New data from Phase 3 studies showed superior SVR (viral cure) rates achieved with telaprevir-based combination therapy in people with hepatitis C, regardless of race or stage of liver disease

Boston, MA, October 30, 2010 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new data from its Phase 3 studies of people with genotype 1 chronic hepatitis C who have not been treated previously. In these studies, the majority of people achieved superior sustained viral response (SVR or viral cure) rates with a telaprevir-based combination regimen, compared to current therapies, regardless of race/ethnicity or stage of liver fibrosis (factors known to limit response to current hepatitis C treatments). Patients in the ADVANCE and ILLUMINATE studies were given telaprevir with pegylated-interferon and ribavirin for the first 12 weeks of the studies, followed by treatment with pegylated-interferon and ribavirin alone for a total of either 24 weeks or 48 weeks based on their response to treatment at weeks 4 and 12. Final data from the Phase 3 ADVANCE and ILLUMINATE studies are being presented at the 61st Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which takes place in Boston October 29 to November 2.

"In our Phase 3 program, starting people with 12 weeks of telaprevir-based combination therapy resulted in significant improvements in viral cure rates, regardless of race, extent of liver damage or experience with prior treatment," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer for Vertex. "The results of the ADVANCE and ILLUMINATE studies represent a major milestone in the development of telaprevir and offer hope for doctors and the millions of people living with hepatitis C who need new and more effective medicines."

Vertex Pharmaceuticals Incorporated is developing telaprevir in collaboration with Tibotec and Mitsubishi Tanabe Pharma.

Results from ADVANCE & ILLUMINATE

Overall in ADVANCE, 75% of people treated with a telaprevir-based combination regimen for 12 weeks, followed by an additional 12 or 36 weeks of pegylated-interferon and ribavirin alone, achieved SVR, compared to 44% of people treated with 48 weeks of pegylated-interferon and ribavirin alone. New data from this study showed that 62% of African Americans/Blacks achieved SVR with telaprevir compared to 25% of African Americans/Blacks who were treated with pegylated-interferon and ribavirin alone. Additionally, 62% of people with advanced liver fibrosis or cirrhosis (scarring of the liver) achieved SVR with telaprevir compared to 33% who were treated with pegylated-interferon and ribavirin alone.

Response-guided therapy was used in ADVANCE, whereby patients whose virus was undetectable at weeks 4 and 12 of treatment with telaprevir-based therapy were eligible to reduce their treatment time by half – from 48 weeks to 24 weeks. ILLUMINATE was designed to confirm both the use of response-guided therapy and to evaluate whether there was any benefit in extending therapy from 24 weeks to 48 weeks in people who met these criteria. In ADVANCE and ILLUMINATE, 58% and 65% of people, respectively, met these criteria for 24-week total treatment. In ILLUMINATE there was no benefit in extending therapy to 48 weeks.

In ILLUMINATE, 72% of people overall achieved SVR with telaprevir-based therapy. New data from this study showed that 60% of African Americans/Blacks and 63% of people with advanced liver fibrosis or cirrhosis achieved SVR with telaprevir-based therapy in the overall study analysis. Of African Americans/Blacks whose virus was undetectable at weeks 4 and 12, 88% of people achieved SVR in both the 24-week and 48-week randomized treatment arms. There was no control arm of pegylated-interferon and ribavirin alone in ILLUMINATE.

The safety and tolerability profile of telaprevir was consistent in both trials, with low discontinuation rates of all drugs during the telaprevir treatment phase due to adverse events.

"Less than half of people with the most common form of hepatitis C - genotype 1- achieve a viral cure with currently approved medicines, and factors such as race and extent of liver fibrosis can further limit cure rates to less than a third," said Ira Jacobson, M.D., Chief of the Division of Gastroenterology and Hepatology, at New York-Presbyterian Hospital/Weill Cornell Medical Center, and the Vincent Astor Distinguished Professor of Medicine at Weill Cornell Medical College. "After treatment with telaprevir-based combination therapy in the ADVANCE study, 75% of people overall achieved a viral cure. Importantly, 62% of African Americans/Blacks and people with extensive liver disease achieved a viral cure – nearly twice as many as those who received pegylated-interferon and ribavirin alone."

ADVANCE Treatment Group RVR+ eRVR++ SVR

Overall Percent of patients w/eRVR who achieved SVR Bridging Fibrosis/Cirrhosis African American/Black Hispanic/
Latino 12-week
TVR- based- arm*
(n=363) 68%
(n=246/363) 58%
(n=212/363) 75%+/-
(n=271/363) 89%
(n=189/212) 62%
(n=45/73) 62%
(n=16/26) 74%
(n=26/35) 8-week TVR-based- arm**(n=364) 66%
(n=242/364) 57%
(n=207/364) 69%+/-
(n=250/364) 83%
(n=171/207) 53%
(n=45/85) 58%
(n=23/40) 66%
(n=29/44) Control arm***(n=361) 9%
(n=34/361) 8%
(n=29/361) 44%
(n=158/361) 97%
(n=28/29) 33%
(n=24/73) 25%
(n=7/28) 39%
(n=15/38) * 12 weeks of telaprevir (TVR), Pegasys(R) (PEG, pegylated-interferon alfa-2a) and Copegus(R) (RBV, ribavirin) followed by 12 or 36 weeks of only PEG and RBV, based on response to treatment at week 4 and week 12

** 8 weeks of telaprevir (TVR) Pegasys (PEG, pegylated-interferon alfa-2a) and Copegus (RBV, ribavirin) followed by 16 or 40 weeks of only PEG and RBV, based on response to treatment at weeks 4 and 12

*** 48 weeks of PEG and RBV

+ RVR: rapid viral response; undetectable ( END