Vertex Enters Agreement with Bristol-Myers Squibb for Phase 2 All-Oral
Studies of VX-135 in Combination with Daclatasvir for the Treatment of
-Two Phase 2 studies to evaluate once-daily combination of Vertex's
investigational nucleotide analogue VX-135 and BMS’ investigational NS5A
replication complex inhibitor daclatasvir-
-Study in people with genotype 1 hepatitis C planned to begin in second
quarter of 2013-
-Study in people with genotypes 1, 2 and 3 hepatitis C, including people with
cirrhosis, planned for second half of 2013-
CAMBRIDGE, Mass. -- April 05, 2013
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced it has
entered into a non-exclusive agreement with Bristol-Myers Squibb Company
(NYSE: BMY) to conduct Phase 2 studies of once-daily all-oral treatment
regimens containing Vertex’s nucleotide analogue hepatitis C virus (HCV)
polymerase inhibitor VX-135 and Bristol-Myers Squibb’s NS5A replication
complex inhibitor daclatasvir for the treatment of hepatitis C. As part of the
agreement, Vertex plans to conduct two Phase 2 studies of the combination,
including an initial study in treatment-naïve people with genotype 1 HCV
infection planned for the second quarter of 2013. Vertex plans to begin a
subsequent study in treatment-naïve people infected with genotype 1, 2 or 3
HCV, including those with cirrhosis, in the second half of 2013, pending data
from the initial study.
“With more than 170 million people infected worldwide, there is a critical
need for new hepatitis C medicines that can offer people simpler and more
tolerable treatment regimens that provide high cure rates,” said Robert
Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at
Vertex. “These studies with daclatasvir will provide the first opportunity to
evaluate VX-135 as part of all-oral regimens in people with multiple hepatitis
C genotypes and in people with cirrhosis.”
Clinical Development Plans for VX-135 with Daclatasvir
Under the terms of the agreement, Vertex will conduct two Phase 2 studies of
VX-135 in combination with daclatasvir. The first study will enroll
approximately 20 non-cirrhotic, treatment-naïve people with chronic genotype 1
HCV infection and is expected to begin in the second quarter of 2013. In the
second half of 2013, Vertex plans to conduct a subsequent study in
approximately 250 treatment-naïve people with chronic genotype 1, 2 or 3 HCV
infection, including those with cirrhosis. Each of these studies is expected
to evaluate safety, tolerability, pharmacokinetics and viral cure rates (SVR4
and SVR12) of multiple all-oral regimens of VX-135 and daclatasvir dosed once
daily, pending regulatory discussions. Vertex will also conduct co-formulation
activities to evaluate the potential for development of a once-daily
fixed-dose combination regimen. Further clinical development activities beyond
the Phase 2 studies are not covered as part of this agreement.
VX-135 is a uridine nucleotide analogue pro-drug designed to inhibit the
replication of the hepatitis C virus by acting on the NS5B polymerase. In
people with genotype 1, treatment with a 200mg once-daily dose of VX-135 in a
7-day viral kinetic study was well-tolerated, with no discontinuations due to
adverse events, and resulted in a 4.54 log median reduction from baseline
in HCV RNA. Data from a 7-day viral kinetic study of VX-135 in people with
genotypes 2, 3 and 4 were consistent with data observed in people with
genotype 1 and have been submitted for presentation at a future medical
Vertex gained worldwide rights to ALS-2200, known as VX-135 in Phase 2
studies, through an exclusive licensing agreement signed with Alios BioPharma,
Inc. in June 2011. The agreement also includes a research program that focuses
on the discovery of additional nucleotide analogues that act on hepatitis C
polymerase. Vertex has the option to select additional compounds for
development emerging from the research program.
Daclatasvir is an NS5A replication complex inhibitor that is being extensively
studied as a key component of potential DAA-based hepatitis C treatment
regimens. Studied in more than 4,100 patients to date, daclatasvir is in Phase
Daclatasvir is part of a portfolio of investigational compounds with different
mechanisms of action that Bristol-Myers Squibb is developing for the treatment
of hepatitis C. These compounds are being studied as part of multiple novel
treatment regimens with the goal of increasing SVR rates across diverse
patient types and geographies.
About Hepatitis C
Hepatitis C is a serious liver infection caused by the hepatitis C virus,
which is spread through direct contact with the blood of infected people and
ultimately affects the liver.^1 Chronic hepatitis C can lead to serious and
life-threatening liver problems, including liver damage, cirrhosis, liver
failure or liver cancer.^1 Though many people with hepatitis C may not
experience symptoms, others may have symptoms such as fatigue, fever, jaundice
and abdominal pain.^1 Unlike HIV and hepatitis B virus, chronic hepatitis C
can be cured.^2 If treatment is not successful and a person does not achieve a
viral cure, they remain at an increased risk for progressive liver
More than 170 million people worldwide are chronically infected with hepatitis
C.^5 In the United States, up to 5 million people have chronic hepatitis C and
75 percent of them are unaware of their infection.^6,7 Hepatitis C is four
times more prevalent in the United States compared to HIV.^7 The majority of
people with hepatitis C in the United States were born between 1945 and 1965,
accounting for three fourths of people with the infection.^8 Hepatitis C is
the leading cause of liver transplantations in the United States and is
reported to contribute to 15,000 deaths annually.^9,10 By 2029, total annual
medical costs in the United States for people with hepatitis C are expected to
more than double, from $30 billion in 2009 to approximately $85 billion.^11
Vertex creates new possibilities in medicine. Our team discovers, develops and
commercializes innovative therapies so people with serious diseases can lead
Vertex scientists and our collaborators are working on new medicines to cure
or significantly advance the treatment of hepatitis C, cystic fibrosis,
rheumatoid arthritis and other life-threatening diseases.
Founded more than 20 years ago in Cambridge, Mass., we now have ongoing
worldwide research programs and sites in the U.S., U.K. and Canada. Today,
Vertex has more than 2,000 employees around the world, and for three years in
a row, Science magazine has named Vertex one of its Top Employers in the life
Vertex Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, Dr. Kauffman's statements in the second paragraph of the press
release and statements regarding Vertex's expectations with respect to the
timing and structure of studies evaluating the combination of VX-135 and
daclatasvir. While Vertex believes the forward-looking statements contained in
this press release are accurate, there are a number of factors that could
cause actual events or results to differ materially from those indicated by
such forward-looking statements. Those risks and uncertainties include, among
other things, that the initiation of planned studies may be delayed or
prevented, that the outcomes of Vertex's planned clinical studies may not be
favorable and other risks listed under Risk Factors in Vertex's annual report
and quarterly reports filed with the Securities and Exchange Commission and
available through the company's website at www.vrtx.com. Vertex disclaims any
obligation to update the information contained in this press release as new
information becomes available.
^1 Centers for Disease Control and Prevention. Hepatitis C Fact Sheet: CDC
Viral Hepatitis. Available at:
http://www.cdc.gov/hepatitis/HCV/PDFs/HepCGeneralFactSheet.pdf Updated June
2010. Accessed September 21, 2012.
^2 Pearlman BL and Traub N. Sustained Virologic Response to Antiviral Therapy
for Chronic Hepatitis C Virus Infection: A Cure and So Much More. Clin Infect
Dis. 2011 Apr;52(7):889-900.
^3 Morgan TR, Ghany MG, Kim HY, Snow KK, Lindsay K, Lok AS. Outcome of
sustained virological responders and non-responders in the Hepatitis C
Antiviral Long-Term Treatment Against Cirrhosis (HALT-C) trial. Hepatology.
2008;50(Suppl 4):357A (Abstract 115).
^4 Veldt BJ, Heathcote J, Wedmeyer H. Sustained virologic response and
clinical outcomes in patients with chronic hepatitis C and advanced fibrosis.
Annals of Internal Medicine. 2007; 147: 677-684.
^5 Ghany MG, Strader DB, Thomas DL, Seeff, LB. Diagnosis, management and
treatment of hepatitis C; An update. Hepatology. 2009;49 (4):1-40.
^6 Chak, E, et. al. Hepatitis C Virus Infection In USA: An Estimate of True
Prevalence. Liver Intl. 2011;1096 -1098.
^7 Institute of Medicine of the National Academies. Hepatitis and liver
cancer: a national strategy for prevention and control of hepatitis B and C.
Colvin HM and Mitchell AE, ed. Available at:
Updated January 11, 2010. Accessed September 21, 2012.
^8 Centers for Disease Control and Prevention. Recommendations for the
Identification of Chronic Hepatitis C Virus Infection Among Persons Born
During 1945–1965. Morbidity and Mortality Weekly Report. August 17, 2012;
^9 Volk MI, Tocco R, Saini S, Lok, ASF. Public health impact of antiviral
therapy for hepatitis C in the United States. Hepatology.
^10 Ly KN, et al. The Increasing Burden of Mortality From Viral Hepatitis in
the United States Between 1999 and 2007. Ann Intern Med. 2012;156:271-278.
^11 Pyenson B, Fitch K, and Iwasaki K. Consequences of Hepatitis C Virus
(HCV): Costs of a Baby Boomer Epidemic of Liver Disease. Milliman, Inc. May
2009. Available at: http://www.vrtx.com/assets/pdfs/MillimanReport.pdf
VRTX – GEN
Vertex Pharmaceuticals Incorporated
Zach Barber, 617-341-6470
Michael Partridge, 617-341-6108
Kelly Lewis, 617-961-7530
Press spacebar to pause and continue. Press esc to stop.