— Product revenue of $2.69 billion, a 13%
increase compared to Q1 2023; reiterated full year 2024 financial
guidance, including product revenue guidance of $10.55 to $10.75
billion —
— Submitted NDA and MAA filings for vanzacaftor
triple in CF to FDA and EMA, respectively —
— Initiated rolling NDA submission for
suzetrigine (VX-548) in moderate-to-severe acute pain and on track
to complete this quarter —
— Entered into agreement to acquire Alpine
Immune Sciences, including povetacicept, a Phase 3-ready asset in
IgA nephropathy and potential pipeline-in-a-product —
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
reported consolidated financial results for the first quarter ended
March 31, 2024, and reiterated full year 2024 financial
guidance.
“Vertex delivered a strong start to 2024 with 13 percent product
revenue growth and outstanding execution across the business. This
quarter, we continued to expand our leadership in CF including
completion of the regulatory submissions for the vanzacaftor
triple, advanced the global launch of CASGEVY™, and initiated the
rolling submission for suzetrigine in moderate-to-severe acute
pain, while progressing our broad and deep pipeline of potentially
transformative medicines,” said Reshma Kewalramani, M.D., Chief
Executive Officer and President of Vertex. “We also recently
announced the acquisition of Alpine Immune Sciences, a compelling
strategic fit. We look forward to welcoming the Alpine team and
together accelerating the development of povetacicept, a potential
best-in-class treatment for IgAN, and fully exploring
povetacicept’s pipeline-in-a-product potential.”
First Quarter 2024
Results
Product revenue increased 13% to $2.69 billion compared
to the first quarter of 2023, primarily driven in ex-U.S. markets
by the continued strong uptake of TRIKAFTA®/KAFTRIO®, including
label extensions in younger age groups, and in the U.S. by the
continued performance of TRIKAFTA, including the uptake in children
with CF 2 to 5 years of age. Net product revenue in the first
quarter of 2024 increased 8% to $1.52 billion in the U.S. and
increased 21% to $1.17 billion outside the U.S., compared to the
first quarter of 2023.
Combined GAAP and Non-GAAP R&D, Acquired IPR&D and
SG&A expenses were $1.2 billion and $1.0 billion,
respectively, compared to $1.3 billion and $1.2 billion,
respectively, in the first quarter of 2023. The decreases were due
to lower Acquired IPR&D expenses partially offset by increased
investments to support launches of Vertex's therapies globally and
continued investment in support of multiple programs that have
advanced in mid- and late-stage clinical development.
GAAP effective tax rate was 14.0% compared to 21.5% for
the first quarter of 2023, primarily due to a discrete adjustment
to Vertex’s income tax reserves as well as tax benefits related to
stock-based compensation.
Non-GAAP effective tax rate was 17.4% compared to 21.3%
for the first quarter of 2023, primarily due to a discrete
adjustment to Vertex’s income tax reserves. Please refer to Note 1
for further details on Vertex’s GAAP to Non-GAAP tax
adjustments.
GAAP and Non-GAAP net income increased by 57% and 56%,
respectively, compared to the first quarter of 2023, primarily due
to higher product revenues and lower Acquired IPR&D
expenses.
Cash, cash equivalents and total marketable securities as
of March 31, 2024 were $14.6 billion, compared to $13.7 billion as
of December 31, 2023. The increase was primarily due to cash from
operations driven by strong revenue growth, partially offset by
business development payments, and repurchases of Vertex’s common
stock.
Full Year 2024 Financial
Guidance
Vertex today reiterated its full year 2024 financial guidance,
including product revenue guidance of $10.55 to $10.75 billion.
Vertex’s product revenue guidance includes expectations for
continued growth in CF as well as for the launch of CASGEVY in
approved indications and geographies. Vertex’s combined Non-GAAP
R&D, Acquired IPR&D and SG&A expense guidance of $4.3
billion to $4.4 billion includes expectations for continued
investment in multiple mid- and late-stage clinical development
programs, commercial and manufacturing capabilities, and
approximately $125 million of upfront and milestone payments. The
recently announced acquisition of Alpine Immune Sciences is
expected to close in the second quarter. Vertex does not anticipate
adjusting its guidance for Alpine’s operating expenses, other than
the potential impact of purchase accounting.
Vertex’s financial guidance is summarized below:
Current FY 2024
Previous FY 2024
Total product revenues
Unchanged
$10.55 to $10.75 billion
Combined GAAP R&D, Acquired
IPR&D and SG&A expenses (2)
Unchanged
$4.9 to $5.1 billion
Combined Non-GAAP R&D, Acquired
IPR&D and SG&A expenses (2)
Unchanged
$4.3 to $4.4 billion
Non-GAAP effective tax rate
Unchanged
20% to 21%
Key Business Highlights
Marketed Products and Potential
Near-Term Launch Opportunities
Cystic Fibrosis (CF)
Portfolio
Vertex anticipates the number of CF patients taking our
medicines will continue to grow through new approvals and
reimbursement for the treatment of younger patients. Recent and
anticipated progress includes:
- The European Commission has granted approval for KALYDECO for
the treatment of infants with CF ages 1 month to less than 4 months
with specific mutations in the CFTR gene. KALYDECO now represents
the first and only medicine approved in Europe to treat the
underlying cause of CF for this age group.
- In the first quarter, Vertex shared positive data from the
pivotal studies for the next-generation triple combination of
vanzacaftor/tezacaftor/deutivacaftor (the “vanzacaftor triple”),
showing that the two randomized studies in patients 12 years and
older met the primary endpoint and all key secondary endpoints, and
the results in the single-arm study in children ages 6 to 11 were
even more pronounced.
- Vertex has submitted regulatory marketing applications for the
once-daily vanzacaftor triple in people with CF 6 years and older
to the U.S. Food and Drug Administration (FDA), using a priority
review voucher, and to the European Medicines Agency (EMA). Vertex
intends to complete regulatory submissions to the MHRA in Great
Britain, Health Canada, SwissMedic, the Australian Therapeutic
Goods Administration (TGA) and the New Zealand Ministry of Health
for people with CF 6 years of age and older later this year.
CASGEVY for the treatment of sickle
cell disease (SCD) and transfusion-dependent beta thalassemia
(TDT):
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell
therapy for eligible patients with SCD or TDT that has been shown
to reduce or eliminate vaso-occlusive crises (VOCs) for patients
with SCD and transfusion requirements for patients with TDT.
- CASGEVY is approved in the U.S., Great Britain, the European
Union (EU), the Kingdom of Saudi Arabia (KSA), and the Kingdom of
Bahrain (Bahrain) for the treatment of both SCD and TDT. Vertex has
completed regulatory submissions for CASGEVY for SCD and TDT in
Switzerland and Canada; the submission in Canada was granted
priority review.
- As of mid-April, Vertex has activated more than 25 authorized
treatment centers (ATCs) globally and multiple patients across all
regions have initiated cell collection.
- Vertex has signed multiple agreements with both commercial and
government health insurance providers in the U.S. to provide access
to CASGEVY.
- Vertex has also secured reimbursed access for people with SCD
or TDT in KSA and Bahrain, as well as for people with TDT in France
through an early access program.
Suzetrigine (VX-548) for the treatment
of moderate to severe acute pain:
Vertex has discovered multiple selective small molecule
inhibitors of NaV1.8 with the goal of creating a new class of pain
medicines that has the potential to provide effective pain relief
across a variety of pain states, without the limitations of opioids
and other currently available medicines.
- In the first quarter, Vertex shared positive results from the
three Phase 3 trials of suzetrigine in moderate-to-severe acute
pain.
- The FDA has granted a rolling New Drug Application (NDA)
submission to suzetrigine in moderate-to-severe acute pain, and
Vertex has started the rolling submission process. The submission
is on track to be completed in the second quarter of 2024.
Suzetrigine has also been granted FDA Fast Track and Breakthrough
Therapy designations in moderate-to-severe acute pain.
Select Clinical-Stage R&D
Pipeline
Cystic Fibrosis
Vertex continues to pursue next-in-class, small molecule, oral
CFTR modulators for the ~90% of patients who may benefit from such
an approach, as well as a nebulized mRNA therapy for the more than
5,000 people with CF who do not make CFTR protein and cannot
benefit from CFTR modulators.
- Vanzacaftor/tezacaftor/deutivacaftor, the next-in-class
triple oral small molecule combination, in cystic fibrosis
- Vertex plans to initiate a new cohort in the Phase 3 study,
RIDGELINE, in the second half of 2024 in children with cystic
fibrosis ages 2 to 5 years who have at least one F508del mutation
or a mutation responsive to triple combination CFTR
modulators.
- Nebulized mRNA therapy:
- Vertex continues to enroll and dose patients in the multiple
ascending dose (MAD) portion of the Phase 1/2 study of VX-522 in
people with CF.
- Vertex expects to share data from this study in late 2024 or
early 2025.
Sickle Cell Disease and Transfusion-Dependent Beta
Thalassemia
- Vertex has completed enrollment in two global Phase 3 studies
of CASGEVY in people 5 to 11 years of age with SCD or TDT.
- Vertex continues to work on preclinical assets for gentler
conditioning for CASGEVY, which could broaden the eligible patient
population to more than 150,000 people in the U.S. and Europe.
Acute Pain
- Vertex plans to initiate a Phase 2 study with an oral
formulation of VX-993, a next-generation selective NaV1.8
inhibitor, for the treatment of moderate to severe acute pain in
2024.
- The FDA cleared the investigational new drug (IND) application
for an intravenous formulation of VX-993 for the treatment of
moderate to severe acute pain, and Vertex has initiated a Phase 1
trial.
- Consistent with its commitment to serial innovation and
leadership in pain, Vertex also continues to develop NaV1.7
inhibitors, for stand-alone use or in combination with NaV1.8
inhibitors, for both acute and peripheral neuropathic pain.
Peripheral Neuropathic Pain (PNP)
- Following a successful end-of-Phase 2 meeting with the FDA,
Vertex is on track to initiate the Phase 3 pivotal program of
suzetrigine in patients with painful diabetic peripheral neuropathy
(DPN), a type of PNP, in the second half of 2024. The FDA recently
granted suzetrigine Breakthrough Therapy designation in this
indication.
- Vertex continues to enroll and dose patients in its Phase 2
study of suzetrigine in painful lumbosacral radiculopathy (LSR),
representing ~40% of the PNP category. Vertex is on track to
complete enrollment in the Phase 2 study by the end of 2024.
- Vertex anticipates initiating a Phase 2 study with an oral
formulation of VX-993 for the treatment of PNP in 2024.
APOL1-Mediated Kidney Disease (AMKD)
Vertex has discovered and advanced multiple oral, small molecule
inhibitors of APOL1 function, pioneering a new class of medicines
that targets an underlying genetic driver of this kidney
disease.
- Based on the totality of the unblinded data reviewed by the
Independent Data Safety Monitoring Committee (IDMC), Vertex
advanced into the Phase 3 portion of the global Phase 2/3 pivotal
clinical trial in patients with AMKD, in which a 45 mg once-daily
oral dose of inaxaplin will be compared to placebo, on top of
standard of care.
- In addition, based on the IDMC review, the trial has been
expanded to include adolescents 10 to 17 years of age with
AMKD.
- The study is designed to have a pre-planned interim analysis at
Week 48 evaluating eGFR slope, supported by a percent change from
baseline in proteinuria, in the inaxaplin arm versus placebo. If
positive, the interim analysis may serve as the basis for Vertex to
seek accelerated approval in the U.S.
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.
- VX-880, fully differentiated islet cells with standard
immunosuppression:
- Based on the totality of evidence reviewed by the IDMC, the
Phase 1/2 study in people with T1D and impaired awareness of
hypoglycemia and recurrent hypoglycemic events has resumed
dosing.
- Vertex has completed enrollment in Parts A, B, and C of the
global 17-patient study and expects to complete dosing soon.
- Vertex plans to present updated data from the ongoing Phase 1/2
study at the American Diabetes Association 84th Scientific Sessions
Conference in June 2024.
- VX-264, fully differentiated islet cells encapsulated in an
immunoprotective device:
- The clinical trial for VX-264, which encapsulates the same
VX-880 islet cells in a novel device designed to eliminate the need
for immunosuppressants, is a multi-part, Phase 1/2 study.
- Vertex has completed Part A of the study and initiated Part
B.
- 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 islet
cells 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.
Myotonic Dystrophy Type 1 (DM1)
Vertex is evaluating multiple approaches that target the
underlying cause of DM1, the most prevalent muscular dystrophy in
adults, with ~110,000 people living with the disease in the U.S.
and Europe, and no approved therapies. Vertex’s lead approach,
VX-670, was in-licensed from Entrada Therapeutics in February 2023
and is an oligonucleotide connected to a cyclic peptide to promote
effective delivery into cells, which holds the potential to address
the underlying cause of DM1.
- The IND in the U.S. for VX-670 has cleared, as have the
clinical trial applications (CTAs) in Canada, the U.K. and the EU,
and the clinical trial notification (CTN) in Australia.
- Enrollment and dosing are underway.
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Vertex is developing small molecule correctors that restore
function to PC1 missense variants, with the goal to address the
underlying cause of ADPKD, the most common genetic kidney disease,
affecting approximately 250,000 people in the U.S. and Europe.
- Vertex has initiated a Phase 1 clinical trial in healthy
volunteers for VX-407, a first-in-class small molecule corrector
that targets the underlying cause of ADPKD in patients with a
subset of PKD1 variants, estimated at ~25,000 (or ~10 percent) of
the overall ~250,000 ADPKD patient population.
Investments in External Innovation
- Vertex and Alpine Immune Sciences entered into a definitive
agreement under which Vertex will acquire Alpine for approximately
$4.9 billion in cash. Vertex has commenced a tender offer to
purchase all of the outstanding shares of common stock of Alpine
for $65 per share in cash. The transaction is anticipated to close
in the second quarter of 2024. Alpine’s lead molecule,
povetacicept, is a highly potent and effective dual antagonist of
BAFF (B cell activating factor) and APRIL (a proliferation-inducing
ligand). Through Phase 2 development, povetacicept has shown
potential best-in-class efficacy in IgA nephropathy (IgAN), a
serious, progressive, autoimmune disease of the kidney that can
lead to end-stage-renal disease. There are currently no approved
therapies that target the underlying cause of IgAN, which is the
most common cause of primary (idiopathic) glomerulonephritis
worldwide, affecting approximately 130,000 people in the U.S.
alone. Povetacicept is on track to enter Phase 3 clinical
development in IgAN in the second half of 2024. Phase 1b/2 studies
in autoimmune renal diseases and cytopenias are ongoing with data
expected later this year.
- Vertex achieved a clinical milestone for VX-670 in DM1 in the
first quarter of 2024, resulting in a $75 million milestone payable
to Entrada.
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) intangible asset
amortization expense, (iii) gains or losses related to the fair
value of the company's strategic investments, (iv) increases or
decreases in the fair value of contingent consideration, (v)
acquisition-related costs, 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 March
31,
2024
2023
Product revenues, net
$
2,690.6
$
2,374.8
Costs and expenses:
Cost of sales
342.6
266.9
Research and development expenses
789.1
742.6
Acquired in-process research and
development expenses
76.8
347.1
Selling, general and administrative
expenses
342.7
241.1
Change in fair value of contingent
consideration
(0.1
)
(1.9
)
Total costs and expenses
1,551.1
1,595.8
Income from operations
1,139.5
779.0
Interest income
181.2
122.6
Interest expense
(10.4
)
(11.4
)
Other (expense) income, net
(31.2
)
1.3
Income before provision for income
taxes
1,279.1
891.5
Provision for income taxes
179.5
191.7
Net income
$
1,099.6
$
699.8
Net income per common share:
Basic
$
4.26
$
2.72
Diluted
$
4.21
$
2.69
Shares used in per share calculations:
Basic
258.2
257.4
Diluted
261.1
260.3
Vertex Pharmaceuticals
Incorporated
Product Revenues
(in millions)(unaudited)
Three Months Ended March
31,
2024
2023
TRIKAFTA/KAFTRIO
$
2,483.6
$
2,096.7
Other CF products
207.0
278.1
Product revenues, net
$
2,690.6
$
2,374.8
Vertex Pharmaceuticals
Incorporated
Reconciliation of GAAP to
Non-GAAP Financial Information
(in millions, except
percentages)(unaudited)
Three Months Ended March
31,
2024
2023
GAAP cost of sales
$
342.6
$
266.9
Stock-based compensation expense
(1.8
)
(1.9
)
Intangible asset amortization expense
(5.0
)
—
Non-GAAP cost of sales
$
335.8
$
265.0
GAAP research and development
expenses
$
789.1
$
742.6
Stock-based compensation expense
(119.4
)
(76.3
)
Acquisition-related costs
—
(2.8
)
Non-GAAP research and development
expenses
$
669.7
$
663.5
Acquired in-process research and
development expenses
$
76.8
$
347.1
GAAP selling, general and
administrative expenses
$
342.7
$
241.1
Stock-based compensation expense
(70.7
)
(44.2
)
Non-GAAP selling, general and
administrative expenses
$
272.0
$
196.9
Combined non-GAAP R&D, Acquired
IPR&D and SG&A expenses
$
1,018.5
$
1,207.5
GAAP other (expense) income,
net
$
(31.2
)
$
1.3
Decrease (increase) in fair value of
strategic investments
27.0
(6.4
)
Non-GAAP other expense, net
$
(4.2
)
$
(5.1
)
GAAP provision for income taxes
$
179.5
$
191.7
Tax adjustments (1)
81.6
22.7
Non-GAAP provision for income
taxes
$
261.1
$
214.4
GAAP effective tax rate
14.0
%
21.5
%
Non-GAAP effective tax rate
17.4
%
21.3
%
Vertex Pharmaceuticals
Incorporated
Reconciliation of GAAP to
Non-GAAP Financial Information (continued)
(in millions, except per share
amounts)(unaudited)
Three Months Ended March
31,
2024
2023
GAAP operating income
$
1,139.5
$
779.0
Stock-based compensation expense
191.9
122.4
Intangible asset amortization expense
5.0
—
Decrease in fair value of contingent
consideration
(0.1
)
(1.9
)
Acquisition-related costs
—
2.8
Non-GAAP operating income
$
1,336.3
$
902.3
GAAP net income
$
1,099.6
$
699.8
Stock-based compensation expense
191.9
122.4
Intangible asset amortization expense
5.0
—
Decrease (increase) in fair value of
strategic investments
27.0
(6.4
)
Decrease in fair value of contingent
consideration
(0.1
)
(1.9
)
Acquisition-related costs
—
2.8
Total non-GAAP adjustments to pre-tax
income
223.8
116.9
Tax adjustments (1)
(81.6
)
(22.7
)
Non-GAAP net income
$
1,241.8
$
794.0
Net income per diluted common share:
GAAP
$
4.21
$
2.69
Non-GAAP
$
4.76
$
3.05
Shares used in diluted per share
calculations:
GAAP and Non-GAAP
261.1
260.3
Notes
1: In the three months ended March 31, 2024 and 2023,
"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.
2: The difference between the company’s full year 2024
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 $600 million to $700 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.
Vertex Pharmaceuticals
Incorporated
Condensed Consolidated Balance
Sheets
(in millions)(unaudited)
March 31, 2024
December 31, 2023
Assets
Cash, cash equivalents and marketable
securities
$
10,171.3
$
11,218.3
Accounts receivable, net
1,793.2
1,563.4
Inventories
813.1
738.8
Prepaid expenses and other current
assets
511.1
623.7
Total current assets
13,288.7
14,144.2
Property and equipment, net
1,172.8
1,159.3
Goodwill and intangible assets, net
1,922.9
1,927.9
Deferred tax assets
1,963.0
1,812.1
Operating lease assets
312.9
293.6
Long-term marketable securities
4,381.4
2,497.8
Other long-term assets
875.7
895.3
Total assets
$
23,917.4
$
22,730.2
Liabilities and Shareholders'
Equity
Accounts payable and accrued expenses
$
3,147.3
$
3,020.2
Other current liabilities
648.6
527.2
Total current liabilities
3,795.9
3,547.4
Long-term finance lease liabilities
361.5
376.1
Long-term operating lease liabilities
359.8
348.6
Other long-term liabilities
853.6
877.7
Shareholders' equity
18,546.6
17,580.4
Total liabilities and shareholders'
equity
$
23,917.4
$
22,730.2
Common shares outstanding
258.3
257.7
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 approved medicines that
treat the underlying causes of multiple chronic, life-shortening
genetic diseases — cystic fibrosis, sickle cell disease and
transfusion-dependent beta thalassemia — and continues to advance
clinical and research programs in these diseases. Vertex also 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 APOL1-mediated kidney
disease, acute and neuropathic pain, type 1 diabetes, myotonic
dystrophy type 1 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, Latin America and the
Middle East. 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 that are
subject to risks, uncertainties and other factors. All statements
other than statements of historical fact are statements that could
be deemed forward-looking statements, including all statements
regarding the intent, belief, or current expectation of Vertex and
members of the Vertex senior management team. Forward-looking
statements are not purely historical and may be accompanied by
words such as “anticipates,” “may,” “forecasts,” “expects,”
“intends,” “plans,” “potentially,” “believes,” “seeks,”
“estimates,” and other words and terms of similar meaning. Such
statements include, without limitation, Dr. Kewalramani's
statements in this press release, the information provided
regarding future financial performance and operations, the section
captioned “Full Year 2024 Financial Guidance” and statements
regarding (i) expectations for Vertex’s continued growth in CF,
including through new approvals and reimbursements for the
treatment of younger patients, (ii) expectations, plans, and status
of the potential near-term commercial launch of the vanzacaftor
triple, including our plans to submit regulatory filings in Great
Britain, Canada, Switzerland, Australia and New Zealand in 2024,
(iii) our beliefs regarding the anticipated benefits of CASGEVY,
expectations around activation of ATCs, and plans to secure
additional reimbursed access outside of the U.S., (iv) expectations
regarding the potential benefits and commercial success of the
product candidates in our pain program, (v) expectations, plans,
and status of the potential near-term commercial launch of
suzetrigine for the treatment of moderate-to-severe acute pain,
including our plans to complete regulatory submissions in the
second quarter of 2024, (vi) plans to initiate a Phase 3 RIDGELINE
study for the vanzacaftor triple in children with CF ages 2 to 5 in
the second half of 2024, (vii) expectations for our VX-522 Phase
1/2 study, including the potential benefits of this nebulized mRNA
therapy and expectations to share data in late 2024 or early 2025,
(viii) expectations regarding our SCD and TDT program, including
expectations that a gentler conditioning for CASGEVY could broaden
the eligible patient population to more than 150,000 people, (ix)
expectations to initiate the Phase 3 pivotal program of suzetrigine
in patients with DPN in the second half of 2024, expectations to
complete enrollment in the Phase 2 study of suzetrigine in LSR by
the end of 2024, and expectations to initiate a Phase 2 study with
an oral formulation of VX-993 for the treatment of PNP in 2024, (x)
plans to initiate a Phase 2 study with an oral formulation of
VX-993 for the treatment of acute pain in 2024, and plans to
continue to develop NaV1.7 inhibitors for both acute pain and PNP,
(xi) expectations regarding the potential benefits of our AMKD
program, including plans for our global Phase 2/3 pivotal clinical
trial evaluating inaxaplin in patients with AMKD, study designs and
our expectations that the interim analysis of this study may serve
as the basis for accelerated approval in the U.S., (xii)
expectations regarding our T1D programs, the status of our Phase
1/2 study of VX-880, including expectations for completion of
dosing and plans to present data at an upcoming medical conference,
(xiii) the potential benefits and clinical status of VX-670 for the
treatment in people with DM1, (xiv) expectations regarding our
ADPKD program, including the potential benefits of VX-407 as a
first-in-class small molecule corrector and our beliefs regarding
the targeted patient population, and (xv) expectations, plans, and
the anticipated timeline for the pending acquisition of Alpine
Immune Sciences, Inc., including with respect to Alpine, and the
therapeutic scope of and the potential benefits of povetacicept,
our beliefs regarding povetacicept’s target patient population, and
our beliefs regarding the clinical progress and availability of
clinical data from the current Alpine pipeline. 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 2024 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 adequate reimbursement or additional regulatory
approvals for CASGEVY on the expected timeline, or at all, that we
are unable to successfully develop, obtain approval or
commercialize suzetrigine as a treatment for acute or neuropathic
pain, 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 Vertex may not be able to complete, successfully integrate, or
profit from the acquisition of Alpine Immune Sciences, Inc., 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, and that anticipated
commercial launches may be delayed, if they occur at all.
Forward-looking statements in this press release should be
evaluated together with the many uncertainties that affect Vertex’s
business, particularly those risks listed under the heading “Risk
Factors” and the other cautionary factors discussed in Vertex’s
periodic reports filed with the SEC, including Vertex’s annual
report on Form 10-K for the year ended December 31, 2023, and its
quarterly reports on Form 10-Q and current reports on Form 8-K, all
of which are 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.
Additional Information about the Acquisition and Where to
Find It
The tender offer for the outstanding shares of common stock of
Alpine Immune Sciences, Inc. referenced in this communication
commenced on April 22, 2024. This communication is for
informational purposes only and is neither an offer to purchase nor
a solicitation of an offer to sell shares of Alpine Immune
Sciences, Inc., nor is it a substitute for any tender offer
materials that Vertex or Alpine Immune Sciences, Inc. have filed
with the SEC. On April 22, 2024, when the tender offer commenced,
Vertex filed with the SEC a Tender Offer Statement on Schedule TO
which included an Offer to Purchase, a related Letter of
Transmittal and certain other tender offer documents (together, the
“Tender Offer Materials”), and Alpine
Immune Sciences, Inc. filed with the SEC a
Solicitation/Recommendation Statement on Schedule 14D-9 (the
“Solicitation/Recommendation
Statement”) with respect to the tender offer. ALPINE IMMUNE
SCIENCES, INC. SECURITY HOLDERS ARE URGED TO READ THE TENDER OFFER
MATERIALS AND THE SOLICITATION/RECOMMENDATION STATEMENT BECAUSE
THEY CONTAIN IMPORTANT INFORMATION WHICH SHOULD BE READ CAREFULLY
BEFORE ANY DECISION IS MADE WITH RESPECT TO THE TENDER OFFER. The
Tender Offer Materials and the Solicitation/Recommendation
Statement are available for free at the SEC’s website at
www.sec.gov. Additional copies of the Tender Offer Materials can be
obtained free of charge under the “Investors” section of Vertex’s
website at
https://investors.vrtx.com/financial-information/sec-filings or by
contacting Vertex by phone at (617) 341-6108, by email at
Investorinfo@VRTX.com, or by directing requests for such materials
to the information agent for the offer, which is named in the
Tender Offer Materials. In addition to the Tender Offer Materials
and the Solicitation/Recommendation Statement, Alpine Immune
Sciences, Inc. and Vertex file periodic reports and other
information with the SEC. Vertex’s and Alpine Immune Sciences,
Inc.’s filings with the SEC are also available for free to the
public from commercial document-retrieval services, at the website
maintained by the SEC at www.sec.gov, and their respective investor
relations websites.
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 First Quarter 2024 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/20240506449207/en/
Vertex: 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)
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