Vertex Enters Agreement with GlaxoSmithKline for Phase 2 All-Oral Study of
VX-135 and GSK2336805 for the Treatment of Hepatitis C
-Companies to evaluate two-drug combination of Vertex’s investigational
nucleotide analogue VX-135 and GSK’s investigational NS5A inhibitor
-Phase 2 proof-of-concept study to begin in early 2013 to evaluate safety,
tolerability and viral cure rates of 12-week treatment regimen-
CAMBRIDGE, Mass. -- November 01, 2012
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced it has
entered into a non-exclusive agreement with GlaxoSmithKline (GSK) to conduct a
Phase 2 proof-of-concept study of an all-oral regimen for the treatment of
hepatitis C containing Vertex’s nucleotide analogue hepatitis C virus (HCV)
polymerase inhibitor VX-135 and GSK’s NS5A inhibitor GSK2336805. Vertex and
GSK plan to initiate the study in early 2013, pending discussions with
regulatory authorities. The study is expected to evaluate safety, tolerability
and viral cure rates using a 12-week combination of VX-135 and GSK2336805. The
companies will jointly fund costs associated with the study. There are no
up-front or milestone payments associated with the agreement. VX-135 is an
investigational uridine nucleotide analogue pro-drug designed to inhibit the
replication of the hepatitis C virus by acting on the NS5B polymerase.
GSK2336805 is an investigational NS5A replication complex inhibitor being
developed by GSK for the treatment of hepatitis C.
“This agreement underscores our broad commitment to develop all-oral treatment
regimens for people with hepatitis C using medicines within our own pipeline
and by working in collaboration with other companies like GSK who share our
commitment to further improve treatment of this disease,” said Robert
Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at
Vertex. “We are focused on the development of new all-oral treatments for
hepatitis C that have the potential to provide a high cure rate with only 12
weeks of treatment, and we look forward to the start of this study with GSK.”
Clinical Development Plans
The Phase 2 proof-of-concept study is expected to begin in early 2013, pending
discussions with regulatory authorities, and will enroll people who have
chronic non-cirrhotic genotype 1 hepatitis C and have not previously been
treated (treatment-naïve). The study will be conducted in the U.S. by Vertex,
and costs associated with the study will be split equally between the two
companies. The goals of the study will be to evaluate safety, tolerability and
viral cure rates (SVR12; undetectable hepatitis C virus 12 weeks after the end
of treatment) of multiple 12-week combination regimens of VX-135 and
GSK2336805, with and without ribavirin. Additional information on the Phase 2
study will be provided upon initiation of the study. Further clinical
development activities beyond the Phase 2 proof-of-concept study are not
covered as part of this agreement.
VX-135 (ALS-2200) is a uridine nucleotide analogue pro-drug that appears to
have a high barrier to drug resistance based on in vitro studies. It is
designed to inhibit the replication of the hepatitis C virus by acting on the
NS5B polymerase. In vitro studies of the compound showed antiviral activity
across all genotypes, or forms, of the hepatitis C virus, including genotypes
more prevalent outside of the United States.
Earlier this year, Vertex announced the first 7-day viral kinetic data for
VX-135. Based on these data, the company plans to initiate multiple all-oral,
Phase 2 proof-of-concept studies, including a study of VX-135 and ribavirin
and a study of VX-135 and telaprevir, the company's approved protease
inhibitor marketed as INCIVEK for people with chronic genotype 1 hepatitis C.
Vertex is on track to initiate the study of VX-135 in combination with
ribavirin by the end of 2012, followed by the study with telaprevir in early
2013. The studies will evaluate safety, tolerability and viral cure rates
(SVR12; undetectable hepatitis C virus 12 weeks after the end of treatment) of
12-week combination regimens in people with chronic non-cirrhotic genotype 1
hepatitis C who have not previously been treated (treatment-naïve).
Vertex gained worldwide rights to VX-135 through an exclusive licensing
agreement signed with Alios BioPharma, Inc. in June 2011. The agreement also
includes a research program that will focus 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
About Hepatitis C
Hepatitis C is a serious liver disease 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 However,
approximately 60 percent of people do not achieve SVR,^3,4,5 or viral cure,^6
after treatment with 48 weeks of pegylated-interferon and ribavirin alone. If
treatment is not successful and a person does not achieve a viral cure, they
remain at an increased risk for progressive liver disease.^7,8
More than 170 million people worldwide are chronically infected with hepatitis
C.^6 In the United States, up to 5 million people have chronic hepatitis C and
75 percent of them are unaware of their infection.^9,10 Hepatitis C is four
times more prevalent in the United States compared to HIV.^10 The majority of
people with hepatitis C in the United States were born between 1945 and 1965,
accounting for 82 percent of people with the disease.^11 Hepatitis C is the
leading cause of liver transplantations in the United States and is reported
to contribute to 15,000 deaths annually.^12,13 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.^10
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
IMPORTANT SAFETY INFORMATION FOR INCIVEK
INCIVEK® (telaprevir) is a prescription medicine used with the medicines
peginterferon alfa and ribavirin to treat chronic (lasting a long time)
hepatitis C genotype 1 infection in adults with stable liver problems, who
have not been treated before or who have failed previous treatment. It is not
known if INCIVEK is safe and effective in children under 18 years of age.
Important Safety Information:
INCIVEK should always be taken in combination with peginterferon alfa and
ribavirin. Ribavirin may cause birth defects or death of an unborn baby.
Therefore, a patient should not take INCIVEK combination treatment if she is
pregnant or may become pregnant, or if he is a man with a sexual partner who
is pregnant. Patients must use two forms of effective birth control during
treatment and for the 6 months after treatment with these medicines. Hormonal
forms of birth control, including birth control pills, vaginal rings, implants
or injections, may not work during treatment with INCIVEK.
INCIVEK and other medicines can affect each other and can also cause side
effects that can be serious or life threatening. There are certain medicines
patients cannot take with INCIVEK combination treatment. Patients should tell
their healthcare providers about all the medicines they take, including
prescription and non-prescription medicines, vitamins and herbal supplements.
INCIVEK can cause serious side effects including skin reactions, rash and
anemia that can be severe. The most common side effects of INCIVEK include
itching, nausea, diarrhea, vomiting, anal or rectal problems, taste changes
and tiredness. There are other possible side effects of INCIVEK, and side
effects associated with peginterferon alfa and ribavirin also apply to INCIVEK
combination treatment. Patients should tell their healthcare providers about
any side effect that bothers them or doesn't go away.
Please see full Prescribing Information for INCIVEK including the Medication
Guide, available at www.INCIVEK.com.
^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 Manns MP, McHutchison JG, Gordon SC, et al. Peginterferon alfa-2b plus
ribavirin compared with interferon alfa-2b plus ribavirin for initial
treatment of chronic hepatitis C: a randomised trial. Lancet.
^4 Fried MW, Shiffman ML, Reddy KR, et al. Peginterferon alfa-2a plus
ribavirin for chronic hepatitis C virus infection. N Engl J Med.
^5 McHutchison JG, Lawitz EJ, Shiffman ML, et al; IDEAL Study Team.
Peginterferon alfa-2b or alfa-2a with ribavirin for treatment of hepatitis C
infection. N Engl J Med. 2009;361:580-593.
^6 Ghany MG, Strader DB, Thomas DL, Seeff, LB. Diagnosis, management and
treatment of hepatitis C; An update. Hepatology. 2009;49 (4):1-40.
^7 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).
^8 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.
^9 Chak, E, et. al. Hepatitis C Virus Infection In USA: An Estimate of True
Prevalence. Liver Intl. 2011;1096 -1098.
^10 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.
^11 Smith, BD, et al. Hepatitis C Virus Antibody Prevalence, Correlates and
Predictors among Persons Born from 1945 through 1965, United States,
1999-2008. AASLD 2011 Annual Meeting.
^12 Volk MI, Tocco R, Saini S, Lok, ASF. Public health impact of antiviral
therapy for hepatitis C in the United States. Hepatology.
^13 S.D. Holmberg, K.N. Ly., et.al. The Growing Burden of Mortality Associated
with Viral Hepatitis in the United States, 1999-2007. AASLD 2011 Annual
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 a Phase 2 study evaluating the combination of VX-135
and GSK2336805 and other planned all-oral Phase 2 studies with VX-135. 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
VRTX – GEN
Vertex Pharmaceuticals Incorporated
Zach Barber, 617-444-6470
Michael Partridge, 617-444-6108
Kelly Lewis, 617-444-7530
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