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Data from Phase 2 Combination Study of VX-809 and Ivacaftor in People with Cystic Fibrosis Who Have the Most Common Genetic Mutation (F508del) Presented at North American Cystic Fibrosis Conference
-- Additional KALYDECO™ (ivacaftor) and CF pipeline presentations highlight Vertex's commitment to advancing CF treatment by targeting the underlying cause of the disease --
"These study results are an exciting step for the cystic fibrosis community, and we look forward to the start of this pivotal program early next year," said Dr. Boyle. "We are particularly encouraged to make such important progress in efforts to treat the underlying cause of the disease in people with two copies of the F508del mutation, who account for the single largest group of people with CF."
Shortly after Dr. Boyle's presentation, the slides will be available on
the NACFC-Handout Hub website at: http://nacfcdl.cff.org/Pages/library.aspx.
Vertex will also host a webcast today,
Updates to Vertex's
For more than 14 years, Vertex has been working to develop new medicines
to treat the underlying cause of CF in as many people as possible. CF is
caused by defective or missing CFTR proteins resulting from mutations in
the CFTR gene. Earlier this year, Vertex received regulatory
Vertex's ongoing work in CF includes additional studies of ivacaftor monotherapy in patients who may benefit from improved CFTR protein function; the evaluation of a combination of a CFTR corrector and the CFTR potentiator, ivacaftor, in patients with the F508del mutation; and continued research aimed at discovering additional potential medicines that could be evaluated as part of future combination treatments. Vertex has multiple studies planned and underway to support these goals, and the company today provided the following updates:
Ivacaftor Monotherapy Studies
Study of Ivacaftor in People with the R117H Mutation: In
July 2012, Vertex initiated a Phase 3 study of ivacaftor in people with CF ages 6 and older who have at least one copy of the R117H mutation. Approximately 3 percent of people with CF in the United Stateshave at least one R117H mutation.
Study of Ivacaftor in People with Gating Mutations: In
July 2012, Vertex initiated a Phase 3 study of ivacaftor in people with CF ages 6 and older who have at least one non-G551D CFTR gating mutation. Approximately 1 percent of people with CF in the United Stateshave at least one non-G551D gating mutation.
Study of Ivacaftor in People with Residual CFTR Function: Vertex
recently initiated a Phase 2 proof-of-concept study of ivacaftor in
people with clinical evidence of residual CFTR function. This is the
first study to evaluate the efficacy of ivacaftor based on a person's
clinical symptoms and characteristics, or phenotype, rather than
solely on their CFTR mutation, or genotype. Between 5 percent
and 10 percent of people with CF in
the United Stateshave residual CFTR function.
- Study of Ivacaftor in Children Ages 2 to 5: Vertex recently initiated a Phase 3 study of ivacaftor in children with CF ages 2 to 5 who have a gating mutation.
"KALYDECO was approved less than ten months ago in the U.S., and since
then, the majority of eligible patients have started treatment. We also
recently received approval in
Combination Treatment for People with the Most Common Mutation, F508del
- Study of VX-661 and Ivacaftor in People with the F508del Mutation: A Phase 2 study of VX-661 in combination with ivacaftor is underway in people with two copies of the F508del mutation. Final data from this study are expected in 2013.
- Part 3 (Cohort 3) of VX-809 and Ivacaftor Study: Enrollment is complete in the third part of the Phase 2 study of VX-809 alone and in combination with ivacaftor. Part 3 of this study is evaluating the pharmacokinetics, safety and tolerability of a twice-daily (q12h) combination of VX-809 (400mg) and ivacaftor (250mg). Vertex expects to use data from this part of the study to support the pivotal program for VX-809 and ivacaftor.
Continued Productivity in
Multiple Vertex and other scientific presentations at NACFC are focused
on the use of CFTR correctors to treat the underlying cause of CF.
Vertex has an active and ongoing research program that has identified
next-generation correctors. This research is being conducted as part of
the company's collaboration with
Vertex will conduct a webcast today,
KALYDECO™ (ivacaftor) is the first treatment to target the underlying
cause of CF in people with the G551D mutation in the CFTR gene.
Known as a CFTR potentiator, KALYDECO 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. KALYDECO
(150mg, q12h) was first approved by the
Vertex retains worldwide rights to develop and commercialize KALYDECO.
KALYDECO is under Priority Review by the
Indication and Important Safety Information for KALYDECO™ (ivacaftor)
KALYDECO (150mg tablets) is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene.
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.
High liver enzymes (transaminases, ALT and AST) have been reported in patients receiving KALYDECO. It is recommended that ALT and AST be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal. Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing. Moderate transaminase elevations are common in subjects with CF. Overall, the incidence and clinical features of transaminase elevations in clinical trials was similar between subjects in the KALYDECO and placebo treatment groups. In the subset of patients with a medical history of elevated transaminases, increased ALT or AST have been reported more frequently in patients receiving KALYDECO compared to placebo.
Use of KALYDECO with medicines that are strong CYP3A inducers such as the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort substantially decreases exposure of KALYDECO, which may diminish effectiveness. Therefore, co-administration is not recommended.
The dose of KALYDECO must be adjusted when concomitantly used with potent and moderate CYP3A inhibitors.
KALYDECO can cause serious adverse reactions including abdominal pain and high liver enzymes in the blood. The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (the common cold), including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; and dizziness. These are not all the possible side effects of KALYDECO. A list of the adverse reactions can be found in the full product labeling for each country where KALYDECO is approved. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 30,000 people in
CF is caused by defective or missing CFTR proteins 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 1,800 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 absence of working CFTR proteins results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs.
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
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit www.cff.org.
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 and other life-threatening diseases.
Founded more than 20 years ago in
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. Boyle's statements in the second paragraph of this press
release, Dr. Mueller's statements in the tenth paragraph of this press
release and statements regarding (i) Vertex's commitment to advancing CF
treatment by targeting the underlying cause of the disease, (ii)
Vertex's ongoing and planned clinical trials of ivacaftor alone and in
combination with its CFTR corrector compounds, including its plans to
initiate a pivotal program in early 2013 that is expected to evaluate
VX-809 in combination with ivacaftor and (iii) Vertex's ongoing research
program for the discovery and development of CFTR corrector compounds.
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 outcomes of ongoing or planned
clinical trials may not be favorable; that the initiation of a pivotal
program to evaluate VX-809 in combination with ivacaftor may be
prevented or delayed; that the company may not be able to successfully
develop new treatments for CF; and other risks listed under Risk Factors
in Vertex's annual report and quarterly reports filed with the
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