— Product revenue of $2.48 billion, a 6%
increase compared to Q3 2022 —
— Company raises full year 2023 product revenue
guidance to approximately $9.85 billion —
— U.S. FDA Advisory Committee meeting for
exa-cel in SCD completed; PDUFA date for exa-cel in SCD is December
8, 2023 —
— Pipeline continues to advance: Phase 3 data
in CF and acute pain expected in early 2024; Phase 2 data in
neuropathic pain expected in late 2023 —
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
reported consolidated financial results for the third quarter ended
September 30, 2023, raised full year 2023 guidance for product
revenue and reiterated full year 2023 guidance for operating
expenses.
“Vertex has delivered another strong quarter across the
business. We remain relentless in our commitment to reach more
patients with our cystic fibrosis medicines, while preparing for
the potential launch of exa-cel in multiple geographies,” said
Reshma Kewalramani, M.D., Chief Executive Officer and President of
Vertex. “Our R&D pipeline continues to make remarkable progress
and we have a milestone-rich period coming up, with multiple major,
near-term milestones, including completion of the Phase 3 pivotal
trials for the vanzacaftor triple in cystic fibrosis and VX-548 in
acute pain, as well as the Phase 2 VX-548 study data read-out in
diabetic peripheral neuropathy.”
Third Quarter 2023
Results
Product revenue increased 6% to $2.48 billion compared to
the third quarter of 2022, primarily driven by the continued
performance of TRIKAFTA in the U.S., including the launch in
children with CF 2 to 5 years of age and continued strong uptake of
TRIKAFTA/KAFTRIO in ex-U.S. markets with recently achieved
reimbursements, as well as label extensions in younger age groups.
Net product revenue in the third quarter of 2023 increased 7% to
$1.55 billion in the U.S. and increased 6% to $929 million outside
the U.S., compared to the third quarter of 2022.
Combined GAAP and Non-GAAP R&D, Acquired IPR&D and
SG&A expenses were $1.1 billion and $993 million,
respectively, compared to $921 million and $758 million,
respectively, in the third quarter of 2022. The increases were due
to increased investment in support of multiple programs that have
advanced in mid- and late-stage clinical development, the costs to
support launches of Vertex's therapies globally, and increased
acquired IPR&D expenses.
GAAP effective tax rate was 12.2% compared to 20.9% for
the third quarter of 2022 as a result of increased R&D tax
credits for the current and prior years.
Non-GAAP effective tax rate was 19.4% compared to 21.4%
for the third quarter of 2022 as a result of increased R&D tax
credits for the current year. Please refer to Note 1 for further
details on our GAAP to Non-GAAP tax adjustments.
GAAP and Non-GAAP net income increased by 11% and 2%,
respectively, compared to the third quarter of 2022. Strong revenue
growth, higher interest income and lower income tax expense in the
third quarter of 2023 were partially offset by increased investment
in our mid- and late-stage clinical pipeline, the costs to support
launches of Vertex’s therapies globally, and higher acquired
IPR&D expenses.
Cash, cash equivalents and total marketable securities as
of September 30, 2023 were $13.6 billion, compared to $10.9 billion
as of December 31, 2022. The increase was primarily driven by
strong revenue growth and operating cash flow, partially offset by
our payments to Entrada Therapeutics, CRISPR Therapeutics and other
collaboration partners, repurchases of our common stock pursuant to
our share repurchase program, and income tax payments.
Full Year 2023 Financial
Guidance
Vertex today raised its full year 2023 CF product revenue
guidance to approximately $9.85 billion. Vertex’s CF product
revenue guidance includes expectations in the U.S. for continued
performance of TRIKAFTA in ages 6+ and the launch of TRIKAFTA in
the 2-5 age group, as well as the continued uptake of
TRIKAFTA/KAFTRIO in multiple countries internationally. This
guidance continues to include an approximate 150-basis-point
negative impact from changes in foreign currency rates, inclusive
of our foreign exchange risk management program. Vertex is also
reiterating its guidance for full year 2023 GAAP and non-GAAP
combined R&D, acquired IPR&D and SG&A expenses and
updating its guidance for full year non-GAAP effective tax
rate.
Vertex’s financial guidance is summarized below:
Current FY 2023
Previous FY 2023
CF product revenues
~$9.85 billion
$9.7 to $9.8 billion
Combined GAAP R&D, Acquired
IPR&D and SG&A expenses (2)
Unchanged
$4.55 to $4.8 billion
Combined Non-GAAP R&D, Acquired
IPR&D and SG&A expenses (2)
Unchanged
$4.1 to $4.2 billion
Non-GAAP effective tax rate
20 to 21%
21% to 22%
Key Business Highlights
Cystic Fibrosis (CF) Marketed
Products
Vertex anticipates the number of CF patients taking our
medicines will continue to grow, including through new approvals
and reimbursement for the treatment of younger patients. Recent and
anticipated progress includes:
- Health Canada granted market authorization for the use of
TRIKAFTA in children with CF 2 to 5 years of age who have at least
one F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. With this approval,
approximately 330 patients are now eligible for the first time for
a medicine that treats the underlying cause of their disease.
Vertex is currently working with government and private payers in
Canada to support access for this new patient population as soon as
possible.
- The European Medicines Agency’s (EMA) Committee for Medicinal
Products for Human Use (CHMP) adopted a positive opinion for the
label extension of KAFTRIO in children with CF 2 to 5 years of age
who have at least one F508del mutation in the CFTR gene. If this
label extension is approved by the European Commission, more than
1,200 children would be newly eligible for treatment.
Potential Near-Term Launch
Opportunities
Vertex is preparing for the following near-term potential new
product launches:
- Exagamglogene autotemcel (exa-cel) in sickle cell disease
(SCD) and transfusion-dependent beta thalassemia (TDT): Exa-cel
is a precise non-viral ex vivo CRISPR gene-editing therapy, which
is being developed in collaboration with CRISPR Therapeutics as a
potential one-time functional cure for SCD and TDT.
- The FDA has assigned exa-cel Prescription Drug User Fee Act
(PDUFA) action dates of December 8, 2023, for SCD and March 30,
2024, for TDT. Exa-cel's BLA for SCD was granted Priority Review by
the FDA.
- Reviews of the filings for exa-cel with the EMA in the E.U. and
the MHRA in the U.K. are well underway, with regulatory decisions
expected in the coming months.
- Vertex submitted a marketing authorization application for
exa-cel to the Saudi Food and Drug Authority (SFDA). Exa-cel is the
first investigational medicine to receive Breakthrough Designation
from the SFDA, reflecting the high unmet need for patients with SCD
and TDT in the Kingdom of Saudi Arabia.
- Clinical data from the CLIMB-111 and CLIMB-121 Phase 1/2/3
studies in TDT and SCD, respectively, were accepted for oral
presentation at the upcoming American Society of Hematology (ASH)
Annual Meeting and Exposition. Additionally, five abstracts were
accepted for poster presentation.
- Vanzacaftor/tezacaftor/deutivacaftor, the next-in-class
triple combination, in cystic fibrosis.
- Vertex remains on track to complete the pivotal SKYLINE 102 and
SKYLINE 103 studies, which are evaluating the efficacy and safety
of vanzacaftor/tezacaftor/deutivacaftor relative to TRIKAFTA in
patients with CF 12 years of age and older, and the RIDGELINE study
of vanzacaftor/tezacaftor/deutivacaftor in children with CF 6 to 11
years of age, by the end of 2023.
- Vertex expects to share the results of all three studies in
early 2024.
- VX-548 in acute pain: Vertex has discovered multiple
selective small molecule inhibitors of NaV1.8 with the objective of
creating a new class of pain medicines that have the potential to
provide effective pain relief across a variety of pain states,
including acute pain, without the limitations of opioids and other
currently available medicines.
- For its lead compound, VX-548, for the treatment of moderate to
severe acute pain, Vertex has completed the randomized, controlled
Phase 3 pivotal trial in abdominoplasty and continues to enroll the
randomized, controlled Phase 3 trial in bunionectomy and a
single-arm safety and effectiveness trial. Vertex remains on track
to complete the pivotal program for acute pain in late 2023.
- Vertex expects to share the results of all three studies in
early 2024.
R&D Pipeline
Vertex is delivering on a diversified pipeline of potentially
transformative medicines for serious diseases utilizing a range of
modalities. Recent and anticipated progress for programs in
clinical development is summarized below.
Cystic Fibrosis
Vertex continues to pursue an mRNA therapy for the approximately
5,000 patients who cannot benefit from CFTR modulators alone, as
well as next-in-class, small molecule CFTR modulator therapies.
- Vertex is enrolling patients in a single ascending dose (SAD)
clinical trial for VX-522, the CFTR mRNA therapeutic that Vertex is
developing in collaboration with Moderna and expects to complete
the SAD and initiate a multiple ascending dose (MAD) study by the
end of 2023.
- Consistent with its overall strategy, Vertex is advancing
additional CFTR potentiators and correctors through clinical
development with the goal of bringing more patients to carrier
levels of CFTR function. Vertex takes a portfolio approach to all
of its programs and is also advancing additional research-stage
CFTR modulators and genetic therapies for CF.
Sickle Cell Disease and Beta Thalassemia
- Vertex continues to enroll and dose patients in two global
Phase 3 studies of exa-cel in patients 5 to 11 years of age with
SCD or TDT.
- Additionally, Vertex continues to work on preclinical assets
for gentler conditioning for exa-cel, which could broaden the
eligible patient population for exa-cel to more than 150,000
people.
Acute and Neuropathic Pain
- The Phase 2, 12-week, dose-ranging study of VX-548 in patients
with diabetic peripheral neuropathy (DPN), a common form of
peripheral neuropathic pain, has completed. Vertex expects to share
results in late 2023. DPN represents approximately 20 percent of
the total peripheral neuropathic pain patient population.
- In addition, Vertex plans to initiate another Phase 2 study of
VX-548 in peripheral neuropathic pain in late 2023. This study will
evaluate the efficacy of VX-548 in patients with lumbosacral
radiculopathy (LSR), a second type of peripheral neuropathic pain
with high unmet need and no approved therapies. LSR represents over
40 percent of the total peripheral neuropathic pain patient
population.
- Consistent with its overall strategy, Vertex takes a portfolio
approach with all its programs and is advancing additional NaV1.8
inhibitors, as well as NaV1.7 inhibitors, through research and
earlier stages of development for pain.
APOL1-Mediated Kidney Disease (AMKD)
Vertex has discovered multiple oral, small molecule inhibitors
of APOL1 function, pioneering a new class of medicines that target
an underlying genetic driver of kidney disease.
- Vertex continues to enroll and dose patients in the pivotal
program for inaxaplin, a single Phase 2/3 clinical trial in
patients with AMKD and expects to complete enrollment in the Phase
2B dose-ranging portion of the study in 2023.
Type 1 Diabetes (T1D)
Vertex is evaluating cell therapies using stem-cell derived,
fully differentiated, insulin-producing islet cells to replace the
endogenous insulin-producing islet cells that are destroyed in
people with T1D, with the goal of developing a potential one-time
functional cure for this disease. Vertex has three programs that
use these fully differentiated cells.
- VX-880, fully differentiated islet cells with standard
immunosuppression:
- Vertex established proof-of-concept for VX-880 in 2022. More
recently, Vertex presented positive, updated clinical data from the
ongoing VX-880 Phase 1/2 study at the European Association for the
Study of Diabetes (EASD) Annual Meeting in October.
- The Phase 1/2 study is designed as a sequential, three-part
clinical trial to evaluate the safety and efficacy of VX-880.
Vertex has completed enrollment in Part C of the study.
- VX-264, fully differentiated islet cells encapsulated in
immunoprotective device:
- VX-264 uses the same stem cell-derived, fully differentiated
islets from the VX-880 program, which are encapsulated in a novel
device designed to shield the cells from the body’s immune system
and obviate the need for immunosuppressive therapy.
- Vertex is studying VX-264 in a Phase 1/2 clinical trial that is
a sequential, multi-part study to evaluate the safety,
tolerability, and efficacy of VX-264.
- Hypoimmune, edited fully differentiated islet cells:
- Vertex’s hypoimmune cell program involves using CRISPR/Cas9 to
gene edit the same stem cell-derived, fully differentiated islets
used in the VX-880 and VX-264 programs to cloak the cells from the
immune system. This program is progressing through the research
stage.
Alpha-1 Antitrypsin Deficiency
Vertex is working to address the underlying genetic cause of
alpha-1 antitrypsin (AAT) deficiency by developing novel small
molecule correctors of Z-AAT protein folding, with a goal of
increasing the secretion of functional AAT into the blood and
addressing both the lung and the liver aspects of AAT
deficiency.
- Vertex has discontinued development of VX-864, a
first-generation AATD corrector, due to non-serious rash events in
some patients.
- Vertex continues to enroll and dose healthy volunteers with
VX-634 and VX-668, the next-wave of investigational small molecule
AAT correctors with significantly improved potency and drug-like
properties compared with the first-generation AATD correctors.
Duchenne Muscular Dystrophy
Vertex is pursuing preclinical research in Duchenne muscular
dystrophy (DMD), using innovative approaches to target the
underlying cause of disease, with the goal of transforming the
lives of these patients by restoring near-full-length dystrophin
and muscle function.
- Based on pre-clinical data generated to date, Vertex has
determined that additional in vitro and animal studies of the
delivery system for its gene editing components will be required
prior to advancing the program into clinical development.
- Consistent with its portfolio approach to research and
development, Vertex is also using the learnings from its
first-generation vectors to design next-generation delivery systems
for in vivo gene editing in DMD.
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 from Vertex's pre-tax income (i)
stock-based compensation expense, (ii) gains or losses related to
the fair value of the company's strategic investments, (iii)
increases or decreases in the fair value of contingent
consideration, (iv) acquisition-related costs, (v) an intangible
asset impairment charge and (vi) other adjustments. The company's
non-GAAP financial results also exclude from its provision for
income taxes the estimated tax impact related to its non-GAAP
adjustments to pre-tax income described above and certain discrete
items. These results should not be viewed as a substitute for the
company’s GAAP results and are provided as a complement to results
provided in accordance with GAAP. 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 that the company believes is
helpful to an understanding of its ongoing business. Management
also uses these non-GAAP financial measures to establish budgets
and operational goals that are communicated internally and
externally, to manage the company's business and to evaluate its
performance. The company’s calculation of non-GAAP financial
measures likely differs from the calculations used by other
companies. A reconciliation of the GAAP financial results to
non-GAAP financial results is included in the attached financial
information.
The company provides guidance regarding combined R&D,
Acquired IPR&D and SG&A expenses and effective tax rate on
a non-GAAP basis. Unless otherwise noted, the guidance regarding
combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A
expenses does not include estimates associated with any potential
future business development transactions, including collaborations,
asset acquisitions and/or licensing of third-party intellectual
property rights. The company does not provide guidance regarding
its GAAP effective tax rate because it is unable to forecast with
reasonable certainty the impact of excess tax benefits related to
stock-based compensation and the possibility of certain discrete
items, which could be material.
Vertex Pharmaceuticals
Incorporated
Consolidated Statements of
Income
(in millions, except per share
amounts)(unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
Product revenues, net
$
2,483.5
$
2,334.3
$
7,351.5
$
6,628.0
Costs and expenses:
Cost of sales
318.7
289.4
894.2
797.0
Research and development expenses
810.0
645.0
2,338.3
1,846.2
Acquired in-process research and
development expenses
51.7
29.0
509.3
92.9
Selling, general and administrative
expenses
263.8
246.8
767.5
677.3
Change in fair value of contingent
consideration
1.2
(2.6
)
(1.3
)
(59.3
)
Total costs and expenses
1,445.4
1,207.6
4,508.0
3,354.1
Income from operations
1,038.1
1,126.7
2,843.5
3,273.9
Interest income
167.9
46.2
435.2
58.6
Interest expense
(10.9
)
(13.7
)
(33.5
)
(43.2
)
Other (expense) income, net
(15.9
)
17.2
(13.0
)
(133.7
)
Income before provision for income
taxes
1,179.2
1,176.4
3,232.2
3,155.6
Provision for income taxes
143.9
245.9
581.4
652.5
Net income
$
1,035.3
$
930.5
$
2,650.8
$
2,503.1
Net income per common share:
Basic
$
4.01
$
3.63
$
10.29
$
9.78
Diluted
$
3.97
$
3.59
$
10.18
$
9.68
Shares used in per share calculations:
Basic
258.0
256.5
257.7
255.8
Diluted
260.6
259.5
260.4
258.7
Vertex Pharmaceuticals
Incorporated
Product Revenues
(in millions)(unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
TRIKAFTA/KAFTRIO
$
2,274.3
$
2,010.5
$
6,611.4
$
5,665.3
Other CF products
209.2
323.8
740.1
962.7
Product revenues, net
$
2,483.5
$
2,334.3
$
7,351.5
$
6,628.0
Vertex Pharmaceuticals
Incorporated
Reconciliation of GAAP to
Non-GAAP Financial Information
(in millions, except
percentages)(unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
GAAP cost of sales
$
318.7
$
289.4
$
894.2
$
797.0
Stock-based compensation expense
(1.7
)
(2.4
)
(5.4
)
(7.0
)
Non-GAAP cost of sales
$
317.0
$
287.0
$
888.8
$
790.0
GAAP research and development
expenses
$
810.0
$
645.0
$
2,338.3
$
1,846.2
Stock-based compensation expense
(81.1
)
(80.0
)
(231.9
)
(229.9
)
Intangible asset impairment charge (3)
—
—
—
(13.0
)
Acquisition-related costs (4)
(2.9
)
(16.5
)
(8.5
)
(22.1
)
Non-GAAP research and development
expenses
$
726.0
$
548.5
$
2,097.9
$
1,581.2
Acquired in-process research and
development expenses
$
51.7
$
29.0
$
509.3
$
92.9
GAAP selling, general and
administrative expenses
$
263.8
$
246.8
$
767.5
$
677.3
Stock-based compensation expense
(48.1
)
(53.2
)
(135.3
)
(142.9
)
Acquisition-related costs (4)
—
(13.2
)
—
(13.2
)
Non-GAAP selling, general and
administrative expenses
$
215.7
$
180.4
$
632.2
$
521.2
Combined non-GAAP R&D, Acquired
IPR&D and SG&A expenses
$
993.4
$
757.9
$
3,239.4
$
2,195.3
GAAP other (expense) income,
net
$
(15.9
)
$
17.2
$
(13.0
)
$
(133.7
)
Decrease (increase) in fair value of
strategic investments
6.2
(16.7
)
0.2
143.1
Non-GAAP other (expense) income,
net
$
(9.7
)
$
0.5
$
(12.8
)
$
9.4
GAAP provision for income taxes
$
143.9
$
245.9
$
581.4
$
652.5
Tax adjustments (1)
112.9
37.1
159.2
138.0
Non-GAAP provision for income
taxes
$
256.8
$
283.0
$
740.6
$
790.5
GAAP effective tax rate
12.2
%
20.9
%
18.0
%
20.7
%
Non-GAAP effective tax rate
19.4
%
21.4
%
20.5
%
21.6
%
Vertex Pharmaceuticals
Incorporated
Reconciliation of GAAP to
Non-GAAP Financial Information (continued)
(in millions, except per share
amounts)(unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
GAAP operating income
$
1,038.1
$
1,126.7
$
2,843.5
$
3,273.9
Stock-based compensation expense
130.9
135.6
372.6
379.8
Increase (decrease) in fair value of
contingent consideration (3)
1.2
(2.6
)
(1.3
)
(59.3
)
Intangible asset impairment charge (3)
—
—
—
13.0
Acquisition-related costs (4)
2.9
29.7
8.5
35.3
Non-GAAP operating income
$
1,173.1
$
1,289.4
$
3,223.3
$
3,642.7
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
GAAP net income
$
1,035.3
$
930.5
$
2,650.8
$
2,503.1
Stock-based compensation expense
130.9
135.6
372.6
379.8
Decrease (increase) in fair value of
strategic investments
6.2
(16.7
)
0.2
143.1
Increase (decrease) in fair value of
contingent consideration (3)
1.2
(2.6
)
(1.3
)
(59.3
)
Intangible asset impairment charge (3)
—
—
—
13.0
Acquisition-related costs (4)
2.9
29.7
8.5
35.3
Total non-GAAP adjustments to pre-tax
income
141.2
146.0
380.0
511.9
Tax adjustments (1)
(112.9
)
(37.1
)
(159.2
)
(138.0
)
Non-GAAP net income
$
1,063.6
$
1,039.4
$
2,871.6
$
2,877.0
Net income per diluted common share:
GAAP
$
3.97
$
3.59
$
10.18
$
9.68
Non-GAAP
$
4.08
$
4.01
$
11.03
$
11.12
Shares used in diluted per share
calculations:
GAAP and Non-GAAP
260.6
259.5
260.4
258.7
Vertex Pharmaceuticals
Incorporated
Condensed Consolidated Balance
Sheets
(in millions)(unaudited)
September 30, 2023
December 31, 2022
Assets
Cash, cash equivalents and marketable
securities
$
11,928.2
$
10,778.5
Accounts receivable, net
1,538.7
1,442.2
Inventories
688.7
460.6
Prepaid expenses and other current
assets
540.2
553.5
Total current assets
14,695.8
13,234.8
Property and equipment, net
1,124.0
1,108.4
Goodwill and intangible assets
1,691.6
1,691.6
Deferred tax assets
1,729.1
1,246.9
Operating lease assets
310.5
347.4
Long-term marketable securities
1,700.0
112.2
Other long-term assets
475.2
409.6
Total assets
$
21,726.2
$
18,150.9
Liabilities and Shareholders'
Equity
Accounts payable and accrued expenses
$
3,283.2
$
2,430.6
Other current liabilities
316.2
311.5
Total current liabilities
3,599.4
2,742.1
Long-term finance lease liabilities
390.3
430.8
Long-term operating lease liabilities
354.4
379.5
Other long-term liabilities
869.3
685.8
Shareholders' equity
16,512.8
13,912.7
Total liabilities and shareholders'
equity
$
21,726.2
$
18,150.9
Common shares outstanding
257.8
257.0
Notes and Explanations
1: In the three and nine months ended September 30, 2023
and 2022, "Tax adjustments" included the estimated income taxes
related to non-GAAP adjustments to the company's pre-tax income and
excess tax benefits related to stock-based compensation. “Tax
adjustments” also included a $74 million discrete benefit related
to prior tax years resulting from a R&D tax credit study that
was completed during the third quarter of 2023.
2: The difference between the company’s full year 2023
combined GAAP R&D, Acquired IPR&D and SG&A expenses and
combined non-GAAP R&D, Acquired IPR&D and SG&A expenses
guidance relates primarily to $475 million to $590 million of
stock-based compensation expense. Unless otherwise noted, the
guidance regarding combined GAAP and non-GAAP R&D, Acquired
IPR&D and SG&A expenses does not include estimates
associated with any potential future business development
transactions, including collaborations, asset acquisitions and/or
licensing of third-party intellectual property rights.
3: In the three months ended June 30, 2022, the company
revised the scope of certain acquired programs, resulting in a $13
million “Intangible asset impairment charge” and a decrease in the
associated fair value of contingent consideration.
4: "Acquisition-related costs" in the three and nine
months ended September 30, 2023 and 2022 related to costs
associated with the company's acquisition of Exonics and
ViaCyte.
Note:
Amounts may not foot due to rounding.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious diseases. The company has multiple approved medicines
that treat the underlying cause of cystic fibrosis (CF) — a rare,
life-threatening genetic disease — and has several ongoing clinical
and research programs in CF. Beyond CF, Vertex has a robust
clinical pipeline of investigational therapies across a range of
modalities in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1-mediated kidney disease, acute and neuropathic
pain, type 1 diabetes and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in
Boston, with international headquarters in London. Additionally,
the company has research and development sites and commercial
offices in North America, Europe, Australia and Latin America.
Vertex is consistently recognized as one of the industry's top
places to work, including 14 consecutive years on Science
magazine's Top Employers list and one of Fortune’s 100 Best
Companies to Work For. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
LinkedIn, Facebook, Instagram, YouTube and Twitter/X.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, Dr. Kewalramani's
statements in this press release, the information provided
regarding future financial performance and operations, the section
captioned "Full Year 2023 Financial Guidance" and statements
regarding (i) expectations for continued growth in the number of
people eligible and treated with our CF medicines, including newly
eligible younger children, and expansion of treatment options for
the patients who cannot benefit from CFTR modulators alone, (ii)
the expectations, development plans and anticipated timelines for
the company's products and product candidates and pipeline
programs, including expectations for multiple additional near-term
clinical milestones, study designs, patient enrollment, data
availability, potential launches and timing thereof, (iii) the
expectations, plans, and status of potential near-term product
commercial launches, including those for exa-cel in SCD and TDT,
vanzacaftor/tezacaftor/deutivacaftor in CF, and VX-548 in acute
pain, (iv) the expectations related to our exa-cel regulatory
filings and potential approvals in the U.S., E.U., U.K., and the
Kingdom of Saudi Arabia, including anticipated timing of regulatory
decisions, expectations regarding the potential benefits of exa-cel
as a functional cure for SCD and TDT, and our expectation that a
gentler conditioning for exa-cel could broaden the eligible patient
population for exa-cel, (v) expectations to complete both SKYLINE
studies and the RIDGELINE study by the end of 2023 and share the
results of these studies in early 2024, (vi) expectations regarding
our collaboration with Moderna to develop CFTR mRNA therapeutics,
and plans to complete the single-ascending dose study and initiate
the multiple-ascending dose study for VX-522 by the end of 2023,
(vii) expectations regarding the potential benefits and objectives
of our pain program and products, including expectations to
complete the VX-548 pivotal program for the treatment of moderate
to severe acute pain in late 2023 and share results from these
studies in early 2024, expectations to share the results of the
recently completed study of VX-548 in patients with DPN and plans
to initiate a second Phase 2 study in patients with LSR, (viii)
expectations regarding the potential benefits of our AMKD program,
and plans regarding our Phase 2/3 study of inaxaplin, including
expectations to complete enrollment in the Phase 2B dose-ranging
portion of the study in 2023, (ix) expectations regarding our T1D
programs, including the potential benefits of our T1D programs that
use stem-cell derived, fully differentiated islet cells, and
expectations for the advancement of our T1D programs, including
clinical trial designs and clinical progress, (x) our expectations
regarding our goals and the potential benefits of our AAT
deficiency program, plans to continue to advance VX-634 and VX-668
in clinical trials, (xi) our plans and expectations for our DMD
programs, and (xii) plans with respect to our additional earlier
stage research and development programs. 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 risks and uncertainties that could cause actual events or
results to differ materially from those expressed or implied by
such forward-looking statements. Those risks and uncertainties
include, among other things, that the company's expectations
regarding its 2023 full year product revenues, expenses and
effective tax rates may be incorrect (including because one or more
of the company's assumptions underlying its expectations may not be
realized), that the company may not be able to receive regulatory
approval for exa-cel on the expected timeline, or at all, that
external factors may have different or more significant impacts on
the company's business or operations than the company currently
expects, that data from preclinical testing or clinical trials,
especially if based on a limited number of patients, may not be
indicative of final results or available on anticipated timelines,
that patient enrollment in our trials may be delayed, that the
company may not realize the anticipated benefits from our
collaborations with third parties, that data from the company's
development programs may not support registration or further
development of its potential medicines in a timely manner, or at
all, due to safety, efficacy or other reasons, that anticipated
commercial launches may be delayed, if they occur at all, and other
risks listed under the heading “Risk Factors” in Vertex's annual
report and subsequent quarterly reports filed with the Securities
and Exchange Commission (SEC) and available through the company's
website at www.vrtx.com and on the SEC’s website at www.sec.gov.
You should not place undue reliance on these statements, or the
scientific data presented. 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 at 4:30 p.m.
ET. To access the call, please dial (833) 630-2124 (U.S.) or
+1(412) 317-0651 (International) and reference the “Vertex
Pharmaceuticals Third Quarter 2023 Earnings Call.”
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. To ensure a timely connection, it is
recommended that participants register at least 15 minutes prior to
the scheduled webcast. An archived webcast will be available on the
company's website.
(VRTX-E)
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version on businesswire.com: https://www.businesswire.com/news/home/20231106937081/en/
Vertex Contacts: Investor Relations: Susie Lisa,
CFA, 617-341-6108 Manisha Pai, 617-961-1899 Miroslava Minkova,
617-341-6135
Media: 617-341-6992 mediainfo@vrtx.com
Vertex Pharmaceuticals (NASDAQ:VRTX)
過去 株価チャート
から 6 2024 まで 7 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
過去 株価チャート
から 7 2023 まで 7 2024