– Treatment with the once-daily vanza triple
CFTR modulator regimen met all primary and key secondary endpoints
in two randomized controlled trials in people with CF ages 12 years
and older –
– Results were more pronounced in the
single-arm study in children ages 6 to 11 years, demonstrating the
potential that treating early in life may prevent disease
development –
– Vanza triple was generally well tolerated
across all three studies –
– Vertex plans to file for approval with global
regulators for people with CF ages 6 years and older by mid-2024,
and use priority review voucher in the U.S. –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced positive results from its once-daily
vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”) program,
the most comprehensive Phase 3 pivotal program ever conducted by
Vertex for the treatment of cystic fibrosis (CF), a progressive,
multi-organ disease caused by dysfunction of the CFTR protein. The
Phase 3 program included two randomized, double-blind,
active-controlled, 52-week trials, SKYLINE 102 and SKYLINE 103,
evaluating the efficacy of vanzacaftor (20 mg)/tezacaftor (100
mg)/deutivacaftor (250 mg) once daily in people with CF ages 12
years and older who have at least one F508del mutation or a
mutation responsive to triple combination CFTR modulators (CFTRm),
compared to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor). A third Phase 3 single-arm, 24‑week, open-label study,
RIDGELINE 105, evaluated the safety and efficacy of the vanza
triple in children with CF ages 6 to 11 years with at least one
mutation responsive to triple combination CFTRm.
In SKYLINE 102 and SKYLINE 103, following a 4-week run-in on
TRIKAFTA, baseline measurements of percent predicted forced
expiratory volume in 1 second (ppFEV1), sweat chloride (SwCl) and
other efficacy parameters were obtained, after which patients were
randomized to either the vanza triple or TRIKAFTA. As in the
SKYLINE trials, all children in the RIDGELINE 105 study received at
least 4 weeks of TRIKAFTA to establish a baseline for ppFEV1, SwCl
and other efficacy parameters prior to receiving vanza triple.
In both SKYLINE 102 and SKYLINE 103, the primary endpoint of
absolute change from baseline in ppFEV1 through week 24 was met and
showed that treatment with vanza triple was non-inferior to
treatment with TRIKAFTA.
The key secondary endpoints in SKYLINE 102 and SKYLINE 103 were
absolute change from baseline in SwCl through week 24 compared to
TRIKAFTA; proportion of patients pooled across the two trials, with
SwCl below 60 mmol/L through week 24 compared to TRIKAFTA; and
proportion of patients pooled across the two trials, with SwCl
below 30 mmol/L through week 24 compared to TRIKAFTA.
Head-to-head against TRIKAFTA, on the first key secondary
endpoint, the vanza triple was superior in reducing SwCl levels in
SKYLINE 102 and SKYLINE 103. In the second and third key secondary
endpoints, which were pooled across SKYLINE 102 and SKYLINE 103,
the vanza triple achieved superiority in the proportion of patients
below 60 mmol/L (the diagnostic threshold for CF) and below 30
mmol/L (carrier level) compared to TRIKAFTA.
The results from other secondary endpoints were consistent with
results of the primary and key secondary endpoints. Additionally,
the results at 52 weeks were consistent with results at 24
weeks.
The primary endpoint in the RIDGELINE 105 study in children 6 to
11 years old was safety. On the secondary endpoint measuring the
absolute change in mean SwCl levels through week 24, the vanza
triple reduced SwCl by -8.6 mmol/L compared to a baseline on
TRIKAFTA. 95% of children achieved SwCl levels below 60 mmol/L and
the majority of children treated with the vanza triple achieved
normal levels of CFTR function with SwCl levels below 30
mmol/L.
Treatment with the vanza triple was well tolerated in all three
studies, and the safety was similar between the vanza triple and
TRIKAFTA treatment groups in SKYLINE 102 and SKYLINE 103. The
safety of the vanza triple in children 6 to 11 years old was
similar to the safety in people 12 years of age and older.
“We are very pleased with today’s results, which demonstrate the
vanza triple is non-inferior to TRIKAFTA in improving lung function
and superior to TRIKAFTA in lowering levels of sweat chloride in
people living with CF, setting a new standard for the level of CFTR
protein function achievable, and raising the very high bar set by
TRIKAFTA,” said Carmen Bozic, M.D., Executive Vice President,
Global Medicines Development and Medical Affairs, and Chief Medical
Officer at Vertex. “We look forward to submitting our application
to regulators with the aim of bringing this potential medicine to
patients as quickly as possible.”
“I have been working as a pediatric pulmonologist for more than
four decades and have seen first-hand the dramatic impact of CFTR
modulators on people with CF, transforming CF from a
life-shortening disease to today, where we see the potential for
halting the disease before it starts. These results were
particularly striking in the pediatric study where 95% of children
achieved a SwCl level below 60 mmol/L, the diagnostic cut-off for a
positive test for CF, and more than 50% of children achieved a SwCl
level below carrier levels where they may see no symptoms of
disease at all,” said Bonnie Ramsey, M.D., Professor Emerita of
Pediatrics, University of Washington School of Medicine, Senior
Consultant to the CF Foundation Therapeutics Development Network
and Co-Chair of Vertex’s CFTR Modulator Steering Committee. “The
efficacy seen with the vanza triple gives me great hope for CF
patients in the future.”
Efficacy Results:
In both active-controlled trials SKYLINE 102 and SKYLINE 103 in
people with CF ages 12 years and older, the primary endpoint of
absolute change from baseline in ppFEV1 through week 24 was met.
All patients received TRIKAFTA during a 4-week run-in prior to
randomization, and baseline values were measured at the end of the
TRIKAFTA run-in. The vanza triple was shown to be non-inferior to
TRIKAFTA (SKYLINE 102: LS mean difference of 0.2; 95% CI: -0.7,
1.1; 1-sided P<0.0001 and SKYLINE 103: LS mean difference of
0.2; 95% CI: -0.5, 0.9; 1-sided P<0.0001).
For the first key secondary endpoint, in the trial of people
with CF heterozygous for F508del and a minimal function mutation
(F/MF) (SKYLINE 102), the absolute mean change from baseline in
SwCl through week 24 was ‑7.5 mmol/L for those taking the vanza
triple, compared to +0.9 mmol/L for those taking TRIKAFTA,
demonstrating a statistically significant and clinically meaningful
improvement in CFTR function (LS mean difference of -8.4; 95% CI:
-10.5, -6.3; P<0.0001). In the trial of people with CF with
other mutations responsive to triple combination CFTRm (SKYLINE
103), the absolute mean change from baseline in SwCl through week
24 was ‑5.1 mmol/L for those taking the vanza triple, compared to
-2.3 mmol/L for those taking TRIKAFTA, again demonstrating
statistically significant and clinically meaningful improvement in
CFTR function (LS mean difference of -2.8; 95% CI: -4.7, -0.9;
P=0.0034).
For the second and third key secondary endpoints based on the
pooled analysis across SKYLINE 102 and SKYLINE 103, following
treatment with the vanza triple, 86% of people with CF across both
trials had a SwCl level below the diagnostic threshold of 60 mmol/L
through 24 weeks, compared to 77% of people treated with TRIKAFTA
(odds ratio 2.21; 95% CI: 1.55, 3.15; P<0.0001). Following
treatment with the vanza triple, 31% of people across both trials
had a SwCl level below carrier level of 30 mmol/L through 24 weeks,
compared to 23% of people treated with TRIKAFTA (odds ratio 2.87;
95% CI: 2.00, 4.12; P<0.0001). Consequently, in these combined
trials, there was about a two-times greater likelihood in the odds
of achieving a SwCl level below 60 mmol/L for those treated with
the vanza triple compared to TRIKAFTA and about a three-times
greater likelihood in the odds of achieving a SwCl level below 30
mmol/L for those treated with the vanza triple compared to
TRIKAFTA.
In the single-arm, open-label study in children 6 to 11 years
old with CF and at least one mutation responsive to triple
combination CFTRm (RIDGELINE 105), the vanza triple demonstrated
safety, the primary endpoint. Secondary endpoints included
evaluation of absolute change in SwCl from baseline through week
24, absolute change in ppFEV1 from baseline through week 24, the
proportion of children with SwCl levels of <60 mmol/L through
week 24, and the proportion of children with SwCl <30 mmol/L
through week 24, among other endpoints. Prior to receiving the
vanza triple, all children were on TRIKAFTA for at least 4 weeks,
and the baseline values were measured at the end of the 4-week
run-in. Children with CF in the study maintained their baseline
level of lung function (ppFEV1 of 99.7) with an absolute LS mean
change from baseline through week 24 of 0.0 (95% CI: ‑2.0, 1.9) and
had an absolute mean change in sweat chloride of -8.6 mmol/L (95%
CI: -11.0, -6.3) from baseline levels of 40.4 mmol/L while on
TRIKAFTA.
Following treatment with the vanza triple, 95% of children in
the study had a SwCl level below 60 mmol/L through 24 weeks (95%
CI: 87%, 99%) and 53% of children had a SwCl level below 30 mmol/L
(95% CI: 41%, 64%).
Primary and Key Secondary Outcomes in Active-Controlled Phase
3 Trials in People with CF Ages 12 Years and Older (SKYLINE 102 and
SKYLINE 103)
Primary Endpoint: Absolute Change from
Baseline* in ppFEV1 through Week 24†
Baseline mean (SD)
LS mean change
(SE)
LS mean difference vs.
TRIKAFTA (95% CI)
P for
non-inferiority, one-sided
SKYLINE 102
TRIKAFTA
N=202
67.2 (14.6)
0.3 (0.3)
--
--
Vanza Triple
N=196
67.0 (15.3)
0.5 (0.3)
0.2 (-0.7, 1.1)
<0.0001
SKYLINE 103
TRIKAFTA
N=289
66.4 (14.9)
0.0 (0.2)
--
--
Vanza Triple
N=284
67.2 (14.6)
0.2 (0.3)
0.2 (-0.5, 0.9)
<0.0001
First Key Secondary Endpoint: Absolute
Change from Baseline* in Sweat Chloride through Week 24†
Baseline mean (SD)
LS mean change
(SE)
LS mean difference vs.
TRIKAFTA (95% CI)
P for superiority,
two‑sided
SKYLINE 102
TRIKAFTA
N=202
54.3 (18.2)
0.9 (0.8)
--
--
Vanza Triple
N=196
53.6 (17.0)
-7.5 (0.8)
-8.4 (-10.5, -6.3)
<0.0001
SKYLINE 103
TRIKAFTA
N=289
42.1 (17.9)
-2.3 (0.7)
--
--
Vanza Triple
N=284
43.4 (18.5)
-5.1 (0.7)
-2.8 (-4.7, -0.9)
0.0034
Second Key Secondary Endpoint:
Proportion of Patients with Sweat Chloride <60 mmol/L through
Week 24† (Pooled Across SKYLINE 102 and SKYLINE 103)
Baseline* %
patients
Through Week 24
% patients
Odds ratio** vs.
TRIKAFTA (95% CI)
P for superiority,
two-sided
Pooled TRIKAFTA
N=491
74%
77%
--
--
Pooled Vanza Triple
N=480
76%
86%
2.21 (1.55, 3.15)
<0.0001
Third Key Secondary Endpoint:
Proportion of Patients with Sweat Chloride <30 mmol/L through
Week 24† (Pooled Across SKYLINE 102 and SKYLINE 103)
Baseline* %
patients
Through Week 24
% patients
Odds ratio** vs.
TRIKAFTA (95% CI)
P for superiority,
two-sided
Pooled TRIKAFTA
N=491
21%
23%
--
--
Pooled Vanza Triple
N=480
19%
31%
2.87 (2.00, 4.12)
<0.0001
* Baseline values were measured after a
4-week run-in on TRIKAFTA. † Through Week 24 was defined as average
of measurements at Weeks 16 and 24. ** Estimated by generalized
estimating equation model; odds ratio >1 favors the vanza
triple. CI = confidence interval; LS = least squares; SD = standard
deviation; SE = standard error.
Efficacy Outcomes in a Single Arm, Open-Label Study in
Children with CF Ages 6 to 11 Years (RIDGELINE 105)
Absolute Change from Baseline* in
ppFEV1 through Week 24†
Baseline mean (SD)
LS mean change (SE)
95% CI
Vanza Triple
N=78
99.7 (15.1)
0.0 (1.0)
-2.0, 1.9
Absolute Change from Baseline* in Sweat
Chloride through Week 24†
Baseline mean (SD)
LS mean change (SE)
95% CI
Vanza Triple
N=78
40.4 (20.9)
-8.6 (1.2)
-11.0, -6.3
Proportion of Children with Sweat
Chloride < 60 mmol/L through Week 24†
Baseline* %
children
Through Week 24 %
children
95% CI
Vanza Triple
N=78
84%
95%
87%, 99%
Proportion of Children with Sweat
Chloride < 30 mmol/L through Week 24†
Baseline* %
children
Through Week 24 %
children
95% CI
Vanza Triple
N=78
39%
53%
41%, 64%
* Baseline values were measured
after at least 4 weeks on TRIKAFTA. †Through Week 24 was defined as
average of measurements at Weeks 16 and 24. CI = confidence
interval; LS = least squares; SE = standard error; SD = standard
deviation.
Safety Results:
In SKYLINE 102 and SKYLINE 103, the majority of adverse events
(AEs) were mild to moderate and generally consistent with the
manifestations of CF. The incidence of serious adverse events
(SAEs) and the incidence of AEs leading to treatment
discontinuation was low and balanced between the vanza triple and
TRIKAFTA groups. Overall, the safety data were generally consistent
between the vanza triple and TRIKAFTA.
Common TEAEs* (≥10% in any Treatment Group, Pooled SKYLINE
102 and SKYLINE 103)
Preferred Term
TRIKAFTA N=491 n
(%)
Vanza Triple N=480
n (%)
Subjects with any TEAEs
469 (95.5)
459 (95.6)
Infective pulmonary exacerbation of cystic
fibrosis
158 (32.2)
133 (27.7)
Cough
101 (20.6)
108 (22.5)
COVID-19
127 (25.9)
107 (22.3)
Nasopharyngitis
95 (19.3)
102 (21.3)
Headache
63 (12.8)
76 (15.8)
Upper respiratory tract infection
67 (13.6)
72 (15.0)
Oropharyngeal pain
60 (12.2)
69 (14.4)
Diarrhea
59 (12.0)
58 (12.1)
Influenza
26 (5.3)
52 (10.8)
Pyrexia
50 (10.2)
52 (10.8)
Fatigue
46 (9.4)
51 (10.6)
Nasal congestion
47 (9.6)
48 (10.0)
Sputum increased
50 (10.2)
45 (9.4)
*TEAE: treatment-emergent adverse
events.
In RIDGELINE 105, the majority of AEs were also mild to moderate
and generally consistent with the manifestations of CF. The
incidence of SAEs was low, and the overall safety of the vanza
triple was similar in children 6 to 11 years old as in people ages
12 years and older. The incidence and nature of AEs was also
similar to those seen in previous CFTRm studies of children 6 to 11
years old.
Next Steps
Vertex is on track to make global regulatory submissions by
mid-2024 including a New Drug Application (NDA) to the Food and
Drug Administration and a Marketing Authorization Application with
the European Medicines Agency for people with CF ages 6 years and
older. The company will use a priority review voucher in the U.S.
The priority review voucher entitles the holder to designate an NDA
for priority review, which provides an expedited 6‑month review
instead of the standard 10-month review.
The full data set from these studies will be presented at future
medical meetings later this year.
About the Vanza Triple Phase 3 Program The Phase 3
program in people with cystic fibrosis ages 12 years and older
consisted of two randomized, double-blind, active-controlled
52-week trials, SKYLINE 102 and SKYLINE 103, which evaluated the
safety and efficacy of the vanza triple in comparison to TRIKAFTA.
SKYLINE 102 randomized and dosed 398 people with CF ages 12 years
and older with one F508del mutation and one minimal function
mutation (F/MF). SKYLINE 103 randomized and dosed 573 people with
CF ages 12 years and older who were homozygous for F508del
mutations (F/F), heterozygous for F508del and a gating (F/G) or a
residual function mutation (F/RF) or have at least one other
mutation responsive to triple combination CFTR modulators and no
F508del mutation. All patients received TRIKAFTA during a 4-week
run-in prior to randomization, and the baseline values for all
endpoints were measured at the end of the TRIKAFTA run-in.
The primary endpoint of both SKYLINE 102 and SKYLINE 103 was the
absolute change from baseline in ppFEV1 through week 24, and the
primary analysis was non-inferiority of the vanza triple compared
to TRIKAFTA. The first key secondary endpoint in SKYLINE 102 and
SKYLINE 103 was absolute change from baseline in sweat chloride
through week 24 compared to TRIKAFTA; the second key secondary
endpoint was the proportion of patients pooled across the two
studies, with SwCl below 60 mmol/L through week 24 compared to
TRIKAFTA; and the third key secondary endpoint was the proportion
of patients pooled across two studies, with SwCl below 30 mmol/L
through week 24 compared to TRIKAFTA. All three key secondary
endpoints were evaluated in a hierarchical manner and multiplicity
controlled for superiority to TRIKAFTA.
A third Phase 3 single-arm, open-label study, RIDGELINE 105,
dosed 78 children with CF ages 6 through 11 years and evaluated the
pharmacokinetics, safety and tolerability, and efficacy of the
vanza triple for 24 weeks, after a baseline established on
TRIKAFTA. Children received TRIKAFTA for at least 4 weeks prior to
receiving vanza triple, and the baseline values for all endpoints
were measured at the end of the TRIKAFTA run-in. The primary
endpoint was safety and tolerability. The secondary endpoints were
efficacy endpoints and included absolute change in SwCl from
baseline through week 24, absolute change in ppFEV1 from baseline
through week 24, the proportion of children with SwCl <60 mmol/L
through week 24, and the proportion of children with SwCl <30
mmol/L through week 24, among other endpoints.
About vanzacaftor/tezacaftor/deutivacaftor (the “vanza
triple”) In people with CF, mutations in the CFTR gene lead to
decreased quantity and/or function of the CFTR protein channel at
the cell surface. Vanzacaftor and tezacaftor are correctors
designed to increase the amount of CFTR protein at the cell surface
by facilitating the processing and trafficking of the CFTR protein.
Deutivacaftor is a potentiator designed to increase the channel
open probability of the CFTR protein delivered to the cell surface
to improve the flow of salt and water across the cell membrane.
Investigational vanzacaftor/tezacaftor/deutivacaftor was granted
Fast Track and Orphan Drug Designations from the U.S. Food and Drug
Administration for the treatment of cystic fibrosis.
The vanza triple will be subject to a meaningfully lower
single-digit royalty obligation, compared to the rate payable on
Vertex’s current CF portfolio.
About Cystic Fibrosis Cystic fibrosis (CF) is a rare,
life-shortening genetic disease affecting more than 92,000 people
globally. CF is a progressive, multi-organ disease that affects the
lungs, liver, pancreas, GI tract, sinuses, sweat glands and
reproductive tract. CF is caused by a defective and/or missing CFTR
protein resulting from certain mutations in the CFTR gene. People
with CF must inherit two defective CFTR genes — one from each
parent — to have CF, and these mutations can be identified by a
genetic test. While there are many different types of CFTR
mutations that can cause the disease, the vast majority of people
with CF have at least one F508del mutation. CFTR mutations lead to
CF by causing CFTR protein to be defective or by leading to a
shortage or absence of CFTR protein at the cell surface. The
defective function and/or absence of CFTR protein results in poor
flow of salt and water into and out of the cells in a number of
organs. In the lungs, this leads to the buildup of abnormally
thick, sticky mucus, chronic lung infections and progressive lung
damage that eventually leads to death for many patients. The median
age of death is in the 30s, but with treatment, projected survival
is improving.
Diagnosis of CF is often made by genetic testing and is
confirmed by testing sweat chloride (SwCl), which measures CFTR
protein dysfunction. The diagnostic threshold for CF is SwCl ≥60
mmol/L, while levels between 30-59 indicate CF is possible and more
testing may be needed to make the diagnosis of CF. A SwCl level of
<30 mmol/L is seen in people who carry one copy of the CF gene
but do not have any manifestation of disease (carriers). Higher
levels of SwCl are associated with more severe disease. Restoring
CFTR function leads to lower levels of SwCl. SwCl levels below 60
mmol/L are associated with improved outcomes such as better and
more stable lung function, fewer pulmonary exacerbations, better
quality of life and improved survival. Restoring SwCl levels below
30 mmol/L has long been the ultimate treatment goal for Vertex, as
levels below 30 mmol/L are considered normal and are typical of CF
carriers who do not have disease.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor) U.S. INDICATIONS AND USAGE
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a
prescription medicine used for the treatment of cystic fibrosis
(CF) in patients aged 2 years and older who have at least one copy
of the F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene or another mutation that is
responsive to treatment with TRIKAFTA. Patients should talk to
their doctor to learn if they have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under
2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking TRIKAFTA, patients should tell their doctor
about all of their medical conditions, including if they: are
allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney
problems, have or have had liver problems, are pregnant or plan to
become pregnant because it is not known if TRIKAFTA will harm an
unborn baby, or are breastfeeding or planning to breastfeed because
it is not known if TRIKAFTA passes into breast milk.
Patients should tell their doctor about all the medicines
they take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements. TRIKAFTA may affect
the way other medicines work, and other medicines may affect how
TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when
taken with certain medicines. Patients should ask their doctor or
pharmacist for a list of these medicines if they are not sure.
Patients should especially tell their doctor if they take:
antibiotics such as rifampin or rifabutin; seizure medicines such
as phenobarbital, carbamazepine, or phenytoin; St. John’s wort;
antifungal medicines including ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; antibiotics including
telithromycin, clarithromycin, or erythromycin.
Patients should avoid food or drink that contains
grapefruit while taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
Liver damage and worsening of liver function in patients
with severe liver disease that can be serious and may require
transplantation. Liver damage has also happened in patients without
liver disease.
High liver enzymes in the blood, which is a common side
effect in patients treated with TRIKAFTA. These can be
serious and may be a sign of liver injury. The patient’s doctor
will do blood tests to check their liver before they start
TRIKAFTA, every 3 months during the first year of taking TRIKAFTA,
and every year while taking TRIKAFTA. 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 the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine.
Serious allergic reactions have happened to patients who
are treated with TRIKAFTA. Call your healthcare provider or go to
the emergency room right away if you have any symptoms of an
allergic reaction. Symptoms of an allergic reaction may include:
rash or hives; tightness of the chest or throat or difficulty
breathing; swelling of the face, lips and/or tongue; difficulty
swallowing; and light-headedness or dizziness.
Abnormality of the eye lens (cataract) has been noted in
some children and adolescents treated with TRIKAFTA. If the patient
is a child or adolescent, their doctor should perform eye
examinations before and during treatment with TRIKAFTA to look for
cataracts.
The most common side effects of TRIKAFTA include
headache, upper respiratory tract infection (common cold) including
stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash,
increase in liver enzymes, increase in a certain blood enzyme
called creatine phosphokinase, flu (influenza), inflamed sinuses,
and increase in blood bilirubin.
Patients should tell their doctor if they have any side effect
that bothers them or that does not go away. These are not all the
possible side effects of TRIKAFTA. For more information, patients
should ask their doctor or pharmacist.
Please click here to see the full Prescribing Information for
TRIKAFTA.
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 as defined in the
Private Securities Litigation Reform Act of 1995, as amended,
including, without limitation, statements by Carmen Bozic, M.D.,
and Bonnie Ramsey, M.D., in this press release, and statements
regarding our beliefs about the potential benefits of
vanzacaftor/tezacaftor/deutivacaftor, our plans to make global
regulatory submissions by mid-2024, including a New Drug
Application to the U.S. Food and Drug Administration and a
Marketing Authorization Application with the European Medicines
Agency, our plans to use a priority review voucher in the U.S. and
our expectations for an expedited review, and our plans to present
the full data set from these studies. 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 regulatory submissions may not be
completed on the anticipated timeline, or at all, that data from
the company's research and development programs may not support
registration or further development of its compounds due to safety,
efficacy, and other risks listed under the heading “Risk Factors”
in Vertex's most recent annual report and subsequent quarterly
reports filed with the Securities and Exchange Commission at
www.sec.gov and available through the company's website at
www.vrtx.com. 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.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or
Heather Nichols: +1 617-839-3607 or International: +44 20 3204
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Grafico Azioni Vertex Pharmaceuticals (NASDAQ:VRTX)
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Da Giu 2023 a Giu 2024