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Vertex Outlines 2015 Business Priorities to Support the Development, Approval and Launch of New Medicines for the Treatment of People with Cystic Fibrosis
-Priority Review granted for combination of lumacaftor and ivacaftor
in people with cystic fibrosis ages 12 and older who have two copies of
the F508del mutation; PDUFA date of
-More than 3,700 people with CF expected to be eligible to receive
KALYDECO by end of 2015 supporting continued growth in KALYDECO global
revenues; 2015 KALYDECO net revenue guidance of
-Pivotal Phase 3 program of VX-661 in combination with ivacaftor to begin in February in people with cystic fibrosis who have either one or two copies of the F508del mutation-
"Vertex enters a year where the potential approval and launch of the
combination of lumacaftor and ivacaftor, and continued geographic and
label expansion for KALYDECO, are expected to significantly increase the
number of people treated with our medicines," said
Vertex today provided the following updates:
At the start of 2014, more than 2,200 people with CF ages 6 and older
with the G551D mutation were eligible for treatment with KALYDECO in
Vertex today announced that its New Drug Application (NDA) for the use
of ivacaftor in children ages 2 to 5 who have the G551D or one of the
eight additional gating mutations was accepted for filing by the
Lumacaftor in Combination with Ivacaftor in People with Two Copies of the F508del Mutation
Vertex today announced that the
Vertex also plans to initiate a study of lumacaftor in combination with ivacaftor in children ages 6 to 11 who have two copies of the F508del mutation in the first half of 2015. The study is expected to evaluate the combination regimen as part of a single-arm, open-label design in approximately 50 children. The primary endpoint of the study will be safety and pharmacokinetics.
VX-661 in Combination with Ivacaftor in People with One or Two Copies of the F508del Mutation
Vertex is currently conducting a 12-week Phase 2 study of VX-661 in
combination with ivacaftor in people with CF who have two copies of the
F508del mutation. Vertex completed enrollment in the study in
Vertex plans to initiate a Phase 3 pivotal program of VX-661 in combination with ivacaftor in February. The initiation of the Phase 3 program is based on safety and efficacy data from Phase 2 studies of VX-661, including interim data from the ongoing 12-week Phase 2 study and previously completed studies of VX-661 in combination with ivacaftor in people with two copies of the F508del mutation and in people with one copy of the F508del mutation and one copy of the G551D mutation, and recent regulatory discussions regarding the design of the Phase 3 program. The program will consist of four studies that will evaluate VX-661 dosed as 100 mg once daily (QD) in combination with ivacaftor dosed as 150 mg every 12 hours (q12h). The first of the studies is expected to begin in February. Additional details on each of the four planned studies are provided below:
Two Copies of the F508del Mutation: In February, Vertex plans
to begin a Phase 3 study to evaluate the combination of VX-661 and
ivacaftor in people ages 12 and older who have two copies of the
F508del mutation. The primary endpoint of the study will be absolute
change in ppFEV1 through six months of treatment versus
placebo. The study will enroll approximately 500 patients in
North Americaand Europe. The majority of the study sites will be in Europe, including the countries of Denmark, France, Germany, the Republic of Ireland, Italy, the Netherlands, Spain, Sweden, Switzerlandand the United Kingdom.
One Copy of the F508del Mutation and a Second Mutation that Results
in a Gating Defect in the CFTR Protein: In the second quarter of
2015, Vertex plans to begin a Phase 3 study to evaluate the
combination of VX-661 and ivacaftor in people ages 12 and older who
have one copy of the F508del mutation and a second mutation that
results in a gating defect in the CFTR protein. The primary endpoint
of the study will be absolute change in ppFEV1 through
eight weeks of treatment with VX-661 and ivacaftor versus ivacaftor
alone. The study will enroll approximately 200 patients in
North Americaand Europe.
One Copy of the F508del Mutation and a Second Mutation That Results
in Residual CFTR Function: In the second quarter of 2015, Vertex
plans to begin a Phase 3 study to evaluate the combination of VX-661
and ivacaftor in people ages 12 and older who have one copy of the
F508del mutation and a second mutation that results in residual CFTR
function. This study will also evaluate ivacaftor dosed without
VX-661. The primary endpoint of the study will be absolute change in
ppFEV1 through eight weeks of treatment as part of a
crossover design. The study will enroll approximately 300 patients in
North Americaand Europe.
- One Copy of the F508del Mutation and A Second Mutation That Results in Minimal CFTR Function: In the second quarter of 2015, Vertex plans to begin a Phase 3 study to evaluate the combination of VX-661 and ivacaftor in people ages 12 and older who have one copy of the F508del mutation and a second mutation that results in minimal CFTR function. The study will initially enroll approximately 120 patients, and the primary endpoint will be absolute change in ppFEV1 through 12 weeks of treatment versus placebo. Expansion of the study to an additional approximately 150 patients will depend on an interim futility analysis of efficacy data from the initial approximately 120 patients.
Vertex has multiple next-generation correctors in the lead-optimization stage of research and expects to begin clinical development of a next-generation corrector in 2015. In vitro data showed that a triple combination of VX-661, ivacaftor and a next-generation corrector resulted in increased chloride transport in human bronchial epithelial cells with one or two copies of the F508del mutation, as compared to the use of a single corrector in combination with ivacaftor.
Research and Early-stage Development Programs
In addition to its ongoing CF research activities, Vertex is advancing multiple additional research and early development programs aimed at the discovery of future transformative medicines for serious diseases, with a focus on specialty markets. The company's research and early development efforts are focused on oncology and multiple neurological diseases. Vertex expects multiple compounds in oncology and neurological diseases to be in clinical development in 2015.
"We expect 2015 to be a year of rapid and significant growth in revenues
based on treating many more people with CF following the potential
approval and launch of the combination of lumacaftor and ivacaftor and
further label and geographic expansion for KALYDECO," said
Vertex today provided the following financial outlook and will provide
complete financial guidance on its year-end conference call on
Vertex entered 2015 with approximately
$1.4 billionin cash, cash equivalents and marketable securities. In July 2014, Vertex entered into a credit agreement that provides for a secured loan of up to $500 million, $300 millionof which Vertex received in July 2014and is currently outstanding.
Vertex expects total 2015 KALYDECO net revenues of
$560to $580 million. Vertex expects to report total 2014 KALYDECO net revenues of approximately $460 millionand fourth quarter KALYDECO net revenues of approximately $120 million. Anticipated 2015 KALYDECO net revenues reflect:
Use of KALYDECO by eligible patients in
Australiafollowing the completion of reimbursement discussions in late 2014
Use of KALYDECO in people in
the United Stateswith the R117H mutation following FDAapproval in late 2014
- The completion of reimbursement discussions for gating mutations in certain European countries
Use of KALYDECO in children with CF ages 2 to 5 with the G551D or
other gating mutations in
the United States, based on potential approval in March 2015
- Use of KALYDECO by eligible patients in
Vertex expects that its combined non-GAAP R&D and SG&A expenses in
2015 will be in the range of
$1.05to $1.1 billion. The increase as compared to 2014 is primarily a result of launch preparation activities for lumacaftor in combination with ivacaftor and the planned pivotal Phase 3 development program for VX-661 in combination with ivacaftor. Vertex's expected non-GAAP R&D and SG&A expenses exclude stock-based compensation expense and certain other expenses recorded in 2015.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO® (ivacaftor)
Ivacaftor (150 mg tablets) is a cystic fibrosis transmembrane conductance regulatory (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene.
Ivacaftor is not effective in patients with CF with 2 copies of the F508del mutation (F508del/F508del) in the CFTR gene. The safety and efficacy of ivacaftor in children with CF younger than 6 years of age have not been established.
Elevated liver enzymes (transaminases; ALT and AST) have been reported in patients receiving ivacaftor. It is recommended that ALT and AST be assessed prior to initiating ivacaftor, 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 ivacaftor dosing.
Use of ivacaftor 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 ivacaftor and may diminish effectiveness. Therefore, co-administration is not recommended. The dose of ivacaftor must be adjusted when used concomitantly with strong and moderate CYP3A inhibitors or when used in patients with moderate or severe hepatic disease.
Cases of non-congenital lens opacities/cataracts have been reported in pediatric patients up to 12 years of age treated with ivacaftor. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating ivacaftor treatment.
Ivacaftor can cause serious adverse reactions including abdominal pain and high liver enzymes in the blood. The most common side effects associated with ivacaftor 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 ivacaftor. A list of the adverse reactions can be found in the product labeling for each country where ivacaftor is approved. Patients should tell their healthcare providers about any side effect that bothers them or does not go away.
Please see KALYDECO (ivacaftor) U.S. Prescribing Information, EU Summary of Product Characteristics, Canadian Product Monograph, Australian Consumer Medicine Information and Product Information, Swiss Prescribing Information and Patient Information, and the New Zealand Datasheet and Consumer Medicine Information.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening 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 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 buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.
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
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
The company will webcast its corporate presentation at the 33rd
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