|[January 06, 2013]
Vertex Outlines Corporate Strategy and Defines Key 2013 Business Priorities
SAN FRANCISCO --(Business Wire)--
Pharmaceuticals Incorporated (Nasdaq: VRTX) 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 (News - Alert) 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
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. KALYDECOTM (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
3. Maximizing revenues and cash flow from the appropriate use of INCIVEK®
(telaprevir) and KALYDECO in the U.S. and Canada and from the growth of
KALYDECO in Europe and other countries
4. Maintaining financial strength to support future growth and
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.
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
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 cop 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
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
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,
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
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.
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
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
Completion of Phase 2 evaluation of VX-509 in RA
Investment in research programs aimed at the creation of future
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® (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® (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
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 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.
VRTX - GEN
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