-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-
Company Website:
http://www.vrtx.com
BOSTON -- (Business Wire)
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)
TABLETS
ORKAMBI 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)
View source version on businesswire.com: http://www.businesswire.com/news/home/20170919006215/en/
Contacts:
Vertex Pharmaceuticals Incorporated
Investors:
Michael
Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Zach
Barber, 617-341-6470
or
Media:
mediainfo@vrtx.com
or
North
America:
Megan Goulart, + 1-617-341-6992
or
Europe &
Australia:
Rebecca Hunt, +44 7718 962 690
Source: Vertex Pharmaceuticals Incorporated
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