Vertex and Tibotec Start Phase 3 Study of Telaprevir for Treatment-Naive Genotyp

[Guest guest]

Vertex and Tibotec Start Phase 3 Study of Telaprevir for Treatment-

Naive Genotype 1 Hepatitis C Patients

On March 13, Vertex Pharmaceuticals and Tibotec announced the

initiation of a new Phase 3 trial, called ADVANCE, which will

evaluate the experimental oral HCV protease inhibitor telaprevir (VX-

950) in previously untreated patients with genotype 1 chronic

hepatitis C virus (HCV) infection.

Below is an excerpt from the companies' press release describing the

new study:

Vertex Pharmaceuticals and Tibotec Announce Start of Phase

3 'ADVANCE' Study with Telaprevir in Treatment-Naive, Genotype 1 HCV

Patients

-- First hepatitis C protease inhibitor to begin Phase 3 clinical

development -- Trial designed to confirm potential of telaprevir to

increase sustained viral response (SVR) rates with 24-week treatment

duration

CAMBRIDGE, Mass., Mar 13, 2008 -- Vertex Pharmaceuticals Incorporated

(Nasdaq: VRTX) and Tibotec today announced that patient screening has

begun in the ADVANCE study, a pivotal Phase 3 clinical study with the

hepatitis C virus (HCV) protease inhibitor telaprevir in combination

therapy for treatment-naive patients with chronic HCV infection.

Telaprevir is the most advanced HCV protease inhibitor in clinical

development targeting treatment of hepatitis C, a disease that

afflicts more than 3 million people in the United States alone, and

170 million worldwide.

The ADVANCE trial will enroll 1,050 treatment-naive genotype 1 HCV

patients and will evaluate two 24-week telaprevir-based regimens in

comparison to a 48-week control arm. The primary endpoint of the

study is sustained viral response (SVR), defined as undetectable HCV

RNA (<10 IU/mL) 24 weeks after the completion of treatment. In this

study, rapid viral response (RVR) criteria will be used to determine

which telaprevir patients can stop all treatment at 24 weeks.

" This is the first Phase 3 study conducted to evaluate whether an

investigational medicine for HCV may be able to both increase the

rate of sustained viral response and shorten the duration of therapy

to 24 weeks in patients with genotype 1 HCV infection compared to

current treatment of 48 weeks. This is important given the

expectation that approximately 40 to 50 percent of people with

genotype 1 HCV who undergo treatment with current therapies achieve

SVR, " said McHutchison, MD, Associate Director, Duke Clinical

Research Institute and a principal investigator for the ADVANCE

study. " We're interested to see whether this trial will confirm the

encouraging results seen thus far in Phase 2 studies of telaprevir.

This study is another important step forward in the evaluation of

novel medicines for the treatment of HCV. "

" Following discussions with the U.S. FDA in January, we have been

able to rapidly finalize the Phase 3 pivotal trial protocol and begin

patient screening at the first sites, " said Alam, MD, Executive

Vice President, Medicines Development and Chief Medical Officer of

Vertex. " The initiation of the ADVANCE trial underscores our

commitment to evaluating the potential for telaprevir to address

significant unmet medical needs in HCV. "

The ADVANCE Study

The ADVANCE study (A New Direction in HCV Care: A Study of Treatment-

Naive Hepatitis C Patients with telaprevir) will be conducted at more

than 100 centers in the U.S., [European Union] and certain other

countries. Patient recruitment is being initiated in the U.S., while

sites in other countries will start recruitment as national Clinical

Trial Applications (CTAs) for each country are approved. The study

arms will include:

• 24 weeks of therapy, with telaprevir dosed at 750 mg every eight

hours (q8h) for 12 weeks in combination with standard doses of

pegylated interferon alfa-2a (peg-IFN) [Pegasys] and ribavirin (RBV)

for 12 weeks, then continuing for another 12 weeks with peg-IFN and

RBV alone;

• 24 weeks of therapy, with telaprevir dosed at 750 mg every eight

hours (q8h) for 8 weeks in combination with standard doses of peg-IFN

and RBV for 8 weeks, then continuing for another 16 weeks with peg-

IFN and RBV alone; and

• A control arm with standard doses of peg-IFN and RBV dosed for 48

weeks.

Patients in both telaprevir arms who achieve RVR, defined in this

study as undetectable (less than 10 IU/mL) HCV RNA levels by the end

of week 4, and who stay undetectable at week 12 will receive 24 weeks

of treatment. Patients in these treatment arms who do not meet these

RVR criteria but are undetectable at week 24 will continue on peg-IFN

and RBV for a total duration of 48 weeks.

Updates on the status of Vertex and Tibotec's clinical trials of

telaprevir are available at www.clinicaltrials.gov.

About Telaprevir

Telaprevir (VX-950) is an investigational oral inhibitor of HCV

protease, an enzyme essential for viral replication, and is one of

the most advanced investigational antiviral agents in development

that specifically targets HCV. The types of adverse events that have

been commonly observed with peg-IFN and RBV were seen across all

treatment arms in Phase 2b trials of telaprevir. The most common

adverse events, regardless of treatment assignment, were fatigue,

rash, headache, and nausea, with rash being the most common reason

for treatment discontinuation. Gastrointestinal disorders, skin

adverse events (rash, pruritus) and anemia were more common in the

telaprevir arms compared to the control arm over the dosing period.

Vertex Pharmaceuticals

Tibotec