- New clinical data show TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor) continues to
deliver significant benefit across a variety of outcomes and over
the long term in people with CF -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that multiple abstracts on the company’s portfolio of
cystic fibrosis (CF) medicines will be presented in posters and
oral presentations at this year’s North American Cystic Fibrosis
Conference (NACFC), including studies demonstrating the clinical
benefits and long-term safety of TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor).
Vertex will present new long-term safety and efficacy data on
TRIKAFTA from an open-label 192-week extension study in people 12
years and older with CF and at least one F508del allele. Based on
an interim analysis through Week 144 of the 192-week study, people
receiving TRIKAFTA showed maintained improvements in lung function,
respiratory symptoms and cystic fibrosis transmembrane conductance
regulator (CFTR) function. TRIKAFTA also continues to be generally
well tolerated (Poster #170 and Oral Workshop: W21.2). These data
will also be discussed during the second plenary on November 4,
2022, at NACFC.
“These long-term data at 144 weeks demonstrate remarkable health
effects in the longest study of TRIKAFTA. The data show sustainment
of the historic improvements in lung function, respiratory symptoms
and sweat chloride, a marker of CFTR function,” said Deepika
Polineni, M.D., MPH, Associate Professor of Pediatrics and Cystic
Fibrosis Center Director at Washington University School of
Medicine in St. Louis, and a co-investigator of the 445-105 study
(Poster #170). “CFTR modulators like TRIKAFTA have pivotally
changed standard of care therapy in CF, and the CF community
continues to benefit from ongoing collection and evaluation of
long-term data.”
Vertex will also present data for the first time from its
investigational Phase 3 open-label study designed to evaluate the
safety, pharmacokinetics and efficacy of TRIKAFTA in children 2
through 5 years of age with CF and at least one F508del allele
(Poster #693). The study shows that treatment with TRIKAFTA led to
improvements in sweat chloride concentration and lung function, as
measured by the lung clearance index, and stable nutritional status
in children 2 through 5 years of age. TRIKAFTA was generally well
tolerated, with a safety profile generally consistent with older
age groups. Based on these results, Vertex recently submitted a New
Drug Application (NDA) with the U.S. Food and Drug Administration
for this age range and will be filing for approvals with the
European Medicines Agency (EMA) and the Medicines and Healthcare
products Regulatory Agency (MHRA) by the end of this year.
Additionally, Vertex will present data from a pooled analysis
from multiple Phase 3 studies with CFTR modulators evaluating how
the restoration of CFTR-mediated chloride transport, as reflected
by changes in sweat chloride concentration, impacts clinical
outcomes in people with CF treated with CFTR modulators (Poster
#694). The study showed that people with CF ages 12 years and older
treated with CFTR modulators achieved higher levels of CFTR
activity, as reflected in lower levels of sweat chloride. Those
with greater improvements in sweat chloride demonstrated greater
improvements in lung function, respiratory symptoms, body mass
index, pulmonary exacerbations, and had better lung function
trajectory over time. These new data demonstrate that higher levels
of CFTR function as measured by a reduction of sweat chloride are
associated with improved clinical outcomes. The best outcomes were
seen in those achieving a sweat chloride concentration <60
mmol/L.
In addition, Vertex will present findings from the first fully
decentralized study in people with CF designed to explore the
feasibility of wearable technology in evaluating clinical outcomes
such as cough count and physical activity (Poster #169 and Oral
Workshop: W30.1).
Vertex will also present interim results from Phase 3 open-label
extension studies evaluating the long-term safety and efficacy of
TRIKAFTA in children with CF ages 6 years and older (Poster #163
and Thematic Poster: TPS01.2) and people with CF 12 years and older
with F508del-gating or F508del-residual function genotypes (Poster
#185 and Thematic Poster: TPS01.5).
“These new data add to the growing body of evidence
demonstrating the benefits of our CFTR modulators across multiple
clinical measures over the long-term, and the significant impact
these medicines are having on patients,” said Carmen Bozic, M.D.,
Executive Vice President, Global Medicines Development and Medical
Affairs, and Chief Medical Officer, Vertex.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 83,000 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. Children 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 early 30s.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor)
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
TABLETS
TRIKAFTA is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 6 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 6 years of age.
Patients should not take TRIKAFTA if they take certain
medicines or herbal supplements, such as: antibiotics such as
rifampin or rifabutin; seizure medicines such as phenobarbital,
carbamazepine, or phenytoin; St. John’s wort.
Before taking TRIKAFTA, patients should tell their doctor
about all of their medical conditions, including if they: 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.
TRIKAFTA may affect the way other medicines work, and other
medicines may affect how TRIKAFTA works. Therefore, the dose of
TRIKAFTA may need to be adjusted when taken with certain medicines.
Patients should especially tell their doctor if they take:
antifungal medicines including ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; antibiotics including
telithromycin, clarithromycin, or erythromycin.
TRIKAFTA may cause dizziness in some people who take it.
Patients should not drive a car, operate machinery, or do anything
that requires alertness until they know how TRIKAFTA affects
them.
Patients should avoid food or drink that contains
grapefruit while they are taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
Liver damage and worsening of liver function in people
with severe liver disease that can be serious and may require
transplantation. Liver damage has also happened in people without
liver disease.
High liver enzymes in the blood, which is a common side
effect in people 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.
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, diarrhea, upper respiratory tract infection (common cold)
including stuffy and runny nose, stomach (abdominal) pain, inflamed
sinuses, increase in liver enzymes, increase in a certain blood
enzyme called creatine phosphokinase, rash, flu (influenza), and
increase in blood bilirubin.
These are not all the possible side effects of TRIKAFTA.
Please click here to see the full U.S. Prescribing
Information for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and
ivacaftor).
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
pipeline of investigational small molecule, cell and genetic
therapies in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes,
alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is 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 13 consecutive years on Science magazine's Top
Employers list and one of Fortune’s Best Workplaces in
Biotechnology and Pharmaceuticals and Best Workplaces for Women.
For company updates and to learn more about Vertex's history of
innovation, visit www.vrtx.com or follow us on Facebook, Twitter,
LinkedIn, YouTube and Instagram.
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 Dr. Carmen
Bozic and Dr. Deepika Polineni in this press release, statements
regarding the potential benefits, safety and efficacy of TRIKAFTA,
our plans to present data about our portfolio of CF medicines at
the NACFC, including data from our TRIKAFTA Phase 3 open-label
studies and data from a pooled analysis from multiple Phase 3
studies with CFTR modulators, and additional scientific
presentations regarding TRIKAFTA, including long-term safety and
efficacy data, expectations to present findings from a study
designed to explore wearable technology in evaluating clinical
outcomes, and expectations to file regulatory approvals with the
EMA and MHRA by the end of this year. 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 data from a limited number of
patients may not be indicative of final clinical trial results,
that the company may not be able to submit the anticipated
regulatory filings on the expected 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 or Manisha Pai: +1 617-961-1899
Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992 or
Heather Nichols: +1 617-839-3607 or International: +44 20 3204
5275
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