Vertex is focused on discovering and developing small molecule drugs for diseases that include hepatitis C, cystic fibrosis, and inflammatory and autoimmune disorders. Its strategy is to retain US development and marketing rights to product candidates for hepatitis C and cystic fibrosis, and to partner candidates for other disease areas. Its lead product is Telaprevir, a protease inhibitor for hepatitis C. Data from two large Phase II trials, PROVE-1 and PROVE-2, showed sustained viral response (SVR) rates of 61% and 69%, respectively, following twelve weeks of triple therapy plus twelve weeks of standard therapy. These results compare with SVR rates of 40% to 50% achieved with the current standard of care. The shorter treatment time and better cure rate with Telaprevir have raised the hopes of many hepatitis C patients. In addition, Telaprevir showed highly encouraging data in patients who have failed standard therapy, offering the chance for a cure for this large, underserved patient population. Results from Phase III trials released in 2010 confirmed this strong efficacy, with 75% of treatmentnaïve patients experiencing SVR and 58% able to stop therapy after just 24 weeks, and with 65% of treatment-refractory patients experiencing SVR with 48 weeks of therapy. In addition, the safety profile was consistent with earlier data. We expect these positive results to lead to launch of Telaprevir in the US and Europe in 2011. In view of the high market demand, we expect the drug to achieve a rapid launch and significant market success. While there are a number of competing drugs in development, data presented in 2010 continue to suggest that Telaprevir’s profile is very solid.

In addition, the 2009 acquisition of ViroChem gave Vertex a highly potent drug from a different class of agents that it will combine with Telaprevir in the hopes of further improving the treatment paradigm for HCV. We expect results from Phase III trials with VX-770 for patients with cystic fibrosis in the first half of 2011. If positive, the drug could be the first to address one of the underlying defects in a subgroup of patients with this devastating disease.

Back