Vertex Announces Collaboration with Janssen To Initiate Phase 2 All-Oral
Study of VX-135 and Simeprevir (TMC435) for the Treatment of Hepatitis C
-Companies to evaluate two-drug combination of Vertex’s investigational
nucleotide analogue VX-135 and Janssen’s investigational protease 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 that it has
entered into a non-exclusive collaboration with Janssen Pharmaceuticals, Inc.
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 Janssen’s protease inhibitor
simeprevir (TMC435). As part of the collaboration, Janssen will conduct a
drug-drug interaction study with VX-135 and simeprevir to support the planned
initiation of a Phase 2 proof-of-concept study in early 2013, pending
discussions with regulatory authorities. The Phase 2 study is expected to
evaluate safety, tolerability and viral cure rates using a 12-week combination
of VX-135 and simeprevir. The companies will jointly fund development costs
associated with the collaboration. 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. Simeprevir is a potent, once-daily
investigational hepatitis C protease inhibitor, currently in Phase 3 trials,
being jointly developed by Janssen R&D Ireland and Medivir AB.
“This collaboration is an important step forward in our commitment to develop
new all-oral treatment regimens for people with hepatitis C – both in
collaboration with other companies like Janssen and with medicines within our
own pipeline,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and
Chief Medical Officer at Vertex. “We believe that an all-oral regimen of
VX-135 and simeprevir has the potential to achieve high cure rates with only
12 total weeks of treatment for people with hepatitis C, and we look forward
to the start of the Phase 2 proof-of-concept study with Janssen."
Clinical Development Plans
Vertex and Janssen expect to initiate a Phase 2 proof-of-concept study of
VX-135 and simeprevir in early 2013, following the completion of a drug-drug
interaction (DDI) study and pending discussions with regulatory authorities.
Costs associated with the studies will be shared 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
treatment) of multiple 12-week combination regimens of VX-135 and simeprevir,
with and without ribavirin, in people who have chronic non-cirrhotic genotype
1 hepatitis C and have not previously been treated (treatment-naïve). Further
clinical development activities beyond the Phase 2 proof-of-concept study are
not covered as part of this collaboration. Additional information on the Phase
2 study will be provided upon initiation of the study.
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 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 Simeprevir (TMC435)
Simeprevir is a once-daily potent investigational hepatitis C protease
inhibitor in late Phase 3 clinical development being jointly developed by
Janssen R&D Ireland and Medivir AB to treat chronic hepatitis C virus
infections. Simeprevir is being investigated in combination with PegIFN/RBV in
Phase 3 trials and is also being evaluated with Direct-acting Antiviral (DAA)
agents in three other Phase 2 interferon-free combinations both with and
without ribavirin (RBV). For further details please visit
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
At Janssen we are dedicated to addressing and solving some of the most
important unmet medical needs of our time in oncology, immunology,
neuroscience, infectious diseases and vaccines, and cardiovascular and
metabolic diseases. Driven by our commitment to patients, we develop
innovative products, services and healthcare solutions to help people
throughout the world. Janssen R&D Ireland and Janssen Pharmaceuticals, Inc.
are part of the Janssen Pharmaceutical Companies of Johnson & Johnson. Please
visit http://www.janssenrnd.com for more information.
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 studies evaluating the combination of VX-135 and
simeprevir (TMC435) 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 becomes available.
VRTX – GEN
Vertex Pharmaceuticals Incorporated
Zach Barber, 617-444-6470
Michael Partridge, 617-444-6108
Kelly Lewis, 617-444-7530
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