-Full-year 2016 product revenues of
approximately $703 million for KALYDECO and $979 million for
ORKAMBI; total 2016 CF product revenues of $1.68 billion compared
to $983 million in 2015-
-Company provides 2017 financial guidance for
KALYDECO product revenues of $690 to $710 million and ORKAMBI
product revenues of $1.1 to $1.3 billion-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
provided an update on its business performance, including
preliminary financial results for 2016 and a financial outlook for
2017, and an update on its ongoing research and development
programs. Jeffrey Leiden, M.D., Ph.D., Chairman, President and
Chief Executive Officer of Vertex, will discuss these updates as
part of a webcast presentation at the 35th Annual J.P. Morgan
Healthcare Conference in San Francisco on Monday, January 9 at 9:30
a.m. PT (12:30 p.m. ET). The presentation will be available on
Vertex’s website, www.vrtx.com.
“In 2016, the number of people with cystic fibrosis treated with
ORKAMBI and KALYDECO increased significantly and we advanced our
broad pipeline of medicines in development for CF,” said Dr.
Leiden. “Entering 2017, we expect to continue to increase the
number of people treated with our medicines and to generate
important data from multiple medicines across our CF pipeline. Our
progress has positioned us well to reach our long-term goal of
treating all patients with CF with medicines that treat the
underlying cause of the disease.”
2016 Financial Highlights and 2017
Financial Outlook
“We have seen total CF product revenues grow from $983 million
in 2015 to $1.68 billion in 2016, and we anticipate revenue growth
in 2017 and beyond,” said Ian Smith, Executive Vice President,
Chief Operating Officer and Chief Financial Officer. “A key driver
of continued revenue growth in 2017 will be to treat more patients
with ORKAMBI by completing multiple reimbursement agreements in
Europe and treating children ages 6 to 11 in the U.S.”
The company will announce its complete fourth quarter and
full-year 2016 financial results on January 25, 2017 and today
provided preliminary 2016 selected financial results, as summarized
below:
Preliminary 2016 Selected Financial
Results*
Fourth-Quarter
2016
Full-year2016
ORKAMBI **
$276M
$979M
KALYDECO
$177M
$703M
TOTAL CF PRODUCT
REVENUES
$453M
$1.68B
For the full year 2016, Vertex expects to report combined GAAP
R&D and SG&A expenses of approximately $1.48 billion and
non-GAAP R&D and SG&A expenses of approximately $1.21
billion.
* The above preliminary financial results are unaudited and
are provided as approximations in advance of the company's complete
financial results announcement on January 25, 2017.
** 2016 ORKAMBI revenues do not include any revenues from
France. In France, approximately 1,000 of the 1,500 eligible
patients have initiated therapy as of the end of 2016.
Approximately €70 million was collected through early access
programs in France during 2016, and approximately €30 million of
these funds was collected in the fourth quarter of 2016.
Vertex expects that revenues from these early access programs
will be recognized in the period that a formal reimbursement
agreement in France is reached based on the terms of such
agreement.
The company entered 2017 with approximately $1.43 billion in
cash, cash equivalents and marketable securities. As of December
31, 2016, Vertex had $300 million outstanding from a credit
agreement.
Vertex today provided full-year 2017 net product revenue
guidance for KALYDECO and ORKAMBI, and guidance for combined
non-GAAP R&D and SG&A expenses, as summarized below:
- KALYDECO: Vertex anticipates
full-year 2017 global KALYDECO net product revenues of $690 to $710
million.
- ORKAMBI: The company anticipates
full-year 2017 ORKAMBI net product revenues of $1.1 to $1.3
billion. This range includes an estimate of potential additional
European revenues in 2017 that is largely dependent on which
European countries complete reimbursement agreements in 2017 and
when these agreements become effective. The company expects
first-quarter 2017 ORKAMBI net product revenues to be similar to
fourth-quarter 2016 ORKAMBI net product revenues.
- Combined Non-GAAP R&D and
SG&A Expenses: Vertex expects that its combined non-GAAP
R&D and SG&A expenses in 2017 will be in the range of $1.25
to $1.30 billion. The increase as compared to 2016 primarily
reflects increased costs related to ongoing and planned CF
development efforts and global commercial support for ORKAMBI and
KALYDECO.
Approved Medicines for
CF
ORKAMBI
Planned submission for approval to treat
children ages 6 to 11 in the EU: On November 7, 2016, Vertex
announced that a Phase 3 study evaluating ORKAMBI in children ages
6 through 11 who have two copies of the F508del mutation met its
primary endpoint of absolute change in lung clearance index
(LCI2.5) through 24 weeks of treatment. Based on these data, Vertex
plans to submit a Marketing Authorization Application (MAA) line
extension to the European Medicines Agency (EMA) in the first half
of 2017 for approval of ORKAMBI in children ages 6 through 11.
There are approximately 3,400 children ages 6 through 11 who have
two copies of the F508del mutation in Europe.
Phase 3 study in children ages 2 to 5:
Vertex is currently conducting a Phase 3 study of ORKAMBI in
children ages 2 through 5 who have two copies of the F508del
mutation. Enrollment of the study is expected to be complete in
mid-2017.
KALYDECO
Phase 3 study in children under two years
of age: Vertex is currently conducting a Phase 3 study
evaluating the safety of KALYDECO in children under 2 years of age.
The study is enrolling infants with one of the 10 mutations for
which KALYDECO is currently approved and will evaluate the effect
of KALYDECO on markers of CF in young children. The study will
utilize a weight-based dose of KALYDECO granules that can be mixed
in soft foods or liquids.
Medicines in Development for
CF
Tezacaftor (VX-661)
In the first half of 2017, Vertex expects to
obtain data from Phase 3 studies of tezacaftor to support the
planned submission of a New Drug Application (NDA) to the U.S. Food
and Drug Administration (FDA) in the second half of 2017 for
tezacaftor in combination with ivacaftor. The Phase 3 studies
include:
Two Copies of the F508del Mutation:
Enrollment is complete in a study evaluating 24 weeks of treatment
with tezacaftor in combination with ivacaftor in approximately 500
people with CF who have two copies of the F508del mutation.
One Copy of the F508del Mutation and a
Second Mutation that Results in Residual CFTR Function:
Enrollment is complete in a study evaluating tezacaftor in
combination with ivacaftor in approximately 200 people with
residual function mutations. This crossover study includes two
8-week dosing periods, separated by an 8-week washout period. The
study includes an arm of ivacaftor monotherapy, in addition to an
arm evaluating tezacaftor in combination with ivacaftor and a
placebo arm.
One Copy of the F508del Mutation and One
Copy of a Mutation that Results in Minimal CFTR Protein
Function: The planned NDA submission will include safety data
from a Phase 3 study of tezacaftor and ivacaftor in people with one
copy of the F508del mutation and one copy of a mutation that
results in minimal CFTR protein function. As previously announced,
this study was discontinued in mid-2016 based on a planned interim
futility analysis that showed the combination of tezacaftor and
ivacaftor did not result in a pre-specified improvement in lung
function.
One Copy of the F508del Mutation and a
Second Mutation that Results in a Gating Defect in the CFTR
Protein: In the first half of 2017, Vertex expects to complete
enrollment in a study evaluating tezacaftor in combination with
ivacaftor in people with one copy of the F508del mutation and a
second mutation that results in a gating defect in the CFTR protein
that has been shown to be responsive to ivacaftor alone. The study
is evaluating 8 weeks of treatment with tezacaftor in combination
with ivacaftor. Data from this study are not expected to be part of
the initial regulatory submissions planned for
tezacaftor/ivacaftor.
Phase 3 study in children ages 6 to
11: Vertex is currently conducting a Phase 3 open-label study
evaluating the safety and tolerability of tezacaftor in combination
with ivacaftor in children ages 6 through 11 with two copies of the
F508del mutation, and in children ages 6 through 11 with one copy
of the F508del mutation and one copy of a mutation that has been
clinically demonstrated to be ivacaftor responsive, including
gating and residual function mutations.
Next-Generation Correctors
Vertex expects to have four different
triple-combination regimens in Phase 1 or 2 clinical development
during the first quarter of 2017. Clinical data in CF patients for
three of these regimens are expected in the second half of
2017.
Dosing is underway in two Phase 2 studies
evaluating the next-generation correctors VX-440 and VX-152 in
triple combination regimens with tezacaftor and ivacaftor in people
with CF. The Phase 2 study of VX-440 is designed to evaluate the
safety and efficacy of 4-week dosing of VX-440 in combination with
tezacaftor and ivacaftor in approximately 40 people with CF who
have one F508del mutation and one minimal function mutation and
approximately 25 people with two copies of the F508del mutation.
The Phase 2 study of VX-152 will evaluate 2 weeks of triple
combination dosing in approximately 35 people with CF who have one
F508del mutation and one minimal function mutation and
approximately 25 people with two copies of the F508del mutation.
Both VX-440 and VX-152 have received Fast Track designation from
the FDA.
The first data from these studies are
expected in the second half of 2017. These data are intended to
support the initiation of Phase 3 development for VX-440 and of a
longer-duration Phase 2b or registrational program for VX-152.
As part of the company’s strategy to develop
multiple next-generation correctors, Vertex is also developing the
additional next-generation correctors VX-659 and VX-445. Dosing is
now underway for a Phase 1 study of VX-659 in healthy volunteers,
and dosing in CF patients is planned in the first half of 2017. The
Phase 1 study of VX-659 will evaluate single ascending doses,
multiple ascending doses and triple combination dosing in healthy
volunteers, and includes an arm to evaluate triple combination
dosing in CF patients who have one F508del mutation and one minimal
function mutation. Dosing of a fourth next generation corrector,
VX-445, is expected to begin in the first quarter of 2017. Pending
data from both Phase 1 studies, Vertex plans to begin Phase 2
development for one or both of these next-generation correctors in
the second half of 2017.
VX-371 (ENaC inhibitor)
Phase 2 study of VX-371 in combination
with ORKAMBI ongoing: Enrollment is ongoing in a study
evaluating VX-371 in combination with ORKAMBI, both with and
without the addition of hypertonic saline, in patients with CF ages
12 and older who have two copies of the F508del mutation. The
primary endpoints of this study are safety and mean absolute change
from baseline in FEV1 at day 28 compared to placebo. Data are
expected in the second half of 2017.
Ongoing Research and Development
Programs in Other Diseases
In addition to clinical development programs focused on CF,
Vertex has ongoing development programs for potential medicines
aimed at other serious and life-threatening diseases, including
VX-371 for the treatment of primary ciliary dyskinesia (PCD),
VX-210 for the treatment of acute cervical spinal cord injury and
VX-150 for the treatment of pain. Additionally, Vertex is
evaluating three compounds designed to inhibit DNA repair pathways
that are fundamental to the survival and proliferation of certain
cancers, including the lead compound, VX-970, an ATR inhibitor
being evaluated in 10 ongoing Phase 1 and 2 studies, VX-803, a
second ATR inhibitor, and VX-984, an inhibitor of DNA-dependent
protein kinase that also targets the DNA damage repair system.
Non-GAAP Financial
Measures
In this press release, Vertex's financial results and financial
guidance are provided in accordance with accounting principles
generally accepted in the United States (GAAP) and using certain
non-GAAP financial measures. In particular, the combined non-GAAP
R&D and SG&A expenses and guidance exclude stock-based
compensation expense, expenses related to variable interest
entities and certain payments related to business development
activities included in research expenses. This information is
provided as a complement to results provided in accordance with
GAAP because management believes these non-GAAP financial measures
help indicate underlying trends in the company's business, are
important in comparing current results with prior period results
and provide additional information regarding the company's
financial position. Management also uses these non-GAAP financial
measures to establish budgets and operational goals that are
communicated internally and externally and to manage the company's
business and to evaluate its performance. The company is not
providing guidance regarding 2017 GAAP R&D and SG&A
expenses because of the difficulty of estimating stock-based
compensation expenses, costs associated with variable interest
entities and predicting whether or not there will be additional
expense items for which adjustments are appropriate, including for
example adjustments with respect to business development
activities. A reconciliation of the 2016 GAAP financial results to
2016 non-GAAP financial results is included below:
Preliminary Reconciliation of Non-GAAP
Information
Twelve Months
Ended
December 31,
2016
Combined GAAP R&D and SG&A expenses
$1.48B
Adjustments*
($0.27B)
Combined non-GAAP R&D and SG&A expenses*
$1.21B
* Adjustments include stock-based compensation expense,
expenses related to variable interest entities and certain payments
related to business development activities included in research
expenses, and other adjustments.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR
KALYDECO® (ivacaftor)
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 2 years and older
who have one of the following mutations in their CF gene: G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or
R117H. KALYDECO is not for use in people with CF due to other
mutations in the CF gene. KALYDECO is not effective in patients
with CF with two copies of the F508del mutation (F508del/F508del)
in the CF gene. It is not known if KALYDECO is safe and effective
in children under 2 years of age.
Patients should not take KALYDECO if they are taking certain
medicines or herbal supplements such as: the antibiotics
rifampin or rifabutin; seizure medications such as phenobarbital,
carbamazepine, or phenytoin; or St. John’s wort.
Before taking KALYDECO, patients should tell their doctor if
they: have liver or kidney problems; drink grapefruit juice, or
eat grapefruit or Seville oranges; are pregnant or plan to become
pregnant because it is not known if KALYDECO will harm an unborn
baby; and are breastfeeding or planning to breastfeed because is
not known if KALYDECO passes into breast milk.
KALYDECO may affect the way other medicines work, and other
medicines may affect how KALYDECO works. Therefore the dose of
KALYDECO may need to be adjusted when taken with certain
medications. Patients should especially tell their doctor if they
take antifungal medications such as ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it.
Patients should not drive a car, use machinery, or do anything that
needs them to be alert until they know how KALYDECO affects them.
Patients should avoid food containing grapefruit or Seville oranges
while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood have been reported in
patients receiving KALYDECO. The patient’s doctor will do blood
tests to check their liver before starting KALYDECO, every 3 months
during the first year of taking KALYDECO, and every year while
taking KALYDECO. For patients who have had high liver enzymes in
the past, the doctor may do blood tests to check the liver more
often. Patients should call their doctor right away if they have
any of the following symptoms of liver problems: pain or discomfort
in the upper right stomach (abdominal) area; yellowing of their
skin or the white part of their eyes; loss of appetite; nausea or
vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving KALYDECO. The patient’s doctor
should perform eye examinations prior to and during treatment with
KALYDECO to look for cataracts. The most common side effects
include headache; upper respiratory tract infection (common cold),
which includes sore throat, nasal or sinus congestion, and runny
nose; stomach (abdominal) pain; diarrhea; rash; nausea; and
dizziness.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing
Information for KALYDECO (ivacaftor).
INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor) TABLETS
ORKAMBI is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients age 6 years and older who have two
copies of the F508del mutation (F508del/F508del) in their CFTR
gene. ORKAMBI should only be used in these patients. It is not
known if ORKAMBI is safe and effective in children under 6 years of
age.
Patients should not take ORKAMBI if they are taking certain
medicines or herbal supplements, such as: the antibiotics
rifampin or rifabutin; the seizure medicines phenobarbital,
carbamazepine, or phenytoin; the sedatives/anti-anxiety medicines
triazolam or midazolam; the immunosuppressant medicines everolimus,
sirolimus, or tacrolimus; or St. John’s wort.
Before taking ORKAMBI, patients should tell their doctor if
they: have or have had liver problems; have kidney problems;
have had an organ transplant; are using birth control (hormonal
contraceptives, including oral, injectable, transdermal or
implantable forms). Hormonal contraceptives should not be used as a
method of birth control when taking ORKAMBI. Patients should tell
their doctor if they are pregnant or plan to become pregnant (it is
unknown if ORKAMBI will harm the unborn baby) or if they are
breastfeeding or planning to breastfeed (it is unknown if ORKAMBI
passes into breast milk).
ORKAMBI may affect the way other medicines work and other
medicines may affect how ORKAMBI works. Therefore, the dose of
ORKAMBI or other medicines may need to be adjusted when taken
together. Patients should especially tell their doctor if they
take: antifungal medicines such as ketoconazole, itraconazole,
posaconazole, or voriconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
When taking ORKAMBI, patients should tell their doctor if
they stop ORKAMBI for more than 1 week as the doctor may need to
change the dose of ORKAMBI or other medicines the patient is
taking. It is unknown if ORKAMBI causes dizziness. Patients should
not drive a car, use machinery, or do anything requiring alertness
until the patient knows how ORKAMBI affects them.
ORKAMBI can cause serious side effects including:
High liver enzymes in the blood, which can be a sign of liver
injury, have been reported in patients receiving ORKAMBI. The
patient’s doctor will do blood tests to check their liver before
they start ORKAMBI, every three months during the first year of
taking ORKAMBI, and annually thereafter. The patient should call
the doctor right away if they have any of the following symptoms of
liver problems: pain or discomfort in the upper right stomach
(abdominal) area; yellowing of the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine; or confusion.
Respiratory events such as shortness of breath or chest
tightness were observed in patients when starting ORKAMBI. If a
patient has poor lung function, their doctor may monitor them more
closely when starting ORKAMBI.
An increase in blood pressure has been seen in some patients
treated with ORKAMBI. The patient’s doctor should monitor their
blood pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving ORKAMBI and ivacaftor, a
component of ORKAMBI. For children and adolescents, the
patient’s doctor should perform eye examinations prior to and
during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include: shortness of
breath and/or chest tightness; upper respiratory tract infection
(common cold), including sore throat, stuffy or runny nose;
gastrointestinal symptoms including nausea, diarrhea, or gas; rash;
fatigue; flu or flu-like symptoms; increase in muscle enzyme
levels; and irregular, missed, or abnormal menstrual periods and
heavier bleeding.
Please click here to see the full Prescribing
Information for ORKAMBI.
About Vertex
Vertex is a global biotechnology company that aims to discover,
develop and commercialize innovative medicines so people with
serious diseases can lead better lives. In addition to our clinical
development programs focused on cystic fibrosis, Vertex has more
than a dozen ongoing research programs aimed at other serious and
life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research
and development sites and commercial offices in the United States,
Europe, Canada and Australia. For seven years in a row, Science
magazine has named Vertex one of its Top Employers in the life
sciences. For additional information and the latest updates from
the company, please visit www.vrtx.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 paragraph of the press release, the information provided in
the section captioned "2016 Financial Highlights and 2017 Financial
Outlook" and statements regarding (i) preliminary financial
information for the quarter and year ended December 31, 2016 and
guidance for 2017; (ii) the timing and amount of ORKAMBI revenue
that may be recognized from France; (iii) the timing of regulatory
applications, including MAAs and NDAs and (iv) the expected timing,
clinical trial designs and results for ongoing and planned clinical
studies of ORKAMBI, KALYDECO, tezacaftor, VX-440, VX-152, VX-659,
VX-445 and VX-371 and clinical studies related to VX-210, VX-150,
VX-970, VX-803 and VX-984. 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 2016 financial results are
preliminary and subject to adjustment, that the company's
expectations regarding its 2017 revenues and expenses may be
incorrect (including because one or more of the company's
assumptions underlying its expectations may not be realized), that
data from the company's development programs may not support
registration or further development of its compounds due to safety,
efficacy or other reasons, 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.
Webcast Information
The company will webcast its corporate presentation at the 35th
Annual J.P.Morgan Healthcare Conference on Monday, January 9 at
9:30 a.m. PT (12:30 p.m. ET). The audio portion of management’s
remarks can be accessed live through Vertex’s website at
www.vrtx.com in the “Investors” section under the “Events and
Presentations” page.
(VRTX-GEN)
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version on businesswire.com: http://www.businesswire.com/news/home/20170108005029/en/
Vertex Pharmaceuticals IncorporatedInvestors:Michael
Partridge, 617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.com
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