-- Median 5.5 log10 reduction in HCV RNA achieved in patients receiving VX-950
and peg-IFN in 14-day clinical study --
CAMBRIDGE, Mass., Jan. 9 /PRNewswire-FirstCall/ -- New data announced
today by Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) show that VX-950,
an investigational oral hepatitis C virus (HCV) protease inhibitor, dosed in
combination with pegylated interferon alfa-2a (Pegasys(R); peg-IFN), achieved
a rapid and dramatic reduction in plasma viral RNA levels in patients with
chronic genotype 1 HCV infection. In this Phase Ib study, the combination of
VX-950 and peg-IFN produced an initial median reduction in plasma HCV RNA of
more than 3 log10 in the first two days, followed by continued decline to a
median 5.5 log10 reduction in HCV RNA at day 14, which equates to a 300,000-
fold reduction in viral levels. The majority of patients (6 of 8) receiving
the combination achieved HCV RNA levels below the limit of quantitation (30
IU/mL, as measured by the Roche TaqMan(R) assay) at 14 days, with 4 of 8
patients achieving HCV RNA levels below the limit of detection (10 IU/mL,
Roche TaqMan(R)). The antiviral activity of the combination through 14 days
was significantly greater than the activity of VX-950 administered as a single
agent, and much greater than peg-IFN alone. In addition, VX-950 appeared to
be well-tolerated when dosed alone and in combination with peg-IFN in the
study. The full data set will be presented at a medical conference later this
year.
"These data show that VX-950, in combination with pegylated interferon,
produced a very rapid viral response in each of these genotype 1 patients, who
are historically the most difficult to treat effectively," said Henk W.
Reesink, MD, Associate Professor of Medicine at Academic Medical Center in
Amsterdam, and a lead investigator for the study. "The profound decreases in
viral load strongly support the evaluation of VX-950 in combination with
pegylated interferon as part of a three-month treatment paradigm to achieve
sustained viral responses (SVR) in HCV patients."
Study Design and Results
The 14-day, randomized, blinded, placebo-controlled Phase Ib study
enrolled 20 treatment-naive patients with genotype 1 HCV, the most prevalent
and difficult to treat form of HCV infection. Patients were randomized to
receive a new tablet formulation of VX-950 at a dose of 750 mg every eight
hours (q8h) in combination with a standard dose of peg-IFN (n=8), the same
dose of VX-950 administered alone (n=8), or a standard dose of peg-IFN alone
(n=4). The median viral load for all patients at study entry was 6.65 log10
IU/mL HCV RNA (approximately 4.4 million IU/mL). Available interim results
indicate:
* A median 5.5 log10 reduction in HCV RNA in patients receiving VX-950 and
peg-IFN for 14 days; 6 of 8 patients had viral levels below the limit of
quantitation (30 IU/mL) at 14 days, and 4 of 8 also achieved viral
levels below the limit of detection (10 IU/mL).
* A median 4.0 log10 reduction in HCV RNA in patients receiving VX-950
alone for 14 days; 1 of 8 patients had viral levels below the limit of
detection (10 IU/mL).
* A median 1.0 log10 reduction in HCV RNA in patients receiving peg-IFN
alone for 14 days; no patients had viral levels below the limit of
quantitation (30 IU/mL) at 14 days.
Safety
A preliminary safety review has been conducted that indicates that the
treatment was well tolerated. All patients completed dosing and no serious
adverse events were reported. All adverse events in the patients receiving
VX-950 alone were reported as mild. Typical interferon-related side effects,
of mild to moderate severity, were reported in the patients that received peg-
IFN along with VX-950 or placebo. In addition, laboratory-related adverse
events of neutropenia in one patient and thrombocytopenia in one patient were
reported among the patients who received peg-IFN. Neutropenia and
thrombocytopenia have previously been reported in patients receiving peg-IFN
alone. It is not known if the two patients in whom these events occurred were
also receiving VX-950, because the full safety database has not yet been
unblinded. A complete safety analysis will be conducted once the study is
fully unblinded.
"The data announced today provide further support for VX-950's potential
to transform the standard of care in HCV," said Joshua Boger, Ph.D., Chairman,
President and Chief Executive Officer of Vertex. "We look forward to pursuing
additional clinical studies in 2006 that will evaluate the ability of VX-950,
in combination with pegylated interferon, to achieve sustained viral responses
in HCV patients, with a shorter duration of treatment compared to the current
standard of care."
Clinical Plans
Vertex is conducting a broad Phase II development program designed to
establish the safety and antiviral activity of VX-950 in studies of up to
three months duration. In the next few months, Vertex expects to initiate a
three-month Phase II trial with more than 200 participants that will study VX-
950 dosed in combination with peg-IFN, both with and without ribavirin,
another standard HCV treatment. This three-month study will include a
comparison to the current standard of care in HCV treatment. Additionally, a
12-patient, 28-day Phase II trial of VX-950 plus peg-IFN and ribavirin has now
completed enrollment and preliminary data from this study are anticipated in
the first quarter. In December 2005, VX-950 received Fast Track designation
from the U.S. Food and Drug Administration.
About Hepatitis C
Hepatitis C is a liver disease caused by the hepatitis C virus, which is
found in the liver and blood of people with the disease. HCV, a serious
public health concern affecting 3.4 million individuals in the United States,
is spread primarily through direct contact with the blood of infected people.
Though many people with hepatitis C may not experience symptoms nor be aware
of their infection, others may have symptoms such as jaundice and fatigue.
Hepatitis C significantly increases a person's risk for developing chronic
liver disease, cirrhosis, the need for liver transplantation, liver cancer, or
death. Current treatments are commonly dosed for six to 12 months and may be
associated with significant adverse events.
About VX-950 and Previous Clinical Data
VX-950 is an oral inhibitor of hepatitis C virus protease, an enzyme
essential for viral replication. In 2005, Vertex reported results from a 14-
day, Phase Ib study of VX-950 dosed as a single agent in genotype 1 HCV
patients. In this prior study, VX-950 displayed potent antiviral activity.
After 14 days of dosing, patients in the dose group with the best response
(750 mg every 8 hours) achieved a median reduction in HCV RNA of 4.4 log10, a
25,000-fold reduction in viral levels. Adverse events observed in patients
receiving VX-950 that were considered possibly related to the drug were mild,
and generally similar in frequency to events in patients receiving placebo.
The most common adverse events reported in both placebo and VX-950 patients
were headache, frequent urination and gastrointestinal symptoms.
Vertex researchers were the first to solve the three-dimensional crystal
structure of HCV protease, and have used structural insights to enable the
design of small molecule HCV protease inhibitors, including VX-950.
About Vertex
Vertex Pharmaceuticals Incorporated is a global biotechnology company
committed to the discovery and development of breakthrough small molecule
drugs for serious diseases. The Company's strategy is to commercialize its
products both independently and in collaboration with major pharmaceutical
companies. Vertex's product pipeline is principally focused on viral
diseases, inflammation, autoimmune diseases and cancer. Vertex co-promotes
the HIV protease inhibitor, Lexiva, with GlaxoSmithKline.
Safe Harbor Statement
This press release may contain forward-looking statements, including
statements that (i) VX-950 has the potential to transform the standard of care
in HCV infection; (ii) Vertex will pursue additional clinical studies in 2006,
including a three-month Phase II trial with more than 200 patients planned for
commencement in the next few months, that will evaluate the ability of VX-950
to achieve sustained viral responses (SVR) in HCV patients; and (iii) Vertex
will receive preliminary data from its ongoing 28-day Phase II clinical trial
in the first quarter of 2006. While management makes its best efforts to be
accurate in making forward-looking statements, such statements are subject to
risks and uncertainties that could cause Vertex's actual results to vary
materially. These risks and uncertainties include, among other things, the
risks that (i) full analysis of the data, or further testing, will not reflect
the interim results reported in this press release, or support any or all of
the conclusions provided in this press release; and (ii) clinical trials for
VX-950 may not proceed as planned due to technical, scientific, or patient
enrollment issues, clinical trial results may not be available when expected,
or expected regulatory filings may not occur or may be delayed due to adverse
clinical or non-clinical trial developments or unanticipated FDA action; and
other risks listed under Risk Factors in Vertex's Form 10-K filed with the
Securities and Exchange Commission on March 16, 2005.
Lexiva is a registered trademark of the GlaxoSmithKline group of
companies, and Pegasys is a registered trademark of Hoffman-La Roche Inc.
Vertex's press releases are available at http://www.vrtx.com.
Vertex Contacts:
Lynne H. Brum, Vice President, Strategic Communications, (617) 444-6614
Michael Partridge, Director, Corporate Communications, (617) 444-6108
Lora Pike, Manager, Investor Relations, (617) 444-6755
Zachry Barber, Specialist, Media Relations, (617) 444-6470
SOURCE Vertex Pharmaceuticals Incorporated
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Related links:
http://www.vrtx.com
Company News On-Call:
http://www.prnewswire.com/comp/938395.html
CONTACT:
Lynne H. Brum, Vice President, Strategic
Communications, +1-617-444-6614, or Michael Partridge, Director,
Corporate Communications, +1-617-444-6108, or Lora Pike, Manager,
Investor Relations, +1-617-444-6755, or Zachry Barber,
Specialist, Media Relations, +1-617-444-6470, all of Vertex
Pharmaceuticals Incorporated
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