- Presentation of data from a Phase 3 study of
ORKAMBI in children ages 6-11 with two copies of the F508del
mutation demonstrated improvements in lung function and sweat
chloride; study also published online in The Lancet Respiratory
Medicine today -
- ECFS data presentations demonstrate that
treating the underlying cause of cystic fibrosis with CFTR
modulators can modify the progression of the disease -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced nine presentations of data on ORKAMBI®
(lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) at the 40th
European Cystic Fibrosis Society (ECFS) Conference, being held June
7-10, 2017. Data from a Phase 3 placebo-controlled study of ORKAMBI
in children with cystic fibrosis (CF) ages 6 through 11 who have
two copies of the F508del mutation were presented at the meeting
and published online today in The Lancet Respiratory Medicine. In
addition, results from a study of ORKAMBI in people ages 12 and
older who have two copies of the F508del mutation and advanced lung
disease as well as a post-hoc analysis of long-term use of ORKAMBI
in three Phase 3 studies were also presented at the meeting. The
data presented at the Conference demonstrate that treating the
underlying cause of CF with CFTR modulators can modify the
progression of the disease.
“In nearly 20 years of research in collaboration with the cystic
fibrosis community, we’ve made remarkable progress in efforts to
change the way CF is treated by developing medicines that address
the underlying cause of the disease, not just the symptoms,” said
Jeffrey Chodakewitz, M.D., Executive Vice President and Chief
Medical Officer at Vertex. “Thousands of patients around the world
are benefitting from KALYDECO and ORKAMBI, which have both shown
the ability to modify the progression of CF. The data presented at
this meeting further demonstrate that treatment with CFTR
modulators can deliver early and sustained benefits for eligible
patients.”
ORKAMBI in children ages 6 to 11 (“Efficacy and safety of
lumacaftor/ivacaftor in patients aged 6-11 years with cystic
fibrosis homozygous for F508del-CFTR: a randomized
placebo-controlled Phase 3 trial.” WS13.4.)
Data were presented for the first time from a Phase 3
randomized, double-blind, placebo-controlled study that evaluated
ORKAMBI in 204 children with CF ages 6 through 11 who have two
copies of the F508del mutation. At the start of the study, the
average baseline predicted forced expiratory volume in one second
(ppFEV1) was approximately 90. In the study, all children received
a twice-daily fixed-dose combination of lumacaftor (200mg) and
ivacaftor (250mg) for 24 weeks. As announced in November 2016, the
study met its primary endpoint of absolute change in lung clearance
index (LCI2.5) through 24 weeks of treatment, demonstrating a
statistically significant improvement in LCI2.5 among those treated
with ORKAMBI compared to placebo. The study also demonstrated
significant improvements in ppFEV1 and sweat chloride in children
receiving ORKAMBI compared with those receiving placebo.
Overall, safety data were similar to those observed in a
previous Phase 3 open-label safety study in children ages 6 through
11. In the placebo-controlled study, the most common adverse events
that occurred more frequently among those receiving ORKAMBI
compared to placebo were infective pulmonary exacerbation,
productive cough, nasal congestion, oropharyngeal pain, abdominal
pain upper, headache, upper respiratory tract infection and sputum
increased. The incidence of liver enzyme elevations and respiratory
events were slightly higher in the ORKAMBI group compared to
placebo. Respiratory events were mild to moderate in severity and
the majority were resolved without interrupting treatment.
Treatment discontinuations due to adverse events were low across
those receiving placebo (n=2) and those receiving ORKAMBI (n=3)
through 24 weeks.
Additional details from the study will be presented at ECFS as
part of Workshop 13, New therapies targeting CFTR: what's new from
the clinical trials pipeline? and as part of an invited talk during
Symposium 22, Best of Journal of Cystic Fibrosis and The Lancet
Respiratory Medicine Symposium. The data were also published online
today in The Lancet Respiratory Medicine.
In the U.S., ORKAMBI was approved in September 2016 for use in
children with CF ages 6 through 11 who have two copies of the
F508del mutation. In the EU, Vertex submitted a Marketing
Authorization Application (MAA) line extension in March 2017 for
the use of ORKAMBI in these children.
ORKAMBI in advanced lung disease (“Lumacaftor/ivacaftor
in patients with cystic fibrosis and advanced lung disease
homozygous for F508del-CFTR: a 24-week open-label study.” Poster
55.)
Data were presented for the first time from a Phase 3b
open-label study that evaluated ORKAMBI in people with CF ages 12
and older who have two copies of the F508del mutation and advanced
lung disease, defined as ppFEV1 less than 40 at screening. At the
start of the 24-week study, the average baseline ppFEV1 was 29.1.
Overall, the incidence of respiratory adverse events was higher
than in other studies of patients who had higher baseline ppFEV1.
Aside from respiratory adverse events, the safety profile of
ORKAMBI seen in the study was generally consistent with the
established safety profile from other Phase 3 studies.
In the study, a subset of eighteen patients initiated treatment
with a half-dose of ORKAMBI (lumacaftor 200mg q12h / ivacaftor
125mg q12h) for one to two weeks and then transitioned to the full
dose (lumacaftor 400mg q12h / ivacaftor 125mg q12h). An analysis of
data from this study showed that these patients had a lower
incidence and shorter duration of respiratory adverse events
compared to those who initiated treatment on the full dose.
Correlation between Rate of Lung Function Decline and Acute
Improvements in Lung Function with ORKAMBI (“Relationship
between rate of percent predicted FEV1 decline and baseline and
acute change in percent predicted FEV1 in patients with cystic
fibrosis treated with lumacaftor/ivacaftor.” Poster 54.)
Progressive loss of lung function is the leading cause of death
in people with CF; therefore, slowing the decline of lung function
is a key goal of CF treatment. As previously reported, up to 120
weeks of ORKAMBI treatment in the Phase 3 TRAFFIC, TRANSPORT and
PROGRESS studies resulted in a reduced annual rate of ppFEV1
decline and mean ppFEV1 that remained above baseline. A post-hoc
analysis of these studies evaluated whether there is any
correlation between acute improvement in lung function and the
long-term rate of lung function decline. Results presented at the
meeting showed that treatment with ORKAMBI produces two effects on
lung function – an acute improvement in ppFEV1 and a reduced rate
of decline over the long term. The magnitude of the acute
improvement was not correlated with the reduction in the rate of
lung function decline. These data, together with similar results
previously reported for KALYDECO in patients with the G551D
mutation, suggest that baseline ppFEV1 or the magnitude of acute
ppFEV1 change are not predictors of the potential for disease
modification, measured in this case by a reduced rate of decline in
ppFEV1, with CFTR modulation.
Vertex continues to progress its CF development program.
The company is on track to submit a New Drug Application to the
U.S. Food and Drug Administration and an MAA to the European
Medicines Agency in the third quarter of 2017 for the
tezacaftor/ivacaftor combination treatment in people with CF ages
12 and older who have two copies of the F508del mutation and in
people who have one mutation that results in residual CFTR function
and one F508del mutation. In addition, studies evaluating four
different next-generation correctors in combination with tezacaftor
and ivacaftor are underway. Data in people with CF are expected in
the second half of 2017 for the studies evaluating the
next-generation correctors VX-440, VX-152 and VX-659 as part of
triple combination regimens with tezacaftor and ivacaftor.
About ORKAMBI® (lumacaftor/ivacaftor)
In people with two copies of the F508del mutation, the CFTR
protein is not processed and trafficked normally within the cell,
resulting in little-to-no CFTR protein at the cell surface.
Patients with two copies of the F508del mutation are easily
identified by a simple genetic test.
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. It is an oral
pill taken every 12 hours - once in the morning and once in the
evening.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About KALYDECO® (ivacaftor)
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, ivacaftor is an oral medicine that aims to help
the CFTR protein function more normally once it reaches the cell
surface, to help hydrate and clear mucus from the airways.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
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.
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) and tezacaftor were
discovered by Vertex as part of this collaboration.
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 seven 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 the
expected timing of (i) regulatory applications, including NDAs,
MAAs and MAA line extensions and (ii) the expected timing, clinical
trial designs and results for ongoing clinical studies of
next-generation correctors in combination with tezacaftor and
ivacaftor. 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
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)
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Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orEric Rojas, +1 617-961-7205orZach Barber, +1
617-341-6470orMedia:Europe & Australia:Megan Goulart, +44 20
3204 5275orNorth America:David Whitrap, +1 617 341
6992mediainfo@vrtx.com
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