-Full-year 2015 total non-GAAP revenues of
$1.01 billion, including net product revenues of $351 million for
ORKAMBI® (lumacaftor/ivacaftor) and $632 million for KALYDECO®
(ivacaftor) in cystic fibrosis-
-Vertex reiterates 2016 financial guidance for
KALYDECO net product revenues of $670 to $690 million and for
non-GAAP operating expenses of $1.18 to $1.23 billion-
-Phase 3 study of ORKAMBI in children ages 6 to
11 meets primary endpoint and supports planned supplemental New
Drug Application in the second quarter of 2016; Approximately 2,400
children ages 6 to 11 would be eligible for treatment with ORKAMBI
in the U.S.-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
reported consolidated financial results for the quarter ended
December 31, 2015. Vertex also reiterated its financial
guidance for total 2016 KALYDECO® (ivacaftor) net revenues and
non-GAAP operating expenses. Key financial results include:
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 % Change 2015
2014 % Change (in millions, except per
share and percentage data)
ORKAMBI product revenues, net $
220 $ — N/A $ 351 $ — N/A
KALYDECO product revenues, net $
181 $ 124 45 % $ 632 $ 464 36 %
GAAP net loss $ (76 ) $ (177
) (57 )% $ (558 ) $ (739 ) (24 )%
GAAP net loss per share $
(0.31 ) $ (0.74 ) (58 )% $ (2.31 ) $ (3.14 ) (26 )%
Non-GAAP net
income (loss) $ 43 $ (132 ) N/A $ (268 ) $ (511 ) (48 )%
Non-GAAP net income (loss) per share $ 0.17 $ (0.55 ) N/A $
(1.11 ) $ (2.17 ) (49 )%
"Entering 2016, Vertex is in a strong financial position to
support continued investment in our business to create future
medicines for serious diseases and value for our shareholders,"
said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief
Executive Officer of Vertex. "We look forward to multiple important
milestones over the coming year that will mark continued progress
toward our goal of discovering and developing potential new
medicines for all people with CF."
On January 10, 2016, Vertex provided a comprehensive update on
its development programs in cystic fibrosis (CF) and other serious
diseases. The company today provided the following updates and
highlighted upcoming milestones:
Phase 3 Data Support Submission of Supplemental New Drug
Application for ORKAMBI in Children Ages 6 to 11 with Two Copies of
the F508del Mutation in the U.S.
Vertex recently completed a Phase 3 clinical study that was
conducted to support the potential approval of ORKAMBI for children
with CF as young as six years of age with two copies of the F508del
mutation in the U.S. Children in the study received a twice-daily
fixed-dose combination of lumacaftor (200mg) and ivacaftor (250mg)
for six months. The study enrolled 58 children ages 6 to 11 with
two copies of the F508del mutation, and the primary endpoint of the
study was safety. The study did not include a placebo control arm.
Secondary and exploratory endpoints evaluated the effect on
multiple efficacy endpoints including forced expiratory volume in
one second (FEV1), lung clearance index and others.
The study met its primary safety endpoint, and safety data from
the study showed that the combination was generally well-tolerated.
The most common adverse events were cough, headache, infective
pulmonary exacerbation, nasal congestion, abdominal pain, increased
sputum and elevated liver enzymes. Two patients (3.4%) discontinued
treatment because of adverse events.
Based on these data, Vertex plans to submit a supplemental New
Drug Application (sNDA) to the U.S. Food and Drug Administration
(FDA) in the second quarter of 2016. There are approximately 2,400
children ages 6-11 who have two copies of the F508del mutation in
the U.S.
In addition to meeting its primary safety endpoint, the study
showed a 2.5 percentage point improvement in FEV1 (p=0.067), a
secondary endpoint, and a -0.88 improvement in lung clearance index
(p=0.0018), which was an exploratory endpoint. Improvements in
secondary endpoints of body mass index, the respiratory domain of
the CF questionnaire-revised (CFQ-R) and sweat chloride were also
observed in the study.
To support approval in the European Union, a six-month Phase 3
efficacy study is ongoing to evaluate lumacaftor/ivacaftor in
approximately 200 children. The primary endpoint of this study is
the absolute change in lung clearance index. There are
approximately 3,400 children ages 6-11 who have two copies of the
F508del mutation in the European Union.
Supplemental New Drug Application in Residual Function
Mutations
The FDA set a target review date of February 7, 2016 for its
decision regarding a supplemental New Drug Application for the use
of KALYDECO in people ages two and older with one of 23 residual
function mutations. The sNDA was based on preclinical data for
ivacaftor in certain residual function mutations, the established
clinical profile of KALYDECO and on previously reported data from
an exploratory Phase 2a study. Eight of the 23 mutations proposed
for approval in the sNDA were represented in the Phase 2a study.
More than 1,500 people with CF in the U.S. ages two and older have
the mutations represented in the sNDA.
Approval of ORKAMBI in Canada
On January 26, Vertex announced that announced that Health
Canada approved ORKAMBI for use in people ages 12 and older with
two copies of the F508del mutation. It is only indicated for these
patients, who can be identified with a genetic test. Of the
approximately 4,000 people in Canada with CF, approximately 1,500
are ages 12 and older and have two copies of the F508del mutation.
Health Canada approval is the first step in the process for
securing funding through Canada's public drug programs for a new
medicine. Once a new medicine receive approval from Health Canada,
it enters the Canadian Agency for Drugs and Technologies (CADTH)
Common Drug Review (CDR) process. Following its review, CADTH will
send a recommendation to participating public drug programs, and
participating jurisdictions may then proceed to reimbursement
discussions with the manufacturer through the Pan-Canadian
Pharmaceutical Alliance (pCPA). When an agreement has been reached
between the manufacturer and the pCPA, each province and territory
determines how the new medicine will be funded.
Fourth Quarter 2015 Financial
Highlights
Revenues:
- Net Product Revenues from ORKAMBI were
$219.9 million. As of December 31, 2015, more than 4,500
people with CF had started treatment with ORKAMBI.
- Net Product Revenues from KALYDECO were
$180.7 million compared to $124.4 million for the fourth quarter of
2014.
Expenses:
- Total combined non-GAAP cost of product
revenues and royalty expenses (COR) were $64.2 million, compared to
$13.2 million for the fourth quarter of 2014. GAAP COR expenses
were $64.4 million, compared to $14.0 million for the fourth
quarter of 2014. Fourth quarter 2015 COR expenses included a $13.9
million sales milestone earned by Cystic Fibrosis Foundation
Therapeutics, Inc.
- Non-GAAP research and development
(R&D) expenses were $203.8 million compared to $175.7 million
for the fourth quarter of 2014. The increased R&D expenses for
the fourth quarter of 2015 were primarily the result of increased
costs related to the pivotal Phase 3 program for VX-661 in
combination with ivacaftor, which includes four Phase 3 studies in
more than 1,000 patients. GAAP R&D expenses were $310.4 million
compared to $201.5 million for the fourth quarter of 2014.
- Non-GAAP sales, general and
administrative (SG&A) expenses were $78.1 million compared to
$60.4 million for the fourth quarter of 2014. The increased
SG&A expenses were primarily the result of increased investment
in global commercial support for the planned launch of ORKAMBI.
GAAP SG&A expenses were $97.1 million compared to $78.5 million
for the fourth quarter of 2014.
Net Income (Loss) Attributable to Vertex:
- Non-GAAP net income was $42.7 million,
or $0.17 per diluted share, compared to a non-GAAP net loss of
$131.8 million, or $0.55 per diluted share, for the fourth quarter
of 2014. The net income was the result of ORKAMBI product revenues
and increased KALYDECO product revenues, partially offset by
increased operating expenses. The GAAP net loss was $75.5 million,
or $0.31 per diluted share, compared to Vertex's fourth quarter
2014 GAAP net loss of $176.7 million, or $0.74 per diluted
share.
Full Year 2015 Financial
Highlights
Revenues:
- Net Product Revenues from ORKAMBI were
$350.7 million.
- Net Product Revenues from KALYDECO were
$631.7 million compared to $463.8 million for the full year
2014.
Expenses:
- Total combined non-GAAP cost of product
revenues and royalty expenses (COR) were $124.9 million, compared
to $45.0 million for the full year 2014. GAAP COR expenses were
$125.5 million compared to $61.0 million for the full year
2014.
- Non-GAAP research and development
(R&D) expenses were $764.4 million compared to $694.2 million
for the full year 2014. The increased R&D expenses for the full
year 2015 were primarily the result of increased costs related to
the pivotal Phase 3 program for VX-661 in combination with
ivacaftor, which includes four Phase 3 studies in more than 1,000
patients. GAAP R&D expenses were $996.2 million compared to
$855.5 million for the full year 2014.
- Non-GAAP sales, general and
administrative (SG&A) expenses were $295.4 million compared to
$225.6 million for the full year 2014. This increased SG&A
expenses were primarily the result of increased investment in
global commercial support for the planned launch of ORKAMBI. GAAP
SG&A expenses were $377.1 million compared to $305.4 million
for the full year 2014.
Net Loss Attributable to Vertex:
- Non-GAAP net loss was $268.3 million,
or $1.11 per diluted share, compared to a non-GAAP net loss of
$511.2 million, or $2.17 per diluted share, for the full year 2014.
The decreased net loss was the result of the first half year of
ORKAMBI product revenues and increased KALYDECO product revenues,
partially offset by increased operating expenses. The GAAP net loss
was $558.1 million, or $2.31 per diluted share, compared to
Vertex's full year 2014 GAAP net loss of $738.6 million, or $3.14
per diluted share.
Cash Position:
- As of December 31, 2015, Vertex
had $1.04 billion in cash, cash equivalents and marketable
securities compared to $1.39 billion in cash, cash equivalents and
marketable securities as of December 31, 2014.
- As of December 31, 2015, Vertex
had $300 million outstanding from a credit agreement that provides
for a secured loan of up to $500 million.
2016 Financial Guidance:
Vertex today reiterated its 2016 net product revenue guidance
for KALYDECO and guidance for non-GAAP operating expenses,
excluding cost of revenues. The company also today provided
financial guidance for the non-GAAP R&D and SG&A components
of non-GAAP operating expenses. The financial guidance is
summarized below, together with information regarding the company's
expectation for providing ORKAMBI net revenue guidance in 2016, as
previously discussed on January 10, 2016:
- KALYDECO: As announced on
January 10, 2016, Vertex anticipates total 2016 KALYDECO net
product revenues of $670 to $690 million, which exclude any
revenues related to the potential approval of KALYDECO for people
in the U.S. who have residual function mutations. Anticipated 2016
KALYDECO net revenues reflect the expectation for approximately 200
patients with a gating mutation to enroll in a Phase 3 clinical
study of VX-661 in combination with ivacaftor who would otherwise
receive KALYDECO, which will thus reduce 2016 KALYDECO
revenues.
- ORKAMBI: The company expects to
provide net product revenue guidance for ORKAMBI during 2016 after
gaining additional information on the launch of ORKAMBI in the
U.S., including:
- The total proportion of the 8,500
eligible patients who begin treatment with ORKAMBI in 2016.
- The rate at which patients initiate
treatment in 2016.
- The proportion of initiated patients
who remain on treatment.
- The compliance rate for patients who
remain on treatment.
As of December 31, more than 4,500 people had begun treatment
with ORKAMBI in the U.S. since the approval of the medicine in July
2015. Vertex expects the vast majority of eligible patients in the
U.S. will begin treatment by the end of 2016.
Vertex expects to recognize revenues from sales of ORKAMBI in
the U.S. and Germany in 2016. In Germany, there are approximately
2,500 people with CF ages 12 and older with two copies of the
F508del mutation. The company does not anticipate any other
significant revenues from other countries in 2016.
- Operating Expenses, Excluding Cost
of Revenues (Combined Non-GAAP R&D and SG&A Expenses):
Vertex expects 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.
The increase as compared to 2015 is primarily a result of expanded
development efforts related to the pivotal Phase 3 development
program for VX-661 in combination with ivacaftor and for multiple
Phase 1 and 2 studies of Vertex's early-stage and mid-stage
pipeline of potential CF medicines and anticipated costs to support
the launch of ORKAMBI in new global markets. The components of
Vertex's non-GAAP operating expenses include:
- Non-GAAP R&D Expenses:
Vertex expects that full-year 2016 non-GAAP R&D expenses will
be in the range of $850 to $880 million. The development component
of 2016 non-GAAP R&D expenses is expected to increase as
compared to 2015 primarily as a result of the ongoing pivotal Phase
3 program for VX-661 and other ongoing and planned Phase 1 and
Phase 2 studies in CF.
- Non-GAAP SG&A Expenses:
Vertex expects that full-year 2016 non-GAAP SG&A expenses will
be in the range of $330 to $350 million. The expected increase in
SG&A is a result of ongoing investment in the company's
commercial infrastructure to support the global launch of ORKAMBI
in new markets.
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
including a one-time 2014 cash payment related to a lease
agreement, hepatitis C-related revenues and costs, $75.0 million
payment related to our collaboration with CRISPR Therapeutics AG
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
Fourth Quarter Results
Consolidated Statements of Operations
Data
(in thousands, except per share
amounts)
(unaudited)
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 2015
2014 Revenues: Product revenues, net $ 406,550 $ 124,942 $
1,000,324 $ 487,821 Royalty revenues 6,331 8,785 23,959 40,919
Collaborative revenues 5,054 10,829 8,053
51,675 Total revenues 417,935 144,556 1,032,336 580,415
Costs and expenses: Cost of product revenues 63,122 11,290 118,181
39,725 Royalty expenses 1,293 2,737 7,361 21,262 Research and
development expenses 310,429 201,463 996,170 855,506 Sales, general
and administrative expenses 97,054 78,527 377,080 305,409
Restructuring expenses 1,524 4,164 2,206
50,925 Total costs and expenses 473,422 298,181
1,500,998 1,272,827 Loss from operations
(55,487 ) (153,625 ) (468,662 ) (692,412 ) Interest expense, net
(20,654 ) (21,177 ) (84,206 ) (72,863 ) Other (expenses) income,
net (1,688 ) (3,792 ) (6,713 ) 30,400
Loss from continuing operations before
provision forincome taxes
(77,829 ) (178,594 ) (559,581 ) (734,875 ) (Benefit from) provision
for income taxes (1,379 ) 2,043 30,381 6,958
Loss from continuing operations (76,450 ) (180,637 ) (589,962 )
(741,833 ) Loss from discontinued operations, net of tax —
(209 ) — (912 ) Net loss (76,450 ) (180,846 ) (589,962 )
(742,745 ) Loss attributable to noncontrolling interest 938
4,190 31,847 4,190 Net loss attributable to
Vertex $ (75,512 ) $ (176,656 ) $ (558,115 ) $ (738,555 )
Amounts attributable to Vertex: Loss from continuing operations $
(75,512 ) $ (176,447 ) $ (558,115 ) $ (737,643 ) Loss from
discontinued operations — (209 ) — (912 ) Net loss
attributable to Vertex $ (75,512 ) $ (176,656 ) $ (558,115 ) $
(738,555 )
Amounts per share attributable to Vertex
commonshareholders:
Net loss from continuing operations: Basic and diluted $ (0.31 ) $
(0.74 ) $ (2.31 ) $ (3.14 ) Net loss from discontinued operations:
Basic and diluted $ — $ — $ — $ — Net loss: Basic and diluted $
(0.31 ) $ (0.74 ) $ (2.31 ) $ (3.14 ) Shares used in per share
calculations: Basic and diluted 242,987 238,272 241,312 235,307
Reconciliation of GAAP to Non-GAAP Net
Loss
Fourth Quarter Results
(in thousands, except per share
amounts)
(unaudited)
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 2015
2014 GAAP loss attributable to Vertex $ (75,512 ) $
(176,656 ) $ (558,115 ) $ (738,555 ) Stock-based compensation
expense 45,439 42,381 231,817 177,542 Real estate restructuring
costs and income (Note 1) 454 3,660 (1,748 ) 40,963 HCV related
revenues and costs (Note 2) (5,510 ) (1,920 ) (23,716 ) 2,245 Other
adjustments (Note 3) 77,786 703 83,424 6,587
Non-GAAP net income (loss) attributable
toVertex (Note 5)
$ 42,657 $ (131,832 ) $ (268,338 ) $ (511,218 )
Amounts per diluted share attributable to
Vertexcommon shareholders:
GAAP $ (0.31 ) $ (0.74 ) $ (2.31 ) $ (3.14 ) Non-GAAP $ 0.17 $
(0.55 ) $ (1.11 ) $ (2.17 ) Shares used in diluted per share
calculations: GAAP 242,987 238,272 241,312 235,307 Non-GAAP 246,635
238,272 241,312 235,307
Reconciliation of GAAP to Non-GAAP
Revenues and Expenses
Fourth Quarter Results
(in thousands)
(unaudited)
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 2015
2014 GAAP total revenues $ 417,935 $ 144,556 $
1,032,336 $ 580,415 HCV related revenues (Note 2) (6,071 ) (3,968 )
(21,449 ) (44,626 ) Other adjustments (Note 3) (1,509 ) —
(2,888 ) —
Non-GAAP total revenues $ 410,355 $
140,588 $ 1,007,999 $ 535,789
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 2015 2014
GAAP cost of product revenues
androyalty expenses
$ 64,415 $ 14,027 $ 125,542 $ 60,987 HCV related costs (Note 2)
(209 ) (801 ) (631 ) (16,036 )
Non-GAAP cost of product revenues and
royaltyexpenses
$ 64,206 $ 13,226 $ 124,911 $ 44,951
GAAP research and
development expenses $ 310,429 $ 201,463 $ 996,170 $ 855,506
Stock-based compensation expense (28,696 ) (25,714 ) (153,245 )
(116,998 ) Real estate restructuring costs (Note 1) — — — (25,094 )
HCV related costs (Note 2) (213 ) (159 ) 493 (14,993 ) Other
adjustments (Note 3) (77,762 ) 100 (78,984 ) (4,229 )
Non-GAAP research and development expenses $ 203,758 $
175,690 $ 764,434 $ 694,192
GAAP sales, general and
administrative expenses $ 97,054 $ 78,527 $ 377,080 $ 305,409
Stock-based compensation expense (16,743 ) (16,667 ) (78,572 )
(60,544 ) Real estate restructuring costs (Note 1) — (122 ) —
(4,645 ) HCV related costs (Note 2) — (879 ) 2,807 (14,095 ) Other
adjustments (Note 3) (2,176 ) (491 ) (5,892 ) (491 )
Non-GAAP sales, general and
administrativeexpenses
$ 78,135 $ 60,368 $
295,423 $ 225,634
Combined non-GAAP R&D and
SG&Aexpenses
$ 281,893 $ 236,058 $ 1,059,857 $ 919,826
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 2015 2014 GAAP interest
expense, net and other expense, net $ (22,342 ) $ (24,969 ) $
(90,919 ) $ (42,463 ) Real estate restructuring income (Note 1) —
— — (36,685 )
Non-GAAP interest expense, net and
otherexpense, net
$ (22,342 ) $ (24,969 ) $ (90,919 ) $ (79,148 )
GAAP
(benefit from) provision for income taxes $ (1,379 ) $ 2,043 $
30,381 $ 6,958 Other adjustments (Note 3) 636 (3,876 )
(29,731 ) (3,876 )
Non-GAAP (benefit from) provision for
incometaxes
$ (743 ) $ (1,833 ) $ 650 $ 3,082
Condensed Consolidated Balance Sheets
Data
(in thousands)
(unaudited)
December 31, 2015
December 31, 2014 Assets Cash, cash
equivalents and marketable securities $ 1,042,462 $ 1,387,106
Restricted cash and cash equivalents (VIE) (Note 4) 78,910 8,418
Accounts receivable, net 177,639 75,964 Inventories 56,083 30,848
Property and equipment, net 697,715 715,812 Intangible assets and
goodwill 334,724 68,915 Other assets 109,512 47,616
Total
assets $ 2,497,045 $ 2,334,679
Liabilities and
Shareholders' Equity Other liabilities $ 431,944 $ 307,023
Deferred tax liability 110,439 15,044 Accrued restructuring expense
15,358 45,855 Deferred revenues 26,010 45,276 Capital leases 52,124
57,099 Fan Pier lease obligation 473,043 473,424 Senior secured
term loan 295,447 294,775 Shareholders' equity 1,092,680
1,096,183
Total liabilities and shareholders'
equity
$ 2,497,045 $ 2,334,679 Common shares outstanding
246,307 241,764
Note 1: In the three and twelve months ended December 31,
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 twelve months ended December 31, 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 charges 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 twelve months ended December 31,
2015 and 2014, "HCV related revenues and costs" included in the
company's loss from continuing operations consisted of:
Three Months EndedDecember
31,
Twelve Months EndedDecember
31,
2015 2014 2015
2014 (in millions) Net product revenues from Incivek $ 6.0
$ 0.6 $ 18.0 $ 24.1 Royalty revenues from Incivo (0.2
) 0.6 1.5 13.5 HCV collaborative revenues 0.3 2.8 1.9 7.1 COR
expenses (0.2 ) (0.8 ) (0.6 ) (16.0 ) R&D and SG&A credits
(including pharma fee) (0.2 ) (1.0 ) 3.3 (29.1 ) Restructuring
expenses (0.1 ) — (0.4 ) (0.8 )
Note 3: In the three and twelve months ended December 31,
2015, "Other adjustments" was primarily attributable to a $75.0
million payment related to our collaboration with CRISPR
Therapeutics AG that was recorded as R&D expense, as well as
activity related to two variable interest entities ("VIEs") that
are not associated with the company's collaborations with our
collaborators. In each of the three and twelve months ended
December 31, 2014, "Other adjustments" was primarily attributable
to development cost associated with VX-509.
Note 4: The company consolidates the financial statements
of two of its collaborators as VIEs as of December 31, 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 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.
Note 5: In each of the three and twelve months ended
December 31, 2015 and 2014, the company excludes from its non-GAAP
loss attributable to Vertex restructuring expense (income). In
addition, in the three and twelve months ended December 31, 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 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 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 Guidance” and statements
regarding (i) the sNDA for ORKAMBI for the treatment of patients
six to eleven years of age who are homozygous for the F508del
mutation and the ongoing Phase 3 study in this same patient
population and (ii) the target date for the FDA to review the sNDA
for the use of KALYDECO in patients ages two and older with one of
23 residual function mutations. 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
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/20160127006283/en/
Vertex Contacts:Investors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:617-341-6992mediainfo@vrtx.com
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