CAMBRIDGE, Mass. -- Vertex Pharmaceuticals
Incorporated announce the initiation of a Phase I
clinical trial for VX-950, an investigational oral protease inhibitor for the
treatment of hepatitis C virus (HCV) infection. The objective of this trial
is to assess safety, tolerability and pharmacokinetics in escalating single
doses of VX-950 in healthy volunteers. Approximately 35 healthy subjects will
participate in the study, which is being conducted in Europe. Successful
completion of the Phase I clinical trial will enable a first study of VX-950
in HCV-infected patients. Such a study is currently planned to start in the
fourth quarter of 2004.
VX-950 is Vertex's lead oral HCV protease inhibitor and one of a new class
of direct antivirals in development for the treatment of HCV. Preclinical
studies have shown that VX-950 significantly reduces levels of HCV RNA in both
an in vitro replicon system and infectious virus assays. At a scientific
conference in October 2003, Vertex scientists reported that VX-950 reduced HCV
RNA 10,000-fold (4 log 10) in nine days in an in vitro replicon assay.
Preclinical pharmacokinetic studies have indicated that VX-950 is orally
bioavailable and achieves excellent exposure in the liver, the target organ
for HCV treatment. The initiation of clinical testing of VX-950 represents a
first step towards establishing the safety and tolerability in humans.
"Preclinical data to date have indicated that direct antivirals such as
VX-950 may represent a powerful new approach to the treatment of HCV
infection," stated John J. Alam, MD, senior vice president of drug
evaluation and approval at Vertex. "Initiation of human clinical trials for
VX-950 reflects Vertex's commitment to leadership in the development and
commercialization of novel antivirals for the treatment of HCV infection, and
it is one of several important clinical milestones for Vertex's proprietary
development programs in 2004."
Chronic hepatitis C virus (HCV) infection is a serious public health
concern affecting approximately 2.7 million people in the United States. HCV
causes inflammation of the liver, which may lead to fibrosis and cirrhosis,
liver cancer, and ultimately, liver failure. Cirrhosis of the liver resulting
from chronic HCV infection is the leading indication for liver transplantation
in the U.S. Due to the asymptomatic nature of HCV infection, it often goes
undetected for up to 20 years following initial infection. Worldwide, the
disease strikes as many as 185 million people. Each year, 8,000 to 10,000
people in the U.S. die from complications of HCV.
The current standard of care in HCV treatment is a combination of weekly
injections of pegylated interferon alpha (peg-IFN) and daily oral dosing of
ribavirin. This combination therapy provides a sustained viral response for
only 40 to 50 percent of patients chronically infected with genotype 1 HCV,
the most difficult viral strain to treat and the most common form in the U.S.
Vertex's drug development portfolio includes two different approaches for
advancing the future standard-of-care in HCV. In addition to VX-950, Vertex
is developing merimepodib, an IMPDH inhibitor in combination with pegylated
interferon alpha (peg-IFN) and ribavirin. Addition of merimepodib to standard
therapy has the potential to enhance antiviral activity and improve clinical
outcomes for a larger percentage of patients. Vertex owns worldwide
development and commercialization rights for both merimepodib and VX-950.
Source: Vertex Pharmaceuticals Incorporated