-Third-quarter 2017 cystic fibrosis product
revenues of $550 million, up 34% versus Q3 2016; $336 million for
ORKAMBI and $213 million for KALYDECO-
-Company increases total 2017 CF product
revenue guidance to $2.10 to $2.15 billion; increases ORKAMBI
revenue guidance to $1.29 to $1.32 billion and KALYDECO revenue
guidance to $810 to $830 million-
-Provides update on clinical development
programs, including top-line results for three clinical studies in
CF: Phase 3 ORKAMBI in ages 2 to 5; Phase 3 tezacaftor/ivacaftor
combination in gating mutations; Phase 2 VX-371 + ORKAMBI in
F508del homozygous patients-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
reported consolidated financial results for the third quarter ended
September 30, 2017. Vertex also increased its total 2017 CF
product revenue guidance, including revenue guidance for ORKAMBI®
(lumacaftor/ivacaftor) and KALYDECO® (ivacaftor), and reiterated
its total 2017 combined GAAP and non-GAAP R&D and SG&A
expense guidance.
In addition, the company today reported top-line results for
three clinical studies in CF, including: a Phase 3 study of ORKAMBI
in children with CF ages 2 to 5 who have two copies of the F508del
mutation; a Phase 3 study of the tezacaftor/ivacaftor combination
in people with CF with one copy of the F508del mutation and one
copy of a gating mutation; and a Phase 2 study of the ENaC
inhibitor VX-371 in combination with ORKAMBI in people with CF who
have two copies of the F508del mutation.
Key financial results include:
Three Months Ended September 30, %
2017
2016 Change (in millions, except per share and
percentage data)
ORKAMBI product revenues, net $ 336 $ 234
44%
KALYDECO product revenues, net $
213
$
176
22%
TOTAL CF product revenues, net $
550
$
410
34%
GAAP net loss $ (103 ) $ (39 ) n/a
GAAP
net loss per share - diluted $ (0.41 ) $ (0.16 ) n/a
Non-GAAP net income $ 136 $ 43 216%
Non-GAAP net income per share -
diluted
$ 0.53 $ 0.17 212%
"Vertex has never been stronger than it is today with
significant progress across all aspects of our business," said
Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief
Executive Officer of Vertex. "We are now treating more patients
with our approved medicines than ever before, resulting in
significant revenues and earnings growth. We expect this financial
trajectory to continue, driven by our pipeline of transformative CF
medicines."
Dr. Leiden continued, "We look forward to continued progress in
2018 with the anticipated approval of our third CF medicine, and
advancement into pivotal development of our portfolio of triple
combination regimens, which have the potential to treat nearly all
CF patients in the future."
Financial Highlights
Revenues:
- Total CF net product revenues were
$549.6 million compared to $409.7 million for the third quarter of
2016.
- Net product revenues from ORKAMBI were
$336.2 million compared to $234.0 million for the third quarter of
2016. The increase in ORKAMBI revenues was driven by a number of
factors, including the continued uptake in children with CF ages 6
to 11 in the U.S. and the addition of revenues from European
countries where ORKAMBI is currently reimbursed.
- Net product revenues from KALYDECO were
$213.5 million compared to $175.6 million for the third quarter of
2016. The increase in KALYDECO revenues was driven by the approval
and uptake among people ages 2 and older in the U.S. who have
certain residual function mutations.
Expenses:
- Combined GAAP R&D and SG&A
expenses were $575.7 million compared to $378.4 million for the
third quarter of 2016. Combined non-GAAP R&D and SG&A
expenses were $333.8 million compared to $295.0 million for the
third quarter of 2016.
- GAAP R&D expenses were $454.9
million compared to $272.4 million for the third quarter of 2016.
The increase in GAAP R&D expenses was primarily due to an
upfront payment of $160.0 million related to the acquisition of
VX-561 (previously known as CTP-656), an investigational once-daily
CFTR potentiator, from Concert Pharmaceuticals. Non-GAAP R&D
expenses were $243.2 million compared to $211.0 million for the
third quarter of 2016. The increase in non-GAAP R&D expenses
was primarily attributable to the clinical development of the
company's triple combination regimens for CF.
- GAAP SG&A expenses were $120.7
million compared to $106.1 million for the third quarter of 2016.
Non-GAAP SG&A expenses were $90.6 million compared to $84.0
million for the third quarter of 2016. The increase in GAAP and
non-GAAP SG&A expenses was driven by the global support for
KALYDECO and ORKAMBI.
Net Income (Loss) Attributable to Vertex:
- GAAP net loss was $(103.0) million, or
$(0.41) per diluted share, for the third quarter of 2017, compared
to a net loss of $(38.8) million, or $(0.16) per diluted share, for
the third quarter of 2016. The GAAP net loss in the third quarter
of 2017 was primarily due to an upfront payment of $160.0 million
related to the acquisition of VX-561 from Concert Pharmaceuticals.
Non-GAAP net income was $136.4 million, or $0.53 per diluted share,
for the third quarter of 2017, compared to $43.1 million, or $0.17
per diluted share, for the third quarter of 2016. Third quarter
2017 non-GAAP net income growth was driven by increased CF product
revenues.
Intangible Asset Impairment:
- Based upon Phase 2 data evaluating
VX-371 in combination with ORKAMBI (reported below), Vertex
concluded that the intangible asset had become fully impaired, and
also resulted in the deconsolidation of Parion Sciences. This
impairment caused a write down of the assets, including the
intangible asset, related to Parion, offset by the benefit from
income taxes and the reversal of non-controlling interest, which
resulted in an increase in GAAP net loss of $7.1 million for the
third quarter of 2017 and had no impact on non-GAAP net
income.
Cash Position:
- As of September 30, 2017, Vertex
had $1.81 billion in cash, cash equivalents and marketable
securities compared to $1.43 billion in cash, cash equivalents and
marketable securities as of December 31, 2016.
2017 Financial Guidance:
Vertex today increased its total 2017 CF product revenue
guidance, including ORKAMBI and KALYDECO revenue guidance, and
reiterated its combined GAAP and non-GAAP R&D and SG&A
expense guidance:
- Total CF Product Revenues:
Vertex now expects total 2017 CF product revenues of $2.10 to $2.15
billion, an increase from its previously announced guidance of
$1.87 to $2.10 billion.
- ORKAMBI: The company now expects
total 2017 product revenues for ORKAMBI of $1.29 to $1.32 billion,
an increase from its previously announced guidance of $1.1 to $1.3
billion. The updated guidance reflects the strong underlying demand
for ORKAMBI throughout the year among people with CF ages 6 and
older in the U.S. and is based on estimates of potential revenues
from countries where ORKAMBI is currently reimbursed. This guidance
does not assume recognition of any ORKAMBI product revenues from
France in 2017.
- KALYDECO: The company now
expects total 2017 product revenues for KALYDECO of $810 to $830
million, an increase from its previously announced guidance of $770
to $800 million. The updated guidance reflects the rapid uptake
among people with CF ages 2 and older in the U.S. who have certain
residual function mutations, following recent label expansions for
these patients.
- Combined Non-GAAP and GAAP R&D
and SG&A Expenses: Vertex continues to expect that total
2017 combined GAAP R&D and SG&A expense will be in the
range of $1.79 to $1.92 billion and combined non-GAAP R&D and
SG&A expense will be in the range of $1.33 to $1.36
billion.
Clinical Update
Vertex today provided updates on a number of its clinical
development programs, including top-line results for three clinical
studies in CF:
KALYDECO
Label expansion for people ages 2 and
older with residual function mutations: On August 1, 2017,
Vertex announced that the U.S. Food and Drug Administration (FDA)
approved KALYDECO for more than 600 people with CF ages 2 and older
who have one of five residual function mutations that result in a
splicing defect in the CFTR gene. This approval followed the FDA's
approval of KALYDECO in May 2017 for 23 other residual
function mutations.
Phase 3 study in children under two years
of age: Vertex today announced that enrollment is complete in
the 12 to 24-month age group of the Phase 3 study evaluating the
safety of KALYDECO in children under 2 years of age with one of 10
gating and R117H mutations.
ORKAMBI
Phase 3 results in children ages 2 to
5: Vertex today announced results from a 2-part, open-label
Phase 3 study of ORKAMBI in 60 children ages 2 to 5 with CF who
have two copies of the F508del mutation. The study met its primary
endpoint of safety showing that ORKAMBI was generally well
tolerated and there were no new safety concerns compared to prior
studies of ORKAMBI in people ages 6 through 11. Secondary endpoints
showed decreases in sweat chloride and improvements in nutritional
status as measured by change in weight (weight-for-age z score) and
body mass index (BMI-for-age z score).
Based on results from this study, Vertex
expects to submit a New Drug Application (NDA) to the FDA and a
Marketing Authorization Application (MAA) line extension to the
European Medicines Agency (EMA) in the first quarter of 2018.
TEZACAFTOR/IVACAFTOR
Regulatory submissions accepted for people
ages 12 and older: On August 24, 2017, Vertex announced that
the FDA granted Priority Review of the NDA for the use of the
tezacaftor/ivacaftor combination treatment studied in people with
CF ages 12 and older who have two copies of the F508del mutation or
one F508del mutation and one residual function mutation that is
responsive to tezacaftor/ivacaftor and set an action date of
February 28, 2018.
Additionally, the EMA has validated the MAA
for the tezacaftor/ivacaftor combination, confirming that the
submission is complete. The company expects approval in the EU in
the second half of 2018.
Phase 3 results in people with one copy of
the F508del mutation and one copy of a gating mutation: Vertex
announced today top-line results from a Phase 3, randomized,
double-blind, parallel-group study evaluating the addition of
tezacaftor in people with CF ages 12 and older who were already
receiving ivacaftor monotherapy and who have one copy of the
F508del mutation and one copy of a gating mutation. The study
enrolled 151 CF patients throughout sites in the U.S., Canada,
Australia and the EU.
The study did not meet its primary endpoint
of absolute change in percent predicted forced expiratory volume in
one second (ppFEV1) from baseline through 8 weeks. For those
receiving tezacaftor in addition to ivacaftor, ppFEV1 improved 0.5
percentage points compared to 0.2 percentage points in those
receiving placebo in addition to ivacaftor (p=0.5846). Safety data
from the study showed that the addition of tezacaftor to ivacaftor
was generally well tolerated and consistent to prior Phase 3
studies of the tezacaftor/ivacaftor combination. Key secondary
endpoints were changes in sweat chloride and change in CFQ-R. Sweat
Chloride decreased 5.8 mmol/L in those who received tezacaftor in
addition to ivacaftor compared to placebo in addition to ivacaftor
(p=0.0216). There was no change in CFQ-R compared to the placebo
group.
Based on the results from this study, Vertex
does not plan to seek regulatory approval for the
tezacaftor/ivacaftor combination in people with CF ages 12 and
older with one copy of the F508del mutation and one copy of a
gating mutation, the vast majority of whom are today eligible for
KALYDECO.
TRIPLE COMBINATION REGIMENS
Vertex continues to evaluate four different
next-generation correctors to be included in an investigational
triple combination regimen. The company expects to initiate pivotal
development of up to two triple combination regimens in the first
half of 2018 pending discussions with regulatory agencies and
additional Phase 2 data for VX-152, VX-659 and VX-445, which are
expected in early 2018.
Vertex recently amended its Phase 2 studies
evaluating VX-659 and VX-445 to add additional cohorts of patients
in order to evaluate each of these next-generation correctors in
combination with tezacaftor and VX-561 as a potential once-daily
triple combination regimen. VX-561 was acquired from Concert
Pharmaceuticals in the third quarter of 2017. These additional
4-week study arms will evaluate once-daily triple combination
dosing in people with CF who have one copy of the F508del mutation
and one copy of a mutation that results in minimal CFTR
function.
ENaC
Phase 2 study of VX-371 in patients
already receiving ORKAMBI: Vertex today announced results from
a Phase 2, 28-day study of the inhaled epithelial sodium channel
(ENaC) inhibitor, VX-371 (P-1037), being developed in collaboration
with Parion Sciences. The study primarily evaluated VX-371 +
hypertonic saline versus hypertonic saline alone in CF patients who
were already receiving ORKAMBI and who continued to receive ORKAMBI
throughout the study. The study dosed 142 CF patients ages 12 and
older who are homozygous for the F508del mutation. The study did
not meet its primary efficacy endpoint. In patients being treated
with ORKAMBI, the addition of hypertonic saline resulted in a
decrease in ppFEV1 of 0.1 percentage points at Day 28. In patients
being treated with ORKAMBI, the addition of VX-371 + hypertonic
saline resulted in an increase in ppFEV1 of 0.1 percentage points
at Day 28. Safety data from the study showed that the addition of
VX-371, with or without hypertonic saline, was generally well
tolerated in patients already receiving ORKAMBI, and the safety
profile was consistent with that observed in prior studies of
VX-371 monotherapy. A Phase 2 study of VX-371 monotherapy in
patients with primary ciliary dyskinesia (PCD) is ongoing.
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 and guidance exclude (i) stock-based compensation expense,
(ii) revenues and expenses related to business development
transactions including collaboration agreements and asset
acquisitions, (iii) revenues and expenses related to consolidated
variable interest entities, including asset impairment charges and
related income tax benefits and the effects of the deconsolidation
of a variable interest entity and (iv) 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. The company
adjusts, where appropriate, for both revenues and expenses in order
to reflect the company's operations. The company provides guidance
regarding product revenues in accordance with GAAP and provides
guidance regarding combined research and development and sales,
general, and administrative expenses on both a GAAP and a non-GAAP
basis. The guidance regarding GAAP research and development
expenses and sales, general and administrative expenses does not
include estimates regarding expenses associated with any potential
future business development activities. A reconciliation of the
GAAP financial results to non-GAAP financial results is included in
the attached financial information.
Vertex Pharmaceuticals
Incorporated
Third-Quarter Results
Consolidated Statements of Operations
Data
(in thousands, except per share
amounts)
(unaudited)
Three Months EndedSeptember
30,
Nine Months EndedSeptember
30,
2017 2016 2017 2016
Revenues: Product revenues, net $ 549,642 $ 409,689 $ 1,544,252 $
1,229,750 Royalty revenues 2,231 3,835 6,643 12,713 Collaborative
revenues (Note 1) 26,292 259 286,123 1,008
Total revenues
578,165 413,783 1,837,018 1,243,471 Costs and expenses: Cost of
product revenues 72,186 53,222 188,963 147,165 Royalty expenses 688
855 2,104 2,813 Research and development expenses 454,947 272,370
1,017,961 799,238 Sales, general and administrative expenses
120,710 106,055 361,285 322,921 Restructuring expenses 337 8 13,859
1,038 Intangible asset impairment charge (Note 2) 255,340 —
255,340 — Total costs and expenses 904,208
432,510 1,839,512 1,273,175 Loss from
operations (326,043 ) (18,727 ) (2,494 ) (29,704 ) Interest
expense, net (13,574 ) (20,140 ) (45,003 ) (60,993 ) Other
(expenses) income, net (Note 2) (77,553 ) (167 ) (80,634 ) 3,025
Loss from operations before (benefit from) provision for
income taxes (Note 2) (417,170 ) (39,034 ) (128,131 ) (87,672 )
(Benefit from) provision for income taxes (Note 2) (125,903 ) 503
(117,581 ) 24,118 Net loss (291,267 ) (39,537 )
(10,550 ) (111,790 ) Loss (income) attributable to noncontrolling
interest (Note 2) 188,315 696 173,350 (33,207
) Net (loss) income attributable to Vertex $ (102,952 ) $ (38,841 )
$ 162,800 $ (144,997 ) Amounts per share attributable
to Vertex common shareholders: Net (loss) income: Basic $ (0.41 ) $
(0.16 ) $ 0.66 $ (0.59 ) Diluted $ (0.41 ) $ (0.16 ) $ 0.64 $ (0.59
) Shares used in per share calculations: Basic 250,268 244,920
247,963 244,529 Diluted 250,268 244,920 252,095 244,529
Reconciliation of GAAP to Non-GAAP Net
Income (Loss)
Third-Quarter Results
(in thousands, except per share
amounts)
(unaudited)
Three Months EndedSeptember
30,
Nine Months EndedSeptember
30,
2017 2016 2017 2016
GAAP (loss) income attributable to Vertex $ (102,952 ) $
(38,841 ) $ 162,800 $ (144,997 )
Stock-based compensation expense
73,770 61,209 215,334 178,623 Concert upfront and transaction
expenses (Note 3) 160,962 — 165,057 — Revenues and expenses related
to VIEs (Note 2) 7,093 1,200 14,083 59,350 Other collaborative and
transaction revenue and expenses (Note 4) (3,236 ) 22,000 (236,570
) 33,000 Other adjustments (Note 5) 770 (2,437 ) 16,006
(2,451 )
Non-GAAP net income attributable to Vertex $
136,407 $ 43,131 $ 336,710 $ 123,525
Amounts per diluted share attributable to Vertex common
shareholders: GAAP $ (0.41 ) $ (0.16 ) $ 0.64 $ (0.59 ) Non-GAAP $
0.53 $ 0.17 $ 1.33 $ 0.50 Shares used in diluted per share
calculations: GAAP 250,268 244,920 252,095 244,529 Non-GAAP 255,792
248,009 252,095 247,433
Reconciliation of GAAP to Non-GAAP
Revenues and Expenses
Third-Quarter Results
(in thousands)
(unaudited)
Three Months EndedSeptember
30,
Nine Months EndedSeptember
30,
2017 2016 2017 2016
GAAP total revenues $ 578,165 $ 413,783 $ 1,837,018 $
1,243,471 Revenues related to VIEs (Note 2) (21,082 ) (203 )
(42,879 ) (850 ) Other collaborative and transaction revenue (Note
4) (5,209 ) — (243,096 ) — Other adjustments (Note 5) — (43
) — (405 )
Non-GAAP total revenues $ 551,874 $
413,537 $ 1,551,043 $ 1,242,216
Three Months EndedSeptember
30,
Nine Months EndedSeptember
30,
2017 2016 2017 2016 GAAP cost of
product revenues and royalty expenses $ 72,874 $ 54,077 $
191,067 $ 149,978 Other adjustments (Note 5) — 16 —
(117 )
Non-GAAP cost of product revenues and royalty
expenses $ 72,874 $ 54,093 $ 191,067 $ 149,861
GAAP
research and development expenses $ 454,947 $ 272,370 $
1,017,961 $ 799,238 Stock-based compensation expense (46,186 )
(39,980 ) (134,855 ) (115,068 ) Concert upfront payment (Note 3)
(160,000 ) — (160,000 ) — Expenses related to VIEs (Note 2) (3,548
) (1,885 ) (6,762 ) (3,791 ) Other collaborative and transaction
expenses (Note 4) (1,865 ) (22,000 ) (5,684 ) (33,000 ) Other
adjustments (Note 5) (136 ) 2,461 (408 ) 3,305
Non-GAAP research and development expenses $ 243,212 $
210,966 $ 710,252 $ 650,684
GAAP sales, general and
administrative expenses $ 120,710 $ 106,055 $ 361,285 $ 322,921
Stock-based compensation expense (27,584 ) (21,229 ) (80,479 )
(63,555 ) Concert transaction expenses (Note 3) (962 ) — (5,057 ) —
Expenses related to VIEs (Note 2) (1,201 ) (758 ) (3,361 ) (2,999 )
Other collaborative and transaction expenses (Note 4) (109 ) — (842
) — Other adjustments (Note 5) (297 ) (76 ) (1,739 ) (106 )
Non-GAAP sales, general and administrative expenses $ 90,557
$ 83,992 $ 269,807 $ 256,261
Combined non-GAAP R&D and SG&A expenses $ 333,769
$ 294,958 $ 980,059 $ 906,945
Three Months EndedSeptember
30,
Nine Months EndedSeptember
30,
2017 2016 2017 2016 GAAP interest
expense, net and other expense, net $ (91,127 ) $ (20,307 ) $
(125,637 ) $ (57,968 ) Expenses (income) related to VIEs (Note 2)
76,581 (36 ) 76,507 138
Non-GAAP interest
expense, net and other expense, net $ (14,546 ) $ (20,343 ) $
(49,130 ) $ (57,830 )
GAAP (benefit from) provision for
income taxes $ (125,903 ) $ 503 $ (117,581 ) $ 24,118 Income
taxes related to VIEs (Note 2) 120,181 509 111,658
(20,063 )
Non-GAAP (benefit from) provision for income
taxes $ (5,722 ) $ 1,012 $ (5,923 ) $ 4,055
Condensed Consolidated Balance Sheets
Data
(in thousands)
(unaudited)
September 30, 2017 December 31,
2016 Assets Cash, cash equivalents and marketable
securities $ 1,812,248 $ 1,434,557 Restricted cash and cash
equivalents (VIE) (Note 2) 1,803 47,762 Accounts receivable, net
263,493 201,083 Inventories 98,192 77,604 Property and equipment,
net 759,978 698,362 Intangible assets and goodwill (Note 2) 79,384
334,724 Other assets 183,227 102,695
Total assets $
3,198,325 $ 2,896,787
Liabilities and
Shareholders' Equity Accounts payable and accruals $ 455,692 $
376,700 Other liabilities 381,167 260,984 Deferred tax liability
(Note 2) 10,682 134,063 Construction financing lease obligation
547,540 486,849 Debt — 300,000 Shareholders' equity 1,803,244
1,338,191
Total liabilities and shareholders' equity
$ 3,198,325 $ 2,896,787 Common shares outstanding
252,683 248,301
Note 1: In the nine months ended September 30, 2017,
collaborative revenues were primarily attributable to a $230
million up-front payment earned from our collaboration with Merck
KGaA, Darmstadt, Germany. During the three and nine months ended
September 30, 2017, collaborative revenues also includes $20.0
million and $40.0 million, respectively, that one of the company's
consolidated variable interest entities ("VIEs") received from a
collaboration agreement with a third party.
Note 2: The company consolidated the financial statements
of two of its collaborators as VIEs during 2016 and through
September 30, 2017. These VIEs were 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. "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 payments by Vertex to these collaborators. Any increase
in the fair value of these contingent 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 payments is evaluated each quarter and any
change in the fair value is reflected in the company's statement of
operations.
In the third quarter of 2017, the company determined that the
value of Parion’s pulmonary ENaC platform had become impaired and
that the fair value of the intangible asset was zero as of
September 30, 2017. Accordingly, an impairment charge of $255.3
million and a benefit from income taxes of $126.2 million resulting
from this charge and subsequent deconsolidation of Parion
attributable to noncontrolling interest was recorded in the third
quarter of 2017. The total impact of this transaction on a GAAP
basis was a $198.7 million loss attributable to noncontrolling
interest and a $7.1 million loss attributable to Vertex and had no
impact on Vertex’s non-GAAP net income in the third quarter of
2017.
As of September 30, 2017, the company has a $29.0 million
intangible asset related to its collaboration agreement with
BioAxone Biosciences, Inc.
Note 3: In July 2017, the company completed the
acquisition of VX-561 (formerly CTP-656) from Concert
Pharmaceuticals, Inc. The company paid Concert $160.0
million in cash to acquire VX-561, which was recorded as a
research and development expense in the three and nine months ended
September 30, 2017.
Note 4: In the three and nine months ended
September 30, 2017, "Other collaboration and transaction
revenues and expenses" primarily consisted of revenues and expenses
associated with the company's oncology program including the
company's collaboration with Merck KGaA, Darmstadt, Germany
including the $230 million upfront payment earned pursuant to the
collaboration. In the three and nine months ended September 30,
2016, "Other collaboration and transaction revenues and expenses"
primarily consisted of collaboration and asset acquisition payments
for early-stage research assets. The company has not adjusted its
prior year Reconciliation of GAAP to Non-GAAP Revenues and Expenses
for the three and nine months ended September 30, 2016 for $5.0
million and $14.9 million, respectively, of operating expenses
related to its oncology program.
Note 5: In the three and nine months ended
September 30, 2017, "Other adjustments" primarily consisted of
restructuring charges related to the company's decision to
consolidate its research activities into its Boston, Milton Park
and San Diego locations and to close our research site in Canada.
In the three and nine months ended September 30, 2016, "Other
adjustments" primarily consisted of revenues and operating costs
and expenses related to HCV as well as restructuring charges
related to the company's relocation from Cambridge to Boston,
Massachusetts.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO®
(ivacaftor)
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 2 years and older
who have one mutation in their CF gene that is responsive to
KALYDECO. Patients should talk to their doctor to learn if they
have an indicated CF gene mutation. It is not known if KALYDECO is
safe and effective in children under 2 years of age.
Patients should not take KALYDECO if they are taking certain
medicines or herbal supplements such as: the antibiotics
rifampin or rifabutin; seizure medications such as phenobarbital,
carbamazepine, or phenytoin; or St. John’s wort.
Before taking KALYDECO, patients should tell their doctor if
they: have liver or kidney problems; drink grapefruit juice, or
eat grapefruit or Seville oranges; are pregnant or plan to become
pregnant because it is not known if KALYDECO will harm an unborn
baby; and are breastfeeding or planning to breastfeed because is
not known if KALYDECO passes into breast milk.
KALYDECO may affect the way other medicines work, and other
medicines may affect how KALYDECO works. Therefore the dose of
KALYDECO may need to be adjusted when taken with certain
medications. Patients should especially tell their doctor if they
take antifungal medications such as ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it.
Patients should not drive a car, use machinery, or do anything that
needs them to be alert until they know how KALYDECO affects them.
Patients should avoid food containing grapefruit or Seville oranges
while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood have been reported in
patients receiving KALYDECO. The patient’s doctor will do blood
tests to check their liver before starting KALYDECO, every 3 months
during the first year of taking KALYDECO, and every year while
taking KALYDECO. For patients who have had high liver enzymes in
the past, the doctor may do blood tests to check the liver more
often. Patients should call their doctor right away if they have
any of the following symptoms of liver problems: pain or discomfort
in the upper right stomach (abdominal) area; yellowing of their
skin or the white part of their eyes; loss of appetite; nausea or
vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving KALYDECO. The patient’s doctor
should perform eye examinations prior to and during treatment with
KALYDECO to look for cataracts. The most common side effects
include headache; upper respiratory tract infection (common cold),
which includes sore throat, nasal or sinus congestion, and runny
nose; stomach (abdominal) pain; diarrhea; rash; nausea; and
dizziness.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing
Information for KALYDECO (ivacaftor).
INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor) TABLETS
ORKAMBI is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients age 6 years and older who have two
copies of the F508del mutation (F508del/F508del) in their CFTR
gene. ORKAMBI should only be used in these patients. It is not
known if ORKAMBI is safe and effective in children under 6 years of
age.
Patients should not take ORKAMBI if they are taking certain
medicines or herbal supplements, such as: the antibiotics
rifampin or rifabutin; the seizure medicines phenobarbital,
carbamazepine, or phenytoin; the sedatives/anti-anxiety medicines
triazolam or midazolam; the immunosuppressant medicines everolimus,
sirolimus, or tacrolimus; or St. John’s wort.
Before taking ORKAMBI, patients should tell their doctor if
they: have or have had liver problems; have kidney problems;
have had an organ transplant; are using birth control (hormonal
contraceptives, including oral, injectable, transdermal or
implantable forms). Hormonal contraceptives should not be used as a
method of birth control when taking ORKAMBI. Patients should tell
their doctor if they are pregnant or plan to become pregnant (it is
unknown if ORKAMBI will harm the unborn baby) or if they are
breastfeeding or planning to breastfeed (it is unknown if ORKAMBI
passes into breast milk).
ORKAMBI may affect the way other medicines work and other
medicines may affect how ORKAMBI works. Therefore, the dose of
ORKAMBI or other medicines may need to be adjusted when taken
together. Patients should especially tell their doctor if they
take: antifungal medicines such as ketoconazole, itraconazole,
posaconazole, or voriconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
When taking ORKAMBI, patients should tell their doctor if
they stop ORKAMBI for more than 1 week as the doctor may need to
change the dose of ORKAMBI or other medicines the patient is
taking. It is unknown if ORKAMBI causes dizziness. Patients should
not drive a car, use machinery, or do anything requiring alertness
until the patient knows how ORKAMBI affects them.
ORKAMBI can cause serious side effects including:
High liver enzymes in the blood, which can be a sign of liver
injury, have been reported in patients receiving ORKAMBI. The
patient’s doctor will do blood tests to check their liver before
they start ORKAMBI, every three months during the first year of
taking ORKAMBI, and annually thereafter. The patient should call
the doctor right away if they have any of the following symptoms of
liver problems: pain or discomfort in the upper right stomach
(abdominal) area; yellowing of the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine; or confusion.
Respiratory events such as shortness of breath or chest
tightness were observed in patients when starting ORKAMBI. If a
patient has poor lung function, their doctor may monitor them more
closely when starting ORKAMBI.
An increase in blood pressure has been seen in some patients
treated with ORKAMBI. The patient’s doctor should monitor their
blood pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving ORKAMBI and ivacaftor, a
component of ORKAMBI. For children and adolescents, the
patient’s doctor should perform eye examinations prior to and
during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include: shortness of
breath and/or chest tightness; upper respiratory tract infection
(common cold), including sore throat, stuffy or runny nose;
gastrointestinal symptoms including nausea, diarrhea, or gas; rash;
fatigue; flu or flu-like symptoms; increase in muscle enzyme
levels; and irregular, missed, or abnormal menstrual periods and
heavier bleeding.
Please click here to see the full Prescribing
Information for ORKAMBI.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is
now located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada and Australia. Vertex is consistently
recognized as one of the industry's top places to work, including
being named to Science magazine's Top Employers in the life
sciences ranking for eight years in a row.
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 third
paragraph of the press release, the information provided in the
section captioned "2017 Financial Guidance" and statements
regarding (i) the timing and expected outcome of regulatory
applications, including NDAs, MAAs and MAA line extensions and (ii)
the development plan and timelines for our product development
candidates, including tezacaftor in combination with ivacaftor and
our next-generation triple combination regimens. 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
2017 revenues and expenses may be incorrect (including because one
or more of the company's assumptions underlying its expectations
may not be realized), that data from the company's development
programs may not support registration or further development of its
compounds due to safety, efficacy or other reasons, and other risks
listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the Securities and Exchange Commission and
available through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
Conference Call and
Webcast
The company will host a conference call and webcast today at
4:30 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-E)
View source
version on businesswire.com: http://www.businesswire.com/news/home/20171025006319/en/
Vertex Pharmaceuticals IncorporatedInvestors: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 Feb 2024 to Mar 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Mar 2023 to Mar 2024