Vertex Announces Data Presentations at European Cystic Fibrosis Society (ECFS) Conference
June 11 2015 - 9:00AM
Business Wire
-Analysis from TRAFFIC and TRANSPORT extension
study of lumacaftor in combination with ivacaftor showed that
improvements in lung function and other measures of disease were
maintained through 48 weeks in people with cystic fibrosis who have
two copies of the F508del mutation-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced data from PROGRESS, the long-term extension study of the
investigational regimen ORKAMBI™ (lumacaftor/ivacaftor). The
ongoing extension study enrolled people with cystic fibrosis (CF)
ages 12 and older who have two copies of the F508del mutation and
completed 24 weeks of treatment in the Phase 3 TRAFFIC and
TRANSPORT studies (lumacaftor/ivacaftor combination and placebo
treatment groups) and met certain other eligibility criteria. These
data are included in one of nine presentations at the 38th European
Cystic Fibrosis Society (ECFS) Conference, June 10-12, 2015, in
Brussels, Belgium.
Lumacaftor in combination with ivacaftor in patients with
cystic fibrosis who are homozygous for the F508del-CFTR
mutation (ECFS Abstract WS01.3, oral presentation during
Workshop 1 - Strategies to correct CFTR defects.)
One thousand thirty-one people who completed 24 weeks of
treatment in either of the Phase 3 studies, TRAFFIC or TRANSPORT,
entered the 96-week PROGRESS Phase 3 extension study in which
everyone received one of two lumacaftor/ivacaftor combination
regimens. An interim analysis was conducted once all patients
completed 24 weeks in PROGRESS for a total of 48 total weeks of
treatment (48 weeks of treatment with a combination regimen for
patients who received a combination regimen in TRAFFIC and
TRANSPORT; 24 weeks of treatment with a combination regimen for
patients who received placebo in TRAFFIC and TRANSPORT).
These data showed that the initial improvements in lung function
(percent predicted forced expiratory volume in one second, or
ppFEV1) observed in the 24-week TRAFFIC and TRANSPORT studies among
those treated with a lumacaftor/ivacaftor combination were
sustained through 48 weeks of treatment across all patients.
Reduced rates of pulmonary exacerbations and improvements in body
mass index (BMI) and patient-reported respiratory symptoms as
measured by the respiratory domain of the Cystic Fibrosis
Questionnaire Revised (CFQ-R) were also maintained over 48 weeks.
In addition, the pattern and magnitude of response observed after
the initiation of combination treatment across all patients who
received placebo in TRAFFIC and TRANSPORT and subsequently received
a combination regimen in PROGRESS were similar to those seen among
patients who received a combination regimen in TRAFFIC and
TRANSPORT.
At the time of this analysis, the safety and tolerability
results, including the type and frequency of adverse events and
serious adverse events, were consistent with those observed in
TRAFFIC and TRANSPORT, and no new safety concerns were identified.
Over 48 weeks, the most common adverse events were infective
pulmonary exacerbation, cough and increased sputum. The incidence
of serious adverse events during PROGRESS was generally similar to
TRAFFIC and TRANSPORT.
Other data presented at the Conference include:
- “Lumacaftor/ivacaftor combination
therapy in CF patients homozygous for F508del-CFTR with severe lung
dysfunction.” Poster 143.
- “VX-661 in combination with ivacaftor
in patients with cystic fibrosis and the F508del-CFTR mutation.”
ECFS Abstract WS01.4, oral presentation during Workshop 1 -
Strategies to correct CFTR defects.
- “R117H-CFTR has a defect in channel
gating activity that can be potentiated by ivacaftor.” ECFS
Abstract WS06.2, oral presentation during Workshop 6 – Fixing ion
transport.
- “An open-label study of the safety,
pharmacokinetics, and pharmacodynamics of ivacaftor in patients
aged 2 to 5 years with cystic fibrosis and a CFTR gating mutation:
the KIWI study.” ECFS Abstract WS01.5, oral presentation during
Workshop 1 - Strategies to correct CFTR defects.
- “Prevalence of cataracts in a
population of cystic fibrosis patients homozygous for the F508del
mutation.” Poster 196.
- “Manifestation and progression of
illness in young children with cystic fibrosis: a targeted
literature review.” Poster 186.
- “Frequency and costs of pulmonary
exacerbations and association with % predicted FEV1 in patients
with cystic fibrosis.” Poster 190.
- “Lung function and health care resource
utilization in patients with cystic fibrosis.” Poster 191.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening 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 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 proteins
at the cell surface. The defective or missing CFTR protein 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 build-up of
abnormally thick, sticky mucus that can cause chronic lung
infections and progressive lung damage that eventually leads to
death. 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.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation. This
collaboration was expanded to support the accelerated discovery and
development of Vertex's CFTR modulators.
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 five 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, statements regarding data that will
be presented at ECFS. While Vertex believes the forward-looking
statements contained in this press release are accurate, 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 regulatory authorities may not approve, or approve on
a timely basis, lumacaftor in combination with ivacaftor 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,
+1 617-341-6108orKelly Lewis, +1 617-961-7530orEric Rojas, +1
617-961-7205orMedia:mediainfo@vrtx.comEurope & Australia: +41
22 593 6066orNorth America: +1 617-341-6992
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