, it is conducting a phase-IIa clinical trial to evaluate multiple doses of VX-222 in combination with only peg-IFN and RBV. This Phase 2a clinical trial evaluate the safety, tolerability and antiviral activity of two dose levels of VX-222 (400 milligrams and 750 milligrams) in a total of 50 patients with genotype-I HCV infection. Patients in the clinical trial are receiving VX-222 in combination with peg-IFN and RBV for 12 weeks, followed by peg-IFN and RBV for 36 weeks.
VX-770
VX-770 is an investigational oral drug candidate, which increases chloride ion transport across cell membranes by partially restoring the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein on the surface of the cells. The VX-770 registration program focuses on patients with the G551D mutation. The registration program consists of three clinical trials designed to evaluate the safety and efficacy of VX-770.
The primary clinical trial, which is referred to as STRIVE, is a phase-III clinical trial of VX-770 that enrolled approximately 170 patients 12 years of age and older with the G551D mutation on at least one of the patient's two CFTR genes, or alleles. In this randomized, placebo-controlled, double-blind, parallel-group clinical trial, patients received either VX-770 or placebo for 48 weeks. The second clinical trial, which is referred to as ENVISION, is a phase-III clinical trial of VX-770 in patients between 6 to 11 years of age with the G551D mutation on at least one allele. ENVISION is a two-part, randomized, placebo-controlled, double-blind, parallel-group clinical trial of VX-770. It has completed part-I of ENVISION, which evaluated single-dose pharmacokinetics to determine the dose selection for children ages 6 to 11. Part-II of the ENVISION trial enrolled approximately 50 patients who are receiving either VX-770 or placebo for 48 weeks.
VX-809
The Company is evaluating VX-809, an oral corrector compound. During 2010, the Company completed a phase-IIa, 28-day clinical trial of VX-809 as a single agent in 89 patients 18 years of age or older with the F508del mutation on both alleles. This phase-IIa clinical trial was a randomized, double-blind, placebo-controlled, multiple dose clinical trial. Patients received one of four doses of VX-809, or placebo, in addition to standard therapies for 28 days. The trial was designed to evaluate the safety and tolerability of VX-809. Multiple secondary endpoints were utilized to determine any effect of VX-809 on CFTR protein function and lung function.
VX-509
VX-509 is designed to inhibit Janus kinase 3 (JAK3), which is involved in signaling pathways that control the survival and proliferation of a type of white blood cell referred to as a lymphocyte. During 2010, it initiated a phase-IIa clinical trial of VX-509 in patients with moderate-to-severe rheumatoid arthritis (RA). The primary endpoints of this clinical trial are to evaluate safety and to measure clinical signs and symptoms of RA in patients after 12 weeks of treatment.
VX-765
VX-765 is designed to inhibit Caspase, which is an enzyme that controls the generation of two cytokines, IL-1b and IL-18. VX-765 has been shown to inhibit acute seizures in preclinical models. In addition, VX-765 has shown activity in preclinical models of chronic epilepsy. VX-765 had been dosed in over 100 patients in phase-I and phase-IIa clinical trials relating to other diseases, including a 28-day phase-IIa clinical trial in patients with psoriasis. It has completed the treatment phase of a phase-IIa clinical trial of VX-765 that enrolled approximately 75 patients with treatment-resistant epilepsy. The double-blind, randomized, placebo-controlled clinical trial was designed to evaluate the safety, tolerability and clinical activity of VX-765. The Company is analyzing data from this trial.
The Company competes with Merck & Co., Inc., Abbot Laboratories, Bristol-Myers Squibb Company, Gilead Sciences, Inc., Intermune, Inc., Pharmasset, Inc. and Hoffman-La Roche, PTC Therapeutics, Inc. and Genzyme Corporation.
OVERALL
Beta: 0.59
Market Cap (Mil.): $11,840.76
Shares Outstanding (Mil.): 206.00
Annual Dividend: --
Yield (%): --
FINANCIALS
VRTX.O Industry Sector
P/E (TTM): -- 31.20 43.63
EPS (TTM): -8.22 -- --
ROI: -57.10 -0.95 6.34
ROE: -110.75 -0.83 7.67
Name Age Since Current Position
Matthew Emmens 59 2009 President, Chief Executive Officer, Chairman of the Board
Ian Smith 45 2008 Chief Financial Officer, Executive Vice President
Peter Mueller 54 2009 Executive Vice President - Global Research and Development, Chief Scientific Officer
Nancy Wysenski 53 2009 Chief Commercial Officer, Executive Vice President
Kenneth Boger 64 2001 Senior Vice President, General Counsel
Lisa Kelly-Croswell 44 2007 Senior Vice President - Human Resources
Amit Sachdev 43 2007 Senior Vice President - Corporate Affairs and Public Policy and Commercial Business Lead, Canada
Christiana Stamoulis 40 2010 Senior Vice President - Corporate Strategy and Business Development
Paul Silva 44 2008 Vice President, Corporate Controller
Jeffrey Leiden 55 2010 Lead Independent Director
Joshua Boger 59 2009 Director
Stuart Collinson 51 2001 Independent Director
Bruce Sachs 51 1998 Independent Director
Elaine Ullian 63 1997 Independent Director
Eugene Cordes 74 2005 Independent Director
Dennis Winger 64 2009 Independent Director
Wayne Riley 51 2010 Independent Director
COMPANY ADDRESS
Vertex Pharmaceuticals Inc
130 Waverly Street
Cambridge MA 02139
In November 2010, Vertex Pharmaceuticals completed its first NDA under its own name for the drug Telaprevir, a novel oral treatment of Hepatitis C. Development and commercialization of Telaprevir is shared with Johnson and Johnson for European distribution and Mitsubishi for the far east. Telaprevir, a protease inhibitor, is the first in this class to reach NDA status, ahead of Merk's boceprevir.
Vertex Pharmaceuticals Incorporated (Vertex), incorporated in 1989, is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of diseases. The Company is engaged in phase-I clinical trials and/or nonclinical activities with respect to a range of additional drug candidates, including compounds intended for the treatment of hepatitis C virus (HCV) infection, cystic fibrosis (CF) and influenza. In November 2010, the Company submitted a new drug application (NDA), requesting approval to market telaprevir in the United States for the treatment of patients with chronic HCV infection.
Telaprevir (VX-950)
Telaprevir, the Company’s lead drug candidate, is an orally-administered hepatitis C protease inhibitor. Telaprevir works by inhibiting the NS3-4A serine protease, an enzyme necessary for HCV replication. It has collaboration agreements with Janssen Pharmaceutica, N.V. (Janssen), a Johnson & Johnson company, and Mitsubishi Tanabe Pharma Corporation (Mitsubishi Tanabe), relating to the development and commercialization of telaprevir. On November 23, 2010, the Food and Drug Administration (FDA) received its NDA for telaprevir. In January 2011, the telaprevir NDA was accepted for filing by the FDA. Its registration program for telaprevir include the REALIZE clinical trial, a phase-III clinical trial in patients infected with genotype-I HCV who failed to achieve an sustained viral response (SVR) with prior interferon-based treatment, and two phase-III clinical trials, ADVANCE and ILLUMINATE for the treatment of patients infected with genotype-I HCV. REALIZE was a pivotal three-arm double-blinded placebo-controlled clinical trial of telaprevir-based treatment regimens. REALIZE included patient groups, such as null responders, partial responders and relapsers.
ADVANCE was a pivotal three-arm double-blinded placebo-controlled clinical trial that enrolled patients with genotype-I HCV infection. ADVANCE had two telaprevir-based treatment arms, one in which patients received 12 weeks of telaprevir-based triple combination therapy and one in which patients received 8 weeks of telaprevir-based triple combination therapy. ILLUMINATE was a supplemental phase-III clinical trial that included evaluation of 24-week and 48-week total treatment durations for patients infected with genotype-I HCV who achieved an extended rapid viral response (eRVR) in response to a telaprevir-based treatment regimen. This clinical trial was a randomized, open-label trial that enrolled 540 patients. ILLUMINATE was designed to supplement SVR data obtained from ADVANCE by evaluating the benefits and risks, for patients achieving an eRVR, of extending total treatment duration from 24 to 48 weeks.
VX-222
HCV polymerase inhibitors, including the Company’s HCV polymerase inhibitor VX-222, are direct-acting antiviral agents that inhibit the replication of HCV, but through a mechanism distinct from HCV protease inhibitors, such as telaprevir. The Company is conducting a phase-IIa clinical trial in patients with genotype-I HCV designed to evaluate response-guided combination treatment regimens of telaprevir and VX-222. Dosing in this clinical trial began in August 2010. This trial included two treatment arms of patients receiving two-drug treatment regimens consisting of telaprevir and VX-222 and two treatment arms of patients receiving four-drug treatment regimens consisting of telaprevir, VX-222, pegylated-interferon (peg-IFN) and ribavirin (RBV). During the year ended December 31, 2010, it discontinued both of the two-drug treatment arms. The remaining two original treatment arms, with four-drug treatment regimens, are continuing without modification. In addition to the clinical trial evaluating VX-222 in combination with telaprevir, it is conducting a phase-IIa clinical trial to evaluate multiple doses of VX-222 in combination with only peg-IFN and RBV. This Phase 2a clinical trial evaluate the safety, tolerability and antiviral activity of two dose levels of VX-222 (400 milligrams and 750 milligrams) in a total of 50 patients with genotype-I HCV infection. Patients in the clinical trial are receiving VX-222 in combination with peg-IFN and RBV for 12 weeks, followed by peg-IFN and RBV for 36 weeks.
VX-770
VX-770 is an investigational oral drug candidate, which increases chloride ion transport across cell membranes by partially restoring the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein on the surface of the cells. The VX-770 registration program focuses on patients with the G551D mutation. The registration program consists of three clinical trials designed to evaluate the safety and efficacy of VX-770.
The primary clinical trial, which is referred to as STRIVE, is a phase-III clinical trial of VX-770 that enrolled approximately 170 patients 12 years of age and older with the G551D mutation on at least one of the patient's two CFTR genes, or alleles. In this randomized, placebo-controlled, double-blind, parallel-group clinical trial, patients received either VX-770 or placebo for 48 weeks. The second clinical trial, which is referred to as ENVISION, is a phase-III clinical trial of VX-770 in patients between 6 to 11 years of age with the G551D mutation on at least one allele. ENVISION is a two-part, randomized, placebo-controlled, double-blind, parallel-group clinical trial of VX-770. It has completed part-I of ENVISION, which evaluated single-dose pharmacokinetics to determine the dose selection for children ages 6 to 11. Part-II of the ENVISION trial enrolled approximately 50 patients who are receiving either VX-770 or placebo for 48 weeks.
VX-809
The Company is evaluating VX-809, an oral corrector compound. During 2010, the Company completed a phase-IIa, 28-day clinical trial of VX-809 as a single agent in 89 patients 18 years of age or older with the F508del mutation on both alleles. This phase-IIa clinical trial was a randomized, double-blind, placebo-controlled, multiple dose clinical trial. Patients received one of four doses of VX-809, or placebo, in addition to standard therapies for 28 days. The trial was designed to evaluate the safety and tolerability of VX-809. Multiple secondary endpoints were utilized to determine any effect of VX-809 on CFTR protein function and lung function.
VX-509
VX-509 is designed to inhibit Janus kinase 3 (JAK3), which is involved in signaling pathways that control the survival and proliferation of a type of white blood cell referred to as a lymphocyte. During 2010, it initiated a phase-IIa clinical trial of VX-509 in patients with moderate-to-severe rheumatoid arthritis (RA). The primary endpoints of this clinical trial are to evaluate safety and to measure clinical signs and symptoms of RA in patients after 12 weeks of treatment.
VX-765
VX-765 is designed to inhibit Caspase, which is an enzyme that controls the generation of two cytokines, IL-1b and IL-18. VX-765 has been shown to inhibit acute seizures in preclinical models. In addition, VX-765 has shown activity in preclinical models of chronic epilepsy. VX-765 had been dosed in over 100 patients in phase-I and phase-IIa clinical trials relating to other diseases, including a 28-day phase-IIa clinical trial in patients with psoriasis. It has completed the treatment phase of a phase-IIa clinical trial of VX-765 that enrolled approximately 75 patients with treatment-resistant epilepsy. The double-blind, randomized, placebo-controlled clinical trial was designed to evaluate the safety, tolerability and clinical activity of VX-765. The Company is analyzing data from this trial.
The Company competes with Merck & Co., Inc., Abbot Laboratories, Bristol-Myers Squibb Company, Gilead Sciences, Inc., Intermune, Inc., Pharmasset, Inc. and Hoffman-La Roche, PTC Therapeutics, Inc. and Genzyme Corporation.
OVERALL
Beta: 0.59
Market Cap (Mil.): $11,840.76
Shares Outstanding (Mil.): 206.00
Annual Dividend: --
Yield (%): --
FINANCIALS
VRTX.O Industry Sector
P/E (TTM): -- 31.20 43.63
EPS (TTM): -8.22 -- --
ROI: -57.10 -0.95 6.34
ROE: -110.75 -0.83 7.67
Name Age Since Current Position
Matthew Emmens 59 2009 President, Chief Executive Officer, Chairman of the Board
Ian Smith 45 2008 Chief Financial Officer, Executive Vice President
Peter Mueller 54 2009 Executive Vice President - Global Research and Development, Chief Scientific Officer
Nancy Wysenski 53 2009 Chief Commercial Officer, Executive Vice President
Kenneth Boger 64 2001 Senior Vice President, General Counsel
Lisa Kelly-Croswell 44 2007 Senior Vice President - Human Resources
Amit Sachdev 43 2007 Senior Vice President - Corporate Affairs and Public Policy and Commercial Business Lead, Canada
Christiana Stamoulis 40 2010 Senior Vice President - Corporate Strategy and Business Development
Paul Silva 44 2008 Vice President, Corporate Controller
Jeffrey Leiden 55 2010 Lead Independent Director
Joshua Boger 59 2009 Director
Stuart Collinson 51 2001 Independent Director
Bruce Sachs 51 1998 Independent Director
Elaine Ullian 63 1997 Independent Director
Eugene Cordes 74 2005 Independent Director
Dennis Winger 64 2009 Independent Director
Wayne Riley 51 2010 Independent Director
COMPANY ADDRESS
Vertex Pharmaceuticals Inc
130 Waverly Street
Cambridge MA 02139
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