-First quarter 2016 cystic fibrosis product
revenues of $394 million; $223 million for ORKAMBI®
(lumacaftor/ivacaftor) and $171 million for KALYDECO®
(ivacaftor)-
-Provides 2016 guidance for ORKAMBI product
revenues of $1.0 to $1.1 billion; increases 2016 guidance for
KALYDECO product revenues to $685 to $705 million-
-Provides update on approved CF medicines and
pipeline of investigational medicines-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
reported consolidated financial results for the quarter ended
March 31, 2016 and provided an update on its approved CF
medicines and other investigational medicines. Vertex also provided
financial guidance for 2016 ORKAMBI® net revenues and increased its
prior guidance for 2016 KALYDECO® net revenues. Key financial
results include:
Three Months Ended March 31,
2016 2015 % Change (in millions, except
per share and percentage data)
ORKAMBI product revenues, net
$ 223 $ — N/A
KALYDECO product revenues, net $
171
$
130
31%
TOTAL CF product revenues, net $
394
$
130
202%
GAAP net loss $ (42 ) $ (199 ) (79)%
GAAP net
loss per share $ (0.17 ) $ (0.83 ) (80)%
Non-GAAP net income
(loss) $ 22 $ (148 ) N/A
Non-GAAP net income (loss) per
share $ 0.09 $ (0.62 ) N/A
"2016 marks an important transition for Vertex following the
launch of ORKAMBI. With recent approvals and label expansions,
there are now approximately 27,000 people with CF eligible to take
ORKAMBI or KALYDECO. The number of CF patients eligible for and
initiating treatment is driving significant revenue growth for a
second straight year, and we expect this trend to continue in 2017
and beyond," said Jeffrey Leiden, M.D., Ph.D., Chairman, President
and Chief Executive Officer of Vertex. “In addition, we believe our
portfolio of approved and pipeline medicines has the potential to
treat the vast majority of people with CF. We remain focused on
investing to create transformative future medicines and generating
continued earnings growth in the years ahead.”
CF Medicines and Pipeline
Update
Vertex today provided the following updates for ORKAMBI,
KALYDECO and the company’s progress toward developing new medicines
with the goal of treating all people with CF:
ORKAMBI
Additional Regulatory Approvals Support
Expansion Efforts: During the first quarter of 2016, Vertex
received regulatory approval for ORKAMBI for the treatment of
people with CF ages 12 and older who have two copies of the F508del
mutation in Canada and Australia, where together there are
approximately 2,500 people who are eligible for treatment with
ORKAMBI. Vertex has now begun the reimbursement approval process in
these countries.
Supplemental New Drug Application in
Children Ages 6 to 11: In late March 2016, Vertex submitted a
supplemental New Drug Application (sNDA) to the FDA for the
approval of ORKAMBI for treatment of children with CF ages 6 to 11
who have two copies of the F508del mutation. The submission
included a request for Priority Review, which if granted would
shorten the FDA's anticipated review time from 10 to six months.
There are approximately 2,400 children ages 6 to 11 who have two
copies of the F508del mutation in the U.S.
Vertex has submitted data from an open label
Phase 3 clinical safety study of ORKAMBI in children ages 6 to 11
who have two copies of the F508del mutation for presentation at the
39th European Cystic Fibrosis Society Conference (ECFS), June 8 -
11 in Basel, Switzerland.
Enrollment Complete in Phase 3 Study of
Children Ages 6 to 11: Vertex has completed enrollment in a
six-month Phase 3 efficacy study evaluating ORKAMBI in
approximately 200 children ages 6 to 11 who have two copies of the
F508del mutation. Pending data from the study, Vertex plans to
submit a Marketing Authorization Application (MAA) variation in
Europe in the first half of 2017 for approval of ORKAMBI for use in
this age group. There are approximately 3,400 children ages 6 to 11
who have two copies of the F508del mutation in the European
Union.
KALYDECO
Study in Children Under Two Years of
Age: Vertex has initiated a Phase 3 clinical study of KALYDECO
in children under 2 years of age to 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. The study will enroll infants with one of the 10
mutations for which KALYDECO is currently approved.
Regulatory Filing for Patients with
Residual Function Mutations: In October 2015, Vertex submitted
an sNDA for approval of KALYDECO for treatment of people with CF
ages 2 and older who have one of 23 residual function mutations.
The company is in ongoing discussions with the FDA regarding a
Complete Response Letter it received in February 2016. There are
approximately 1,500 people ages 2 and older in the U.S. who have
one of the 23 residual function mutations included in the sNDA.
Pipeline of Investigational Medicines
for CF
VX-661 - Broad Phase 3 program ongoing in multiple groups of
people with CF
Vertex provided the following updates on the
Phase 3 studies of the investigational combination of VX-661 and
ivacaftor in multiple different groups of people with CF who have
at least one copy of the F508del mutation:
- Enrollment in the study in people with
two copies of the F508del mutation is expected to be complete in
mid-2016, and data from this Phase 3 study are expected in early
2017.
- Enrollment is ongoing in the Phase 3
study of VX-661 in combination with ivacaftor in patients with one
copy of the F508del mutation and a second mutation that results in
a gating defect. Vertex plans to complete enrollment of this study
in late 2016 or early 2017.
- Vertex has revised its enrollment
target for the Phase 3 study of VX-661 in combination with
ivacaftor in patients with one copy of the F508del mutation and a
second mutation that results in residual CFTR function. The
original expectation was for up to 300 patients to enroll in this
study. Vertex now plans to enroll approximately 200 patients.
Enrollment is expected to be complete in the second half of
2016.
- Enrollment is complete in Part A of the
study in people with one copy of the F508del mutation and a second
mutation that results in minimal CFTR function. An interim futility
analysis of efficacy data from Part A of this study is expected to
be completed in the third quarter of 2016.
In addition to evaluating the efficacy of the
combination regimen, these Phase 3 studies will also provide safety
data on the combination of VX-661 and ivacaftor to support the
planned development of a triple combination regimen that includes a
next-generation corrector in combination with VX-661 and
ivacaftor.
VX-371 - Enrollment ongoing in Phase 2 study of VX-371 in
combination with ORKAMBI
Vertex today announced data from an
exploratory Phase 2, 14-day study of its inhaled epithelial sodium
channel (ENaC) inhibitor, VX-371 (P-1037), being developed in
collaboration with Parion Sciences. The study dosed 142 people ages
12 and older with a confirmed diagnosis of CF. There was no
restriction based on CFTR mutation. 136 people completed the study.
Patients were not using any CFTR modulator therapy immediately
before or during the study. The primary endpoint of the study was
safety compared to placebo. Secondary endpoints evaluated the
effect on mean absolute forced expiratory volume in one second
(FEV1) and patient-reported respiratory symptoms as reported in the
CF questionnaire-revised (CFQ-R). The study met its primary safety
endpoint, and safety data from the study showed that VX-371 was
generally well tolerated. There were no statistically significant
changes in FEV1 or CFQ-R for those who received VX-371.
The clinical safety data announced today
provide support for the company's ongoing placebo-controlled Phase
2a study evaluating VX-371 in combination with ORKAMBI, both with
and without the addition of hypertonic saline. This study is
expected to enroll approximately 150 people with CF ages 12 and
older who have two copies of the F508del mutation. The primary
endpoints of the Phase 2a study are safety and mean absolute change
from baseline in FEV1 at day 28 compared to placebo.
In vitro, VX-371 showed a meaningful change
in cilia beat frequency when VX-371 was used in combination with
ORKAMBI in human bronchial epithelial cells with two copies of the
F508del mutation, but did not show a meaningful change in cilia
beat frequency when VX-371 was used alone.
Next-Generation Correctors - Phase 1 studies in healthy
volunteers progressing as planned
In the fourth quarter of 2015, Vertex
initiated clinical development of two next-generation correctors
known as VX-152 and VX-440. Both VX-152 and VX-440 are being
evaluated alone and as part of a triple combination with VX-661 and
ivacaftor in ongoing Phase 1 studies in healthy volunteers.
Pending successful completion of the Phase 1
studies of VX-152 and VX-440, the company expects to begin Phase 2
proof-of-concept studies in combination with VX-661 and ivacaftor
in the second half of 2016.
CRISPR Collaboration - Gene editing collaboration focused on
discovering potential treatments to address the mutations and genes
known to cause and contribute to CF
In October 2015, Vertex entered into a
strategic research collaboration with CRISPR Therapeutics focused
on the use of CRISPR's gene editing technology, known as
CRISPR-Cas9, to discover and develop potential new treatments aimed
at the underlying genetic causes of human disease. The
collaboration will evaluate the use of CRISPR-Cas9 across multiple
diseases where targets have been validated through human genetics.
As part of the collaboration, Vertex and CRISPR will evaluate the
use of CRISPR-Cas9 to potentially correct the mutations in the CFTR
gene known to result in the defective protein that causes CF and to
edit other genes that contribute to the disease.
Other Research and Development
Programs
Beyond CF, Vertex is advancing research and development programs
focused on the treatment of key mechanisms in serious diseases. The
company today provided the following updates to its pipeline
programs:
Oncology
VX-970: VX-970 is an inhibitor of ATR,
a critical regulator of the DNA damage repair system. Vertex
presented data from a Phase 1 trial of VX-970 in combination with
cisplatin in patients with advanced solid tumors at the Annual
Association for Cancer Research (AACR) meeting on April 17,
2016.
Vertex is currently conducting two Phase 1/2
studies that are enrolling specific cohorts of triple-negative
breast cancer patients and non-small cell lung cancer patients. In
these studies, VX-970 is being dosed in combination with commonly
used DNA-damaging repair therapies.
Vertex has also entered into two cooperative
research and development agreements (CRADAs) with the National
Cancer Institute to support evaluation of VX-970 across other types
of cancers. The CRADA enables NCI to conduct multiple clinical
studies that will evaluate treatment with VX-970 in people with
small cell lung, head and neck, bladder, ovarian and other
cancers.
Pain
VX-150: Vertex is developing VX-150 as
a potential medicine for the treatment of pain. VX-150 is designed
to block pain signaling through inhibition of a sodium channel
known as NaV 1.8. In the first quarter of 2016, Vertex initiated a
six-week crossover Phase 2 proof-of-concept study of VX-150 in
approximately 100 people with symptomatic osteoarthritis of the
knee. Vertex expects to complete enrollment of this study in the
second half of 2016.
Acute Spinal Cord Injury
VX-210: In the first quarter of 2016,
Vertex initiated a randomized, double-blind, placebo controlled
Phase 2b/3 study to evaluate the efficacy and safety of VX-210 in
patients with certain acute cervical spinal cord injuries. Vertex
is developing VX-210 as a potential medicine for acute spinal cord
injury. VX-210 is designed to inhibit a protein known as Rho that
blocks neural regeneration after injury.
First Quarter 2016 Financial
Highlights
Revenues:
- Net product revenues from ORKAMBI were
$223.1 million. ORKAMBI was launched in the U.S. in July 2015.
- Net product revenues from KALYDECO were
$170.5 million, compared to $130.2 million for the first quarter of
2015.
Expenses:
- Non-GAAP research and development
(R&D) expenses were $222.0 million compared to $177.2 million
for the first quarter of 2015. The increase was primarily driven by
increased investment to progress our portfolio of CF medicines.
GAAP R&D expenses, including stock-based compensation expense,
were $255.9 million compared to $215.6 million for the first
quarter of 2015.
- Non-GAAP sales, general and
administrative (SG&A) expenses were $83.7 million compared to
$69.1 million for the first quarter of 2015. The increase was
primarily driven by increased investment to support the global
launch of ORKAMBI. GAAP SG&A expenses, including stock-based
compensation expense, were $105.2 million compared to $85.9 million
for the first quarter of 2015.
Net Income (Loss) Attributable to Vertex:
- Non-GAAP net income was $22.4 million,
or $0.09 per diluted share, compared to a non-GAAP net loss of
$148.4 million, or $0.62 per diluted share, for the first quarter
of 2015. The GAAP net loss, including stock-based compensation
expense, was $41.6 million, or $0.17 per diluted share, compared to
Vertex's GAAP net loss of $198.6 million, or $0.83 per diluted
share, for the first quarter of 2015.
Cash Position:
- As of March 31, 2016, Vertex had
$1.03 billion in cash, cash equivalents and marketable securities
compared to $1.04 billion in cash, cash equivalents and marketable
securities as of December 31, 2015.
- As of March 31, 2016, Vertex had
$300 million outstanding from a credit agreement, repayable by the
end of the third quarter of 2017. The agreement allows for the
facility to increase to up to $500 million.
2016 Financial Guidance:
Vertex today provided 2016 revenue guidance for ORKAMBI and
increased 2016 revenue guidance for KALYDECO. The company also
reiterated guidance for its 2016 combined non-GAAP R&D and
SG&A expenses. The guidance is summarized below:
ORKAMBI: The company anticipates total
2016 product revenues for ORKAMBI of $1.0 to $1.1 billion. This
guidance is based on the company's understanding of treatment
patterns from the launch of ORKAMBI to date, including:
- Uptake: Approximately 65% of the
8,500 eligible patients in the U.S. have initiated treatment as of
March 31, 2016. Vertex continues to expect the vast majority of
eligible patients ages 12 and older in the U.S. will initiate
treatment by the end of 2016.
- Persistence: Of the patients who
have started on treatment, approximately 15% discontinued treatment
within the first three months of initiation. The company projects
that the proportion of all patients who initiate and remain on
treatment will stabilize at approximately 70% to 80%.
- Compliance: The overall
compliance rate, which reflects the number of pills actually taken
by a patient in a given month, is expected to be between 70% to
80%.
2016 ORKAMBI guidance also reflects potential
revenues from the anticipated approval of ORKAMBI in the U.S. for
the treatment of people ages 6 to 11 who have two copies of the
F508del mutation in the second half of 2016 and revenues from sales
of ORKAMBI outside the U.S., primarily in Germany.
KALYDECO: Vertex today increased its
guidance for 2016 revenues of KALYDECO. The company now expects
product revenues of $685 to $705 million. The prior range, provided
on January 10, 2016, was for KALYDECO product revenues of $670 to
$690 million for 2016.
The change in KALYDECO guidance reflects:
- A continued increase in the number of
patients initiating treatment with KALYDECO globally
- A reduced impact from the VX-661 Phase
3 program
2016 guidance for KALYDECO currently excludes
any revenues related to the potential approval of KALYDECO for
people in the U.S. who have residual function mutations.
Operating Expenses, Excluding Cost of
Revenues (Combined Non-GAAP R&D and SG&A Expenses):
Vertex continues to expect that its combined non-GAAP R&D and
SG&A expenses in 2016 will be in the range of $1.18 to $1.23
billion. Vertex's expected non-GAAP R&D and SG&A expenses
exclude stock-based compensation expense and certain other
expenses.
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, non-GAAP financial
results exclude stock-based compensation expense, costs and credits
related to the relocation of the company's corporate headquarters
and hepatitis C-related revenues and costs and other adjustments.
These results are 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. A
reconciliation of the GAAP financial results to non-GAAP financial
results is included in the attached financial information.
Vertex Pharmaceuticals
Incorporated
First Quarter Results
Consolidated Statements of Operations
Data
(in thousands, except per share
amounts)
(unaudited)
Three Months Ended March 31, 2016
2015 Revenues: Product revenues, net $ 394,410 $ 130,875
Royalty revenues 3,596 6,792 Collaborative revenues 74 842
Total revenues 398,080 138,509 Costs and expenses: Cost of
product revenues (Note 1) 49,789 9,381 Royalty expenses 860 2,926
Research and development expenses 255,860 215,599 Sales, general
and administrative expenses 105,214 85,860 Restructuring expenses
(income) 687 (3,272 ) Total costs and expenses 412,410
310,494 Loss from operations (14,330 ) (171,985 )
Interest expense, net (20,698 ) (21,307 ) Other income (expenses),
net 4,411 (5,113 ) Loss from operations before provision for
income taxes (30,617 ) (198,405 ) Provision for income taxes 5,485
299 Net loss (36,102 ) (198,704 ) (Income) loss
attributable to noncontrolling interest (5,529 ) 98 Net loss
attributable to Vertex $ (41,631 ) $ (198,606 ) Amounts per
share attributable to Vertex common shareholders: Net loss: Basic
and diluted $ (0.17 ) $ (0.83 ) Shares used in per share
calculations: Basic and diluted 243,831 239,493
Reconciliation of GAAP to Non-GAAP Net
Income/(Loss)
First Quarter Results
(in thousands, except per share
amounts)
(unaudited)
Three Months Ended March 31, 2016
2015 GAAP loss attributable to Vertex $ (41,631 ) $
(198,606 ) Stock-based compensation expense 55,472 57,384 Real
estate restructuring costs and income (Note 2) 436 (3,567 ) HCV
related revenues and costs (Note 3) (1,436 ) (4,469 ) Other
adjustments (Notes 4 and 5) 9,581 882
Non-GAAP net
income (loss) attributable to Vertex $ 22,422 $ (148,376
) Amounts per diluted share attributable to Vertex common
shareholders: GAAP $ (0.17 ) $ (0.83 ) Non-GAAP $ 0.09 $ (0.62 )
Shares used in diluted per share calculations: GAAP 243,831 239,493
Non-GAAP 246,680 239,493
Reconciliation of GAAP to Non-GAAP
Revenues and Expenses
First Quarter Results
(in thousands)
(unaudited)
Three Months Ended March 31, 2016
2015 GAAP total revenues $ 398,080 $ 138,509 HCV
related revenues (Note 3) (851 ) (2,869 ) Other adjustments (Note
4) (74 ) (200 )
Non-GAAP total revenues $ 397,155 $
135,440
Three Months Ended March 31,
2016 2015 GAAP cost of product revenues and
royalty expenses $ 50,649 $ 12,307 HCV related costs (Note 3)
(139 ) (1,596 )
Non-GAAP cost of product revenues and royalty
expenses $ 50,510 $ 10,711
GAAP research and
development expenses $ 255,860 $ 215,599 Stock-based
compensation expense (34,448 ) (38,217 ) HCV related costs (Note 3)
826 488 Other adjustments (Note 4) (192 ) (696 )
Non-GAAP
research and development expenses $ 222,046 $ 177,174
GAAP sales, general and administrative expenses $ 105,214 $
85,860 Stock-based compensation expense (21,024 ) (19,167 ) HCV
related costs (Note 3) 32 2,904 Other adjustments (Note 4) (543 )
(448 )
Non-GAAP sales, general and administrative expenses $
83,679 $ 69,149
Combined non-GAAP R&D and
SG&A expenses $ 305,725 $ 246,323
Three Months Ended March 31, 2016 2015
GAAP interest expense, net and other expense, net $ (16,287
) $ (26,420 ) Other adjustments (Note 4) 211 —
Non-GAAP interest expense, net and other expense, net $
(16,076 ) $ (26,420 )
GAAP provision for income taxes
$ 5,485 $ 299 Other adjustments (Note 4) (3,063 ) 63
Non-GAAP provision for income taxes $ 2,422 $ 362
Condensed Consolidated Balance Sheets
Data
(in thousands)
(unaudited)
March 31, 2016 December 31, 2015 Assets
Cash, cash equivalents and marketable securities $ 1,025,618 $
1,042,462 Restricted cash and cash equivalents (VIE) (Note 5)
76,273 78,910 Accounts receivable, net 181,878 177,639 Inventories
63,200 57,207 Property and equipment, net 690,521 697,715
Intangible assets and goodwill 334,724 334,724 Other assets 115,692
109,930
Total assets $ 2,487,906 $ 2,498,587
Liabilities and Shareholders' Equity Other
liabilities $ 397,379 $ 426,482 Deferred tax liability 112,259
110,439 Accrued restructuring expense 13,935 15,358 Deferred
revenues 23,195 26,010 Capital leases 56,178 58,468 Fan Pier lease
obligation 472,940 473,043 Senior secured term loan 295,822 295,159
Shareholders' equity 1,116,198 1,093,628
Total
liabilities and shareholders' equity $ 2,487,906 $
2,498,587 Common shares outstanding 247,287 246,307
Note 1 : Cost of product revenues includes the second and
final $13.9 million commercial milestone that was earned by CFFT in
the first quarter of 2016 related to sales of ORKAMBI.
Note 2: The company excludes from its non-GAAP income
(loss) attributable to Vertex restructuring expense (income). In
the three months ended March 31, 2016, "Real estate restructuring
costs and income" consisted of restructuring charges, related to
the company's relocation from Cambridge to Boston, Massachusetts.
In the three months ended March 31, 2015, "Real estate
restructuring costs and income" consisted of restructuring credits
of $3.6 million primarily related to the company's relocation from
Cambridge to Boston, Massachusetts.
Note 3: In the three months ended March 31, 2016 and
2015, "HCV related revenues and costs" included net product
revenues from Incivek, royalty revenues from Incivo, HCV
collaborative revenues and operating costs and expenses related to
HCV. The Company withdrew Incivek from the market in the United
States in 2014.
Note 4: In the three months ended March 31, 2016, "Other
adjustments" was primarily attributable to changes in the fair
value of contingent milestone payments and royalties payable by
Vertex to two variable interest entities ("VIEs"). In the three
months ended March 31, 2015, "Other adjustments" was primarily
attributable to development costs associated with VX-509.
Note 5: The company consolidates the financial statements
of two of its collaborators as VIEs as of March 31, 2016 and
December 31, 2015. These VIEs are consolidated because Vertex
has licensed the rights to develop the company's collaborators'
most significant intellectual property assets. The company's
interest and obligations with respect to these VIEs' assets and
liabilities are limited to those accorded to the company in its
collaboration agreements with these collaborators. Restricted cash
and cash equivalents (VIE) reflects the VIEs’ cash and cash
equivalents, which Vertex does not have any interest in and which
will not be used to fund the collaboration. Each reporting period
Vertex estimates the fair value of the contingent milestone
payments and royalties payable by Vertex to these collaborators.
Any increase in the fair value of these contingent milestone and
royalty payments results in a decrease in net income attributable
to Vertex (or an increase in net loss attributable to Vertex) on a
dollar-for-dollar basis. The fair value of contingent milestone and
royalty payments is evaluated each quarter and any change in the
fair value is reflected in the company's statement of
operations.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor) TABLETS
ORKAMBI is a combination of lumacaftor and ivacaftor indicated
for the treatment of cystic fibrosis (CF) in patients age 12 years
and older who are homozygous for the F508del mutation in the CFTR
gene. The efficacy and safety of ORKAMBI have not been established
in patients with CF other than those homozygous for the F508del
mutation.
Worsening of liver function, including hepatic encephalopathy,
in patients with advanced liver disease has been reported in some
patients with CF while receiving ORKAMBI.
Serious adverse reactions related to elevated transaminases have
been reported in patients with CF receiving ORKAMBI and, in some
instances, associated with concomitant elevations in total serum
bilirubin.
Respiratory events (e.g., chest discomfort, shortness of breath,
and chest tightness) were observed more commonly in patients during
initiation of ORKAMBI compared to those who received placebo.
Clinical experience in patients with percent predicted FEV1 < 40
is limited, and additional monitoring of these patients is
recommended during initiation of therapy.
Co-administration of ORKAMBI with sensitive CYP3A substrates or
CYP3A substrates with a narrow therapeutic index is not recommended
as ORKAMBI may reduce their effectiveness. ORKAMBI may
substantially decrease hormonal contraceptive exposure, reducing
their effectiveness and increasing the incidence of
menstruation-associated adverse reactions. Co-administration with
strong CYP3A inducers is not recommended as they may reduce the
therapeutic effectiveness of ORKAMBI.
Abnormalities of the eye lens (cataracts) have been reported in
pediatric patients treated with ivacaftor, a component of
ORKAMBI.
The most common adverse reactions associated with ORKAMBI
include shortness of breath, sore throat, nausea, diarrhea, upper
respiratory tract infection, fatigue, chest tightness, increased
blood creatinine phosphokinase, rash, flatulence, runny nose, and
influenza.
Please see the full prescribing information for ORKAMBI.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
KALYDECO® (ivacaftor)
KALYDECO is a cystic fibrosis transmembrane conductance
regulatory (CFTR) potentiator indicated for the treatment of cystic
fibrosis (CF) in patients age 2 years and older who have one of the
following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R,
G551S, S1251N, S1255P, S549N, S549R or R117H.
KALYDECO is not effective in patients with CF with 2 copies of
the F508del mutation (F508del/F508del) in the CFTR gene. The safety
and efficacy of KALYDECO in children with CF younger than 2 years
of age have not been studied. The use of KALYDECO in children under
the age of 2 years is not recommended.
High liver enzymes (transaminases; ALT and AST) have been
reported in patients with CF receiving KALYDECO.
Use of KALYDECO with medicines that are strong CYP3A inducers
substantially decreases exposure of KALYDECO and may diminish
effectiveness. Therefore, co-administration is not recommended. The
dose of KALYDECO must be adjusted when used concomitantly with
strong and moderate CYP3A inhibitors or when used in patients with
moderate or severe hepatic disease.
Cases of non-congenital lens opacities/cataracts have been
reported in pediatric patients treated with KALYDECO.
The most common side effects associated with KALYDECO include
headache; upper respiratory tract infection (common cold),
including sore throat, nasal or sinus congestion, and runny nose;
stomach (abdominal) pain; diarrhea; rash; nausea; and
dizziness.
Please see the full prescribing information for KALYDECO.
About VertexVertex 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 six 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 StatementsThis
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 Guidance” and statements regarding (i)
2016 guidance for ORKAMBI and KALYDECO, (ii) the expected timing
and clinical trial designs for ongoing and planned clinical studies
of ORKAMBI, KALYDECO, VX-661 VX-371, VX-152, VX-440, VX-970, VX-150
and VX-210, (iii) Vertex's expectations regarding uptake,
persistence and compliance with respect to ORKAMBI, (iv) the timing
of regulatory applications, including sNDAs and MAAs and the status
of interactions with regulatory authorities and (v) Vertex's plans
to submit and/or present data at scientific conferences. While
Vertex believes the forward-looking statements contained in this
press release are accurate, these forward-looking statements
represent the company's beliefs only as of the date of this press
release and 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 its
2016 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.
Conference Call and
WebcastThe company will host a conference call and
webcast today at 5:00 p.m. ET. To access the call, please dial
(866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). The
conference call will be webcast live and a link to the webcast can
be accessed through Vertex's website at www.vrtx.com in the
"Investors" section under "Events and Presentations." To ensure a
timely connection, it is recommended that users register at least
15 minutes prior to the scheduled webcast. An archived webcast will
be available on the company's website.
(VRTX-GEN)
View source
version on businesswire.com: http://www.businesswire.com/news/home/20160427006603/en/
Vertex Contacts:Investors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:617-341-6992mediainfo@vrtx.com
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Aug 2024 to Sep 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Sep 2023 to Sep 2024