We are collaborating with Tibotec, a Janssen (Johnson & Johnson) company headquartered in Mechelen, Belgium, to develop and commercialize our investigational hepatitis C virus (HCV) protease inhibitor telaprevir (VX-950). Under the agreement, Janssen will have exclusive rights in Europe, South America, the Middle East, Africa and Australia. Vertex received an upfront payment of $165 million and has received $100 million in milestones based on successful development and launch of telaprevir in the Janssen territories. Vertex and Janssen also share development costs for telaprevir. The agreement also includes a royalty on product sales in Europe and other territories outside of North America and the Far East. Vertex retains commercial rights in North America and continues to lead the global development plan for telaprevir.

Vertex initiated its CF research program in 1998 as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), a non-profit, donor-supported drug discovery and development affiliate of the CF Foundation. Vertex and CFFT expanded the agreement in 2000 and again in 2004, and in 2006, entered into a collaboration for the accelerated development of VX-770, a novel, oral drug candidate for the treatment of cystic fibrosis (CF). In addition, Vertex and CFFT entered into an expanded research collaboration in January 2006 to discover novel compounds known as correctors, which may work by increasing the number of CFTR channels on the cell surface.

VX-770, an investigational CFTR potentiator, is intended to increase chloride ion transport through the defective Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. VX-770 was advanced into preclinical development based on successful collaborative research with CFFT that incorporated capabilities and proprietary ion channel research from Vertex's San Diego research site.

Based on positive results announced in 2008 from a Phase 2 trial of VX-770 in patients with CF who carry the G551D mutation in CFTR, in May 2009 Vertex initiated the Phase 3 ENDEAVOR registration program designed to support registration of VX-770 in patients with the G551D mutation. The G551D mutation is present in approximately 4% of CF patients.

The VX-770 registration program consists of three clinical trials, including a primary 48-week Phase 3 trial of patients aged 12 years and older who carry the G551D mutation on at least one allele. Two additional trials will evaluate VX-770 in patients aged 6 to 11 years with the G551D mutation on at least one allele and in patients homozygous for the F508del mutation, respectively. Click to learn more about VX-770.

VX-809, an investigational oral CFTR corrector compound, is Vertex's second drug candidate for the treatment of CF. Corrector compounds such as VX-809 are designed to increase the amount of F508del CFTR on the surface of cells lining the airway, which may result in an increase in chloride transport across the cell surface in patients with the F508del CFTR mutation. Vertex recently completed a Phase 2a clinical trial for VX-809. The trial was designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial was also designed to evaluate the potential effect of VX-809 on measures of CFTR function. The trial enrolled 89 patients homozygous for the F508del mutation in the CFTR gene, the most common mutation in CF patients. Based on the Phase 2a data, Vertex is now focused on generating key data that may support the initiation of a combination trial of VX-809 with the CFTR potentiator VX-770 in patients with the F508del mutation.

In June 2004, Vertex signed an agreement with Osaka-based Mitsubishi Tanabe Pharma Corporation to develop and commercialize telaprevir (VX-950), Vertex’s investigational oral protease inhibitor for the treatment of hepatitis C virus (HCV) infection, in Japan and certain Far East countries. The companies amended the agreement in July 2009. Under the amended agreement, Mitsubishi Tanabe received rights in its territory for telaprevir-based combination therapy with interferon and ribavirin, as well as rights to manufacture telaprevir for sale in its territory. Vertex expects to receive a $105 million payment upon signing and has the potential to receive additional bonus milestone payments in lieu of royalties, that if realized would range between $15 million and $65 million. Mitsubishi Tanabe is currently conducting Phase 3 registration studies with telaprevir in combination with pegylated interferon and ribavirin in Japan in approximately 300 treatment-naïve and treatment-failure genotype 1 hepatitis C patients.

Janssen Pharmaceutica, a Johnson & Johnson Company, has exclusive rights to develop and commercialize telaprevir in Europe, South America, the Middle East, Africa and Australia. Vertex retains exclusive commercial rights to telaprevir in North America and continues to lead the global development for telaprevir.