-VX-440 to be evaluated as part of 4-week
triple combination dosing with tezacaftor (VX-661) and ivacaftor;
VX-152 to be evaluated as part of 2-week triple combination
dosing-
-Studies to enroll people with cystic fibrosis
who have one copy of the F508del mutation and a minimal function
mutation and also people with two copies of the F508del
mutation-
-Additional next-generation correctors
advancing into Phase 1 development; VX-659 Phase 1 clinical study
expected to begin in 2016 and will enroll healthy volunteers and CF
patients-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the planned initiation of two Phase 2 studies to evaluate
the next-generation correctors VX-440 and VX-152 in triple
combination regimens with tezacaftor (VX-661) and ivacaftor in
people with cystic fibrosis (CF). The Phase 2 study of VX-440 is
designed to evaluate the safety and efficacy of 4-week dosing of
VX-440 in combination with tezacaftor and ivacaftor in
approximately 40 people with CF who have one F508del mutation and
one minimal function mutation and approximately 25 people with two
copies of the F508del mutation. The first data from this study are
expected in the second half of 2017 and are intended to support the
initiation of Phase 3 development for VX-440. The Phase 2 study of
VX-152 will evaluate 2 weeks of triple combination dosing in
approximately 35 people with CF who have one F508del mutation and
one minimal function mutation and approximately 25 people with two
copies of the F508del mutation. Data from the study of VX-152 are
also expected in the second half of 2017 and are intended to
support the initiation of a longer-duration Phase 2b or
registrational program for VX-152.
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Table 1 (Graphic: Vertex)
Vertex has submitted Investigational New Drug applications to
the U.S. Food and Drug Administration (FDA) for both VX-440 and
VX-152 and expects to start both studies by the end of 2016.
Vertex also announced today that it plans to begin Phase 1
development of an additional next-generation corrector, VX-659, by
the end of 2016. The Phase 1 study is expected to enroll healthy
volunteers and will also include an arm to evaluate triple
combination dosing in CF patients who have one F508del mutation and
one minimal function mutation. Based on data from this study,
Vertex plans to start a Phase 2 study of VX-659 in the second half
of 2017. The company also expects to advance a fourth
next-generation corrector into Phase 1 development in 2017.
“We are committed to advancing multiple next-generation
correctors in parallel to bring the best potential treatments to
all people with CF who have at least one F508del mutation,” said
Jeffrey Chodakewitz, M.D., Executive Vice President and Chief
Medical Officer at Vertex. “We believe that the combination of a
next-generation corrector with tezacaftor and ivacaftor has the
potential to benefit a broad range of people with this disease,
including those with minimal function mutations who do not yet have
a medicine to treat the underlying cause of their CF.”
“KALYDECO and ORKAMBI are significant medical advances for many
people with CF, however a very large number of our patients still
do not have medicine to treat the cause of their disease,” said
Patrick Flume, M.D., Director of the Medical University of South
Carolina Cystic Fibrosis Center and Principal Investigator for
the Phase 2 VX-152 study. “The studies announced today are a
promising step forward for patients and for the treatment of this
devastating disease. I look forward to working with Vertex to move
these potential medicines through development and toward patients
as rapidly as possible.”
The initiation of the Phase 2 studies of VX-440 and VX-152 is
based on data from preclinical studies as well as recently
completed Phase 1 studies for VX-440 and VX-152 that each enrolled
approximately 100 healthy volunteers. Additional details for each
Phase 2 study are provided below.
About the VX-440 Phase 2
Study
The Phase 2 study of VX-440 includes three parts. Part A of the
study is designed to evaluate triple combination dosing for 4 weeks
in approximately 40 patients ages 18 and older with one F508del
mutation and one minimal function mutation. Part B of the study is
designed to evaluate triple combination dosing for 4 weeks in
approximately 25 patients ages 18 and older with two copies of the
F508del mutation. A detailed study design is provided in Table
1.
Part A Cohort 1a will evaluate low-dose VX-440 in combination
with tezacaftor and ivacaftor. Cohort 1a is designed to confirm
that the safety and pharmacokinetic properties observed in healthy
volunteers are similar in people with CF. The doses for Part A
Cohort 1b and Part B will be confirmed based on review of data from
Part A Cohort 1a.
The primary endpoints of the study are safety and tolerability
and the absolute change in percent predicted forced expiratory
volume in one second (ppFEV1). Secondary endpoints will evaluate
relative improvement in ppFEV1, change in sweat chloride and change
in the CF questionnaire-revised (CFQ-R) respiratory domain score,
among others. Women of childbearing potential who enroll in the
Phase 2 study of VX-440 will be required to use pre-specified,
non-hormonal methods of contraception.
Pending completion of enrollment, Vertex expects to report data
from both cohorts of Part A and from Part B in the second half of
2017. The initiation of Phase 3 development is pending data from
Parts A and B and discussions with regulatory authorities.
A third part of the study (Part C) is designed to evaluate
triple combination dosing for 12 weeks in approximately 130
patients ages 12 and older with one F508del mutation and one
minimal function mutation. Part C will be initiated based on data
from Part A and Part B and would run in parallel with potential
Phase 3 development. Part C is intended to generate 12-week safety
and efficacy data and to demonstrate the contribution of the
components to the overall effect of the triple combination
regimen.
About the VX-152 Phase 2
Study
The Phase 2 study of VX-152 includes two parts. Part A of the
study includes up to three dose escalation cohorts and is designed
to evaluate triple combination dosing for 2 weeks in approximately
35 patients ages 18 and older with one F508del mutation and one
minimal function mutation. Part B of the study includes up to two
cohorts and is designed to evaluate triple combination dosing for 2
weeks in approximately 25 patients ages 18 and older with two
copies of the F508del mutation. A detailed study design is provided
in Table 2.
The primary endpoint of the study is safety and tolerability.
Secondary endpoints will evaluate absolute and relative improvement
in ppFEV1, change in sweat chloride, and change in the CFQ-R
respiratory domain score, among others. Part A Cohort 1a, will
evaluate low-dose VX-152 in combination with tezacaftor and
ivacaftor. The doses for Part A Cohort 1b, Cohort 1c and Part B
will be based on review of data from prior cohorts.
Pending completion of enrollment, Vertex expects to report data
from Parts A and B of this study in the second half of 2017. These
data are intended to support the initiation of a longer-duration
Phase 2b or registrational program.
Advancing Additional Next-Generation
Correctors into Phase 1
As part of Vertex’s commitment to find new medicines to treat
the cause of CF for all people with the disease, the company is
evaluating multiple next-generation correctors in parallel, both in
research and development, to maximize the probability of rapidly
advancing the best regimens to help the greatest number of people
with CF. Consistent with that strategy, Vertex today announced that
it plans to begin clinical development of a third next-generation
corrector, VX-659, in 2016.
In human bronchial epithelial (HBE) cells with multiple
different CFTR genotypes, including cells with two copies of the
F508del mutation and cells with one copy of the F508del mutation
and one copy of a mutation known to result in minimal CFTR
function, the triple combination of VX-659, tezacaftor and
ivacaftor exhibited higher maximal efficacy (as measured by percent
of normal chloride transport) and enhanced potency compared to
other next-generation correctors in triple combination.
A Phase 1 study of VX-659 is expected to begin by the end of
2016 and will evaluate single ascending doses, multiple ascending
doses and triple combination dosing with tezacaftor and ivacaftor
in healthy volunteers. The Phase 1 study will also include an arm
to evaluate triple combination, or placebo, dosing in people with
CF who have one copy of the F508del mutation and one copy of a
minimal function mutation. Pending results of this study, Vertex
plans to initiate a Phase 2 study to evaluate VX-659 in people with
CF in the second half of 2017.
In addition to VX-659, Vertex expects to move a fourth
next-generation corrector from research into clinical development
in 2017.
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 2 years and older
who have one of the following mutations in their CF gene: G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or
R117H. KALYDECO is not for use in people with CF due to other
mutations in the CF gene. KALYDECO is not effective in patients
with CF with two copies of the F508del mutation (F508del/F508del)
in the CF gene. 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 (ivacaftor).
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 Vertex
Vertex is a global biotechnology company that aims to discover,
develop and commercialize innovative medicines so people with
serious diseases can lead better lives. In addition to our clinical
development programs focused on cystic fibrosis, Vertex has more
than a dozen ongoing research programs aimed at other serious and
life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research
and development sites and commercial offices in the United States,
Europe, Canada and Australia. For six years in a row, Science
magazine has named Vertex one of its Top Employers in the life
sciences. For additional information and the latest updates from
the company, please visit www.vrtx.com.
Investor Conference Call and
Webcast
Vertex will host a conference call and webcast today at 4:30
p.m. ET to review its third quarter financial results and also
discuss the clinical development plans for VX-440, VX-152 and
VX-659. To access the call, please dial (866) 501-1537 (U.S.) or +1
(720) 545-0001 (International). The conference call will be webcast
live and a link to the webcast can be accessed through Vertex's
website at www.vrtx.com in the "Investors" section under "Events
and Presentations." To ensure a timely connection, it is
recommended that users register at least 15 minutes prior to the
scheduled webcast. An archived webcast will be available on the
company's website.
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. Chodakewitz's statements in the
fourth paragraph of the press release, Dr. Flume’s statements in
the fifth paragraph of the press release and statements regarding
the expected timing and clinical trial designs for planned clinical
studies of VX-440, VX-152, VX-659 and any additional
next-generation corrector. 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 further development or registration of VX-440, VX-152 or
VX-659 (including combination regimens containing such compounds)
or its other 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)
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Vertex Pharmaceuticals IncorporatedInvestors:Michael
Partridge, 617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:US: 617-341-6992Europe & Australia:
+44 20 3204 5275mediainfo@vrtx.com
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