-Second quarter 2015 total revenues of $166
million, including net product revenues of approximately $155
million for KALYDECO® (ivacaftor) in cystic fibrosis-
-Vertex increases guidance for total 2015
KALYDECO net revenues; now expects KALYDECO revenues of $575 to
$590 million-
-ORKAMBI™ (lumacaftor/ivacaftor) launch
underway following FDA approval on July 2-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
reported consolidated financial results for the quarter ended
June 30, 2015. Vertex also increased its financial guidance
for total 2015 KALYDECO® (ivacaftor) revenues and reiterated its
prior guidance for non-GAAP operating expenses. Key financial
results include:
Three Months Ended June 30,
2015 2014 % Change
(in millions, except per share and percentage data)
KALYDECO
product revenues, net $ 154.9 $ 113.1 37 %
GAAP net loss
$ (188.8 ) $ (159.4 ) 18 %
GAAP net loss per share $ (0.78 )
$ (0.68 ) 15 %
Non-GAAP net loss $ (130.7 ) $ (141.7 ) (8 )%
Non-GAAP net loss per share $ (0.54 ) $ (0.61 ) (11 )%
"With the recent approval of ORKAMBI and continued label and
geographic expansion for KALYDECO, we have made significant
progress toward our goals of treating many more people with cystic
fibrosis and positioning the company for long-term revenue and
earnings growth," said Jeffrey Leiden, M.D., Ph.D., Chairman,
President and Chief Executive Officer of Vertex. "As we enter the
second half of the year, we remain focused on advancing key
pipeline programs in CF including VX-661, the ENaC inhibitor VX-371
(P-1037) and our next-generation correctors, and on bringing
forward potential new medicines in multiple other diseases."
Vertex today provided the following updates:
ORKAMBI™ (lumacaftor/ivacaftor)
On July 2, 2015, the U.S. Food and Drug Administration (FDA)
approved ORKAMBI (lumacaftor/ivacaftor) for the treatment of cystic
fibrosis (CF) in people ages 12 and older with two copies of the
F508del mutation. Patients have now begun to receive ORKAMBI.
Outside of the U.S., Vertex has submitted ORKAMBI for regulatory
approval in the European Union, Australia and Canada. A decision by
the European Medicines Agency (EMA) is anticipated by the end of
2015. Reviews by Health Canada and Australia's Therapeutic Goods
Administration (TGA) are ongoing.
Studies of Lumacaftor in Combination with Ivacaftor in
Children Ages 6 to 11
Vertex is currently conducting two Phase 3 clinical studies of
lumacaftor in combination with ivacaftor in children 6 to 11 years
of age. The first study is evaluating lumacaftor in combination
with ivacaftor in approximately 50 children in the U.S. to support
the potential FDA approval in children ages 6 to 11. The primary
endpoint of this six-month study is safety. This study is fully
enrolled, and pending data, Vertex plans to submit a supplemental
New Drug Application (sNDA) to the FDA in the first half of 2016.
In Europe, an efficacy study is required to support approval in
children ages 6 to 11, and Vertex recently initiated a study to
evaluate lumacaftor in combination with ivacaftor in these patients
to support potential approval in Europe. The six-month study will
evaluate lumacaftor in combination with ivacaftor in approximately
200 children at sites in North America, Europe and Australia. The
primary endpoint of the second study is the absolute change in lung
clearance index.
KALYDECO® (ivacaftor)
Throughout the first half of 2015, Vertex completed
reimbursement discussions in multiple key countries in Europe to
enable patients with non-G551D gating mutations to receive
KALYDECO. Vertex is currently awaiting a decision on its
applications for European Union approval of KALYDECO for use in
people ages 18 and older with the R117H mutation and in children
ages 2 to 5 with one of nine gating mutations.
Phase 3 Studies Investigating VX-661 in Combination with
Ivacaftor
Vertex has initiated four 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.
The studies are evaluating VX-661 dosed as 100 mg once daily (QD)
in combination with ivacaftor dosed as 150 mg every 12 hours
(q12h). Additional details on these four studies are noted
below:
- Two Copies of the F508del
Mutation: In the first quarter of 2015, Vertex began a Phase 3
study to evaluate the combination of VX-661 and ivacaftor in people
ages 12 and older who have two copies of the F508del mutation.
Enrollment of approximately 500 patients in North America and
Europe is ongoing.
- One Copy of the F508del Mutation and
a Second Mutation that Results in a Gating Defect in the CFTR
Protein: Vertex recently began a Phase 3 study to evaluate the
combination of VX-661 and ivacaftor in people ages 12 and older who
have one copy of the F508del mutation and a second mutation that
results in a gating defect in the CFTR protein. Enrollment of
approximately 200 patients in North America and Europe is
ongoing.
- One Copy of the F508del Mutation and
a Second Mutation That Results in Residual CFTR Function:
Vertex recently began a Phase 3 study to evaluate the combination
of VX-661 and ivacaftor in people ages 12 and older who have one
copy of the F508del mutation and a second mutation that results in
residual CFTR function. This study is also evaluating ivacaftor
dosed without VX-661. Enrollment of approximately 300 patients in
North America, Europe and Australia is ongoing.
- One Copy of the F508del Mutation and
A Second Mutation That Results in Minimal CFTR Function: Vertex
today announced the initiation of a Phase 3 study to evaluate the
combination of VX-661 and ivacaftor in people ages 12 and older who
have one copy of the F508del mutation and a second mutation that
results in minimal CFTR function. The study will enroll
approximately 150 patients, and expansion of the study to an
additional approximately 150 patients will depend on an interim
futility analysis of efficacy data from the initial 150
patients.
Development of Investigational VX-371 (P-1037)
In the second quarter, Vertex and Parion Sciences entered into a
collaboration to develop investigational epithelial sodium channel
(ENaC) inhibitors, including VX-371 (P-1037), for the potential
treatment of CF and other pulmonary diseases. Parion is conducting
an exploratory Phase 2a study of inhaled VX-371 in approximately
120 people with CF. The study is enrolling people with a confirmed
diagnosis of CF and any CFTR mutation, including those who have
mutations not expected to respond to ivacaftor alone. Data from
this study are expected in mid-2016. In addition, Vertex plans to
begin a Phase 2a study to evaluate whether the addition of VX-371
to the combination of lumacaftor and ivacaftor in people with CF
who have two copies of the F508del mutation provides additional
benefit as compared to the combination of lumacaftor and ivacaftor
alone. This Phase 2a study is expected to begin in early 2016.
Second Quarter 2015 Non-GAAP Financial
Results
The non-GAAP financial results for the second quarter 2015 and
second quarter 2014 exclude stock-based compensation expense, costs
related to the relocation of the company's corporate headquarters,
hepatitis C-related revenues and costs and other adjustments.
Total Non-GAAP Revenues: Total non-GAAP revenues for the
second quarter of 2015 were $159.9 million, including $154.9
million in net product revenues from KALYDECO and $5.0 million from
royalty revenues.
- Net Product Revenues from
KALYDECO: Vertex's second quarter 2015 net product revenues
from KALYDECO were $154.9 million compared to $113.1 million for
the second quarter of 2014. The increased KALYDECO net product
revenues, compared to the second quarter of 2014, resulted
primarily from additional people being treated with KALYDECO in
both U.S. and ex-U.S. markets.
Non-GAAP Cost of Product Revenues and Royalty Expenses
(COR): Total combined non-GAAP COR expenses for the second
quarter of 2015 were $16.5 million, compared to $11.1 million for
the second quarter of 2014.
Non-GAAP Research and Development (R&D) Expenses and
Sales, General and Administrative (SG&A) Expenses: Total
combined non-GAAP R&D and SG&A expenses for the second
quarter of 2015 were $253.9 million, compared to $237.4 million for
the second quarter of 2014. The components were:
- R&D Expenses: Non-GAAP
R&D expenses were $181.9 million for the second quarter of
2015, compared to $179.5 million in non-GAAP R&D expenses for
the second quarter of 2014. The R&D expenses for the second
quarter of 2015 were similar to the second quarter of 2014 as a
result of the completion of the Phase 3 program for the combination
of lumacaftor and ivacaftor in the first half of 2014, offset by
increased costs related to the initiation of the pivotal Phase 3
program for VX-661 in combination with ivacaftor in the first half
of 2015.
- SG&A Expenses: Non-GAAP
SG&A expenses were $72.0 million for the second quarter of
2015, compared to $57.9 million in non-GAAP SG&A expenses for
the second quarter of 2014. This increase was primarily the result
of increased investment in global commercial support for the
planned launch of ORKAMBI.
Non-GAAP Net Loss Attributable to Vertex: Vertex's second
quarter 2015 non-GAAP net loss was $130.7 million, or $0.54 per
diluted share, compared to a non-GAAP net loss of $141.7 million,
or $0.61 per diluted share, for the second quarter of 2014. The
non-GAAP net loss for the second quarter of 2015 was similar to the
second quarter of 2014 as a result of increased KALYDECO product
revenues, offset by increased operating expenses and interest
expense.
Cash Position at June 30,
2015
As of June 30, 2015, Vertex had $1.0 billion in cash, cash
equivalents and marketable securities compared to $1.4 billion in
cash, cash equivalents and marketable securities as of
December 31, 2014. As of June 30, 2015, Vertex had $300
million outstanding from a credit agreement that provides for a
secured loan of up to $500 million.
2015 Financial Guidance
This section contains forward-looking guidance about the
financial outlook for Vertex.
Vertex today increased its financial guidance for total 2015
KALYDECO revenues and reiterated its guidance for non-GAAP
operating expenses:
- KALYDECO Net Revenues: Vertex
now expects KALYDECO net revenues of $575 to $590 million for 2015.
The prior range, first provided on January 28, 2015, was for
KALYDECO net revenues of $560 to $580 million for 2015.
- Non-GAAP R&D and SG&A
Expenses: Vertex reiterated its guidance for combined non-GAAP
R&D and SG&A expenses in 2015 of $1.05 to $1.10
billion.
Vertex's expected combined non-GAAP R&D and SG&A
expenses exclude stock-based compensation expense and certain other
expenses recorded in 2015.
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
including a one-time 2014 cash payment related to a lease
agreement, 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.
Second Quarter 2015 GAAP Financial
Results
Total Revenues: Total revenues for the second quarter of
2015 were $166.1 million compared with $138.4 million in total
revenues for the second quarter of 2014. Second quarter 2015
revenues were comprised primarily of $154.9 million in KALYDECO net
product revenues and an aggregate of $11.2 million in net product
revenues from INCIVEK, royalty revenues and collaborative revenues.
For the second quarter of 2014, Vertex reported $113.1 million in
net product revenues from KALYDECO and an aggregate of $25.4
million in net product revenues from INCIVEK, royalty revenues and
collaborative revenues.
Operating Costs and Expenses: Total operating costs and
expenses for the second quarter of 2015 were $337.2 million,
including certain charges of $66.8 million, compared to $319.0
million for the second quarter of 2014, including certain charges
of $70.5 million. GAAP operating costs and expenses included:
- COR Expenses: COR expenses were
$16.9 million for the second quarter of 2015, including $0.4
million of certain charges, compared to $17.3 million for the
second quarter of 2014, including $6.2 million of certain
charges.
- R&D Expenses: R&D
expenses were $223.9 million for the second quarter of 2015,
including $41.9 million of certain charges, compared to $224.5
million for the second quarter of 2014, including $45.0 million of
certain charges.
- SG&A Expenses: SG&A
expenses were $94.4 million for the second quarter of 2015,
including $22.4 million of certain charges, compared to $77.4
million for the second quarter of 2014, including $19.6 million of
certain charges.
Net Loss Attributable to Vertex: Vertex's second quarter
2015 net loss was $188.8 million, or $0.78 per diluted share,
including net charges of $58.2 million. Vertex's second quarter
2014 net loss was $159.4 million, or $0.68 per diluted share,
including net charges of $17.7 million.
Vertex Pharmaceuticals
Incorporated
Second Quarter Results
Condensed Consolidated Statements of
Operations Data
(in thousands, except per share
amounts)
(unaudited)
Three Months Ended June 30, Six Months Ended June
30, 2015 2014 2015
2014 Revenues: Product revenues, net $ 160,388 $ 122,319 $
291,263 $ 225,780 Royalty revenues 5,077 13,015 11,869 23,748
Collaborative revenues 611 3,087 1,453 7,344
Total revenues 166,076 138,421 304,585 256,872 Costs and
expenses: Cost of product revenues 15,409 9,655 24,790 18,227
Royalty expenses 1,451 7,645 4,377 14,549 Research and development
expenses 223,858 224,487 439,457 463,104 Sales, general and
administrative expenses 94,394 77,446 180,254 151,658 Restructuring
expenses (income) 2,128 (270 ) (1,144 ) 5,918 Total
costs and expenses 337,240 318,963 647,734
653,456 Loss from operations (171,164 ) (180,542 ) (343,149
) (396,584 ) Interest expense, net (21,111 ) (15,585 ) (42,418 )
(31,302 ) Other income (expenses), net 1,414 37,731
(3,699 ) 38,182 Loss from continuing operations before
provision for income taxes (190,861 ) (158,396 ) (389,266 )
(389,704 ) Provision for income taxes 30,131 693
30,430 1,496 Loss from continuing operations (220,992
) (159,089 ) (419,696 ) (391,200 ) Loss from discontinued
operations, net of tax — (293 ) — (639 ) Net loss
(220,992 ) (159,382 ) (419,696 ) (391,839 ) Loss attributable to
noncontrolling interest 32,144 — 32,242 —
Net loss attributable to Vertex $ (188,848 ) $ (159,382 ) $
(387,454 ) $ (391,839 ) Amounts attributable to Vertex: Loss
from continuing operations $ (188,848 ) $ (159,089 ) $ (387,454 ) $
(391,200 ) Loss from discontinued operations — (293 ) —
(639 ) Net loss attributable to Vertex $ (188,848 ) $
(159,382 ) $ (387,454 ) $ (391,839 ) Amounts per share
attributable to Vertex common shareholders: Net loss from
continuing operations: Basic and diluted $ (0.78 ) $ (0.68 ) $
(1.61 ) $ (1.68 ) Net loss: Basic and diluted $ (0.78 ) $ (0.68 ) $
(1.61 ) $ (1.68 ) Shares used in per share calculations: Basic and
diluted 240,757 233,808 240,129 233,353
Reconciliation of GAAP to Non-GAAP Net
Loss
Second Quarter Results
(in thousands, except per share
amounts)
(unaudited)
Three Months Ended June 30, Six Months Ended June
30, 2015 2014 2015
2014 GAAP loss attributable to Vertex $ (188,848 ) $
(159,382 ) $ (387,454 ) $ (391,839 ) Stock-based compensation
expense 63,261 42,444 120,645 89,024 Real estate restructuring
costs and income (Note 1) 1,178 (26,037 ) (2,400 ) (6,095 ) HCV
related revenues and costs (Note 2) (6,004 ) (2,327 ) (10,473 )
8,993 Other adjustments (Note 3) (270 ) 3,584 623
6,909
Non-GAAP net loss attributable to Vertex $
(130,683 ) $ (141,718 ) $ (279,059 ) $ (293,008 ) Amounts
per diluted share attributable to Vertex common shareholders: GAAP
$ (0.78 ) $ (0.68 ) $ (1.61 ) $ (1.68 ) Non-GAAP $ (0.54 ) $ (0.61
) $ (1.16 ) $ (1.26 ) Shares used in diluted per share
calculations: GAAP and Non-GAAP 240,757 233,808 240,129 233,353
Reconciliation of GAAP to Non-GAAP
Revenues and Expenses
Second Quarter Results
(in thousands)
(unaudited)
Three Months Ended June 30, Six Months Ended June
30, 2015 2014 2015
2014 GAAP total revenues $ 166,076 $ 138,421 $
304,585 $ 256,872 HCV related revenues (Note 2) (6,094 ) (16,445 )
(8,963 ) (26,715 ) Other adjustments (Note 3) (74 ) — (274 )
—
Non-GAAP total revenues $ 159,908 $ 121,976
$ 295,348 $ 230,157
Three
Months Ended June 30, Six Months Ended June 30,
2015 2014 2015 2014 GAAP cost of
product revenues and royalty expenses $ 16,860 $ 17,300 $
29,167 $ 32,776 HCV related costs (Note 2) (371 ) (6,233 ) (1,968 )
(12,273 )
Non-GAAP cost of product revenues and royalty
expenses $ 16,489 $ 11,067 $ 27,199 $ 20,503
GAAP
research and development expenses $ 223,858 $ 224,487 $ 439,457
$ 463,104 Stock-based compensation expense (41,632 ) (27,253 )
(79,849 ) (60,153 ) Real estate restructuring costs (Note 1) —
(9,382 ) — (21,583 ) HCV related costs (Note 2) 512 (4,756 ) 1,000
(13,407 ) Other adjustments (Note 3) (827 ) (3,584 ) (1,520 )
(6,909 )
Non-GAAP research and development expenses $
181,911 $ 179,512 $ 359,088 $ 361,052
GAAP sales, general
and administrative expenses $ 94,394 $ 77,446 $ 180,254 $
151,658 Stock-based compensation expense (21,629 ) (15,191 )
(40,796 ) (28,871 ) Real estate restructuring costs (Note 1) —
(1,706 ) — (3,906 ) HCV related costs (Note 2) (54 ) (2,666 ) 2,851
(8,572 ) Other adjustments (Note 3) (695 ) — (1,147 ) —
Non-GAAP sales, general and administrative expenses $
72,016 $ 57,883 $ 141,162 $ 110,309
Combined Non-GAAP R&D and SG&A expenses $ 253,927
$ 237,395 $ 500,250 $ 471,361
Three Months Ended June 30, Six Months Ended June
30, 2015 2014 2015 2014 GAAP
interest expense, net and other income (expense), net $ (19,697
) $ 22,146 $ (46,117 ) $ 6,880 Real estate restructuring income
(Note 1) — (36,685 ) — (36,685 )
Non-GAAP interest
expense, net and other income (expense), net $ (19,697 ) $
(14,539 ) $ (46,117 ) $ (29,805 )
GAAP provision for
income taxes $ 30,131 $ 693 $ 30,430 $ 1,496 Other adjustments
(Note 3) (29,653 ) — (29,589 ) —
Non-GAAP
provision for income taxes $ 478 $ 693 $ 841 $ 1,496
Condensed Consolidated Balance Sheets
Data
(in thousands)
(unaudited)
June 30, 2015 December 31, 2014 Assets
Cash, cash equivalents and marketable securities $ 1,016,450 $
1,387,106 Restricted cash and cash equivalents (VIE) (Note 4)
88,318 8,418 Accounts receivable, net 94,519 75,964 Inventories
42,113 30,848 Property and equipment, net 713,378 715,812
Intangible assets and goodwill 334,724 68,915 Other assets 84,571
47,616
Total assets $ 2,374,073 $ 2,334,679
Liabilities and Shareholders' Equity Other
liabilities $ 333,185 $ 307,374 Deferred tax liability 112,413
15,044 Accrued restructuring expense 19,843 45,855 Deferred
revenues 35,949 45,276 Capital leases 56,821 57,099 Fan Pier lease
obligation 472,834 473,073 Senior secured term loan 294,812 294,775
Shareholders' equity 1,048,216 1,096,183
Total
liabilities and shareholders' equity $ 2,374,073 $
2,334,679 Common shares outstanding 244,342 241,764
Note 1: In the three and six months ended June 30, 2015,
"Real estate restructuring costs and income" consisted of
restructuring charges and credits, respectively, related to the
company's relocation from Cambridge to Boston, Massachusetts. In
the three and six months ended June 30, 2014, "Real estate
restructuring costs and income" consisted of (i) transition costs
related to the company's relocation that were recorded as R&D
and SG&A, (ii) restructuring credits and charges, respectively,
related to this relocation and (iii) credits recorded to other
(expense) income, net to record the effect of the one-time cash
payment received related to a lease agreement in the second quarter
of 2014.
Note 2: In the three and six months ended June 30, 2014
and 2015, "HCV related revenues and costs" included in the
company's loss from continuing operations consisted of:
Three Months Ended
June 30, Six Months Ended June 30, 2015
2014 2015 2014 (in
millions) Net product revenues from Incivek $ 5.5 $ 9.3 $
6.2 $ 13.2 Royalty revenues from Incivo 0.1 5.7 1.6 10.6 HCV
collaborative revenues 0.5 1.5 1.2 2.9 COR expenses (0.4 ) (6.2 )
(2.0 ) (12.3 ) R&D and SG&A credits (including pharma fee)
0.5 (7.4 ) 3.9 (22.0 ) Restructuring expenses (0.2 ) (0.2 ) (0.4 )
(0.8 )
Note 3: In each of the three and six months ended June
30, 2014 and 2015, "Other adjustments" consisted of development
cost associated with VX-509. In addition, in the three and six
months ended June 30, 2015, "Other adjustments" included amounts
related to two variable interest entities ("VIEs").
Note 4: The company consolidates the financial statements
of two of its collaborators as VIEs as of June 30, 2015 and
consolidated a single VIE as of December 31, 2014. These VIEs
are consolidated because Vertex has licensed the rights to develop
the company's collaborator's 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.
Note 5: In each of the three and six months ended June
30, 2014 and 2015, the company excludes from its non-GAAP loss
attributable to Vertex restructuring expense (income). In addition,
in the three and six months ended June 30, 2014 discontinued
operations related to the effect of the company's relationship with
Alios are excluded from its non-GAAP loss attributable to
Vertex.
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 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 five 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 "2015 Financial Guidance," and statements
regarding the expected timing of potential approval of ORKAMBI in
ex-U.S. markets and the expected timing and clinical trial designs
of the (i) Phase 3 clinical studies of lumacaftor in combination
with ivacaftor in children 6 to 11 years of age, (ii) Phase 3
program of VX-661 in combination with ivacaftor and (iii) Phase 2a
clinical studies of VX-371 (P-1037). 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 2015 revenues
and financial results and its 2015 non-GAAP operating expenses may
be incorrect (including because one or more of the company's
assumptions underlying its revenue or expense expectations may not
be realized), that regulatory authorities outside of the United
States may not approve, or approve on a timely basis, ORKAMBI, 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
Webcast
The 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)
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version on businesswire.com: http://www.businesswire.com/news/home/20150729006530/en/
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orKelly Lewis,
617-961-7530orMedia:Zach Barber
617-341-6992mediainfo@vrtx.com
Vertex Pharmaceuticals (NASDAQ:VRTX)
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