press release
Jan. 6, 2013, 5:00 p.m. EST
Vertex Outlines Corporate Strategy and Defines Key 2013 Business Priorities
-2013 investment focused on key development programs in cystic fibrosis, hepatitis C and autoimmune diseases-
Hepatitis C
-- Hepatitis C: Vertex's strategy in hepatitis C is to develop new all-oral treatment regimens of 12 weeks or less in duration with a goal of providing a high viral cure rate and improved tolerability. In 2013, Vertex plans to conduct multiple Phase 2 studies of 12-week all-oral treatment regimens that include the company's nucleotide analogue VX-135. These studies are expected to provide safety and viral cure rate data in the second half of 2013 to support the start of pivotal development of one or more all-oral regimens in 2014.
Vertex plans to initiate multiple studies of the nucleotide analogue VX-135 as part of 12-week all-oral combination treatment regimens. Vertex expects to generate data from these studies in the second half of 2013 to enable progression into pivotal development in 2014. The all-oral studies for VX-135 include:
VX-135 in Combination with Ribavirin
-- In the first quarter of 2013, Vertex expects to begin dosing in a study of VX-135 and ribavirin as part of a 12-week all-oral treatment regimen. The study will evaluate safety, tolerability and viral cure rates (SVR12; undetectable hepatitis C virus 12 weeks after the end of treatment).
Collaborative Agreements for All-Oral Studies of VX-135
-- In late 2012, Vertex entered into two non-exclusive agreements to conduct Phase 2 proof-of-concept studies of VX-135 in combination with simeprevir (TMC435), a once-daily protease inhibitor being jointly developed by Janssen R&D Ireland and Medivir AB, and with GSK2336805, a once-daily NS5A inhibitor in development by GlaxoSmithKline (GSK). These Phase 2 studies will evaluate safety, tolerability and viral cure rates (SVR12; undetectable hepatitis C virus 12 weeks after the end of treatment) using 12-week combination regimens. A drug-drug interaction study with simeprevir will be initiated shortly, and Vertex expects the Phase 2 studies to be initiated in the first half of the year.
SAN FRANCISCO, Jan 06, 2013 (BUSINESS WIRE) -- ---First two Breakthrough
Therapy Designations from U.S. FDA granted to ivacaftor monotherapy and to the
combination regimen of VX-809 and ivacaftor for the treatment of cystic
fibrosis-
Vertex Pharmaceuticals Incorporated
/quotes/zigman/79675/quotes/nls/vrtx VRTX
+1.55% today
outlined the company's strategy and defined key 2013 business priorities to
support continued growth and the creation of long-term shareholder value.
Vertex's Chairman, President and Chief Executive Officer, Jeffrey Leiden, M.D.,
Ph.D., will discuss the company's strategy and 2013 priorities as part of a live
presentation at the 31st Annual J.P. Morgan Healthcare Conference in San
Francisco on Monday, January 7 at 10:00 a.m. PT (1:00 p.m. ET). The presentation
will be webcast on Vertex's website, www.vrtx.com .
"Since the approval of our first medicine less than two years ago, Vertex has
undergone a rapid evolution that has positioned the company to move forward with
a clear focus on using innovative science to develop transformative medicines
for serious diseases in specialty markets," said Dr. Leiden.
"In cystic fibrosis and hepatitis C, KALYDECO and INCIVEK are just the
beginning of what we hope to provide to people with these diseases. In CF,
multiple ongoing and planned studies of KALYDECO monotherapy and other
combinations of our CF medicines in development aim to help many more people
with this disease. In hepatitis C, we are focused on developing multiple
all-oral regimens that could further improve treatment. In addition to our
development programs, we continue to invest in research for future medicines,
with a focus on serious diseases where we have significant scientific expertise
and commercial capabilities."
Entering 2013, Vertex's key strategies and business priorities include:
1. Focusing investment on key development programs for:
-- Cystic Fibrosis (CF): Vertex's CF strategy is to provide benefit to as
many CF patients as possible, and to maximize the benefit for these patients,
with our approved and investigational medicines. KALYDECO(TM) (ivacaftor) is
currently approved for people with CF ages 6 and older who have at least one
copy of the G551D mutation in the CFTR gene (approximately 2,000 people with CF
worldwide). In 2013, the company is conducting multiple Phase 3 label-expansion
and other proof-of-concept studies of ivacaftor monotherapy in people with
certain mutations not studied in prior Phase 3 studies. Vertex also expects to
initiate a pivotal Phase 3 development program for a combination regimen of
VX-809 and ivacaftor in people with CF who have two copies of the F508del
mutation in the first quarter of 2013.
-- Hepatitis C: Vertex's strategy in hepatitis C is to develop new all-oral
treatment regimens of 12 weeks or less in duration with a goal of providing a
high viral cure rate and improved tolerability. In 2013, Vertex plans to conduct
multiple Phase 2 studies of 12-week all-oral treatment regimens that include the
company's nucleotide analogue VX-135. These studies are expected to provide
safety and viral cure rate data in the second half of 2013 to support the start
of pivotal development of one or more all-oral regimens in 2014.
-- Autoimmune Diseases: Vertex's strategy in autoimmune diseases is to
maximize the value of VX-509 across multiple autoimmune diseases globally. The
company will evaluate collaborative opportunities that provide funding and
capabilities to broaden and accelerate global development of VX-509.
2. Investing in innovative research programs to support development of
additional specialty medicines for serious diseases
-- Vertex's research efforts are concentrated on additional advancements in
CF and other genetic diseases and additional serious diseases in specialty
markets.
3. Maximizing revenues and cash flow from the appropriate use of INCIVEK(R)
(telaprevir) and KALYDECO in the U.S. and Canada and from the growth of
KALYDECOin Europe and other countries
4. Maintaining financial strength to support future growth and shareholder
returns
Cystic Fibrosis
In CF, Vertex is rapidly advancing multiple studies aimed at expanding the
number of people who may benefit from our approved and investigational CF
medicines, including ivacaftor monotherapy for people with certain mutations not
evaluated in prior studies and a combination of VX-809 and ivacaftor in people
with two copies of the F508del mutation. The company also is advancing its
second-generation corrector research program, which may lead to further
improvements in CF treatment in the coming years. Vertex today provided the
following updates to its CF development program:
Vertex Receives First Two Breakthrough Therapy Designations from U.S. FDA
-- Vertex today announced that the U.S. Food and Drug Administration (FDA)
granted the first two Breakthrough Therapy Designations to ivacaftor monotherapy
and the combination regimen of VX-809 with ivacaftor for CF. Enacted as part of
the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA),
Breakthrough Therapy Designation is intended to expedite the development and
review of a potential new medicine if it is "intended, alone or in combination
with one or more other drugs, to treat a serious or life-threatening disease or
condition and preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over existing therapies on one or more
clinically significant endpoints, such as substantial treatment effects observed
early in clinical development."(1)
-- The FDA granted Breakthrough Therapy Designation to ivacaftor for
potential additional indications beyond the currently approved use of this
medicine for people with CF ages 6 and older who have the G551D mutation. The
designation for ivacaftor was based on data from clinical and pre-clinical
studies, including Phase 3 data in people with the G551D mutation and
pre-clinical data in the G551D mutation as well as a number of other mutations.
The Breakthrough Therapy Designation for the combination regimen of VX-809 with
ivacaftor was based on the Phase 2 combination data announced in 2012. Multiple
studies are currently underway in an effort to determine whether patients with
other CFTR mutations may benefit from ivacaftor alone, and Vertex is also
preparing to start a pivotal program of VX-809 in combination with ivacaftor in
people who have two copies of the most common CF mutation (F508del).
-- The implications of Breakthrough Therapy Designation cannot be determined
at this time. Vertex is working with the FDA and other global regulatory
agencies to determine any potential implications of the Breakthrough Therapy
Designations to its ongoing and planned development activities, and subsequent
regulatory submissions, for ivacaftor monotherapy and the combination regimen of
VX-809 with ivacaftor.
KALYDECO (ivacaftor) Reimbursement Progress in Europe
-- Since approval of KALYDECO in January 2012, the vast majority of the
eligible patients with the G551D mutation in the U.S. have initiated treatment
with KALYDECO. Growth in 2013 KALYDECO revenues will be dependent on completion
of reimbursement discussions in other countries outside the U.S. The company is
now seeking reimbursement for KALYDECO in multiple countries in Europe. In
December, the National Health Service (NHS) recommended funding of KALYDECO in
England. In England, Vertex will make ivacaftor available to eligible people
with CF as quickly as possible as part of a patient access program with the NHS
and anticipates reimbursement to begin in the second quarter of 2013.
Ivacaftor Label-Expansion Studies
-- There are three Phase 3 label expansion trials and one Phase 2
proof-of-concept study underway for ivacaftor monotherapy: -- A Phase 3 study of
ivacaftor is ongoing in people with CF ages 6 and older who have at least one
copy of the R117H mutation. Approximately 3 percent of people with CF in the
U.S. have at least one R117H mutation.
-- A Phase 3 study of ivacaftor is ongoing in people with CF ages 6 and older
who have at least one non-G551D CFTR gating mutation. Approximately 1 percent of
people with CF in the U.S. have at least one non-G551D gating mutation.
-- A Phase 3 study of ivacaftor was recently initiated in children with CF
ages 2 to 5 who have a gating mutation. Enrollment of this study is underway.
-- A Phase 2 proof-of-concept study is underway evaluating ivacaftor in
people with CF who have clinical evidence of residual CFTR function. This is the
first study to evaluate the safety and efficacy of ivacaftor based on a person's
clinical symptoms and characteristics, or phenotype, rather than solely on their
CFTR mutation, or genotype. Between 5 and 10 percent of people with CF in the
U.S. may have residual CFTR function.
-- Vertex expects to obtain the first data from these studies in the second
half of 2013. In 2013, Vertex expects to discuss with the U.S. FDA any potential
implications of Breakthrough Therapy Designation on the timing and content of
regulatory submissions in the U.S. to support expansion of the KALYDECO label.
The company will also discuss plans for submission of these data with other
global regulatory authorities.
Combination of VX-809 and ivacaftor for People with Two Copies of the F508del
Mutation
-- Vertex recently completed an end-of-Phase 2 meeting with the FDA and is on
track to submit final protocols and initiate a pivotal Phase 3 program of VX-809
in combination with ivacaftor in the first quarter of 2013, pending regulatory
approval.
Combination of VX-661 and ivacaftor for People with Two Copies of the F508del
Mutation
-- A Phase 2 study of VX-661 and ivacaftor is ongoing in people with two
copies of the F508del mutation. Data from this study are expected in the first
half of 2013.
Research to Identify Additional CF Treatment Regimens
-- Vertex has an active and ongoing research program that has identified
next-generation correctors. This research is being conducted as part of the
company's collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., and
is focused on the accelerated discovery and development of correctors that could
play a role in a variety of future combination treatments, including a dual
corrector approach, among others.
Hepatitis C
In hepatitis C, Vertex plans to initiate multiple studies of the nucleotide
analogue VX-135 as part of 12-week all-oral combination treatment regimens.
Vertex expects to generate data from these studies in the second half of 2013 to
enable progression into pivotal development in 2014. The all-oral studies for
VX-135 include:
VX-135 in Combination with Ribavirin
-- In the first quarter of 2013, Vertex expects to begin dosing in a study of
VX-135 and ribavirin as part of a 12-week all-oral treatment regimen. The study
will evaluate safety, tolerability and viral cure rates (SVR12; undetectable
hepatitis C virus 12 weeks after the end of treatment).
Collaborative Agreements for All-Oral Studies of VX-135
-- In late 2012, Vertex entered into two non-exclusive agreements to conduct
Phase 2 proof-of-concept studies of VX-135 in combination with simeprevir
(TMC435), a once-daily protease inhibitor being jointly developed by Janssen
R&D Ireland and Medivir AB, and with GSK2336805, a once-daily NS5A inhibitor
in development by GlaxoSmithKline (GSK). These Phase 2 studies will evaluate
safety, tolerability and viral cure rates (SVR12; undetectable hepatitis C virus
12 weeks after the end of treatment) using 12-week combination regimens. A
drug-drug interaction study with simeprevir will be initiated shortly, and
Vertex expects the Phase 2 studies to be initiated in the first half of the
year.
Autoimmune Diseases
In mid-2012, Vertex initiated a Phase 2b study of VX-509, a selective JAK3
inhibitor, in people with moderate to severe rheumatoid arthritis (RA). This
study is ongoing, with data expected in the second half of 2013. Vertex believes
there is a global opportunity for VX-509 to treat multiple autoimmune diseases.
Vertex will evaluate collaborative opportunities that would enable the company
to maximize the value of VX-509 by providing funding and capabilities to broaden
and accelerate the company's evaluation of VX-509 across multiple additional
autoimmune diseases globally.
Financial Strength to Invest in Research Innovation and Development of New
Medicines
Vertex plans to announce fourth quarter and full-year 2012 financial results,
and 2013 financial guidance, in late January. The company expects total non-GAAP
operating expenses (that exclude cost of revenues, stock-based compensation
expense, and Alios expenses related to the collaboration with Vertex) for 2013
to be similar to 2012. We anticipate a reduction in our 2013 SG&A expenses
compared to 2012. The company's 2013 R&D expenses will primarily support:
-- Investment in broad development activities for our late-stage CF and
hepatitis C programs, including formulation and commercial supply chain
investment
-- Completion of Phase 2 evaluation of VX-509 in RA
-- Investment in research programs aimed at the creation of future medicines
About Vertex
Vertex creates new possibilities in medicine. Our team discovers, develops
and commercializes innovative therapies so people with serious diseases can lead
better lives.
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
sciences.
Indication and Important Safety Information for KALYDECO (ivacaftor)
Ivacaftor (150mg tablets) is indicated for the treatment of cystic fibrosis
(CF) in patients age 6 years and older who have a G551D mutation in the CFTR
gene.
Ivacaftor is not for use in people with CF due to other mutations in the CFTR
gene. It is not effective in CF patients with two copies of the F508del mutation
(F508del/F508del) in the CFTR gene. The efficacy and safety of ivacaftor in
children younger than 6 years of age have not been evaluated.
High liver enzymes (transaminases, ALT and AST) have been reported in
patients receiving ivacaftor. It is recommended that ALT and AST be assessed
prior to initiating ivacaftor, every 3 months during the first year of
treatment, and annually thereafter. Patients who develop increased transaminase
levels should be closely monitored until the abnormalities resolve. Dosing
should be interrupted in patients with ALT or AST of greater than 5 times the
upper limit of normal. Following resolution of transaminase elevations, consider
the benefits and risks of resuming ivacaftor dosing. Moderate transaminase
elevations are common in subjects with CF. Overall, the incidence and clinical
features of transaminase elevations in clinical trials was similar between
subjects in the ivacaftor and placebo treatment groups. In the subset of
patients with a medical history of elevated transaminases, increased ALT or AST
have been reported more frequently in patients receiving ivacaftor compared to
placebo.
Use of ivacaftor with medicines that are strong CYP3A inducers such as the
antibiotics rifampin and rifabutin; seizure medications (phenobarbital,
carbamazepine, or phenytoin); and the herbal supplement St. John's Wort
substantially decreases exposure of ivacaftor, which may diminish effectiveness.
Therefore, co-administration is not recommended.
The dose of ivacaftor must be adjusted when concomitantly used with potent
and moderate CYP3A inhibitors. The dose of ivacaftor must be adjusted when used
in patients with moderate or severe hepatic disease.
Ivacaftor can cause serious adverse reactions including abdominal pain and
high liver enzymes in the blood. The most common side effects associated with
ivacaftor include headache; upper respiratory tract infection (the common cold),
including sore throat, nasal or sinus congestion, and runny nose; stomach
(abdominal) pain; diarrhea; rash; and dizziness. These are not all the possible
side effects of ivacaftor. A list of the adverse reactions can be found in the
full product labeling for each country where ivacaftor is approved. Patients
should tell their healthcare providers about any side effect that bothers them
or doesn't go away.
Please see full U.S. Prescribing Information for KALYDECO at www.KALYDECO.com
, the EU Summary of Product Characteristics for KALYDECO at http://goo.gl/N3Tz4
, and the KALYDECO Canadian Product Monograph at www.vrtx.ca .
Indication and Important Safety Information for INCIVEK (telaprevir)
INCIVEK(R) (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(R) (telaprevir) should always be used in combination with
peginterferon alfa and ribavirin. INCIVEK combination treatment may cause
serious side effects including skin rash and serious skin reactions, anemia (low
red blood cell count) that can be severe, and birth defects or death of an
unborn baby.
Skin rashes are common with INCIVEK combination treatment. Sometimes these
skin rashes and other skin reactions can become serious, require treatment in a
hospital, and may lead to death. Patients should call their healthcare provider
right away if they develop any skin changes during treatment with INCIVEK. Their
healthcare provider will decide if they need treatment or if they need to stop
INCIVEK or any of their other medicines. Patients should not stop taking INCIVEK
combination treatment without talking with their healthcare provider first.
Patients' healthcare providers will do blood tests regularly to check for
anemia. If anemia is severe, the healthcare providers may tell them to stop
taking INCIVEK.
INCIVEK combined with peginterferon alfa and 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. Females who can become pregnant and
females whose male partner takes these medicines must have a negative pregnancy
test before starting treatment, every month during treatment, and for 6 months
after treatment ends. Patients must use two forms of effective birth control
during treatment and for 6 months after all treatment has ended. These two forms
of birth control should not contain hormones, as these 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.
The most common side effects of INCIVEK combination treatment include
itching, nausea, diarrhea, vomiting, anal or rectal problems (including
hemorrhoids, discomfort, burning or itching around or near the anus), 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 provider
about any side effect that bothers them or doesn't go away.
Please see full Prescribing Information including Boxed Warning, and the
Medication Guide for INCIVEK available at www.INCIVEK.com .
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. Leiden's statements in the second and third paragraphs of the press release,
the information provided in the four numbered key strategies and business
priorities for 2013 and statements regarding (i) Vertex rapidly advancing
multiple studies aimed at expanding the number of people who may benefit from
our approved and investigational CF medicines and Vertex's CF research program;
(ii) expectations regarding the submission of final protocols and the initiation
of a pivotal Phase 3 program of VX-809 in combination with ivacaftor; (iii) the
dependence of growth in KALYDECO revenues in 2013 on the completion of
reimbursement discussions in countries outside the U.S. and the anticipation
that reimbursement in England will begin in the second quarter of 2013; (iv)
expectations regarding the timing and structure of all-oral studies of VX-135;
(v) information regarding the company's ongoing and planned studies and the
timing of the availability of data from these studies; (vi) the expectation that
Vertex will generate data from studies of VX-135 in the second half of 2013 to
enable progression into pivotal development in 2014; (vii) potential
collaborative opportunities and (viii) expectations regarding 2013 non-GAAP
operating expenses. 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 company's expectations regarding future KALYDECO revenues
and/or operating expenses may be incorrect, that the outcomes of Vertex's
ongoing and planned clinical studies may not be favorable, that the initiation
of planned studies and/or pivotal programs may be delayed or prevented, that
collaborative arrangements may not be available on acceptable terms 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.
