-KALYDECO: 2014 revenue growth anticipated from geographic
expansion and approval for use in patients with additional CFTR
-Lumacaftor in combination with ivacaftor: results from two
Phase 3 studies, TRAFFIC and TRANSPORT, expected mid-year in people with
two copies of the F508del mutation-
-Company ends 2013 with approximately $1.47 billion in cash, cash
equivalents and marketable securities; expects 2014 KALYDECO net
revenues of $470 to $500 million-
SAN FRANCISCO--(BUSINESS WIRE)--
Pharmaceuticals Incorporated (Nasdaq:VRTX) today outlined key 2014
business priorities aimed at supporting investment in future
opportunities in cystic fibrosis (CF) and other high-potential research
and development programs. Jeffrey Leiden, M.D., Ph.D., Chair, President
and Chief Executive Officer of Vertex, will discuss the company's
corporate strategy and 2014 business priorities as part of a webcast
presentation at the 32nd Annual J.P. Morgan Healthcare
Conference in San Francisco on Tuesday, January 14 at 8:30 a.m. PT
(11:30 a.m. ET). The presentation will be available on Vertex's website, www.vrtx.com.
In conjunction with the conference, Vertex today provided updates to its
development programs in CF and other early research and development
programs and provided a financial outlook for 2014.
"Throughout 2013, we made significant progress across all parts of our
company, and I am pleased that our strategy to focus investment in key
research and development programs and to maintain financial strength has
positioned us well to further advance our pipeline and our business in
2014," said Dr. Leiden. "Over the coming year, we expect important data
from multiple studies across our cystic fibrosis pipeline that may allow
us to help many people with this disease."
Cystic Fibrosis (CF)
"Today, nearly all eligible patients with the G551D mutation have
started treatment with KALYDECO in the United States and Europe. As we
advance through 2014, our goal is to increase the number of people
eligible for KALYDECO by achieving public reimbursement for this
important medicine in additional countries, including Australia and
Canada, and through regulatory approvals for additional mutations.
"Also this year, we will obtain results from our two Phase 3 studies of
lumacaftor and ivacaftor in people with two copies of the F508del
mutation, which will help define the role that these potential medicines
may play for the more than 28,000 people ages 6 and older with the most
common form of the disease," continued Dr. Leiden.
Global Availability of KALYDECO (ivacaftor)
KALYDECO is currently available to eligible patients in the United
States, England, Scotland, Northern Ireland, Wales, the Republic of
Ireland, France, Germany, the Netherlands, Austria, Denmark, Sweden,
Norway, Greece, and Italy. KALYDECO is also approved in Australia and
Canada, and Vertex is in active discussions with relevant agencies in
these countries to expand access to KALYDECO for eligible patients
through public reimbursement. There are approximately 300 people age six
years and older who have the G551D mutation in Australia and Canada.
Additional Studies Aimed at Increasing the Number of People
Eligible for Ivacaftor
Multiple additional studies of ivacaftor are designed to evaluate
whether additional people with CF may benefit from treatment with
ivacaftor alone, including:
Gating Mutations Study: In December 2013, the U.S. Food and
Drug Administration (FDA) accepted Vertex's supplemental New Drug
Application (sNDA) for ivacaftor in people with CF ages 6 and older
who have non-G551D gating mutations and granted the company's request
for Priority Review. A target review date of March 27, 2014 was set
under the Prescription Drug User Fee Act (PDUFA) for the FDA's
approval decision. Vertex has also submitted a Marketing Authorization
Application (MAA) variation in Europe for ivacaftor in people with CF
ages 6 years and older who have non-G551D gating mutations.
Approximately 400 people ages 6 years and older have non-G551D gating
mutations in North America, Australia and Europe.
R117H Study: Vertex recently announced data from a Phase 3
study of people ages 6 years and older with one copy of the R117H
mutation. The company plans to meet with the FDA in early 2014 to
discuss these data and the potential submission of an sNDA for people
with the R117H mutation. R117H is the most common residual function
mutation. In North America, Europe and Australia, approximately 1,100
people with CF ages 6 and older have at least one copy of an R117H
mutation. Detailed information regarding this study was provided in a
separate press release issued on December 19, 2013.
Study in Children Ages 2 to 5 with Gating Mutations: A Phase 3
study of ivacaftor in children with CF ages 2 to 5 with a gating
mutation is ongoing and fully enrolled. Data from this study are
expected in the second quarter of 2014 to support a potential NDA
submission in the second half of 2014. In North America, Europe and
Australia, approximately 300 children ages 2 to 5 have a gating
Residual Function Study: Enrollment is complete in a Phase 2
proof-of-concept study evaluating ivacaftor in people with CF who have
clinical evidence of residual CFTR function. Data from this study are
expected in the second quarter of 2014. In North America, Europe and
Australia, more than 3,000 people ages 6 and older have non-R117H
mutations that result in residual function.
Lumacaftor in Combination with Ivacaftor
Phase 3 Program in People with Two Copies (homozygous) of the
Phase 3 TRAFFIC and TRANSPORT Studies: Vertex completed
enrollment in October 2013 for the global Phase 3 TRAFFIC and
TRANSPORT studies evaluating lumacaftor (VX-809) in combination with
ivacaftor in people with CF ages 12 and older who have two copies
(homozygous) of the F508del mutation. Vertex began dosing in TRAFFIC
and TRANSPORT in June and May 2013, respectively, and expects data
from these studies to be available in mid-2014 to support the
potential submission of an NDA and MAA for the combination therapy in
people homozygous for the F508del mutation in the second half of 2014.
Two additional studies of lumacaftor in combination with ivacaftor are
being conducted as part of the Phase 3 program, including a study in
children with CF ages 6 to 11 who have two copies of the F508del
mutation and a study in people 18 and older with one copy
(heterozygous) of the F508del mutation on one allele and a
second mutation on the other allele that is not expected to respond to
either ivacaftor or lumacaftor alone. The second part of the study in
children is expected to begin in the second half of 2014 and will be
used for potential subsequent registration of the combination in
children ages 6 to 11. The study in people with one copy of the
F508del mutation is ongoing and intended to provide additional safety
and lung function data on the combination in heterozygous patients.
More than 28,000 people ages 6 and older have two copies of the
F508del mutation in North America, Europe and Australia, including
approximately 22,000 ages 12 and older.
VX-661 in Combination with Ivacaftor
12-Week Phase 2 Study of VX-661 and Ivacaftor in People with Two
Copies of the F508del Mutation: Vertex is preparing to conduct a
12-week study of VX-661 in combination with ivacaftor in people with
CF who have two copies of the F508del mutation. The study is designed
to evaluate safety, efficacy and pharmacokinetics to characterize
VX-661 for further clinical development. Vertex has submitted a
protocol to the FDA for this study and expects enrollment to begin in
the first quarter of 2014.
4-Week Phase 2 Study of VX-661 in Combination with KALYDECO in
People with One Copy of the G551D and F508del mutation: Enrollment
is complete in a Phase 2 study evaluating a 4-week regimen of VX-661
in combination with KALYDECO in people with one copy of the G551D
mutation and one copy of the F508del mutation. This study is intended
to explore whether the addition of a corrector to treatment with
KALYDECO can provide greater clinical benefit than treatment with
KALYDECO alone in people with the G551D and F508del mutations. Data
from this study are expected in the first half of 2014.
Early Research and Development Programs
"In addition to our late-stage efforts in cystic fibrosis, we continue
to advance multiple early-stage research and development programs,
including VX-135 as part of potential all-oral regimens for hepatitis C.
Vertex was built upon innovative science, and our commitment to research
will continue to be our growth engine for the future as we seek to
create new transformative medicines for cystic fibrosis, cancer,
multiple sclerosis and other serious and rare diseases," concluded Dr.
Next-Generation Correctors for Cystic Fibrosis:
Vertex's goal is to advance a next-generation corrector into clinical
development by the end of 2014. Next-generation correctors could be
evaluated as part of potential dual-corrector regimens. The proposed
use of a dual-corrector combination regimen is supported by in
vitro data that showed a combination of two correctors with
ivacaftor 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.
Study of VX-135 in Combination with Daclatasvir: Vertex
and Bristol Myers Squibb Company (BMS) recently announced sustained
viral response (SVR4) rate and safety data from the initial cohorts of
a Phase 2a study of VX-135 in combination with daclatasvir, an NS5A
replication complex inhibitor being developed by BMS, in New Zealand.
Vertex is currently evaluating these data with BMS to determine the
potential next steps for this combination in people with hepatitis C.
VX-135 in Combination with Simeprevir: A drug-drug
interaction study of VX-135 in combination with simeprevir in healthy
volunteers is complete. Simeprevir (TMC435) is a once-daily
investigational hepatitis C protease inhibitor being jointly developed
by Janssen R&D Ireland and Medivir AB. Vertex and Janssen are
currently discussing the potential next steps for further evaluation
of VX-135 in combination with simeprevir.
Early-stage research and development programs are ongoing in cystic
fibrosis, cancer, multiple sclerosis and other serious and rare
diseases. Vertex expects to advance one or more compounds for the
treatment of these diseases into clinical development in 2014.
Financial Guidance and Outlook
"Entering 2014, we have aligned our business and investment with the
future potential we see within our cystic fibrosis program and other
high-potential research and development programs," said Ian Smith,
Executive Vice President and Chief Financial Officer for Vertex. "We
believe there is the potential for significant future growth in revenues
from KALYDECO and that we may achieve further revenue growth from the
potential combination regimen of lumacaftor and ivacaftor in people with
two copies of the F508del mutation. As we progress over the coming
months, we remain committed to maintaining balance sheet strength to
support continued investment in our business as we receive data from
multiple clinical studies, including our Phase 3 studies in CF."
Vertex today provided the following financial outlook and will provide
complete financial guidance on its year-end conference call on January
Vertex ended 2013 with approximately $1.47 billion in cash, cash
equivalents and marketable securities.
Vertex expects total 2014 net revenues of $570 to $600 million,
including KALYDECO net revenues of $470 to $500 million for 2014.
Total revenues include net product revenues for KALYDECO and INCIVEK,
as well as royalty and collaborative revenues.
Vertex expects that its 2014 non-GAAP operating expenses will be in
the range of $900 to $950 million. The company's planned 2014
investment includes approximately $40 to $50 million of expense
related to development of an all-oral treatment regimen for hepatitis
C. Additionally, the company expects to record approximately $60
million in effective interest expense in 2014 related to the
accounting treatment for the leases of the company's corporate
headquarters. Vertex's expected non-GAAP operating expense excludes
cost of revenues, stock-based compensation expense, restructuring
charges, transition costs related to the relocation of our corporate
headquarters, Alios expenses related to the accounting for the
collaboration with Vertex and any similar expenses incurred in 2014.
Non-GAAP Financial Measures
In financial press releases, Vertex's financial results and financial
guidance are provided in accordance with accounting principles generally
accepted in the United States (GAAP) and using certain non-GAAP
financial measures. In this press release, Vertex provides its 2014
non-GAAP guidance excluding costs of revenues, stock-based compensation
expense, restructuring charges, transition costs related to the
relocation of our corporate headquarters, Alios expenses related to the
accounting for the collaboration with Vertex and any similar expenses
incurred in 2014. Vertex provides these financial measures as a
complement to results provided in accordance with GAAP because
management believes these non-GAAP financial measures help indicate
underlying trends in the company's business, are important in comparing
current results with prior period results and provide additional
information regarding its financial position. Management also uses these
non-GAAP financial measures to establish budgets and operational goals
that are communicated internally and externally, and to manage the
company's business and to evaluate its performance.
Vertex is a global biotechnology company that aims to discover, develop
and commercialize innovative medicines 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 cystic fibrosis, hepatitis C, rheumatoid arthritis and other
life-threatening diseases. In addition to our clinical development
programs, Vertex has more than a dozen ongoing preclinical 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 four 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
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO™ (ivacaftor)
Ivacaftor (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.
Ivacaftor is not for use in people with CF due to other mutations in
the CFTR gene. It is not effective in patients with CF with 2 copies of
the F508del mutation (F508del/F508del) in the CFTR gene.
The efficacy and safety of ivacaftor in children younger than 6 years of
age have not been evaluated.
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 which may
diminish effectiveness. Therefore, co-administration is not recommended.
The dose of ivacaftor must be adjusted when used concomitantly with
potent and moderate CYP3A inhibitors. The dose of ivacaftor must be
adjusted when used in patients with moderate or severe hepatic disease.
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
Please see full U.S. Prescribing Information for KALYDECO at www.KALYDECO.com,
the EU Summary of Product Characteristics for KALYDECO at http://goo.gl/N3Tz4,
the Canadian Product Monograph for KALYDECO at www.vrtx.ca
and the Australian Consumer Medical Information and Product Information
for KALYDECO (ivacaftor) at http://bit.ly/18wlMld.
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, third and fourth
paragraphs of the press release and the paragraph following the caption
"Early Research and Development Programs," the information provided in
the section captioned "Financial Guidance and Outlook" and statements
regarding (i) expected revenues and expenses for 2014, including growth
in KALYDECO net revenues anticipated from geographic expansion and
approval for use in patients with additional CFTR mutations; (ii) the
timing of initiation and receipt of data from ongoing and planned Phase
2 and Phase 3 studies; (iii) potential regulatory submissions to the FDA
and in Europe and the expected timing of those submissions; (iv) the
company's plans regarding meetings with the FDA regarding the data from
the R117H study; (v) the company's research programs, including its goal
of advancing a next-generation corrector into clinical development by
the end of 2014; (vi) the company's discussions with BMS and Janssen
regarding VX-135; and (vii) expectations regarding the advancement of
one or more research compounds into clinical development in 2014. 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 2014
revenues and expenses may be incorrect (including because one or more of
the company's assumptions underlying its expectations may not be
realized), 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.
Vertex Pharmaceuticals Incorporated
Kelly Lewis, 617-961-7530
Source: Vertex Pharmaceuticals Incorporated
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