Vertex Provides Update on Ongoing Phase 3 Program for VX-661 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis
August 15 2016 - 4:05PM
Business Wire
-Results from Part A of Phase 3 study in people
with a mutation that results in minimal cystic fibrosis
transmembrane conductance regulator (CFTR) protein function do not
support continuation of the study-
-Enrollment complete in Phase 3 study in people
with two copies of the F508del mutation-
-Enrollment expected to be completed in
September in Phase 3 study in people with one F508del mutation and
one residual function mutation-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
provided an update on its ongoing Phase 3 development program of
its investigational compound VX-661 in combination with ivacaftor,
which includes four studies that together are expected to enroll
more than 1,000 people with cystic fibrosis (CF). Based on a
planned interim futility analysis conducted by the study’s
independent Data Safety Monitoring Board (DSMB), Vertex plans to
stop the study of VX-661 and ivacaftor in people with one copy of
the F508del mutation and one copy of a mutation that results in
minimal CFTR protein function (F508del het/min). There were no
safety concerns noted in the DSMB’s review of the data. Vertex also
today announced that enrollment is now complete in the study of
VX-661 and ivacaftor in people with two copies of the F508del
mutation (F508del homozygous) and that the company expects to
complete enrollment in the study of people with one copy of the
F508del mutation and one copy of a residual function mutation in
September.
“While we recognize that people with CF with minimal function
mutations have a form of the disease that is particularly difficult
to treat, we believed it was important to evaluate whether a dual
combination of VX-661 and ivacaftor could provide some benefit to
these patients given they do not today have a medicine to treat the
cause of their disease,” said Jeffrey Chodakewitz, M.D., Executive
Vice President and Chief Medical Officer at Vertex. “These results
suggest that a triple combination regimen may provide this group of
people with CF the best chance at obtaining a meaningful benefit
and we look forward to beginning the first study of a
next-generation corrector together with VX-661 and ivacaftor in
this group of patients later this year, pending data from our
ongoing Phase 1 studies in healthy volunteers.”
The VX-661 Phase 3 program includes four Phase 3 studies in
multiple different groups of people with CF who have at least one
copy of the F508del mutation. The studies are evaluating VX-661
dosed as 100 mg once daily (QD) in combination with ivacaftor dosed
as 150 mg every 12 hours (q12h). These studies include people with
CF with the following mutations:
- Two Copies of the F508del
Mutation: In August, Vertex completed enrollment in a study
evaluating 24 weeks of treatment with VX-661 in combination with
ivacaftor in approximately 500 people with CF who have two copies
of the F508del mutation. Data from this study are expected in the
first half of 2017.
- One Copy of the F508del Mutation and
a Second Mutation that Results in Residual CFTR Function: In
September, Vertex expects to complete enrollment in a study
evaluating VX-661 in combination with ivacaftor in approximately
200 people with residual function mutations. The crossover study
includes two 8-week dosing periods, separated by an 8-week washout
period. The study includes an arm of ivacaftor monotherapy, in
addition to an arm evaluating VX-661 in combination with ivacaftor
and a placebo arm. Data from this study are expected in the first
half of 2017.
- One Copy of the F508del Mutation and
a Second Mutation that Results in a Gating Defect in the CFTR
Protein: Enrollment is ongoing in a study designed to evaluate
VX-661 in combination with ivacaftor in people with gating
mutations that have been shown to be responsive to ivacaftor alone.
The study is expected to enroll approximately 200 patients and is
evaluating 8 weeks of treatment with VX-661 in combination with
ivacaftor. Enrollment is expected to complete in late 2016 or early
2017.
- One Copy of the F508del Mutation and
a Second Mutation that Results in Minimal CFTR Function: In
April, Vertex completed enrollment of approximately 150 people in
Part A of a two-part study evaluating people with mutations that
result in minimal CFTR function. A planned interim futility
analysis was conducted by the study’s independent DSMB after at
least 8 weeks of dosing to determine whether to stop the study or
to continue the study and initiate enrollment in Part B. The
analysis showed that the combination of VX-661 and ivacaftor did
not result in a pre-specified improvement in lung function. The
DSMB recommended that Vertex stop the study and not initiate
enrollment in Part B. There were no safety concerns noted in the
DSMB’s review of the data. Vertex plans to close this study based
on the recommendation of the DSMB, and patients from Part A of the
study who enrolled in the long-term extension study will be
transitioned off the combination of VX-661 and ivacaftor.
Vertex plans to submit a New Drug Application (NDA) to the FDA
for VX-661 in combination with ivacaftor in the second half of
2017, pending data from the Phase 3 program. The NDA is expected to
include data from the study in people with minimal function
mutations.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening genetic disease
affecting approximately 75,000 people in North
America, Europe and Australia. Today, the median
predicted age of survival for a person with CF is between 34 and 47
years, but the median age of death remains in the mid-20s.
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 more than 1,900 known mutations in
the CFTR gene. Some of these mutations, which can be
determined by a genetic, 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 proteins in people with CF
results in poor flow of salt and water into and out of the cell in
a number of organs, including the lungs. This leads to the buildup
of abnormally thick, sticky mucus that can cause chronic lung
infections and progressive lung damage.
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.
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
second paragraph of the press release and statements regarding (i)
the timing of completing enrollment of Phase 3 studies of VX-661 in
combination with ivacaftor and (ii) plans to submit an NDA to the
FDA for VX-661 in combination with ivacaftor. 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 VX-661 in
combination with ivacaftor 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,
617-341-6992orMedia:mediainfo@vrtx.comUS: 617-341-6992Europe
& Australia: +44 20 3204 5275
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