Vertex HCV Drug Studies Update

Vertex HCV Drug Studies Update

Vertex Pharmaceuticals Reports Third Quarter 2009 Financial Results and Highlights Recent Business and Clinical Progress
Mon Oct 26, 2009 4:01pm EDT

-Phase 3 registration programs in hepatitis C and cystic fibrosis on track-

-Vertex to present telaprevir SVR data from Study C208 at AASLD meeting this
week-

CAMBRIDGE, Mass.--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reviewed recent
business and clinical progress and reported consolidated financial results for
the quarter ended September 30, 2009.

"Vertex has made significant advancements across its business and expects to
enter 2010 in a strong financial position that will enable the continued
investment in late-stage development programs in hepatitis C virus infection and
cystic fibrosis," said Matthew Emmens, Chairman, President and Chief Executive
Officer of Vertex Pharmaceuticals. "We remain focused on the completion of the
telaprevir Phase 3 registration program and are on track to submit a telaprevir
New Drug Application in the second half of 2010. In addition, we believe ongoing
clinical trials of telaprevir and of our novel HCV polymerase inhibitor VX-222
will enable the initiation of the first combination trial of these two compounds
in HCV patients in the coming months - underscoring our commitment to improve
patient care in HCV."

Mr. Emmens continued, "Later this week, we expect to present final SVR data from
Study C208 at the AASLD meeting in Boston showing the potential for telaprevir
to be dosed twice-daily as part of a response-guided treatment regimen. Our
confidence in telaprevir`s competitive profile remains high, and we look forward
to the presentation of data from C208 and other clinical trials in the coming
days.

"In cystic fibrosis, we recently completed enrollment in a Phase 2 trial of
VX-809, our novel CFTR corrector compound, and we continue to enroll patients
across the three trials of the Phase 3 registration program for VX-770, our
novel CFTR potentiator. VX-770 and VX-809 aim to address the underlying
defective protein responsible for this orphan disorder, with the goal of
enabling cystic fibrosis patients to live a more normal life," Mr. Emmens said.

Broad Commitment to Hepatitis C

Phase 3 registration program ongoing:ADVANCE, ILLUMINATE and REALIZE trials

* The ADVANCE, ILLUMINATE and REALIZE trials are evaluating telaprevir-based
regimens as part of a global Phase 3 registration program in more than 2,200
genotype 1 treatment-naïve and treatment-failure patients with hepatitis C virus
(HCV) infection.
* Vertex expects sustained viral response (SVR) data to become available from
ADVANCE and ILLUMINATE in the first half of 2010 and from REALIZE in mid-2010.
Vertex plans to submit a New Drug Application (NDA) for telaprevir in the second
half of 2010.
* The Phase 3 ADVANCE trial is evaluating telaprevir, or placebo, as part of a
24-week telaprevir-based response-guided treatment regimen in combination with
pegylated interferon (peg-IFN) and ribavirin (RBV) in more than 1,050
treatment-naïve HCV patients. The response-guided trial design is utilizing
rapid viral response (RVR) criteria to determine which telaprevir patients can
stop all treatment at 24 weeks.
* The Phase 3 ILLUMINATE trial is evaluating response-guided telaprevir-based
regimens, or placebo, in more than 500 treatment-naïve HCV patients. This trial
is designed to supplement SVR data obtained from the pivotal Phase 3 ADVANCE
trial. The aim of the ILLUMINATE trial is to characterize whether there is an
additional benefit to extending treatment from 24 to 48 weeks in treatment-naïve
patients who achieved undetectable virus levels at weeks 4 and 12 of treatment
(eRVR).
* The Phase 3 REALIZE trial is evaluating 48-week telaprevir-based regimens, or
placebo, in more than 650 patients with genotype 1 HCV who did not achieve an
SVR with a previous peg-IFN-based treatment. The REALIZE trial enrolled all
major treatment-failure groups, including null responders.

SVR data from telaprevir twice-daily dosing to be presented at AASLD this week

* Vertex expects that final SVR data from Study C208, which is evaluating
twice-daily telaprevir dosing, will be presented at a Presidential Plenary
session at the upcoming Annual Meeting of the American Association for the Study
of Liver Diseases (AASLD), Oct. 30 - Nov. 3 in Boston. The C208 presentation at
AASLD represents the first SVR data for telaprevir-based regimens, including SVR
results from twice-daily dosing of telaprevir, as part of a response-guided
therapy trial design, similar to that being used in the ADVANCE and ILLUMINATE
Phase 3 trials of telaprevir. Study C208 is an exploratory Phase 2, open-label
clinical study conducted by Tibotec in Europe that evaluated a twice-daily
(1125mg q12h) dosing schedule of telaprevir in combination with peg-IFN-alfa-2a
(PEGASYS®) or peg-IFN-alfa-2b (PEGINTRON®) and RBV, as compared to the current
three-times-daily (750mg q8h) telaprevir dosing schedule.

Additional telaprevir clinical studies in patients who failed prior HCV therapy

* Vertex has completed PROVE 3, a Phase 2b clinical trial of telaprevir-based
combination therapy in patients with genotype 1 HCV who did not achieve an SVR
with a previous peg-IFN-based treatment. Final PROVE 3 data, including 48-week
follow-up SVR rates (SVR48), will be presented at AASLD.
* Vertex is also conducting Study 107, an open-label Phase 2 study to evaluate
telaprevir-based combination regimens in patients who did not achieve an SVR in
the 48-week control arms of the PROVE 1, PROVE 2 and PROVE 3 studies. In Study
107, telaprevir was given in combination with peg-IFN and RBV for 12 weeks
followed by peg-IFN and RBV for 12 weeks or 36 weeks depending on the patient`s
antiviral response to telaprevir in Study 107 and whether the patient was a
prior null-responder, partial-responder or relapser.

On track to initiate STAT-C combination trial as early as Q4 2009

* Vertex is seeking to advance HCV therapy through the development of novel
combinations of Specifically-Targeted Antiviral Therapies for hepatitis C
(STAT-Cs).
* Vertex is currently conducting a three-day, multiple-dose viral kinetic study
to evaluate the antiviral activity, safety, tolerability and pharmacokinetics of
the HCV polymerase inhibitor VX-222. In the trial, VX-222 is being administered
at four different doses as a monotherapy in 32 treatment-naïve patients with
genotype 1 HCV infection. Vertex is also currently conducting a drug-drug
interaction study with VX-222 and telaprevir in healthy volunteers.
* Vertex expects to obtain data from these trials in the fourth quarter of 2009,
which could enable the initiation of a combination trial of telaprevir and
VX-222 in patients with genotype 1 HCV as early as the fourth quarter of 2009.
Vertex expects data from this first STAT-C combination study of telaprevir and
VX-222 to become available by mid-2010.

Additional HCV compounds in clinical development

* Vertex is also evaluating additional HCV compounds, including the HCV protease
inhibitors VX-813 and VX-985 as well as the HCV polymerase inhibitor VX-759.
* Vertex also has an NS5A inhibitor program in preclinical development.
* The goal of these programs is to identify compounds that are appropriate for
further development, including combination therapy.

AASLD

* The upcoming AASLD meeting, being held Oct. 30 - Nov. 3 in Boston, is expected
to include three telaprevir-related clinical presentations, including
presentations on SVR results from Study C208, final SVR48 results from PROVE 3
and results from a pooled analysis of PROVE 1 and PROVE 2 in "difficult-to-cure"
patients.