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Vertex to Present Data at ECFS Conference on Potential Impact of Early Treatment and Long-Term Treatment with CFTR Modulators on the Underlying Cause of CF

BOSTON–(BUSINESS WIRE)–#cfVertex
Pharmaceuticals Incorporated
(Nasdaq: VRTX) today announced that
data from six scientific abstracts from the company’s portfolio of
cystic fibrosis (CF) medicines will be presented at the 42nd
European Cystic Fibrosis Conference, taking place June 5-8, 2019 in
Liverpool, UK.

These abstracts include data being presented from the ongoing Phase 3
ARRIVAL study of the pharmacokinetics (PK) and safety of KALYDECO®
(ivacaftor) in a cohort of patients 6 to <12 months old with at least
one CFTR gating mutation. The study showed a safety profile
consistent with that observed in children aged 12 to <24 months and
demonstrated substantial improvements in sweat chloride. Improvements in
exploratory biomarkers of pancreatic disease, including increases in
fecal elastase-1 (FE-1) and reductions in serum immunoreactive
trypsinogen (IRT), suggest improvements in exocrine pancreatic function
and inflammation with ivacaftor.

Additionally, a 96-week open-label extension study of ORKAMBI®
(lumacaftor/ivacaftor) in patients aged 6 to 11 years with two F508del
mutations (F/F) confirmed that lumacaftor/ivacaftor was generally
well tolerated for up to 120 weeks, showing a safety profile consistent
with previous studies.

CF science continues to evolve rapidly, and it is vital that we help
deepen our understanding of the potential long-term impact of our
medicines, particularly in younger patient populations,” said Reshma
Kewalramani, M.D., Executive Vice President and Chief Medical Officer at
Vertex. “By presenting these data at ECFS, we are excited to build on
the available data on CFTR modulators and identify new areas of
scientific exploration.”

                 
    Abstract Title   Presentation Type   Presenting Author   Date/ Time
                 
IVA   Ivacaftor (IVA) treatment in patients 6 to < 12 months old with
cystic fibrosis with a CFTR gating mutation: results of a
2-part, single-arm, phase 3 study
  Oral Presentation / Hot off the press: new data from drug trials   Jane Davies  

June 6, 2019
17:00-18:30

TEZ / IVA   Change in low-dose chest Computed Tomography (CT) scores after 72
weeks of tezacaftor/ivacaftor (TEZ/IVA) in patients (pts) with
cystic fibrosis and ppFEV1 ≥70%: an exploratory phase 2
study
  Oral Presentation / Hot off the press: new data from drug trials   Claire Wainwright  

June 6, 2019
17:00-18:30

CF Disease Burden   Disease progression and burden in patients with cystic fibrosis
homozygous for F508del across Europe in an observational registry
(VOICE Study)
  Poster   Edward McKone  

June 7, 2019
14:00-15:00

  Hospitalisations among children with cystic fibrosis aged < 6 years   Poster   Teja Thorat  

June 7, 2019
14:00-15:00

LUM / IVA   Real-world outcomes among patients with Cystic Fibrosis treated with
lumacaftor/ivacaftor (LUM/IVA) in 2017: an interim analysis of data
from the US CF Foundation Patient Registry (CFFPR)
  Oral Presentation / New Therapies and real life experience   Nataliya Volkova  

June 7, 2019
15:00-16:30

  Long-term safety and efficacy of lumacaftor/ivacaftor therapy in
patients aged 6-11 years with cystic fibrosis homozygous for the
F508del-CFTR
mutation (F/F)
  Oral Presentation / New therapies and real life experience   Mark Chilvers  

June 7, 2019
15:00– 16:30

       

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 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. It is also available as 25 mg,
50 mg and 75 mg granules in pediatric patients ages 6 months to less
than 6 years.

People with CF who have specific mutations in the CFTR gene are
currently indicated for KALYDECO in different countries across North
America, Europe and other International markets.

U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO®
(ivacaftor):

KALYDECO (ivacaftor) is a prescription medicine
used for the treatment of cystic fibrosis (CF) in patients age 6 months
and older who have at least one mutation in their CFTR gene that
is responsive to KALYDECO. Patients should talk to their doctor to learn
if they have an indicated CFTR gene mutation. It is not known if
KALYDECO is safe and effective in children under 6 months of age.

Patients should not take KALYDECO if they take 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.

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 U.S. full Prescribing Information for KALYDECO (ivacaftor).

About ORKAMBI® (lumacaftor/ivacaftor)
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.

U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR ORKAMBI®
(lumacaftor/ivacaftor):

ORKAMBI is a prescription medicine used
for the treatment of cystic fibrosis (CF) in patients age 2 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 2 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 and anti-anxiety medicines triazolam or
midazolam; the immunosuppressant medicines cyclosporine, everolimus,
sirolimus, or tacrolimus; or St. John’s wort.

Before taking ORKAMBI, patients should tell their doctor about all
their medical conditions, including if they:
have or have had liver
problems; have kidney problems; have had an organ transplant; or are
using birth control. Hormonal contraceptives, including oral,
injectable, transdermal, or implantable forms 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.

ORKAMBI can cause serious side effects, including:

Worsening of liver function in people with severe liver disease.
The worsening of liver function can be serious or cause death. Patients
should talk to their doctor if they have been told they have liver
disease as their doctor may need to adjust the dose of ORKAMBI.

High liver enzymes in the blood, which can be a sign of liver
injury. 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.

Breathing problems such as shortness of breath or chest tightness
in patients when starting ORKAMBI, especially in patients who have poor
lung function. If a patient has poor lung function, their doctor may
monitor them more closely when starting ORKAMBI.

An increase in blood pressure in some people receiving ORKAMBI.
The patient’s doctor should monitor their blood pressure during
treatment with ORKAMBI.

Abnormality of the eye lens (cataract) in some children and
adolescents receiving ORKAMBI. For children and adolescents, the
patient’s doctor should perform eye examinations before and during
treatment with ORKAMBI to look for cataracts.

The most common side effects of ORKAMBI include: breathing
problems, such as shortness of breath and chest tightness; nausea;
diarrhea; fatigue; increase in a certain blood enzyme called creatinine
phosphokinase; rash; gas; common cold, including sore throat, stuffy or
runny nose; flu or flu-like symptoms; and irregular, missed, or abnormal
periods (menses) and increase in the amount of menstrual bleeding.

Side effects seen in children are similar to those seen in adults
and adolescents. Additional common side effects seen in children
include: cough with sputum, stuffy nose, headache, stomach pain, and
increase in sputum.

Please click here
to see the full U.S. Prescribing Information for ORKAMBI
(lumacaftor/ivacaftor).

About SYMDEKO® (tezacaftor/ivacaftor and ivacaftor)
Some
mutations result in CFTR protein that is not processed or folded
normally within the cell, and that generally does not reach the cell
surface. SYMDEKO is a combination of tezacaftor and ivacaftor.
Tezacaftor is designed to address the trafficking and processing defect
of the CFTR protein to enable it to reach the cell surface where
ivacaftor can increase the amount of time the protein stays open.

U.S INDICATION AND IMPORTANT SAFETY INFORMATION FOR SYMDEKO®
(tezacaftor/ivacaftor and ivacaftor) tablets

SYMDEKO is a prescription medicine used for the treatment of cystic
fibrosis (CF) in patients aged 12 years and older who have two copies of
the F508del mutation, or who have at least one mutation in the CF gene
that is responsive to treatment with SYMDEKO. Patients should talk to
their doctor to learn if they have an indicated CF gene mutation. It is
not known if SYMDEKO is safe and effective in children under 12 years of
age.

Patients should not take SYMDEKO if they take certain medicines or
herbal supplements such as: 
the antibiotics rifampin or
rifabutin; seizure medicines such as phenobarbital, carbamazepine, or
phenytoin; St. John’s wort.

Before taking SYMDEKO, patients should tell their doctor if they: have
or have had liver problems; have kidney problems; are pregnant or plan
to become pregnant because it is not known if SYMDEKO will harm an
unborn baby; are breastfeeding or planning to breastfeed because it is
not known if SYMDEKO passes into breast milk.

SYMDEKO may affect the way other medicines work, and other medicines
may affect how SYMDEKO works.
 Therefore, the dose of SYMDEKO may
need to be adjusted when taken with certain medicines. Patients should
especially tell their doctor if they take antifungal medicines such as
ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole;
or antibiotics such as telithromycin, clarithromycin, or erythromycin.

SYMDEKO may cause dizziness in some people who take it. Patients
should not drive a car, use machinery, or do anything that requires
alertness until they know how SYMDEKO affects them.

Patients should avoid food or drink that contains grapefruit
or Seville oranges while they are taking SYMDEKO.

SYMDEKO can cause serious side effects, including:

High liver enzymes in the blood, which have been reported in
people treated with SYMDEKO or treated with ivacaftor alone. The
patient’s doctor will do blood tests to check their liver before they
start SYMDEKO, every 3 months during the first year of taking SYMDEKO,
and every year while taking SYMDEKO. 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) in some children and
adolescents treated with SYMDEKO or with ivacaftor alone. If the patient
is a child or adolescent, their doctor should perform eye examinations
before and during treatment with SYMDEKO to look for cataracts.

The most common side effects of SYMDEKO include headache,
nausea, sinus congestion, and dizziness.

These are not all the possible side effects of SYMDEKO. Please
click 
here to
see the full U.S. Prescribing Information for SYMDEKO
(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 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, Australia and Latin America. 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 nine 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), SYMDEKO®/SYMKEVI®
(tezacaftor/ivacaftor and ivacaftor), and VX-445 (elexacaftor)
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,
Dr. Kewalramani’s statements in the fourth paragraph and the information
provided regarding ongoing clinical trials, including the Phase 3
ARRIVAL trial. 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, that data from the company’s development programs may not
support registration or further development of its compounds due to
safety, efficacy or other reasons, 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)

(VRTX-GEN)

Contacts

Vertex Pharmaceuticals Incorporated
Investors:
Michael
Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Zach
Barber, 617-341-6470
or
Media:
[email protected]
or
North
America:
+ 1-617-341-6992
or
Europe & Australia:
Rebecca
Hunt, +44 7718 962 690

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