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Vertex Corrects and Provides Additional Data from Recent Interim Analysis of Phase 2 Combination Study of VX-809 and KALYDECO™ (ivacaftor) in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
- Corrected Data: Responder analysis showed 35% of patients experienced an absolute improvement in lung function (FEV1) of at least 5 percentage points and 19% had at least a 10 percentage-point improvement when treated with VX-809 and KALYDECO -
- Additional Data: Patients treated with VX-809 and KALYDECO experienced an 8.5 percentage point mean absolute improvement in lung function compared to patients treated with placebo (p=0.002) -
- Vertex plans to start a pivotal study of this combination to treat the underlying cause of CF in adults with two copies of the F508del mutation, pending final data and discussions with regulatory agencies -
The actual absolute improvements in lung function for these patients are: approximately 35 percent (13/37) experienced an absolute improvement of 5 percentage points or more and approximately 19 percent (7/37) experienced an absolute improvement of 10 percentage points or more from baseline to Day 56. As previously announced, none of the patients treated with placebo (0/11) achieved a 5 percentage-point or more mean absolute improvement in lung function from baseline to Day 56.
Additional data from the interim analysis are provided today for people with two copies of the F508del mutation. A mean absolute improvement in lung function of 8.5 percentage points was observed among those who were treated with VX-809 and KALYDECO compared to placebo (p=0.002). In addition, the within-group mean absolute improvement from baseline to Day 56 was 4.0 percentage points (p=0.002) for patients treated with the combination. From baseline to Day 56, those treated with placebo experienced a mean absolute decrease in lung function of 4.6 percentage points (p=0.04).
These data are from a planned interim analysis that was conducted after approximately half of the patients completed 56 days of treatment. Evaluation of patients with one copy (heterozygous, n=21) of the F508del mutation is ongoing. All patients have now completed dosing. Analyses are ongoing and complete data, including statistical analyses for all patient groups, will be available mid-year. Vertex plans to start a pivotal study of VX-809 and KALYDECO in people with two copies of the F508del mutation, pending final study results and discussions with regulatory agencies.
Interim Lung Function Data
|VX-809 alone (200mg, 400mg or 600mg; QD) for 28 days followed by the addition of KALYDECO (250mg, q12h) for 28 days (n=37)||
|Mean absolute change in FEV1 from baseline to Day 56 compared to placebo||8.5 (p=0.002)||N/A|
|Mean absolute change in FEV1 from baseline to Day 56 for pooled treatment and placebo groups||4.0 (p=0.002)||-4.6 (p=0.04)|
|≥ 5 percentage point absolute improvement from baseline to Day 56||35% (13/37) *||0%|
|≥ 10 percentage point absolute improvement from baseline to Day 56||19% (7/37) *||0%|
"The improvements in lung function seen to date in this study exceeded
our expectations. We're continuing to move forward as quickly as
possible toward a pivotal study of VX-809 and KALYDECO in people with
two copies of the F508del mutation," said
Safety results are the same as those announced on
About this Phase 2 Study
Data from the first part of this study were announced in 2011. The interim data announced this month are from the second part of the ongoing Phase 2 randomized, double-blind, placebo-controlled study. This part of the study enrolled 108 people with CF ages 18 and older with one or two copies of the F508del mutation who were divided into five treatment groups of approximately 20 patients each. Three groups of homozygous patients were randomized to receive VX-809 alone (200mg, 400mg or 600mg) for 28 days and then in combination with KALYDECO (250mg) for an additional 28 days. One group of heterozygous patients received VX-809 alone (600mg) for 28 days and then in combination with KALYDECO (250mg) for an additional 28 days. The improvements in lung function were primarily observed following the addition of KALYDECO from Day 28 and maintained through Day 56. The placebo group includes both homozygous and heterozygous patients.
Cystic fibrosis is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. Located at the surface of cells, CFTR proteins act as channels to regulate the flow of salt and water into and out of the cells. In people with the F508del mutation in the CFTR gene, little to no CFTR protein reaches the cell surface. As a result, thick, sticky mucus builds up and blocks the passages in many organs, leading to a variety of symptoms. In particular, mucus builds up and clogs the airways in the lungs, causing chronic lung infections and progressive lung damage. VX-809, known as a CFTR corrector, is believed to help CFTR proteins reach the cell surface. KALYDECO, known as a CFTR potentiator, keeps the CFTR protein channels open longer to increase the flow of salt and water into and out of the cell.
VX-809 and KALYDECO were discovered as part of a collaboration with
Conference Call for Media and Investors
Vertex will host a conference call and webcast today,
To listen to the live call on the telephone, dial 1-866-516-1003 (
The conference ID number for the live call and replay is 86428080.
The call will be available for replay via telephone commencing
Following the live webcast, an archived version will be available on
Vertex's website until
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 30,000 people in
In people with the most common mutation in the CFTR gene, F508del, little to no CFTR protein reaches the cell surface. Globally, nearly half (46 percent) of people with CF have two copies of the F508del mutation and one-third (33 percent) have one copy of the F508del mutation.
KALYDECO™ (ivacaftor) is the first treatment to target the underlying
cause of CF. KALYDECO (150mg, q12h) was approved by the
Vertex retains worldwide rights to develop and commercialize KALYDECO.
Indication and Important Safety Information
KALYDECO (150mg, q12h) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a certain mutation in their CFTR gene called the G551D mutation.
KALYDECO is not for use in people with CF due to other mutations in the CFTR gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CFTR gene.
It is not known if KALYDECO is safe and effective in children under 6 years of age.
KALYDECO should not be used with certain medicines, including the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort.
KALYDECO can cause serious side effects. High liver enzymes in the blood have occurred in patients taking KALYDECO as well as those receiving placebo. Regular assessment is recommended.
The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (common cold) including 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. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.
Please see full Prescribing Information for KALYDECO at www.KALYDECO.com.
Collaborative History with
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the
Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis, epilepsy and other life-threatening diseases.
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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 Dr. Wright's
statements in the sixth paragraph of this press release, and statements
regarding (i) Vertex's plan to start a pivotal study of VX-809 and
KALYDECO in people with two copies of the F508del mutation, pending
final data and discussions with regulatory agencies, (ii) the expected
availability of complete data from the study in mid-2012 and (iii) the
evaluation of patients with one copy of the F508del mutation being
ongoing. 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 the final outcomes of
this clinical trial or future clinical trials of VX-809 and KALYDECO may
be less favorable than the interim analysis reported today, or may not
be favorable at all, that final data from heterozygous patients may not
be favorable or may be less favorable than the data from homozygous
patients, that final data and/or discussions with regulatory agencies
regarding the scope and design of future clinical trials may result in
additional clinical trials needing to be conducted before Vertex can
initiate the first pivotal clinical trials evaluating VX-809 in
combination with KALYDECO and other risks listed under Risk Factors in
Vertex's annual report and quarterly reports filed with the
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