-Eleven abstracts from Vertex’s CF program
accepted for presentation-
-Late-breaking abstract submitted with data
from three different triple combination regimens in CF
patients-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that 11 abstracts from its cystic fibrosis (CF) research
and development program will be presented at the annual North
American Cystic Fibrosis Conference (NACFC) in Indianapolis,
November 2 to 4, 2017. Previously announced data from the Phase 3
EVOLVE and EXPAND studies of the investigational
tezacaftor/ivacaftor combination in people with CF ages 12 and
older who have certain mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene will be presented
for the first time. Additionally, data from the Phase 3 extension
study of ORKAMBI® (lumacaftor/ivacaftor) in children with CF ages 6
to 11 who have two copies of the F508del mutation and real-world
KALYDECO® (ivacaftor) data will be presented. The company also
submitted an abstract for the late-breaking poster session with
previously announced Phase 1 and Phase 2 data for three different
next-generation correctors (VX-440, VX-152 and VX-659) in triple
combination regimens with tezacaftor and ivacaftor in people with
CF who have one F508del mutation and one minimal function mutation
and in people with two copies of the F508del mutation.
Collectively, the data at the Conference demonstrate continued
progress across the company’s CF program goals of providing
enhanced treatment options for more people, demonstrating the
disease-modifying effects of CFTR modulators, and expanding CFTR
treatment options to all people with CF through the development of
new medicines.
The accepted abstracts are listed below and are now available in
the online edition of Pediatric Pulmonology.
Vertex Abstracts (Oral presentations will also be
presented as posters)
Tezacaftor/Ivacaftor
Combination
1. “Efficacy and Safety of Tezacaftor/Ivacaftor in Patients aged
≥12 with CF Homozygous for F508del-CFTR: A Randomized Placebo (PBO)
– Controlled Phase 3 Trial.” Poster #247. An oral symposium
presentation is scheduled for November 3, 2017, 10:35 a.m. EDT.
2. “Efficacy and Safety of Tezacaftor/Ivacaftor in Patients aged
≥12 with CF Heterozygous for F508DEL and a Residual Function
Mutation: A randomized, double-blind, Placebo-Controlled, Crossover
Phase 3 Study.” Poster #273. An oral symposium presentation is
scheduled for November 3, 2017, 10:55 a.m. EDT.
3. “Sustained CFTR Correction and Potentiation Predicted during
Transitions between Lumacaftor/Ivacaftor and Tezacaftor/Ivacaftor-
based Regimens.” Poster #253.
4. “Drug-Drug Interaction Profile of Tezacaftor/Ivacaftor in
Healthy Adult Subjects.” Poster #254.
ORKAMBI
5. “Effect of Lumacaftor/Ivacaftor on Total, Bronchiectasis, and
Air Trapping Computed Tomography (CT) Scores in Children Homozygous
for F508del-CFTR: Exploratory Imaging Substudy.” Poster #197. An
oral workshop presentation is scheduled for November 3, 2017, 2:50
p.m. EDT.
6. “Safety and Efficacy of Lumacaftor/Ivacaftor (LUM/IVA) in
Patients aged ≥6 years with CF Homozygous for F508del-CFTR (Phase 3
Extension Study).” Poster #278.
7. “Feasibility of Ultrashort Echo Time (UTE) MRI to Evaluate
the Effect of Lumacaftor/Ivacaftor Therapy in Children with Cystic
Fibrosis (CF) Homozygous for F508del.” Poster #266.
8. “Modeling the Long-Term Health Outcomes of Patients with CF
who are Homozygous for the F508del Mutation treated with
Lumacaftor/Ivacaftor.” Poster #30.
KALYDECO
9. “Real-World Outcomes in Patients with CF Treated with
Ivacaftor: 2015 US and UK CF Registry Analyses.” Poster #496.
10. “Disease Progression in Patients with CF Treated with
Ivacaftor: Analyses of Real-World Data from the US and UK CF
Registries.” Poster #497.
Additional NACFC Presentations
11. “Caregiver Burden due to Pulmonary Exacerbations in CF: A
Survey of Caregivers of Children with CF in the US, UK, Ireland,
and Germany.” Poster #252.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening genetic disease
affecting approximately 75,000 people in North America, Europe and
Australia.
CF is caused by a defective or missing CFTR protein resulting
from mutations in the CFTR gene. Children must inherit two
defective CFTR genes — one from each parent — to have CF. There are
approximately 2,000 known mutations in the CFTR gene. Some of these
mutations, which can be determined by a genetic test, or genotyping
test, lead to CF by creating non-working or too few CFTR protein at
the cell surface. The defective function or absence of CFTR protein
results in poor flow of salt and water into and out of the cell in
a number of organs. In the lungs, this leads to the buildup of
abnormally thick, sticky mucus that can cause chronic lung
infections and progressive lung damage in many patients that
eventually leads to death. The median age of death is in the
mid-to-late 20s.
About ORKAMBI® (lumacaftor/ivacaftor)
In people with two copies of the F508del mutation, the CFTR
protein is not processed and trafficked normally within the cell,
resulting in little-to-no CFTR protein at the cell surface.
Patients with two copies of the F508del mutation are easily
identified by a simple genetic test.
ORKAMBI is a combination of lumacaftor, which is designed to
increase the amount of mature protein at the cell surface by
targeting the processing and trafficking defect of the F508del-CFTR
protein, and ivacaftor, which is designed to enhance the function
of the CFTR protein once it reaches the cell surface. It is an oral
pill taken every 12 hours - once in the morning and once in the
evening.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR
ORKAMBI® (lumacaftor/ivacaftor)
TABLETSORKAMBI is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 6 years and older
who have two copies of the F508del mutation
(F508del/F508del) in their CFTR gene. ORKAMBI should only be used
in these patients. It is not known if ORKAMBI is safe and effective
in children under 6 years of age.
Patients should not take ORKAMBI if they are taking certain
medicines or herbal supplements, such as: the antibiotics
rifampin or rifabutin; the seizure medicines phenobarbital,
carbamazepine, or phenytoin; the sedatives/anti-anxiety medicines
triazolam or midazolam; the immunosuppressant medicines everolimus,
sirolimus, or tacrolimus; or St. John's wort.
Before taking ORKAMBI, patients should tell their doctor if
they: have or have had liver problems; have kidney
problems; have had an organ transplant; are using birth control
(hormonal contraceptives, including oral, injectable, transdermal
or implantable forms). Hormonal contraceptives should not be used
as a method of birth control when taking ORKAMBI. Patients should
tell their doctor if they are pregnant or plan to become pregnant
(it is unknown if ORKAMBI will harm the unborn baby) or if they are
breastfeeding or planning to breastfeed (it is unknown if ORKAMBI
passes into breast milk).
ORKAMBI may affect the way other medicines work and other
medicines may affect how ORKAMBI works. Therefore, the dose of
ORKAMBI or other medicines may need to be adjusted when taken
together. Patients should especially tell their doctor if they
take: antifungal medicines such as ketoconazole, itraconazole,
posaconazole, or voriconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
When taking ORKAMBI, patients should tell their
doctor if they stop ORKAMBI for more than 1 week as the doctor may
need to change the dose of ORKAMBI or other medicines the patient
is taking. It is unknown if ORKAMBI causes dizziness. Patients
should not drive a car, use machinery, or do anything requiring
alertness until the patient knows how ORKAMBI affects them.
ORKAMBI can cause serious side effects including:
High liver enzymes in the blood, which can be a sign of liver
injury, have been reported in patients receiving
ORKAMBI. The patient's doctor will do blood tests to check
their liver before they start ORKAMBI, every three months during
the first year of taking ORKAMBI, and annually thereafter. The
patient should call the doctor right away if they have any of the
following symptoms of liver problems: pain or discomfort in the
upper right stomach (abdominal) area; yellowing of the skin or the
white part of the eyes; loss of appetite; nausea or vomiting; dark,
amber-colored urine; or confusion.
Respiratory events such as shortness of breath or chest
tightness were observed in patients when starting
ORKAMBI. If a patient has poor lung function, their doctor
may monitor them more closely when starting ORKAMBI.
An increase in blood pressure has been seen in some patients
treated with ORKAMBI. The patient's doctor should monitor
their blood pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving ORKAMBI and ivacaftor, a
component of ORKAMBI. For children and adolescents, the
patient's doctor should perform eye examinations prior to and
during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include: shortness of
breath and/or chest tightness; upper respiratory tract infection
(common cold), including sore throat, stuffy or runny nose;
gastrointestinal symptoms including nausea, diarrhea, or gas; rash;
fatigue; flu or flu-like symptoms; increase in muscle enzyme
levels; and irregular, missed, or abnormal menstrual periods and
heavier bleeding.
Please click here to see the full
Prescribing Information for ORKAMBI.
About KALYDECO® (ivacaftor)
KALYDECO (ivacaftor) is the first medicine to treat the
underlying cause of CF in people with specific mutations in the
CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral
medicine designed to keep CFTR proteins at the cell surface open
longer to improve the transport of salt and water across the cell
membrane, which helps hydrate and clear mucus from the airways.
KALYDECO is available as 150 mg tablets for adults and pediatric
patients age 6 years and older, and is taken with fat-containing
food. It is also available as 50 mg and 75 mg granules in pediatric
patients ages 2 to less than 6 years and is administered with
soft-food or liquid with fat-containing food.
People with CF who have specific mutations in the CFTR gene are
currently benefiting from KALYDECO in 27 different countries across
North America, Europe and Australia.
KALYDECO® (ivacaftor) INDICATION AND IMPORTANT
SAFETY INFORMATION
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 2 years and older
who have at least one mutation in their CF gene that is responsive
to KALYDECO. Patients should talk to their doctor to learn if they
have an indicated CF gene mutation. It is not known if KALYDECO is
safe and effective in children under 2 years of age.
Patients should not take KALYDECO if they are taking certain
medicines or herbal supplements such as: the antibiotics
rifampin or rifabutin; seizure medications such as phenobarbital,
carbamazepine, or phenytoin; or St. John's wort.
Before taking KALYDECO, patients should tell their doctor if
they: have liver or kidney problems; drink grapefruit juice, or
eat grapefruit or Seville oranges; are pregnant or plan to become
pregnant because it is not known if KALYDECO will harm an unborn
baby; and are breastfeeding or planning to breastfeed because is
not known if KALYDECO passes into breast milk.
KALYDECO may affect the way other medicines work, and other
medicines may affect how KALYDECO works. Therefore the dose of
KALYDECO may need to be adjusted when taken with certain
medications. Patients should especially tell their doctor if they
take antifungal medications such as ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it.
Patients should not drive a car, use machinery, or do anything that
needs them to be alert until they know how KALYDECO affects them.
Patients should avoid food containing grapefruit or Seville oranges
while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood have been reported in
patients receiving KALYDECO. The patient's doctor will do blood
tests to check their liver before starting KALYDECO, every 3 months
during the first year of taking KALYDECO, and every year while
taking KALYDECO. For patients who have had high liver enzymes in
the past, the doctor may do blood tests to check the liver more
often. Patients should call their doctor right away if they have
any of the following symptoms of liver problems: pain or discomfort
in the upper right stomach (abdominal) area; yellowing of their
skin or the white part of their eyes; loss of appetite; nausea or
vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving KALYDECO. The patient's doctor
should perform eye examinations prior to and during treatment with
KALYDECO to look for cataracts. The most common side effects
include headache; upper respiratory tract infection (common cold),
which includes sore throat, nasal or sinus congestion, and runny
nose; stomach (abdominal) pain; diarrhea; rash; nausea; and
dizziness.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing
Information for KALYDECO.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is
now located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada and Australia. Vertex is consistently
recognized as one of the industry's top places to work, including
being named to Science magazine's Top Employers in the life
sciences ranking for seven years in a row. For additional
information and the latest updates from the company, please visit
www.vrtx.com.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation. KALYDECO®
(ivacaftor), ORKAMBI® (lumacaftor/ivacaftor), tezacaftor, VX-440,
VX-152 and VX-659 were discovered by Vertex as part of this
collaboration.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995,
including, without limitation, statements regarding the
tezacaftor/ivacaftor combination and the next-generation triple
combination regimens. While Vertex believes the forward-looking
statements contained in this press release are accurate, these
forward-looking statements represent the company's beliefs only as
of the date of this press release, and there are a number of
factors that could cause actual events or results to differ
materially from those indicated by such forward-looking statements.
Those risks and uncertainties include, among other things, (i) that
Vertex could experience unforeseen delays in conducting its
development programs relating to triple combination treatments and
in submitting related regulatory filings, (ii) that regulatory
authorities may not approve, or approve on a timely basis, one or
more of these regimens due to safety, efficacy or other reasons,
and (iii) and other risks listed under Risk Factors in Vertex's
annual report and quarterly reports filed with the Securities and
Exchange Commission and available through the company's website at
www.vrtx.com. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals IncorporatedInvestors:Michael
Partridge, 617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.comorNorth America:Megan
Goulart, + 1-617-341-6992orEurope & Australia:Rebecca Hunt, +44
7718 962 690
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