-Data expected in late 2018 from Phase 3
studies of VX-659, tezacaftor and ivacaftor in people with one
F508del mutation and one minimal function mutation and in people
with two F508del mutations-
-Enrollment of two Phase 3 studies of VX-445 in
triple combination with tezacaftor and ivacaftor expected to be
complete in the fourth quarter of 2018; Data for both studies of
VX-445, tezacaftor and ivacaftor expected in the first quarter of
2019-
-Vertex plans to evaluate VX-659 and VX-445
triple combination data to choose the best regimen to submit for
potential regulatory approval; Submission of New Drug Application
planned for no later than mid-2019-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that enrollment is complete for the two Phase 3 studies
of the next-generation corrector VX-659 in triple combination with
tezacaftor and ivacaftor in people with cystic fibrosis (CF) with
one F508del mutation and one minimal function mutation and in
people with two F508del mutations. Based on the completion of
enrollment, Vertex expects to report data from both Phase 3 studies
of the VX-659 triple combination regimen in late 2018. Vertex
expects to complete enrollment of the two Phase 3 studies of the
next-generation corrector VX-445 in triple combination with
tezacaftor and ivacaftor in the fourth quarter of 2018 and to
report data from these studies in the first quarter of 2019. Vertex
plans to evaluate data from both the VX-659 and VX-445 Phase 3
triple combination programs to choose the best regimen to submit
for potential regulatory approval. The data expected in late 2018
for VX-659 and in the first quarter of 2019 for VX-445 are expected
to provide the basis for submission of a New Drug Application (NDA)
to the U.S. FDA for people with one F508del mutation and one
minimal function mutation no later than mid-2019.
“The two Phase 3 studies of VX-659, tezacaftor and ivacaftor
enrolled nearly 500 people with CF in less than five months,
underscoring the significant interest within the CF community to
advance triple combination regimens that have the potential to
treat up to 90 percent of all people with the disease,” Reshma
Kewalramani, M.D., Executive Vice President, Global Medicines
Development and Medical Affairs and Chief Medical Officer at
Vertex. “Our goal is to bring the best triple combination regimen
to people with CF as quickly as possible, and we look forward to
obtaining data later this year and in the first quarter of 2019 to
support a submission for potential approval of a triple combination
regimen.”
The Phase 3 study of the VX-659 triple combination regimen in
patients with one F508del mutation and one minimal function
mutation was designed with a pre-specified interim analysis to
evaluate the primary endpoint of ppFEV1 at 4 weeks and safety
through 12 weeks. This interim analysis will be conducted once all
patients are through the primary efficacy endpoint at week 4. These
efficacy and safety data from the interim analysis are expected in
late 2018 and are intended to support a potential NDA submission
for the VX-659, tezacaftor and ivacaftor triple combination regimen
in people with one F508del mutation and one minimal function
mutation.
Similar to the interim analysis for the VX-659 triple
combination regimen, the Phase 3 study of the VX-445 triple
combination regimen in patients with one F508del mutation and one
minimal function mutation was designed with a pre-specified interim
analysis to evaluate the primary endpoint of ppFEV1 at 4 weeks and
safety through 12 weeks. This interim analysis will also be
conducted once all patients are through the primary efficacy
endpoint at week 4. Data from the VX-445 interim analysis are
expected in the first quarter of 2019. The interim analyses for the
VX-659 and VX-445 triple combination regimens will enable Vertex to
evaluate comparable data from both Phase 3 triple combination
programs and determine the best regimen to submit for potential
regulatory approval.
To preserve the integrity of the Phase 3 studies of VX-659,
tezacaftor and ivacaftor and the Phase 3 studies of VX-445,
tezacaftor and ivacaftor that will be ongoing into mid-2019, Vertex
expects to disclose in late 2018 and the first quarter of 2019 only
the topline results for the primary 4-week efficacy endpoints of
the VX-659 and VX-445 Phase 3 studies, respectively, and whether
the safety and efficacy profiles observed support a potential NDA
submission. In the second half of 2019, Vertex intends to disclose
additional safety and efficacy data, including secondary endpoints,
for each study following the completion of both the VX-659 and
VX-445 Phase 3 triple combination programs.
About the VX-659 Phase 3 Study in People with One F508del
Mutation and One Minimal Function MutationThe ongoing
randomized, double-blind, placebo-controlled Phase 3 study is
evaluating VX-659 in triple combination with tezacaftor and
ivacaftor, or triple placebo, in 385 patients ages 12 and older who
have one F508del mutation and one minimal function mutation. The
primary endpoint of the study is the mean absolute change in
percent predicted forced expiratory volume in one second (ppFEV1)
from baseline at week 4 of triple combination treatment compared to
triple placebo. The study was designed to include a pre-specified
interim analysis to evaluate the primary endpoint at 4 weeks and
safety through 12 weeks. This interim analysis will be conducted
once all patients are through the primary efficacy endpoint at week
4. These data are expected to form the basis of a potential NDA
submission to the U.S. FDA for people with one F508del mutation and
one minimal function mutation. The study is evaluating VX-659 in
combination with tezacaftor and ivacaftor for a total of 24 weeks
of treatment and will generate additional safety data and data for
key secondary endpoints, including the number of pulmonary
exacerbations, change in body mass index, change in sweat chloride,
and change in patient-reported outcomes as measured by the
respiratory domain score of the Cystic Fibrosis
Questionnaire-Revised (CFQ-R), among others.
About the VX-659 Phase 3 Study in People with Two F508del
MutationsThe randomized, double-blind, controlled Phase 3 study
evaluated four weeks of treatment with VX-659 or placebo in
combination with tezacaftor and ivacaftor in 111 patients ages 12
years or older who have two F508del mutations. All patients
received tezacaftor in combination with ivacaftor during a 4-week
run-in prior to the start of the triple combination treatment
period. The primary endpoint of the study is the mean absolute
change in ppFEV1 from baseline (end of the 4-week
tezacaftor/ivacaftor run-in) at week four of treatment with VX-659
in triple combination with tezacaftor and ivacaftor compared to
those who receive placebo, tezacaftor and ivacaftor.
About the VX-445 Phase 3 StudiesVertex expects to
complete enrollment in the fourth quarter of 2018 for two Phase 3
studies evaluating VX-445 in triple combination with tezacaftor and
ivacaftor in people with one F508del mutation and one minimal
function mutation and in people with two F508del mutations. The
study designs, including the pre-specified interim analysis for the
study in people with one F508del mutation and one minimal function
mutation, are the same as were used for the VX-659 Phase 3 program
noted above.
About CFCF 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 proteins
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
cells 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 VertexVertex 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 eight 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™ (tezacaftor/ivacaftor and ivacaftor), VX-659 and VX-445
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
second paragraph and the information provided regarding (i) the
timing of expected data from the Phase 3 studies of VX-659 and
VX-445, (ii) the timing of expected completion of enrollment for
the Phase 3 studies of VX-445, (iii) the company’s expectation
that it will evaluate VX-659 and VX-445 data to choose the best
regimen to submit for potential regulatory approval no later than
mid-2019, (iv) information regarding the planned interim analysis
and (v) information regarding the timing and type of data the
company intends to disclose from the Phase 3 studies. 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
that data from the Phase 3 development programs may not support
continued development or approval of the company's
triple-combination regimens 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:North America:Heather Nichols,
617-341-6992orEurope & Australia:Rebecca Hunt, +44 7718 962
690
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