-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
July 5, 2015-
-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 $560 - $580 million-
-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 Pharmaceuticals Incorporated (Nasdaq:VRTX) today outlined
2015 business priorities to support the development, approval and
launch of new medicines that treat the underlying cause of cystic
fibrosis (CF). The company also provided 2015 guidance for
KALYDECO® (ivacaftor) net revenues and for non-GAAP operating
expenses. These updates were made in conjunction with the 33rd
Annual J.P. Morgan Healthcare Conference that begins tomorrow in
San Francisco. Vertex's Chairman, President and Chief Executive
Officer, Jeffrey Leiden, M.D., Ph.D., will discuss the company's
continued execution of its corporate strategy and 2015 priorities
as part of a live presentation on Monday, January 12 at 9:30 a.m.
PT (12:30 p.m. ET). The presentation will be webcast on Vertex's
website, www.vrtx.com.
“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 Dr. Leiden. “We enter 2015 with a strong cash
position of approximately $1.4 billion, which, when combined with
continued revenue growth, will allow us to invest in key internal
and external opportunities in CF and other diseases to bolster our
pipeline and position the company to advance other transformative
medicines in the coming years.”
Vertex today provided the following updates:
KALYDECO (ivacaftor)
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 North America, Europe and Australia. Following the
expansion of the KALYDECO label throughout 2014, more than 3,100
people ages 6 and older are currently eligible for treatment with
KALYDECO, including people with one of eight gating mutations and,
in the United States, people with the R117H mutation. Vertex
currently expects that by the end of 2015, more than 3,700 people
may be eligible for treatment with KALYDECO, including children
ages 2 to 5 and, in Europe, adults with the R117H mutation.
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 Food and Drug Administration (FDA), and a target
review date of March 17, 2015 was set under the Prescription Drug
User Fee Act (PDUFA) for the FDA's approval decision.
In Europe, discussions are ongoing regarding reimbursement for
patients with non-G551D gating mutations. Additionally, a Marketing
Authorization Application (MAA) variation for ivacaftor in people
ages 18 and older with the R117H mutation and an MAA line extension
for ivacaftor in children ages 2 to 5 with gating mutations have
both been validated by the European Medicines Agency (EMA).
Validation indicates that the application is complete and starts
the regulatory review process by the Committee for Medicinal
Products for Human Use (CHMP). Following its review, the CHMP
issues an opinion that is then considered by the European
Commission, which has the authority to approve medicines for the
European Union. If approved, Vertex would then begin the
country-by-country reimbursement approval process.
In Australia, KALYDECO was listed on the Pharmaceutical Benefits
Scheme (PBS) as of December 1, 2014, which is intended to provide
reimbursement for all eligible patients ages 6 and older with the
G551D or other gating mutations. Patients in Australia have now
begun to receive treatment with KALYDECO.
Lumacaftor in Combination with
Ivacaftor in People with Two Copies of the F508del
Mutation
Vertex today announced that the FDA has accepted its NDA for the
combination of lumacaftor and ivacaftor in people with CF ages 12
and older who have two copies of the F508del mutation. The FDA
granted Vertex’s request for Priority Review, with a target review
(PDUFA) date of July 5, 2015 for the FDA's approval decision. In
Europe, the MAA for this combination regimen has been validated by
the EMA and is under review by the CHMP. The CHMP granted Vertex's
request for Accelerated Assessment of the MAA, which is given to
new medicines of major public health interest and shortens the
review time from approximately 210 to 150 days for the CHMP to give
an opinion following the start of the review. The CHMP opinion is
then reviewed by the European Commission, which generally issues a
final decision within three months. If approved, Vertex would then
begin the country-by-country reimbursement approval process. In
2015, Vertex also plans to submit applications for regulatory
approval of the combination of lumacaftor and ivacaftor in Canada
and Australia. There are approximately 22,000 people with CF ages
12 and older who have two copies of the F508del mutation
in North America, Europe and Australia, including
approximately 8,500 in the United States and 12,000
in Europe.
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 October 2014. The study enrolled approximately 20 patients who
received placebo and approximately 20 patients who received one of
two doses of VX-661 (50 mg q12h or 100 mg QD) in combination with
ivacaftor (150 mg q12h). The primary endpoint of the study is
safety, and additional secondary endpoints will evaluate the
pharmacokinetics and efficacy of VX-661. 12-week data from this
study are expected in the first quarter of 2015.
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 America and 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, Switzerland and 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 America and 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 America and 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.
Next-generation
Correctors
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.
Financial
“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 Ian Smith, Executive Vice President and Chief Financial
Officer for Vertex. “As our revenues increase, we are committed to
managing our operating expense to support high future operating
margins and earnings growth.”
Vertex today provided the following financial outlook and will
provide complete financial guidance on its year-end conference call
on January 28, 2015:
- Vertex entered 2015 with approximately
$1.4 billion in 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 million of which
Vertex received in July 2014 and is currently outstanding.
- Vertex expects total 2015 KALYDECO net
revenues of $560 to $580 million. Vertex expects to report total
2014 KALYDECO net revenues of approximately $460 million and fourth
quarter KALYDECO net revenues of approximately $120 million.
Anticipated 2015 KALYDECO net revenues reflect:
- Use of KALYDECO by eligible patients in
Australia following the completion of reimbursement discussions in
late 2014
- Use of KALYDECO in people in the United
States with the R117H mutation following FDA approval 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
- Vertex expects that its combined
non-GAAP R&D and SG&A expenses in 2015 will be in the range
of $1.05 to $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.
In the United States (U.S.) and Europe, ivacaftor is also
indicated for the treatment of CF in patients age 6 and older who
have one of the following mutations in the
CFTR gene: G1244E, G1349D, G178R, G551S, S1251N, S1255P,
S549N, or S549R. In Canada, ivacaftor is indicated for these
same mutations and additionally for G970R. Additionally, in
the U.S. ivacaftor is indicated for the treatment of CF in patients
age 6 and older who have an R117H 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.
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, Dr. Leiden's statements in the
second paragraph of the press release, the information provided in
the section captioned "Financial" and statements regarding (i) 2015
KALYDECO net revenue guidance; (ii) the expected timing and
clinical trial designs for the pivotal Phase 3 program of VX-661 in
combination with ivacaftor and the other planned studies described
in this press release; (iii) the company’s expectations regarding
the number of people who may be eligible for KALYDECO by the end of
2015; (iv) the company’s expectations regarding potential approvals
for lumacaftor in combination with ivacaftor and additional
approvals for ivacaftor; (v) the target date for the FDA to review
the NDA for lumacaftor in combination with ivacaftor under PDUFA;
(vi) the company’s expectations regarding reimbursement
discussions; (vii) Vertex’s plans to initiate new studies,
including the pivotal Phase 3 program of VX-661 in combination with
ivacaftor and the study to evaluate lumacaftor in combination with
ivacaftor in children 6 to 11; (viii) the company’s expectations
regarding next-generation correctors; and (ix) the company’s
research and early-stage development programs. 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 company's
expectations regarding its 2015 revenues and expenses may be
incorrect (including because one or more of the company's
assumptions underlying its expectations may not be realized), that
regulatory authorities may not approve, or approve on a timely
basis, lumacaftor in combination with ivacaftor, 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.
Webcast Information
The company will webcast its corporate presentation at the 33rd
Annual J.P. Morgan Healthcare Conference on Monday, January 12 at
9:30 a.m. PT (12:30 p.m. ET). The audio portion of management’s
remarks can be accessed live through Vertex’s website at
www.vrtx.com in the “Investors” section under the “Events and
Presentations” page.
(VRTX-GEN)
Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge,
617-341-6108orKelly Lewis, 617-961-7530orEric Rojas,
617-961-7205orMedia:Zach Barber, 617-767-9533mediainfo@vrtx.com
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